Phase 2 Study of EPN-701 for the Treatment of Calciphylaxis
Sagar U. Nigwekar, MD, MMSc
Summary
Calciphylaxis is a rare but serious condition that leads to painful skin lesions. At present, there is no approved treatment for calciphylaxis. This research study is planned to examine the potential benefits of a new treatment (EPN-701) in patients with calciphylaxis. The present early clinical study will also evaluate the pharmacology of EPN-701 and whether it can be safely used in patients with calciphylaxis. Findings will inform the planning of a future definitive study to establish EPN-701 as an approved treatment for calciphylaxis.
Description
This is a phase 2 double-blind RCT that will randomly assign 60 adult dialysis-dependent patients with calciphylaxis to either EPN-701 (10 mg/day administered orally) or a matched placebo in a 1:1 ratio for 24 weeks. Standardized background clinical care will be applied to all participants. Primary objective will be to determine the clinical efficacy and safety of EPN-701 in dialysis-dependent patients with calciphylaxis. The clinical efficacy will be assessed by pre-post (Baseline-Week 24) change in pain intensity and calciphylaxis wound surface area. Secondary objective will be to examine…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Participant must provide written or electronic consent after the nature of the study has been explained, and prior to any research-related procedures, per the International Conference on Harmonization (ICH) Good Clinical Practice (GCP) 2. Clinical diagnosis of calciphylaxis confirmed by the Investigator with presence of at least one ulcerated skin lesion and moderate or severe pain (worst pain intensity in the preceding 24 hours of at least 5 on a 0-10 scale). Consistent with the clinical practice and expert recommendations, histological confirmation of calciphylaxis di…
Interventions
- DrugEPN-701 (Oral)
Randomized subjects will receive EPN-701 or placebo
- DrugPlacebo (Normal Saline)
Randomized subjects will receive EPN-701 or placebo.
Location
- Renal AssociatesBoston, Massachusetts