Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Acho… (NCT03794609) | Clinical Trial Compass
TerminatedNot Applicable
Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia.
Stopped: The study was terminated on 18th November 2022. The decision to terminate the trial was not based on any safety and/or efficacy concerns.
United States315 participantsStarted 2018-06-15
Plain-language 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.
Who can participate
Age range0 Years – 15 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. Written informed consent is obtained from the children's parent(s) / legal guardian(s) before any study-related activity is carried out
✓. 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
✓. The child has been diagnosed as having achondroplasia documented by clinical diagnosis
✓. The child is between 0 years and 10 years of age, inclusive, on the date of consent / assent
✓. The investigator has considered the family and prospective participating child being able to comply with the study procedures
Exclusion criteria
✕. The child has a diagnosis of hypochondroplasia or any short stature condition other than achondroplasia (eg, spondyloepiphyseal dysplasia congenital \[SEDC\], pseudoachondroplasia, trisomy 21)
✕. 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
What they're measuring
1
Change From Baseline in Standing Height
Timeframe: Baseline, 1 year, 2 years, 3 years, 4 years
2
Change From Baseline in Sitting Height
Timeframe: Baseline, 1 year, 2 years, 3 years, 4 years
3
Change From Baseline in Knee Height
Timeframe: Baseline, 1 year, 2 years, 3 years, 4 years
4
Change From Baseline in Head Circumference
Timeframe: Baseline, 1 year, 2 years, 3 years, 4 years
5
Change From Baseline in Arm Span
Timeframe: Baseline, 1 year, 2 years, 3 years, 4 years
6
Number of Participants With Achondroplasia-Related Treatments
Timeframe: Baseline to end of study visit (up to 55 months)
✕. 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
✕. Any surgery that affects the growth plate of the long bones that is planned, or has occurred in the past 18 months
✕. Participation in any interventional study (investigational product or device) for treatment of achondroplasia or short stature
✕. 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
✕. 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.
✕. Any concurrent disease or condition that in the view of the investigator would interfere with study participation