Your session is about to expire
← Back to Search
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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to month 120
Awards & highlights
ASPIRO Trial Summary
This trial will test a new drug, AT132, for safety and effectiveness in treating X-Linked Myotubular Myopathy, in children aged 5 and under. The trial will last 10 years.
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.Check my eligibility
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.See study design
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.
ASPIRO Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am male.
Select...
I am using or open to using a cuffed tracheostomy tube for breathing assessments.
Select...
I have XLMTM due to a confirmed MTM1 gene mutation.
Select...
I need a machine to help me breathe.
Select...
I am under 5 years old.
ASPIRO Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to month 120
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to month 120
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 moreASPIRO 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.
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.
2,777 Previous Clinical Trials
8,063,460 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I am male.You have a high level of AAV8 neutralizing antibodies in your blood.I have a serious liver condition.I have had or will have scoliosis surgery within a year.I can sit unassisted for at least 30 seconds.I was born before 35 weeks of pregnancy and am not yet at my due date based on corrected age.You have a significant health condition aside from XLMTM, as determined by the researcher.I have taken medication for XLMTM, like pyridostigmine, within the last 3 months.I haven't taken immune-modulating drugs recently, except for inhaled steroids for a chronic respiratory condition.I am using or open to using a cuffed tracheostomy tube for breathing assessments.I have a condition like scoliosis that may prevent me from sitting up without help.I have anemia with a hemoglobin level below 10 g/dL.You are currently taking part in a study to treat XLMTM.Only for people in France: You have had heart rhythm problems, inflammation of the heart, or any other heart disease in the past.I had surgery less than 3 months ago or have surgery planned that could affect the study's first 48 weeks.I am allergic or cannot take the study drug due to health reasons.I have XLMTM due to a confirmed MTM1 gene mutation.I currently have a serious lung infection or another active infection.I cannot take ursodiol due to health reasons.You need a ventilator with a maximum positive end-expiratory pressure (PEEP) of at least 8 cm H2O at screening.I need a machine to help me breathe.I cannot take prednisolone due to health reasons.I am under 5 years old.
Research Study Groups:
This trial has the following groups:- Group 1: Lower Dose
- Group 2: Delayed-Treatment Control
- Group 3: Higher Dose
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
What is the intent behind this medical experiment?
"This clinical trial, assessed over a period of 24 weeks, will measure the change in ventilation support from baseline. Additionally, secondary outcomes such as change in respiratory endurance (PImax), ability to sit independently for 30 seconds or more per assessment by an independent physical therapy panel, and time required to reduce invasive ventilator usage to 16 hours or less daily by a separate pulmonary adjudication panel will be monitored."
Answered by AI
How widespread is the implementation of this trial's protocol?
"At present, this clinical trial is accepting patients from 4 locations: Chicago, Bethesda, and Toronto as well as 4 other cities. To reduce travel needs when participating in the study it's best to choose a site closest to you."
Answered by AI
Is the recruitment process for this medical trial open at present?
"According to data hosted on clinicaltrials.gov, this trial is no longer actively enrolling patients; the original post date was August 2nd 2017 and it last updated July 6th 2022. However, there are currently 30 alternative studies that are accepting new recruits at present."
Answered by AI
Recent research and studies
Other Trials to Consider
Popular Categories
Share this study with friends
Copy Link
Messenger