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

Angitia Biopharmaceuticals

Status and phase: Enrolling; Phase 2

Conditions: Osteogenesis Imperfecta (OI)

Treatments: Drug: AGA2115; Other: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT07062588

ACT24-001

Details and patient eligibility

About

This study will determine the effect of treatment of AGA2115 in adults with Type I, III, or IV osteogenesis imperfecta versus placebo.

Full 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.

Enrollment: 80 estimated patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

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)

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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80 participants in 4 patient groups, including a placebo group

AGA2115 Dose Regimen 1

Experimental group

Description:

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.

Treatment:

Drug: AGA2115

AGA2115 Dose Regimen 2

Experimental group

Description:

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.

Treatment:

Drug: AGA2115

AGA2115 Dose Regimen 3

Experimental group

Description:

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.

Treatment:

Drug: AGA2115

Placebo

Placebo Comparator group

Description:

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.

Treatment:

Other: Placebo