In healthy individuals, the ALK2 receptor protein interacts with Bone Morphogenetic Proteins (BMPs). Through this interaction, normal bone is formed. However, in those with FOP, this mutation leads to a malformed overactive protein resulting in the production of extra bone. The disease progresses throughout the lifetime of the patient sequentially affecting muscle, ligaments, tendons and other connective tissues throughout the body.
Mutant ALK2 hyper-responsiveness to BMPs and neo-responsiveness to Activin A provides a rationale for using blocking antibodies to prevent progression of the disease and subsequent ankylosis. DS-6016a is an anti-ALK2 monoclonal antibody developed by Daiichi Sankyo and Saitama Medical University for the treatment of FOP.
About the Clinical Trial
The aim of the DS-6016a
is to assess the safety, tolerability and pharmacokinetics of DS-6016a after subcutaneous injection in healthy Japanese participants. The study design will be a single ascending dose study. DS-6016a is highly specific to ALK2 receptor-mediated FOP and has never been tested for any other indication. Daiichi/Sankyo will recruit 48 Japanese healthy male subjects between the ages of 20 and 45 years old with a body mass index between 18.5 and 25.0 kg/M2 at screening. Women or subjects with a history of hypersensitivity or dependence on alcohol or drugs are excluded from participating in the study.
Eligibility Criteria*
- AGE: ≤ 20, ≤ 45
- DISEASE ACTIVITY: None
Study Design*
- STUDY TYPE: Interventional
- RANDOMIZED STUDY: Yes
- PLACEBO CONTROLLED: Yes
- LENGTH OF PARTICIPATION: 9 months
- NUMBER OF STUDY VISITS: 18
Status
Phase 1, Recruiting
Therapy Approach
Anti-ALK-2 monoclonal antibody
Study Sponsor
Daiichi Sankyo Co., Ltd.
The IFOPA does not endorse nor recommend specific clinical trials. Please speak with your doctor if you are interested in participating in a clinical trial.
