What side effects are associated with this type of intervention?

Common treatments for Facioscapulohumeral Muscular Dystrophy (FSHD) include corticosteroids, physical therapy, and emerging treatments like selective p38 MAP kinase inhibitors such as Losmapimod. Known side effects of corticosteroids can include weight gain, osteoporosis, hypertension, and increased risk of infections. For selective p38 MAP kinase inhibitors like Losmapimod, side effects observed in clinical trials include gastrointestinal issues, liver enzyme elevations, and potential cardiovascular risks. It is important to discuss these potential side effects with a healthcare provider to tailor the treatment plan to the patient's specific needs.

What prior FDA approvals exist?

As of now, there are no FDA-approved treatments specifically for Facioscapulohumeral Muscular Dystrophy (FSHD). Losmapimod, a selective p38 MAP kinase inhibitor, is currently being studied for its efficacy and safety in treating FSHD. This trial represents a novel approach, as there are no existing approved drugs targeting the same pathway for this condition.

What other types of research exist?

As of now, there are no specific novel alternatives to Losmapimod mentioned in the provided context for the treatment of Facioscapulohumeral Muscular Dystrophy (FSHD). Patients should consult with their healthcare provider to discuss ongoing clinical trials and emerging therapies that may be suitable for their condition.

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MAP Kinase Inhibitor

Losmapimod for Facioscapulohumeral Muscular Dystrophy

Phase 3

Waitlist Available

Research Sponsored by Fulcrum Therapeutics

Eligibility Criteria Checklist

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

Must have

Participants must be between 18 and 65 years of age, inclusive

Genetically confirmed diagnosis of FSHD 1 or FSHD 2

Must not have

Known active opportunistic or life-threatening infections including Human Immunodeficiency virus (HIV) and hepatitis B or C

Participants who are on drug(s) or supplements that may affect muscle function, as determined by the Investigator: participants must be on a stable dose of that drug(s) or supplement for at least 3 months prior to the first dose of study drug and remain on that stable dose for the duration of the study

Timeline

Screening 3 weeks

Treatment Varies

Follow Up at week 48

Awards & highlights

Pivotal Trial

Summary

This trial is testing the safety and effectiveness of a drug called losmapimod for people with a muscle disease called Facioscapulohumeral Muscular Dystrophy (FSHD). Participants will take the drug regularly, and those who complete the initial phase can continue taking it to see how it works over a longer duration. The goal is to see if losmapimod can help reduce muscle inflammation and slow down muscle weakening.

Who is the study for?

Adults aged 18-65 with genetically confirmed Facioscapulohumeral Muscular Dystrophy (FSHD1 or FSHD2) and a specific range of muscle strength loss can join. They must not be wheelchair-dependent, have no MRI contraindications, and cannot be on certain drugs affecting muscle function unless stable for 3 months.

What is being tested?

The trial is testing Losmapimod's effectiveness in treating FSHD by comparing it to a placebo. Participants will take the drug or placebo twice daily. After this phase, they may continue in an extension study to assess long-term effects.

What are the potential side effects?

Potential side effects are not explicitly listed but could include reactions related to liver or kidney health due to exclusion criteria regarding these organs' conditions.

Eligibility Criteria

Inclusion Criteria

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

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I am between 18 and 65 years old.

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I have a genetic diagnosis of FSHD type 1 or 2.

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I can walk without needing a wheelchair or walker.

Exclusion Criteria

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

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I do not have HIV, hepatitis B or C, or any severe infections.

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I have been on a stable dose of medication or supplements that affect muscle function for at least 3 months.

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I have severe kidney problems.

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I have a heart rhythm problem that needs medication.

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I am not taking any medications that affect certain liver enzymes or kidney transporters while on losmapimod.

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I have been diagnosed with tuberculosis, whether active or not.

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I have a history of liver disease.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ at week 48

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~at week 48

This trial's timeline: 3 weeks for screening, Varies for treatment, and at week 48 for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Part A: Change from Baseline in total Relative surface area (RSA) Quadrants 1 to 5 (Q1-Q5) with 500 grams (g) wrist weight averaged over both arms as assessed by Reachable workspace (RWS) at Week 48

Secondary study objectives

Part A: Change from Baseline in Quality of Life in Neurologic Disorders upper extremity (Neuro-QoL UE) Scale at Week 48

Part A: Change from Baseline in Whole body (WB) longitudinal composite Muscle Fat Infiltration (MFI) of B muscles at Week 48

Part A: Change from Baseline in average shoulder abductor strength by hand-held quantitative dynamometry at Week 48

+1 more

Side effects data

From 2016 Phase 2 trial • 17 Patients • NCT02000440

29%

Headache

24%

Fatigue

18%

Vomiting

18%

Muscle spasms

18%

Rash

18%

Oropharyngeal pain

18%

Blood creatinine increased

18%

Nausea

18%

Dizziness

12%

Oedema peripheral

12%

Abdominal pain

12%

Dyspepsia

12%

Blood pressure increased

12%

Upper respiratory tract infection

Bronchitis

Dizziness postural

Sinusitis

Nasal congestion

Anaemia

Skin papilloma

Breast pain

Vitamin D decreased

Back pain

Decreased appetite

Hyperglycaemia

Animal bite

Contusion

Confusional state

Proteinuria

Joint stiffness

Hyperhidrosis

Musculoskeletal stiffness

Gout

Dysuria

Multiple allergies

Eosinophils urine

Abdominal pain upper

Diarrhoea

Presyncope

Localised oedema

Ear infection

Gastroenteritis viral

Nasopharyngitis

Pneumonia

Respiratory tract infection viral

Urinary tract infection

Asthma

Cough

Epistaxis

Blood urea increased

Cystatin C increased

Flatulence

Chest discomfort

Palpitations

Ear pain

Pyrexia

Swelling

Anxiety

Blood albumin increased

100%

80%

60%

40%

20%

Study treatment Arm

Losmapimod 7.5 mg BID / 15 mg BID

Awards & Highlights

Pivotal Trial

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

Trial Design

3Treatment groups

Experimental Treatment

Placebo Group

Group I: Part B: Open-label extensionExperimental Treatment1 Intervention

Participants will receive losmapimod, upon completion of all assessments for Part A.

Group II: Part A: Placebo-controlled treatment period: LosmapimodExperimental Treatment1 Intervention

Participants will be randomized to receive losmapimod.

Group III: Part A: Placebo-controlled treatment period: PlaceboPlacebo Group1 Intervention

Participants will be randomized to receive placebo

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Losmapimod

2012

Completed Phase 2

~150

0 / 10Eligibility 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

Losmapimod, a selective p38 MAP kinase inhibitor, works by blocking the p38 MAP kinase pathway, which is involved in inflammatory responses and muscle degeneration. This mechanism is particularly relevant for patients with Facioscapulohumeral Muscular Dystrophy (FSHD) because the disease is characterized by progressive muscle weakness and inflammation. By inhibiting this pathway, Losmapimod aims to reduce inflammation and muscle damage, potentially slowing disease progression and improving muscle function. This approach is significant for FSHD patients as it targets the underlying molecular processes contributing to their symptoms.