A Phase 3 Extension study of MT-7117 in patients with EPP or XLP
Research type
Research Study
Full 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)
IRAS ID
305714
Contact name
Lesley Rhodes
Contact email
Sponsor organisation
Mitsubishi Tanabe Pharma Development America (MTDA), Inc.
Eudract number
2021-001831-17
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 9 months, 1 days
Research summary
Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP) is a genetic metabolic disorder which is characterised by the accumulation of a molecule named protoporphyrin in the body, which leads to pain when skin is exposed to sunlight. This study is looking at a new experimental drug MT-7117 (dersimelagon) for the treatment of EPP or XLP in adults and adolescents.
EPP and XLP are primarily managed by a preventative approach, which consists of avoidance of sun exposure and the use of adequate clothing and sunscreens. Until recently there was no standard of care for treatment of EPP in adults and there is currently an unmet medical need for new effective and safe, orally administered treatments, including paediatric patients as they have currently no treatment options.
The aim of this extension study is to offer the patients who completed the MT-7117-G01 study including the double blinded extension period the opportunity to continue to be treated with dersimelagon for up to a maximum 24 further months or until the drug is commercially available in the first country, whichever time period is shorter.This open-label, long-term extension study is divided into three periods: Screening, an Open-label Treatment Period lasting up to 104 weeks (24 months) and a Follow-up Period, which comes 6 weeks after the End of Trial (EoT) or Early Termination Visit.
Open label means that both the investigators (Doctors) and participants are aware of the drug or treatment given.
Participants will have 10 in-clinic visits. 4 of the study visits can be done remotely at the participants home (a mobile nurse will come to them) or at an alternative convenient location for them.
Participants will be dispensed study drug at clinic visit 2(day 1), visit 3(week 12) and visit 5(week 60).
It is expected that 13 patients in total across 2 clinical centers in the United Kingdom will participate.
REC name
Wales REC 2
REC reference
22/WA/0041
Date of REC Opinion
15 Feb 2022
REC opinion
Favourable Opinion