Osteogenesis Imperfecta Trial of AGA2115 for ADUlts With COL1A1 and/or COL1A2 GeNetic Variations (IDUN)

Not Applicable

Not yet recruiting

• Conditions: Osteogenesis Imperfecta (OI)
• Interventions: Drug: AGA2115; Other: Placebo

• Registration Number: NCT07062588
• Lead Sponsor: Angitia Incorporated Limited

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.

Study Timeline

• Status Verified: 2025-06
• Study First Submitted: 2025-07-01
• Study First Submitted QC: 2025-07-10
• Study First Posted: 2025-07-14
• Last Update Submitted: 2025-07-20
• Last Update Posted: 2025-07-24
• Study Start: 2025-11-01
• Primary Completion: 2027-11
• Study Completion: 2029-02-01

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 80

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)

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
AGA2115 Dose Regimen 1	AGA2115	Participants that complete the double-blind period receiving AGA2115 Dose 1 from study start to Month 12 will continue to a 12-month open-label period. Participants will then receive AGA2115 Dose 2 for Months 12 to 24.
AGA2115 Dose Regimen 2	AGA2115	Participants that complete the double-blind period receiving AGA2115 Dose 2 from study start to Month 12 will continue to a 12-month open-label period. Participants will be kept on the same AGA2115 dose for Months 12 to 24.
AGA2115 Dose Regimen 3	AGA2115	Participants that complete the double-blind period receiving AGA2115 Dose 3 from study start to Month 12 will continue to a 12-month open-label period. Participants will receive the same AGA2115 dose for Months 12 to 24.
Placebo	Placebo	Participants that complete the double-blind period receiving placebo from study start to Month 12 will continue to a 12-month open-label period. Participants will receive AGA2115 Dose 3 for Months 12 to 24.

Primary Outcome Measures

Name	Time	Method
Percent change from Baseline at Month 12 in lumbar spine BMD	12 months

Secondary Outcome Measures

Name	Time	Method
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