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Safety and Proof-of-Concept (POC) Study With AMT-130 in Adults With Early Manifest Huntington's Disease

StatusActive, Not Recruiting
PhasePhase 1/2
Started2019-09-06
View on ClinicalTrials.gov ↗

Amendment history

2025-10-17
Recruiting→Active, Not Recruiting Study Status, Contacts/Locations v40
Trial statusRecruiting→Active, Not Recruiting
Study sites14→12
2025-10-15
Study Status, Study Description, Eligibility, Contacts/Locations v39
Eligibility criteriarevised
[...] cipants 25 to 65 years of age of both sexes Cohorts 1, 2, & 34: Early manifest HD as defined by a UHDRS total functional c [...]
Study description+109 characters
[...] 130 and will receive pre- and post-operative dexamethasone. AllCohorts 1 and 2 participants continue follow-up visits through 6 years after receipt of AMT-130. Cohorts 3 and 4 participants continue follow-up visits through 5 years after receipt of AMT-130.
Study sitesdetails revised at 1 of 14 sites
2025-10-08
Study Status, Study Description, Outcome Measures, Contacts/Locations v38
Secondary endpointsdetails revised at 1 of 1 entry
Study descriptionrevised
[...] thasone. All participants continue follow-up visits through 56 years after receipt of AMT-130.
Study sitesdetails revised at 3 of 14 sites
2025-08-29
Study Status, Contacts/Locations v37
Study sitescontacts updated at 1 of 14 sites
2025-08-19
Study Status, Contacts/Locations v36
Study sitesdetails revised at 10 of 14 sites
Show 35 earlier versions
2025-08-11
Active, Not Recruiting→Recruiting Study Identification, Study Status, Oversight, Study Description, Study Design, Arms and Interventions, Outcome Measures, Eligibility, Contacts/Locations v35
Trial statusActive, Not Recruiting→Recruiting
Primary completion date2029-04→2029-06
Completion date2029-06→2029-12
Enrollment target37→43
Study sites12→14
Study arms4→5
Eligibility criteria-439 characters
[...] procedure Participants 25 to 65 years of age of both sexes 3a. CohortCohorts 1, 2, & 23: Early manifest HD as defined by a UHDRS total functional c [...] l manifest HD (UHDRS question 80) or has cognitive symptoms 3b. Cohort 3: Early manifest HD as defined by a UHDRS TFC score [...] e disorder (Movement Disorder Society Task Force criteria). 4. HTT gene expansion testing with the presence of ≥40 CAG repeats 5. Striatal MRI volume requirements per hemisphere: Cohorts 1, 2, & 3: Putamen ≥2.5 cm3cm^3 (per side); Caudate ≥2.0 cm3cm^3 (per side) 6Cohort 4: Putamen <2.5 cm^3 (on either side); Caudate <2.0 cm^3 [...] [...]
Secondary endpointsdetails revised at 1 of 1 entry
Study description-874 characters
[...] ion of Huntington's Disease signs in animal models. Cohort 1 &, 2 consists of a blinded 12-month Core Study Period to evaluate the safety, and potential impact of AMT-130 on disease progression and an unblinded 4-year Long-Term Period with periodic follow-up visits to evaluate the safety of AMT-130 and disease progression in treated individuals. Cohort 2 Sham participants who do not cross over to receive AMT-130 treatment will have the opp [...] 3 participants will receive either high orevaluated low dose AMT-130. Following completion of the Month 36 visit, they will be unblinded to their treatment a [...] [...]
2025-03-05
Recruiting→Active, Not Recruiting Study Identification, Study Status, Contacts/Locations v34
Trial statusRecruiting→Active, Not Recruiting
Study sites14→12
Study titlerevised
Safety and Proof-of-Concept (POC) Study withWith AMT-130 in Adults withWith Early Manifest Huntington's Disease
2024-12-18
Active, Not Recruiting→Recruiting Study Status, Contacts/Locations v33
Trial statusActive, Not Recruiting→Recruiting
Study sites12→14
2024-10-22
Study Status, Study Design v32
Enrollment target36→37
2024-09-30
Recruiting→Active, Not Recruiting Study Identification, Study Status, Oversight, Contacts/Locations, IPDSharing v31
Trial statusRecruiting→Active, Not Recruiting
Study sites14→12
Study titlerevised
Safety and Proof-of-Concept (POC) Study Withwith AMT-130 in Adults Withwith Early Manifest Huntington's Disease
2024-08-01
Study Status, Contacts/Locations v30
Study sitesdetails revised at 1 of 14 sites
2024-07-26
4 amendments v26-v29
4 re-verifications since 2024-06-28, no change to tracked fields
2024-06-25
Study Status, Contacts/Locations v25
Study sitescontacts updated at 1 of 14 sites
2024-04-29
Study Status, Study Description, Outcome Measures, Eligibility, Contacts/Locations v24
Eligibility criteria+271 characters
Inclusion Criteria: Able and willing to provide written informed consent prior to the study and study-related procedure SubjectsParticipants 25 to 65 years of age of both sexes 3a. Cohort 1 & 2: Early [...] dy visit schedule as outlined in the protocol 8. All female subjectsparticipants of childbearing potential (FOCP) must have a negative serum [...] investigational agents, such as antisense oligonucleotides (ASOASOs), cell transplantation or any other experimental brain surg [...] found clinical and laboratory evaluations or could affect a subjectparticipant's safety or their ability to undergo the neurosurgical [...] [...]
Secondary endpointsdetails revised at 1 of 1 entry
Study description+89 characters
[...] ceive AMT-130 treatment. Cohort 3 participants will receive AMT-130 either high or low dose AMT-130. Following completion of the Month 1236 visit, they will be unblinded to their treatment arm. Cohor [...] ty and exploratory efficacy data of low or high dose AMT-130. Cohort together3 withparticipants peri-will also receive pre and post-operative glucocorticoidsimmunosuppressant therapies composed of dexamethasone, sirolimus, and rituximab.
Study sites14 entries, revised
University of Arizona (Surgical Site Only)
2023-10-16
Study Identification, Study Status, Study Description, Study Design, Arms and Interventions, Outcome Measures, Eligibility, Contacts/Locations v23
Enrollment target44→36
Study sites13→14
Study arms5→4
InterventionsImitation (sham) surgery (Other), intra-striatal rAAV5-miHTT (Genetic), intra-striatal rAAV5-miHTT (Genetic)→Imitation (sham) surgery (Other), intra-striatal rAAV5-miHTT (Genetic)
Eligibility criteria+1,097 characters
[...] ated procedure Subjects 25 to 65 years of age of both sexes 3a. Cohort 1 & 2: Early manifest HD as defined by a UHDRS total functional capacity (TFC) score of 9 to 13 and EITHER a diagnostic classificationconfidence level (DCL) of 4 OR a DCL of 3 if the subject either meets [...] l manifest HD (UHDRS question 80) or has cognitive symptoms 3b. Cohort 3: Early manifest HD as defined by a UHDRS TFC score of ≥ 11 and EITHER a DCL of 4 or a DCL of 3 with either a positive "Yes" response to UHDRS Question 80 (multidimensional manifest diagnosis on motor, cognitive, behavioral, functional) or DSM5 criteria [...] [...]
Study description+249 characters
[...] progression of Huntington's Disease signs in animal models. ThisCohort trial1 & 2 consists of a blinded 12-month (Cohort 1 & 2) and 12-month (Cohort 3A & 3B) Core Study Period to evaluate the safety and potential impa [...] an additional 2 years. Following completion of the 12-month (Cohort 1 & 2) or 12-month (Cohort 3A & 3B) blinded post treatment follow-up period (Cohorts 1 & 2 only), once the crossover has been activated after review of data [...] a will be allowed to crossover to receive AMT-130 treatment. Cohort 3 participants will receive AMT-130 either high or low dose. Following completion of the Mo [...]
2023-06-16
Study Status, Contacts/Locations v22
Study sitesdetails revised at 1 of 13 sites
2023-05-30
Study Status, Contacts/Locations v21
Study sitesdetails revised at 1 of 13 sites
2023-03-16
Study Status, Contacts/Locations v20
Study sites12→13
2022-10-17
Study Identification, Study Status, Study Description, Conditions, Study Design, Arms and Interventions, Outcome Measures, Eligibility, Contacts/Locations v19
Primary completion date2022-12→2029-04
Completion date2026-05→2029-06
Enrollment target26→44
Study arms3→5
InterventionsImitation (sham) surgery (Other), intra-striatal rAAV5-miHTT (Genetic)→Imitation (sham) surgery (Other), intra-striatal rAAV5-miHTT (Genetic), intra-striatal rAAV5-miHTT (Genetic)
Eligibility criteria+1,488 characters
Inclusion Criteria: Able and willing to provide written informed consent prior to the study and study-related procedure Subjects 25 to 65 years of age of both sexes Early manifest HD as defined by a UHDRS total functional cap [...] gene expansion testing with the presence of ≥40 CAG repeats. Striatal MRI volume requirements per hemisphere: Putamen ≥2 [...] ions (addressing motor, behavioral, and cognitive symptoms) must be stable for 3 months prior to Screening. Able andwith willingno change in clinical symptoms requiring change in medication prior to provideanticipated writtenadministration informed conse [...] [...]
Study description+202 characters
[...] n and is associated with decreased progression of Huntington's diseaseDisease signs in animal models. This 5-year trial consists of a blinded 12-month (Cohort 1 & 2) and 12-month (Cohort 3A & 3B) Core Study Period to evaluate the safety and potential impa [...] of AMT-130 and disease progression in treated individuals. Cohort 2 Sham participants who do not cross over to receive AMT-130 treatment will have the opportunity to participate in the Optional Extended Follow-Up Period and will be followed for an additional 2 years. Following completion of the 12-month (Cohort 1 & 2) or 12-month (Cohort 3A & [...] [...]
Study sitescontacts updated at 2 of 12 sites
Study title+2 characters
Safety and Proof-of-Concept (POC) Study With AMT-130 in Adults With Early Manifest Huntington's Disease
2021-12-02
Study Status, Contacts/Locations v18
Study sitesdetails revised at 1 of 12 sites
2021-09-30
Study Status, Contacts/Locations v17
Study sites11→12
2021-09-15
Study Status, Contacts/Locations v16
Study sitescontacts updated at 1 of 11 sites
2021-08-18
Study Status, Contacts/Locations v15
Re-verified, no change to tracked fields
2021-08-11
Study Status, Contacts/Locations v14
Study sitescontacts updated at 1 of 11 sites
2021-07-23
Study Status, Contacts/Locations v13
Study sites10→11
2021-07-08
Study Status, Contacts/Locations v12
Study sites9→10
2021-01-15
Study Status, Contacts/Locations v11
Study sitesdetails revised at 1 of 9 sites
2021-01-08
Study Status, Contacts/Locations v10
Study sitescontacts updated at 1 of 9 sites
2020-10-27
Study Status, Contacts/Locations v9
Study sitesdetails revised at 1 of 9 sites
2020-10-05
Study Status, Contacts/Locations v8
Study sites7→9
2020-09-14
Study Status, Study Description, Study Design, Arms and Interventions, Outcome Measures, Eligibility, Contacts/Locations v7
InterventionsImitation surgery (Procedure), rAAV5-miHTT (Genetic)→Imitation (sham) surgery (Other), intra-striatal rAAV5-miHTT (Genetic)
Eligibility criteria+452 characters
Inclusion Criteria: FirstEarly manifest HD as defined by a UHDRS total functional capacity (TFC) score of 9 to 13 and EITHER a diagnostic classification level (DCL) of 4 subjectsOR ina CohortDCL 1:of early3 if the subject either meets the definition of multidimensional manifest HD (StageUHDRS 2question 80). All remainingor subjectshas incognitive Cohort 1 and all subjects in Cohort 2: early manifest HD (Stages 1 and 2).symptoms HTT gene expansion testing with the presence of ≥4440 CAG repeats. Striatal MRI volume requirements per hemispher [...] ; Caudate ≥2.0 cm3 (per side) All HD concomitant medica [...]
Study description+462 characters
[...] udies have shown that AMT-130 lowers huntingtin protein and improvedis associated with decreased progression of Huntington disease signs in animal models. This 5-year trial consists of a blinded 1812-month Core Study Period to evaluate the safety and potential impact of AMT-130 on disease progression and an unblinded 3.54-year Long-Term Period with annualperiodic follow-up visits to evaluate the safety of AMT-130 and disease progression in treated individuals. Following completion of the 12-month blinded post treatment follow-up period, subjects will be individually unblinded. Once the crossov [...]
Study sitesdetails revised at 1 of 7 sites
2020-08-26
Study Status, Contacts/Locations v6
Study sites6→7
2020-06-05
Study Status, Contacts/Locations v5
Study sites4→6
2020-01-09
Study Status, Contacts/Locations v4
Study sites3→4
2019-11-13
Study Status, Contacts/Locations v3
Re-verified, no change to tracked fields
2019-10-18
Study Status, Contacts/Locations v2
Study sitesdetails revised at 1 of 3 sites
2019-10-11
Not Yet Recruiting→Recruiting Study Status, Contacts/Locations v1
Trial statusNot Yet Recruiting→Recruiting
Start dateestimated 2019-10→confirmed 2019-09-06
Study sites3 entries, revised
Johns HopkinsThe University of Texas
2019-10-07
Original filing
This is the first study of AMT-130 in patients with early manifest HD and is designed to establish safety and proof-of-concept (PoC). CT-AMT-130-01 is a Phase 1/2, multicenter, first-in-human (FIH) study. The first three cohorts of the study have completed enrollment, including the randomized, double-blind, sham-controlled cohorts. Cohort 4 is open-label. Cohort 4 participants will receive high dose AMT-130.
Trial Details
NCT Number NCT04120493
Lead Sponsor UniQure Biopharma B.V.
Conditions Huntington's Disease
Enrollment 43 participants
Start Date 2019-09-06
Primary Completion 2029-06 (estimated)
Study Completion 2029-12 (estimated)
Updated on ClinicalTrials.gov 2025-10-21