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

NCT03794609

Last updated date
Study Location
Los Angeles Biomedical Research Institute At Harbour-UCLA Medical Centre
Los Angeles, California, 90502, United States
Contact
1-800-718-1021

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Eligibility Criteria
condition
The disease, disorder, syndrome, illness, or injury that is being studied.
Achondroplasia
Sex
Females and Males
Age
Pediatric Trials: 0-17 Years
Adult Trials: 18+ Years
0-10
Inclusion Criteria
The factors, or reasons, that allow a person to participate in a clinical study.
Show details

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
The factors, or reasons, that prevent a person from participating in a clinical study.
Show details


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

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AchondroplasiaObservational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia. NCT03794609
  1. Los Angeles, California
  2. Columbia, Missouri
  3. Cincinnati, Ohio
  4. Houston, Texas
  5. Seattle, Washington
  6. Melbourne,
  7. Barcelona,
  8. Lausanne,
  9. Wilmington, Delaware
  10. Baltimore, Maryland
  11. Boston, Massachusetts
  12. Salt Lake City, Utah
  13. Antwerp,
  14. Toronto,
  15. Copenhagen,
  16. Marseille,
  17. Paris,
  18. Cologne,
  19. Magdeburg,
  20. Como,
  21. Genova,
  22. Milan,
  23. Rome,
  24. Coimbra,
  25. Málaga,
  26. Bristol,
  27. London,
  28. Newcastle,
  29. Sheffield,
ALL GENDERS
0+
years
MULTIPLE SITES
Advanced Information
Descriptive Information
Brief Title Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia.
Official Title A Multi-center, Prospective, Longitudinal, Observational Study to Investigate the Clinical and Anthropometric Characteristics of Children With the Diagnosis Achondroplasia.
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.

Detailed Description Not Provided
Study Type Observational
Study Design Observational Model: Case-Only
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population

It is assumed that each of the study sites will enroll approximately 10-15 children of both genders and of various ages (0-10 years old).

The total number of children planned to be enrolled across all sites is approximately 200.

Condition Achondroplasia
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: January 3, 2019)
200
Original Estimated Enrollment Same as current
Estimated Study Completion Date June 15, 2023
Estimated Primary Completion Date June 15, 2023   (Final data collection date for primary outcome measure)
Eligibility Criteria

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
Sex/Gender
Sexes Eligible for Study:All
Ages up to 10 Years   (Child)
Accepts Healthy Volunteers No
Contacts
Contact: Pfizer CT.gov Call Center1-800-718-1021[email protected]
Listed Location Countries Australia,   Belgium,   Canada,   Denmark,   France,   Germany,   Italy,   Portugal,   Spain,   Switzerland,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT03794609
Other Study ID Numbers TA46-002
C4181001 ( Other Identifier: Pfizer )
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD:No
Plan Description: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/d….
Responsible Party Pfizer
Study Sponsor Pfizer
Collaborators Not Provided
Investigators
Study Director:Pfizer CT.gov Call CenterPfizer
PRS Account Pfizer
Verification Date April 2020