NCT05005975 · Mitsubishi Tanabe Pharma America Inc.
Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)
What this study is about
To evaluate the long-term safety and how well patients handle the treatment of taken by mouth dersimelagon.
View original scientific description
To evaluate the long-term safety and tolerability of oral dersimelagon.
Interventions
DRUG
MT-7117
MT-7117
Primary outcome measures
Number of patients with Treatment emergent adverse events (TEAEs) (including serious adverse events [SAEs] and adverse events of special interest [AESIs]).
Time frame: up to 66 further months
Vital signs (blood pressure, respiratory rate, pulse rate, and body temperature), Clinical laboratory examinations (hematology, coagulation, biochemistry, urinalysis, and others), 12-lead electrocardiogram (ECG) parameters (Mean Heart Rate, PR Interval, QRS Duration, QT interval, QTcB and QTcF) will be assessed.
Number of patients with abnormal Physical examination data
Time frame: up to 66 further months
Physical examination consists of assessment of abdominal, respiratory, cardiovascular, general appearance, and others.
Number of patients with Nevi appearance
Time frame: up to 66 further months
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Additional screening criteria check may apply for qualification:
- 1\. Subjects provided written informed consent to participate. For adolescent subjects, both adolescent assent and parental consent will be provided.
- 2\. Subjects 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 amendment 1 or 2.
- 3\. Subjects 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 (including travel).
- 5\. Female subjects 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 subjects of childbearing pote
Where
- Huntington Beach, California
- San Francisco, California
- Miami, Florida
- Boston, Massachusetts
- Brighton, Massachusetts
- Detroit, Michigan
- Kansas City, Missouri
- New York, New York
- Winston-Salem, North Carolina
- Cleveland, Ohio
- Columbus, Ohio
- Philadelphia, Pennsylvania
And 2 more locations — see the full list below.
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Dec 11, 2025 · Source of record for eligibility and locations