Frontotemporal Dementia (FTD)PREVENT-PGRNNCT06711510
Predict to Prevent PGRN Disease
A study testing Clinical, behavioral and cognitive evaluation, blood sampling and brain MRI in people with Frontotemporal Dementia (FTD) and GRN Related Frontotemporal Dementia, run by Assistance Publique - Hôpitaux de Paris.
Trial summary
Progranulin (GRN or PGRN) mutations are among the most common genetic causes of frontotemporal lobar degeneration (FTLD). With the advent of gene-specific therapeutic interventions, an accurate knowledge of the presymptomatic phase of the disease is of utmost importance. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Frontotemporal Dementia (FTD), GRN Related Frontotemporal Dementia
- What is being tested?
- Clinical, behavioral and cognitive evaluation, blood sampling and brain MRI
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Jan 2025to Mar 2030
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- For all :
- signature of informed consent for the study
- being French speaker
- having social security coverage
- absence of any other neurological disease
- For asymptomatic at-risk individuals :
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- being first-degree relative of a GRN mutation carrier OR of a patient suffering from FTD with discovery of a GRN mutation in their family
- absence of any neurological troubles related to the disease
- For patients :
- being carrier of a pathogenic GRN mutation
- diagnosis of behavioural-variant frontotemporal dementia according to Rasckvsky et al. criteria (2011) or any related clinical syndromes, such as primary progressive aphasia according to Gorno-Tempini et al. criteria (2011)
Who cannot join
- For all :
- any contraindication to brain MRI (pace-maker, ferromagnetic heart valve, aneurysm clips or any other ferromagnetic foreign body implanted in brain/eye or other critical locations, non-MRI compatible intrauterine devices, claustrophobia)
- chronic alcohol use / addiction
- known chronic kidney disease
- hypersensitivity to local anaesthetics
- impossibility of laying down for 1 hour without moving
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- pregnant of breastfeeding women, or any beta-HCG positive test for women in childbearing age N.B.: participation in therapeutic essays is not an exclusion criteria, but should be discussed with the study investigator in every case.
- For asymptomatic at-risk individuals :
- Peaple under legal protection
About this study
Progranulin (GRN or PGRN) mutations are among the most common genetic causes of frontotemporal lobar degeneration (FTLD). With the advent of gene-specific therapeutic interventions, an accurate knowledge of the presymptomatic phase of the disease is of utmost importance. Increases of plasma neurofilament light chain (NfL) levels are good predictors of phenoconversion in presymptomatic carriers. However their increase rates remain partially elucidated insofar, with many confounding factors. Another point which deserves further precision is the definition of the biological onset of the disease, via the identification of markers of intraneuronal accumulation of TDP-43 protein. PREVENT-PGRN aims aims at studying the trajectory of plasma NfL changes in presymptomatic GRN mutation carriers in comparison with healthy controls, in partnership with the GENFI-QBS study. Additionally, other disease-related biomarkers, namely associated with TDP-43 pathology, will be investigated in this study, at the presymptomatic and clinical phase.
Read the detailed description
Despite several advances in the understanding of the presymptomatic phase of GRN-associated FTLD, some aspects concerning longitudinal biomarker changes and their capacity to predict phenoconversion have been only partially elucidated. Plasma NfL levels proved to be good tools for monitoring neurodegeneration in the years preceding phenoconversion, but thresholds for cross-sectional values and longitudinal increase rates need to be validated in international studies on large populations. In addition, numerous confounding factors (head trauma, epilepsy, metabolic disorders, etc.) may punctually impact plasma NfL dosages. This may suggest that several measurements spaced over time are best predictors of phenoconversion rather than a single assay. Whether cross-sectional NfL levels or their rate of change during follow-up are the best parameter for individual monitoring in the presymptomatic and clinical phases still has to be determined. This knowledge is an essential prerequisite for all current and future therapeutic trials designed for GRN carriers at the presymptomatic phase.
To answer this question, this project aims to define the trajectory of plasma NfL levels as exactly as possible in carriers of GRN gene mutations during the presymptomatic and clinical phase. NfL dosages will be performed every 3 months over a 2-year period and carriers' trajectories will be compared with those measured in age-matched control individuals. This study takes place in the frame of the international collaborative project GENFI-QBS substudy (quarterly blood sampling), aiming at characterizing the evolution of plasma biomarkers in genetic FTLD by repeated assays every 3 months. Plasma samples will also allow complementary biomarker analyses, providing a broader biomarker panel to better elucidate the pathophysiological mechanisms of the disease. Notably, the validation of in vivo TDP-43 biomarkers and the tracking of the longitudinal progression of the proteinopathy represent other major challenges. Indeed, the advent of such a biomarker in asymptomatic GRN carriers would allow to establish the start of the lesional process (i.e., TDP-43 accumulation) and thus the appropriate moment to start preventive treatments.
Therefore, this study will enrol 90 participants, including 10 patients with early-stage FTD (or other clinically defined syndromes within FTLD spectrum) carrying pathogenic GRN mutations and 80 asymptomatic at-risk individuals (i.e., first-degree relatives of individuals carrying a GRN mutation. These latter will thus be either presymptomatic GRN carriers (~50% risk) or controls. The determination of the genetic status of asymptomatic participants will be done in research setting, and results will be blinded to participants and investigators. A part of study participants will be selected from Predict-PGRN (NCT04014673), a natural history study aiming at characterizing the presymptomatic phase of PGRN disease over 5-year follow-up.
A comprehensive evaluation including neurological examination, cognitive and behavioral assessment, brain MRI, biological blood sampling and olfactory swab sampling will be performed at inclusion, at 12 months and at 24 months. Neuropsychological, behavioral and MRI data will allow to compare additional cognitive-behavioral and neuroimaging markers between carriers and non-carriers, providing further information on the clinical progression and the risk of phenoconversion of the former. As optional assays, upon participants' specific consent, CSF sampling and skin biopsy will be proposed at the same time-points, in order to provide additional substrates to look for biomarkers of TDP-43 accumulation, together with plasma and olfactory mucosa samples. Intermediate visits will be made every 3 months (at month 3, 6, 9, 15, 18 and 21), only for quick clinical follow-up and blood sampling. The purpose of these visits is to enable close monitoring of blood biomarkers, namely NfL, but also to document the occurrence of intercurrent events and to monitor participants' clinical status.
- Being tested
- Clinical, behavioral and cognitive evaluation, blood sampling, brain MRI, olfactory swab sampling with nasal brush, skin punch biopsy
- Keywords
- Frontotemporal dementia, frontotemporal lobar degeneration, GRN, PGRN, mutation, , neurofilament light chain (NfL), biomarker, amyotrophic lateral sclerosis, primary progressive aphasia
- Medical subject headings
- Frontotemporal Dementia, Frontotemporal Lobar Degeneration, Charcot-Marie-Tooth disease, Type 1F, Amyotrophic Lateral Sclerosis, Aphasia, Primary Progressive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Clinical, behavioral and cognitive evaluationOther
Questionnaries and tests
blood samplingOther
blood sampling (56ml max)
brain MRIOther
brain MRI (35 to 70 minutes)
olfactory swab sampling with nasal brushOther
olfactory swab sampling with nasal brush
skin punch biopsyOther
skin punch biopsy (optional intervention)
lumbar puncture for CSF samplingOther
lumbar puncture for CSF sampling (optional intervention)
| Study group | Role | What they receive |
|---|---|---|
| Standardized protocol for all participants | Other | Nine evaluations (every 3 months) over a 2-year follow-up, with the same standardized protocol for all participants, including:
* At the inclusion visit (M0), after 12 months (M12) and after 24 months (M24):
* clinical (neurological) evaluation
* behavioral examination
* cognitive testing
* blood sampling (determination of carrier / non-carrier status by means of GRN testing will be done at M0 visit; results will remain strictly confidential and not disclosed to study participants and investigators)
* brain MRI
* olfactory swab sampling with nasal brush
* skin punch biopsy (optional)
* lumbar puncture for CSF sampling (optional)
* For the remaining visits (M3, M6, M9, M15, M18, M21):
* clinical evaluation
* blood sampling Receives: Other: Clinical, behavioral and cognitive evaluation; Other: blood sampling; Other: brain MRI; Other: olfactory swab sampling with nasal brush; Other: skin punch biopsy; Other: lumbar puncture for CSF sampling |
What is being measured
The primary outcome is the main question the study is designed to answer.
Compare the trajectory of plasma NfL between GRN mutation carriers, during the presymptomatic and clinical phases, and non-carrier controls, to improve identification of early disease phases and guide future therapeutic trials.
Cross-sectional and longitudinal comparisons of NfL assays by Simoa® technique on plasma samples collected every 3 months during the 9 study visits (M0, M3, M6, M9, M12, M15, M18, M21, M24), between GRN mutation carriers and non-carrier controls.
Measured: 2 years
Secondary outcomes (5)
Compare the levels of other known biomarkers of interest in fluids (plasma, CSF) (such as GFAP, for example) between GRN mutation carriers, during the presymptomatic and clinical phases, and non-carrier controls.
Cross-sectional comparisons of other fluid biomarkers known to be associated with FTLD disease (e.g., GFAP) sampled every 3 months for 2 years (9 time-points) between GRN mutation carriers and controls.
Measured: 2 years
Compare the trajectory of other known biomarkers of interest in fluids (plasma, CSF) mentioned above (such as GFAP, for example) between carriers of GRN gene mutations, during the presymptomatic and clinical phases, and non-carrier controls.
Longitudinal comparisons of the trajectories of other fluid biomarkers known to be associated with FTLD disease (e.g., GFAP) sampled every 3 months for 2 years (9 time-points) between GRN mutation carriers and controls.
Measured: 2 years
Discover new fluid biomarkers (plasma, CSF) and peripheral biomarkers (nasal mucosa, fibroblasts) targeting, in particular, the detection of the TDP-43 protein (by RT-QuIC, among others).
Comparisons of the level of new fluid (plasma, CSF) and peripheral (nasal brush, skin biopsy) biomarkers targeting TDP-43 pathology between patients, presymptomatic carriers and controls.
Measured: 2 years
Establish correlations between biomarker trajectories and neuropsychological profiles
Correlations between the levels of measured biomarkers with neuropsychological and behavioral scores obtained at each yearly visit (3 time-points).
Measured: 2 years
Establish correlations between biomarker trajectories and neuroimaging profiles
Correlations between the levels of measured biomarkers with structural and functional neuroimaging data obtained at each yearly visit (3 time-points).
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Dec 2024
- Study startJan 2025 (estimated)
- Primary completionMar 2030 (estimated)
- Study completionMar 2030 (estimated)
Why it stopped: loss of funding
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Frontotemporal Dementia (FTD)Studies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Nine evaluations (every 3 months) over a 2-year follow-up, with the same standardized protocol for all participants, including: * At the inclusion visit (M0), after 12 months (M12) and after 24 months (M24): * clinical (neurological) evaluation * behavioral examination * cognitive testing * blood sampling (determination of carrier / non-carrier status by means of GRN testing will be done at M0 visit; results will remain strictly confidential and not disclosed to study participants and investigators) * brain MRI * olfactory swab sampling with nasal brush * skin punch biopsy (optional) * lumbar puncture for CSF sampling (optional) * For the remaining visits (M3, M6, M9, M15, M18, M21): * clinical evaluation * blood sampling
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06711510
- Sponsor's protocol ID
- APHP240482
- Acronym
- PREVENT-PGRN
- Status last verified
- Mar 2025
- First submitted
- 21 Nov 2024
- Last updated
- 21 Mar 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Predict to Prevent PGRN Disease
- ClinicalTrials.gov ID
- NCT06711510
- Acronym / sponsor ID
- PREVENT-PGRN / APHP240482
- Keywords
- Frontotemporal dementia, frontotemporal lobar degeneration, GRN, PGRN, mutation, , neurofilament light chain (NfL), biomarker, amyotrophic lateral sclerosis
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: loss of funding
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- Jan 2025 (estimated)
- Primary completion
- Mar 2030 (estimated)
- Study completion
- Mar 2030 (estimated)
- First posted
- 2 Dec 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Progranulin (GRN or PGRN) mutations are among the most common genetic causes of frontotemporal lobar degeneration (FTLD). With the advent of gene-specific therapeutic interventions, an accurate knowledge of the presymptomatic phase of the disease is of utmost importance. Increases of plasma neurofilament light chain (NfL) levels are good predictors of phenoconversion in presymptomatic carriers. However their increase rates remain partially elucidated insofar, with many confounding factors. Another point which deserves further precision is the definition of the biological onset of the disease, via the identification of markers of intraneuronal accumulation of TDP-43 protein. PREVENT-PGRN aims aims at studying the trajectory of plasma NfL changes in presymptomatic GRN mutation carriers in comparison with healthy controls, in partnership with the GENFI-QBS study. Additionally, other disease-related biomarkers, namely associated with TDP-43 pathology, will be investigated in this study, at the presymptomatic and clinical phase.
Detailed description
Despite several advances in the understanding of the presymptomatic phase of GRN-associated FTLD, some aspects concerning longitudinal biomarker changes and their capacity to predict phenoconversion have been only partially elucidated. Plasma NfL levels proved to be good tools for monitoring neurodegeneration in the years preceding phenoconversion, but thresholds for cross-sectional values and longitudinal increase rates need to be validated in international studies on large populations. In addition, numerous confounding factors (head trauma, epilepsy, metabolic disorders, etc.) may punctually impact plasma NfL dosages. This may suggest that several measurements spaced over time are best predictors of phenoconversion rather than a single assay. Whether cross-sectional NfL levels or their rate of change during follow-up are the best parameter for individual monitoring in the presymptomatic and clinical phases still has to be determined. This knowledge is an essential prerequisite for all current and future therapeutic trials designed for GRN carriers at the presymptomatic phase.
To answer this question, this project aims to define the trajectory of plasma NfL levels as exactly as possible in carriers of GRN gene mutations during the presymptomatic and clinical phase. NfL dosages will be performed every 3 months over a 2-year period and carriers' trajectories will be compared with those measured in age-matched control individuals. This study takes place in the frame of the international collaborative project GENFI-QBS substudy (quarterly blood sampling), aiming at characterizing the evolution of plasma biomarkers in genetic FTLD by repeated assays every 3 months. Plasma samples will also allow complementary biomarker analyses, providing a broader biomarker panel to better elucidate the pathophysiological mechanisms of the disease. Notably, the validation of in vivo TDP-43 biomarkers and the tracking of the longitudinal progression of the proteinopathy represent other major challenges. Indeed, the advent of such a biomarker in asymptomatic GRN carriers would allow to establish the start of the lesional process (i.e., TDP-43 accumulation) and thus the appropriate moment to start preventive treatments.
Therefore, this study will enrol 90 participants, including 10 patients with early-stage FTD (or other clinically defined syndromes within FTLD spectrum) carrying pathogenic GRN mutations and 80 asymptomatic at-risk individuals (i.e., first-degree relatives of individuals carrying a GRN mutation. These latter will thus be either presymptomatic GRN carriers (~50% risk) or controls. The determination of the genetic status of asymptomatic participants will be done in research setting, and results will be blinded to participants and investigators. A part of study participants will be selected from Predict-PGRN (NCT04014673), a natural history study aiming at characterizing the presymptomatic phase of PGRN disease over 5-year follow-up.
A comprehensive evaluation including neurological examination, cognitive and behavioral assessment, brain MRI, biological blood sampling and olfactory swab sampling will be performed at inclusion, at 12 months and at 24 months. Neuropsychological, behavioral and MRI data will allow to compare additional cognitive-behavioral and neuroimaging markers between carriers and non-carriers, providing further information on the clinical progression and the risk of phenoconversion of the former. As optional assays, upon participants' specific consent, CSF sampling and skin biopsy will be proposed at the same time-points, in order to provide additional substrates to look for biomarkers of TDP-43 accumulation, together with plasma and olfactory mucosa samples. Intermediate visits will be made every 3 months (at month 3, 6, 9, 15, 18 and 21), only for quick clinical follow-up and blood sampling. The purpose of these visits is to enable close monitoring of blood biomarkers, namely NfL, but also to document the occurrence of intercurrent events and to monitor participants' clinical status.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Nine evaluations (every 3 months) over a 2-year follow-up, with the same standardized protocol for all participants, including: * At the inclusion visit (M0), after 12 months (M12) and after 24 months (M24): * clinical (neurological) evaluation * behavioral examination * cognitive testing * blood sampling (determination of carrier / non-carrier status by means of GRN testing will be done at M0 visit; results will remain strictly confidential and not disclosed to study participants and investigators) * brain MRI * olfactory swab sampling with nasal brush * skin punch biopsy (optional) * lumbar puncture for CSF sampling (optional) * For the remaining visits (M3, M6, M9, M15, M18, M21): * clinical evaluation * blood sampling
| Arm | Type | Receives |
|---|---|---|
| Standardized protocol for all participants | Other | Other: Clinical, behavioral and cognitive evaluation, Other: blood sampling, Other: brain MRI, Other: olfactory swab sampling with nasal brush, Other: skin punch biopsy, Other: lumbar puncture for CSF samplingNine evaluations (every 3 months) over a 2-year follow-up, with the same standardized protocol for all participants, including: * At the inclusion visit (M0), after 12 months (M12) and after 24 months (M24): * clinical (neurological) evaluation * behavioral examination * cognitive testing * blood sampling (determination of carrier / non-carrier status by means of GRN testing will be done at M0 visit; results will remain strictly confidential and not disclosed to study participants and investigators) * brain MRI * olfactory swab sampling with nasal brush * skin punch biopsy (optional) * lumbar puncture for CSF sampling (optional) * For the remaining visits (M3, M6, M9, M15, M18, M21): * clinical evaluation * blood sampling |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- For all :
- For asymptomatic at-risk individuals :
- For patients :
Key exclusion criteria (9 listed)
- For all :
- For asymptomatic at-risk individuals :
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Clinical, behavioral and cognitive evaluationQuestionnaries and tests | Other | Standardized protocol for all participants |
| blood samplingblood sampling (56ml max) | Other | Standardized protocol for all participants |
| brain MRIbrain MRI (35 to 70 minutes) | Other | Standardized protocol for all participants |
| olfactory swab sampling with nasal brusholfactory swab sampling with nasal brush | Other | Standardized protocol for all participants |
| skin punch biopsyskin punch biopsy (optional intervention) | Other | Standardized protocol for all participants |
| lumbar puncture for CSF samplinglumbar puncture for CSF sampling (optional intervention) | Other | Standardized protocol for all participants |
Primary outcome
Compare the trajectory of plasma NfL between GRN mutation carriers, during the presymptomatic and clinical phases, and non-carrier controls, to improve identification of early disease phases and guide future therapeutic trials.
Time frame: 2 years
Cross-sectional and longitudinal comparisons of NfL assays by Simoa® technique on plasma samples collected every 3 months during the 9 study visits (M0, M3, M6, M9, M12, M15, M18, M21, M24), between GRN mutation carriers and non-carrier controls.
5 secondary outcomes
Compare the levels of other known biomarkers of interest in fluids (plasma, CSF) (such as GFAP, for example) between GRN mutation carriers, during the presymptomatic and clinical phases, and non-carrier controls.
Time frame: 2 years
Compare the trajectory of other known biomarkers of interest in fluids (plasma, CSF) mentioned above (such as GFAP, for example) between carriers of GRN gene mutations, during the presymptomatic and clinical phases, and non-carrier controls.
Time frame: 2 years
Discover new fluid biomarkers (plasma, CSF) and peripheral biomarkers (nasal mucosa, fibroblasts) targeting, in particular, the detection of the TDP-43 protein (by RT-QuIC, among others).
Time frame: 2 years
Establish correlations between biomarker trajectories and neuropsychological profiles
Time frame: 2 years
Establish correlations between biomarker trajectories and neuroimaging profiles
Time frame: 2 years
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Mar 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A study testing Clinical, behavioral and cognitive evaluation, blood sampling and brain MRI in people with Frontotemporal Dementia (FTD) and GRN Related Frontotemporal Dementia, run by Assistance Publique - Hôpitaux de Paris.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 21 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Compare the trajectory of plasma NfL between GRN mutation carriers, during the presymptomatic and clinical phases, and non-carrier controls, to improve identification of early disease phases and guide future therapeutic trials. (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Mar 2025.