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BN44621 Hinalea 2

  • Status
    Accepting Candidates
  • Age
    3 Months - 24 Months
  • Sexes
    All
  • Healthy Volunteers
    No
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Objective

This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene abeparvovec and experience a plateau or decline in function. Participants to be enrolled are children

Details

Full study title A Phase IV Open-Label Study Evaluating The Effectiveness And Safety of Risdiplam Administered In Pediatric Patients With Spinal Muscular Atrophy Who Experienced A Plateau Or Decline In Function After Gene Therapy
Protocol number OCR45770
ClinicalTrials.gov ID NCT05861999
Phase Phase 4

Eligibility

Inclusion Criteria:

-

Lead researcher

  • Carmen Leon Astudillo, MD
    Pediatric Neuromuscular Rehabilitation Specialist, Pediatric Pulmonologist (Child Lung Specialist)
    Carmen Leon Astudillo

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  1. Step
    1

    Contact the research team

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    Primary contact

  2. Step
    2

    Get screened to confirm eligibility

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  3. Step
    3

    Provide your consent to participate

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  4. Step
    4

    Participate

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