Observational Study Investigating Clinical & Anthropometr... | NCT03794609

ID	Type	Description	Link
C4181001	Other Identifier	Alias Study Number

Status Module

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Change From Baseline in Standing Height	Standing height (for participants aged ≥2 years) is an assessment of maximum vertical size. Standing height was measured using a stadiometer with a fixed vertical backboard and an adjustable head piece. Height in supine position (or length for participants aged <2 years) was measured using measuring board. Three valid measurements were performed at each visit and the average of the 3 valid measurements was reported and used in descriptive summaries. Data is reported according to age group (<2 years and ≥2 years) and gender of the participants.	Baseline, 1 year, 2 years, 3 years, 4 years
Change From Baseline in Sitting Height	Sitting height (for participants aged ≥2 years) is a measure of the trunk of the body from the buttocks to the top of the head when the participant is sitting upright. Sitting height was measured using a stadiometer and sitting height table. Crown-rump length (for participants aged <2 years) was measured using measuring board. Three valid measurements were performed at each visit and the average of the 3 valid measurements was reported and used in descriptive summaries. Data is reported according to age group (<2 years and ≥2 years) and gender of the participants.	Baseline, 1 year, 2 years, 3 years, 4 years
Change From Baseline in Knee Height	Knee height means lower segment length (lower segment=standing height - sitting height). Knee height was measured using knee height caliper. Three valid measurements were performed at each visit and the average of the 3 valid measurements was reported and used in descriptive summaries. Data is reported according to age group (<2 years, 2-10 years, and ≥10 years) and gender of the participants.	Baseline, 1 year, 2 years, 3 years, 4 years
Change From Baseline in Head Circumference	Head circumference is a measurement of the circumference of the child's head at its largest area, above the eyebrows and ears and around the back of the head. Head circumfernce was measured using measuring tape. Three valid measurements were performed at each visit and the average of the 3 valid measurements was reported and used in descriptive summaries. Data is reported according to age group (<2 years, 2-10 years, and ≥10 years) and gender of the participants.

Secondary Outcomes

Measure	Description	Time Frame
Baseline and Post-baseline Measurements of Serum Collagen X Marker	Collagen X Marker (CXM), is a degradation by-product of endochondral ossification that is released into the circulation in proportion to overall growth plate activity. This marker corresponds to the rate of linear bone growth at time of measurement as recently demonstrated in children of normal average growth. Data is reported according to age group (<2 years, 2-10 years, and ≥10 years) and gender of the participants.

Eligibility Module

Eligibility Criteria

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
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

Name	Affiliation	Role
Pfizer Pfizer CT.gov Call Center	Pfizer	Study Director

Facility	Status	City	State	ZIP	Country	Contacts
Los Angeles Biomedical Research Institute At Harbour-UCLA Medical Centre		Los Angeles	California	90502	United States

Label	URL
To obtain contact information for a study center near you, click here.	View source

IPD Sharing Statement Module

Results Section

Participant Flow Module

Arm/Group Information

ID	Title	Description
FG000	Enrolled Participants	Participants with achondroplasia were followed over 4 years with respect to achondroplasia symptoms, tests and treatments, anthropometric parameters and biomarkers of bone growth. No study intervention was administered, and no other type of therapy was provided.

Baseline Characteristics Module

Outcome Measures Module

Adverse Events Module

More Info Module

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot	Yes	No	No	Study Protocol	Apr 20, 2020	Jan 10, 2024

Baseline Measures

Title

Description

Population Description

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Denominator Units Selected

Denominators

Classes

Age, Customized

Count of Participants

Participants

Title

Denominators

Categories

0-2 Years

Title	Measurements
	BG00082

2-10 Years

Title	Measurements
	BG000213

≥10 Years

Sex: Female, Male

Count of Participants

Participants

Title

Denominators

Categories

Title	Measurements
Female	BG000137
Male	BG000178

Ethnicity (NIH/OMB)

Count of Participants

Participants

Title

Denominators

Categories

Title	Measurements
Hispanic or Latino	BG00022
Not Hispanic or Latino	BG000288

Race/Ethnicity, Customized

Count of Participants

Participants

Title

Denominators

Categories

Title	Measurements
Asian	BG00052
Black or African American	BG0002

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

Primary

Change From Baseline in Standing Height

Standing height (for participants aged ≥2 years) is an assessment of maximum vertical size. Standing height was measured using a stadiometer with a fixed vertical backboard and an adjustable head piece. Height in supine position (or length for participants aged <2 years) was measured using measuring board. Three valid measurements were performed at each visit and the average of the 3 valid measurements was reported and used in descriptive summaries. Data is reported according to age group (<2 years and ≥2 years) and gender of the participants.

Analysis population included all enrolled participants, among whom, 2 participants have not been included due to missing age. The "Overall Number of Participants Analyzed" was based on age at baseline. The "Number Analyzed" in a row was based on actually analyzed participants at each visit.

Posted

Mean

Standard Deviation

centimeter (cm)

Baseline, 1 year, 2 years, 3 years, 4 years

ID	Title	Description
OG000	Male (Aged <2 Years)	Participants with achondroplasia were followed over a five-year period with respect to achondroplasia symptoms, tests and treatments, anthropometric parameters and biomarkers of bone growth. No study intervention was administered, and no other type of therapy was provided.
OG001	Female (Aged <2 Years)	Participants with achondroplasia were followed over a five-year period with respect to achondroplasia symptoms, tests and treatments, anthropometric parameters and biomarkers of bone growth. No study intervention was administered, and no other type of therapy was provided.
OG002	Male (Aged ≥2 Years)	Participants with achondroplasia were followed over a five-year period with respect to achondroplasia symptoms, tests and treatments, anthropometric parameters and biomarkers of bone growth. No study intervention was administered, and no other type of therapy was provided.
OG003	Female (Aged ≥2 Years)	Participants with achondroplasia were followed over a five-year period with respect to achondroplasia symptoms, tests and treatments, anthropometric parameters and biomarkers of bone growth. No study intervention was administered, and no other type of therapy was provided.

Units	Counts
Participants	OG00045 OG00137 OG002131 OG003

Title	Denominators	Categories
Baseline	ParticipantsOG00045 ParticipantsOG00135 ParticipantsOG002125 ParticipantsOG003

Primary

Change From Baseline in Sitting Height

Sitting height (for participants aged ≥2 years) is a measure of the trunk of the body from the buttocks to the top of the head when the participant is sitting upright. Sitting height was measured using a stadiometer and sitting height table. Crown-rump length (for participants aged <2 years) was measured using measuring board. Three valid measurements were performed at each visit and the average of the 3 valid measurements was reported and used in descriptive summaries. Data is reported according to age group (<2 years and ≥2 years) and gender of the participants.

Posted

Mean

Standard Deviation

Baseline, 1 year, 2 years, 3 years, 4 years

ID	Title	Description
OG000	Male (Aged <2 Years)	Participants with achondroplasia were followed over a five-year period with respect to achondroplasia symptoms, tests and treatments, anthropometric parameters and biomarkers of bone growth. No study intervention was administered, and no other type of therapy was provided.
OG001	Female (Aged <2 Years)	Participants with achondroplasia were followed over a five-year period with respect to achondroplasia symptoms, tests and treatments, anthropometric parameters and biomarkers of bone growth. No study intervention was administered, and no other type of therapy was provided.

Primary

Change From Baseline in Knee Height

Knee height means lower segment length (lower segment=standing height - sitting height). Knee height was measured using knee height caliper. Three valid measurements were performed at each visit and the average of the 3 valid measurements was reported and used in descriptive summaries. Data is reported according to age group (<2 years, 2-10 years, and ≥10 years) and gender of the participants.

Posted

Mean

Standard Deviation

Baseline, 1 year, 2 years, 3 years, 4 years

ID	Title	Description
OG000	Male (Aged <2 Years)	Participants with achondroplasia were followed over a five-year period with respect to achondroplasia symptoms, tests and treatments, anthropometric parameters and biomarkers of bone growth. No study intervention was administered, and no other type of therapy was provided.
OG001	Female (Aged <2 Years)	Participants with achondroplasia were followed over a five-year period with respect to achondroplasia symptoms, tests and treatments, anthropometric parameters and biomarkers of bone growth. No study intervention was administered, and no other type of therapy was provided.

Primary

Change From Baseline in Head Circumference

Head circumference is a measurement of the circumference of the child's head at its largest area, above the eyebrows and ears and around the back of the head. Head circumfernce was measured using measuring tape. Three valid measurements were performed at each visit and the average of the 3 valid measurements was reported and used in descriptive summaries. Data is reported according to age group (<2 years, 2-10 years, and ≥10 years) and gender of the participants.

Posted

Mean

Standard Deviation

Baseline, 1 year, 2 years, 3 years, 4 years

ID	Title	Description
OG000	Male (Aged <2 Years)	Participants with achondroplasia were followed over a five-year period with respect to achondroplasia symptoms, tests and treatments, anthropometric parameters and biomarkers of bone growth. No study intervention was administered, and no other type of therapy was provided.
OG001	Female (Aged <2 Years)	Participants with achondroplasia were followed over a five-year period with respect to achondroplasia symptoms, tests and treatments, anthropometric parameters and biomarkers of bone growth. No study intervention was administered, and no other type of therapy was provided.

Primary

Change From Baseline in Arm Span

Arm span measurement is the distance between fingertips when the arms are outstretched. All participants should be measured supine lying on the measurement grid. Three valid measurements were performed at each visit and the average of the 3 valid measurements was reported and used in descriptive summaries. Data is reported according to age group (<2 years, 2-10 years, and ≥10 years) and gender of the participants.

Posted

Mean

Standard Deviation

Baseline, 1 year, 2 years, 3 years, 4 years

ID	Title	Description
OG000	Male (Aged <2 Years)	Participants with achondroplasia were followed over a five-year period with respect to achondroplasia symptoms, tests and treatments, anthropometric parameters and biomarkers of bone growth. No study intervention was administered, and no other type of therapy was provided.
OG001	Female (Aged <2 Years)	Participants with achondroplasia were followed over a five-year period with respect to achondroplasia symptoms, tests and treatments, anthropometric parameters and biomarkers of bone growth. No study intervention was administered, and no other type of therapy was provided.

Primary

Number of Participants With Achondroplasia-Related Treatments

Achondroplasia-related treatments were collected in a prospective and standardized fashion to allow characterization of the natural history of the disease course in the youngest participants in relation to the level of actual disease burden over time due to treatments, as well as data to support identification of possible risk factors. Data is reported according to the gender of the participants.

Analysis population included all enrolled participants.

Posted

Count of Participants

Participants

Baseline to end of study visit (up to 55 months)

ID	Title	Description
OG000	Male (All Ages)	Participants with achondroplasia were followed over a five-year period with respect to achondroplasia symptoms, tests and treatments, anthropometric parameters and biomarkers of bone growth. No study intervention was administered, and no other type of therapy was provided.
OG001	Female (All Ages)	Participants with achondroplasia were followed over a five-year period with respect to achondroplasia symptoms, tests and treatments, anthropometric parameters and biomarkers of bone growth. No study intervention was administered, and no other type of therapy was provided.

Secondary

Baseline and Post-baseline Measurements of Serum Collagen X Marker

Collagen X Marker (CXM), is a degradation by-product of endochondral ossification that is released into the circulation in proportion to overall growth plate activity. This marker corresponds to the rate of linear bone growth at time of measurement as recently demonstrated in children of normal average growth. Data is reported according to age group (<2 years, 2-10 years, and ≥10 years) and gender of the participants.

Posted

Mean

Standard Deviation

nanogram per liter (ng/L)

Baseline, 1 year, 2 years, 3 years

ID	Title	Description
OG000	Male (Aged <2 Years)	Participants with achondroplasia were followed over a five-year period with respect to achondroplasia symptoms, tests and treatments, anthropometric parameters and biomarkers of bone growth. No study intervention was administered, and no other type of therapy was provided.
OG001	Female (Aged <2 Years)	Participants with achondroplasia were followed over a five-year period with respect to achondroplasia symptoms, tests and treatments, anthropometric parameters and biomarkers of bone growth. No study intervention was administered, and no other type of therapy was provided.

ID	Term
D004392	Dwarfism
D001848	Bone Diseases, Developmental
D001847	Bone Diseases
D009140	Musculoskeletal Diseases
D010009	Osteochondrodysplasias
D030342	Genetic Diseases, Inborn
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities