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Phase I/II Safety and Tolerability Study of striatal administration of AB-1001 in adult subjects with Early Manifest Huntington's Disease (HD)
EU CTIS ID: 2024-510895-18-00
What this study is testing
Assess the safety and tolerability of a one- time, bilateral administration of AB-1001 into the caudate nucleus and putamen; Select the dose of AB-1001 to be utilized in the Expansion Part
- Phase I and Phase II (Integrated)- First administration to humans
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 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 compatible with the local delivery of the study drug: Putamen ≥ 2.3 cm3 (per side); Caudate ≥ 1.7 cm3 on Screening MRI.
- All HD concomitant medications are stable for at least 30 days prior to screening at the investigator’s discretion.
You likely can't join if
- Subjects with prior metastatic neoplasms
- Subjects with active psychosis
- Subjects with confused state
- Subjects with violent and/or suicidal behavior
- Subjects with prior strokes
- Subjects with intracranial hemorrhage
See the full eligibility criteria
- 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 compatible with the local delivery of the study drug: Putamen ≥ 2.3 cm3 (per side); Caudate ≥ 1.7 cm3 on Screening MRI.
- All HD concomitant medications are stable for at least 30 days prior to screening at the investigator’s discretion.
- Subjects with prior metastatic neoplasms
- Subjects with active psychosis
- Subjects with confused state
- Subjects with violent and/or suicidal behavior
- Subjects with prior strokes
- Subjects with intracranial hemorrhage
- Subjects with active infection
- Subjects with contraindication to MRI or gadolinium and any other medical condition that in the investigator’s opinion would interfere with the study conduct and assessments will be excluded.
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 65+ years. The study team makes the final eligibility decision.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.