Clinical trials

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Search and review clinical trials. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Recruiting

A Study of AAV2-GDNF in Adults With Moderate Parkinson's Disease (REGENERATE-PD)

The objective of this randomized, surgically controlled, double-blinded, Phase 2 study is to evaluate the safety and efficacy of AAV2-GDNF delivered to the putamen in subjects with moderate Parkinson's Disease.

Participants needed: 127
Trial details
Phase: Phase 2Age: 45-75Biological sex: AllType: InterventionalSponsor: AskBio IncUpdated: Jun 29, 2026Locations: 46
Eligibility criteria

Male and female adults 45-75 years of age inclusive, at the time of signing of i... [+6]

Known history or current evidence of medical, genetic, or neurological condition... [+8]

Status: Not yet recruiting

Prescriptive Infusion Algorithm (PIA)

This study is being done to test a new way of delivering AB-1005 into the brain. The goal is to make the procedure easier and quicker to perform, while providing similar amounts of drug to the part of the brain that needs treatment. Technical (Stage 0) To test if the new delivery method (prefrontal surgical approach) can consistently deliver AB-1005 to the putamen using MRI monitoring. Technical (Stage 1) To test if the new delivery method (PIA-based infusion) can consistently deliver AB-1005 to the putamen using brain imaging by MRI. Technical (Stage 2) To confirm the new delivery method works without brain imaging by MRI, using standard operating room tools including brain imaging by CT. Safety (Stage 1 and 2) To assess the safety and tolerability of the new delivery method for AB-1005 up to 6 months after surgery

Participants needed: 18
Trial details
Phase: Phase 2Age: 45-75Biological sex: AllType: InterventionalSponsor: AskBio IncUpdated: Jun 26, 2026Locations: 1
Eligibility criteria

Participant must be 45 to 75 years of age inclusive, at the time of signing the... [+13]

Evidence of secondary or atypical parkinsonism (as determined by the neurologist... [+31]

Status: Recruiting

A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late Onset Pompe Disease (PROGRESS-GT LOPD)

This is a single-arm, open-label, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of AB-1009 in adult participants with late-onset Pompe disease (LOPD).

Participants needed: 12
Trial details
Phase: Phase 1, Phase 2Age: 18+Biological sex: AllType: InterventionalSponsor: AskBio IncUpdated: May 28, 2026Locations: 9
Eligibility criteria

Participant must be ≥18 years of age at the time of signing the informed consent... [+6]

Severe cardiomyopathy, defined as left ventricular ejection fraction (LVEF) <40%... [+9]

Status: Recruiting

A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)

The purpose of this study is to evaluate the safety and tolerability of a single intravenous infusion of AB-1003 in adults diagnosed with limb girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). Participants will be treated in sequential, dose-level cohorts. (Part 1)

Participants needed: 10
Trial details
Phase: Phase 1, Phase 2Age: 18-65Biological sex: AllType: InterventionalSponsor: AskBio IncUpdated: Feb 20, 2026Locations: 6
Eligibility criteria

Male and female subjects aged 18 and 65 years with clinical diagnosis of LGMD2I/... [+3]

Significant cardiomyopathy as defined by echocardiogram (left ventricular ejecti... [+12]