Huntington DiseasePhase 1/2NCT05541627
A Study to Evaluate AB-1001 Striatal Administration in Adults With Early Manifest Huntington's Disease
A Phase 1/2 study testing AB-1001 Gene Therapy in people with Huntington Disease, run by AskBio France, SAS, a subsidiary of AskBio Inc. at 1 site in France.
Trial summary
A Phase I/II Dose-Finding Study to Evaluate Striatal Administration of AB-1001 (previously BV-101) in Adults with Early Manifest Huntington's Disease Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Huntington Disease
- What is being tested?
- AB-1001 Gene Therapy
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteFrance
- Study dates
- 12 Oct 2022to Apr 2028
- Phase
- Phase 1/2
- 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
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or Female subjects between ages 18 and 65 years (both inclusive) at time of consenting, able to provide Informed Consent and able to understand and comply with all study procedures.
- Documented genetic confirmation of pathological CAG expansion in the huntingtin gene ≥40.
- Early manifest HD as defined by a UHDRS total functional capacity (TFC) score of 9 to 13 and a diagnostic classification level (DCL) of 4, or a DCL of 3 if present with cognitive impairment and clear evidence of disease progression.
- Striatal MRI volumes per hemisphere: Putamen ≥ 2.3 cm3 (per side); Caudate ≥ 1.7 cm3 (per side) on Screening MRI.
- All HD concomitant medications stable for at least 30 days prior to screening at the investigator's discretion.
Who cannot join
- Prior or ongoing medical condition, physical findings, ECG findings, or laboratory abnormality that, in the investigator's opinion, would impact subject's safety and compliance with the study procedures.
- Metastatic neoplasms within the five years prior to screening.
- Presence of clinically relevant immunologic, hematologic, hepatic, cardiac, or renal disease at the time of screening as per investigator's clinical judgment.
- Current untreated and unstable depressive disorder or a serious mood disorder requiring hospitalization.
- History of prior suicide attempt or imminent risk of self-harm based on investigator's judgment or with a "yes" answer on item 4 or 5 on the Columbia Suicide Severity Rating Scale (C-SSRS).
- Patients with history of confirmed stroke, known intracranial neoplasms, vascular malformations, or intracranial hemorrhage.
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- Subjects not deemed suitable for the surgical procedure as per the Neurosurgeon's judgment.
- Any history of gene therapy, cell transplantation or any other experimental brain surgery.
- Any RNA or DNA targeted HD specific investigational agents such as antisense oligonucleotides within 6 months prior to screening.
- Subjects unable to tolerate or unwilling to undergo multiple lumbar punctures.
- Participation in any clinical trial of an approved or non-approved investigational drug or intervention within 12 weeks or 5 half-lives whichever is longer prior to treatment.
Trial locations and contacts
1 site in 1 country.
France1 site
Institut du Cerveau (ICM), Hôpital La Pitié Salpêtrière APHP
Paris, Île-de-France Region, 75013
About this study
Official title: An Open-Label Phase I/II Dose Finding Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Striatal Administration of AB-1001 in Adult Subjects With Early Manifest Huntington's Disease (HD)
A Phase I/II Dose-Finding Study to Evaluate Striatal Administration of AB-1001 (previously BV-101) in Adults with Early Manifest Huntington's Disease
Read the detailed description
This is a Phase I/II, first-in-human, open-label study to evaluate the safety, tolerability, and preliminary efficacy signals in subjects with early manifest HD following treatment with one-time intracerebral bilateral injections of AB-1001 within the striatum (caudate and putamen).
This study consists of 2 parts: Dose-Finding Part and Expansion Part; each part consists of 3 phases: Screening Phase (8 weeks, with extension to 12 weeks to accommodate scheduling if needed), Treatment and Initial Follow-Up Phase (52 weeks) and Long-Term Follow-Up Phase (4 years). In the Dose-Finding Part, 2 dose titers will be tested in 3-6 subjects in each cohort. Once a dose is selected based on Dose-Limiting Toxicities, an additional 6 subjects will be enrolled into the Dose Expansion Part.
- Conditions
- Huntington Disease
- Being tested
- AB-1001 Gene Therapy
- Keywords
- Huntington's Disease, Early Manifest Huntington's Disease, Brain Diseases, Central Nervous System Diseases, Nervous System Diseases, Movement Disorders, Neurodegenerative Diseases, Genetic Diseases, Inborn, Cognition Disorders, Neurocognitive Disorders, Gene Therapy, AAV (adeno-associated virus), Viral Vector, Cholesterol 24-Hydroxylase, CYP46A1, AB-1001
- Medical subject headings
- Huntington Disease, Brain Diseases, Central Nervous System Diseases, Nervous System Diseases, Movement Disorders, Neurodegenerative Diseases, Genetic Diseases, Inborn, Cognition Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AB-1001 Gene TherapyGenetic
Also called AAVrh10.CAG.hCYP46A1 (previously BV-101)
One-time intracerebral bilateral injections of AB-1001 (AAVrh10.CAG.hCYP46A1), an adeno-associated viral vector serotype Rh10 containing the human cholesterol 24-hydroxylase gene
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | Low-dose of AB-1001 Receives: Genetic: AB-1001 Gene Therapy |
| Cohort 2 | Experimental | High-dose of AB-1001 Receives: Genetic: AB-1001 Gene Therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Dose-Limiting Toxicities (DLTs), Treatment-Emergent Adverse Events (TEAEs), and Serious Adverse Events (SAEs)
The incidence of DLTs, TEAEs, and SAEs will be measured according to protocol specifications.
Measured: Through Week 52
Secondary outcomes (7)
Anatomical and volumetric measures of brain regions impacted by HD as assessed by MRI
The magnitude and variability of change from baseline in anatomical and volumetric measures of brain regions impacted by HD as assessed by MRI will be measured
Measured: At Week 52
Composite Unified Huntington Disease Rating Scale (cUHDRS)
The change from baseline in the cUHDRS will be measured (a higher score indicates better functioning)
Measured: At Week 52
Mutant Huntingtin protein (mHTT)
The change from baseline in mHTT in blood and cerebrospinal fluid (CSF) will be measured
Measured: At Week 52
Neurofilament light chain (NfL)
The change from baseline in blood and CSF NfL will be measured
Measured: At Week 52
24OH cholesterol
The change from baseline in blood and CSF 24OH cholesterol will be measured
Measured: At Week 52
Magnetic resonance spectroscopy (MRS) metabolic profile
Change from baseline in MRS metabolic profile
Measured: At Week 52
Positron emission tomography (PET) fluoro-deoxyglucose (FDG) striatal profile
Change from baseline in PET FDG striatal profile
Measured: At Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Sep 2022
- Study start12 Oct 2022
- Primary completion4 Apr 2024
- Study completionApr 2028 (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.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- AskBio France, SAS, a subsidiary of AskBio Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05541627
- Sponsor's protocol ID
- ASK-HD-01-CS-101
- Status last verified
- Sep 2025
- First submitted
- 13 Sep 2022
- Last updated
- 14 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- 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 14 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Phase I/II Dose Finding Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Striatal Administration of AB-1001 in Adult Subjects With Early Manifest Huntington's Disease (HD)
- ClinicalTrials.gov ID
- NCT05541627
- Acronym / sponsor ID
- ASK-HD-01-CS-101
- Conditions
- Huntington Disease
- Keywords
- Huntington's Disease, Early Manifest Huntington's Disease, Brain Diseases, Central Nervous System Diseases, Nervous System Diseases, Movement Disorders, Neurodegenerative Diseases, Genetic Diseases, Inborn
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 5 participants (actual)
- Lead sponsor
- AskBio France, SAS, a subsidiary of AskBio Inc.Industry (pharma, biotech, devices)
- Study start
- 12 Oct 2022 (actual)
- Primary completion
- 4 Apr 2024 (actual)
- Study completion
- Apr 2028 (estimated)
- First posted
- 15 Sep 2022 (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.
A Phase I/II Dose-Finding Study to Evaluate Striatal Administration of AB-1001 (previously BV-101) in Adults with Early Manifest Huntington's Disease
Detailed description
This is a Phase I/II, first-in-human, open-label study to evaluate the safety, tolerability, and preliminary efficacy signals in subjects with early manifest HD following treatment with one-time intracerebral bilateral injections of AB-1001 within the striatum (caudate and putamen).
This study consists of 2 parts: Dose-Finding Part and Expansion Part; each part consists of 3 phases: Screening Phase (8 weeks, with extension to 12 weeks to accommodate scheduling if needed), Treatment and Initial Follow-Up Phase (52 weeks) and Long-Term Follow-Up Phase (4 years). In the Dose-Finding Part, 2 dose titers will be tested in 3-6 subjects in each cohort. Once a dose is selected based on Dose-Limiting Toxicities, an additional 6 subjects will be enrolled into the Dose Expansion Part.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Genetic: AB-1001 Gene TherapyLow-dose of AB-1001 |
| Cohort 2 | Experimental | Genetic: AB-1001 Gene TherapyHigh-dose of AB-1001 |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Male or Female subjects between ages 18 and 65 years (both inclusive) at time of consenting, able to provide Informed Consent and able to understand and comply with all study procedures.
- Documented genetic confirmation of pathological CAG expansion in the huntingtin gene ≥40.
- Early manifest HD as defined by a UHDRS total functional capacity (TFC) score of 9 to 13 and a diagnostic classification level (DCL) of 4, or a DCL of 3 if present with cognitive impairment and clear evidence of disease progression.
- Striatal MRI volumes per hemisphere: Putamen ≥ 2.3 cm3 (per side); Caudate ≥ 1.7 cm3 (per side) on Screening MRI.
- All HD concomitant medications stable for at least 30 days prior to screening at the investigator's discretion.
Key exclusion criteria (11 listed)
- Prior or ongoing medical condition, physical findings, ECG findings, or laboratory abnormality that, in the investigator's opinion, would impact subject's safety and compliance with the study procedures.
- Metastatic neoplasms within the five years prior to screening.
- Presence of clinically relevant immunologic, hematologic, hepatic, cardiac, or renal disease at the time of screening as per investigator's clinical judgment.
- Current untreated and unstable depressive disorder or a serious mood disorder requiring hospitalization.
- History of prior suicide attempt or imminent risk of self-harm based on investigator's judgment or with a "yes" answer on item 4 or 5 on the Columbia Suicide Severity Rating Scale (C-SSRS).
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 |
|---|---|---|
| AB-1001 Gene TherapyAlso: AAVrh10.CAG.hCYP46A1 (previously BV-101)One-time intracerebral bilateral injections of AB-1001 (AAVrh10.CAG.hCYP46A1), an adeno-associated viral vector serotype Rh10 containing the human cholesterol 24-hydroxylase gene | Genetic | Cohort 1, Cohort 2 |
Primary outcome
Incidence of Dose-Limiting Toxicities (DLTs), Treatment-Emergent Adverse Events (TEAEs), and Serious Adverse Events (SAEs)
Time frame: Through Week 52
The incidence of DLTs, TEAEs, and SAEs will be measured according to protocol specifications.
7 secondary outcomes
Anatomical and volumetric measures of brain regions impacted by HD as assessed by MRI
Time frame: At Week 52
Composite Unified Huntington Disease Rating Scale (cUHDRS)
Time frame: At Week 52
Mutant Huntingtin protein (mHTT)
Time frame: At Week 52
Neurofilament light chain (NfL)
Time frame: At Week 52
24OH cholesterol
Time frame: At Week 52
Magnetic resonance spectroscopy (MRS) metabolic profile
Time frame: At Week 52
Positron emission tomography (PET) fluoro-deoxyglucose (FDG) striatal profile
Time frame: At Week 52
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 14 May 2026.
- 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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing AB-1001 Gene Therapy in people with Huntington Disease, run by AskBio France, SAS, a subsidiary of AskBio Inc. 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 5 inclusion and 11 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: under way, not looking for participants, as of the sponsor's last update on 14 May 2026. 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.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
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?
Incidence of Dose-Limiting Toxicities (DLTs), Treatment-Emergent Adverse Events (TEAEs), and Serious Adverse Events (SAEs) (time frame: Through Week 52). 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 14 May 2026.