Clinical trials: Severe Osteopetrosis
Infantile Malignant Osteopetrosis
A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis
The primary objective of this Phase 1 study is to evaluate the therapeutic safety and feasibility of the investigational product (IP), RP-L401.
- Trial name
- NCT04525352
- Testing
- RP-L401
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Osteopetrosis
Post Marketing Surveillance Study of Actimmune in Patients With Severe, Malignant Osteopetrosis
The purpose of this study is to establish a registry of all children with severe, malignant osteopetrosis who are treated with Actimmune (IFN-g 1b or Interferon gamma-1b) to monitor the effects of IFN-g 1b on preventing…
- Trial name
- NCT00043329
- Testing
- Actimmune Registry
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- InterMune