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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT05231668
Registration number
NCT05231668
Ethics application status
Date submitted
31/01/2022
Date registered
9/02/2022
Date last updated
15/03/2024
Titles & IDs
Public title
Single Ascending Dose Study of SAR439459 in Adults With Osteogenesis Imperfecta (OI)
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Scientific 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
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Secondary ID [1]
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U1111-1269-6569
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Secondary ID [2]
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SAD17378
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Osteogenesis Imperfecta
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Condition category
Condition code
Musculoskeletal
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Other muscular and skeletal disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Injuries and Accidents
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Fractures
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - SAR439459
Treatment: Drugs - Placebo
Experimental: SAR439459 - Participants will receive a single dose of SAR439459
Placebo Comparator: Placebo - Participants will receive a single dose of placebo
Treatment: Drugs: SAR439459
Powder for solution for infusion; IV infusion
Treatment: Drugs: Placebo
Solution for infusion; IV infusion
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Number of participants with adverse events (AEs)/treatment-emergent adverse events (TEAEs)
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Assessment method [1]
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Timepoint [1]
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From baseline to Week 24
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Secondary outcome [1]
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Assessment of PK parameters: area under the curve (AUC)
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Assessment method [1]
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Timepoint [1]
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From baseline to Week 24
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Secondary outcome [2]
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Assessment of PK parameters: maximum serum concentration observed (Cmax)
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Assessment method [2]
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Timepoint [2]
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From baseline to Week 24
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Secondary outcome [3]
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Assessment of PK parameters: time to reach maximum concentration observed (tmax)
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Assessment method [3]
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Timepoint [3]
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From baseline to Week 24
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Secondary outcome [4]
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Titer of anti-SAR439459 antibodies (if detected)
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Assessment method [4]
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Timepoint [4]
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From baseline to Week 24
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Secondary outcome [5]
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Percent change from baseline in bone mineral density (BMD)
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Assessment method [5]
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Timepoint [5]
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From baseline to Week 24
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Eligibility
Key inclusion criteria
- Participants who are clinically categorized as Type I or IV osteogenesis imperfecta
with a previously documented pathogenic genetic variant in human collagen type 1 alpha
1 gene (COL1A1) or human collagen type 1 alpha 2 gene (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.
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Minimum age
18
Years
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Maximum age
65
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
- Previously installed rods or metal hardware that would prevent bone mineral density
evaluation of the lumbar spine (note: only two of the L1-L4 vertebrae are necessary
for evaluation).
- History of moderate (25-40°) to severe (>40°) scoliosis assessed as Cobb angle (unless
scoliosis does not impact assessment of bone mineral density in the lumbar vertebrae
in the opinion of the investigator).
- Postmenopausal women who:
- Are within 5 years of the onset of menopause (for example less than 5 years from
their last menstruation or post-hysterectomy), however if the person has been on
hormone replacement therapy for more than 1 year prior to enrollment, then they
are eligible regardless of time from onset of menopause. The person must be
willing to continue hormone replacement therapy throughout the study duration. OR
- Were previously on hormone replacement therapy but have stopped within the past 5
years.
- 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 central nervous system (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.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people analysing the results/data
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Intervention assignment
Other
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Other design features
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Phase
Phase 1
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
25/08/2022
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
30/06/2025
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Actual
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Sample size
Target
24
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
NSW,VIC
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Recruitment hospital [1]
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Westmead Hospital_Site Number :0360003 - Westmead
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Recruitment hospital [2]
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Department of Medicine/ School of Clinical Sciences at Monash Health Monash University_246 Clayton Road_Site Number :0360002 - Clayton
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Recruitment postcode(s) [1]
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2145 - Westmead
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Recruitment postcode(s) [2]
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3168 - Clayton
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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California
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Country [2]
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United States of America
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State/province [2]
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Connecticut
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Country [3]
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United States of America
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State/province [3]
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Indiana
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Country [4]
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United States of America
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State/province [4]
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Maryland
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Country [5]
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United States of America
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State/province [5]
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Ohio
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Country [6]
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United States of America
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State/province [6]
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Tennessee
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Country [7]
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United States of America
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State/province [7]
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Texas
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Country [8]
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Canada
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State/province [8]
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Ontario
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Country [9]
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Canada
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State/province [9]
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Toronto
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Country [10]
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France
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State/province [10]
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Lyon
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Country [11]
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France
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State/province [11]
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Paris
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Sanofi
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
SAR439459 is a human anti-Transforming growth factor ß (TGFß) 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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT05231668
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Clinical Sciences & Operations
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Address
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Sanofi
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Trial Transparency email recommended (Toll free for US & Canada)
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Address
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Country
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Phone
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800-633-1610
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Fax
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Email
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[email protected]
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT05231668
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