Active — Not RecruitingPhase 1/2

Safety and Efficacy of AMT-130 in European Adults With Early Manifest Huntington's Disease

Poland, United Kingdom14 participantsStarted 2021-10-07

Plain-language summary

This is the second study of AMT-130 in patients with early manifest HD and is designed as part of an integrated two-study phase I/II program under a single data safety monitoring board (DSMB) with staggered enrollment based upon continued demonstration of safety of AMT-130 administration. Cohort 3 participants will receive either high or low dose (1:1 randomization). Participants enrolled in Cohort 3 will also receive an immunosuppression regimen consisting of dexamethasone, sirolimus, and rituximab.

Who can participate

Age range25 Years – 65 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Able and willing to provide written informed consent prior to the study and study-related procedure.
✓. Male and female participants 25-65 years of age.
✓. Cohorts 1 \& 2:
✓. a DCL of 4 OR
✓. a DCL of 3 with either a positive ("Yes") response to the UHDRS Question 80 (multidimensional manifest diagnosis on motor, cognitive, behavioral, functional) or DSM5 criteria for cognitive disorder (American Psychiatric Association, 2013; MDS Task Force criteria).
✓. Cohort 3:
✓. a DCL of 4 OR
✓. a DCL of 3 with either a positive ("Yes") response to the UHDRS Question 80 (multidimensional manifest diagnosis on motor, cognitive, behavioral, functional) or DSM5 criteria for cognitive disorder (American Psychiatric Association, 2013; MDS Task Force criteria).

Exclusion criteria

✕. Evidence of suicide risk, defined as:
✕. Suicide attempt within 1 year prior to Screening (Visit 1/1A)
✕. Suicidal ideation as defined by a positive response to question 5 on Columbia-Suicide Severity Rating Scale (C-SSRS) Suicidal Ideation Section within 60 days prior to Screening (Visit 1/1A)
✕. Significant risk of suicide as judged by the Investigator

What they're measuring

Evaluate the safety and tolerability of bilateral striatal delivery of AMT-130 as a total HTT gene lowering therapy in adult subjects with early manifest HD assessed by Adverse Events

Timeframe: 6 months

Evaluate the safety and tolerability of bilateral striatal delivery of AMT-130 as a total HTT gene lowering therapy in adult subjects with early manifest HD assessed by Vital Signs - Blood Pressure

Timeframe: 6 months

Evaluate the safety and tolerability of bilateral striatal delivery of AMT-130 as a total HTT gene lowering therapy in adult subjects with early manifest HD assessed by Vital Signs - Respiratory Rate

Timeframe: 6 months

Evaluate the safety and tolerability of bilateral striatal delivery of AMT-130 as a total HTT gene lowering therapy in adult subjects with early manifest HD assessed by Vital Signs - Heart Rate

Timeframe: 6 months

Evaluate the safety and tolerability of bilateral striatal delivery of AMT-130 as a total HTT gene lowering therapy in adult subjects with early manifest HD assessed by Electrocardiograms

Timeframe: 6 months

Evaluate the safety and tolerability of bilateral striatal delivery of AMT-130 as a total HTT gene lowering therapy in adult subjects with early manifest HD assessed by changes documented in the neurological examinations

Timeframe: 6 months

Trial details

NCT IDNCT05243017

SponsorUniQure Biopharma B.V.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2029-03

View on ClinicalTrials.gov More Huntington Disease trials

Plain-language summary

Who can participate

Age range25 Years – 65 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Able and willing to provide written informed consent prior to the study and study-related procedure.
✓. Male and female participants 25-65 years of age.
✓. Cohorts 1 \& 2:
✓. a DCL of 4 OR
✓. a DCL of 3 with either a positive ("Yes") response to the UHDRS Question 80 (multidimensional manifest diagnosis on motor, cognitive, behavioral, functional) or DSM5 criteria for cognitive disorder (American Psychiatric Association, 2013; MDS Task Force criteria).
✓. Cohort 3:
✓. a DCL of 4 OR
✓. a DCL of 3 with either a positive ("Yes") response to the UHDRS Question 80 (multidimensional manifest diagnosis on motor, cognitive, behavioral, functional) or DSM5 criteria for cognitive disorder (American Psychiatric Association, 2013; MDS Task Force criteria).

Exclusion criteria

✕. Evidence of suicide risk, defined as:
✕. Suicide attempt within 1 year prior to Screening (Visit 1/1A)
✕. Suicidal ideation as defined by a positive response to question 5 on Columbia-Suicide Severity Rating Scale (C-SSRS) Suicidal Ideation Section within 60 days prior to Screening (Visit 1/1A)
✕. Significant risk of suicide as judged by the Investigator

What they're measuring

Evaluate the safety and tolerability of bilateral striatal delivery of AMT-130 as a total HTT gene lowering therapy in adult subjects with early manifest HD assessed by Adverse Events

Timeframe: 6 months

Evaluate the safety and tolerability of bilateral striatal delivery of AMT-130 as a total HTT gene lowering therapy in adult subjects with early manifest HD assessed by Vital Signs - Blood Pressure

Timeframe: 6 months

Evaluate the safety and tolerability of bilateral striatal delivery of AMT-130 as a total HTT gene lowering therapy in adult subjects with early manifest HD assessed by Vital Signs - Respiratory Rate

Timeframe: 6 months

Evaluate the safety and tolerability of bilateral striatal delivery of AMT-130 as a total HTT gene lowering therapy in adult subjects with early manifest HD assessed by Vital Signs - Heart Rate

Timeframe: 6 months

Evaluate the safety and tolerability of bilateral striatal delivery of AMT-130 as a total HTT gene lowering therapy in adult subjects with early manifest HD assessed by Electrocardiograms

Timeframe: 6 months