Osteogenesis Imperfecta (OI)
Conditions
Brief summary
This study will determine the effect of treatment of AGA2115 in adults with Type I, III, or IV osteogenesis imperfecta versus placebo.
Detailed description
This Phase 2 dose-ranging study will evaluate the safety and efficacy of AGA2115 at a range of doses in adults with Type I, III, or IV osteogenesis imperfecta. The study will last 27 months with a 24-month treatment period and a 3-month follow-up period. During the first 12 months of the study, participants will be randomized 1:1:1:1 to receive either placebo or one of three dose levels of AGA2115 doses; treatment assignment will be double-blind. Months 12 to 24 will be open-label, and all participants will receive AGA2115. Participants will attend visits where safety and efficacy parameters will be evaluated.
Interventions
DRUGAGA2115
Subcutaneous injection
OTHERPlacebo
Subcutaneous injection
Sponsors
Angitia Incorporated Limited
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
* Male or female adults (aged 18 to 75 years inclusive) with a clinical diagnosis of osteogenesis imperfecta Type I, III, or IV with documented genetic testing confirmation of genetic variations in the COL1A1 or COL1A2 genes * 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 * BMD T-score of ≤ -1.0 at the lumbar spine, total hip, or femoral neck
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 or osteomalacia or any skeletal condition (other than OI) leading to long-bone deformities and/or increased risk of fractures * Treatment with bisphosphonates within the past 6 months * Treatment with teriparatide, abaloparatide, strontium ranelate, or hormone replacement therapy within the past 12 months * Treatment with denosumab (or denosumab biosimilars) within the past 2 years * Treatment with 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 * Participation in any clinical study within the past 12 months during which the participant was administered any IP (participant must also agree not to enroll in any other clinical study concurrently in which IP is administered)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Percent change from Baseline at Month 12 in lumbar spine BMD | 12 months |
Secondary
| Measure | Time frame |
|---|---|
| Percent change from Baseline at Month 3 and 6 in lumbar spine, total hip, femoral neck, one-third distal radius, and total body (minus head) BMD | 3 and 6 months |
| Percent change from Baseline at Month 12 in total hip, femoral neck, one-third distal radius, and total body (minus head) BMD | 12 months |
| Percent change from Baseline at Week 1 and Month 1, 3, 6, 9, and 12 in P1NP and CTX-1 | Week 1, Month 1, 3, 6, 9, and 12 |
| Percentage of participants with fractures during the double-blind treatment period | up to 12 months |
| Annualized fracture rate for incident fractures occurring during the double-blind treatment period (total, long-bone, vertebral, peripheral) | up to 12 months |
Countries
Argentina, Australia, Canada, Denmark, France, Netherlands, United Kingdom, United States
Contacts
CONTACTKimberly Brown
STUDY_DIRECTORHeather Byers, MD
Angitia Incorporated Limited
Outcome results
None listed