RECRUITING

Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)

Conditions

Quick Navigation

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

To evaluate the long-term safety and tolerability of oral dersimelagon.

Official Title

A Phase 3, Multicenter, Open-label, Long-term, Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)

Quick Facts

Study Start:2021-08-10

Study Completion:2027-12

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT05005975

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:12 Years to 75 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
* 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 potential and male subjects with partner of childbearing potential must agree to use 2 effective methods of contraception including barrier method (especially for female subjects, one method must be highly effective method)	* 1. History or presence of photodermatoses other than EPP or XLP. * 2. Presence of clinically significant hepatobiliary disease at Screening, determined as clinically significant by the Investigator. * 3. Subjects with AST, ALT, ALP ≥ 3.0 × upper limit of normal (ULN) or TB \> 1.5 × ULN at Screening. The TB level of \> 1.5 × ULN listed in this exclusion criteria may not be applicable to subjects with a documented medical history of Gilbert's syndrome. Please consult with the Sponsor for eligibility of subjects with elevated levels due to Gilbert's syndrome. * 4. Subjects with or having a history (in the last 2 years) of excessive alcohol intake in the opinion of the Investigator. * 5. History of melanoma. * 6. 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. * 7. 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. * 8. 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/1.73m2 as calculated by the CKD-EPI creatinine equation (2009) for adults and by the Schwartz creatinine equation for adolescents (2009). MDRD can be used for adults per local recommendations. * 9. 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. * 10. Female subjects who are pregnant, lactating, or intending to become pregnant during the study. * 11. Treatment with phototherapy or afamelanotide within 3 months before baseline (Visit 2 or Re-entry Visit 2). * 12. Treatment with cimetidine or antioxidant agents at doses which, in the opinion of the Investigator, may affect study endpoints (including but not limited to beta-carotene, cysteine, pyridoxine) within 4 weeks before baseline (Visit 2 or Re-entry Visit 2). * 13. Chronic treatment with opioids, ketamine, or medical formulations or derivatives of cannabis within 4 weeks before baseline (Visit 2). Note: This exclusion criterion may not be applicable to subjects at Re-entry Visits. Acute use of scheduled analgesics more than 3 months before baseline (Visit 2) is allowed. * 14. 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. * 15. Previous treatment with any investigational agent other than dersimelagon within 12 weeks before Screening OR 5 half-lives of the investigational product (whichever is longer). * 16. History of any hypersensitivity to the active ingredient and/or excipients (lactose monohydrate, hydroxypropyl cellulose, carmellose calcium, magnesium stearate, hypromellose, titanium dioxide, talc, polyethylene glycol, iron oxide yellow, iron oxide red, and iron oxide black). * 17. 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. * 18. Using the following drugs (including but not limited to) within 1 week of baseline (Visit 2 or Re-entry Visit 2): 1. Drugs known to be predominantly metabolized by cytochrome P450 (CYP) 3A4 with a narrow therapeutic index for which elevated plasma concentrations are associated with clinical safety concern or significant medical events. 2. Drugs that are known substrates of P-glycoprotein (P-gp), breast cancer resistance protein (BCRP), organic anion transporting polypeptide (OATP)1B1, or OATP1B3 for which elevated plasma concentrations are associated with significant medical events.

Inclusion Criteria

Exclusion Criteria

* 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 potential and male subjects with partner of childbearing potential must agree to use 2 effective methods of contraception including barrier method (especially for female subjects, one method must be highly effective method)

* 1. History or presence of photodermatoses other than EPP or XLP.
* 2. Presence of clinically significant hepatobiliary disease at Screening, determined as clinically significant by the Investigator.
* 3. Subjects with AST, ALT, ALP ≥ 3.0 × upper limit of normal (ULN) or TB \> 1.5 × ULN at Screening. The TB level of \> 1.5 × ULN listed in this exclusion criteria may not be applicable to subjects with a documented medical history of Gilbert's syndrome. Please consult with the Sponsor for eligibility of subjects with elevated levels due to Gilbert's syndrome.
* 4. Subjects with or having a history (in the last 2 years) of excessive alcohol intake in the opinion of the Investigator.
* 5. History of melanoma.
* 6. 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.
* 7. 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.
* 8. 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/1.73m2 as calculated by the CKD-EPI creatinine equation (2009) for adults and by the Schwartz creatinine equation for adolescents (2009). MDRD can be used for adults per local recommendations.
* 9. 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.
* 10. Female subjects who are pregnant, lactating, or intending to become pregnant during the study.
* 11. Treatment with phototherapy or afamelanotide within 3 months before baseline (Visit 2 or Re-entry Visit 2).
* 12. Treatment with cimetidine or antioxidant agents at doses which, in the opinion of the Investigator, may affect study endpoints (including but not limited to beta-carotene, cysteine, pyridoxine) within 4 weeks before baseline (Visit 2 or Re-entry Visit 2).
* 13. Chronic treatment with opioids, ketamine, or medical formulations or derivatives of cannabis within 4 weeks before baseline (Visit 2). Note: This exclusion criterion may not be applicable to subjects at Re-entry Visits. Acute use of scheduled analgesics more than 3 months before baseline (Visit 2) is allowed.
* 14. 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.
* 15. Previous treatment with any investigational agent other than dersimelagon within 12 weeks before Screening OR 5 half-lives of the investigational product (whichever is longer).
* 16. History of any hypersensitivity to the active ingredient and/or excipients (lactose monohydrate, hydroxypropyl cellulose, carmellose calcium, magnesium stearate, hypromellose, titanium dioxide, talc, polyethylene glycol, iron oxide yellow, iron oxide red, and iron oxide black).
* 17. 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.
* 18. Using the following drugs (including but not limited to) within 1 week of baseline (Visit 2 or Re-entry Visit 2):
1. Drugs known to be predominantly metabolized by cytochrome P450 (CYP) 3A4 with a narrow therapeutic index for which elevated plasma concentrations are associated with clinical safety concern or significant medical events.
2. Drugs that are known substrates of P-glycoprotein (P-gp), breast cancer resistance protein (BCRP), organic anion transporting polypeptide (OATP)1B1, or OATP1B3 for which elevated plasma concentrations are associated with significant medical events.

Contacts and Locations

Study Contact

Clinical Trials Information Desk, To prevent mis-communication,

CONTACT

please e-mail

information@mt-pharma-us.com

Principal Investigator

Head of Medical Science

STUDY_DIRECTOR

Mitsubishi Tanabe Pharma America Inc.

Study Locations (Sites)

Marvel Clinical Research, LLC

Huntington Beach, California, 92647

United States

University of California at San Francisco - CSF Porphyria Center

San Francisco, California, 94143

United States

University Of Miami School Of Medicine, Center For Liver Diseases

Miami, Florida, 33136

United States

Massachusetts General Hospital

Boston, Massachusetts, 02114

United States

MetroBoston Clinical Partners, LLC

Brighton, Massachusetts, 02135-3211

United States

Henry Ford Health System

Detroit, Michigan, 48202

United States

Kansas City Research Institute

Kansas City, Missouri, 64131

United States

Icahn School of Medicine at Mount Sinai (ISSMS)-The Mount Sinai Hospital (MSH)

New York, New York, 10029

United States

Wake Forest University Baptist Health

Winston-Salem, North Carolina, 27157

United States

Cleveland Clinic - Taussig Cancer Institute

Cleveland, Ohio, 44106

United States

Remington-Davis Clinical Research

Columbus, Ohio, 43215

United States

Thomas Jefferson University

Philadelphia, Pennsylvania, 19107

United States

Einstein Medical Center, Philadelphia

Philadelphia, Pennsylvania, 19141

United States

The University of Texas Medical Branch (UTMB)

Galveston, Texas, 77555

United States

University of Washington-Seattle Cancer Care Alliance

Seattle, Washington, 98109

United States

Collaborators and Investigators

Sponsor: Mitsubishi Tanabe Pharma America Inc.

Head of Medical Science, STUDY_DIRECTOR, Mitsubishi Tanabe Pharma America Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2021-08-10

Study Completion Date2027-12

Study Record Updates

Study Start Date2021-08-10

Study Completion Date2027-12

Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)

Conditions

Quick Navigation

Study Overview

Description

Official Title

Quick Facts

Study ID

Participation Criteria

Contacts and Locations

Study Contact

Principal Investigator

Study Locations (Sites)

Collaborators and Investigators

Study Record Dates

Study Registration Dates

Study Record Updates

Terms related to this study

Additional Relevant MeSH Terms