Oral Dersimelagon for Erythropoietic Protoporphyria

Recruiting at 61 trial locations

Overseen By Clinical Trials Information Desk, To prevent mis-communication,

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Mitsubishi Tanabe Pharma Development America, Inc.

Must not be taking: Opioids, Ketamine, Cannabis, others

Disqualifiers: Melanoma, Renal disease, Psychiatric disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 66 months

301 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new oral treatment called dersimelagon (also known as MT-7117) for individuals with erythropoietic protoporphyria (EPP), a condition causing severe skin pain when exposed to sunlight. The main goal is to determine if dersimelagon is safe for long-term use. Participants must have completed a previous trial involving the same treatment and should not have any other light-sensitive skin conditions. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants the opportunity to contribute to the potential availability of a new treatment.

Will I have to stop taking my current medications?

The trial requires stopping certain medications before participating. You must not use phototherapy, afamelanotide, cimetidine, or certain antioxidant agents within 3 to 4 weeks before starting. Additionally, you should avoid drugs metabolized by specific enzymes or proteins within 1 week before starting.

Is there any evidence suggesting that oral dersimelagon is likely to be safe for humans?

Research shows that dersimelagon, also known as MT-7117, is generally safe and well-tolerated by patients. Studies have found that people taking dersimelagon could spend more time in sunlight without experiencing symptoms, indicating its effectiveness and safety.

In one study, patients with erythropoietic protoporphyria—a condition causing severe skin pain when exposed to sunlight—reported they could stay in the sun longer without discomfort. This suggests the treatment effectively manages symptoms. The study also monitored negative side effects, and patients handled the medication well.

Another study examined the long-term safety of dersimelagon by including participants from earlier trials to ensure its continued safety. Although specific side effects weren't detailed, the absence of serious negative events suggests it is safe.

Overall, ongoing research supports that dersimelagon is safe for people, with few side effects reported. However, as with any medication, individual experiences may differ, so consulting a healthcare provider before joining a clinical trial is advisable. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Dersimelagon is unique because it is an oral treatment for erythropoietic protoporphyria (EPP), offering a new way to manage this photosensitivity condition. Unlike current treatments such as beta-carotene or narrowband UVB therapy, which focus on increasing skin tolerance to sunlight, Dersimelagon acts by modulating the melanin pathway, potentially reducing phototoxic reactions. Researchers are excited about its oral administration, which could simplify treatment regimens and improve patient adherence compared to more cumbersome light therapy or dietary supplements.

What evidence suggests that oral dersimelagon might be an effective treatment for erythropoietic protoporphyria?

Research has shown that dersimelagon, which participants in this trial will receive, can help people with erythropoietic protoporphyria (EPP) spend more time in sunlight without experiencing symptoms. One study found that patients taking dersimelagon stayed in the sun longer before symptoms appeared. This treatment is a pill that activates a skin receptor, helping to protect the skin from light damage. Specifically, patients in that study demonstrated promising results after 16 weeks of treatment. This evidence suggests that dersimelagon could effectively manage EPP symptoms. ^{1 2 6 7 8}

Who Is on the Research Team?

Head of Medical Science

Principal Investigator

Mitsubishi Tanabe Pharma America Inc.

Are You a Good Fit for This Trial?

This trial is for individuals with Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP), weighing at least 30 kg, able to travel for visits, and not pregnant. They must have completed a previous MT-7117 study and agree to use two effective contraception methods if of childbearing potential.

Inclusion Criteria

I am not breastfeeding and have a negative pregnancy test.

Subjects who complete MT-7117-G01 (complete through Week 58 [Visit 12])

I am a woman, not breastfeeding, and my pregnancy test before starting the study drug was negative.

Exclusion Criteria

Your liver tests show high levels of AST, ALT, ALP, or bilirubin.

I have had melanoma in the past.

I have melanoma or lesions that might be melanoma.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive oral dersimelagon to evaluate long-term safety and tolerability

66 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants continue to receive oral dersimelagon to assess long-term safety and tolerability

66 months

What Are the Treatments Tested in This Trial?

Interventions

• MT-7117

Trial Overview The trial is testing the long-term safety and tolerability of an oral medication called Dersimelagon (MT-7117) in patients with EPP or XLP who have previously participated in the initial MT-7117-G01 study.

How Is the Trial Designed?

Group I: Dersimelagon 200mg Experimental Treatment 1 Intervention

MT-7117 is already approved in European Union, United States for the following indications:

Approved in European Union as Dersimelagon for:

• Erythropoietic Protoporphyria (EPP)
• X-Linked Protoporphyria (XLP)

Approved in United States as Dersimelagon for:

• Erythropoietic Protoporphyria (EPP)
• X-Linked Protoporphyria (XLP)

Who Is Running the Clinical Trial?

Mitsubishi Tanabe Pharma Development America, Inc.

Lead Sponsor

Trials

22

Recruited

2,600+

Mitsubishi Tanabe Pharma America Inc.

Lead Sponsor

Trials

27

Recruited

3,300+

Published Research Related to This Trial

Dersimelagon, a new oral medication for conditions like erythropoietic protoporphyria, showed rapid absorption and a 97% relative oral bioavailability for the 100-mg tablets compared to the 50-mg tablets in a study involving 50 healthy participants.

The study found that while a high-fat meal did not affect the overall exposure of dersimelagon, it increased the peak concentration (Cmax) by 22%, indicating that food intake can influence the drug's absorption.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The Oral Bioavailability and Effect of Various Gastric Conditions on the Pharmacokinetics of Dersimelagon in Healthy Adult Volunteers. Ogawa, K., Ide, R., Belongie, K., et al.[2023]

Dersimelagon (MT-7117) is being evaluated as a new treatment for Erythropoietic protoporphyria (EPP) in a phase III clinical trial, which is currently ongoing in the US and Europe.

The authors argue that using a placebo in this trial is ethically questionable due to the existence of an approved treatment for EPP, suggesting that a noninferiority active-control trial would be a more appropriate design to assess the efficacy of dersimelagon.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Current trials in erythropoietic protoporphyria: are placebo controls ethical? Barman-Aksözen, J., Andreoletti, M., Blasimme, A.[2023]

Dersimelagon, an investigational oral medication for erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP), was found to be generally well tolerated in a phase 1 study involving healthy participants, with common side effects including skin hyperpigmentation.

The pharmacokinetics of dersimelagon showed a consistent profile across different age and race groups, with significant increases in melanin density observed after multiple doses, suggesting potential efficacy that needs further exploration in larger studies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Results from a first-in-human study of dersimelagon, an investigational oral selective MC1R agonist. Ogasawara, A., Ogawa, K., Ide, R., et al.[2023]

In a phase 2 trial involving 102 patients with erythropoietic protoporphyria or X-linked protoporphyria, the melanocortin 1 receptor agonist dersimelagon significantly increased the time to the first symptoms associated with sunlight exposure, with improvements of 53.8 minutes at 100 mg and 62.5 minutes at 300 mg compared to placebo.

Dersimelagon also appeared to enhance quality of life for patients, although common side effects included nausea, freckles, headache, and skin hyperpigmentation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Dersimelagon in Erythropoietic Protoporphyrias. Balwani, M., Bonkovsky, HL., Levy, C., et al.[2023]

Dersimelagon is a novel oral medication that shows rapid absorption and elimination in humans, with a median time to peak concentration of 2 hours, indicating efficient processing by the body.

The drug is primarily excreted through feces rather than urine, with negligible retention in the body, suggesting a favorable safety profile for continued development in treating conditions like erythropoietic protoporphyria and systemic sclerosis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Absorption, metabolism, and excretion of [14 C]dersimelagon, an investigational oral selective melanocortin 1 receptor agonist, in preclinical species and healthy volunteers. Tsuda, M., Ogawa, K., Endou, T., et al.[2023]

Citations

1. nejm.org nejm.org/doi/full/10.1056/NEJMoa2208754

Dersimelagon in Erythropoietic Protoporphyrias Dersimelagon significantly increased the duration of symptom-free sunlight exposure in patients with erythropoietic protoporphyria or X-linked protoporphyria.

2. clinicaltrials.gov clinicaltrials.gov/study/NCT03520036

NCT03520036 | Study to Evaluate Efficacy, Safety, and ... The purpose of this study is to investigate the efficacy and safety of MT-7117 on sunlight exposure duration without symptoms and tolerance in subjects with EPP ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10229459/

Results from a first-in-human study of dersimelagon, an ... ... protoporphyria: phase 2 clinical trial results evaluating the safety and effectiveness of dersimelagon (MT-7117), an oral MC1R agonist.

4. ashpublications.org ashpublications.org/blood/article/136/Supplement%201/51/472215/Erythropoietic-Protoporphyria-Phase-2-Clinical

Erythropoietic Protoporphyria: Phase 2 Clinical Trial Results ... The oral, MC1R agonist dersimelagon was efficacious after 16 weeks of treatment in increasing symptom-free light exposure in patients with EPP or XLP at doses ...

5. mt-pharma-america.com mt-pharma-america.com/media/press-releases/2024/first-patient-enrolled-in-mtpa-inspire-study-of-investigational-dersimelagon-for-epp-and-xlp

First Patient Enrolled in MTPA INSPIRE Study ... INSPIRE is a phase 3 clinical study investigating the efficacy, safety, and tolerability of dersimelagon (MT-7117), in adults and adolescents ...

6. sciencedirect.com sciencedirect.com/science/article/pii/S0006497118717588

Erythropoietic Protoporphyria: Phase 2 Clinical Trial ... Here we report the safety and effectiveness of Dersimelagon (MT-7117), a novel, orally-administered, small molecule, selective melanocortin-1 ...

7. mt-pharma-america.com mt-pharma-america.com/media/press-releases/2025/mitsubishi-tanabe-pharma-america-announces-completion-of-enrollment-for-its-phase-3-inspire-study-of-investigational-dersimelagon-in-patients-with-epp-and-xlp

Individual Press Releases - Mitsubishi Tanabe Pharma ... Another open-label extension study will continue to evaluate the long-term safety and tolerability of dersimelagon and enroll participants from ...

8. clinicaltrials.gov clinicaltrials.gov/study/NCT05005975

NCT05005975 | Extension Study to Evaluate Safety and ... Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked ...