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NCT05230459RECRUITINGIRB Ready

A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)

Sponsored by AskBio Inc

Quick Self-Assessment

See if you qualify and express your interest

Preparing your pre-screening questions...
๐Ÿ“Š
1 of 10 participants interested
10% interest

Express Your Interest Today

Interested in learning more? Complete the form below.

Secure & Confidential

Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Advance Medical Science

    Contribute to groundbreaking research

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel (varies by study)

  • Help Others

    Benefit future patients

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3In-person assessment if eligible
  4. 4Begin participation

About This Study

The purpose of this study is to evaluate the safety and tolerability of a single intravenous infusion of AB-1003 in adults diagnosed with limb girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). Participants will be treated in sequential, dose-level cohorts.

Who Can Participate

Inclusion Criteria

Male and female subjects aged 18 and 65 years with clinical diagnosis of LGMD2I/R9 and confirmation of FKRP gene mutation. 2. Ability to ascend 4 stairs between 2.5 and 10 seconds. 3. Ability to walk/run 10 meters in \<30 seconds. 4. Able to understand and comply with all study procedures. 5. Sexually active females of childbearing potential and female and male partners of male subjects receiving study intervention must use a barrier method of contraception for the first 6 months after dosing.

Exclusion Criteria

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. 2. Contraindication to MRI or hypersensitivity to contrast dyes, shellfish or iodine. 3. Implanted spinal rods, cardiac pacema

โ“ Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility during the screening process.

Study Details

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Study Type

INTERVENTIONAL

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Target Enrollment

10 participants

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Age Range

18 Years - 65 Years

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Gender

ALL

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Sponsor

AskBio Inc

Study Locations

Choose your preferred location or select flexible during enrollment

RECRUITING

Irvine

California

Location available
View Irvine location page
RECRUITING

Iowa City

Iowa

Location available
View Iowa City location page
RECRUITING

Kansas City

Kansas

Location available
View Kansas City location page
RECRUITING

Baltimore

Maryland

Location available
View Baltimore location page
RECRUITING

Richmond

Virginia

Location available
View Richmond location page
RECRUITING

Seattle

Washington

Location available
View Seattle location page

Frequently Asked Questions

Q:What is a clinical trial?

A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.

Q:Will I be compensated?

Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.

Q:Will I receive a placebo instead of treatment?

When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.

Q:Can I leave a trial if I change my mind?

Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.

Q:How long does a clinical trial last?

Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.

Still have questions? Our study coordinators are here to help.

Ready to Make a Difference?

Express your interest in this groundbreaking clinical trial

๐Ÿ”’ Secure ยท ๐Ÿฅ Expert Care

Looking for LGMD2I Treatment in Irvine?

Join others in California exploring innovative treatment options through clinical research

LGMD2I Treatment Options in Irvine, California

If you're searching for LGMD2I treatment in Irvine, participating in a clinical research study may provide access to innovative approaches under expert medical supervision. This study is actively recruiting participants in Irvine, Iowa City, Kansas City and surrounding areas.

Clinical trials offer participants the opportunity to receive cutting-edge treatments while contributing to medical research that may help future patients with LGMD2I. All study-related care is provided at no cost to participants.

Local Sites
3 locations in California
Now Enrolling
Up to 10 participants
Quick Start
Screening available now

Why Consider a Clinical Trial for LGMD2I?

Potential Benefits

  • โœ“Access to new treatment approaches before public availability
  • โœ“Close monitoring by experienced medical professionals
  • โœ“Study-related care provided at no cost
  • โœ“Contribute to medical research for LGMD2I

What to Expect

  • โ†’Initial screening to determine eligibility
  • โ†’Regular check-ups and monitoring visits
  • โ†’Possible compensation for time and travel
  • โ†’You can withdraw at any time

Frequently Asked Questions About This LGMD2I Study

Important Clinical Trial Information

This information is provided for educational purposes and does not constitute medical advice. Clinical trial participation involves potential risks and benefits. Eligibility requirements apply and will be assessed during the screening process.

Study identifier: NCT05230459. For complete study details, visit ClinicalTrials.gov. Always consult with your healthcare provider before making decisions about your medical care or participating in clinical research.