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First-in-human study of AT132 in X-Linked Myotubular Myopathy (XLMTM) Patients
EU CTIS ID: 2024-512637-32-00
What this study is testing
To determine the therapeutic dose of AT132 To confirm the safety and efficacy of the therapeutic dose of AT132
- Phase II and Phase III (Integrated)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1. Subject has a diagnosis of XLMTM resulting from a genetically confirmed mutation in the MTM1 gene as assessed by a Sponsor- approved testing facility.* * = Inclusion/exclusion criteria for delayed treatment control subjects before receiving AT132.
- 2. Subject is male.*
- 3. Subject is aged less than 5 years old at dosing*
- 4. Subject requires mechanical ventilatory support: Part 1: Subject requires some mechanical ventilatory support (eg., ranging from 24 hours per day full time mechanical ventilation, to noninvasive support such as continuous positive airway pressure [CPAP] or bilevel positive airway pressure [BiPAP] during sleeping hours). Part 2: Subject requires invasive mechanical ventilatory support ranging from 20 to 24 hours per day at screening (confirmed by daytime polysomnographic study).
- 5. Subject requiring invasive mechanical ventilator support is fitted with or willing to be fitted with a cuffed tracheostomy tube for some respiratory assessments.*
- 6. Subject has ventilator maximum positive end-expiratory pressure (PEEP) < 8 cm H2O at screening.*
You likely can't join if
- 1. Subject is participating in an interventional study designed to treat XLMTM.* * = If a subject is a delayed-treatment control, this inclusion/exclusion criterion must be met before receiving AT132.
- 10.Subject has a contraindication to prednisolone.*
- 11.Subject has a contraindication to study drug or ingredients.*
- 12. Subject has previous scoliosis repair surgery/procedure, or planned/expected scoliosis repair surgery/procedure, in the 12 months following Day 1 (Part 2 including any subjects enrolles under protocol v8 and beyond ).
- 13. Subject has contractures, scoliosis, or other medical condition that would limit the potential to achieve unassisted sitting, in the opinion of the Investigator (Part 2 including any subjects enrolles under protocol v8 and beyond ).
- 14. Subject is able to sit without assistance for at least 30 seconds at screening, in the opinion of the Investigator (Part 2 including any subjects enrolles under protocol v8 and beyond ).
See the full eligibility criteria
Who can join
- 1. Subject has a diagnosis of XLMTM resulting from a genetically confirmed mutation in the MTM1 gene as assessed by a Sponsor- approved testing facility.* * = Inclusion/exclusion criteria for delayed treatment control subjects before receiving AT132.
- 2. Subject is male.*
- 3. Subject is aged less than 5 years old at dosing*
- 4. Subject requires mechanical ventilatory support: Part 1: Subject requires some mechanical ventilatory support (eg., ranging from 24 hours per day full time mechanical ventilation, to noninvasive support such as continuous positive airway pressure [CPAP] or bilevel positive airway pressure [BiPAP] during sleeping hours). Part 2: Subject requires invasive mechanical ventilatory support ranging from 20 to 24 hours per day at screening (confirmed by daytime polysomnographic study).
- 5. Subject requiring invasive mechanical ventilator support is fitted with or willing to be fitted with a cuffed tracheostomy tube for some respiratory assessments.*
- 6. Subject has ventilator maximum positive end-expiratory pressure (PEEP) < 8 cm H2O at screening.*
- 7. Signed informed consent by the parent(s) or legally authorized representative(s) (LAR) (when applicable).*
- 8. Subject and parent(s)/LAR(s) are willing and able to comply with study visits and study procedures.*
- 9. Subject's range is ≥ 4.8 kg (UNIQUE to France)
What rules you out
- 1. Subject is participating in an interventional study designed to treat XLMTM.* * = If a subject is a delayed-treatment control, this inclusion/exclusion criterion must be met before receiving AT132.
- 10.Subject has a contraindication to prednisolone.*
- 11.Subject has a contraindication to study drug or ingredients.*
- 12. Subject has previous scoliosis repair surgery/procedure, or planned/expected scoliosis repair surgery/procedure, in the 12 months following Day 1 (Part 2 including any subjects enrolles under protocol v8 and beyond ).
- 13. Subject has contractures, scoliosis, or other medical condition that would limit the potential to achieve unassisted sitting, in the opinion of the Investigator (Part 2 including any subjects enrolles under protocol v8 and beyond ).
- 14. Subject is able to sit without assistance for at least 30 seconds at screening, in the opinion of the Investigator (Part 2 including any subjects enrolles under protocol v8 and beyond ).
- 15. Subject has a clinically important condition, including CTCAE v4.03 Grade ≥ 2 anemia (< 10 g/dL hemoglobin).*
- 16. Subject has a contraindication to ursodiol (ursodeoxycholic acid).*
- 17. Subject has a prior diagnosis or history of cardiac arrhytmias, myocarditis, or any other cardiac disease (UNIQUE to FRANCE).
- 18. Subject has a contradiction to general anesthesia and to muscle biopsy procedure (UNIQUE to France).
- 2. Subject born < 35 weeks gestation who is still not term as per corrected age.
- 3. Subject tests positive for AAV8 neutralizing antibody with titers > 1:20 (subjects under the age of 18 months may be retested in cases where antibodies may have been maternally acquired and titers may decline in the first months of life).*
- 4. Subject had recent surgery (< 3 months before Day 1) or has planned surgery that may confound data collection during the first 48 weeks of the study.
- 5. Subject has a clinically important condition or life-threatening disease, other than XLMTM, in the opinion of the Investigator.*
- 6. Subject has a clinically significant underlying liver disease, defined as: • ≥ Grade 3 aspartate aminotransferase (AST) (> 5.0 x upper limit of normal [ULN]; Common Terminology Criteria for Adverse Events [CTCAE] v. 4.03)* • ≥ Grade 3 alanine aminotransferase (ALT) (> 5.0 x ULN; CTCAE v. 4.03)* • Hepatic peliosis or any other clinically significant structural abnormality detected by ultrasound*
- 7. Subject is currently experiencing a clinically important respiratory infection or other active infection.*
- 8.Subject has received pyridostigmine or any medication to treat XLMTM within 3 months before Day 1.*
- 9.Other than as required per protocol, subject has received immunemodulating agents within 3 months before Day 1 (use of inhaled corticosteroids to manage chronic respiratory conditions is allowed); use of other concomitant medications to manage chronic conditions must have beenstable for at least 4 weeks before dosing.*
The study team makes the final eligibility decision.
Where it's taking place
- Canada
- United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Canada; United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.