What side effects are associated with this type of intervention?

Common treatments for Limb-Girdle Muscular Dystrophy (LGMD) that target genetic or molecular pathology, such as gene therapy or antisense oligonucleotides, can have side effects including immune reactions (fever, chills, severe immune responses), off-target effects (muscle weakness, fatigue), and potential long-term risks like organ toxicity or malignancy. These treatments aim to correct or mitigate the genetic defects causing the disease but require careful monitoring for adverse effects.

What prior FDA approvals exist?

As of now, there are no specific FDA-approved treatments for Limb-Girdle Muscular Dystrophy (LGMD) that target the genetic or molecular pathology of the disease, such as LGMD2I/R9. However, investigational therapies like AB-1003, which involve gene therapy or molecular approaches, are being studied to address the underlying genetic defects. These approaches are similar to treatments being explored for other muscular dystrophies, such as eteplirsen for Duchenne Muscular Dystrophy, which aims to increase dystrophin production through exon skipping. The field is actively researching and developing new therapies that target the genetic causes of these conditions.

What other types of research exist?

Promising novel alternatives to AB-1003 for LGMD2I/R9 patients include gene therapies such as SRP-9003, which targets the genetic mutations causing the disease. Additionally, CRISPR-Cas9 based gene editing therapies are being explored for their potential to correct genetic defects at the DNA level. Other molecular therapies, such as antisense oligonucleotides, are also under investigation for their ability to modulate gene expression and improve muscle function. Patients should discuss these options with their healthcare provider to determine the most appropriate treatment based on the latest research and clinical trial availability.

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Gene Therapy

LION-101 for Limb-Girdle Muscular Dystrophy

Phase 1 & 2

Recruiting

Research Sponsored by Asklepios Biopharmaceutical, Inc.

Eligibility Criteria Checklist

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

Must have

Ability to walk/run 10 meters in < 30 seconds

Male and female subjects aged 18 and 65 years with clinical diagnosis of LGMD2I/R9 and confirmation of FKRP gene mutation

Must not have

Requirement for daytime ventilatory support

Significant cardiomyopathy as defined by echocardiogram (left ventricular ejection fraction <40%), evidence of conduction defect (increased PR and RR intervals, left bundle branch block and QTcF >480m/sec), NYHA Class 3 or 4 heart failure, or MRI gadolinium enhancement evidence of clinically important myocardial fibrosis

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 0-52 weeks

Summary

This trial is testing the safety and tolerability of an IV dose of AB-1003 in adults with a specific type of muscular dystrophy (LGMD2I/R9). The goal is to see if the treatment can help improve muscle function or slow down muscle damage.

Who is the study for?

This trial is for adults aged 18-65 with a confirmed diagnosis of LGMD2I/R9 due to FKRP gene mutation. Participants should be able to climb stairs and walk 10 meters within specific times, understand study procedures, and use barrier contraception if sexually active. Exclusions include significant heart issues, MRI contraindications, other clinical trial participation, certain antibody levels, pregnancy or breastfeeding women, liver/renal diseases, cancer history in the last 5 years (except some skin cancers), and those needing ventilatory support.

What is being tested?

The study tests the safety of LION-101 in treating limb girdle muscular dystrophy type 2I/R9 through a single intravenous infusion. It's conducted in sequential groups receiving different doses of LION-101 versus placebo to determine tolerability.

What are the potential side effects?

While not explicitly listed here, potential side effects may include reactions at the infusion site such as pain or swelling; general symptoms like fever or chills; allergic reactions; and possibly effects on muscle tissue where the drug acts.

Eligibility Criteria

Inclusion Criteria

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

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I can walk or run 10 meters in less than 30 seconds.

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I am between 18 and 65 years old and have been diagnosed with LGMD2I/R9 with a confirmed FKRP gene mutation.

Exclusion Criteria

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

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I need help with breathing during the day.

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I have serious heart issues, including weak heart pumping or specific heart rhythm problems.

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My kidney function is reduced.

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I have a history of liver disease or currently have abnormal liver function.

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I haven't had any life-threatening diseases or cancer, except for skin cancer, in the last 5 years.

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I haven't taken any experimental drugs or had gene therapy recently.

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I am not pregnant, planning to become pregnant within a year, or breastfeeding.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 0-52 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~0-52 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 0-52 weeks for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Adverse Events

Trial Design

3Treatment groups

Experimental Treatment

Placebo Group

Group I: AB-1003 Cohort 2Experimental Treatment1 Intervention

Group II: AB-1003 Cohort 1Experimental Treatment1 Intervention

Group III: Placebo (Cohorts 1 and 2)Placebo Group1 Intervention

0 / 9Eligibility 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

The most common treatments for Limb-Girdle Muscular Dystrophy (LGMD), particularly those targeting genetic or molecular pathology like AB-1003, often involve gene therapy and molecular interventions. These treatments aim to correct or compensate for the defective genes responsible for the disease. For instance, gene therapy may introduce a functional copy of the mutated gene, while molecular therapies might enhance the expression of compensatory proteins or inhibit pathways that lead to muscle degradation. These approaches are crucial for LGMD patients as they address the root cause of muscle weakness and degeneration, potentially improving muscle function and slowing disease progression.

Pharmacologic and genetic therapy for childhood muscular dystrophies.