A Study to Evaluate the Safety of LION-101 in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)
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|ClinicalTrials.gov Identifier: NCT05230459|
Recruitment Status : Recruiting
First Posted : February 9, 2022
Last Update Posted : May 23, 2023
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|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 Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||10 participants|
|Intervention Model:||Sequential Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|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|
|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)||
Single intravenous infusion of Placebo
- Adverse Events [ Time Frame: 0-52 weeks ]Treatment Emergent Adverse Events, Serious Adverse Events, Dose Limiting Toxicity
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.
|Ages Eligible for Study:||18 Years to 65 Years (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- Male and female subjects between the ages 18 and 65 years with clinical diagnosis of LGMD2I/R9 and confirmation of FKRP gene mutation.
- Ability to ascend 4 stairs between 2.5 and 10 seconds.
- Ability to walk/run 10 meters in < 30 seconds.
- Able to understand and comply with all study procedures.
- 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.
- 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.
- Contraindication to MRI or hypersensitivity to contrast dyes, shellfish or iodine.
- Implanted spinal rods, cardiac pacemaker or other implantation that would distort cardiac MRI images.
- 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).
- Abnormal renal function (GFR < 60 ml/min, using the MDRD equation).
- 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).
- In the opinion of the investigator, a pre-existing medical condition that predisposes the subject to risks that outweighs the potential benefits.
- Requirement for daytime ventilatory support.
- Change in glucocorticosteroid treatment within 3 months prior to baseline visit.
- Exposure to another investigational drug within 3 months prior to study treatment or any previous treatment with gene therapy.
- Ongoing participation in any other therapeutic clinical trial.
- Neutralizing antibody titer to AAV9 ≥ 1:5.
- Female subjects who are pregnant, plan to become pregnant in the next 12 months, or breastfeeding.
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
|Contact: Medical Affairs at AskBio||(919) 561-6210||AskFirst@askbio.com|
|United States, California|
|University of California - Irvine||Recruiting|
|Irvine, California, United States, 92697|
|Contact: UCI Alpha Clinic firstname.lastname@example.org|
|Principal Investigator: Tahseen Mozaffar, MD|
|United States, Iowa|
|University of Iowa||Recruiting|
|Iowa City, Iowa, United States, 52242|
|Contact: Ciara Gibbs email@example.com|
|Principal Investigator: Katherine Mathews, MD|
|United States, Virginia|
|Richmond, Virginia, United States, 23298|
|Contact: Abel Bulti firstname.lastname@example.org|
|Principal Investigator: Nicholas E Johnson, MD|
|Responsible Party:||Asklepios Biopharmaceutical, Inc.|
|Other Study ID Numbers:||
|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|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
gene augmentation therapy
fukutin related protein
Muscular Dystrophies, Limb-Girdle
Muscular Disorders, Atrophic
Nervous System Diseases
Genetic Diseases, Inborn