The classic website will no longer be available as of June 25, 2024. Please use the modernized ClinicalTrials.gov.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 1 for:    LION-101
Previous Study | Return to List | Next Study

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05230459
Recruitment Status : Recruiting
First Posted : February 9, 2022
Last Update Posted : April 3, 2024
Sponsor:
Information provided by (Responsible Party):
Asklepios Biopharmaceutical, Inc.

Study Description
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Brief 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)

Condition or disease Intervention/treatment Phase
Limb Girdle Muscular Dystrophy Limb-Girdle Muscular Dystrophy Type 2 LGMD2I Muscular Dystrophy LGMD2 LGMD FKRP FKRP Mutation Fukutin Related Protein Genetic: AB-1003 dose level 1 Genetic: AB-1003 dose level 2 Other: Placebo Phase 1 Phase 2

Study Design
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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)
Actual Study Start Date : March 12, 2023
Estimated Primary Completion Date : December 2028
Estimated Study Completion Date : December 2028

Resource links provided by the National Library of Medicine


Arms and Interventions
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Arm Intervention/treatment
Experimental: AB-1003 Cohort 1 Genetic: AB-1003 dose level 1
Single intravenous infusion of AB-1003 gene therapy at dose level 1
Experimental: AB-1003 Cohort 2 Genetic: AB-1003 dose level 2
Single intravenous infusion of AB-1003 gene therapy at dose level 2
Placebo Comparator: Placebo (Cohorts 1 and 2) Other: Placebo
Single intravenous infusion of Placebo


Outcome Measures
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Primary Outcome Measures :
  1. Adverse Events [ Time Frame: 0-52 weeks ]
    Treatment Emergent Adverse Events, Serious Adverse Events, Dose Limiting Toxicity


Eligibility Criteria
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.
Contacts and Locations
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05230459


Contacts
Layout table for location contacts
Contact: Medical Affairs at AskBio 919-561-6210 AskFirst@askbio.com
Contact: myTomorrows (see link below in reference section)

Locations
Layout table for location information
United States, California
University of California - Irvine Recruiting
Irvine, California, United States, 92697
Contact: UCI Alpha Clinic       stemcell@uci.edu   
Principal Investigator: Tahseen Mozaffar, MD         
United States, Iowa
University of Iowa Recruiting
Iowa City, Iowa, United States, 52242
Contact: Ciara Gibbs       ciara-gibbs@uiowa.edu   
Principal Investigator: Katherine Mathews, MD         
United States, Kansas
University of Kansas Medical Center Recruiting
Kansas City, Kansas, United States, 66160
Contact: Andrea Klempnauer       atenney@kumc.edu   
Principal Investigator: Jeffrey Statland, MD         
United States, Maryland
Kennedy Krieger Institute Recruiting
Baltimore, Maryland, United States, 21205
Contact: Mary Yep       yep@kennedykrieger.org   
Principal Investigator: Doris Leung, MD, PhD         
United States, Virginia
VCU Recruiting
Richmond, Virginia, United States, 23298
Contact: Anarosa Rezeq       anarosa.rezeq@vcuhealth.org   
Principal Investigator: Nicholas E Johnson, MD         
United States, Washington
University of Washington Medical Center Recruiting
Seattle, Washington, United States, 98195
Contact: Mike Willis       mwillis5@uw.edu   
Principal Investigator: B. Jane Distad, MD         
Sponsors and Collaborators
Asklepios Biopharmaceutical, Inc.
More Information
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Additional Information:

Layout table for additonal information
Responsible Party: Asklepios Biopharmaceutical, Inc.
ClinicalTrials.gov Identifier: NCT05230459    
Other Study ID Numbers: LION-CS101
First Posted: February 9, 2022    Key Record Dates
Last Update Posted: April 3, 2024
Last Verified: April 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Asklepios Biopharmaceutical, Inc.:
gene therapy
LGMD2I
LGMD2I/R9
gene augmentation therapy
 FKRP
fukutin related protein
FKRP mutation
Additional relevant MeSH terms:
Layout table for MeSH terms
Muscular Dystrophies
Muscular Dystrophies, Limb-Girdle
Muscular Disorders, Atrophic
Muscular Diseases
 Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn