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Study of EPN-701 for the Treatment of Calciphylaxis

ConditionCalciphylaxis
Trial statusNot yet recruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorSagar U. Nigwekar, MD, MMSc

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial design

Design model

Parallel

Treatments tested in this trial

  • EPN-701 (Oral)

    Drug

    Randomized subjects will receive EPN-701 or placebo

  • Placebo (Normal Saline)

    Drug

    Randomized subjects will receive EPN-701 or placebo.

Treatment groups

60 Participants
are divided into 2 treatment groups
Group A: EPN-701Active comparator 1 intervention
Group B: PlaceboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

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

Time frame
Baseline-24 weeks
2

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

Time frame
Baseline-24 weeks

Secondary outcomes

Other outcomes

1

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

Pre-post (Baseline-Week 24) change in plasma uncarboxylated matrix Gla protein (uc-MGP) among the trial arms

Time frame
Baseline-24 weeks
2

Change in calcification burden

Pre-post (Baseline-Week 24) change in radiotracer uptake on 18-sodium fluoride positron emission tomography/computerized tomography (18F-NaF PET/CT)

Time frame
Baseline-24 weeks
3

Changes in additional biomarkers and plasma proteomic

Pre-post (Baseline-Week 24) changes in biomarkers such as plasma Protein induced by vitamin K absence or antagonist-II (PIVKA- II), fetuin A, and novel markers identified via plasma proteomic analyses

Time frame
Baseline-24 weeks
4

Change in pain interference

Pre-post (Baseline-Week 24) change in the pain interference score recorded on Brief Pain Inventory/Short Form (BPI/SF). Score of 0 indicates no pain interference. Score of 10 indicates most severe pain interference.

Time frame
Baseline-24 weeks

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Sagar U. Nigwekar, MD, MMSc

Lead sponsor

Massachusetts General Hospital

Sponsor institution

Epizon Pharma, Inc.

Collaborator

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: