New pill could let EPP patients enjoy the sun without pain
NCT ID NCT03520036
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
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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102 people
The number who actually took part.
- Started
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Jul 2018
- Finished
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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
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18 to 75 years
- Sex
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Anyone
- Healthy volunteers
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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 Hide 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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Genom att skicka in godkänner du våra Användarvillkor
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
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ACTCA, A Member of the Alliance, Inc.
Los Angeles, California, 90036, United States
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Ichan School of Medicine at Mount Sinai
New York, New York, 10029, United States
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Metro Boston Clinical Partners, LLC
Brighton, Massachusetts, 02135, United States
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Remington-Davis, Inc
Columbus, Ohio, 43215, United States
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University of California at San Francisco
San Francisco, California, 94143, United States
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University of Miami Miller School of Medicine
Miami, Florida, 33136, United States
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University of Texas Medical Branch Porphyria Center
Galveston, Texas, 77555, United States
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University of Utah
Salt Lake City, Utah, 84108, United States
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Wake Forest Baptist Medical Center
Winston-Salem, North Carolina, 27157, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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