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Single Ascending Dose Study of SAR439459 in Adults With Osteogenesis Imperfecta (OI)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05231668
Recruitment Status : Recruiting
First Posted : February 9, 2022
Last Update Posted : December 7, 2022
Sponsor:
Information provided by (Responsible Party):
Sanofi

Brief Summary:

SAR439459 is a human anti-TGFβ monoclonal antibody. This phase 1 clinical study investigates the safety, tolerability, and activity of a single dose of SAR439459 in adult participants with OI.

Participants will receive a single IV dose of SAR439459 with safety, pharmacokinetic (PK), and pharmacodynamic (PD) assessments over 24 weeks.

There will be up to 3 dose cohorts. In addition to safety, tolerability, and PK assessments, bone mineral density (BMD) will be evaluated by dual-energy Xray absorptimetry (DXA) scan and a series of blood biomarkers will be monitored to document pharmacodynamic effects of the single dose of SAR439459.


Condition or disease Intervention/treatment Phase
Osteogenesis Imperfecta Drug: SAR439459 Drug: Placebo Phase 1

Detailed Description:

The duration of the study for all participants will be approximately 29 weeks:

  • Up to 5 weeks from initiation of screening to dose administration
  • Treatment on Day 1
  • Follow-up and observation of safety and PD for 24 weeks
  • Final study visit at Week 24

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 24 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: A Phase 1b, Single Ascending Dose, Randomized, Double-blind Study to Evaluate the Safety, Tolerability, and Activity of SAR439459 in Adults With Osteogenesis Imperfecta
Actual Study Start Date : August 25, 2022
Estimated Primary Completion Date : April 15, 2024
Estimated Study Completion Date : April 15, 2024


Arm Intervention/treatment
Experimental: SAR439459
Participants will receive a single dose of SAR439459
Drug: SAR439459
Powder for solution for infusion; IV infusion

Placebo Comparator: Placebo
Participants will receive a single dose of placebo
Drug: Placebo
Solution for infusion; IV infusion




Primary Outcome Measures :
  1. Number of participants with adverse events (AEs)/treatment-emergent adverse events (TEAEs) [ Time Frame: From baseline to Week 24 ]

Secondary Outcome Measures :
  1. Assessment of PK parameters: area under the curve (AUC) [ Time Frame: From baseline to Week 24 ]
  2. Assessment of PK parameters: maximum serum concentration observed (Cmax) [ Time Frame: From baseline to Week 24 ]
  3. Assessment of PK parameters: time to reach maximum concentration observed (tmax) [ Time Frame: From baseline to Week 24 ]
  4. Titer of anti-SAR439459 antibodies (if detected) [ Time Frame: From baseline to Week 24 ]
  5. Percent change from baseline in bone mineral density (BMD) [ Time Frame: From baseline to Week 24 ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Participants who are clinically categorized as Type I or IV osteogenesis imperfecta with a previously documented pathogenic genetic variant in COL1A1 or COL1A2.
  • Participants who have experienced at least 1 bone fracture in the past 10 years OR 2 or more (≥2) fractures since the age of 18.
  • Body weight ≥30.0 kg.
  • Contraception for sexually active male participants or female patient; not pregnant or breastfeeding; no sperm donating for male participant.
  • Signed written informed assent/consent.

Exclusion Criteria:

  • Previously installed rods or metal hardware that would prevent bone mineral density evaluation of the lumbar spine.
  • History of moderate (25-40°) to severe (>40°) scoliosis assessed as Cobb angle.
  • Postmenopausal women.
  • History of treatment with denosumab, anti-sclerostin antibody, parathyroid hormone, bisphosphonates, or any other experimental therapy for OI within 6 months prior to any study baseline assessment.
  • Known bleeding disorder.
  • History of significant bleeding event that required hospitalization, surgery, or a blood transfusion that was possibly associated with increased bleeding tendency.
  • Any major surgery within the last 28 days prior to investigational medicinal product (IMP) administration.
  • Elective surgery or invasive procedure anticipated within 6 months after the IMP administration.
  • Therapeutic doses of anticoagulants or antiplatelet agents (eg, 1 mg/kg bid of enoxaparin, 300 mg of aspirin daily, and 75 mg of clopidogrel daily or equivalent) within 7 days prior to the IMP administration.
  • Any known CNS or intraocular lesion that has a risk of bleeding.
  • Prior history of skin cancers including melanoma, squamous cell carcinoma, or basal cell carcinoma.
  • Clinically significant cardiac valvular disorder or symptomatic heart failure.
  • Vitamin D (25-hydoxyvitamin D) <15 ng/dL; rescreening will be allowed after supplementation.

The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05231668


Contacts
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Contact: Trial Transparency email recommended (Toll free for US & Canada) 800-633-1610 ext option 6 Contact-US@sanofi.com

Locations
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United States, Connecticut
Yale University - Site Number:8400007 Recruiting
New Haven, Connecticut, United States, 06510
Contact: Jacqueline Prinz    203-785-5977    jacqueline.prinz@yale.edu   
Principal Investigator: Clemens Bergwitz, Dr.         
United States, Indiana
Indiana University School of Medicine_Site Number: 8400002 Recruiting
Indianapolis, Indiana, United States, 46202
Contact: Amy Katz    317-948-2601    katzamy@iu.edu   
Principal Investigator: Erik Imel, Doctor         
United States, New Mexico
New Mexico Clinical Research & Osteoporosis Center-Site Number:8400001 Recruiting
Albuquerque, New Mexico, United States, 87106
Contact: Mary Niles    505-855-5505    mniles@nmbonecare.com   
Principal Investigator: Michael Lewiecki, Dr         
United States, New York
Columbia University Irving Medical Center_Site Number: 8400009 Not yet recruiting
New York, New York, United States, 10032
Contact: Joanne Carroll    212-305-5508    Treatment_program@cumc.columbia.edu   
Principal Investigator: Wendy Chung, Doctor         
United States, Texas
Baylor College of Medicine - Site Number:8400003 Recruiting
Houston, Texas, United States, 77030
Contact: Dianne Nguyen    713-798-6694    diannen@bcm.edu   
Principal Investigator: Sandesh Nagamani, Dr.         
Australia, New South Wales
Westmead Hospital_Site Number :0360003 Recruiting
Westmead, New South Wales, Australia, 2145
Contact: Yang Song    8890 3791    yang.song@health.nsw.gov.au   
Principal Investigator: Christian Girgis, Dr         
Australia, Victoria
Department of Medicine/ School of Clinical Sciences at Monash Health Monash University_246 Clayton Road_Site Number :0360002 Recruiting
Clayton, Victoria, Australia, 3168
Contact: Cat Shore-Lorenti    +61 (0)3 8572 2582    cat.shore-lorenti@monash.edu   
Principal Investigator: Peter Ebeling, Prof         
Canada, Ontario
Bone Research and Education Centre_Site Number :1240003 Recruiting
Oakville, Ontario, Canada, L6M 1M1
Contact: Shehryar Mehmood    905-844-5677    shehryar@boneresearch.ca   
Principal Investigator: Aliya Khan, Dr         
France
Hopital Edouard Herriot _Site Number :2500002 Recruiting
Lyon, France, 69003
Contact: Catherine Planckaert    33 4 72 11 74 40    c.planckaert@pmo-lyon.fr   
Principal Investigator: Roland Chapurlat, Prof         
Hopital Lariboisiere_Site Number :2500001 Recruiting
Paris, France, 75010
Contact: Sylvie Fernandez    33 6 60 93 17 75    sylvie.fernandez-ext@aphp.fr   
Principal Investigator: Martine Cohen Solal, Prof         
Sponsors and Collaborators
Sanofi
Investigators
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Study Director: Clinical Sciences & Operations Sanofi
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Responsible Party: Sanofi
ClinicalTrials.gov Identifier: NCT05231668    
Other Study ID Numbers: SAD17378
U1111-1269-6569 ( Registry Identifier: ICTRP )
2021-004914-21 ( EudraCT Number )
First Posted: February 9, 2022    Key Record Dates
Last Update Posted: December 7, 2022
Last Verified: December 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Osteogenesis Imperfecta
Osteochondrodysplasias
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Genetic Diseases, Inborn
Collagen Diseases
Connective Tissue Diseases