A Trial of PF-06252616 in Ambulatory Participants With LGMD2I

Phase 1/2 Completed 19 enrolled Kathryn Wagner Pfizer · collab

Non-randomizedParallel-groupOpen-labelTreatment

Summary

The investigational product PF 06252616, a humanized anti myostatin monoclonal antibody that neutralizes myostatin (GDF8) is in development for the treatment of Limb Girdle Muscular Dystrophy 2I (LGMD2I) to preserve and/or improve muscle function. This study will provide the clinical assessment of the safety, tolerability, Pharmacokinetics and Pharmacodynamics of PF 06252616 following repeat IV doses in ambulatory adults with LGMD2I.

Timeline

Start: 2016-07
Primary completion: 2019-01
Completion: 2019-01

Publications

Leung DG, Bocchieri AE, Ahlawat S, Jacobs MA, Parekh VS, Braverman V, Summerton K, Mansour J, Bibat G, Morris C, Marraffino S, Wagner KR. Longitudinal functional and imaging outcome measures in FKRP limb-girdle muscular dystrophy. BMC Neurol. 2020 May 19;20(1):196. doi: 10.1186/s12883-020-01774-5.

Drugs

Evaluation	Drug	Modality	Dose	Route
Comparator	domagrozumab	Monoclonal antibody	5 mg/kg	Intravenous
Comparator	domagrozumab	Monoclonal antibody	20 mg/kg	Intravenous
Comparator	domagrozumab	Monoclonal antibody	40 mg/kg	Intravenous

A Trial of PF-06252616 in Ambulatory Participants With LGMD2I

Summary

Timeline

Publications

Drugs

Indications