ACHONDROPLASIA
Clinical trials for ACHONDROPLASIA explained in plain language.
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Could this pill help kids with dwarfism grow faster?
Disease control CompletedThis study tested a drug called infigratinib in 114 children aged 3 to 18 with achondroplasia, the most common form of dwarfism. The goal was to see if it could safely increase their growth rate over one year compared to a placebo. Participants had already been in an earlier obse…
Matched conditions: ACHONDROPLASIA
Phase: PHASE3 • Sponsor: QED Therapeutics, a BridgeBio company • Aim: Disease control
Last updated May 17, 2026 06:52 UTC
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Weekly shot shows promise for boosting growth in kids with dwarfism
Disease control CompletedThis study tested a weekly injection called TransCon CNP in 84 children aged 2 to 11 with achondroplasia, the most common form of dwarfism. The goal was to see if it safely improves growth rate over a year compared to a placebo. After the blinded phase, all children could receive…
Matched conditions: ACHONDROPLASIA
Phase: PHASE2, PHASE3 • Sponsor: Ascendis Pharma Growth Disorders A/S • Aim: Disease control
Last updated May 15, 2026 11:58 UTC
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Could this pill help kids with dwarfism grow faster?
Disease control CompletedThis study tested a drug called infigratinib in 84 children aged 3 to 11 with achondroplasia, the most common form of dwarfism. The goal was to see if the drug could safely increase growth rate by targeting a specific protein linked to the condition. Participants took the drug da…
Matched conditions: ACHONDROPLASIA
Phase: PHASE2 • Sponsor: QED Therapeutics, a BridgeBio company • Aim: Disease control
Last updated May 15, 2026 11:56 UTC
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No treatment, just tracking: PROPEL study follows kids with achondroplasia
Knowledge-focused CompletedThis study followed 271 children with achondroplasia (ages 2.5 to 17) to understand their growth, medical issues, and quality of life over time. No medication was given. The goal was to gather natural history data to help design future treatments.
Matched conditions: ACHONDROPLASIA
Sponsor: QED Therapeutics, a BridgeBio company • Aim: Knowledge-focused
Last updated May 17, 2026 06:50 UTC