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A Study to Evaluate the Safety of 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 : May 23, 2023
Sponsor:
Information provided by (Responsible Party):
Asklepios Biopharmaceutical, Inc.

Study Description
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Brief Summary:
The purpose of this study is to evaluate the safety and tolerability of a single intravenous infusion of LION-101 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: LION-101 dose level 1 Genetic: LION-101 dose level 2 Other: Placebo Phase 1 Phase 2

Study Design
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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 Phase 1/2, Double-blind, Randomized, Placebo-controlled, Dose-escalation Study to Evaluate the Safety of LION-101 Gene Therapy in Adult Subjects (18-65 Years) With Genetic Confirmation of Limb Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9) - Part 1
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
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Arm Intervention/treatment
Experimental: LION-101 Cohort 1 Genetic: LION-101 dose level 1
Single intravenous infusion of LION-101 gene therapy at dose level 1
Experimental: LION-101 Cohort 2 Genetic: LION-101 dose level 2
Single intravenous infusion of LION-101 gene therapy at dose level 2
Placebo Comparator: Placebo (Cohorts 1 and 2) Other: Placebo
Single intravenous infusion of Placebo


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


Eligibility Criteria
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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 between the ages 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 LION-101 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
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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
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Contact: Medical Affairs at AskBio (919) 561-6210 AskFirst@askbio.com

Locations
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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, Virginia
VCU Recruiting
Richmond, Virginia, United States, 23298
Contact: Abel Bulti       abel.bulti@vcuhealth.org   
Principal Investigator: Nicholas E Johnson, MD         
Sponsors and Collaborators
Asklepios Biopharmaceutical, Inc.
More Information
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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: May 23, 2023
Last Verified: May 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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:
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Muscular Dystrophies
Muscular Dystrophies, Limb-Girdle
Muscular Disorders, Atrophic
Muscular Diseases
 Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn