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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03794609




Registration number
NCT03794609
Ethics application status
Date submitted
19/11/2018
Date registered
7/01/2019
Date last updated
21/04/2020

Titles & IDs
Public title
Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia.
Scientific title
A Multi-center, Prospective, Longitudinal, Observational Study to Investigate the Clinical and Anthropometric Characteristics of Children With the Diagnosis Achondroplasia.
Secondary ID [1] 0 0
C4181001
Secondary ID [2] 0 0
TA46-002
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Achondroplasia 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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
Timepoint [1] 0 0
Up to 5 Years
Primary outcome [2] 0 0
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
Timepoint [2] 0 0
Up to 5 Years
Primary outcome [3] 0 0
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
Timepoint [3] 0 0
Up to 5 Years
Secondary outcome [1] 0 0
Measurement of biomarkers for bone growth - changes from Baseline in blood samples of collagen fragments
Timepoint [1] 0 0
Baseline, Month 12, Month 24, Month 36, Month 48, Month 60

Eligibility
Key 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
Minimum age
No limit
Maximum age
10 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Childrens Hospital Melbourne - Melbourne
Recruitment postcode(s) [1] 0 0
- Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Delaware
Country [3] 0 0
United States of America
State/province [3] 0 0
Maryland
Country [4] 0 0
United States of America
State/province [4] 0 0
Massachusetts
Country [5] 0 0
United States of America
State/province [5] 0 0
Missouri
Country [6] 0 0
United States of America
State/province [6] 0 0
Ohio
Country [7] 0 0
United States of America
State/province [7] 0 0
Texas
Country [8] 0 0
United States of America
State/province [8] 0 0
Utah
Country [9] 0 0
United States of America
State/province [9] 0 0
Washington
Country [10] 0 0
Belgium
State/province [10] 0 0
Antwerp
Country [11] 0 0
Canada
State/province [11] 0 0
Toronto
Country [12] 0 0
Denmark
State/province [12] 0 0
Copenhagen
Country [13] 0 0
France
State/province [13] 0 0
Marseille
Country [14] 0 0
France
State/province [14] 0 0
Paris
Country [15] 0 0
Germany
State/province [15] 0 0
Cologne
Country [16] 0 0
Germany
State/province [16] 0 0
Magdeburg
Country [17] 0 0
Italy
State/province [17] 0 0
Como
Country [18] 0 0
Italy
State/province [18] 0 0
Genova
Country [19] 0 0
Italy
State/province [19] 0 0
Milan
Country [20] 0 0
Italy
State/province [20] 0 0
Rome
Country [21] 0 0
Portugal
State/province [21] 0 0
Coimbra
Country [22] 0 0
Spain
State/province [22] 0 0
Barcelona
Country [23] 0 0
Spain
State/province [23] 0 0
Málaga
Country [24] 0 0
Switzerland
State/province [24] 0 0
Lausanne
Country [25] 0 0
United Kingdom
State/province [25] 0 0
Bristol
Country [26] 0 0
United Kingdom
State/province [26] 0 0
London
Country [27] 0 0
United Kingdom
State/province [27] 0 0
Newcastle
Country [28] 0 0
United Kingdom
State/province [28] 0 0
Sheffield

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Pfizer
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/show/NCT03794609
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Pfizer CT.gov Call Center
Address 0 0
Pfizer
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Pfizer CT.gov Call Center
Address 0 0
Country 0 0
Phone 0 0
1-800-718-1021
Fax 0 0
Email 0 0
ClinicalTrials.gov_Inquiries@pfizer.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT03794609