INcreased Sun Exposure Without Pain In Research Participants With EPP or XLP (INSPIRE)

• ClinicalTrials.gov Identifier: NCT06144840
• Recruitment Status: Recruiting
• First Posted: November 22, 2023
• Last Update Posted: May 13, 2024
• Sponsor: Mitsubishi Tanabe Pharma America Inc.
• Information provided by (Responsible Party): Mitsubishi Tanabe Pharma America Inc.

Tracking Information

• First Submitted Date: November 17, 2023
• First Posted Date: November 22, 2023
• Last Update Posted Date: May 13, 2024
• Actual Study Start Date: December 11, 2023
• Estimated Primary Completion Date: April 2026 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: November 17, 2023)

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 16 [ Time Frame: Week 16 ]

The comparison between MT-7117 treatment group and placebo group will be performed.

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: November 17, 2023)

• Patient Global Impression of Change (PGIC) at Week 16. [ Time Frame: Week 16 ]
  The comparison between MT-7117 treatment group and placebo group will be performed.
• Total number of sunlight-induced pain events defined as prodromal symptoms (burning, tingling, itching, or stinging) with pain rating of 1-10 on the Likert scale during the 16-week double-blind treatment period. [ Time Frame: Week 16 ]
  The comparison between MT-7117 treatment group and placebo group will be performed.
• Total number of sunlight-induced non-prodrome, phototoxic reactions during the 16-week double-blind treatment period. [ Time Frame: Week 16 ]
  The comparison between MT-7117 treatment group and placebo group will be performed.

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: INcreased Sun Exposure Without Pain In Research Participants With EPP or XLP
• Official 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
• Brief Summary: To investigate the efficacy of MT-7117 on time to onset and severity of first prodromal symptoms (burning, tingling, itching, or stinging) associated with sunlight exposure in adults and adolescents with EPP or XLP.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment

Condition

• Erythropoietic Protoporphyria (EPP)
• X-Linked Protoporphyria (XLP)

Intervention

• Drug: Dersimelagon
  MT-7117
• Drug: Placebo
  Placebo

Study Arms

• Experimental: MT-7117
  Intervention: Drug: Dersimelagon
• Placebo Comparator: Placebo
  Intervention: Drug: Placebo

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: November 17, 2023): 150
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: June 2026
• Estimated Primary Completion Date: April 2026 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

1. Subjects provided written informed consent to participate. For minor subjects, both minor's assent and parental consent will be required.
2. Male and female subjects with a confirmed diagnosis of EPP or XLP based on medical history.
3. Subjects aged 12 years to 75 years, inclusive, at Screening.
4. Subjects are willing and able to travel to the study sites for all scheduled visits.
5. In the Investigator's opinion, subject can 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 and receiving direct sunlight exposure as much as possible).
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.

Additional screening criteria check may apply for qualification.

Exclusion Criteria:

1. History or presence of photodermatoses other than EPP or XLP.
2. Subjects who are unwilling or unable to go outside in sunlight during daylight hours most days (e.g., between 1-hour post-sunrise and 1 hour pre-sunset) during the study.
3. Presence or history of any hepatobiliary disease, including druginduced liver injury at screening, determined as clinically significant by the Investigator after the discussion with the Sponsor Medical Monitor.
4. Subjects with aspartate aminotransferase (AST), alanine aminotransferase (ALT), alkaline phosphatase (ALP) ≥ 2.0 × upper limit of normal (ULN) or total bilirubin >1.5 × ULN at Screening.
5. History (in the last 2 years) or presence of alcohol abuse, or abuse of illicit drugs in the opinion of the Investigator.
6. History of melanoma.
7. 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.
8. 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.
9. Presence of clinically significant acute or chronic renal disease or subjects with an estimated glomerular filtration rate (eGFR) <60 mL/min as calculated by the Chronic Kidney DiseaseEpidemiology Collaboration (CKD-EPI) creatinine equation (2021) for adults and by the Schwartz creatinine equation for adolescents (2009). Modification of Diet in Renal Disease can be used for adults per local recommendations.
10. 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.
11. Female subjects who are pregnant, lactating, or intending to become pregnant during the study.

12. Treatment with any of the following medications or therapy within each period before Randomization (Visit 2);

    • Afamelanotide within 3 months
    • Phototherapy within 3 months
    • Cimetidine within 4 weeks
    • Antioxidant agents within 4 weeks, at doses which, in the opinion of the Investigator, may affect study endpoints (including but not limited to beta-carotene, cysteine, pyridoxine).
    • 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.

    Note: Acute use of scheduled narcotics more than 3 months prior to randomization are allowed. Non-steroidal anti-inflammatory drug, aspirin for analgesia, or prior temporary use of scheduled agents within 3 months of screening is allowed.

13. Dermatological treatments 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 at screening, such as, for example, tanning agents.
14. Subjects who participated in any previous MT-7117 clinical studies.
15. Previous treatment with any investigational agent such as bitopertin, within 12 weeks before Screening or 5 half-lives of the investigational product (whichever is longer).
16. Use of sunscreens with zinc oxide. Note: Sunscreens without zinc oxide are allowed, however their use, in frequency, quantity and body surface area should be maintained relatively stable throughout the duration of the study.
17. History of any hypersensitivity to the active ingredient and/or excipients (lactose monohydrate, hydroxypropylcellulose, carmellose calcium, magnesium stearate, hypromellose, titanium dioxide, talc, polyethylene glycol, iron oxide yellow, iron oxide red, and iron oxide black). (EU ONLY)
18. Subjects 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, symptomatic inflammatory bowel disease, or partial or complete bowel obstruction.(EU ONLY)
19. History of any hypersensitivity to the active ingredient and/or excipients contained in MT-7117 IMP (lactose monohydrate, hydroxypropyl cellulose, carmellose calcium, magnesium stearate, hypromellose, titanium dioxide, talc, polyethylene glycol, iron oxide yellow, iron oxide red, and iron oxide black). (UK ONLY)

Additional screening criteria check may apply for qualification.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 12 Years to 75 Years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Contacts

Contact: Clinical Trials Information Desk, To prevent mis-communication,; please email:; information@mt-pharma-us.com

• Listed Location Countries: Australia, Bulgaria, Czechia, France, Italy, Japan, Netherlands, Poland, Spain, United Kingdom, United States

Administrative Information

• NCT Number: NCT06144840
• Other Study ID Numbers: MT-7117-A-302; jRCT2031230656 ( Registry Identifier: Japan Registry of Clinical Trials (jRCT) )
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Mitsubishi Tanabe Pharma America Inc.
• Original Responsible Party: Same as current
• Current Study Sponsor: Mitsubishi Tanabe Pharma America Inc.
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Study Director: Head of Medical Science; Mitsubishi Tanabe Pharma America Inc.

• PRS Account: Mitsubishi Tanabe Pharma America Inc.
• Verification Date: May 2024