Study to Evaluate Cipaglucosidase Alfa/Miglustat in IOPD (Rossella, ATB200-08)
Rossella: A Study to Evaluate the Safety, PK, Efficacy, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
About: This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ERT-experienced and ERT-naïve pediatric subjects with classic IOPD.
Ages Eligible: 0 to <18 years at age of Screening
Sexes Eligible for Study: All
Status: Actively recruiting participants.
For more information about this study, including eligibility criteria, please go to the study’s ClinicalTrials.gov page. If you are interested in participating in this study or have questions about this research please contact Samantha Norman, Clinical Research Coordinator
A Study to Evaluate the PK, Safety, Efficacy, and PD With ATB200/AT2221 in LOPD Subjects (ATB200-04)
An Open-label Study of the Pharmacokinetics, Safety, Efficacy, and Pharmacodynamics of ATB200/AT2221 in Pediatric Subjects aged 12 to < 18 years with Late-onset Pompe Disease
About: The purpose of the study is to validate doses for ATB200/AT2221 in pediatrics and to evaluate changes in key clinical outcome measures (eg, motor, respiratory, fatigue) in late-onset Pompe disease (LOPD) pediatric subjects.
Ages Eligible for Study: 12 Years to 18 Years (Pediatrics)
Sexes Eligible for Study: All
Status: Actively Recruiting Participants.
For more information about this study, including eligibility criteria, please go to the study’s ClinicalTrials.gov page. If you are interested in participating in this study or have questions about this research please contact Nicole Fernandez, Clinical Research Coordinator.
First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221 (ATB200-02)
An Open-Label, Fixed-Sequence, Ascending-Dose, First-in-Human Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of Intravenous Infusions of ATB200 Co-Administered With Oral AT2221 in Adult Subjects With Pompe Disease
About: This study is an international, multi-center, study of Pompe disease patients that are currently receiving enzyme-replacement therapy (ERT). The purpose of this study is to find out if the co-administration of investigational new drugs ATB200 and AT2221 is safe in adults with Pompe disease.
Ages Eligible for Study: 18 Years to 75 Years (Adult, Senior)
Sexes Eligible for Study: All
Status: This study is ongoing but no longer recruiting participants. Accepting transfers.
For more information about this study, including eligibility criteria, please go to the study’s ClinicalTrials.gov page. If you are interested in participating in this study or have questions about this research please contact Nicole Fernandez, Clinical Research Coordinator.
Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease (AAV9-GAA_IM)
Evaluation of Re-administration of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV9-DES-hGAA) in Patients With Late-Onset Pompe Disease (LOPD)
About: A recombinant AAV vector has been generated to carry the codon-optimized acid alpha-glucosidase (coGAA) gene expressed from a human desmin enhancer/promoter (DES). The proposed clinical trial is a within-participant, double-blind, randomized, phase I controlled study evaluating the toxicology, biodistribution and potential activity of re-administration of rAAV9-DES-hGAA injected intramuscularly into the TA. Nine participants (18 to 50-years old) who reside within the United States with Late-Onset Pompe Disease (LOPD) will be included. The goal of the immune modulation strategy is to ablate B-cells (Rituximab and Sirolimus) prior to the initial exposure to the study agent in one leg and the subsequent exposure of the same vector to the contralateral leg after four months. At each study agent dosing, the contralateral leg will receive excipient. Patients will act as their own controls. Repeated measures, at baseline and during the following 3 months after each injection, will assess the safety, biochemical and functional impact of the vector.
Ages Eligible for Study: 18 Years to 50 Years (Adult)
Sexes Eligible for Study: All
Status: Actively Recruiting Participants.
For more information about this study, including eligibility criteria, please go to the study’s ClinicalTrials.gov page. If you are interested in participating please contact Samantha Norman, Clinical Study Coordinator.
Response to Diaphragmatic Pacing in Subjects with Pompe Disease
About: This study follows people with Pompe Disease who are receiving diaphragm pacemaker for six months following implantation to assess changes in their breathing function.
Ages Eligible for Study: 2 Years to 65 Years (Child, Adult)
Sexes Eligible for Study: All
Status: Actively Recruiting Participants. Accepting Transfers.
For more information about this study, including eligibility criteria, please go to the study’s ClinicalTrials.gov page. If you are interested in participating please contact Juliette Clavier, Clinical Study Coordinator.
Pompe Disease Registry
About: The Pompe Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Pompe disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient’s treating physician.
The objectives of the Registry are:
- To enhance the understanding of the variability, progression, and natural history of the key manifestations of Pompe disease;
- To assist the Pompe medical community with the development of recommendations for monitoring patients and reports on patient outcomes to help optimize patient care;
- To characterize and describe the Pompe disease population as a whole; and
- To evaluate the long-term effectiveness and safety of available treatment options including ERT(Enzyme Replacement Therapy) with Myozyme
Ages Eligible for Study: Child, Adult, Senior
Sexes Eligible for Study: All
Status: Actively Recruiting Participants.
For more information about this study, including eligibility criteria, please go to the study’s ClinicalTrials.gov page. If you are interested in participating please contact Samantha Norman, Clinical Research Coordinator.