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Gene Therapy

Gene Therapy (AT132) for Myotubular Myopathy (ASPIRO Trial)

Phase 2 & 3

Waitlist Available

Research Sponsored by Astellas Gene Therapies

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Subject is male

Subject requiring invasive mechanical ventilator support is fitted with or willing to be fitted with a cuffed tracheostomy tube for some respiratory assessments

Must not have

Subject has a clinically significant underlying liver disease

Subject has previous scoliosis repair surgery/procedure, or planned/expected scoliosis repair surgery/procedure in the 12 months following Day 1

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to month 120

Awards & highlights

Summary

This trial tests AT132, a gene therapy, in children under 5 with a rare muscle disease. The therapy aims to deliver a healthy gene to improve muscle function. Participants will receive one dose and be monitored for safety and effectiveness over several years. AT132 has been used in previous gene therapy trials for muscle diseases.

Who is the study for?

This trial is for boys under 5 with X-Linked Myotubular Myopathy (XLMTM) confirmed by a specific gene mutation. They must need breathing support, weigh over 4.8 kg in France, and have no recent surgeries or infections. Those with heart issues, anemia, liver disease, or certain medication use are excluded.

What is being tested?

The study tests AT132's safety and effectiveness in treating XLMTM. Participants receive one dose of AT132 and are monitored for ten years. It's multinational with ascending doses compared to delayed treatment controls.

What are the potential side effects?

Potential side effects aren't specified but may include reactions related to gene therapy such as immune responses, site inflammation, or complications from the delivery method used.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am male.

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I am using or open to using a cuffed tracheostomy tube for breathing assessments.

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I have XLMTM due to a confirmed MTM1 gene mutation.

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I need a machine to help me breathe.

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I am under 5 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have a serious liver condition.

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I have had or will have scoliosis surgery within a year.

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I was born before 35 weeks of pregnancy and am not yet at my due date based on corrected age.

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I have taken medication for XLMTM, like pyridostigmine, within the last 3 months.

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I have anemia with a hemoglobin level below 10 g/dL.

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I am allergic or cannot take the study drug due to health reasons.

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I currently have a serious lung infection or another active infection.

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I cannot take ursodiol due to health reasons.

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I cannot take prednisolone due to health reasons.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to month 120

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to month 120

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to month 120 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Change from baseline in hours of ventilation support at Week 24

Secondary outcome measures

Change from baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) at Week 24

Change from baseline in Pediatric Quality of Life Inventory (PedsQL) assessment at Week 24

Change from baseline in maximal inspiratory pressure (MIP) at Week 24

+8 more

Trial Design

3Treatment groups

Experimental Treatment

Active Control

Group I: Lower DoseExperimental Treatment1 Intervention

1.0 x 10^14 vg/kg of AT132 defined with the use of 1st generation vg titer assay delivered intravenously one time. 1.3 x10^14 vg/kg of AT132 defined with the use of 2nd generation vg titer assay delivered intravenously one time.

Group II: Higher DoseExperimental Treatment1 Intervention

3.0 x 10^14 vg/kg of AT132 defined with the use of 1st generation vg titer assay delivered intravenously one time. 3.5 x 10^14 vg/kg of AT132 defined with the use of 2nd generation vg titer assay delivered intravenously one time

Group III: Delayed-Treatment ControlActive Control1 Intervention

Delayed-Treatment Control subjects will generally have the same assessments as treated subjects. After the follow up period, eligible delayed-treatment control subjects will be dosed with AT132 and initiate the same post-dose procedures as subjects who received AT132.

0 / 14Eligibility criteria met

Find a Location

Who is running the clinical trial?

Astellas Gene TherapiesLead Sponsor

4 Previous Clinical Trials

307 Total Patients Enrolled

Audentes TherapeuticsLead Sponsor

7 Previous Clinical Trials

247 Total Patients Enrolled

Medical DirectorStudy DirectorAstellas Pharma Global Development, Inc.

2,801 Previous Clinical Trials

8,070,304 Total Patients Enrolled

Media Library

AT132 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03199469 — Phase 2 & 3

Myotubular Myopathy Research Study Groups: Lower Dose, Delayed-Treatment Control, Higher Dose

Myotubular Myopathy Clinical Trial 2023: AT132 Highlights & Side Effects. Trial Name: NCT03199469 — Phase 2 & 3

AT132 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03199469 — Phase 2 & 3

~3 spots leftby Sep 2025

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