What is the mechanism of action of this treatment?

Lymphoproliferative Syndrome (LPS) treatments often target specific pathways involved in the proliferation and survival of lymphocytes. Common treatments include Bruton tyrosine kinase (BTK) inhibitors, which block B-cell receptor signaling, and monoclonal antibodies like rituximab that target CD20 on B-cells, leading to their destruction. These treatments are crucial for LPS patients as they help control abnormal lymphocyte growth and reduce disease symptoms. While melanocortin receptor agonists like Dersimelagon are not directly mentioned for LPS, their role in modulating immune responses and inflammation could offer potential therapeutic benefits in managing immune dysregulation seen in LPS.

What side effects are associated with this type of intervention?

Common treatments for Lymphoproliferative Syndrome include chemotherapy, immunotherapy, and targeted therapies. Chemotherapy can cause side effects such as nausea, vomiting, hair loss, and increased risk of infections. Immunotherapy, including immune checkpoint inhibitors, may lead to immune-related adverse events like colitis, dermatitis, and endocrinopathies. Targeted therapies, such as kinase inhibitors, can result in side effects like hypertension, fatigue, and hand-foot syndrome. While specific data on Melanocortin Receptor Agonists like Dersimelagon are limited, they are generally studied for their safety and tolerability in other conditions.

What prior FDA approvals exist?

FDA-approved treatments for Lymphoproliferative Syndrome typically include immunosuppressive therapies and targeted treatments such as monoclonal antibodies. Examples include rituximab, a monoclonal antibody targeting CD20, and various chemotherapeutic agents. While dersimelagon is a melanocortin receptor agonist, there are no direct FDA-approved treatments for Lymphoproliferative Syndrome that function through this mechanism. Therefore, the focus remains on immunosuppressive and targeted therapies.

What other types of research exist?

Promising novel alternatives to Dersimelagon for Lymphoproliferative Syndrome patients in 2024 may include immune checkpoint inhibitors like pembrolizumab and nivolumab, as well as monoclonal antibodies targeting PD-1/PD-L1 pathways. These therapies have shown efficacy in various lymphoproliferative and immune-related conditions and are being actively studied in clinical trials.

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Oral Dersimelagon for Erythropoietic Protoporphyria

Phase 3

Recruiting

Research Sponsored by Mitsubishi Tanabe Pharma Development America, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Female subjects who are non-lactating and have a negative urine pregnancy test at baseline visit prior to receiving the first dose of study drug.

6. Female subjects who are non-lactating and have a negative urine pregnancy test at baseline visit prior to receiving the first dose of study drug.

Must not have

Treatment with phototherapy or afamelanotide within 3 months before baseline (Visit 2).

History of melanoma.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 66 further months

Awards & highlights

Pivotal Trial

No Placebo-Only Group

Summary

This trial is testing a pill called dersimelagon to see if it is safe and tolerable for patients who need long-term treatment.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

While specific side effects are not listed here, participants will be monitored for any adverse reactions due to long-term use of Dersimelagon as it relates to their liver function, skin conditions including melanoma risk, alcohol intake effects, psychiatric health stability, and overall organ function.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am not breastfeeding and have a negative pregnancy test.

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

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My body weight is at least 30 kg.

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My body weight is at least 30 kg.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I haven't had phototherapy or taken afamelanotide in the last 3 months.

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I have had melanoma in the past.

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I have melanoma or lesions that might be melanoma.

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I am not pregnant, breastfeeding, nor planning to become pregnant during the study.

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I do not have any skin conditions caused by light, except for EPP or XLP.

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I haven't taken any experimental drugs recently, except dersimelagon.

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Two or more of my close family members have had melanoma.

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I have kidney problems or am on dialysis.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 66 further months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 66 further months

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 66 further months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Number of patients with Treatment emergent adverse events (TEAEs) (including serious adverse events [SAEs] and adverse events of special interest [AESIs]).

Number of patients with abnormal Physical examination data

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Dersimelagon 200mgExperimental Treatment1 Intervention

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

MT-7117

2021

Completed Phase 2

~470

0 / 12Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Lymphoproliferative Syndrome (LPS) treatments often target specific pathways involved in the proliferation and survival of lymphocytes. Common treatments include Bruton tyrosine kinase (BTK) inhibitors, which block B-cell receptor signaling, and monoclonal antibodies like rituximab that target CD20 on B-cells, leading to their destruction. These treatments are crucial for LPS patients as they help control abnormal lymphocyte growth and reduce disease symptoms. While melanocortin receptor agonists like Dersimelagon are not directly mentioned for LPS, their role in modulating immune responses and inflammation could offer potential therapeutic benefits in managing immune dysregulation seen in LPS.

[Staging and Treatment Response Evaluation in Malignant Lymphomas - Czech Lymphoma Study Group Recommendations According to Criteria Revised in 2014 (Lugano Classification)].