Registry for patients with achondroplasia and hypochondroplasia

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational registry collects clinical data from patients diagnosed with achondroplasia and hypochondroplasia. The aim is to gather comprehensive information that can help in understanding these conditions better. By documenting patient experiences and clinical outcomes, the registry seeks to support future research and treatment approaches. Participation involves providing informed consent and sharing relevant health information.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Confirmed diagnosis of achondroplasia/hypochondroplasia
* Patients (and/or Parents legal guardian when required) able to provide informed consent

Exclusion Criteria:

* Absence of diagnosis of achondroplasia/hypochondroplasia
* Patients (and/or Parents legal guardian when required) not able to provide informed consent

Where it's running

• Medical Genetics Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico Milan
  Milan, Italy Recruiting

Study contacts

• Study coordinator: Maria Francesca Bedeschi, MD
• Email: mariafrancesca.bedeschi@policlinico.mi.it
• Phone: +390255032150

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Measuring limb movement in people with FGFR3-related skeletal dysplasia

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• BMN 333 versus daily vosoritide for growth in children with achondroplasia

  This trial tests whether weekly BMN 333 injections help children with achondroplasia grow as well as daily vosoritide.

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• Observing children with achondroplasia over time

  Collecting growth, clinical, and quality-of-life information from children with achondroplasia aged 2.5 to under 11 to see how the condition and treatments affect them over 6 months to 2 years.

  Recruiting at 3 sites Abbisko Therapeutics Co, Ltd · Beijing, Beijing Municipality and 7 other locations · NCT07301463

• Infigratinib treatment for children under 3 with achondroplasia

  This trial tests whether daily oral infigratinib helps children younger than 3 years who have achondroplasia.

  Recruiting at 12 sites Phase 2 QED Therapeutics, a BridgeBio company · Oakland, California and 11 other locations · NCT07169279

• Daily vosoritide versus placebo for infants and toddlers with hypochondroplasia

  This trial will test whether daily vosoritide injections for 52 weeks help infants and toddlers under 36 months with genetically confirmed hypochondroplasia grow better and stay healthier.

  Recruiting at 17 sites Phase 2 BioMarin Pharmaceutical · Phoenix, Arizona and 25 other locations · NCT07126262

• Evaluating TYRA-300 for children with achondroplasia

  This study is testing a new medication called TYRA-300 to see if it is safe and helps children aged 3 to 10 with achondroplasia grow better.

  Recruiting at 25 sites Phase 2 Tyra Biosciences, Inc · Torrance, California and 24 other locations · NCT06842355

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.