A Multicenter Multinational Observational Study of Children With

See also
Status	Clinical Trial	Phase
Recruiting	`NCT05328050` - Registry for Patients With Achondroplasia / Hypochondroplasia (OMPR-Ach/Hy)
Completed	`NCT01111019` - Efficacy and Safety Evaluation of Recombinant Human Growth Hormone (r-hGH), Saizen®, on a Population of Children With Hypochondroplasia, Treated at Least 3 Years or Until Near Final Height, When Applicable, in Comparison With a Historic Cohort of Non-treated Children	Phase 2
Completed	`NCT01541306` - C-Type Natriuretic Peptide and Achondroplasia	N/A

See also

Status

Clinical Trial

Phase

Recruiting

NCT05328050 - Registry for Patients With Achondroplasia / Hypochondroplasia (OMPR-Ach/Hy)

Completed

NCT01111019 - Efficacy and Safety Evaluation of Recombinant Human Growth Hormone (r-hGH), Saizen®, on a Population of Children With Hypochondroplasia, Treated at Least 3 Years or Until Near Final Height, When Applicable, in Comparison With a Historic Cohort of Non-treated Children

Phase 2

Completed

NCT01541306 - C-Type Natriuretic Peptide and Achondroplasia

N/A

Interventional studies are often prospective and are specifically tailored to evaluate direct impacts of treatment or preventive measures on disease.

Observational studies are often retrospective and are used to assess potential causation in exposure-outcome relationships and therefore influence preventive methods.

Expanded access is a means by which manufacturers make investigational new drugs available, under certain circumstances, to treat a patient(s) with a serious disease or condition who cannot participate in a controlled clinical trial.

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

NCT number	NCT06212947
Study type	Observational
Source	BioMarin Pharmaceutical
Contact	Trial Specialist
Phone	1-800-938-4587
Email	medinfo@bmrn.com
Status	Recruiting
Phase
Start date	November 27, 2023
Completion date	December 31, 2043

A Multicenter Multinational Observational Study of Children With Hypochondroplasia

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms