Osteogenesis ImperfectaPhase 1NCT05231668
Single Ascending Dose Study of SAR439459 in Adults With Osteogenesis Imperfecta (OI)
A Phase 1 study testing SAR439459 in people with Osteogenesis Imperfecta, run by Sanofi at 13 sites in 4 countries.
Trial summary
SAR439459 is a human anti-Transforming growth factor β (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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Osteogenesis Imperfecta
- What is being tested?
- SAR439459
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 16 participants
- Where
- 13 sitesin 4 countries
- Study dates
- 25 Aug 2022to 12 Nov 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Show all 16Show fewer
- 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.
Trial locations and contacts
13 sites in 4 countries.
13 sites in 4 countries
United States7 sites
Kennedy Krieger Institute_Site number 8400004
Baltimore, Maryland, 21205
Cincinnati Children's Hospital Medical Center Site Number : 8400010
Cincinnati, Ohio, 45229
Baylor College of Medicine - Site Number:8400003
Houston, Texas, 77030
Indiana University School of Medicine_Site Number: 8400002
Indianapolis, Indiana, 46202
UCLA Health_Site Number: 8400006
Los Angeles, California, 90095
Vanderbilt University Site Number : 8400011
Nashville, Tennessee, 37232
Yale University - Site Number:8400007
New Haven, Connecticut, 06510
Australia2 sites
Department of Medicine/ School of Clinical Sciences at Monash Health Monash University_246 Clayton Road_Site Number :0360002
Clayton, Victoria, 3168
Westmead Hospital_Site Number :0360003
Westmead, New South Wales, 2145
Canada2 sites
Bone Research and Education Centre_Site Number :1240003
Oakville, Ontario, L6M 1M1
Toronto general Hospital_Site Number :1240002
Toronto, M5G 2C4
France2 sites
Hopital Edouard Herriot _Site Number :2500002
Lyon, 69003
Hopital Lariboisiere_Site Number :2500001
Paris, 75010
About this study
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
SAR439459 is a human anti-Transforming growth factor β (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.
Read the 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
- Conditions
- Osteogenesis Imperfecta
- Being tested
- SAR439459, Placebo
- Medical subject headings
- Osteogenesis Imperfecta
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SAR439459Drug
Powder for solution for infusion; IV infusion
PlaceboDrug
Solution for infusion; IV infusion
| Study group | Role | What they receive |
|---|---|---|
| SAR439459 | Experimental | Participants will receive a single dose of SAR439459 Receives: Drug: SAR439459 |
| Placebo | Placebo comparator | Participants will receive a single dose of placebo Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with adverse events (AEs)/treatment-emergent adverse events (TEAEs)
Measured: From baseline to Week 24
Secondary outcomes (5)
Assessment of PK parameters: area under the curve (AUC)
Measured: From baseline to Week 24
Assessment of PK parameters: maximum serum concentration observed (Cmax)
Measured: From baseline to Week 24
Assessment of PK parameters: time to reach maximum concentration observed (tmax)
Measured: From baseline to Week 24
Titer of anti-SAR439459 antibodies (if detected)
Measured: From baseline to Week 24
Percent change from baseline in bone mineral density (BMD)
Measured: From baseline to Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Feb 2022
- Study start25 Aug 2022
- Primary completion12 Nov 2024
- Study completion12 Nov 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Osteogenesis ImperfectaStudies of the same condition, open and under way.
- More trials run by SanofiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sanofi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences & Operations, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05231668
- Sponsor's protocol ID
- SAD17378
- Other IDs
- U1111-1269-6569, 2024-511369-12-00, 2021-004914-21
- Status last verified
- Sep 2025
- First submitted
- 31 Jan 2022
- Last updated
- 11 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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
Study at a glance
From the registry record, last updated by the sponsor on 11 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05231668
- Acronym / sponsor ID
- SAD17378
- Conditions
- Osteogenesis Imperfecta
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 16 participants (actual)
- Lead sponsor
- SanofiIndustry (pharma, biotech, devices)
- Study start
- 25 Aug 2022 (actual)
- Primary completion
- 12 Nov 2024 (actual)
- Study completion
- 12 Nov 2024 (actual)
- First posted
- 9 Feb 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
SAR439459 is a human anti-Transforming growth factor β (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.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| SAR439459 | Experimental | Drug: SAR439459Participants will receive a single dose of SAR439459 |
| Placebo | Placebo comparator | Drug: PlaceboParticipants will receive a single dose of placebo |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (16 listed)
- 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:
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SAR439459Powder for solution for infusion; IV infusion | Drug | SAR439459 |
| PlaceboSolution for infusion; IV infusion | Drug | Placebo |
Primary outcome
Number of participants with adverse events (AEs)/treatment-emergent adverse events (TEAEs)
Time frame: From baseline to Week 24
5 secondary outcomes
Assessment of PK parameters: area under the curve (AUC)
Time frame: From baseline to Week 24
Assessment of PK parameters: maximum serum concentration observed (Cmax)
Time frame: From baseline to Week 24
Assessment of PK parameters: time to reach maximum concentration observed (tmax)
Time frame: From baseline to Week 24
Titer of anti-SAR439459 antibodies (if detected)
Time frame: From baseline to Week 24
Percent change from baseline in bone mineral density (BMD)
Time frame: From baseline to Week 24
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Sep 2025.
- SAD17378 Plain Language Results SummaryLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. 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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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Sciences & Operations study director
Sanofi
13 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing SAR439459 in people with Osteogenesis Imperfecta, run by Sanofi at 13 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 11 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in United States, Australia, Canada, France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early. Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with adverse events (AEs)/treatment-emergent adverse events (TEAEs) (time frame: From baseline to Week 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Sep 2025.