GRN FDA Survey Please enable JavaScript in your browser to complete this form.Please enable JavaScript in your browser to complete this form.Research Title: GRN-FTD Community Survey Protocol #: NB400226 Researcher: Carrie Milliard, MS, CGC, CCRC Contact Information: 415-646-4456 x105, cmilliard@ftdregistry.org Research Sponsor: FTD Disorders Registry, LLC Purpose, Procedure, and Duration: In collaboration with the FTD Disorders Registry, CureGRN, the Association for Frontotemporal Degeneration (AFTD), and the Bluefield Project to Cure FTD are conducting a critical survey to better understand the perspectives of individuals affected by FTD caused by variants in the GRN (progranulin) gene. Your anonymous responses will be used in upcoming meetings to inform the Food and Drug Administration (FDA) and other key stakeholders about community priorities and experiences. This is a vital opportunity to make your voice heard! Benefits: You may not benefit personally from being in this study, but your answers could help us understand more about the experiences of individuals impacted by FTD-GRN. Risks: Some of our questions may make you feel uncomfortable or upset, but you can skip any question you don’t want to answer. You can also stop the survey at any time. The survey is anonymous, and we will make an effort to safeguard the data you provide. Alternative Opportunities: We know of no alternative except not to participate in our study. If you would like to learn about more supports, information, and research opportunities related to FTD-GRN, please visit: https://www.curegrn.org/ https://www.theaftd.org/ https://www.bluefieldproject.org/ https://ftdregistry.org/ Privacy and Future Use: Your responses to the research survey are anonymous. That means we won’t know which responses are yours. We won’t collect names, internet addresses, email addresses, or any other identifiable information. Complaints or Concerns: If you have questions about the study, please contact Carrie Milliard, MS, CGC, CCRC, at 415-646-4456 x105, cmilliard@ftdregistry.org. This research is being overseen by North Star Review Board. An IRB is a group of people who perform independent reviews of research studies. You may contact North Star Review Board at 877-673-8439 (toll free) or info@northstarreviewboard.org if: You have questions, concerns, or complaints that are not being answered by the research team. You are not getting answers from the research team. You cannot reach the research team. You want to talk to someone else about the research study. You have questions about your rights as a research subject. Thank you for taking the time to consider our study. You do not have to participate in our study, but we hope you will. You may have received this link from several people or organizations. PLEASE ONLY TAKE THIS SURVEY ONE TIME. By proceeding to complete this survey, you are indicating your consent to participate. NextThank you for taking the time to complete this survey. We are seeking input from individuals affected by GRN-related frontotemporal dementia (FTD) to better understand their experiences, needs, and priorities. Your participation is entirely optional and anonymous. Any information you provide will be reported in aggregate, meaning individual responses will not be identified. The information collected through this survey will be used for research purposes and may be shared with the US Food and Drug Administration (FDA) and other stakeholders to help inform understanding of the GRN family experience. We are grateful for your time, perspective, and willingness to contribute to this important effort. Have you, or has a loved one, been diagnosed with FTD, and do you know or suspect that the diagnosis is or was associated with a variant in the GRN (progranulin) gene?YesNoVariants in the GRN gene reduce progranulin protein levels, causing brain cell damage that leads to frontotemporal degeneration (FTD) – a progressive condition affecting personality, language, and behavior. (Learn more about here: https://www.curegrn.org/about-grn-ftd)Thank you for your interest, but you are not eligible for this survey. Please visit any of these non-profit organizations for more information and resources on FTD: CureGRN - https://www.curegrn.org/ The Association for Frontotemporal Degeneration (AFTD) - https://www.theaftd.org/ The Bluefield Project to Cure FTD - https://www.bluefieldproject.org/ The FTD Disorders Registry - https://ftdregistry.org/I am (select all that apply)Diagnosed with FTD-GRNAt risk for developing FTD-GRNA current or former care partner of someone with FTD-GRNWhat is your age? Selected Value: 0 Where did you hear of this survey? (select all that apply)CureGRNThe Association for Frontotemporal Degeneration (AFTD)The Bluefield Project to Cure FTDThe FTD Disorders RegistryFamily/friendOtherHeard of survey from - OtherWhat country do you currently reside in?Choose your country from the dropdown listUnited StatesAfghanistanAlbaniaAlgeriaAndorraAngolaAntigua & DepsArgentinaArmeniaAustraliaAustriaAzerbaijanBahamasBahrainBangladeshBarbadosBelarusBelgiumBelizeBeninBhutanBoliviaBosnia HerzegovinaBotswanaBrazilBruneiBulgariaBurkinaBurundiCambodiaCameroonCanadaCape VerdeCentral African RepChadChileChinaColombiaComorosCongoCongo {Democratic Rep}Costa RicaCroatiaCubaCyprusCzech RepublicDenmarkDjiboutiDominicaDominican RepublicEast TimorEcuadorEgyptEl SalvadorEquatorial GuineaEritreaEstoniaEthiopiaFijiFinlandFranceGabonGambiaGeorgiaGermanyGhanaGreeceGrenadaGuatemalaGuineaGuinea-BissauGuyanaHaitiHondurasHungaryIcelandIndiaIndonesiaIranIraqIreland {Republic}IsraelItalyIvory CoastJamaicaJapanJordanKazakhstanKenyaKiribatiKorea NorthKorea SouthKosovoKuwaitKyrgyzstanLaosLatviaLebanonLesothoLiberiaLibyaLiechtensteinLithuaniaLuxembourgMacedoniaMadagascarMalawiMalaysiaMaldivesMaliMaltaMarshall IslandsMauritaniaMauritiusMexicoMicronesiaMoldovaMonacoMongoliaMontenegroMoroccoMozambiqueMyanmar, {Burma}NamibiaNauruNepalNetherlandsNew ZealandNicaraguaNigerNigeriaNorwayOmanPakistanPalauPanamaPapua New GuineaParaguayPeruPhilippinesPolandPortugalQatarRomaniaRussian FederationRwandaSt Kitts & NevisSt LuciaSaint Vincent & the GrenadinesSamoaSan MarinoSao Tome & PrincipeSaudi ArabiaSenegalSerbiaSeychellesSierra LeoneSingaporeSlovakiaSloveniaSolomon IslandsSomaliaSouth AfricaSouth SudanSpainSri LankaSudanSurinameSwazilandSwedenSwitzerlandSyriaTaiwanTajikistanTanzaniaThailandTogoTongaTrinidad & TobagoTunisiaTurkeyTurkmenistanTuvaluUgandaUkraineUnited Arab EmiratesUnited KingdomUruguayUzbekistanVanuatuVatican CityVenezuelaVietnamYemenZambiaZimbabweOtherCountry - OtherDo you live in a rural, suburban, or urban area?RuralSuburbanUrbanPrefer not to answerWants and Risk Tolerance of the FTD-GRN CommunityHow concerned are you about developing FTD symptoms in the future related to a known or possible GRN variant?Rate 1 out of 5Rate 2 out of 5Rate 3 out of 5Rate 4 out of 5Rate 5 out of 51 star = Not concerned | 5 stars = Greatly concernedBefore symptoms appear, would you be willing to receive experimental treatments to potentially prevent or delay symptoms?YesNoWould you be comfortable with accepting medical risks if an approved or experimental therapy was believed to be able to delay or prevent disease onset?Rate 1 out of 5Rate 2 out of 5Rate 3 out of 5Rate 4 out of 5Rate 5 out of 51 star = Not comfortable | 5 stars = Very comfortableBefore symptoms appear, would you be comfortable participating in a trial that required daily consumption by mouth of a pill or capsule?Rate 1 out of 5Rate 2 out of 5Rate 3 out of 5Rate 4 out of 5Rate 5 out of 51 star = Not comfortable | 5 stars = Very comfortableBefore symptoms appear, would you be comfortable participating in a trial that infused lab-produced progranulin via IV?Rate 1 out of 5Rate 2 out of 5Rate 3 out of 5Rate 4 out of 5Rate 5 out of 51 star = Not comfortable | 5 stars = Very comfortableBefore symptoms appear, would you be comfortable participating in a trial that Replaced unhealthy cells in the brain with healthy ones?Rate 1 out of 5Rate 2 out of 5Rate 3 out of 5Rate 4 out of 5Rate 5 out of 51 star = Not comfortable | 5 stars = Very comfortableBefore symptoms appear, would you be comfortable participating in a trial that used a neurosurgical procedure to deliver a working copy of the GRN gene to the brain?Rate 1 out of 5Rate 2 out of 5Rate 3 out of 5Rate 4 out of 5Rate 5 out of 51 star = Not comfortable | 5 stars = Very comfortableBefore symptoms appear, would you be comfortable participating in a trial that made alterations to your DNA to increase the production of progranulin? Rate 1 out of 5Rate 2 out of 5Rate 3 out of 5Rate 4 out of 5Rate 5 out of 51 star = Not comfortable | 5 stars = Very comfortableWould you be comfortable enrolling in a placebo-controlled trial in which you might receive the placebo instead of the active therapy?YesNoWhy not comfortable in placebo trialBefore symptoms appear, would you be comfortable undergoing monitoring procedures during the course of a clinical trial, such as ... 12345 Brain imaging1Brain imaging 12Brain imaging 23Brain imaging 34Brain imaging 45Brain imaging 5Lumbar punctures1Lumbar punctures 12Lumbar punctures 23Lumbar punctures 34Lumbar punctures 45Lumbar punctures 5Blood draws1Blood draws 12Blood draws 23Blood draws 34Blood draws 45Blood draws 5Neurological testing*1Neurological testing* 12Neurological testing* 23Neurological testing* 34Neurological testing* 45Neurological testing* 5Skin biopsy1Skin biopsy 12Skin biopsy 23Skin biopsy 34Skin biopsy 45Skin biopsy 5 On a scale of 1 to 5 1 being Not comfortable and 5 being Very comfortable *Neurological testing: Such as memory and thinking skills questionnairesWould contributing to research that may help future generations motivate you to consider participation?Rate 1 out of 5Rate 2 out of 5Rate 3 out of 5Rate 4 out of 5Rate 5 out of 51 star = Would not motivate me | 5 stars = Would greatly motivate meWould any of the potential side effects described below, particularly those that could affect your work or family responsibilities, strongly influence your decision to participate in a clinical trial before symptoms appear?Mild side effects such as headache, nausea or GI disturbance.Moderate side effects such as vomiting or pain that make me unable to go to work or fulfill family responsibilities.Severe side effects that may require a hospital stay.I'm willing to take part regardless of potential side effects.Which of the following factors would make you more likely to consider participating in a clinical trial?I knew I would not receive a placebo.I was confident that the clinical team would treat me with respect and compassion.I was confident that privacy would be guaranteed.I would get my individual results for various tests completed during the study.The outcome of the study would be shared with trial participants and the scientific community at large.I would be reimbursed for all expenses.I could participate in some aspects of the study at my local medical center.I could participate in some aspects of the study from home.OtherMore likely to participate - OtherDo you find the inclusion criteria for entering an FTD-GRN clinical trial of symptomatic patients to be too stringent?YesNoWhy you feel the inclusion criteria are too stringent?Are you concerned that you may not be able to afford participating in a clinical trial?YesNoPlease outline your affordability concern(s) and what could lessen these concerns?Are you concerned about the amount of time required to participate in a clinical trial?YesNoPlease outline your time concern(s) and what could lessen these concerns?What would you like the FDA and/or other health authorities to know when making decisions about new potential treatments for FTD-GRN?Would you be comfortable participating in a clinical trial that may involve receiving a placebo surgery, meaning some participants would undergo a surgical procedure but would not receive the active investigational therapy?Rate 1 out of 5Rate 2 out of 5Rate 3 out of 5Rate 4 out of 5Rate 5 out of 51 star = Not comfortable | 5 stars = Very comfortableA placebo surgery is a procedure in which a participant undergoes surgery but does not receive the active therapy. In placebo-controlled studies, participants are not told whether they received the active study treatment or the placebo. Participants who receive placebo surgery may still face risks associated with the procedure, such as anesthesia, surgical drilling, infection, and other surgical complications.What are your views on the use of placebo surgeries as controls in clinical trials studying therapies administered directly to the brain or cerebrospinal fluid?If you were eligible to enroll in an experimental clinical trial, would you be more likely to participate if the trial sponsor agreed to share your de-identified data with other scientists to help advance the search for FTD-GRN treatments?YesNoUnsureWhy are you against or unsure about sharing your data? What would you need to know to feel more comfortable sharing data?What would the opportunity to participate in a clinical trial before developing symptoms mean to you? How might it affect your outlook on the future, your decisions about genetic testing, or your sense of peace?If a clinical trial were not available to you in your home country, would you be willing to travel to participate in a trial in another country?YesNoUnsureFTD-GRN Survey Participant/Treatment DemographicsIf loved one(s) were diagnosed with FTD-GRN, what form of the disease were they diagnosed with? (select all that apply)bvFTD - behavioral variant FTD, frontotemporal dementia, FTD dementia, or Pick's diseaseCBD or CBS - corticobasal degeneration, or corticobasal syndromePPA - primary progressive aphasia (including nfvPPA, svPPA, lvPPA)Primary Progressive Apraxia of SpeechPSP - progressive supranuclear palsy, or Richardson's syndromeI'm not sureIf loved one(s) were diagnosed with FTD-GRN, did they experience changes in these areas? (select all that apply)Language (speaking, finding words, understanding, knowing the meaning of objects)Memory (remembering recent events, learning new information)Thinking (solving problems, making judgments, organizing)Spatial (judging distances, perceiving objects)Personality (acting differently or inappropriately in a social situation)Relationships (getting along with others)Mood (anxious, not interested, depressed, irritable, emotional outbursts)Motor (tremor, balance, performing movements, walking)Eating or drinking (cravings, alcohol intake, weight gain)Behavior (repetitive or compulsive behavior, rigid routines)Sleep (not sleeping through the night, bad dreams, sleeping too much)Delusions or hallucinationsIncontinenceOtherSingle Line TextIf you were treated before symptom onset, which potential clinical trial outcomes would be most meaningful to you? (select all that apply)Delaying the onset of symptoms for years or decades.Preventing symptoms from ever developing.Slowing the progression of disease after symptoms have begun, so that daily functions and independence can be maintained longer even if they eventually decline.Reducing the impact of the one or two symptoms that are most distressing, even if the disease continues to progress and lifespan is not extended.Improving or stabilizing biomarkers, such as progranulin or neurofilament levels, even without noticeable changes in symptoms.If a loved one was diagnosed with FTD-GRN, were they prescribed any medications to address their symptoms?YesNoUnsureWhat medications helped alleviate symptoms?Have you done any of the following to attempt to prevent symptoms? (select all that apply)Diet modificationExerciseTaken supplementsMeditationSupport GroupSpeech-language therapyOccupational therapyPhysical therapyResearch involving an experimental medicationResearch or medical care involving brain stimulation (electrodes or magnets placed on head)In your opinion, have any of the above interventions delayed the onset of symptoms? If so, where did you hear about these interventions?If a loved one was diagnosed with FTD-GRN, how long was the period of time between the development of symptoms and their diagnosis?If a loved one was diagnosed and passed away from FTD-GRN, how long was the period of time between the development of symptoms and their passing?Wants and Risk Tolerance of the FTD-GRN CommunityWould you be comfortable participating in a trial that required daily consumption by mouth of a pill or capsule?Rate 1 out of 5Rate 2 out of 5Rate 3 out of 5Rate 4 out of 5Rate 5 out of 51 star = Not comfortable | 5 stars = Very comfortableWould you be comfortable participating in a trial that infused lab-produced progranulin via IV?Rate 1 out of 5Rate 2 out of 5Rate 3 out of 5Rate 4 out of 5Rate 5 out of 51 star = Not comfortable | 5 stars = Very comfortableWould you be comfortable participating in a trial that Replaced unhealthy cells in the brain with healthy ones?Rate 1 out of 5Rate 2 out of 5Rate 3 out of 5Rate 4 out of 5Rate 5 out of 51 star = Not comfortable | 5 stars = Very comfortableWould you be comfortable participating in a trial that used a neurosurgical procedure to deliver a working copy of the GRN gene to the brain?Rate 1 out of 5Rate 2 out of 5Rate 3 out of 5Rate 4 out of 5Rate 5 out of 51 star = Not comfortable | 5 stars = Very comfortableWould you be comfortable participating in a trial that made alterations to your DNA to increase the production of progranulin?Rate 1 out of 5Rate 2 out of 5Rate 3 out of 5Rate 4 out of 5Rate 5 out of 51 star = Not comfortable | 5 stars = Very comfortableWould you be comfortable enrolling in a placebo-controlled trial in which you might receive the placebo instead of the active therapy?YesNoWhy not comfortable in placebo trialWould you be comfortable undergoing monitoring procedures during the course of a clinical trial, such as ... 12345 Brain imaging1Brain imaging 12Brain imaging 23Brain imaging 34Brain imaging 45Brain imaging 5Lumbar punctures1Lumbar punctures 12Lumbar punctures 23Lumbar punctures 34Lumbar punctures 45Lumbar punctures 5Blood draws1Blood draws 12Blood draws 23Blood draws 34Blood draws 45Blood draws 5Neurological testing*1Neurological testing* 12Neurological testing* 23Neurological testing* 34Neurological testing* 45Neurological testing* 5Skin biopsy1Skin biopsy 12Skin biopsy 23Skin biopsy 34Skin biopsy 45Skin biopsy 5 On a scale of 1 to 5 1 being Not comfortable and 5 being Very comfortable *Neurological testing: Such as memory and thinking skills questionnairesWould contributing to research that may help future generations motivate you to consider participation?Rate 1 out of 5Rate 2 out of 5Rate 3 out of 5Rate 4 out of 5Rate 5 out of 51 star = Would not motivate me | 5 stars = Would greatly motivate meWould any of the potential side effects described below, particularly those that could affect your work or family responsibilities, strongly influence your decision to participate in a clinical trial?Mild side effects such as headache, nausea or GI disturbance.Moderate side effects such as vomiting or pain that make me unable to go to work or fulfill family responsibilities.Severe side effects that may require a hospital stay.I'm willing to take part regardless of potential side effects. If you are eligible to participate in a FTD-GRN clinical trial and haven't, what are your reasons for abstaining? (select all that apply)I am not eligible for a trial.There were no trials available near where I live.Travel would be too difficult.It would be too difficult emotionally to participate.The procedures involved in clinical trials would be too difficult.I'm concerned about potential side effects.I am waiting for a more promising clinical trial option.I am skeptical of clinical researchers.I am concerned about potential expenses related to participation.I would miss too much work.I don't know where to find a clinical trial.Not applicable; I participate(d) in a clinical trial.OtherReason for abstaining from trial - OtherIf you are unlikely or unwilling to participate in a clinical trial, would you be more likely to participate if any of the following were guaranteed? (select all that apply)I knew I would not receive a placebo.I was confident that the clinical team would treat me with respect and compassion.I was confident that privacy would be guaranteed.I would get my individual results for various tests completed during the study.The outcome of the study would be shared with trial participants and the scientific community at large.I would be reimbursed for all expenses.I could participate in some aspects of the study at my local medical center.I could participate in some aspects of the study from home.Not applicable; I participate(d) in a clinical trial.OtherMore likely to participate - OtherDo you find the inclusion criteria for entering an FTD-GRN clinical trial to be too stringent? YesNoPlease explain why you feel the inclusion criteria for entering an FTD-GRN clinical trial are too stringent?Are you concerned that you may not be able to afford participating in a clinical trial?YesNoPlease outline your affordability concern(s) and what could lessen these concerns?Even if costs were reimbursed, are you still concerned that participation in a clinical trial may be cost prohibitive?YesNoPlease outline your reimbursement concern(s) and what could lessen these concerns?Are you concerned about the amount of time required to participate in a clinical trial?YesNoPlease outline your time concern(s) and what could lessen these concerns?Have you been a participant in a previous FTD-GRN trial that is over? YesNoWould you like to enter another FTD-GRN trial?Rate 1 out of 5Rate 2 out of 5Rate 3 out of 5Rate 4 out of 5Rate 5 out of 51 star = Not very likely | 5 stars = Very LikelyIf you enrolled in a trial in the past, do you regret that choice?YesNoWhat would you have done differently?What would you like the FDA and/or other health authorities to know when making decisions about new potential treatments for FTD-GRN?Would you be comfortable participating in a clinical trial that may involve receiving a placebo surgery, meaning some participants would undergo a surgical procedure but would not receive the active investigational therapy?Rate 1 out of 5Rate 2 out of 5Rate 3 out of 5Rate 4 out of 5Rate 5 out of 51 star = Not very likely | 5 stars = Very Likely A placebo surgery is a procedure in which a participant undergoes surgery but does not receive the active therapy. In placebo-controlled studies, participants are not told whether they received the active study treatment or the placebo. Participants who receive placebo surgery may still face risks associated with the procedure, such as anesthesia, surgical drilling, infection, and other surgical complications.What are your views on the use of placebo surgeries as controls in clinical trials studying therapies administered directly to the brain or cerebrospinal fluid?If you were eligible to enroll in an experimental clinical trial, would you be more likely to participate if the trial sponsor agreed to share your de-identified data with other scientists to help advance the search for FTD-GRN treatments?YesNoUnsureWhy are you against or unsure about sharing your data? What would you need to know to feel more comfortable sharing data?If a clinical trial were not available to you in your home country, would you be willing to travel to participate in a trial in another country?YesNoUnsureFTD-GRN Survey Participant/Treatment DemographicsWhat form of FTD-GRN were you diagnosed with? (select all that apply)bvFTD - behavioral variant FTD, frontotemporal dementia, FTD dementia, or Pick's diseaseCBD or CBS - corticobasal degeneration, or corticobasal syndromePPA - primary progressive aphasia (including nfvPPA, svPPA, lvPPA)Primary Progressive Apraxia of SpeechPSP - progressive supranuclear palsy, or Richardson's syndromeI'm not sureIf loved one(s) were diagnosed with FTD-GRN, what form of the disease were they diagnosed with? (select all that apply)bvFTD - behavioral variant FTD, frontotemporal dementia, FTD dementia, or Pick's diseaseCBD or CBS - corticobasal degeneration, or corticobasal syndromePPA - primary progressive aphasia (including nfvPPA, svPPA, lvPPA)Primary Progressive Apraxia of SpeechPSP - progressive supranuclear palsy, or Richardson's syndromeI'm not sureWhat symptoms have you experienced with your FTD-GRN diagnosis? (select all that apply)Language (speaking, finding words, understanding, knowing the meaning of objects)Memory (remembering recent events, learning new information)Thinking (solving problems, making judgments, organizing)Spatial (judging distances, perceiving objects)Personality (acting differently or inappropriately in a social situation)Relationships (getting along with others)Mood (anxious, not interested, depressed, irritable, emotional outbursts)Motor (tremor, balance, performing movements, walking)Eating or drinking (cravings, alcohol intake, weight gain)Behavior (repetitive or compulsive behavior, rigid routines)Sleep (not sleeping through the night, bad dreams, sleeping too much)Delusions or hallucinationsIncontinenceOtherAffected - Symptoms in me - OtherIf loved one(s) were diagnosed with FTD-GRN, did they experience changes in these areas? (select all that apply)Language (speaking, finding words, understanding, knowing the meaning of objects)Memory (remembering recent events, learning new information)Thinking (solving problems, making judgments, organizing)Spatial (judging distances, perceiving objects)Personality (acting differently or inappropriately in a social situation)Relationships (getting along with others)Mood (anxious, not interested, depressed, irritable, emotional outbursts)Motor (tremor, balance, performing movements, walking)Eating or drinking (cravings, alcohol intake, weight gain)Behavior (repetitive or compulsive behavior, rigid routines)Sleep (not sleeping through the night, bad dreams, sleeping too much)Delusions or hallucinationsIncontinenceOtherAffected - Symptoms in loved one - OtherWhich potential clinical trial outcomes would be most meaningful to you? (select all that apply)Delaying the onset of symptoms for years or decades in people who are not yet symptomatic.Preventing symptoms from ever developing in people who are not yet symptomatic.Slowing the progression of disease after symptoms have begun, so that daily functions and independence can be maintained longer even if they eventually decline.Reducing the impact of the one or two symptoms that are most distressing, even if the disease continues to progress and lifespan is not extended.Improving or stabilizing biomarkers, such as progranulin or neurofilament levels, even without noticeable changes in symptoms.If loved one(s) were diagnosed with FTD-GRN, which potential clinical trial outcomes would be most meaningful to them? (select all that apply)Delaying the onset of symptoms for years or decades in people who are not yet symptomatic.Preventing symptoms from ever developing in people who are not yet symptomatic.Slowing the progression of disease after symptoms have begun, so that daily functions and independence can be maintained longer even if they eventually decline.Reducing the impact of the one or two symptoms that are most distressing, even if the disease continues to progress and lifespan is not extended.Improving or stabilizing biomarkers, such as progranulin or neurofilament levels, even without noticeable changes in symptoms.Were you prescribed medications to address your symptoms?YesNoUnsureIn your opinion did any of these medications help alleviate symptoms?If a loved one was diagnosed with FTD-GRN, were they prescribed any medications to address their symptoms?YesNoUnsureWhat medications helped alleviate symptoms?Have you done any of the following to help manage symptoms? (select all that apply)Diet modificationExerciseTaken supplementsMeditationSupport GroupSpeech-language therapyOccupational therapyPhysical therapyResearch involving an experimental medicationResearch or medical care involving brain stimulation (electrodes or magnets placed on head)In your opinion, have any of the above interventions alleviated symptoms? If so, where did you hear about these interventions?Have there been financial impacts associated with your diagnosis (ex: cost of care, lost wages, negative impact on personal finances, debt accumulation)?If a loved one was diagnosed with FTD-GRN, how long was the period of time between the development of symptoms and their diagnosis?If a loved one was diagnosed and passed away from FTD-GRN, how long was the period of time between the development of symptoms and their passing?Wants and Risk Tolerance of the FTD-GRN CommunityWould you be comfortable enrolling your loved one in a trial that required daily consumption by mouth of a pill or capsule?Rate 1 out of 5Rate 2 out of 5Rate 3 out of 5Rate 4 out of 5Rate 5 out of 51 star = Not comfortable | 5 stars = Very comfortable Would you be comfortable enrolling your loved one in a trial that infused lab-produced progranulin via IV?Rate 1 out of 5Rate 2 out of 5Rate 3 out of 5Rate 4 out of 5Rate 5 out of 51 star = Not comfortable | 5 stars = Very comfortable Would you be comfortable enrolling your loved one in a trial that Replaced unhealthy cells in the brain with healthy ones?Rate 1 out of 5Rate 2 out of 5Rate 3 out of 5Rate 4 out of 5Rate 5 out of 51 star = Not comfortable | 5 stars = Very comfortable Would you be comfortable enrolling your loved one in a trial that used a neurosurgical procedure to deliver a working copy of the GRN gene to the brain?Rate 1 out of 5Rate 2 out of 5Rate 3 out of 5Rate 4 out of 5Rate 5 out of 51 star = Not comfortable | 5 stars = Very comfortable Would you be comfortable enrolling your loved one in a trial that made alterations to your DNA to increase the production of progranulin?Rate 1 out of 5Rate 2 out of 5Rate 3 out of 5Rate 4 out of 5Rate 5 out of 51 star = Not comfortable | 5 stars = Very comfortableWould you be comfortable enrolling your loved one in a placebo-controlled trial in which they might receive the placebo instead of the active therapy?YesNoWhy not comfortable in placebo trialWould you be comfortable with your loved one undergoing monitoring procedures during the course of a clinical trial, such as ... 12345 Brain imaging1Brain imaging 12Brain imaging 23Brain imaging 34Brain imaging 45Brain imaging 5Lumbar punctures1Lumbar punctures 12Lumbar punctures 23Lumbar punctures 34Lumbar punctures 45Lumbar punctures 5Blood draws1Blood draws 12Blood draws 23Blood draws 34Blood draws 45Blood draws 5Neurological testing*1Neurological testing* 12Neurological testing* 23Neurological testing* 34Neurological testing* 45Neurological testing* 5Skin biopsy1Skin biopsy 12Skin biopsy 23Skin biopsy 34Skin biopsy 45Skin biopsy 5 On a scale of 1 to 5 1 being Not comfortable and 5 being Very comfortable *Neurological testing: Such as memory and thinking skills questionnairesWould contributing to research that may help future generations motivate you to consider your loved one's participation?Rate 1 out of 5Rate 2 out of 5Rate 3 out of 5Rate 4 out of 5Rate 5 out of 51 star = Would not motivate me | 5 stars = Would greatly motivate me Would any of the outlined potential side effects strongly influence your decision to enroll your loved one in a clinical trial? (select all that apply)Mild side effects such as headache, nausea or GI disturbance.Moderate side effects such as vomiting or pain.Severe side effects that may require a hospital stay.I'm willing for my loved one to take part regardless of potential side effects. If your loved one is eligible to participate in a FTD-GRN clinical trial and hasn't, what are the reasons for abstaining? (select all that apply)My loved one is not eligible for a trial.There were no trials available near where my loved one lives.Travel would be too difficult.It would be too difficult emotionally to participate.The procedures involved in clinical trials would be too difficult.I'm concerned about potential side effects.I am waiting for a more promising clinical trial option.I am skeptical of clinical researchers.There is concerned about potential expenses related to participation.I would miss too much work.I don't know where to find a clinical trial.OtherWhat other reasons do you have for abstaining from a clinical trial?If you are unlikely or unwilling to enroll your loved one in a clinical trial, would you be more likely to enroll them if any of the following were guaranteed? (select all that apply) I knew my loved one would not receive a placebo.I was confident that the clinical team would treat me and my loved one with respect and compassion.I was confident that privacy would be guaranteed.My loved one would get their individual results for various tests completed during the study.The outcome of the study would be shared with trial participants and the scientific community at large.I would be reimbursed for all expenses.My loved one could participate in some aspects of the study at a local medical center.My loved one could participate in some aspects of the study from home.OtherWhat else would make you be more likely to enroll them in a clinical trial?Did your loved one attempt to enroll in a clinical trial and were found to be ineligible? If so, why were they found to be ineligible?Do you find the inclusion criteria for entering an FTD-GRN clinical trial to be too stringent? YesNoPlease explain why you feel the inclusion criteria would be too stringent?Are you concerned that you may not be able to afford participating in a clinical trial?YesNoPlease outline your concern(s) and what could lessen these affordability concerns?Even if costs were reimbursed, are you still concerned that participation in a clinical trial may be cost prohibitive?YesNoPlease outline your concern(s) and what could lessen these cost concerns?Are you concerned about the amount of time required to participate in a clinical trial?YesNoPlease outline your concern(s) and what could lessen these time concerns?Has your loved one participated in a previous FTD-GRN trial that is over? YesNoWould you like to enroll them in another FTD-GRN trial?Rate 1 out of 5Rate 2 out of 5Rate 3 out of 5Rate 4 out of 5Rate 5 out of 51 star = Not likely | 5 stars = Very likelyIf you enrolled your loved one in a trial in the past, did you regret that choice?YesNoWhat would you have done differently?What would you like the FDA and/or other health authorities to know when making decisions about new potential treatments for FTD-GRN?Would you be comfortable participating in a clinical trial that may involve receiving a placebo surgery, meaning some participants would undergo a surgical procedure but would not receive the active investigational therapy?Rate 1 out of 5Rate 2 out of 5Rate 3 out of 5Rate 4 out of 5Rate 5 out of 51 star = Not comfortable | 5 stars = Very comfortable A placebo surgery is a procedure in which a participant undergoes surgery but does not receive the active therapy. In placebo-controlled studies, participants are not told whether they received the active study treatment or the placebo. Participants who receive placebo surgery may still face risks associated with the procedure, such as anesthesia, surgical drilling, infection, and other surgical complications.What are your views on the use of placebo surgeries as controls in clinical trials studying therapies administered directly to the brain or cerebrospinal fluid?If your loved one was enrolled in a clinical trial, would you be comfortable allowing researchers to share their de-identified data (information with your loved one's name and personal information removed) with other scientists to help speed up the search for FTD-GRN treatments?YesNoUnsureWhy are you against or unsure about sharing their data? What would you need to know to feel more comfortable sharing data?If a clinical trial were not available to your loved one in their home country, would they be willing to travel to participate in a trial in another country?YesNoUnsureFTD-GRN Survey Participant/Treatment DemographicsWhat form of FTD-GRN was your loved one diagnosed with? (select all that apply)bvFTD - behavioral variant FTD, frontotemporal dementia, FTD dementia, or Pick's diseaseCBD or CBS - corticobasal degeneration, or corticobasal syndromeFTD with ALS (amyotrophic lateral sclerosis), or FTD with MND (motor neuron disease)PPA - primary progressive aphasia (including nfvPPA, svPPA, lvPPA)PSP - progressive supranuclear palsy, or Richardson's syndromeI'm not sureIf other members of your loved one's family were diagnosed with FTD-GRN, what form of the disease were they diagnosed with? (select all that apply)bvFTD - behavioral variant FTD, frontotemporal dementia, FTD dementia, or Pick’s diseaseCBD or CBS - corticobasal degeneration, or corticobasal syndromePPA - primary progressive aphasia (including nfvPPA, svPPA, lvPPA)PSP - progressive supranuclear palsy, or Richardson's syndromeI'm not sureWhat symptoms has your loved one experienced with their FTD-GRN diagnosis? (select all that apply)Language (speaking, finding words, understanding, knowing the meaning of objects)Memory (remembering recent events, learning new information)Thinking (solving problems, making judgments, organizing)Spatial (judging distances, perceiving objects)Personality (acting differently or inappropriately in a social situation)Relationships (getting along with others)Mood (anxious, not interested, depressed, irritable, emotional outbursts)Motor (tremor, balance, performing movements, walking)Eating or drinking (cravings, alcohol intake, weight gain)Behavior (repetitive or compulsive behavior, rigid routines)Sleep (not sleeping through the night, bad dreams, sleeping too much)Delusions or hallucinationsIncontinenceOtherCaregiver - Changes in loved one - OtherWhich potential clinical trial outcomes would be most meaningful to you? (select all that apply)Delaying the onset of symptoms for years or decades in people who are not yet symptomatic.Preventing symptoms from ever developing in people who are not yet symptomatic.Slowing the progression of disease after symptoms have begun, so that daily functions and independence can be maintained longer even if they eventually decline.Reducing the impact of the one or two symptoms that are most distressing, even if the disease continues to progress and lifespan is not extended.Improving or stabilizing biomarkers, such as progranulin or neurofilament levels, even without noticeable changes in symptoms.Was your loved one prescribed medications to address their symptoms?YesNoUnsureIn your opinion did any of these medications help alleviate their symptoms?Has your loved one done any of the following to help manage their symptoms? (select all that apply)Diet modificationExerciseTaken supplementsMeditationSupport GroupSpeech-language therapyOccupational therapyPhysical therapyResearch involving an experimental medicationResearch or medical care involving brain stimulation (electrodes or magnets placed on head)In your opinion, have any of the above interventions alleviated their symptoms?YesNoWhere did you hear about these interventions?Have there been financial impacts associated with your loved one's diagnosis?YesNo(example: cost of care, lost wages, negative impact on personal finances, debt accumulation)Please describe the financial impacts.How long was the period of time between the development of symptoms and your loved one's diagnosis of FTD-GRN?If a loved one was diagnosed and passed away from FTD-GRN, how long was the period of time between the development of symptoms and their passing?Submit
