Ignite Creation Date:
2024-05-06 @ 5:14 PM
Last Modification Date:
2024-10-26 @ 2:25 PM
Study NCT ID:
NCT05243017
Status:
RECRUITING
Last Update Posted:
2024-06-03
First Post:
2021-11-01
Brief Title:
Safety and Efficacy of AMT-130 in European Adults with Early Manifest Huntingtons Disease
Sponsor:
UniQure Biopharma BV
Organization:
UniQure Biopharma BV
Study Overview
Official Title:
A Phase IbII Randomized Double-Blind Study to Explore Safety Tolerability and Efficacy Signals of Multiple Doses of Striatally-Administered RAAV5-miHTT Total Huntingtin Gene HTT Lowering Therapy AMT 130 in Early Manifest Huntingtons Disease
Status:
RECRUITING
Status Verified Date:
2024-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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 III 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 11 randomization Participants enrolled in Cohort 3 will also receive an immunosuppression regimen consisting of dexamethasone sirolimus and rituximab
Cohort 3 participants will receive either high or low dose 11 randomization Participants enrolled in Cohort 3 will also receive an immunosuppression regimen consisting of dexamethasone sirolimus and rituximab
Detailed Description:
The aim of the European study is to build upon the safety demonstrated in the first human dose FHD randomized double blind sham-controlled sequential dose escalation study CT-AMT-130-01 clinicaltrialsgov NCT04120493 being conducted in the US and expand the number of patients exposed to the two doses to provide sufficient sample size for comparisons of safety and efficacy CT-AMT-130-02 is a Phase IbII open-label Cohorts 1 2 randomized Cohort 3 only sequential multiple dose study that will be conducted in approximately 5 to 8 European HD centers 2 of these centers will serve as surgical sites Both studies will share a common set of clinical safety imaging and biomarker evaluations over 5 years of follow-up The DSMB will evaluate safety and other parameters to enable the staggered treatment of patients within each of the dosing cohorts
Cohort 3 participants will receive either high or low dose AMT-130 Following completion of the Month 36 visit they will be unblinded to their treatment arm Cohort 3 will further evaluate the safety and exploratory efficacy data of low or high dose AMT-130 Cohort 3 participants will also receive pre and post-operative immunosuppressant therapies composed of dexamethasone sirolimus and rituximab
Cohort 3 participants will receive either high or low dose AMT-130 Following completion of the Month 36 visit they will be unblinded to their treatment arm Cohort 3 will further evaluate the safety and exploratory efficacy data of low or high dose AMT-130 Cohort 3 participants will also receive pre and post-operative immunosuppressant therapies composed of dexamethasone sirolimus and rituximab
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None