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

Part of paid clinical trials in Irvine, California.

Sponsor
AskBio Inc
Study ID
NCT05230459
Phase
PHASE1/PHASE2
Status
Recruiting
Apply to this trial

Conditions

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

Eligibility Criteria

Sex
ALL
Age
18 Years - 65 Years
Healthy Volunteers
Not accepted

Interventions

  • AB-1003 dose level 1 — GENETIC
    Single intravenous infusion of AB-1003 gene therapy at dose level 1
  • AB-1003 dose level 2 — GENETIC
    Single intravenous infusion of AB-1003 gene therapy at dose level 2
  • Placebo — OTHER
    Single intravenous infusion of Placebo

Study Details

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)

Key Dates

Start date
May 15, 2023
Status verified
Feb 2026
Primary completion
Dec 31, 2028
Completion
Dec 31, 2032

Study Design

Enrollment
10 participants (estimated)
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: AB-1003 Cohort 1
  • Experimental: AB-1003 Cohort 2
  • Placebo Comparator: Placebo (Cohorts 1 and 2)

Primary Outcome Measure

Adverse Events [ Time Frame: 0-52 weeks ]

Central Contacts

  • AskFirst Patient Engagement
    919-561-6210
    [email protected]
  • myTomorrows (see link below in reference section)

Locations (6)

FacilityCityStateZIPSite coordinators
University of California - IrvineIrvineCalifornia92697
UCI Alpha Clinic
[email protected]
Tahseen Mozaffar, MD (PRINCIPAL_INVESTIGATOR)
University of IowaIowa CityIowa52242
Katherine Mathews, MD (PRINCIPAL_INVESTIGATOR)
University of Kansas Medical CenterKansas CityKansas66160
Andrea Klempnauer
[email protected]
Jeffrey Statland, MD (PRINCIPAL_INVESTIGATOR)
Kennedy Krieger InstituteBaltimoreMaryland21205
Doris Leung, MD, PhD (PRINCIPAL_INVESTIGATOR)
VCURichmondVirginia23298
Anarosa Rezeq
[email protected]
Nicholas E Johnson, MD (PRINCIPAL_INVESTIGATOR)
University of Washington Medical CenterSeattleWashington98195
Piya Modalavalasa
[email protected]
B. Jane Distad, MD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Irvine, CA

By research site
University of California - Irvine· Irvine, CAUniversity of Iowa· Iowa City, IAUniversity of Kansas Medical Center· Kansas City, KSKennedy Krieger Institute· Baltimore, MDVCU· Richmond, VAUniversity of Washington Medical Center· Seattle, WA

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