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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT05230459
Other study ID # LION-CS101
Secondary ID
Status Recruiting
Phase Phase 1/Phase 2
First received
Last updated
Start date March 12, 2023
Est. completion date December 2028

Study information

Verified date April 2024
Source Asklepios Biopharmaceutical, Inc.
Contact Medical Affairs at AskBio
Phone 919-561-6210
Email AskFirst@askbio.com
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

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)


Recruitment information / eligibility

Status Recruiting
Enrollment 10
Est. completion date December 2028
Est. primary completion date December 2028
Accepts healthy volunteers No
Gender All
Age group 18 Years to 65 Years
Eligibility Inclusion 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 AB-1003 must use a barrier method of contraception for the first 6 months after dosing. Exclusion Criteria: 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 chronic liver disease (e.g. hepatitis, HIV, steatosis) or abnormal liver function (abnormal GGT and/or abnormal total/direct bilirubin and/or AST and ALT >2 ULN). 5. Abnormal renal function (GFR < 60 ml/min, using the MDRD 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 baseline 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.

Study Design


Related Conditions & MeSH terms


Intervention

Genetic:
AB-1003 dose level 1
Single intravenous infusion of AB-1003 gene therapy at dose level 1
AB-1003 dose level 2
Single intravenous infusion of AB-1003 gene therapy at dose level 2
Other:
Placebo
Single intravenous infusion of Placebo

Locations

Country Name City State
United States Kennedy Krieger Institute Baltimore Maryland
United States University of Iowa Iowa City Iowa
United States University of California - Irvine Irvine California
United States University of Kansas Medical Center Kansas City Kansas
United States VCU Richmond Virginia
United States University of Washington Medical Center Seattle Washington

Sponsors (1)

Lead Sponsor Collaborator
Asklepios Biopharmaceutical, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Adverse Events Treatment Emergent Adverse Events, Serious Adverse Events, Dose Limiting Toxicity 0-52 weeks
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