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

Erythropoietic Protoporphyria (EPP) Clinical Trial

— INSPIRE  

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06144840
• Other study ID #: MT-7117-A-302
• Secondary ID: jRCT2031230656
• Status: Recruiting
• Phase: Phase 3
• Start date: December 11, 2023
• Est. completion date: June 2026

Study information

• Verified date: June 2024
• Source: Mitsubishi Tanabe Pharma America Inc.
• Contact: Clinical Trials Information Desk, To prevent mis-communication,
• Phone: please email:
• Email: information[@]mt-pharma-us.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 150
• Est. completion date: June 2026
• Est. primary completion date: April 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years to 75 Years

Eligibility

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.

Related Conditions & MeSH terms

• Erythropoietic Protoporphyria (EPP)
• Genetic Diseases, X-Linked

Intervention

Drug:
• Dersimelagon
MT-7117
• Placebo
Placebo

Locations

Country	Name	City	State
Australia	Royal Prince Alfred Hospital	Camperdown	New South Wales
Australia	Royal Melbourne Hospital (RMH)	Parkville	Victoria
Bulgaria	University Multi-Profile Hospital for Active Treatment (UMHAT) St. Ivan Rilski - Porphyria Center	Sofia
Czechia	Institute for Clinical and Experimental Medicine - IKEM	Prague
France	Centre Hospitalier Universitaire de Bordeaux - Hopital Saint - Andre	Bordeaux
France	CHU Nantes	Nantes
France	Assistance Publique-Hopitaux de Paris (AP-HP) - Hopital Louis-Mourier	Paris
France	Hôpital Bichat - Hospital Bichat-Hopitaux Universitaires Paris Nord Val de Seine	Paris
Italy	Azienda Ospedaliera Spedali Civili di Brescia-Universita degli Studi Di Brescia	Brescia
Italy	Azienda Sanitaria Ospedaliera Santa Croce E Carle - Cuneo	Cuneo
Italy	Ospedalle Galliera	Genova
Italy	Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano	Milan
Italy	U.O.C. Medicina Interna Azienda ospedaliero Universitaria Policlinico di Modena	Modena
Italy	IFO-San Gallicano IRCCS	Rome
Italy	Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Materno-Infantile - Burlo Garofolo - Clinica Pediatrica	Trieste
Japan	Mazda Hospital	Aki-gun	Hiroshima-ken
Japan	Hamamatsu University Hospital	Hamamatsu	Shizuoka
Japan	Tokyo Saiseikai Central Hospital	Tokyo
Netherlands	Universitair Medisch Centrum Rotterdam	Rotterdam
Poland	Instytut Hematologii I Transfuzjologii	Warsaw
Spain	Hospital Universitario 12 de Octubre	Madrid
Spain	Hospital General Universitario De Valencia	Valencia
United Kingdom	Guy's Hospital	London
United Kingdom	Southampton General Hospital - University Hospital Southampton NHS Foundation Trust	Southamption
United States	MGH	Boston	Massachusetts
United States	MetroBoston Clinical Partners, LLC	Brighton	Massachusetts
United States	Remington-Davis Clinical Research	Columbus	Ohio
United States	Henry Ford Health System	Detroit	Michigan
United States	The University of Texas Medical Branch (UTMB)	Galveston	Texas
United States	Marvel Clinical Research, LLC	Huntington Beach	California
United States	Kansas City Research Institute	Kansas City	Missouri
United States	University Of Miami School Of Medicine, Center For Liver Diseases	Miami	Florida
United States	Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH)	New York	New York
United States	Einstein Medical Center (EMC)	Philadelphia	Pennsylvania
United States	University of California at San Francisco	San Francisco	California
United States	University of Washington	Seattle	Washington
United States	Wake Forest University Baptist Health	Winston-Salem	North Carolina

Sponsors (1)

Lead Sponsor	Collaborator
Mitsubishi Tanabe Pharma America Inc.

Countries where clinical trial is conducted

United States,  Australia,  Bulgaria,  Czechia,  France,  Italy,  Japan,  Netherlands,  Poland,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	The comparison between MT-7117 treatment group and placebo group will be performed.	Week 16
Secondary	Patient Global Impression of Change (PGIC) at Week 16.	The comparison between MT-7117 treatment group and placebo group will be performed.	Week 16
Secondary	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.	The comparison between MT-7117 treatment group and placebo group will be performed.	Week 16
Secondary	Total number of sunlight-induced non-prodrome, phototoxic reactions during the 16-week double-blind treatment period.	The comparison between MT-7117 treatment group and placebo group will be performed.	Week 16