Phase 3 study of MT-7117 in patients with EPP or XLP

• Research type

  Research Study

• Full 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

• IRAS ID

  282256

• Contact name

  Lesley Rhodes

• Contact email

  lesley.e.rhodes@manchester.ac.uk

• Sponsor organisation

  Mitsubishi Tanabe Pharma Development America (MTDA), Inc.

• Eudract number

  2019-004226-16

• Clinicaltrials.gov Identifier

  NCT04402489

• Duration of Study in the UK

  1 years, 0 months, 1 days

• Research summary

  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 primary aim of the study is to test the effectiveness of dersimelagon in treating patients aged between 12 and 75 years old. In this Study, 2 out of every 3 patients will receive active medicine (66% chance) and 1 out of 3 will receive placebo (33% chance) in the form of an oral tablet.
  This phase 3 study is divided into separate parts. A screening phase, a 26 week double-blind treatment phase and a 6 week follow up period. Participants can also participate in an additional 26 week double blind extension period.
  During the study, patients will have 6 or 8 in clinic visits to assess eligibility, obtain consent, conduct blood/urine tests and electrocardiogram as well as 2-4 remote visits for additional blood sample collection. Patients will also need to complete electronic diaries to document their sun exposure.
  It is expected that 14 patients in total across 4 clinical centers in the United Kingdom will participate.

  Summary of Results:

  For the primary efficacy endpoint, 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 (Visit 7), MT-7117 200 mg did not demonstrate statistically significant improvement over placebo (LSMD 22.70 minutes, p = 0.116).
  However, clinical improvements were seen during the study. There was nominally significant improvement in the MT-7117 200 mg group compared with the placebo group at Week 16 and nominally significant improvements were observed in the MT-7117 200 mg group compared to the placebo group at most of the timepoints (Week 6, 8, 14, 16, 20, 22, and 24). The MT-7117 100 mg dose group showed numerical improvement over placebo during the 26-weeks treatment, although the difference was not nominally significant compared to placebo

  Short and long-term use of MT-7117 is well-tolerated and has a safety profile that supports use in patients with EPP/XLP.

• REC name

  South Central - Berkshire Research Ethics Committee

• REC reference

  20/SC/0331

• Date of REC Opinion

  4 Nov 2020

• REC opinion

  Further Information Favourable Opinion