Osteogenesis Imperfecta (OI)
Conditions
Keywords
Osteogenesis Imperfecta, dose regimen finding, Phase 2
Brief summary
This study is to evaluate the safety and efficacy of AGA2115 at three different dose regimens in Chinese adults and adolescents with Type I, III, or IV Osteogenesis imperfecta (OI).
Detailed description
This Phase 2 study will evaluate the safety and efficacy of AGA2115 in three different dosing regimens in Chinese adults and adolescents with Type I, III, or IV OI. Participants will be in the study for 24 or 27 months depending on their assigned cohort. During the first 12 months of the study, adult and adolescent participants will be randomized separately in a 1:1:1:1 ratio to one of three AGA2115 dosing regimens or control cohort. During months 12 to 24 or 27, all participants will receive AGA2115 and attend visits for the evaluation of safety and efficacy parameters.
Interventions
DRUGAGA2115
Participants will receive AGA2115 administered by subcutaneous injection
Sponsors
Angitia Biopharmaceuticals Guangzhou Limited
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
* Adults (18-75 years) or adolescents (12-17 years) with a confirmed diagnosis of Osteogenesis Imperfecta (OI) Type I, III, or IV with genetic confirmation of pathogenic variants in COL1A1 or COL1A2 genes * BMD T-score of ≤-1.0 at the lumbar spine, total hip, or femoral neck (adults) or BMD Z-score of ≤-1.0 at the lumbar spine, total hip, or femoral neck (adolescents) * Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol
Exclusion criteria
* Vitamin D deficiency * Concomitant uncontrolled diseases or conditions that could affect bone metabolism such as hypo-/hyperparathyroidism, hypo-/hyperthyroidism, abnormal thyroid function or thyroid disease, or other endocrine disorders. * Current hyper- or hypocalcemia. * History of rickets, osteomalacia, or other significant skeletal disorders (excluding OI) leading to long-bone deformities and/or increased risk of fractures. * Use of bisphosphonates within the past 6 months. * Use of teriparatide, abaloparatide, strontium ranelate, or hormone replacement therapy within the past 12 months. * Use of denosumab (or denosumab biosimilars) within the past 2 years. * Use of anti-sclerostin antibody medications (romosozumab, setrusumab, blosozumab) at any time. * History of myocardial infarction or stroke (or other cardiovascular associated event deemed significant) within the past 12 months. * Malignancy within the last 5 years. * Pregnant or breastfeeding women, or women planning to become pregnant during the study or within 4 months after the last dose of IP.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Occurrence of Treatment-Emergent Adverse Events (TEAEs) | Baseline to Month 27 (Cohorts 1 and 5); Baseline to Month 24 (Cohorts 2, 3, 4, 6, 7 and 8) |
Secondary
| Measure | Time frame |
|---|---|
| Percent change from Baseline at Month 3, 6, 9 and 12 in Bone Mineral Density (BMD) at lumbar spine, total hip, femoral neck, one-third distal radius, and total body (minus head) for adults and adolescents. | Months 3, 6, 9, and 12 |
| Change from Baseline at Month 3, 6, 9, and 12 in BMD Z-score at lumbar spine, total hip, femoral neck, one-third distal radius, and total body (minus head) for adolescents. | Month 3, 6, 9, and 12 |
| Percent Change from Baseline at Week 1 and Month 1, 3, 6, 9, and 12 in bone turnover markers CTX-1 and P1NP | Week 1, Month 1, 3, 6, 9, and 12 |
| Percentage of participants with fractures between Baseline and Month 12 | Baseline to Month 12 |
| Annualized fracture rate for incident fractures occurring between Baseline and Month 12 | Baseline to Month 12 |
| AGA2115 observed concentration for the treatment groups | Day 1 to Month 27 (Cohorts 1 and 5); Day 1 to Month 24 (Cohorts 2, 3, 4, 6, 7 and 8). |
| Serum anti-AGA2115 antibodies | Day 1 to Month 27 (Cohorts 1 and 5); Day 1 to Month 24 (Cohorts 2, 3, 4, 6, 7 and 8). |
Countries
China
Contacts
CONTACTYolanda Liu
Outcome results
None listed