Clinical Trials

           The Penn Neurogenetics Therapy Center (PNTC) partners with both Penn investigators and biomedical companies to deliver early phase clinical trials of novel genetic-based therapies to patients with neurological disorders. The center leverages Penn resources and streamlines internal startup operations to efficiently establish and conduct first-in-human clinical trials with the highest standards of care and research. Our team of trialists, operations specialists, and supporting clinical research staff provides focused, coordinated expert care and management. To conduct complex, multi-disciplinary studies, we rely on well-established collaborations with other centers, departments and clinical entities at Penn. Since PNTC’s launch in March 2020, we have been conducting or are now in the process of activating clinical trials of gene and molecular therapies for several neurological indications, including ALS, Charcot-Marie-Tooth type 1A (CMT1A), facioscapulohumeral muscular dystrophy (FSHD), epilepsy, FTD, HD, myasthenia gravis (MG), myotonic dystrophy type 1 (DM1), PD and spinocerebellar ataxia (SCA). We are completely committed to bringing precision gene-based medicine to our patients.

Our Trials

  • A Phase 1/2a Open-Label Ascending Dose Study to Evaluate the Safety and Effects of LY3884961 in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)
    • Study Name: Propel
    • Sponsor: Prevail Therapeutics, a wholly-owned subsidiary of Eli Lilly and Company
    • Site PI: Andres Deik Acosta Madiedo, MD
    • Link to ClinicalTrials.gov

 

  • A Phase 1/2 Ascending Dose Study to Evaluate the Safety and Effects on Progranulin Levels of PR006A in Patients with Frontotemporal Dementia with Progranulin Mutations (FTD-GRN) (PROCLAIM)
    • Study Name: Proclaim
    • Sponsor: Prevail Therapeutics, a wholly-owned subsidiary of Eli Lilly and Company
    • Site PI: David Irwin, MD
    • Link to ClinicalTrials.gov

 

  • A Phase 1b Open-Label, Multicenter, Dose-Escalation Study to Assess the Safety, Tolerability, and Pharmacodynamic Effects of a Single Dose of PBFT02 Delivered into the Cisterna Magna (ICM) of Adult Subjects with Frontotemporal Dementia (FTD) and Mutations in the Progranulin Gene (GRN)

  • A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Adult Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)

 

  • A Phase 1/2 Multiple-Ascending-Dose Study With a Long-Term Open-Label Extension to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Effect on Disease Progression of BIIB105 Administered Intrathecally to Adults With Amyotrophic Lateral Sclerosis With or Without Poly-CAG Expansion in the Ataxin-2 Gene

 

  • A Phase 1 Single- and Multiple-Ascending-Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB094 Administered Intrathecally to Adults With Parkinson's Disease

 

  • A Phase 3b Study to Evaluate Higher Dose Nusinersen (BIIB058) inPatients with Spinal Muscular Atrophy Previously Treated with Risdiplam

  • Autologous T-cells Expressing a Chimeric Antigen Receptor Directed to B-Cell Maturation Antigen (BCMA) in patients with Generalized Myasthenia Gravis (GMG)

  • A Randomized, Double-Blind, Placebo-Controlled Dose Range Finding Study with Open-Label Extension to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of LMI070/branaplam when Administered as Weekly Oral Doses in Participants with Early Manifest Huntington’s Disease

 

  • A Phase 1, Blinded, Randomized, Placebo-controlled Study to Investigate the Safety, Tolerability, and Pharmacokinetics of Multiple Ascending Doses of BIIB132 Administered Intrathecally to Adults With Spinocerebellar Ataxia 3

For More Information

What are clinical trials?

What are gene therapies?

What are the uses of gene therapies?