Recifercept for Achondroplasia

"This clinical trial is enrolling 63 participants, aged between three months and ten years, that have been diagnosed with achondroplasia. In addition to this criteria, the patients must meet additional requirements: they must be at Tanner stage 1 based on physical examination; capable of standing independently for height measurements (if two or older); if less than 2 years old at enrollment then an MRI brain/cervical spine needs to be documented within one year; completion of the C4181001 natural history study with valid measurements taken 3 months apart prior to enrollment in this trial."

"Accurate. Per clinicaltrials.gov, this trial is presently recruiting volunteers and was initially listed on December 2nd 2020 with the latest update occurring November 17th 2022."

"This clinical trial's primary objective, measured between the Baseline and Day 57 time frames is to assess Height. Further, secondary goals include evaluating Number of Participants With Laboratory Abnormalities (hematology & blood chemistry), Change from Baseline in Lower Leg Length, as well as Number of Participants With Change From Baseline in Vital Signs (blood pressure, respiration rate, radial pulse and body temperature)."

"This trial is seeking participants aged 3 months to 10 years old. In total, 12 studies are available for minors and three trials have been developed specifically with elderly patients in mind."

"Our experts at Power judged the safety of Recifercept to be a 2, as this is an early-stage trial with evidence for its protection, but no data attesting to its efficacy."