Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoetic Protoporphyria or X-Linked Protoporphyria
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|ClinicalTrials.gov Identifier: NCT04402489|
Recruitment Status : Active, not recruiting
First Posted : May 26, 2020
Last Update Posted : June 14, 2021
|Condition or disease||Intervention/treatment||Phase|
|EPP XLP||Drug: Placebo Drug: MT-7117 Low Dose Drug: MT-7117 High Dose||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||159 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Triple (Participant, Care Provider, Investigator)|
|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|
|Actual Study Start Date :||June 1, 2020|
|Estimated Primary Completion Date :||September 2021|
|Estimated Study Completion Date :||May 2022|
Placebo Comparator: Placebo Comparator
Oral tablet of placebo once a day.
Experimental: MT-7117 Low Dose
Oral tablet of MT-7117 Low Dose once a day.
Drug: MT-7117 Low Dose
MT-7117 Low Dose
Other Name: Dersimelagon
Experimental: MT-7117 High Dose
Oral tablet of MT-7117 High Dose once a day.
Drug: MT-7117 High Dose
MT-7117 High Dose
Other Name: Dersimelagon
- 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. [ Time Frame: Baseline (Week 0) and 26 weeks ]
- Patient Global Impression of Change (PGIC). [ Time Frame: Week 26 ]PGIC: Scale from 1 to 7, where 7 is worse.
- Total number of sunlight-induced pain events with pain rating of 1-10 on the Likert scale during the 26-week double-blind treatment period. [ Time Frame: Baseline (Week 0) and Week 26 ]
- Change from baseline for total score in the domain of pain intensity in the PROMIS-57. [ Time Frame: Baseline (Week 0) and Week 26 ]Pain intensity: 0 to 10, where 10 is worst pain imaginable.
- The percentage of subjects who are responders based on average daily sunlight exposure time to first prodromal symptom associated with sunlight exposure between 1 hour post sunrise and 1 hour pre-sunset defined by within-subject meaningful change. [ Time Frame: Week 26 ]
- Change from baseline for total score in the domain of physical function in the PROMIS-57. [ Time Frame: Baseline (Week 0) and Week 26 ]Physical function: 1-5, where 5 is without any difficulty.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04402489
|Study Director:||Head of Medical Science||Mitsubishi Tanabe Pharma Development America, Inc.|