Actively Recruiting
A Multicenter Multinational Observational Study of Children With Hypochondroplasia
Led by BioMarin Pharmaceutical · Updated on 2025-04-03
400
Participants Needed
43
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
This research aims to observe and track growth patterns and the clinical progression of Hypochondroplasia (HCH) in children. It focuses on collecting various growth measurements and other relevant data over time to better understand this condition. The study is sponsored by BioMarin Pharmaceutical and involves children diagnosed with Hypochondroplasia, up to 15 years old. Participants include children confirmed to have Hypochondroplasia through genetic testing. The study is observational, meaning there are no experimental treatments involved. Children will be monitored regularly to record changes in growth and body proportions, with data collection occurring every six months over the course of the study, which may last up to 15 years. Throughout the study, children will undergo assessments every six months to measure growth velocity, height, body mass index (BMI), and body segment ratios. Quality of life and severity impressions from both patients and caregivers will be evaluated annually. Researchers will also track medical events, use of growth hormone treatments, and any limb lengthening surgeries. This long-term follow-up allows for comprehensive monitoring of growth and health outcomes in children with Hypochondroplasia.
CONDITIONS
Brief Title
A Multicenter Multinational Observational Study of Children With Hypochondroplasia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants must be 64 15 years old at the time of signing the informed consent
- Participants must have genetic confirmation of Hypochondroplasia diagnosis
You will not qualify if you...
- Have a diagnosis of another genetic short stature condition other than Hypochondroplasia or a genetic variant known to cause another genetic syndrome associated with short stature
- Received an investigational product or medical device within 6 months before the Screening visit
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 15 years
Participants are observed with assessments every 6 months to track growth and health changes over time.
Visits every 6 months with additional yearly assessments
Trial Site Locations
Total: 43 locations
1
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
Actively Recruiting
2
Cedars-Sinai Medical Center
Los Angeles, California, United States, 90048
Not Yet Recruiting
3
UCSF Benioff Children's Hospital
Oakland, California, United States, 94609
Actively Recruiting
4
Nemours Alfred I. Dupont Hospital for Children
Wilmington, Delaware, United States, 19803
Actively Recruiting
5
Children's National Health System
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
6
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
7
The Johns Hopkins University School of Medicine
Baltimore, Maryland, United States, 21218
Not Yet Recruiting
8
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
9
University of Minnesota Masonic Children's Hospital
Minneapolis, Minnesota, United States, 55454
Actively Recruiting
10
University of Missouri
Columbia, Missouri, United States, 65212
Actively Recruiting
11
Washington University School of Medicine in St. Louis
St Louis, Missouri, United States, 63110
Actively Recruiting
12
Mount Sinai Kravis Children's Hospital
New York, New York, United States, 10029
Actively Recruiting
13
Akron Children's Hospital
Akron, Ohio, United States, 44307
Not Yet Recruiting
14
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Not Yet Recruiting
15
Cook Children's Endocrinology
Fort Worth, Texas, United States, 76104
Actively Recruiting
16
Medical College of Wisconsin, Children's Hospital
Milwaukee, Wisconsin, United States, 53226
Actively Recruiting
17
Hospital de Pediatria SAMIC Prof. Dr. Juan P Garrahan
Buenos Aires, Argentina
Not Yet Recruiting
18
Royal Children's Hospital Melbourne
Parkville, Victoria, Australia, 3052
Actively Recruiting
19
Hospital de Clinicas de Porto Algre
Porto Alegre, Rio Grande Do Sol, Brazil
Not Yet Recruiting
20
Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto
Ribeirão Preto, São Paulo, Brazil
Not Yet Recruiting
21
Stollery Children's Hospital
Edmonton, Alberta, Canada
Actively Recruiting
22
SickKids - The Hospital for Sick Children
Toronto, Ontario, Canada
Actively Recruiting
23
Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec, Canada
Not Yet Recruiting
24
Hospices Civils de Lyon - Hôpital Femme Mère Enfant
Lyon, France
Actively Recruiting
25
Hopital de la Timone
Marseille, France
Actively Recruiting
26
Hôpital Necker-Enfants Malades
Paris, France
Actively Recruiting
27
CHU de Toulouse - Hopital des Efants
Toulouse, France
Actively Recruiting
28
Uniklinik Koln
Cologne, Germany
Actively Recruiting
29
Universitatskinderklinik Magdeburg
Magdeburg, Germany
Actively Recruiting
30
Istituto Giannina Gaslini
Genoa, Liguria, Italy
Actively Recruiting
31
Ospedale Pediatrico Bambino Gesù
Roma, Rome, Italy
Not Yet Recruiting
32
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, Italy
Actively Recruiting
33
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Rome, Italy
Actively Recruiting
34
Kumamoto University Hospital
Kumamoto, Kumamoto, Japan
Actively Recruiting
35
Osaka University Hospital
Suita, Osaka, Japan
Actively Recruiting
36
Tottori University Hospital
Yonago, Tottori, Japan
Actively Recruiting
37
Tokushima University Hospital
Tokushima, Japan
Not Yet Recruiting
38
Institute of Science Tokyo Hospital
Tokyo, Japan
Actively Recruiting
39
Hospital Universitario La Paz
Madrid, Spain
Actively Recruiting
40
Vithas Hospital San Jose
Vitoria-Gasteiz, Spain
Actively Recruiting
41
Great Ormond Street Hospital for Children NHS Foundation Trust
London, England, United Kingdom
Actively Recruiting
42
Myriad Trials Ltd
London, England, United Kingdom
Not Yet Recruiting
43
NHS Greater Glasgow and Clyde
Glasgow, Scottland, United Kingdom
Actively Recruiting
Research Team
T
Trial Specialist
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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