Study of EPN-701 for the Treatment of Calciphylaxis

NCT07583745 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: 2026P000834
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Calciphylaxis

Keywords

calciphylaxis; Massachusetts General Hospital; MGH; MGB; Mass General Brigham

Outcome Measures

Primary Outcomes (2)

• Change in pain intensity (Alternate Primary Endpoint)

  Pre-post (Baseline-Week 24) change in the worst calciphylaxis associated pain intensity as measured on the 0 to 10 scale among the trial arms. Score of 0 indicates no pain. Score of 10 indicates most severe pain

  Baseline-24 weeks

• Change in total calciphylaxis lesion(s) surface area (Alternate Primary Endpoint)

  Pre-post (Baseline-Week 24) change in total surface area of ulcerated skin lesions of calciphylaxis among the trial arms

  Baseline-24 weeks

Other Outcomes (7)

• Plasma level of uncarboxylated matrix Gla protein (uc-MGP)

  Baseline-24 weeks

• Change in calcification burden

  Baseline-24 weeks

• Changes in additional biomarkers and plasma proteomic

  Baseline-24 weeks

Study Arms (2)

EPN-701

ACTIVE COMPARATOR

EPN-701 will be administered daily by mouth

Drug: EPN-701 (Oral)

Placebo

PLACEBO COMPARATOR

Placebo will be taken daily by mouth

Drug: Placebo (Normal Saline)

Interventions

EPN-701 (Oral) DRUG

Randomized subjects will receive EPN-701 or placebo

EPN-701

Placebo (Normal Saline) DRUG

Randomized subjects will receive EPN-701 or placebo.

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)
• 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 diagnosis by a skin biopsy is not a requirement for participation.
• ESKD requiring thrice weekly hemodialysis
• Male or female aged ≥18 years
• Women of childbearing potential (WOCBP) as defined in Clinical Trials Facilitation and Coordination Group (CTFG, 2020) must have a negative serum pregnancy test (defined as serum hCG ≤25 mIU/mL \[Haninger-Vacariu, 2020\]) at Screening
• Women of childbearing potential (WOCBP) must be using or agree to use one highly effective form of contraception and a barrier method from Screening through 30 days after the last dose of study treatment. See Appendix C for further information about highly effective forms of contraception.
• Males who are sexually active must agree to use condoms from the period following first dose of study treatment through four weeks after the last dose of study treatment. Males with a WOCBP partner must agree to use one highly effective form of contraception in addition to condoms from Screening through 30 days after the last dose of study treatment.
• Males must agree to not donate sperm from the period following the first dose of study treatment through 4 weeks after last dose of study treatment. WOCBP who are participants or partners of fertile male participants must agree not to donate ova from the period following the first dose of study treatment through 4 weeks after last dose of study treatment.

You may not qualify if:

• In the opinion of the Investigator, presence of any clinically significant disease outside of calciphylaxis that may impact study participation and/or confound interpretation of the study results
• Hospitalized at the time of Screening. Patients discharged from a hospital admission ≥24 hours prior to Screening may be eligible for participation.
• Septic shock
• Hospice or comfort care including withdrawal from dialysis
• Malignancy within the last year, except non-melanoma skin cancers, cervical carcinoma in situ, or localized prostate cancer.
• Concurrent participation in another interventional clinical study and/or receipt of any other investigational new drug within four weeks prior to the first dose of study treatment, or use of an investigational device, through completion of participation in the study
• Previous participation in a study of EPN-701
• Participants who are pregnant, trying to become pregnant, or breastfeeding
• Participants who are trying to father a child
• Peritoneal dialysis participants
• Kidney transplant recipients who are receiving immunosuppression at the time of Screening and patients who have an imminent transplant plan. Patients who have received a kidney transplant that has failed, necessitating dialysis, and who are no longer on immunosuppression may participate.
• History of or current diagnosis of cirrhosis
• Current use of prohibited medications, including vitamin K-containing medications and warfarin. Patients who have discontinued warfarin treatment upon the diagnosis of calciphylaxis will be eligible for Screening.

Sponsors & Collaborators

• Sagar U. Nigwekar, MD, MMSc: lead

Study Sites (1)

Renal Associates

Boston, Massachusetts, 02114, United States

Location

MeSH Terms

Conditions

Calciphylaxis

Interventions

Saline Solution

Condition Hierarchy (Ancestors)

Calcinosis; Calcium Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Crystalloid Solutions; Isotonic Solutions; Solutions; Pharmaceutical Preparations

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Assistant Professor

Study Record Dates

• First Submitted: April 29, 2026
• First Posted: May 13, 2026
• Study Start (Estimated): July 1, 2026
• Primary Completion (Estimated): June 30, 2030
• Study Completion (Estimated): June 30, 2030
• Last Updated: May 13, 2026

Record last verified: 2026-04

Locations

US (1)