The trial is taking place at:
V

Virginia Commonwealth University | VCU

Veeva-enabled site

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

A

Asklepios Biopharmaceutical

Status and phase

Enrolling
Phase 2
Phase 1

Conditions

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

Treatments

Genetic: AB-1003 dose level 2
Genetic: AB-1003 dose level 1
Other: Placebo

Study type

Interventional

Funder types

Industry

Identifiers

NCT05230459
LION-CS101

Details and patient eligibility

About

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)

Enrollment

10 estimated patients

Sex

All

Ages

18 to 65 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

  • Male and female subjects aged 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 AB-1003 must use a barrier method of contraception for the first 6 months after dosing.

Exclusion criteria

  • 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.

Trial design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Sequential Assignment

Masking

Quadruple Blind

10 participants in 3 patient groups, including a placebo group

AB-1003 Cohort 1
Experimental group
Treatment:
Genetic: AB-1003 dose level 1
AB-1003 Cohort 2
Experimental group
Treatment:
Genetic: AB-1003 dose level 2
Placebo (Cohorts 1 and 2)
Placebo Comparator group
Treatment:
Other: Placebo

Trial contacts and locations

6

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Central trial contact

Medical Affairs at AskBio; myTomorrows (see link below in reference section)

Data sourced from clinicaltrials.gov

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