Menu
Recruiting NCT07557446

A Dose REgimen-Finding Study of AGA2115 in Chinese Patients With Osteogenesis ImpeRfecta (EIR)

Phase II Interventional Osteogenesis Imperfecta (OI)

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: AGA2115.
Who it may be relevant to
Registry conditions: Osteogenesis Imperfecta (OI). Basic parameters: 12 years — 75 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
China
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

A Phase 2 Multi-center, Randomized, Open-Label, Dose Regimen-Finding Study of AGA2115 in Chinese Adults and Adolescents With Type I, III, or IV Osteogenesis Imperfecta

Overview

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

  • Drug AGA2115
    Participants will receive AGA2115 administered by subcutaneous injection

Primary outcome measures

  • Occurrence of Treatment-Emergent Adverse Events (TEAEs) [Time frame: Baseline to Month 27 (Cohorts 1 and 5); Baseline to Month 24 (Cohorts 2, 3, 4, 6, 7 and 8)]
Secondary outcome measures (7)
  • 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. [Time frame: 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. [Time frame: 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 [Time frame: Week 1, Month 1, 3, 6, 9, and 12]
  • Percentage of participants with fractures between Baseline and Month 12 [Time frame: Baseline to Month 12]
  • Annualized fracture rate for incident fractures occurring between Baseline and Month 12 [Time frame: Baseline to Month 12]
  • AGA2115 observed concentration for the treatment groups [Time frame: 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 [Time frame: Day 1 to Month 27 (Cohorts 1 and 5); Day 1 to Month 24 (Cohorts 2, 3, 4, 6, 7 and 8).]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Open label
Primary purpose
Treatment

Study locations

China · 4 centers
  • Chinese Academy of Medical Sciences, Peking Union Medical College Hospital — Beijing
  • The University of Hong Kong-Shenzhen Hospital — Shenzhen
  • Children's Hospital of Soochow University — Suzhou
  • Shanghai Sixth People's Hospital — Shanghai

Identifiers

NCT: NCT07557446 · ACT24-003

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗