Clinical trials: Rhizomelic Chondrodysplasia Punctata
Rhizomelic Chondrodysplasia Punctata
A First-in-Human Phase 1 Study of Plasmalogen Precursor PPI-1011 in Healthy Adult Volunteers to Assess Safety, Tolerability, and Pharmacokinetics
PPI-1011 is being developed as a docosahexaenoic acid (DHA) containing plasmalogen precursor with good long-term stability, specifically for the treatment of rhizomelic chondrodysplasia punctata (RCDP), which is an ultra rare…
- Trial name
- NCT05969977
- Testing
- PPI-1011
- Where
- Canada
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- MED-LIFE DISCOVERIES LP
Rhizomelic Chondrodysplasia Punctata
RCDP Natural History Study
A prospective, longitudinal observational trial in patients with RCDP. Study participants will be evaluated at baseline and approximately every 6 months by the study team.
- Trial name
- NCT04031287
- Where
- United States
- Volunteers
- 75 people
- Sponsor
- MED-LIFE DISCOVERIES LP