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

Sponsor

Mitsubishi Tanabe Pharma Development America, Inc. (Industry)

Overall Status

Recruiting

CT.gov ID

NCT05005975

Collaborator

(none)

175

Enrollment

Locations

Arm

24.7

Anticipated Duration (Months)

5.3

Patients Per Site

0.2

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
EPP XLP	Drug: MT-7117	Phase 3

Study Design

Study Type:

Interventional

Anticipated Enrollment :

175 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

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)

Actual Study Start Date :

Aug 10, 2021

Anticipated Primary Completion Date :

Sep 1, 2023

Anticipated Study Completion Date :

Sep 1, 2023

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Dersimelagon 100mg	Drug: MT-7117 MT-7117 Other Names: Dersimelagon

Arm

Intervention/Treatment

Experimental: Dersimelagon 100mg

Drug: MT-7117

MT-7117

Other Names:

Dersimelagon

Outcome Measures

Primary Outcome Measures

Number of patients with Treatment emergent adverse events (TEAEs) (including serious adverse events [SAEs] and adverse events of special interest [AESIs]). [up to a maximum 24 months]
Vital signs (blood pressure, respiratory rate, pulse rate, and body temperature), Clinical laboratory examinations (hematology, coagulation, biochemistry, urinalysis, and others), 12-lead electrocardiogram (ECG) parameters (Mean Heart Rate, PR Interval, QRS Duration, QT interval, QTcB and QTcF) will be assessed.
Number of patients with abnormal Physical examination data [up to a maximum 24 months]
Physical examination consists of assessment of abdominal, respiratory, cardiovascular, general appearance, and others.
Number of patients with Nevi appearance [up to a maximum 24 months]

Eligibility Criteria

Criteria

Ages Eligible for Study:

12 Years to 75 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

1. Subjects provided written informed consent to participate. For adolescent subjects, both adolescent assent and parental consent will be provided.
1. Subjects who complete MT-7117-G01 (complete through Week 58 [Visit 12])
1. Subjects have a body weight of ≥30 kg.
1. Subjects are willing and able to travel to the study sites for all scheduled visits.
1. 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).
1. 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.
1. 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)

Exclusion Criteria:

A subject will NOT be eligible for this study if ANY of the following criteria apply:

1. History or presence of photodermatoses other than EPP or XLP.
1. Presence of clinically significant hepatobiliary disease at Screening.
1. Subjects with AST, ALT, ALP ≥3.0 × upper limit of normal (ULN) or total bilirubin

1.5 × ULN at Screening.

1. Subjects with or having a history (in the last 2 years) of excessive alcohol intake in the opinion of the Investigator.
1. History of melanoma.
1. Presence of melanoma and/or lesions suspicious for melanoma at Screening.
1. History of familial melanoma (defined as having 2 or more first-degree relatives, such as parents, sibling and/or child).
1. 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.
1. 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.
1. 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.
1. 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.
1. Female subjects who are pregnant, lactating, or intending to become pregnant during the study.
1. Treatment with phototherapy or afamelanotide within 3 months before baseline (Visit 2).
1. 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).
1. 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 baseline (Visit 2). Acute use of scheduled narcotics greater than 3 months prior to baseline, over-the-counter medications (OTCs), such as non-steroidal anti-inflammatory drugs (NSAIDs) or aspirin for analgesia, or prior temporary use of scheduled agents within 3 months of baseline (Visit 2) are not excluded.
1. 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.
1. 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).

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Marvel Clinical Research, LLC	Huntington Beach	California	United States	92647
2	University of California at San Francisco-CSF Porphyria Center	San Francisco	California	United States	94143
3	University of Miami School of Medicne, Center for Liver Diseases	Miami	Florida	United States	33136
4	MetroBoston Clinical Partners, LLC	Brighton	Massachusetts	United States	02135-3211
5	Kansas City Research Institute	Kansas City	Missouri	United States	64131
6	Icahn School of Medicine at Mount Sinai (ISSMS)-The Mount Sinai Hospital (MSH)	New York	New York	United States	10029
7	Wake Forest University Baptist Health	Winston-Salem	North Carolina	United States	27157
8	Remington-Davis Clinical Research	Columbus	Ohio	United States	43215
9	Thomas Jefferson University	Philadelphia	Pennsylvania	United States	19107
10	The University of Texas Medical Branch (UTMB)	Galveston	Texas	United States	77555
11	University of Washington-Seattle Cancer Care Alliance	Seattle	Washington	United States	98109
12	The Wesley Hospital	Brisbane	Queensland	Australia	4066
13	Royal Melbourne Hospital (RMH)	Parkville	Victoria	Australia	3050
14	University of Alberta Hospital	Edmonton	Alberta	Canada	Edmonton AB T6G 2G3
15	Westfaelische Wilhelms-Universitaet Muenster	Muenster	Northrhein Westalien	Germany	48149
16	Charite - Universitaetsmedizin Berlin	Berlin		Germany	10117
17	Azienda Ospedaliera Spedali Civili di Brescia-Universita degli Studi Di Brescia	Brescia		Italy	25100
18	Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano	Milan		Italy	20122
19	U.O.C. Medicina Interna Azienda ospedaliero Universitaria Policlinico di Modena	Modena		Italy	41125
20	I.F.O. Hospital, Centro Porfirie e Malattie Rare	Rome		Italy	144
21	Kobe University Hospital	Kobe	Hyogo	Japan	650-0017
22	Sophia Dermatology Clinic	Kanazawa	Ishikawa	Japan	921-8035
23	Investigator site	Sayama	Osaka	Japan
24	Osaka Medical College Hospital	Takatsuki	Osaka	Japan	569-8686
25	Tokyo Saiseikai Central Hospital	Minato-ku	Tokyo	Japan	108-0073
26	Toyama University Hospital	Sugitani	Toyama	Japan	930-0194
27	Haukeland University Hospital	Bergen		Norway	N5021
28	Hospital Clínic de Barcelona	Barcelona		Spain	8036
29	Hospital Universitario	Madrid		Spain	28041
30	Karolinska University Hospital	Stockholm		Sweden	14186
31	University of Manchester	Salford	Manchester	United Kingdom	M6 8HD
32	Salford Royal NHS Foundation Trust	Manchester	MN	United Kingdom	M6 8HD
33	Evelina London Children's Hospital - Guy's & St Thomas' NHS Foundation Trust	London		United Kingdom	SE1 9RT

Sponsors and Collaborators

Mitsubishi Tanabe Pharma Development America, Inc.

Investigators

Study Director: Head of Medical Science, Mitsubishi Tanabe Pharma Development America, Inc.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Mitsubishi Tanabe Pharma Development America, Inc.

ClinicalTrials.gov Identifier:

NCT05005975

Other Study ID Numbers:

MT-7117-A-301

First Posted:

Aug 16, 2021

Last Update Posted:

Jul 13, 2022

Last Verified:

Jul 1, 2022

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Yes

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Protoporphyria, Erythropoietic

Study Results

No Results Posted as of Jul 13, 2022