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Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of SAR442501 in Pediatric Participants With Achondroplasia

PHASE2RECRUITING

This is a Phase 2, open-label, multicenter, study to evaluate safety, tolerability and efficacy of SAR442501 in children from birth up to 12 years of age with Achondroplasia.

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Study details:

Up to approximately 275 weeks: 3 weeks Screening + 52 weeks primary treatment period + up to approximately 216 weeks extended treatment period+ 4 weeks follow-up.

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Eligibility criteria

Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.

Inclusion criteria

  • Participants must have ACH with a confirmed mutation in the FGFR3 gene
  • Participants and/or parent(s) or legal representative(s) must be willing and able to perform all the study procedures to the best of their physical ability.
  • Parent(s) or legal representative(s) capable of giving signed informed consent and participants capable of giving assent when applicable.

    • Exclusion criteria

  • Have hypochondroplasia (or the N540K mutation) or short stature condition other than ACH (eg, trisomy 21, pseudochondroplasia)
  • Participants have received any dose of medications or investigational product, including human growth hormone, IGF-1, intended to affect participants' stature or body proportions between the completion of OBS16647 and enrollment (Week 0/Day 1/Visit 2).
  • Have a history of growth plate closure.
  • Long bone fracture within 3 months of enrollment (Week 0/Day 1/Visit 2)
  • Current evidence of corneal or retinal disorder/keratopathy.
  • Participants have had a previous surgical intervention involving the foramen magnum (Stage 2 only).
  • Hyperphosphatemia.
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    Eligibility

    Age eligible for study : 0 and older

    Healthy volunteers accepted : No

    Gender eligible for study: All

    Things to know

    Study dates

    Study start: 2023-10-10

    Primary completion: 2027-12-02

    Study completion finish: 2027-12-02

    study type

    Study type

    TREATMENT

    phase

    Phase

      PHASE2

    trial

    Trial ID

    NCT06067425

    Intervention or treatment

    DRUG: SAR442501

    Conditions

    • • Osteochondrodysplasia
    Image related to Osteochondrodysplasia

    • Condition: Osteochondrodysplasia

    • DRUG: SAR442501

    • Parkville, Victoria, Australia

    • Sponsor: Sanofi

    You may be eligible to participate in this trial based on your search. Check eligibility by answering some questions.

    Are you running this trial? If you're a clinic or sponsor, you can claim this study. Claim or learn more.

    Find a site

    Closest Location:

    Investigational Site Number : 0360001

    Research sites nearby

    Select from list below to view details:

    • Investigational Site Number : 0360001

      Parkville, Victoria, Australia

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    Study Plan

    This section provides details of the study plan, including how the study is designed and what the study is measuring.

    How is the study designed?

    Participant Group/ArmIntervention/Treatment
    EXPERIMENTAL: Cohort 1
    • Not Specified
    DRUG: SAR442501
    • Solution for injection; Subcutaneous injection
    EXPERIMENTAL: Cohort 2
    • Not Specified
    DRUG: SAR442501
    • Solution for injection; Subcutaneous injection
    EXPERIMENTAL: Cohort 3
    • Not Specified
    DRUG: SAR442501
    • Solution for injection; Subcutaneous injection

    What is the study measuring?

    Primary outcome

    Primary Outcome MeasurePrimary Outcome DescriptionPrimary Outcome Time Frame
    Number of participants with adverse events (AE), serious adverse events (SAE), and adverse events of special interest (AESI) during the treatment-emergent periodNot SpecifiedBaseline to Week 52

    Secondary outcome

    Secondary Outcome MeasureSecondary Outcome DescriptionSecondary Outcome Time Frame
    Change in annualized growth velocity (AGV) ZscoreNot SpecifiedBaseline to Week 26 and Week 52
    Change in AGV (cm/year)Not SpecifiedBaseline to Week 26 and Week 52
    Change in height Z scoreNot SpecifiedBaseline to Week 26 and Week 52
    Change in upper-to-lower body segment ratioNot SpecifiedBaseline to Week 26 and Week 52
    Change in upper to lower extremity ratioNot SpecifiedBaseline to Week 26 and Week 52
    Change in sitting to standing height ratio (crown-to-rump length to total length for infants)Not SpecifiedBaseline to Week 26 and Week 52
    Change in arm span to height ratioNot SpecifiedBaseline to Week 26 and Week 52
    Change in upper arm to forearm length ratioNot SpecifiedBaseline to Week 26 and Week 52
    Change in upper leg to lower leg ratioNot SpecifiedBaseline to Week 26 and Week 52
    Change in head circumference to height ratioNot SpecifiedBaseline to Week 26 and Week 52
    Change in brainstem parameterChange in surface area of the bony foramen magnum (cm2) as measured by head and neck Magnetic Resonance Imaging (MRI).Baseline to Week 52
    Change in skull parameterChange in dimensions of skull base parameters, and degree of synchondroses fusion as measured by head and neck MRI.Baseline to Week 52
    Change in spine morphometric parameterChange in grading of cord compression and cord constriction as assessed by head and neck MRI.Baseline to Week 52
    Change in volumetric parameterChange in brainstem and spinal cord volume as measured by head and neck MRI.Baseline to Week 52
    Change in overall health-related quality of life score in the PedsQL Inventory Generic Core ScalePedsQL Generic Core Scales, global score ranging from 0-100, with higher scores representing better outcomesBaseline to Week 26 and Week 52
    Change in fatigue score in the PedsQL Multidimensional Fatigue ScalePedsQL Multidimensional Fatigue Scale, global score ranging from 0-100, with higher scores representing better outcomesBaseline to Week 26 and Week 52
    Change in present pain and worst pain rating (PPQ) scorePediatric Pain Questionnaire (PPQ) score value between 0-4. The lower the better.Baseline to Week 26 and Week 52
    Change in mobility and symptom rating (STEMS) scoreScreening Tool for Everyday Mobility and Symptoms (STEMS) score value between 1-5. The lower the better.Baseline to Week 26 and Week 52
    Change in developmental score in the Achondroplasia Developmental Recording FormAchondroplasia Developmental Recording Form to record the age at which participants achieve developmental milestones. The earlier the better.Baseline to Week 52
    Assessment of pharmacokinetic (PK) parameter: plasma concentration of SAR442501Not SpecifiedBaseline to Week 26 and 52
    Assessment of PK parameter: maximum plasma concentration observed (Cmax)Not SpecifiedBaseline to Week 26 and 52
    Assessment of PK parameter: time to reach Cmax (Tmax)Not SpecifiedBaseline to Week 26 and 52
    Assessment of PK parameter: Area under the plasma concentration versus time curve calculated using the trapezoidal method during a dose interval (AUC0-t)Not SpecifiedBaseline to Week 26 and 52
    Assessment of PK parameter: concentration observed before treatment administration during repeated dosing (Ctrough)Not SpecifiedBaseline to Week 26 and 52
    Assessment of pharmacodynamics (PD) parameter: change in collagen X biomarker (CXM) levelsNot SpecifiedBaseline to Week 26 and Week 52
    Assessment of PD parameter: change in osteocalcin levelsNot SpecifiedBaseline to Week 26 and Week 52
    Assessment of PD parameter: change in bone-specific alkaline phosphataseNot SpecifiedBaseline to Week 26 and Week 52
    Assessment of PD parameter: change in procollagen type 1 N-terminal propeptide (P1NP) levelsNot SpecifiedBaseline to Week 26 and Week 52
    Assessment of PD parameter: change in collagen-type 1 C-Telopeptide (CTX) levelsNot SpecifiedBaseline to Week 26 and Week 52
    Number of participants with treatment-emergent anti-drug antibodies (ADA)Not SpecifiedBaseline to Week 26 and Week 52
    Changes in neurological examinationPercentage of participants with changes (i.e. abnormal to normal or normal to abnormal) in neurological examination findingsBaseline through Week 26 and Week 52

    Frequently Asked Questions

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    References

    Clinical Trials Gov: Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of SAR442501 in Pediatric Participants With Achondroplasia

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