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Tests treatment safety and results for Glycogen Storage Disease Type II Infantile Onset
Official title A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
ClinicalTrials.gov ID: NCT04808505
What this study is testing
What is Cipaglucosidase alfa?
Cipaglucosidase alfa is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for glycogen storage disease type ii infantile onset.
Also referred to as ATB200.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This is a Phase 3, open-label, multicenter study to evaluate the safety, efficacy, PK, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ERT-experienced and ERT-naïve pediatric subjects with IOPD.
- Phase 3: a large, late-stage study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages up to 17
You may be able to join if
- Cohort 1:
- Male or female people who are aged 6 months to \< 18 years on Day 1
- Subject must have documentation of IOPD genotype
- Subject must have had hypertrophic cardiomyopathy at the time of diagnosis
- Subject must have received ERT for at least 6 months immediately before enrollment. For people whose ERT dosage has been modified, the subject must...
You likely can't join if
- Cohort 1 and Cohort 2, unless specified
- Subject requires invasive ventilation (eg, tracheostomy)
- Subject is CRIM negative and has not received prophylactic immunomodulation (Cohort 1); Subject is CRIM negative and will not be receiving...
- Subject has a history of life-threatening IARs/hypersensitivity (eg, anaphylaxis and severe cutaneous reactions) to ERT (eg, alglucosidase alfa...
- Subject has prior history of illness or condition known to affect motor function
- Female subject is pregnant (or intends to get pregnant) or breastfeeding at screening (Cohort 1)
See the full eligibility criteria
- Cohort 1:
- Male or female people who are aged 6 months to \< 18 years on Day 1
- Subject must have documentation of IOPD genotype
- Subject must have had hypertrophic cardiomyopathy at the time of diagnosis
- Subject must have received ERT for at least 6 months immediately before enrollment. For people whose ERT dosage has been modified, the subject must have been on the modified dosage and regimen for at least 3 months...
- people aged ≥ 12 to \< 18 years must perform one valid 6-minute walk test (6MWT) (≥ 75 meters) at screening; people aged ≥ 5 to \< 12 years must perform one valid 6MWT (≥ 40 meters) at screening; people aged 18 months...
- people must have experienced a clinical decline on their current rhGAA dose and frequency Cohort 2:
- Male or female people who are aged 0 to \<6 months at Day 1
- Subject must have documentation of IOPD genotype
- Subject must have had hypertrophic cardiomyopathy at the time of diagnosis
- Subject is ERT-naïve Long-term Extension (Cohort 1 or Cohort 2): 1\. Subject must have, in the opinion of the investigator, benefited from therapy with cipaglucosidase alfa/miglustat during the 104-week primary...
- Cohort 1 and Cohort 2, unless specified
- Subject requires invasive ventilation (eg, tracheostomy)
- Subject is CRIM negative and has not received prophylactic immunomodulation (Cohort 1); Subject is CRIM negative and will not be receiving prophylactic immunomodulation (Cohort 2)
- Subject has a 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...
- Subject has prior history of illness or condition known to affect motor function
- Female subject is pregnant (or intends to get pregnant) or breastfeeding at screening (Cohort 1)
The study team makes the final eligibility decision.
Where it's taking place
- Gainesville, Florida, United States
- Atlanta, Georgia, United States
- Durham, North Carolina, United States
- Cincinnati, Ohio, United States
- Pittsburgh, Pennsylvania, United States
- Salt Lake City, Utah, United States
- Giessen, Germany
- Heidelberg, Germany
- Höchheim, Germany
- Münster, Germany
- Naples, Italy
- Rotterdam, Netherlands
- Taipei, Taiwan
- London, United Kingdom
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, up to 17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Gainesville, Florida, United States; Atlanta, Georgia, United States; Durham, North Carolina, United States; Cincinnati, Ohio, United States; Pittsburgh, Pennsylvania, United States; Salt Lake City, Utah, United States and 8 more location(s). 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 ClinicalTrials.gov; the study team decides eligibility.