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

Sponsor

Pfizer (Industry)

Overall Status

Recruiting

CT.gov ID

NCT03794609

Collaborator

(none)

200

Enrollment

Locations

122.7

Anticipated Duration (Months)

5.7

Patients Per Site

Patients Per Site Per Month

Study Details

Study Description

Brief 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.

Condition or Disease	Intervention/Treatment	Phase
Achondroplasia

Study Design

Study Type:

Observational

Anticipated Enrollment :

200 participants

Observational Model:

Case-Only

Time Perspective:

Prospective

Official Title:

A Multi-center, Prospective, Longitudinal, Observational Study to Investigate the Clinical and Anthropometric Characteristics of Children With the Diagnosis Achondroplasia.

Actual Study Start Date :

Jun 15, 2018

Anticipated Primary Completion Date :

Sep 5, 2028

Anticipated Study Completion Date :

Sep 6, 2028

Outcome Measures

Primary Outcome Measures

Collection of Natural History of Achondroplasia Characteristics in a cohort of Children aged 0-10 years old diagnosed with Achondroplasia [Up to 5 Years]
To quantify the number & type of Achondroplasia Characteristics by review of medical records
Collection of Natural History of Achondroplasia Symptoms in a cohort of Children aged 0-10 years old diagnosed with Achondroplasia [Up to 5 Years]
To quantify the number & type of Achondroplasia Symptoms by review of medical records
Collection of Natural History of Achondroplasia related Tests & Treatments in a cohort of Children aged 0-10 years old diagnosed with Achondroplasia [Up to 5 Years]
To quantify the number & type of Achondroplasia related Tests & Treatments by review of medical records

Secondary Outcome Measures

Measurement of biomarkers for bone growth [Baseline, Month 12, Month 24, Month 36, Month 48, Month 60]
changes from Baseline in blood samples of collagen fragments

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A to 10 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Written informed consent is obtained from the children's parent(s) / legal guardian(s) before any study-related activity is carried out
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
The child has been diagnosed as having achondroplasia documented by clinical diagnosis
The child is between 0 years and 10 years of age, inclusive, on the date of consent / assent
The investigator has considered the family and prospective participating child being able to comply with the study procedures

Exclusion Criteria:

The child has a diagnosis of hypochondroplasia or any short stature condition other than achondroplasia (eg, spondyloepiphyseal dysplasia congenital [SEDC], pseudoachondroplasia, trisomy 21)
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
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
Any surgery that affects the growth plate of the long bones that is planned, or has occurred in the past 18 months
Participation in any interventional study (investigational product or device) for treatment of achondroplasia or short stature
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
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.
Any concurrent disease or condition that in the view of the investigator would interfere with study participation

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Los Angeles BioMedical Research Institute at Harbor-UCLA Medical Center	Torrance	California	United States	90502
2	Alfred I. duPont Hospital for Children - Wilmington	Wilmington	Delaware	United States	419803-3607
3	The Johns Hopkins Hospital	Baltimore	Maryland	United States	21287
4	Boston Children's Hospital	Boston	Massachusetts	United States	02115
5	University of Missouri - Columbia	Columbia	Missouri	United States	65203
6	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio	United States	45229
7	Texas Children Hospital	Houston	Texas	United States	77030
8	University of Utah Health	Salt Lake City	Utah	United States	84132
9	Seattle Children's Hospital	Seattle	Washington	United States	98105
10	Murdoch Childrens Research Institute	Parkville	Victoria	Australia	3052
11	Antwerp University Hospital	Antwerp		Belgium	2650
12	The Hospital for Sick Children	Toronto	Ontario	Canada	M5G 1X8
13	CHU Sainte-Justine	Montreal	Quebec	Canada	H3T 1C5
14	The First Affiliated Hospital, Sun Yat-sen University	Guangzhou	Guangdong	China	510080
15	Beijing Children's Hospital, Capital Medical University	Beijing		China	100045
16	Bispebjerg Hospital	Copenhagen		Denmark	2400
17	Centre Hospitalier Univesitaire La Timone	Marseille		France	13385
18	Hopital Necker-Enfants Malades	Paris		France	75015
19	Center for Rare Skeletal Diseases in Childhood and Adolescence	Cologne		Germany	50937
20	Otto-von-Guericke-Universitat Magdeburg	Magdeburg		Germany	39120
21	ASST Lariana Como	San Fermo della Battaglia	Como	Italy	22042
22	IRCCS Istituto Giannina Gaslini	Genova	Genoa	Italy	16147
23	San Raffaele Hospital	Milan		Italy	20132
24	Center for Rare Diseases, Department of Pediatrics, Polo Salute Donna e Bambino	Rome		Italy	00168
25	Osaka Women's and Children's Hospital	Izumi	Osaka	Japan	594-1101
26	Osaka University Hospital	Suita	Osaka	Japan	565-0871
27	Okayama University Hospital	Okayama		Japan	700-8558
28	Hospital Pediátrico de Coimbra	Coimbra		Portugal	3000-602
29	Vall d'Hebron University Hospital	Barcelona		Spain	08035
30	Hospital Quirónsalud Málaga	Malaga		Spain	29004
31	Centre Hospitalier Universitaire Vaudois -CHUV	Lausanne		Switzerland	1011
32	University Hospitals Bristol NHS Foundation Trust	Bristol		United Kingdom	BS2 8BJ
33	Guy's & St Thomas NHS Trust, St Thomas' Hospital	London		United Kingdom	SE1 7EH
34	Newcastle Hospitals NHS Foundation Trust	Newcastle upon Tyne		United Kingdom	NE1 3BZ
35	Sheffield Children's NHS Foundation Trust	Sheffield		United Kingdom	S10 2TH

Sponsors and Collaborators

Pfizer

Investigators

Study Director: Pfizer CT.gov Call Center, Pfizer

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

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

Publications

None provided.

Responsible Party:

Pfizer

ClinicalTrials.gov Identifier:

NCT03794609

Other Study ID Numbers:

TA46-002
C4181001

First Posted:

Jan 7, 2019

Last Update Posted:

Aug 9, 2022

Last Verified:

Aug 1, 2022

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Achondroplasia

Study Results

No Results Posted as of Aug 9, 2022