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Enzyme Replacement Therapy
Cipaglucosidase Alfa + Miglustat for Pompe Disease
Phase 3
Recruiting
Research Sponsored by Amicus Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Subject has a sitting forced vital capacity (FVC) ≥ 30% of the predicted value for healthy Adolescents at screening (Cohort 1 only)
Subject must have a diagnosis of LOPD based on documentation as defined in study protocol
Must not have
Subject has evidence of moderate to severe hypertrophic cardiomyopathy aligning with classic IOPD
Female subject is pregnant or breast-feeding at screening
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 52 weeks
Awards & highlights
Summary
This trial will study the safety and effectiveness of Cipaglucosidase Alfa/Miglustat in treating Pompe disease in children who have either received enzyme replacement therapy before or have not.
Who is the study for?
This trial is for pediatric patients with Pompe disease, aged from birth to less than 18 years. It includes those who have previously received enzyme replacement therapy and those who haven't. Participants must meet certain breathing capacity criteria, agree to use contraception if of reproductive age, and not exceed a weight limit (for older children).
What is being tested?
The study tests the safety and effectiveness of Cipaglucosidase Alfa/Miglustat in treating Pompe disease in children. This Phase 3 trial involves both experienced and new patients to enzyme replacement therapy, assessing how the body processes the drug, its efficacy, pharmacodynamics, and potential immune response.
What are the potential side effects?
While specific side effects are not listed here, common ones for treatments like Cipaglucosidase Alfa/Miglustat may include allergic reactions to ingredients, gastrointestinal symptoms such as nausea or diarrhea, fatigue or headaches. The exact side effects will be monitored throughout the study.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
My lung function is at least 30% of what is expected for a healthy person my age.
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I have been diagnosed with Late-Onset Pompe Disease (LOPD).
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I am diagnosed with LOPD and fall within the specified age range for my cohort.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
I have a severe thickening of my heart muscle.
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I am currently pregnant or breastfeeding.
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I have a history of conditions like Guillain-Barre syndrome that affect my movement.
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I have received gene therapy before.
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I need a ventilator for more than 6 hours a day when I'm awake.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 52 weeks
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~52 weeks
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Secondary study objectives
Assessment of pharmacokinetic parameters
Trial Design
1Treatment groups
Experimental Treatment
Group I: Cipaglucosidase Alfa (ATB200)/Miglustat(AT2221)Experimental Treatment2 Interventions
Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat (AT2221) capsule
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Cipaglucosidase Alfa
2018
Completed Phase 3
~130
Miglustat
2020
Completed Phase 4
~210
Find a Location
Who is running the clinical trial?
Amicus TherapeuticsLead Sponsor
54 Previous Clinical Trials
2,699 Total Patients Enrolled
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Eligibility Criteria:
This trial includes the following eligibility criteria:- I can walk at least 75 meters if I'm 12-17 years old, or at least 40 meters if I'm 5-11 years old.My lung function is at least 30% of what is expected for a healthy person my age.I have been diagnosed with Late-Onset Pompe Disease (LOPD).I have a severe thickening of my heart muscle.I was diagnosed with Pompe disease at birth and show no symptoms.I agree to use effective birth control during and up to 3 months after the study.I am currently pregnant or breastfeeding.I am diagnosed with LOPD and fall within the specified age range for my cohort.I weigh 115 kg or less.I have a history of conditions like Guillain-Barre syndrome that affect my movement.You are allergic to any of the ingredients in ATB200, approved rhGAA, or AT2221.I haven't taken any experimental drugs or treatments recently.I have received gene therapy before.I have not taken any prohibited medications in the last 30 days.I need a ventilator for more than 6 hours a day when I'm awake.
Research Study Groups:
This trial has the following groups:- Group 1: Cipaglucosidase Alfa (ATB200)/Miglustat(AT2221)
Awards:
This trial has 2 awards, including:- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
- 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.
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