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

• ClinicalTrials.gov Identifier: NCT03794609
• Recruitment Status: Active, not recruiting
• First Posted: January 7, 2019
• Last Update Posted: November 18, 2022
• Sponsor: Pfizer
• 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

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

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

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