We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03794609
Recruitment Status : Active, not recruiting
First Posted : January 7, 2019
Last Update Posted : November 18, 2022
Sponsor:
Information provided by (Responsible Party):
Pfizer

Tracking Information
First Submitted Date November 19, 2018
First Posted Date January 7, 2019
Last Update Posted Date November 18, 2022
Actual Study Start Date June 15, 2018
Estimated Primary Completion Date September 29, 2027   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: January 3, 2019)
  • Collection of Natural History of Achondroplasia Characteristics in a cohort of Children aged 0-10 years old diagnosed with Achondroplasia [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: Up to 5 Years ]
    To quantify the number & type of Achondroplasia related Tests & Treatments by review of medical records
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures
 (submitted: January 3, 2019)
Measurement of biomarkers for bone growth [ Time Frame: Baseline, Month 12, Month 24, Month 36, Month 48, Month 60 ]
changes from Baseline in blood samples of collagen fragments
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia.
Official Title An International, Prospective Registry Investigating the Natural History of Participants With 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 Active, not recruiting
Actual Enrollment
 (submitted: November 16, 2022)
315
Original Estimated Enrollment
 (submitted: January 3, 2019)
200
Estimated Study Completion Date September 29, 2027
Estimated Primary Completion Date September 29, 2027   (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 0 Years to 15 Years   (Child)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries Australia,   Belgium,   Canada,   China,   Denmark,   France,   Germany,   Italy,   Japan,   Portugal,   Spain,   Switzerland,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT03794609
Other Study ID Numbers TA46-002
C4181001 ( Other Identifier: Alias Study Number )
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/data_requests.
Current Responsible Party Pfizer
Original Responsible Party Therachon SAS
Current Study Sponsor Pfizer
Original Study Sponsor Therachon SAS
Collaborators Not Provided
Investigators
Study Director: Pfizer Pfizer CT.gov Call Center Pfizer
PRS Account Pfizer
Verification Date November 2022