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

• Register: ClinicalTrials.gov
• Last refreshed on: 31 March 2025
• Main ID: NCT04402489
• Date of registration: 20/05/2020
• Prospective Registration: Yes
• Primary sponsor: Mitsubishi Tanabe Pharma America Inc.
• Public title: Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria or X-Linked Protoporphyria
• Scientific title: A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Adults and Adolescents With Erythropoietic Protoporphyria or X-Linked Protoporphyria
• Date of first enrolment: June 1, 2020
• Target sample size: 184
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT04402489
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Triple (Participant, Care Provider, Investigator).
• Phase: Phase 3

Countries of recruitment

Australia, Canada, Finland, Germany, Italy, Japan, Norway, Spain, Sweden, United Kingdom, United States

Contacts

• Name: Head of Medical Science
• Affiliation: Mitsubishi Tanabe Pharma America Inc.

Key inclusion & exclusion criteria

Additional screening criteria check may apply for qualification.

Inclusion Criteria:

1. Subjects provided written informed consent to participate. For minor subjects, both
minor assent and parental consent will be provided.

2. Male and female subjects with a confirmed diagnosis of EPP or XLP based on medical
history, aged 12 years to 75 years, inclusive, at Screening.

3. Subjects have a body weight of =30 kg.

4. Subjects are willing and able to travel to the study sites for all scheduled visits.

5. 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).

6. 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.

7. Female subjects of childbearing potential and male subjects with partner of
child-bearing potential currently using/willing to use 2 effective methods of
contraception including barrier method as described in the protocol.

Exclusion Criteria:

1. History or presence of photodermatoses other than EPP or XLP.

2. Subjects who are unwilling or unable to go outside during daylight hours most days
(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 of melanoma.

7. Presence of melanoma and/or lesions suspicious for melanoma at Screening.

8. History of familial melanoma (defined as having 2 or more first-degree relatives,
such as parents, sibling and/or child).

9. 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 excision, the subject will be excluded from the
study.

10. 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.

11. 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 as calculated by the Chronic Kidney Disease-Epidemiology
Collaboration (CKDEPI) creatinine equation (2009) for adults and by the Schwartz
creatinine equation for adolescents (2009). Modification of Diet in Renal Disease
(MDRD) can be used for adults per local recommendations.

12. 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.

13. Female subjects who are pregnant, lactating, or intending to become pregnant during
the study.

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 within 4 weeks before Randomization (Visit 2), at
doses which, in the opinion of the Investigator, may affect study endpoints
(including but not limited to beta-carotene, cysteine, pyridoxine).

18. Chronic treatment with any scheduled analgesic agents including, but not limited to,
opioids and opioid derivatives such as morphine, hydrocodone, oxycodone, fentanyl,
or their combination with other unscheduled analgesics or non-steroidal
anti-inflammatory drug (Percocet and Vicodin-like prescription drugs) within 4 weeks
before Randomization (Visit 2).

Acute use of scheduled narcotics greater than 3 months prior to randomization, OTCs,
such as NSAIDs or aspirin for analgesia, or prior temporary use of scheduled agents
within 3 months of screening are not excluded.

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 (this does not include placebo treated subjects).

21. Previous treatment with any investigational agent within 12 weeks before Screening
OR 5 half-lives of the investigational product (whichever is longer).


Age minimum: 12 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

EPP

XLP

Intervention(s)

Drug: MT-7117 High Dose

Drug: MT-7117 Low Dose

Drug: Placebo

Primary Outcome(s)

Change From Baseline in Average Daily Sunlight Exposure Time (Minutes) to First Prodromal Symptom (Burning, Tingling, Itching, or Stinging) Associated With Sunlight Exposure Between 1 Hour Post Sunrise and 1 Hour Pre-sunset at Week 26 (Visit 7) [Time Frame: From 1 hour post-sunrise to 1 hour pre-sunset at Week 26 (Visit 7)]

Secondary Outcome(s)

Change From Baseline for Total Score in the Domain of Pain Intensity in the PROMIS-57 at Week 26 [Time Frame: Baseline (Week 0) and Week 26]

Change From Baseline for Total Score in the Domain of Physical Function in the PROMIS-57 at Week 26 [Time Frame: Baseline (Week 0) and Week 26]

Patient Global Impression of Change (PGIC) at Week 26 [Time Frame: Week 26]

The Percentage of Subjects Who Are Responders [Time Frame: Week 26]

Total Number of Sunlight-induced Pain Events Defined as Prodrome Symptoms (Burning, Tingling, Itching, or Stinging) With Pain Rating of 1-10 on the Likert Scale During the 26-week Double-blind Treatment Period. [Time Frame: During the 26-week double-blind treatment period]

Secondary ID(s)

jRCT2080225355

MT-7117-G01

2019-004226-16

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 25/03/2025
• URL: https://clinicaltrials.gov/ct2/show/results/NCT04402489