MT-7117-A-301

11 evidence topics · 3 sources

Objective

Primary objective

“To evaluate the long-term safety and tolerability of oral dersimelagon.” (opens the source at this quote in a new tab)

Location and study date

Study title, status, and dates

Registry field	Quoted record
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)”
Overall status	“Active, not recruiting”
Start date, actual	“2021-08-10”
Primary completion, estimated	“2027-12”
Enrollment, actual	“286”

Study design

Open-label single-group design

Design field	Quoted record
Allocation	“na”
Intervention model	“Single group”
Masking	“None”
Phase	“Phase 3”

Relationship to the other trials in the program

“Another open-label extension study will continue to evaluate the long-term safety and tolerability of dersimelagon and enroll participants from the INSPIRE study and participants who have completed the study MT-7117-G01, if interested.” (opens the source at this quote in a new tab)

Eligibility criteria

Rollover eligibility from the phase 3 trials

“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.” (opens the source at this quote in a new tab)

Hepatic and dermatologic exclusions

“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.” (opens the source at this quote in a new tab)
“5. History of melanoma.” (opens the source at this quote in a new tab)

Treatment

Dose and route

“Dersimelagon 200mg” (opens the source at this quote in a new tab)

Study outcomes

Rationale for using treatment-emergent adverse events as the primary endpoint

Safety outcome measures and observation period

“Number of patients with Treatment emergent adverse events (TEAEs) (including serious adverse events [SAEs] and adverse events of special interest [AESIs]).” (opens the source at this quote in a new tab)
“Number of patients with Nevi appearance” (opens the source at this quote in a new tab)
“up to 66 further months” (opens the source at this quote in a new tab)

Absence of efficacy endpoints

“E.6.4 Safety Yes E.6.5 Efficacy No” (opens the source at this quote in a new tab)

Statistical analysis description

Number of participants (Planned and analyzed)

Participants enrolled

Registry field	Quoted record
Enrollment, actual	“286”

Description of analysis sets

No evidence found.

Results

Participant disposition

No evidence found.

Baseline characteristics

No evidence found.

Efficacy results

Not applicable.

Health-related quality of life and patient-reported outcomes

Not applicable.

Safety results

No evidence found.

Study limitations

Summary: status of the long-term extension

The study is open-label and single-group, so it provides no controlled comparison. It remains ongoing with estimated completion in December 2027, and no results have been posted to ClinicalTrials.gov or the EU Clinical Trials Register. Enrollment is restricted to participants who completed one of the two phase 3 trials or a previous period of the extension itself, which excludes participants from the phase 2 trial and selects for people who tolerated and continued earlier treatment.