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New pill could let EPP patients enjoy the sun without pain

NCT ID NCT03520036

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 2 trial tested a drug called MT-7117 in 102 adults with erythropoietic protoporphyria (EPP), a rare condition that causes severe pain from sunlight. Participants took either a low dose, high dose, or placebo daily for 16 weeks. The goal was to see if the drug could increase the time they could stay in the sun without symptoms.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
MT-7117
What this could lead to
If it works, this could lead to a daily pill that lets people with EPP spend more time in sunlight without pain.
What could go wrong
This is a small, early-phase trial. The drug may not work better than placebo, and side effects are still being studied.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

102 people

The number who actually took part.

Started

Jul 2018

Finished

Sep 2019

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 75 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Additional screening criteria check may apply for qualification. Inclusion Criteria: * 1\. Subjects provided written informed consent to participate. * 2\. Male and female subjects with a confirmed diagnosis of EPP based on medical history, aged 18 years to 75 years, inclusive, at Screening. * 3\. Subjects are willing and able to travel to the study sites for all scheduled visits. * 4\. In the Investigator's opinion, subject is able to understand the nature of the study and any risks involved in participation, and willing to cooperate and comply with the protocol restrictions and requirements (including travel). Exclusion Criteria: * 1\. History or presence of photodermatoses other than EPP. * 2\. Subjects who are unwilling or unable to go outside during daylight hours (e.g., between 1 hour post sunrise and 1 hour pre-sunset) during the study. * 3\. Presence of clinically significant hepatobiliary disease based on LFT values at Screening. * 4\. Subjects with AST, ALT, ALP ≥3.0 × upper limit of normal (ULN) or total bilirubin \>1.5 × ULN at Screening. * 5\. Subjects with or having a history (in the last 2 years) of excessive alcohol intake in the opinion of the Investigator. * 6\. History or presence of melanoma and/or atypical nevus at Screening. * 7\. History of familial melanoma (defined as having 2 or more first-degree relatives, such as parents, sibling and/or child). * 8\. History or presence of pre-malignant skin lesion squamous cell carcinoma, basal cell carcinoma, or other malignant skin lesions. * 9\. History or presence of psychiatric disease judged to be clinically significant by the Investigator and which may interfere with the study evaluation and/or safety of the subjects. * 10\. Presence of clinically significant acute or chronic renal disease based upon the subject's medical records including hemodialysis; and a serum creatinine level of greater than 1.2 mg/dL or a glomerular filtration rate (GFR) \<60 ml/min. * 11\. Presence of any clinically significant disease or laboratory abnormality which, in the opinion of the Investigator, can interfere with the study objectives and/or safety of the subjects. * 12\. Pregnancy or lactation. * 13\. Females of child bearing potential and male subjects with partners of child-bearing potential unwilling to use adequate contraception measures as described in the protocol. * 14\. Treatment with phototherapy within 3 months before Randomization (Visit 2). * 15\. Treatment with afamelanotide within 3 months before Randomization (Visit 2). * 16\. Treatment with cimetidine within 4 weeks before Randomization (Visit 2). * 17\. Treatment with antioxidant agents at doses which, in the opinion of the Investigator, may affect study endpoints (including but not limited to beta-carotene, cysteine, pyridoxine) within 4 weeks before Randomization (Visit 2). * 18\. Chronic treatment with prescription-based analgesic agents including but not limited to opioids and opioid derivatives such as morphine, hydrocodone, oxycodone or their combination with other analgesics or non-steroidal anti-inflammatory drug (NSAID, as Percocet and Vicodin-like prescription drugs) within 4 weeks before Randomization (Visit 2). * 19\. Treatment with any drugs or supplements which, in the opinion of the Investigator, can interfere with the objectives of the study or safety of the subjects. * 20\. Previous exposure to MT 7117. * 21\. Previous treatment with any investigational agent within 12 weeks before Screening OR 5 half-lives of the investigational product (whichever is longer).

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • ACTCA, A Member of the Alliance, Inc.

    Los Angeles, California, 90036, United States

  • Ichan School of Medicine at Mount Sinai

    New York, New York, 10029, United States

  • Metro Boston Clinical Partners, LLC

    Brighton, Massachusetts, 02135, United States

  • Remington-Davis, Inc

    Columbus, Ohio, 43215, United States

  • University of California at San Francisco

    San Francisco, California, 94143, United States

  • University of Miami Miller School of Medicine

    Miami, Florida, 33136, United States

  • University of Texas Medical Branch Porphyria Center

    Galveston, Texas, 77555, United States

  • University of Utah

    Salt Lake City, Utah, 84108, United States

  • Wake Forest Baptist Medical Center

    Winston-Salem, North Carolina, 27157, United States

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