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Study to evaluate the safety and efficacy of Cipaglucosidase Alfa and Miglustat in pediatric subjects with late-onset Pompe disease
EU CTIS ID: 2022-502547-36-00
What this study is testing
To evaluate the safety and tolerability of cipaglucosidase alfa/miglustat co-administration
- Therapeutic confirmatory (Phase III)
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. (Cohort 1) Male or female subjects (ERT-naïve [have never received a dose of rhGAA] or ERT-experienced [have received rhGAA for at least 6 months immediately before enrollment, and if ERT dosage has been modified, must have been on the modified dosage for at least 3 months before enrollment]) diagnosed with LOPD who are aged 12 to < 18 years at screening.
- 2. (Cohort 1) Subject weighs ≤ 115 kg.
- 3. (Cohort 1) Subject has a sitting forced vital capacity (FVC) ≥ 30% of the predicted value for healthy adolescents (Global Lung Function Initiative) at screening
- 4. (Cohort 1) Subject performs one 6-Minute Walk Test (6MWT) (≥ 75 meters) at screening that is valid, as determined by the clinical evaluator
- 5. (Cohort 2) Male or female subjects (ERT-naïve [have never received a dose of rhGAA] or ERT-experienced [have received rhGAA for at least 6 months immediately before enrollment, and if ERT dosage has been modified, must have been on the modified dosage for at least 3 months before enrollment]) diagnosed with LOPD who are aged 0 months to < 12 years at screening
- 6. (Cohort 2) Subjects aged ≥ 5 to < 12 years who perform one 6MWT (≥ 40 meters) at screening that is valid, as determined by the clinical evaluator
You likely can't join if
- 1. (Cohort 1 & 2) Subject has received any investigational/experimental drug, oral anabolic steroid or derivative, biologic, or device within 30 days or 5 half-lives of the therapy or treatment, whichever is longer, before screening
- 5. (Cohort 1 & 2) Subject has history of life-threatening IARs/hypersensitivity (eg. anaphylaxis and severe cutaneous reactions) to ERT (eg. alglucosidase alfa, cipaglucosidase alfa), miglustat, or other iminosugars, or to any of the excipients, where rechallenge was unsuccessful.
- 6. (Cohort 1 & 2) Female subject is pregnant or breast-feeding at screening
- 7. (Cohort 1 & 2) Subject requires the use of ventilation support for > 6 hours per day while awake
- 12. (Cohort 2) Subject has clinical presentation of classic IOPD (ie, GAA enzyme activity less than 1% of normal).
- 8. (Cohort 1) Subject has moderate to severe hypertrophic cardiomyopathy aligning with classic IOPD
See the full eligibility criteria
Who can join
- 1. (Cohort 1) Male or female subjects (ERT-naïve [have never received a dose of rhGAA] or ERT-experienced [have received rhGAA for at least 6 months immediately before enrollment, and if ERT dosage has been modified, must have been on the modified dosage for at least 3 months before enrollment]) diagnosed with LOPD who are aged 12 to < 18 years at screening.
- 2. (Cohort 1) Subject weighs ≤ 115 kg.
- 3. (Cohort 1) Subject has a sitting forced vital capacity (FVC) ≥ 30% of the predicted value for healthy adolescents (Global Lung Function Initiative) at screening
- 4. (Cohort 1) Subject performs one 6-Minute Walk Test (6MWT) (≥ 75 meters) at screening that is valid, as determined by the clinical evaluator
- 5. (Cohort 2) Male or female subjects (ERT-naïve [have never received a dose of rhGAA] or ERT-experienced [have received rhGAA for at least 6 months immediately before enrollment, and if ERT dosage has been modified, must have been on the modified dosage for at least 3 months before enrollment]) diagnosed with LOPD who are aged 0 months to < 12 years at screening
- 6. (Cohort 2) Subjects aged ≥ 5 to < 12 years who perform one 6MWT (≥ 40 meters) at screening that is valid, as determined by the clinical evaluator
- 7. (Cohort 1 & 2) Subject’s parent or legally authorized representative is willing and able to provide written informed consent and authorization for use and disclosure of personal health information or research-related health information, and subject provides assent, if applicable, based on site and local regulations.
- 8. (Cohort 1 & 2) Subject must have a diagnosis of LOPD based on documentation of at least one of the following: a. deficiency of GAA enzyme b. gene encoding human acid α-glucosidase (GAA) genotyping
- 9. (Cohort 1 & 2) If of reproductive potential and if sexually active, female and male subjects agree to use a highly effective method of contraception throughout the duration of the study and for up to 90 days after their last dose of cipaglucosidase alfa/miglustat
What rules you out
- 1. (Cohort 1 & 2) Subject has received any investigational/experimental drug, oral anabolic steroid or derivative, biologic, or device within 30 days or 5 half-lives of the therapy or treatment, whichever is longer, before screening
- 5. (Cohort 1 & 2) Subject has history of life-threatening IARs/hypersensitivity (eg. anaphylaxis and severe cutaneous reactions) to ERT (eg. alglucosidase alfa, cipaglucosidase alfa), miglustat, or other iminosugars, or to any of the excipients, where rechallenge was unsuccessful.
- 6. (Cohort 1 & 2) Female subject is pregnant or breast-feeding at screening
- 7. (Cohort 1 & 2) Subject requires the use of ventilation support for > 6 hours per day while awake
- 12. (Cohort 2) Subject has clinical presentation of classic IOPD (ie, GAA enzyme activity less than 1% of normal).
- 8. (Cohort 1) Subject has moderate to severe hypertrophic cardiomyopathy aligning with classic IOPD
- 9. (Cohort 1 & 2) In the opinion of the investigator, the parent or legally authorized representative is unlikely or unable to comply with the study requirements
- 12. (Cohort 1 & 2) Subject has been placed in an institution by court or official order.
- 10. (Cohort 1 & 2) Subject has any prior history of illness or condition known to affect motor function, such as, but not limited to, eg. Guillain-Barre syndrome, cerebral palsy
- 11. (Cohort 2) Subject is asymptomatic (ie, showing no signs and symptoms of Pompe disease)
- 2. (Cohort 1 & 2) Subject has received treatment with prohibited medications within 30 days of screening
- 3. (Cohort 1 & 2) Subject has received any gene therapy at any time
- 4. (Cohort 1 & 2) Subject has any intercurrent illness or condition at screening or baseline that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator and/or the medical monitor that the potential subject may have an unacceptable risk by participating in this study
The study team makes the final eligibility decision.
Where it's taking place
- Canada
- Taiwan
- United States
- Japan
- Australia
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 all sexes, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Canada; Taiwan; United States; Japan; Australia. 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.