Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia.

Achondroplasia Clinical Trial

Official title:

An International, Prospective Registry Investigating the Natural History of Participants With Achondroplasia

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT03794609
• Other study ID #: TA46-002
• Secondary ID: C4181001
• Status: Terminated
• Start date: June 15, 2018
• Est. completion date: January 13, 2023

Study information

• Verified date: July 2023
• Source: Pfizer
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

This is a registry study in children with achondroplasia, age 0-10 years, to be conducted at multiple clinical centers in several countries. Information collected will include in anthropometric characteristics, related symptoms, tests, & treatments Children's information will be collected in the registry for a maximum of 5 years.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 315
• Est. completion date: January 13, 2023
• Est. primary completion date: January 13, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 0 Years to 15 Years

Eligibility

Inclusion Criteria:

1. Written informed consent is obtained from the children's parent(s) / legal guardian(s) before any study-related activity is carried out

2. The child is able to provide written informed assent, where this is required according to national legislation, before any study related activity is carried out

3. The child has been diagnosed as having achondroplasia documented by clinical diagnosis

4. The child is between 0 years and 10 years of age, inclusive, on the date of consent / assent

5. The investigator has considered the family and prospective participating child being able to comply with the study procedures

Exclusion Criteria:

1. The child has a diagnosis of hypochondroplasia or any short stature condition other than achondroplasia (eg, spondyloepiphyseal dysplasia congenital [SEDC], pseudoachondroplasia, trisomy 21)

2. The child has any medical condition that may impact growth or where the treatment is known to impact growth, such as but not limited to hypothyroidism or hyperthyroidism, insulin-requiring diabetes mellitus, autoimmune inflammatory disease (including celiac disease, systemic lupus erythematosus [SLE], juvenile dermatomyositis, scleroderma, and others), autonomic neuropathy, or inflammatory bowel disease

3. Treatment in the previous 12 months prior to consent and assent with growth hormone, insulin-like growth factor 1 (IGF-1), anabolic steroids, or any other drug expected to affect growth velocity

4. Any surgery that affects the growth plate of the long bones that is planned, or has occurred in the past 18 months

5. Participation in any interventional study (investigational product or device) for treatment of achondroplasia or short stature

6. Has had bone-related surgery impacting assessment of anthropometric measurements or is expected to have it during the study period. Children with previous limb-lengthening surgery may enroll if surgery occurred at least 18 months prior to the date of consent/assent and healing is complete without sequelae as determined by the investigator

7. Has any condition that in the view of the investigator places the child at high risk of poor compliance with the visit schedule or of not completing the study.

8. Any concurrent disease or condition that in the view of the investigator would interfere with study participation

Related Conditions & MeSH terms

• Achondroplasia

Locations

Country	Name	City	State
Australia	Childrens Hospital Melbourne	Melbourne
Australia	Murdoch Childrens Research Institute	Parkville	Victoria
Belgium	Antwerp University Hospital	Antwerp
Belgium	Universitair Ziekenhuis Antwerpen (UZA)	Edegem
Canada	CHU Sainte-Justine	Montreal	Quebec
Canada	The Hospital for Sick Children	Toronto
Canada	The Hospital for Sick Children	Toronto	Ontario
China	Beijing Children's Hospital, Capital Medical University	Beijing
China	The First Affiliated Hospital, Sun Yat-sen University	Guangzhou	Guangdong
Denmark	Bispebjerg Hospital	Copenhagen
Denmark	Righospitalet	Copenhagen
France	Centre Hospitalier Universitaire La Timone	Marseille
France	Centre Hospitalier Univesitaire La Timone	Marseille
France	Hopital Necker-Enfants Malades	Paris
Germany	Center for Rare Skeletal Diseases in Childhood and Adolescence	Cologne
Germany	University Hospital of Cologne	Cologne
Germany	Otto-von-Guericke- Universitat Magdeburg	Magdeburg
Germany	Otto-von-Guericke-Universitat Magdeburg	Magdeburg
Italy	ASST Lariana Como	Como
Italy	IRCCS Istituto Giannina Gaslini	Genova
Italy	IRCCS Istituto Giannina Gaslini	Genova	Genoa
Italy	San Raffaele Hospital	Milan
Italy	Center for Rare Diseases, Department of Pediatrics, Polo Salute Donna e Bambino	Rome
Italy	Fondazione Policlinico Universitario A.Gemelli	Rome
Italy	ASST Lariana Como	San Fermo della Battaglia	Como
Japan	Osaka Women's and Children's Hospital	Izumi	Osaka
Japan	Okayama University Hospital	Okayama
Japan	Osaka University Hospital	Suita	Osaka
Portugal	Hospital Pediatrico de Coimbra	Coimbra
Portugal	Hospital Pediátrico de Coimbra	Coimbra
Spain	Vall D'Hebron Univeristy Hospital	Barcelona
Spain	Vall d'Hebron University Hospital	Barcelona
Spain	Hospital Quironsalud Malaga	Málaga
Switzerland	Centre Hospitalier Universitaire Vaudois -CHUV	Lausanne
Switzerland	CENTRE HOSPITALIER UNIVERSITAIRE VAUDOIS -CHUV Hôpital Neslé	Lausanne
United Kingdom	Bristol Royal Childrens Hospital	Bristol
United Kingdom	University Hospitals Bristol NHS Foundation Trust	Bristol
United Kingdom	Guy's & St Thomas NHS Trust, St Thomas' Hospital	London
United Kingdom	Guys & St Thomas NHS Trust	London
United Kingdom	Newcastle Hospital NHS Foundation Trust	Newcastle
United Kingdom	Newcastle Hospitals NHS Foundation Trust	Newcastle upon Tyne
United Kingdom	Sheffield Children's NHS Foundation Trust	Sheffield
United Kingdom	Sheffield Childrens NHS Foundation Trust	Sheffield
United States	The John Hopkins	Baltimore	Maryland
United States	The Johns Hopkins Hospital	Baltimore	Maryland
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Boston Childrens Hospital	Boston	Massachusetts
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	Univesity of Missouri - Columbia	Columbia	Missouri
United States	Texas Children Hospital	Houston	Texas
United States	Texas Children'S Hospital	Houston	Texas
United States	Los Angeles Biomedical Research Institute At Harbour-UCLA Medical Centre	Los Angeles	California
United States	University of Utah Health	Salt Lake City	Utah
United States	Seattle Children's Hospital	Seattle	Washington
United States	Los Angeles BioMedical Research Institute at Harbor-UCLA Medical Center	Torrance	California
United States	Alfred I. duPont Hospital for Children	Wilmington	Delaware
United States	Alfred I. duPont Hospital for Children - Wilmington	Wilmington	Delaware

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Countries where clinical trial is conducted

United States,  Australia,  Belgium,  Canada,  China,  Denmark,  France,  Germany,  Italy,  Japan,  Portugal,  Spain,  Switzerland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Collection of Natural History of Achondroplasia Characteristics in a cohort of Children aged 0-10 years old diagnosed with Achondroplasia	To quantify the number & type of Achondroplasia Characteristics by review of medical records	Up to 5 Years
Primary	Collection of Natural History of Achondroplasia Symptoms in a cohort of Children aged 0-10 years old diagnosed with Achondroplasia	To quantify the number & type of Achondroplasia Symptoms by review of medical records	Up to 5 Years
Primary	Collection of Natural History of Achondroplasia related Tests & Treatments in a cohort of Children aged 0-10 years old diagnosed with Achondroplasia	To quantify the number & type of Achondroplasia related Tests & Treatments by review of medical records	Up to 5 Years
Secondary	Measurement of biomarkers for bone growth	changes from Baseline in blood samples of collagen fragments	Baseline, Month 12, Month 24, Month 36, Month 48, Month 60

External Links

•   To obtain contact information for a study center near you, click here.