Huntington DiseaseI2BIO-HDNCT05808153
Innovative Imaging and Cognitive BIOmarkers to Predict Huntington's Disease Progression
A study testing radiotracer injection in people with Huntington Disease, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
Intro Huntington's disease (HD) patients suffer from motor, cognitive and behavioral impairments, with heterogeneous phenotypes and variable time course. This leads to a high variance of HD markers, none of which is currently sensitive enough to 1) measure disease progression from small cohort data, 2) predict disease… Read more about this study
- Who is it for?
- Ages 18 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Huntington Disease
- What is being tested?
- radiotracer injection
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 80 participants
- Where
- 1 siteFrance
- Study dates
- 21 Mar 2024to 2 Feb 2027
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
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 to 65 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- For all participants:
- Age ≥18 years and ≤65 years
- Information and collection of written consent
- Affiliation with a social security plan, beneficiary or beneficiary's right
- Healthy controls
- UHDRS functional score TFC = 13
Show all 21Show fewer
- Motor UHDRS score TMS < 6 With no known genetic disease and no direct relationship to an HD patient or family ancestors carrying the HD mutation (or knowing their genetic status with CAG < 36).
- Manifest carriers
- Number of GACs ≥ 40
- CAP score ≥ 250
- 10 ≤ TFC ≤ 13
- TMS >5 if TFC=13
- Diagnostic confidence level =4
- Age of onset of disease > 20 years
- Patients physically able to sign consent
- Premanifest carriers
- Number of GACs ≥ 40
- CAP score ≥250
- CFT = 13
- TMS < 6
- Patients physically able to sign consent
Who cannot join
- Participant under guardianship or curatorship
- Neurological or psychiatric disorder unrelated to HD
- Intercurrent illness that may impact participant's performance
- Chronic progressive neurological disease
- Claustrophobia
- Brain injury unrelated to HD
Show all 21Show fewer
- Pacemaker, intracorporeal metal, intracerebral clip, any metallic foreign body: implantable cardiac electronic device such as pacemakers, implantable cardioverter defibrillators etc., metallic intraocular foreign bodies, implantable neurostimulation systems, cochlear implants/ear implants, drug infusion pumps (insulin administration, analgesic drugs), or chemotherapy pumps): if possible, the patient should remove the device.
- Catheters with metal components (Swan-Ganz catheter), metal fragments such as bullets, shotgun pellets and metal shrapnel, cerebral artery aneurysm clips, magnetic dental implants, tissue expander, artificial limb, hearing aid, piercing such as pacemaker,
- Known hypersensitivity to the radiopharmaceutical preparation (excipients in the radiopharmaceutical preparation)
- Pregnant or breastfeeding woman
- Person under state medical aid
- Person deprived of liberty
- Person participating or having participated in an interventional study for less than 3 months or without time limit in a trial of neural transplants or gene therapy.
- Person participating or having participated in a research protocol with a radiopharmaceutical injection for less than 12 months.
- Neurological or psychiatric disorder unrelated to HD
- Intercurrent disease that may impact participant's performance
- Chronic progressive neurological disease
- Claustrophobia
- Brain injury unrelated to HD
- Pacemaker, intracorporeal metal, intracerebral clip
- Pregnant, breastfeeding or wanting to procreate during participation in the study.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
France1 site, 1 recruiting
- Recruiting
Hopital Henri MONDOR
Créteil, Île-de-France Region, 94000
About this study
Intro Huntington's disease (HD) patients suffer from motor, cognitive and behavioral impairments, with heterogeneous phenotypes and variable time course. This leads to a high variance of HD markers, none of which is currently sensitive enough to 1) measure disease progression from small cohort data, 2) predict disease entry in carriers of the HD mutation (during the prodromal phase or in patients considered asymptomatic: pre-HD patients), and 3) measure a significant evolution of the state of pre-HD patients over a time window compatible with the realization of clinical trials (about 2/3 years). Moreover, the markers of HD do not allow a fine stratification of the patients.
Hypothesis/Objective Our objectives are 1) to evaluate the sensitivity of new markers and assessment tools for symptomatic (HD) and presymptomatic (pre-HD) patients, 2) to define a model of disease progression, and 3) to establish an enrichment strategy to improve patient selection for future therapeutic trials.
Read more
Method We will evaluate newly developed cognitive tests, multimodal imaging techniques, biological markers and use innovative statistical approaches.
We will follow 60 patients with the mutation responsible for MH (40 presymptomatic pre-MH patients, 20 symptomatic MH patients) and 20 healthy volunteers (controls) over a 24-month period.
- Conditions
- Huntington Disease
- Being tested
- radiotracer injection
- Keywords
- Huntington's disease, Biomarkers, Cognition, Disease progression, Imaging
- Medical subject headings
- Huntington Disease, Disease Progression
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
radiotracer injectionRadiation
MRI with radiotracer injection
| Study group | Role | What they receive |
|---|---|---|
| Symptomatic (MH) and pre-symptomatic (preMH) patients | Experimental | * Number of GAC ≥ 40
* GAP score ≥ 250
* 10 ≤ TFC ≤ 13
* TMS >5 if TFC=13
* Diagnostic confidence level =4
* Age onset of the disease > 20 years
* Patients in physical capacity to sign the consent Receives: Radiation: radiotracer injection |
| Age-matched controls (healthy volunteers) | Active comparator | * TFC functional UHDRS score = 13
* TMS engine UHDRS rating < 6 Receives: Radiation: radiotracer injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Genetic markers
Measured: Visit Month 0
Secondary outcomes (4)
cognitive tests
Cognitive scores - Neurological scores- Psychiatric scores
Measured: Visits Month 0, Month 1, Month 12, Month 24
biological markers
Neuroinflammation markers in blood - Neurodegeneration markers in blood
Measured: Visits Month 0, Month 1, Month 12, Month 24
multimodal imaging techniques
MRI
Measured: Visits Month 0, Month 12, Month 24
multimodal imaging techniques
PET/MRI
Measured: Visits Month 0, Month 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Apr 2023
- Study start21 Mar 2024
- Primary completion2 Apr 2025 (estimated)
- Study completion2 Feb 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Huntington DiseaseStudies 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
- Non-randomised
- Intervention model
- Single group
- Model description
- Model description: Identification, evaluation and validation of new clinical, biological and imaging biomarkers (MRI without contrast product and PET with intravenous administration of a radiotracer) on a prospective cohort of carriers of the mutation responsible for Huntington's disease and healthy volunteers.
- Masking
- None (open label)
- Enrolment
- 80 participants (estimated)
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
- NCT05808153
- Sponsor's protocol ID
- APHP210360
- Other IDs
- 2021-004141-20
- Acronym
- I2BIO-HD
- Status last verified
- Jan 2024
- First submitted
- 15 Dec 2022
- Last updated
- 7 May 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 7 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Innovative Imaging and Cognitive BIOmarkers to Predict Huntington's Disease Progression
- ClinicalTrials.gov ID
- NCT05808153
- Acronym / sponsor ID
- I2BIO-HD / APHP210360
- Conditions
- Huntington Disease
- Keywords
- Huntington's disease, Biomarkers, Cognition, Disease progression, Imaging
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- 21 Mar 2024 (actual)
- Primary completion
- 2 Apr 2025 (estimated)
- Study completion
- 2 Feb 2027 (estimated)
- First posted
- 11 Apr 2023 (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.
Intro Huntington's disease (HD) patients suffer from motor, cognitive and behavioral impairments, with heterogeneous phenotypes and variable time course. This leads to a high variance of HD markers, none of which is currently sensitive enough to 1) measure disease progression from small cohort data, 2) predict disease entry in carriers of the HD mutation (during the prodromal phase or in patients considered asymptomatic: pre-HD patients), and 3) measure a significant evolution of the state of pre-HD patients over a time window compatible with the realization of clinical trials (about 2/3 years). Moreover, the markers of HD do not allow a fine stratification of the patients.
Hypothesis/Objective Our objectives are 1) to evaluate the sensitivity of new markers and assessment tools for symptomatic (HD) and presymptomatic (pre-HD) patients, 2) to define a model of disease progression, and 3) to establish an enrichment strategy to improve patient selection for future therapeutic trials.
Method We will evaluate newly developed cognitive tests, multimodal imaging techniques, biological markers and use innovative statistical approaches.
We will follow 60 patients with the mutation responsible for MH (40 presymptomatic pre-MH patients, 20 symptomatic MH patients) and 20 healthy volunteers (controls) over a 24-month period.
Study design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Model description: Identification, evaluation and validation of new clinical, biological and imaging biomarkers (MRI without contrast product and PET with intravenous administration of a radiotracer) on a prospective cohort of carriers of the mutation responsible for Huntington's disease and healthy volunteers.
| Arm | Type | Receives |
|---|---|---|
| Symptomatic (MH) and pre-symptomatic (preMH) patients | Experimental | Radiation: radiotracer injection* Number of GAC ≥ 40 * GAP score ≥ 250 * 10 ≤ TFC ≤ 13 * TMS >5 if TFC=13 * Diagnostic confidence level =4 * Age onset of the disease > 20 years * Patients in physical capacity to sign the consent |
| Age-matched controls (healthy volunteers) | Active comparator | Radiation: radiotracer injection* TFC functional UHDRS score = 13 * TMS engine UHDRS rating < 6 |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (21 listed)
- For all participants:
- Age ≥18 years and ≤65 years
- Information and collection of written consent
- Affiliation with a social security plan, beneficiary or beneficiary's right
- Healthy controls
Key exclusion criteria (21 listed)
- Participant under guardianship or curatorship
- Neurological or psychiatric disorder unrelated to HD
- Intercurrent illness that may impact participant's performance
- Chronic progressive neurological disease
- Claustrophobia
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 |
|---|---|---|
| radiotracer injectionMRI with radiotracer injection | Radiation | Age-matched controls (healthy volunteers), Symptomatic (MH) and pre-symptomatic (preMH) patients |
Primary outcome
Genetic markers
Time frame: Visit Month 0
4 secondary outcomes
cognitive tests
Time frame: Visits Month 0, Month 1, Month 12, Month 24
biological markers
Time frame: Visits Month 0, Month 1, Month 12, Month 24
multimodal imaging techniques
Time frame: Visits Month 0, Month 12, Month 24
multimodal imaging techniques
Time frame: Visits Month 0, Month 24
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 May 2024.
- Individual participant dataSharing planned: Undecided
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing radiotracer injection in people with Huntington Disease, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
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 21 inclusion and 21 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: looking for participants now, as of the sponsor's last update on 7 May 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Anne-Catherine BACHOUD-LEVI, PhD ((+33)1 49 81 23 10, anne-catherine.bachoud-levi@aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Genetic markers (time frame: Visit Month 0). 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 7 May 2024.