A Phase 2, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Evaluate the Safety and Efficacy of Aga2115 in Adults With Type I, Iii, or Iv Osteogenesis Imperfecta (Oi)

Study of Treatment for Osteogenesis Imperfecta in Adults

E
Erik Imel, MD

Primary Investigator

A Phase 2, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Evaluate the Safety and Efficacy of Aga2115 in Adults With Type I, Iii, or Iv Osteogenesis Imperfecta (Oi)
Recruiting
18 years - 75 years
All
Phase 2
1 participants needed
1 Location

Brief description of study

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

 

Detailed description of study

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.

Eligibility of study

You may be eligible for this study if you meet the following criteria:

  • Conditions: Osteogenesis Imperfecta (OI)
  • Age: 18 years - 75 years
  • Gender: All

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)

This study investigates the effects of an investigational treatment on adults with Type I, III, or IV osteogenesis imperfecta, a condition where bones break easily due to a genetic defect. The purpose of this study is to compare the safety and effectiveness of different doses of the investigational treatment to a placebo.

Participants will be randomly assigned to receive either a placebo or one of three doses of the investigational treatment for the first 12 months. After this, all participants will receive the investigational treatment for another 12 months. During the study, participants will attend visits where their health and the effects of the treatment will be monitored.

  • Who can participate: Adults aged 18 to 75 years with Type I, III, or IV osteogenesis imperfecta and specific genetic variations may participate. They must have a BMD T-score of ≤ -1.0 and be able to give informed consent. Certain health conditions and recent treatments may disqualify participation.
  • Study details: Participants will take part in a study that includes receiving either the investigational treatment or a placebo for the first year. Afterward, all participants will receive the investigational treatment. A placebo is an inactive substance that looks like the investigational treatment but does not contain any medicine.
  • Study timelines: The study will last 27 months.
Updated on 19 Mar 2026. Study ID: ENDO-ANGITIA-AGA2115

Find a site

We have submitted the contact information you provided to the research team at {{SITE_NAME}}. A copy of the message has been sent to your email for your records.
Would you like to be notified about other trials? Sign up for Patient Notification Services.
Sign up

Send a message

Enter your contact details to connect with study team

Investigator Avatar

Primary Contact

First name*
Last name*
Email*
Phone number*
Other language

Interested in the study?

Select a study center that’s convenient for you, and get in touch with the study team.

Connect with the Study Team