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NCT05408715: ROCKET

A Natural History Study in Children With a Type II Collagen Disorder With Short Stature

Recruiting now Last updated 2 October 2023
What this trial tests

trial testing Natural History Study in SEDC in 60 participants. Currently enrolling.

Timeline
29 June 2022
Primary endpoint
31 December 2026
31 December 2026

Quick facts

Lead sponsorInnoskel
StatusRecruiting now
Study typeOBSERVATIONAL
Enrollment60
Start date29 June 2022
Primary completion31 December 2026
Estimated completion31 December 2026
Sites2 locations across France, Spain

Drugs / interventions tested

Conditions studied

Sponsor

Innoskel

Who can join

Under 12, any sex, with SEDC or Hypochondrogenesis. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

There are relatively few data available on type II collagen disorders, and evidence is lacking on the disease course in relation to symptoms and development of complications, the level of actual disease burden over time as well as data to support identification of possible risk factors. This study aims to build a natural history data set through collection of a number of clinical, imaging, and laboratory assessments that may be specific predictors of type II collagen disorder progression and clinical outcome. Having a type II collagen disorder natural history data set can inform potential efficacy endpoints and biomarkers for future clinical trials. This natural history study will follow up to 60 individuals diagnosed with a type II collagen disorder for up to 3 years. Visits will be conducted every 3 months for the first year and then every 6 months, during which several assessments will be performed in order to learn about the natural course of the disease, including changes in clinical and functional outcomes, imaging and biofluid biomarkers. Some of the study activities include: a physical exam, height measurements, vision and breathing tests and x-ray. A blood sample will be collected once or twice each year. Most of the information collected, the tests done, and the schedule of visits in this study are the same as recommended for regular care of children with a type II collagen disorder.

Publications & conference data

No peer-reviewed publications indexed yet for this trial.

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