Safety & Proof-of-Concept (POC) Study With AMT-130 in Adults With Early

Status Recruiting

Clinical Trial Summary

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 I/II, randomized, multicenter, multiple dose, double-blind, imitation surgery, first-in-human (FIH) study. Cohort 3 participants will receive either high or low dose (1:1 randomization).

Clinical Trial Description

AMT-130 is an investigational, single administration gene therapy intended to modify the disease course for HD. Preclinical studies have shown that AMT-130 lowers huntingtin protein and is associated with decreased progression 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 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 blinded post treatment follow-up period (Cohorts 1 & 2 only), once the crossover has been activated after review of data by the DSMB, subjects randomized to the imitation (sham) procedure who continue to meet inclusion/exclusion criteria 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 Month 12 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 together with peri- and post-operative glucocorticoids. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT04120493
Study type	Interventional
Source	UniQure Biopharma B.V.
Contact	Diane Lopez, MS
Phone	781-777-3697
Email	amt130_clinical_trials@uniqure.com
Status	Recruiting
Phase	Phase 1/Phase 2
Start date	September 6, 2019
Completion date	June 2029

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT02956148` - Follow-up Measurement of Brain PDE10A Enzyme Levels in Huntington´s Disease Gene Expansion Carriers	Early Phase 1
Terminated	`NCT02494778` - A Study Evaluating if Pridopidine is Safe, Efficacious, and Tolerable in Patients With Huntington's Disease	Phase 2
Completed	`NCT02197130` - Randomized, Placebo Controlled Study Of The Efficacy And Safety Of PF-02545920 In Subjects With Huntington's Disease	Phase 2
Completed	`NCT02208934` - Study To Assess the Safety and Tolerability of Single Ascending Oral Doses of PBF-999 in Healthy Young Male Volunteers	Phase 1
Completed	`NCT02216474` - Brain Stimulation in Movement Disorders	N/A
Completed	`NCT01806896` - Study Evaluating The Safety, Tolerability And Brain Function Of 2 Doses Of PF-0254920 In Subjects With Early Huntington's Disease	Phase 2
Completed	`NCT01502046` - Neuroprotection by Cannabinoids in Huntington's Disease	Phase 2
Terminated	`NCT00712426` - Creatine Safety, Tolerability, & Efficacy in Huntington's Disease (CREST-E)	Phase 3
Completed	`NCT00670709` - Examination of Quantitative Electroencephalographic (QEEG) Biomarkers in Huntington's Disease
Completed	`NCT00029874` - Minocycline in Patients With Huntington's Disease	Phase 1/Phase 2
Terminated	`NCT02231580` - Study Exploring Safety, Pharmacokinetic and Pharmacodynamic of BN82451 in Male Huntington's Disease Patients	Phase 2
Completed	`NCT02215616` - A Clinical Study in Participants With Huntington's Disease (HD) to Assess Efficacy and Safety of Three Oral Doses of Laquinimod	Phase 2
Not yet recruiting	`NCT02551705` - Functional Imaging of Social Cognition in Premanifest Huntington's Disease	N/A
Active, not recruiting	`NCT02101957` - Multicentric Trial of the Treatment of Huntington's Disease by Cysteamine (RP103)	Phase 2/Phase 3
Completed	`NCT00990613` - A Study Evaluating The Absorption Of Dimebon Into The Body From A Dimebon Solution Applied To The Skin	Phase 1
Completed	`NCT00975481` - A Study To Evaluate The Abuse Potential Of Single Oral Doses Of Dimebon (Latrepirdine) In Healthy Recreational Polydrug Users	Phase 1
Completed	`NCT01521832` - Escalating Dose Study in Healthy Volunteers With SEN0014196	Phase 1
Completed	`NCT00387270` - Safety Study of the Novel Drug Dimebon to Treat Patients With Huntington's Disease	Phase 1/Phase 2
Completed	`NCT00095355` - Effects of Lithium and Divalproex`on Brain-Derived Neurotrophic Factor in Huntington's Disease	Phase 2
Completed	`NCT00026988` - Creatine Therapy for Huntington's Disease	Phase 1/Phase 2

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

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

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms