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Tests treatment safety and results for EPP
Official title Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)
ClinicalTrials.gov ID: NCT05005975
What this study is testing
What is MT-7117?
MT-7117 is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for epp.
Also referred to as Dersimelagon.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- To evaluate the long-term safety and tolerability of oral dersimelagon.
- Phase 3: a large, late-stage study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 12 to 75
You may be able to join if
- Additional screening criteria check may apply for qualification:
- 1\. people provided written informed consent to participate. For adolescent people, both adolescent assent and parental consent will be provided.
- 2\. people who have completed: MT-7117-G01 (completed through Week 58 [Visit 12]) or, MT-7117-A-302 (completed through Week 58 [Visit 10]) or...
- 3\. people are willing and able to travel to the study sites for all scheduled visits.
- 4\. In the Investigator's opinion, subject can understand the nature of the study and any risks involved in participation, and is willing to...
You likely can't join if
- Additional screening criteria check may apply for qualification: A subject will NOT be eligible for this study if ANY of the following criteria apply:
- 1\. History or presence of photodermatoses other than EPP or XLP.
- 2\. Presence of clinically significant hepatobiliary disease at Screening, determined as clinically significant by the Investigator.
- 3\. people with AST, ALT, ALP ≥ 3.0 × upper limit of normal (ULN) or TB \> 1.5 × ULN at Screening. The TB level of \> 1.5 × ULN listed in this...
- 4\. people with or having a history (in the last 2 years) of excessive alcohol intake in the opinion of the Investigator.
- 5\. History of melanoma.
See the full eligibility criteria
- Additional screening criteria check may apply for qualification:
- 1\. people provided written informed consent to participate. For adolescent people, both adolescent assent and parental consent will be provided.
- 2\. people who have completed: MT-7117-G01 (completed through Week 58 [Visit 12]) or, MT-7117-A-302 (completed through Week 58 [Visit 10]) or, MT-7117-A-301 (completed EOT - Week 104 or Week 130) according to protocol...
- 3\. people are willing and able to travel to the study sites for all scheduled visits.
- 4\. In the Investigator's opinion, subject can understand the nature of the study and any risks involved in participation, and is willing to cooperate and comply with the protocol restrictions and requirements...
- 5\. Female people who are non-lactating and have a negative urine pregnancy test at baseline visit prior to receiving the first dose of study drug.
- 6\. Female people of childbearing potential and male people with partner of childbearing potential must agree to use 2 effective methods of contraception including barrier method (especially for female people, one...
- Additional screening criteria check may apply for qualification: A subject will NOT be eligible for this study if ANY of the following criteria apply:
- 1\. History or presence of photodermatoses other than EPP or XLP.
- 2\. Presence of clinically significant hepatobiliary disease at Screening, determined as clinically significant by the Investigator.
- 3\. people with AST, ALT, ALP ≥ 3.0 × upper limit of normal (ULN) or TB \> 1.5 × ULN at Screening. The TB level of \> 1.5 × ULN listed in this exclusion criteria may not be applicable to people with a documented medical...
- 4\. people with or having a history (in the last 2 years) of excessive alcohol intake in the opinion of the Investigator.
- 5\. History of melanoma.
- 6\. Presence of squamous cell carcinoma, basal cell carcinoma, or other malignant skin lesions. Any suspicious lesions or nevi will be evaluated. If the suspicious lesion or nevi cannot be resolved through biopsy or...
- 7\. 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 people.
- 8\. Presence of clinically significant acute or chronic renal disease based upon the subject's medical records including hemodialysis; an estimated glomerular filtration rate (eGFR) \<60 mL/min/1.73m2 as calculated by...
- 9\. 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 people.
- 10\. Female people who are pregnant, lactating, or intending to become pregnant during the study.
- 11\. Treatment with phototherapy or afamelanotide within 3 months before baseline (Visit 2 or Re-entry Visit 2).
- 12\. Treatment with cimetidine or 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...
- 13\. Chronic treatment with opioids, ketamine, or medical formulations or derivatives of cannabis within 4 weeks before baseline (Visit 2). Note: This exclusion criterion may not be applicable to people at Re-entry...
- 14\. 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 people.
- 15\. Previous treatment with any investigational agent other than dersimelagon within 12 weeks before Screening OR 5 half-lives of the investigational product (whichever is longer).
- 16\. History of any hypersensitivity to the active ingredient and/or excipients (lactose monohydrate, hydroxypropyl cellulose, carmellose calcium, magnesium stearate, hypromellose, titanium dioxide, talc, polyethylene...
- 17\. people who are unable to swallow tablets or have diseases significantly affecting the gastrointestinal function such as malabsorption syndrome, resection of the stomach or small bowel, bariatric surgery procedures...
- 18\. Using the following drugs (including but not limited to) within 1 week of baseline (Visit 2 or Re-entry Visit 2):
- Drugs known to be predominantly metabolized by cytochrome P450 (CYP) 3A4 with a narrow therapeutic index for which elevated plasma concentrations are associated with clinical safety concern or significant medical events.
- Drugs that are known substrates of P-glycoprotein (P-gp), breast cancer resistance protein (BCRP), organic anion transporting polypeptide (OATP)1B1, or OATP1B3 for which elevated plasma concentrations are associated...
The study team makes the final eligibility decision.
Where it's taking place
- Huntington Beach, California, United States
- San Francisco, California, United States
- Miami, Florida, United States
- Boston, Massachusetts, United States
- Brighton, Massachusetts, United States
- Detroit, Michigan, United States
- Kansas City, Missouri, United States
- New York, New York, United States
- Winston-Salem, North Carolina, United States
- Cleveland, Ohio, United States
- Columbus, Ohio, United States
- Philadelphia, Pennsylvania, United States
- Galveston, Texas, United States
- Seattle, Washington, United States
- Camperdown, New South Wales, Australia
- Brisbane, Queensland, Australia
- Parkville, Victoria, Australia
- Sophia, Belgium
- Edmonton, Alberta, Canada
- Prague, Czechia
+ 29 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 12 years to 75 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Huntington Beach, California, United States; San Francisco, California, United States; Miami, Florida, United States; Boston, Massachusetts, United States; Brighton, Massachusetts, United States; Detroit, Michigan, United States and 43 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.