CalciphylaxisPhase 2NCT07583745
Study of EPN-701 for the Treatment of Calciphylaxis
A Phase 2 study testing EPN-701 (Oral) in people with Calciphylaxis, run by Sagar U. Nigwekar, MD, MMSc at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Calciphylaxis
- What is being tested?
- EPN-701 (Oral)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 60 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Oct 2026to 30 Sep 2030
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Show all 13Show fewer
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site
Renal Associates
Boston, Massachusetts, 02114
Scott Krinsky, 9784063408, skrinsky1@mgb.org
skrinsky1@mgb.org
About this study
Official title: Phase 2 Study of EPN-701 for the Treatment of Calciphylaxis
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.
Read the detailed 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 the safety of EPN-701 in dialysis-dependent patients with calciphylaxis. Safety will be assessed by active monitoring for adverse events throughout the trial period.
The pharmacodynamic efficacy will be primarily assessed by pre-post (Baseline-Week 12) change in plasma ucMGP among the trial arms. EPN-701 plasma concentration time profiles and other pharmacokinetic assessments will also be assessed.
Exploratory outcomes will be pre-post (Baseline-Week 24) changes in radiotracer uptake indicative of calcification burden, wound severity scores, hospitalization, mortality, and quality of life. The Investigators will also explore novel biomarkers of calciphylaxis via plasma proteomics and examine the associations between changes in ucMGP, radiotracer uptake, and novel biomarkers with patient-oriented clinical outcomes to inform the selection of efficacy endpoints for a future phase 3 RCT.
Findings from the proposed trial will inform the end point selection and design of a future phase 3 trial aimed at establishing a targeted novel therapy for calciphylaxis.
- Conditions
- Calciphylaxis
- Being tested
- EPN-701 (Oral), Placebo (Normal Saline)
- Keywords
- calciphylaxis, Massachusetts General Hospital, MGH, MGB, Mass General Brigham
- Medical subject headings
- Calciphylaxis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EPN-701 (Oral)Drug
Randomized subjects will receive EPN-701 or placebo
Placebo (Normal Saline)Drug
Randomized subjects will receive EPN-701 or placebo.
| Study group | Role | What they receive |
|---|---|---|
| EPN-701 | Active comparator | EPN-701 will be administered daily by mouth Receives: Drug: EPN-701 (Oral) |
| Placebo | Placebo comparator | Placebo will be taken daily by mouth Receives: Drug: Placebo (Normal Saline) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
Measured: Baseline-24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 May 2026
- Study start1 Oct 2026 (estimated)
- Primary completion30 Sep 2030 (estimated)
- Study completion30 Sep 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for CalciphylaxisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sagar U. Nigwekar, MD, MMSc
- Sponsor type
- University, hospital or other
- Collaborators
- Epizon Pharma, Inc.
- Responsible party
- Sponsor-investigator, Sagar U. Nigwekar, MD, MMSc, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07583745
- Sponsor's protocol ID
- 2026P000834
- Status last verified
- Sep 2026
- First submitted
- 29 Apr 2026
- Last updated
- 14 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2 Study of EPN-701 for the Treatment of Calciphylaxis
- ClinicalTrials.gov ID
- NCT07583745
- Acronym / sponsor ID
- 2026P000834
- Conditions
- Calciphylaxis
- Keywords
- calciphylaxis, Massachusetts General Hospital, MGH, MGB, Mass General Brigham
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Sagar U. Nigwekar, MD, MMScUniversity, hospital or other
- Collaborators
- Epizon Pharma, Inc.
- Study start
- 1 Oct 2026 (estimated)
- Primary completion
- 30 Sep 2030 (estimated)
- Study completion
- 30 Sep 2030 (estimated)
- First posted
- 13 May 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed 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 the safety of EPN-701 in dialysis-dependent patients with calciphylaxis. Safety will be assessed by active monitoring for adverse events throughout the trial period.
The pharmacodynamic efficacy will be primarily assessed by pre-post (Baseline-Week 12) change in plasma ucMGP among the trial arms. EPN-701 plasma concentration time profiles and other pharmacokinetic assessments will also be assessed.
Exploratory outcomes will be pre-post (Baseline-Week 24) changes in radiotracer uptake indicative of calcification burden, wound severity scores, hospitalization, mortality, and quality of life. The Investigators will also explore novel biomarkers of calciphylaxis via plasma proteomics and examine the associations between changes in ucMGP, radiotracer uptake, and novel biomarkers with patient-oriented clinical outcomes to inform the selection of efficacy endpoints for a future phase 3 RCT.
Findings from the proposed trial will inform the end point selection and design of a future phase 3 trial aimed at establishing a targeted novel therapy for calciphylaxis.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| EPN-701 | Active comparator | Drug: EPN-701 (Oral)EPN-701 will be administered daily by mouth |
| Placebo | Placebo comparator | Drug: Placebo (Normal Saline)Placebo will be taken daily by mouth |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (13 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EPN-701 (Oral)Randomized subjects will receive EPN-701 or placebo | Drug | EPN-701 |
| Placebo (Normal Saline)Randomized subjects will receive EPN-701 or placebo. | Drug | Placebo |
Primary outcome
Change in pain intensity (Alternate Primary Endpoint)
Time frame: Baseline-24 weeks
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
Change in total calciphylaxis lesion(s) surface area (Alternate Primary Endpoint)
Time frame: Baseline-24 weeks
Pre-post (Baseline-Week 24) change in total surface area of ulcerated skin lesions of calciphylaxis among the trial arms
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Sep 2026.
Related trials for Calciphylaxis
RecruitingObservational study
Partners Calciphylaxis Biobank
Calciphylaxis, Calcific Uremic Arteriolopathy
Not yet recruitingPhase 4
Emerging Indications for Hyperbaric Oxygen Treatment
Post-COVID-19 Condition, Ulcerative Colitis
Not yet recruitingPhase 1
Anti-Interleukin-6 (IL6) in Calciphylaxis
Calciphylaxis
RecruitingObservational study
Calciphylaxis in Patients With Chronic Kidney Disease - A Study of the Danish Calciphylaxis Research Initiative (DanCaRI)
Calciphylaxis, Calcific Uremic Arteriolopathy
RecruitingEarly Phase 1
Human Amniotic-Derived Mesenchymal Stem Cell Therapy for Calciphylaxis
Chronic Kidney Diseases, Calciphylaxis
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing EPN-701 (Oral) in people with Calciphylaxis, run by Sagar U. Nigwekar, MD, MMSc at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 14 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in pain intensity (Alternate Primary Endpoint) (time frame: Baseline-24 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Sep 2026.