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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT03794609
Ethics application status
Date submitted
19/11/2018
Date registered
7/01/2019

Titles & IDs
Public title
Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia.
Scientific title
An International, Prospective Registry Investigating the Natural History of Participants With 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
Change From Baseline in Standing Height
Timepoint [1] 0 0
Baseline, 1 year, 2 years, 3 years, 4 years
Primary outcome [2] 0 0
Change From Baseline in Sitting Height
Timepoint [2] 0 0
Baseline, 1 year, 2 years, 3 years, 4 years
Primary outcome [3] 0 0
Change From Baseline in Knee Height
Timepoint [3] 0 0
Baseline, 1 year, 2 years, 3 years, 4 years
Primary outcome [4] 0 0
Change From Baseline in Head Circumference
Timepoint [4] 0 0
Baseline, 1 year, 2 years, 3 years, 4 years
Primary outcome [5] 0 0
Change From Baseline in Arm Span
Timepoint [5] 0 0
Baseline, 1 year, 2 years, 3 years, 4 years
Primary outcome [6] 0 0
Number of Participants With Achondroplasia-Related Treatments
Timepoint [6] 0 0
Baseline to end of study visit (up to 55 months)
Secondary outcome [1] 0 0
Baseline and Post-baseline Measurements of Serum Collagen X Marker
Timepoint [1] 0 0
Baseline, 1 year, 2 years, 3 years

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
0 Years
Maximum age
15 Years
Sex
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
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
15/06/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
13/01/2023
Sample size
Target
Accrual to date
Final
315
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Murdoch Childrens Research Institute - Parkville
Recruitment hospital [2] 0 0
Childrens Hospital Melbourne - Melbourne
Recruitment postcode(s) [1] 0 0
3052 - Parkville
Recruitment postcode(s) [2] 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
Belgium
State/province [11] 0 0
Edegem
Country [12] 0 0
Canada
State/province [12] 0 0
Ontario
Country [13] 0 0
Canada
State/province [13] 0 0
Quebec
Country [14] 0 0
Canada
State/province [14] 0 0
Toronto
Country [15] 0 0
China
State/province [15] 0 0
Guangdong
Country [16] 0 0
China
State/province [16] 0 0
Beijing
Country [17] 0 0
Denmark
State/province [17] 0 0
Copenhagen
Country [18] 0 0
France
State/province [18] 0 0
Marseille
Country [19] 0 0
France
State/province [19] 0 0
Paris
Country [20] 0 0
Germany
State/province [20] 0 0
Cologne
Country [21] 0 0
Germany
State/province [21] 0 0
Magdeburg
Country [22] 0 0
Italy
State/province [22] 0 0
Como
Country [23] 0 0
Italy
State/province [23] 0 0
Genoa
Country [24] 0 0
Italy
State/province [24] 0 0
Genova
Country [25] 0 0
Italy
State/province [25] 0 0
Milan
Country [26] 0 0
Italy
State/province [26] 0 0
Rome
Country [27] 0 0
Japan
State/province [27] 0 0
Osaka
Country [28] 0 0
Japan
State/province [28] 0 0
Okayama
Country [29] 0 0
Portugal
State/province [29] 0 0
Coimbra
Country [30] 0 0
Spain
State/province [30] 0 0
Barcelona
Country [31] 0 0
Spain
State/province [31] 0 0
Málaga
Country [32] 0 0
Switzerland
State/province [32] 0 0
Lausanne
Country [33] 0 0
United Kingdom
State/province [33] 0 0
Bristol
Country [34] 0 0
United Kingdom
State/province [34] 0 0
London
Country [35] 0 0
United Kingdom
State/province [35] 0 0
Newcastle upon Tyne
Country [36] 0 0
United Kingdom
State/province [36] 0 0
Newcastle
Country [37] 0 0
United Kingdom
State/province [37] 0 0
Sheffield

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

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Pfizer 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
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT03794609