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intra-striatal rAAV5-miHTT

Phase 1

Huntington Disease | Gene therapy | Rare Disease |uniQure N.V.|Last Updated: Mar 10, 2025

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment14
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05243017Safety and Efficacy of AMT-130 in European Adults With Early Manifest Huntington's DiseasePHASE1 ACTIVE NOT_RECRUITING 14 — — Oct 7, 2021Oct 7, 2029Mar 10, 20254 Poland, United Kingdom
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Study Endpoints
Primary Endpoints
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
6 months

Evaluation will be assessed by; \- Type and incidence of Adverse Events (AEs)

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
6 months

Evaluation will be assessed by; \- Changes from baseline in blood pressure (mmHg)

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
6 months

Evaluation will be assessed by; \- Changes from baseline in respiratory rate (BPM)

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
6 months

Evaluation will be assessed by; \- Changes from baseline in heart rate (BPM)

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
6 months

Evaluation will be assessed by; \- Changes from baseline in electrocardiograms (ECGs) for any clinically significant abnormalities or clinically significant worsening. (normal or abnormal)

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
6 months

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)

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 physical examinations
6 months

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)

Evaluate the safety and tolerability by number of participants with clinically significant changes in laboratory tests - Clinical Chemistry
6 months

Evaluation will be assessed by; \- Changes from baseline in Clinical Chemistry laboratory tests with clinical significance.

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 number of participants with clinically significant laboratory tests - hematology
6 months

Evaluation will be assessed by; \- Changes from baseline in hematology laboratory tests with clinical significance.

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 number of participants with clinical significant laboratory tests - urinalysis
6 months

Evaluation will be assessed by; \- Change from baseline in routine urinalysis test with clinical significance.

Evaluate the safety and tolerability o by number of participants with clinical significant changes in cerebrospinal fluid (CSF) analysis
6 months

Evaluation will be assessed by; \- Change from baseline in CSF analysis with clinical significance.

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 vector shedding
6 months

Evaluation will be assessed by; \- Change over time in AAV5 vector shedding

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 laboratory testing serum and CSF for biomarkers
6 months

Evaluation will be assessed by; \- Change over time in microglial activation (YKL-40) (pg/mL)

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 cognitive assessment
6 months

Evaluation will be assessed by; \- Change from baseline to Day 14 and Month 1 in the Montreal Cognitive Assessment (MoCA)

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 number of participants with changes in MRI
6 months

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)

Secondary Endpoints
Duration of persistence of AMT-130 in the brain
Collected for duration of study through month 60
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Cohort 1EXPERIMENTALLow dose AMT-130 (6 × 10\^12 gc/subject)
Cohort 2EXPERIMENTALHigh dose AMT-130 (6 × 10\^13 gc/subject)
Cohort 3EXPERIMENTALLow dose AMT-130 (6 × 10\^12 gc/subject) High dose AMT-130 (6 × 10\^13 gc/subject)
Interventions
NameTypeDescription
intra-striatal rAAV5-miHTTGENETICOne time MRI-guided stereotaxic infusion of rAAV5-miHTT into the brain
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Eligibility Criteria
Age Range25 Years — 65 Years
SexALL
Healthy VolunteersNo
Study Sites4

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 (...

Countries:PolandUnited Kingdom
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Competitive Landscape -Huntington's Disease 9 trials
CompanyTickerTrialsLead PhaseDrugs
Novartis AG Sponsored ADRNVS2PHASE3Votoplam
Neurocrine Biosciences, Inc.NBIX2PHASE3Valbenazine
Alnylam Pharmaceuticals, IncALNY1PHASE1ALN-HTT02
uniQure N.V.QURE2PHASE1intra-striatal rAAV5-miHTT
Sarepta Therapeutics, Inc.SRPT1PHASE1SRP-1005
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT05243017primaryCompletionDate: changed
LOWMay 24, 2026NCT05243017studyFirstPostDate: changed