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Prospective Clinical Assessment Study in Children With Hypochondroplasia
This is a long-term, multicenter, non-interventional study of children ages 2.5 to \<17 years with hypochondroplasia (HCH).
Conditions:
🦠 Hypochondroplasia
🗓️ Study Start (Actual) 5 June 2024
🗓️ Primary Completion (Estimated) October 2026
✅ Study Completion (Estimated) October 2026
👥 Enrollment (Estimated) 150
🔬 Study Type OBSERVATIONAL
📊 Phase N/A
Locations:
📍 Oakland, California, United States
📍 Aurora, Colorado, United States
📍 Washington, District of Columbia, United States
📍 Baltimore, Maryland, United States
📍 Columbia, Missouri, United States
📍 Nashville, Tennessee, United States
📍 Madison, Wisconsin, United States
📍 Parkville, Victoria, Australia
📍 London, Ontario, Canada
📍 Ottawa, Ontario, Canada
📍 Montréal, Quebec, Canada
📍 Bron, France
📍 Paris, France
📍 Toulouse, France
📍 Freiburg, Baden-Wurtemberg, Germany
📍 Magdeburg, Sachsen-Anhalt, Germany
📍 Bergen, Norway
📍 Oslo, Norway
📍 Coimbra, Portugal
📍 Singapore, Singapore
📍 Vitoria-gasteiz, Spain
📍 Solna, Sweden
📍 Bristol, United Kingdom
📍 London, United Kingdom
📍 Manchester, United Kingdom
📍 Sheffield, United Kingdom
More locations available...

📋 Eligibility Criteria

Description

    Inclusion Criteria:

    • Signed informed consent.
    • Aged 2.5 to \<17 years at study entry.
    • Diagnosis of HCH documented clinically by the presence of disproportionate short stature and confirmed with a molecular test.
    • Participants are ambulatory and able to stand without assistance.
    • Study participants and parent(s), guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures.

    Exclusion Criteria:

    • Have ACH or short stature condition other than HCH.
    • In females, having had their menarche. Annualized height growth velocity ≤1.5 cm/year over a period ≥6 months prior to screening.
    • Having a clinically significant disease or condition that in view of the investigator or Sponsor will interfere with the evaluation of growth, with study participation or not be in the best interest of the participant.
    • Clinically significant abnormality in any laboratory test result at screening
    • Have been treated with growth hormone, IGF 1, or anabolic steroids the previous 6 months or long-term treatment (\>3 months) at any time.
    • Current evidence of corneal or retinal disorders.
    • Have used any other investigational product or investigational medical device for the treatment of HCH or short stature.
    • Have had regular long-term treatment (\>1 month) with oral corticosteroids (low-dose ongoing inhaled steroid for asthma is acceptable).
    • Previous limb-lengthening surgery or guided growth surgery with plates still in place or removed within the 6 months prior to screening.
    • Having had a fracture of the long bones or spine within 12 months of screening.
    • History and/or current evidence of extensive ectopic tissue calcification.
    • History of malignancy.
    • Concurrent circumstance, disease, or condition that, in the view of the investigator and/or sponsor, would interfere with study participation, and/or would place the participant at high risk for poor compliance with study activities or for not completing the study.
Ages Eligible for Study: 30 Months to 16 Years (CHILD)
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers: No

🗓️ Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

  • First Submitted 8 May 2024
  • First Submitted that Met QC Criteria 8 May 2024
  • First Posted 13 May 2024

Study Record Updates

  • Last Update Submitted that Met QC Criteria 19 July 2024
  • Last Update Posted 22 July 2024
  • Last Verified July 2024
I'm interested ...!!