NCT05224505

Brief Summary

Phase 1 dose escalation study to assess tolerability and safety of ATA-100 with 5-year follow-up

Trial Health

78
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
6

participants targeted

Target at below P25 for phase_1

Timeline
41mo left

Started Sep 2022

Longer than P75 for phase_1

Geographic Reach
3 countries

3 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress52%
Sep 2022Sep 2029

First Submitted

Initial submission to the registry

December 15, 2021

Completed
2 months until next milestone

First Posted

Study publicly available on registry

February 4, 2022

Completed
7 months until next milestone

Study Start

First participant enrolled

September 1, 2022

Completed
2.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2025

Completed
4.7 years until next milestone

Study Completion

Last participant's last visit for all outcomes

September 30, 2029

Expected
Last Updated

October 6, 2025

Status Verified

October 1, 2025

Enrollment Period

2.4 years

First QC Date

December 15, 2021

Last Update Submit

October 1, 2025

Conditions

LGMDR9

Outcome Measures

Primary Outcomes (1)

  • Safety and tolerability

    Incidence of Adverse Events and significant laboratory changes

    Baseline through 12 months

Secondary Outcomes (10)

  • Combined endpoint (global testing)

    Baseline through 12 months

  • Timed Up and Go (TUG) test

    Baseline through 12 months

  • Change from baseline in velocity as measured by 10MWT

    Baseline through 12 months

  • 2-minute walk distance test

    Baseline through 12 months

  • Cardiac MRI

    Baseline through 12 months

  • +5 more secondary outcomes

Study Arms (2)

Cohort 1

EXPERIMENTAL

Cohort 1: single intravenous injection 9.0E+12 vg/Kg

Biological: ATA-100 (AAV9 encoding FKRP gene)

Cohort 2

EXPERIMENTAL

Cohort 2: single intravenous injection 2.7E+13 vg/Kg

Biological: ATA-100 (AAV9 encoding FKRP gene)

Interventions

ATA-100 (AAV9 encoding FKRP gene)BIOLOGICAL

Single intravenous infusion on Day 0

Cohort 1Cohort 2

Eligibility Criteria

Age16 Years - 99 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • \. Female and male ambulant patients
  • \. Patients ≥ 16 years old
  • \. Documented LGMDR9 diagnosis based on clinical presentation and genotyping confirming the FKRP gene mutations
  • \. Moderate diaphragmatic muscle impairment

You may not qualify if:

  • \. Detectable serum neutralizing antibodies against AAV9
  • \. Cardiomyopathy

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

Rigshospitalet, University of Copenhagen Blegdamsvej 9

Copenhagen, 2100, Denmark

Location

Institute of Myology Pitié-Salpêtrière Hospital 47 Bd de l'Hôpital

Paris, 75013, France

Location

Royal Victoria Infirmary Queen Victoria Road Level 6 Leazes Wing

Newcastle upon Tyne, NE1 4LP, United Kingdom

Location

MeSH Terms

Conditions

Muscular Dystrophy, Limb-Girdle, Type 2I

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Model Details: Single IV administration of ATA-100, open label, 2 sequential cohorts
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 15, 2021

First Posted

February 4, 2022

Study Start

September 1, 2022

Primary Completion

February 1, 2025

Study Completion (Estimated)

September 30, 2029

Last Updated

October 6, 2025

Record last verified: 2025-10

Locations

DK(1)FR(1)GB(1)