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Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)
NCT04035811 · View on ClinicalTrials.gov ↗
Study Summary
This is a long-term, multi-center, observational study in children 2.5 to \<17 years with achondroplasia (ACH). The objective is to evaluate growth, ACH-related medical complications, assessments of health-related quality of life, body pain, functional abilities, cognitive functions, and treatments of study participants. No study medication will be administered.
Conditions Studied
Study Locations (20)
Other
- Hospital de Pediatría SAMIC Prof. Dr. Juan P. Garrahan — Buenos Aires
- Murdoch Children's Research Institute — Parkville
- Hopital Femme Mere Enfant — Lyon
- Hopital Necker-Enfants Malades — Paris
- Hopital des Enfants — Toulouse
- Otto-von-Guericke-University Magdeburg Medical Fakulty — Magdeburg
- Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlino — Milan
- Rare Disease Unit Fondazione Policlinico A Gemelli IRCCS — Rome
Ontario
- Children's Hospital - London Health Sciences Center — London
- University of Ottawa — Ottawa
California
- Benioff Children's Hospital Oakland — Oakland
Colorado
- Children's Hospital Colorado — Aurora
Delaware
- Nemours Alfred I. Dupont Hospital for Children — Wilmington
Maryland
- Johns Hopkins School of Medicine — Baltimore
Missouri
- University of Missouri — Columbia
Ohio
- Cincinnati Children's Hospital — Cincinnati
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 271 participants |
| Start Date | 2019-08-12 |
| Est. Completion | 2026-06 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04035811
The ClinicalTrials.gov registry entry for NCT04035811 describes a study currently listed as active not recruiting. It is categorized as an unspecified phase, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 271 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is QED Therapeutics, a BridgeBio company, which has 3 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Achondroplasia appearing as the primary indexed condition, and to 0 interventions. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT04035811 reports 20 study locations spanning 12 distinct geographic areas — top geographies include Other, Ontario, California. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT04035811 about?
NCT04035811 is a clinical study titled "Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)". This is a long-term, multi-center, observational study in children 2.5 to \<17 years with achondroplasia (ACH). The objective is to evaluate growth, ACH-related medical complications, assessments of health-related quality of life, body pain, functional abilities, cognitive functions, and treatments ...
What is the current status of trial NCT04035811?
This trial is currently active not recruiting. The enrollment target is 271 participants. The study started on 2019-08-12. Estimated completion is 2026-06.
What conditions does trial NCT04035811 study?
This clinical trial studies the following conditions: Achondroplasia. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
Who is sponsoring clinical trial NCT04035811?
This trial is sponsored by QED Therapeutics, a BridgeBio company, which has 3 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT04035811 being conducted?
This trial has 20 study locations across California, Colorado, Delaware, Maryland, Missouri. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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