Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria

NCT03520036 | Completed Phase 2 Results FDA Drug

• 1 other identifier: MT-7117-A01
• Study Type: interventional
• Target: 102
• Locations: 1 country
• Sites: 9

Brief Summary

The purpose of this study is to investigate the efficacy and safety of MT-7117 on sunlight exposure duration without symptoms and tolerance in subjects with EPP.

Conditions

Erythropoietic Protoporphyria (EPP)

Outcome Measures

Primary Outcomes (3)

• Change From Baseline in Average Daily Time (Minutes) to First Prodromal Symptom Associated With Sunlight Exposure Between Hour Post Sunrise and 1 Hour Pre-Sunset at Week 16.

  Duration in minutes, of sunlight exposure between 1 hour post sunrise and 1 hour pre-sunset. The average Duration in minutes, of sunlight exposure before the first prodromal symptom between 1 hour post sunrise and 1 hour pre-sunset. The average duration means that average of daily durations in 14-day windows before Day 1 (or week 16 Day) applied.

  Baseline (Week 0) and Week 16

• Change From Baseline in Average Daily Duration (Minutes) of Sunlight Exposure Between 1 Hour Post Sunrise and 1 Hour Pre-Sunset Without Prodromal Symptoms at Week 16

  Change from baseline to week 16 in Average Daily Duration (Minutes) of Sunlight Exposure sums any sunlight exposure time excluding any overlapped time with prodromal symptoms, including if the patients go out multiple times on the same day after the prodromal symptom had previously ended.

  Baseline (Week 0), and Week 16

• Change From Baseline in Average Daily Mean Duration (Minutes) of Sunlight Exposure Between 1 Hour Post Sunrise and 1 Hour Pre-Sunset Without Prodromal Symptoms at Week 16

  Change from baseline to week 16 in Average Daily Mean Duration (Minutes) of Sunlight Exposure without prodromal symptoms divided by the number of sunlight exposures periods applicable that day.

  Baseline (Week 0), and Week 16

Secondary Outcomes (6)

• Total Number of Sunlight Exposure Episodes With Prodromal Symptoms During 16-Week Double-Blind Treatment Period

  Week 16

• Change From Baseline in Average Daily Mean Intensity of Prodromal Symptoms During 16-week Double-blind Treatment Period in 11-point Likert Scale

  Baseline (Week 0), and Week 16

• Change From Baseline in Average Daily Duration (Minutes) of Prodromal Symptoms at 16-Week Double-Blind Treatment Period

  Baseline (Week 0), and Week 16

• Change From Baseline in Pigmentation as Measured by Melanin Density for Average of 6 Skin Segments at Week 8 and Week 16.

  Baseline (Week 0), Week 8, and Week 16

• Percent Change From Baseline in Pigmentation as Measured by Melanin Density for Average of 6 Skin Segments at Week 8 and Week 16.

  Baseline (Week 0), Week 8, and Week 16

Other Outcomes (3)

• Total Number of Sunlight Exposure Episodes

  Baseline (Week 0) and Week 16

• Change in Pigmentation as Measured by Melanin Density

  Baseline (Week 0) and Week 16

• The Quality of Life as Measured by the Patient Reported Outcomes Measurement Information System (PROMIS) 57

  Baseline (Week 0) and Week 16

Study Arms (3)

MT-7117 low dose

EXPERIMENTAL

Drug: MT-7117 low dose

MT-7117 high dose

EXPERIMENTAL

Drug: MT-7117 high dose

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

MT-7117 low dose DRUG

MT-7117 low dose QD, oral, 16 weeks

MT-7117 low dose

MT-7117 high dose DRUG

MT-7117 high dose QD, oral, 16 weeks

MT-7117 high dose

Placebo DRUG

Placebo QD, oral, 16 weeks

Placebo

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• \. Subjects provided written informed consent to participate.
• \. Male and female subjects with a confirmed diagnosis of EPP based on medical history, aged 18 years to 75 years, inclusive, at Screening.
• \. Subjects are willing and able to travel to the study sites for all scheduled visits.
• \. 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).

You may not qualify if:

• \. History or presence of photodermatoses other than EPP.
• \. 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.
• \. Presence of clinically significant hepatobiliary disease based on LFT values at Screening.
• \. Subjects with AST, ALT, ALP ≥3.0 × upper limit of normal (ULN) or total bilirubin \>1.5 × ULN at Screening.
• \. Subjects with or having a history (in the last 2 years) of excessive alcohol intake in the opinion of the Investigator.
• \. History or presence of melanoma and/or atypical nevus at Screening.
• \. History of familial melanoma (defined as having 2 or more first-degree relatives, such as parents, sibling and/or child).
• \. History or presence of pre-malignant skin lesion squamous cell carcinoma, basal cell carcinoma, or other malignant skin lesions.
• \. 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.
• \. 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.
• \. 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.
• \. Pregnancy or lactation.
• \. 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.
• \. Treatment with phototherapy within 3 months before Randomization (Visit 2).
• \. Treatment with afamelanotide within 3 months before Randomization (Visit 2).

Sponsors & Collaborators

• Tanabe Pharma America, Inc.: lead

Study Sites (9)

ACTCA, A Member of the Alliance, Inc.

Los Angeles, California, 90036, United States

Location

University of California at San Francisco

San Francisco, California, 94143, United States

Location

University of Miami Miller School of Medicine

Miami, Florida, 33136, United States

Location

Metro Boston Clinical Partners, LLC

Brighton, Massachusetts, 02135, United States

Location

Ichan School of Medicine at Mount Sinai

New York, New York, 10029, United States

Location

Wake Forest Baptist Medical Center

Winston-Salem, North Carolina, 27157, United States

Location

Remington-Davis, Inc

Columbus, Ohio, 43215, United States

Location

University of Texas Medical Branch Porphyria Center

Galveston, Texas, 77555, United States

Location

University of Utah

Salt Lake City, Utah, 84108, United States

Location

Related Publications (2)

• Balwani M, Bonkovsky HL, Levy C, Anderson KE, Bissell DM, Parker C, Takahashi F, Desnick RJ, Belongie K; Endeavor Investigators. Dersimelagon in Erythropoietic Protoporphyrias. N Engl J Med. 2023 Apr 13;388(15):1376-1385. doi: 10.1056/NEJMoa2208754.

  PMID: 37043653 RESULT

• Ogasawara A, Ide R, Inoue S, Tsuda M, Teng R. Assessment of Potential Drug-Drug Interactions for Novel Oral Melanocortin-1 Receptor Agonist Dersimelagon. Pharmacol Res Perspect. 2025 Feb;13(1):e70069. doi: 10.1002/prp2.70069.

  PMID: 39887900 DERIVED

MeSH Terms

Conditions

Protoporphyria, Erythropoietic

Condition Hierarchy (Ancestors)

Porphyrias, Hepatic; Liver Diseases; Digestive System Diseases; Skin Diseases, Genetic; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases; Skin and Connective Tissue Diseases; Porphyrias; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Clinical Trials, Information Desk
• Organization: Tanabe Pharma America, Inc.

information.US@mb.tanabe-pharma.com

908-607-1980

Study Officials

• Head of Medical Science

  Tanabe Pharma America, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 23, 2018
• First Posted: May 9, 2018
• Study Start: July 5, 2018
• Primary Completion: September 28, 2019
• Study Completion: September 28, 2019
• Last Updated: December 31, 2025
• Results First Posted: January 20, 2023

Record last verified: 2025-12

Locations

US (9)