RECRUITING

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

Conditions

Limb Girdle Muscular Dystrophy Limb-Girdle Muscular Dystrophy Type 2 LGMD2I Muscular Dystrophy LGMD2 LGMD FKRP FKRP Mutation Fukutin Related Protein gene therapy LGMD2I/R9 gene augmentation therapy

Quick Navigation

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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. (Part 1)

Official Title

A Two-part Multicenter Study: a Randomized, Double-blind, Placebo-controlled Dose-escalation Safety Phase (Part 1) Followed by Double-blind, Placebo-controlled, Adaptive Phase (Part 2) Study to Evaluate the Safety and Efficacy of AB-1003 in Adult Subjects With LGMD2I/R9 Mutations in the Gene Encoding Fukutin Related Protein (FKRP)

Quick Facts

Study Start:2023-05-15

Study Completion:2028-12

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT05230459

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years to 65 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
1. 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.	1. 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 pacemaker or other implantation that would distort cardiac MRI images. 4. History of active, ongoing chronic liver disease (e.g. hepatitis, HIV-related liver disease, hemochromatosis, steatosis, etc.) or abnormal liver function tests (abnormal GGT and/or abnormal total/direct bilirubin \>upper limit of normal \[ULN\] and/or elevated AST and ALT \>2 ULN). 5. Abnormal renal function (GFR \<60 ml/min, using the Modification of Diet in Renal Disease equation). 6. Any life-threatening disease, including malignant neoplasms and medical history or malignant neoplasms within the past 5 years prior to screening (except basal and squamous cell skin cancer). 7. In the opinion of the investigator, a pre-existing medical condition that predisposes the subject to risks that outweighs the potential benefits. 8. Requirement for daytime ventilatory support. 9. Change in glucocorticosteroid treatment within 3 months prior to screening visit. 10. Exposure to another investigational drug within 3 months prior to study treatment or any previous treatment with gene therapy. 11. Ongoing participation in any other therapeutic clinical trial. 12. Neutralizing antibody titer to AAV9 ≥1:5. 13. Female subjects who are pregnant, plan to become pregnant in the next 12 months, or breastfeeding.

Inclusion Criteria

Exclusion Criteria

1. 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.

1. 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 pacemaker or other implantation that would distort cardiac MRI images.
4. History of active, ongoing chronic liver disease (e.g. hepatitis, HIV-related liver disease, hemochromatosis, steatosis, etc.) or abnormal liver function tests (abnormal GGT and/or abnormal total/direct bilirubin \>upper limit of normal \[ULN\] and/or elevated AST and ALT \>2 ULN).
5. Abnormal renal function (GFR \<60 ml/min, using the Modification of Diet in Renal Disease equation).
6. Any life-threatening disease, including malignant neoplasms and medical history or malignant neoplasms within the past 5 years prior to screening (except basal and squamous cell skin cancer).
7. In the opinion of the investigator, a pre-existing medical condition that predisposes the subject to risks that outweighs the potential benefits.
8. Requirement for daytime ventilatory support.
9. Change in glucocorticosteroid treatment within 3 months prior to screening visit.
10. Exposure to another investigational drug within 3 months prior to study treatment or any previous treatment with gene therapy.
11. Ongoing participation in any other therapeutic clinical trial.
12. Neutralizing antibody titer to AAV9 ≥1:5.
13. Female subjects who are pregnant, plan to become pregnant in the next 12 months, or breastfeeding.

Contacts and Locations

Study Contact

Medical Affairs at AskBio

CONTACT

919-561-6210

AskFirst@askbio.com

myTomorrows (see link below in reference section)

CONTACT

Study Locations (Sites)

University of California - Irvine

Irvine, California, 92697

United States

University of Iowa

Iowa City, Iowa, 52242

United States

University of Kansas Medical Center

Kansas City, Kansas, 66160

United States

Kennedy Krieger Institute

Baltimore, Maryland, 21205

United States

VCU

Richmond, Virginia, 23298

United States

University of Washington Medical Center

Seattle, Washington, 98195

United States

Collaborators and Investigators

Sponsor: Asklepios Biopharmaceutical, Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-05-15

Study Completion Date2028-12

Study Record Updates

Study Start Date2023-05-15

Study Completion Date2028-12

Terms related to this study

Keywords Provided by Researchers

gene therapy
LGMD2I
LGMD2I/R9
gene augmentation therapy
FKRP
fukutin related protein
FKRP mutation

Additional Relevant MeSH Terms

Limb Girdle Muscular Dystrophy
Limb-Girdle Muscular Dystrophy Type 2
LGMD2I
Muscular Dystrophy
LGMD2
LGMD
FKRP
FKRP Mutation
Fukutin Related Protein