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Also known as intra-striatal rAAV5-miHTT
AMT-130 · 2 trials · 2 indications
Evaluation will be assessed by; \- Type and incidence of Adverse Events (AEs)
Evaluation will be assessed by; \- Changes from baseline in blood pressure (mmHg)
Evaluation will be assessed by; \- Changes from baseline in respiratory rate (BPM)
Evaluation will be assessed by; \- Changes from baseline in heart rate (BPM)
Evaluation will be assessed by; \- Changes from baseline in electrocardiograms (ECGs) for any clinically significant abnormalities or clinically significant worsening. (normal or abnormal)
Evaluation will be assessed by; \- Changes from baseline in neurological examinations including mental status, cranial nerves, sensory, motor, fine motor, reflexes, and gait (normal or abnormal)
Evaluation will be assessed by; \- Changes from baseline in physical examinations assessed by physical appearance, HEENT, Neck, Chest and Lungs, Cardiovascular, Abdomen, Musculoskeletal, and Genitourinary (normal or abnormal)
Evaluation will be assessed by; \- Changes from baseline in Clinical Chemistry laboratory tests with clinical significance.
Evaluation will be assessed by; \- Changes from baseline in hematology laboratory tests with clinical significance.
Evaluation will be assessed by; \- Change from baseline in routine urinalysis test with clinical significance.
Evaluation will be assessed by; \- Change from baseline in CSF analysis with clinical significance.
Evaluation will be assessed by; \- Change over time in AAV5 vector shedding
Evaluation will be assessed by; \- Change over time in microglial activation (YKL-40) (pg/mL)
Evaluation will be assessed by; \- Change from baseline to Day 14 and Month 1 in the Montreal Cognitive Assessment (MoCA)
Evaluation will be assessed by; \- Change from baseline will be measured by edema, inflammation, volume loss, and structural changes as measured by the following MRI pulse sequences, T1, T2 and diffusion MRI (dMRI)
Safety will be assessed by adverse events (AEs) related to clinical safety laboratory tests, vital signs, electrocardiograms (ECGs), neurological and physical examinations, rAAV5 vector shedding, immunogenicity response (Cohorts 1, 2 \& 3), suicidality risk \[Columbia-Suicide Severity Rating Scale \[C-SSRS)\], changes in global cognitive functioning \[Montreal Cognitive Assessment Scale (MoCA)\] and MRI measures of edema, inflammation, volume loss and structural changes.
| Arm | Type | Description |
|---|---|---|
| Cohort 1 | EXPERIMENTAL | Low dose AMT-130 (6 × 10\^12 gc/subject) |
| Cohort 2 | EXPERIMENTAL | High dose AMT-130 (6 × 10\^13 gc/subject) |
| Cohort 3 | EXPERIMENTAL | Low dose AMT-130 (6 × 10\^12 gc/subject) High dose AMT-130 (6 × 10\^13 gc/subject) |
| Cohorts 1, 2 | SHAM_COMPARATOR | Imitation (sham) surgery |
| Cohort 4 | EXPERIMENTAL | High dose rAAV5-miHTT (6x10\^13 gc/subject). |
| Name | Type | Description |
|---|---|---|
| intra-striatal rAAV5-miHTT | GENETIC | One time MRI-guided stereotaxic infusion of rAAV5-miHTT into the brain |
| Imitation (sham) surgery | OTHER | Simulated surgical procedure with skin incisions only; no intrastriatal injections and no burr holes through the skull |
Inclusion Criteria: 1. Able and willing to provide written informed consent prior to the study and study-related procedure. 2. Male and female participants 25-65 years of age. 3. Cohorts 1 \& 2: 1. a DCL of 4 OR 2. a DCL of 3 with either a positive ("Yes") response to the UHDRS Question 80 (...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Novartis AG Sponsored ADR | NVS | 2 | PHASE3 | Votoplam |
| Neurocrine Biosciences, Inc. | NBIX | 1 | PHASE3 | Valbenazine |
| Hoffmann-La Roche | RHHBY | 3 | PHASE2 | Tominersen, RG6496, SPK-10001 |
| Alnylam Pharmaceuticals, Inc | ALNY | 1 | PHASE1 | ALN-HTT02 |
| uniQure N.V. | QURE | 2 | PHASE1 | AMT-130 |
| Sarepta Therapeutics, Inc. | SRPT | 1 | PHASE1 | SRP-1005 |
AMT-130 is an investigational gene therapy being developed for the treatment of Huntington disease, a hereditary neurodegenerative disorder. It is being studied in adults with early manifest Huntington disease. AMT-130 has not been approved for any indication and remains in clinical development.
AMT-130 is being developed by uniQure N.V., which trades on the Nasdaq under the ticker QURE. The company is running the clinical program for the gene therapy in Huntington disease across sites in the United States and Europe.
AMT-130 is in Phase 1 clinical development. Two Phase 1 trials are currently active and not recruiting, with no completed trials. The program has enrolled a total of 57 participants across both studies. AMT-130 is investigational and has not received FDA approval.
AMT-130 is being evaluated in two Phase 1 trials. NCT04120493 is a safety and proof-of-concept study in adults with early manifest Huntington disease in the United States, enrolling 43 participants. NCT05243017 is a safety and efficacy study in European adults with early manifest Huntington disease in Poland and the United Kingdom, enrolling 14 participants.
Yes, intra-striatal rAAV5-miHTT is another name for AMT-130. It describes the same uniQure gene therapy candidate for Huntington disease, which is delivered directly into the striatum. Both names refer to the investigational product being studied in the two Phase 1 trials.
AMT-130 has received four FDA designations: Regenerative Medicine Advanced Therapy (RMAT), Breakthrough Therapy, Fast Track, and Orphan Drug. These designations relate to its development for Huntington disease. The therapy remains investigational and is still being evaluated in Phase 1 clinical trials.