Late-Onset GSD-II (Pompe Disease) MedDRA version: 20.0 Level: LLT Classification code 10036143 Term: Pompe's disease System Organ Class: 100000004850 MedDRA version: 20.1 Level: LLT Classification code 10045253 Term: Type II glycogen storage disease System Organ Class: 100000004850
Conditions
Interventions
Product Name: VAL-1221
Product Code: VAL-1221
Pharmaceutical Form: Solution for injection/infusion
INN or Proposed INN: VAL-1221
Current Sponsor code: VAL-1221
Concentration unit: mg/ml milligram(s)/m
Sponsors
Valerion Therapeutics, LLC
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male or female patients =18 years 2. Patient is able and willing to provide informed consent prior to any study procedures are performed 3. Diagnosis of Glycogen Storage Disease II (GSD-II) based on one of the following: a. Endogenous cultured skin fibroblast acid alpha glucosidase (GAA) activity 30% and 20% but =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Cardiac involvement in first year of life 2. Anti-GAA antibody titers >1:51,200 at two time points 3. Prior use of chaperone therapy for GSD-II within the last 12 months 4. Use of immunosuppressive medication other than glucocorticoids within 6 months prior to study enrollment 5. Use of invasive ventilatory assistance other than BiPAP at night or during periods of rest 6. Has received any investigational medication or has enrolled in any study involving investigational drugs or therapies within 30 days prior to first dose of study drug 7. Start of or change in usual regimen of albuterol or respiratory muscle training within 30 days prior to first dose of study drug 8. History of sensitivity to any of the constituents of the study drug 9. Patient is planning to become pregnant or to breastfeed during the study or is currently breastfeeding 10. Patient has a medical condition or circumstance that, in the opinion of the investigator, might compromise the patient’s ability to comply with the protocol or the patient’s well-being or safety 11. Patient has any condition that, in the view of the investigator, places the patient at high risk of poor treatment compliance or of not completing the study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Part 1: • To assess the safety, tolerability and immunogenicity of VAL-1221 given IV Part 2: • To assess the long-term safety, tolerability and immunogenicity of VAL-1221 given IV;Secondary Objective: Part 1: • To assess the pharmacokinetics of VAL-1221 given IV • To assess the pharmacodynamics of VAL-1221 given IV Part 2: • To assess the long-term pharmacodynamics of VAL-1221 given IV • To optimize dosing of VAL-1221 given IV;Primary end point(s): Number of patients with adverse events, infusion-associated reactions to VAL-1221 and changes in anti-VAL-1221 antibody titers. Antibodies will be assessed for neutralizing capacity.;Timepoint(s) of evaluation of this end point: Throughout the study | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Part 1: • Pharmacokinetic profile of VAL-1221 using plasma GAA activity and/or plasma GAA concentration. • Change from Baseline to Week 12 in the presence and activity of GAA on muscle biopsy • Change from Baseline to Week 12 in the amount of glycogen on muscle biopsy determined biochemically and histologically • Change from Baseline to Week 12 in urinary hex4 excretion and serum creatine kinase Part 2: • Change from Baseline in Part 1 of the study to Months 12, 24 and 36 - Urinary hex4 excretion and creatinine - Serum creatine kinase (CK);Timepoint(s) of evaluation of this end point: Part 1: • Up to week 12 Part 2: • At Months 12, 24 and 36 | — |
Countries
United Kingdom, United States
Contacts
Public ContactClinical Trial Operations
Voisin Consulting
Outcome results
None listed