Panhematin for Prevention of Acute Attacks of Porphyria
Summary
The purpose of this study is to determine if Panhematin is safe and effective for prevention of acute attacks of porphyria. The study aims to provide high quality evidence for the use on hemin for prevention of acute attacks of porphyria. High quality studies have not been done previously for treating or preventing acute attacks with hemin. The lack of strong evidence for efficacy of hemin for treatment and prevention of attacks limits its availability for patients with acute porphyrias. Funding source: FDA Office of Orphan Product Development (FDA OOPD) FD-R-03720
Timeline
- Start
- 2015-10-30
- Primary completion
- 2023-12-27
- Completion
- 2023-12-27
Outcome
Met primary endpoint
Stopped (Enrollment): “Low accrual”
registry analysis (superiority test); Hemin for Injection vs Placebo; p = 0.029; 2-sided Fisher's Exact Test NCT02922413 ↗
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Hemin | Unknown | 4 mg/kg | Intravenous |