An Operationally Seamless, Randomized Phase 2/3 Study Consisting of a Phase 2 Single-Blind, Dose-Evaluation Phase and a Phase 3 Double-Blind, Placebo-Controlled Phase to Assess the Efficacy and Safety of Setrusumab in Subjects With Osteogenesis Imperfecta

Who is this study for? Patients with Osteogenesis Imperfecta
What treatments are being studied? Setrusumab
Status: Active_not_recruiting
Location: See all (44) locations...
Intervention Type: Biological, Other
Study Type: Interventional
Study Phase: Phase 2/Phase 3
SUMMARY

The primary objectives of the study are to identify a setrusumab dosing strategy in participants with OI and to evaluate the effect of setrusumab vs placebo on reduction in fracture rate.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 5
Maximum Age: 25
Healthy Volunteers: f
View:

• Diagnosis of OI Type I, III, or IV as confirmed by identification of pathogenic or likely pathogenic genetic variants in COL1A1 or COL1A2. If a variant of uncertain significance is identified, then clinical presence of the expected phenotype can be used to confirm the diagnosis

• ≥ 1 fracture in the past 12 months, ≥ 2 fractures in the past 24 months or ≥ 1 tibia, femur or humerus fracture in the past 24 months

• Serum 25-hydroxyvitamin D ≥ 20 ng/mL at the Screening Visit. If 25-hydroxyvitamin D levels are below 20 ng/mL, 25-hydroxyvitamin D testing can repeated after a minimum of 14 days of vitamin D supplementation as directed by the treating physician

• Willing to not receive bisphosphonate therapy during the study

• From the period following informed consent to 60 days after the last dose of the study drug, females of childbearing potential and fertile males must consent to use highly effective contraception. If female, agree not to become pregnant. If male, agree not to father a child or donate sperm

• Willing and able to provide informed consent for subjects greater than or equal to 18 years of age, or provide assent (if possible) and have a legally authorized representative provide informed consent, after the nature of the study has been explained and prior to any research-related procedures

• Willing to provide access to medical records for the collection of radiographic data, fracture data, growth data, and disease history

• Must, in the opinion of the Investigator, be willing and able to complete all aspects of the study, adhere to the study visit schedule, and comply with the assessments

Locations
United States
Arkansas
Arkansas Children's Hospital
Little Rock
California
Children's Hospital Los Angeles
Los Angeles
Shriners Hospital for Children - Northern California
Sacramento
Colorado
Children's Hospital Colorado
Aurora
Connecticut
Connecticut Children's Medical Center
Hartford
Yale New Haven Hospital
New Haven
Washington, D.c.
Children's National Medical Center
Washington D.c.
Delaware
Nemours/ Alfred i. duPoint Hospital for Children
Wilmington
Florida
University of South Florida
Tampa
Illinois
Shriners Hospitals for Children - Chicago
Chicago
Indiana
Indiana University Hospital
Indianapolis
Massachusetts
Boston Children's Hospital
Boston
Maryland
Kennedy Krieger Institute
Baltimore
Missouri
Washington University School of Medicine
St Louis
North Carolina
Atrium Health Levine Children's Hospital
Charlotte
Nebraska
Children's Hospital and Medical Center
Omaha
New Mexico
New Mexico Clinical Research & Osteoporosis Center, Inc.
Albuquerque
Ohio
Nationwide Children's Hospital- Ohio State University College of Medicine
Columbus
Pennsylvania
The Children's Hospital of Philadelphia
Philadelphia
Tennessee
Vanderbilt University Medical Center
Nashville
Texas
Cook Children's Medical Center
Fort Worth
Baylor College of Medicine
Houston
Wisconsin
UW Health University Hospital
Madison
Other Locations
Australia
Royal Children's Hospital
Melbourne
Queensland Paediatric Endocrinology
South Brisbane
Canada
London Health Sciences Center
London
McGill University Health Centre
Montreal
Children's Hospital of Eastern Ontario
Ottawa
University of Toronto- The Hospital for Sick Children (SickKids)
Toronto
France
Institut Imagine
Paris
Germany
University of Cologne
Cologne
Otto von Guericke University Magdeburg
Magdeburg
Musculoskeletal Center Würzburg
Würzburg
Italy
Istituto Ortopedico Rizzoli
Bologna
Azienda Ospedaliera Universitaria Policlinico Umberto I
Rome
Universita Degli Studi Di Verona
Verona
Netherlands
Wilhelmina Children's Hospital
Utrecht
Poland
Uniwersytet Medyczny w Lodzi - Klinika Endokrynologii i Chorob Metabolicznych
Lodz
Portugal
Hospital de Santa Maria
Lisbon
Centro Hospitalar do Porto
Porto
Turkey
Gazi University
Ankara
Marmara University
Istanbul
United Kingdom
Royal Manchester Childrens Hospital
Manchester
Sheffield Children's NHS Foundation Trust
Sheffield
Time Frame
Start Date: 2022-02-21
Completion Date: 2027-04
Participants
Target number of participants: 183
Treatments
Experimental: Low Dose Setrusumab -> Open-Label (OL) Setrusumab Selected Dose
Single-blind setrusumab low dose during Phase 2 followed by open-label setrusumab selected dose~During treatment and treatment extension periods, participants may receive supplementation with calcium and vitamin D to maintain normal values as directed by the treating physician
Experimental: High Dose Setrusumab -> OL Setrusumab Selected Dose
Single-blind setrusumab high dose during Phase 2 followed by open-label setrusumab~During treatment and treatment extension periods, participants may receive supplementation with calcium and vitamin D to maintain normal values as directed by the treating physician
Experimental: Setrusumab Selected Dose -> OL Setrusumab Selected Dose
Double-blind setrusumab selected dose during Phase 3 followed by open-label setrusumab~During treatment and treatment extension periods, participants may receive supplementation with calcium and vitamin D to maintain normal values as directed by the treating physician
Placebo_comparator: Placebo -> OL Setrusumab Selected Dose
Double-blind placebo during Phase 3 followed by open-label setrusumab~During treatment and treatment extension periods, participants may receive supplementation with calcium and vitamin D to maintain normal values as directed by the treating physician
Related Therapeutic Areas
Sponsors
Collaborators: Mereo BioPharma
Leads: Ultragenyx Pharmaceutical Inc

This content was sourced from clinicaltrials.gov