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Questionário sobre acondroplasia
Desenhámos um questionário com o objetivo de conhecer a população ligada à acondroplasia.
O questionário é opcional e anónimo. Os dados obtidos serão usados para cálculos estatísticos e para perceber melhor a história natural da acondroplasia.
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ClinicalTrials.gov Identifier: NCT04265651

Phase 2 trial, multicenter, open-label, dose-escalation, and dose-expansion study to evaluate the safety, tolerability, and efficacy of infigratinib, a fibroblast growth factor receptor (FGFR) 1-3-selective tyrosine kinase inhibitor in children 3 to 11 years of age with Achondroplasia (ACH) who previously participated in the PROPEL study (Protocol QBGJ398-001) for at least 6 months. The study includes dose escalation with extended treatment and dose expansion.

Actual Study Start Date  : March 10, 2020
Estimated Primary Completion Date  : October 2021
Estimated Study Completion Date  : February 2023


Ages Eligible for Study:   3 Years to 11 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

 

Inclusion Criteria:

  1. Signed informed consent by participant or parent(s) or legally authorized representative (LAR) and signed informed assent by the participant (when applicable).
  2. Diagnosis of ACH documented clinically and confirmed by genetic testing.
  3. At least a 6-month period of growth assessment in the PROPEL study (Protocol QBGJ398-001) before study entry.
  4. Ambulatory and able to stand without assistance
  5. Able to swallow oral medication.

Recruitment centers
Australia, Victoria
Murdoch Children's Hosptial  
Parkville, Victoria, Australia, 3052

Contact: QED Therapeutics Senior Medical Director 1-877-280-5655 Este endereço de email está protegido contra piratas. Necessita ativar o JavaScript para o visualizar.
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Obrigado.