Brief Summary

This is a long-term, multi-center, longitudinal, observational study in children with achondroplasia (ACH). The aim is to study height velocity and comorbidities in children with ACH. This is a natural history study and no study medication will be administered.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

260

participants targeted

Target at P75+ for all trials

Timeline

Completed

Started Jun 2019

Longer than P75 for all trials

Geographic Reach

14 countries

34 active sites

Status

completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

4.6 years study duration

First Submitted

Initial submission to the registry

March 7, 2019

Completed

7 days until next milestone

First Posted

Study publicly available on registry

March 14, 2019

Completed

3 months until next milestone

Study Start

First participant enrolled

June 19, 2019

Completed

4.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 12, 2024

Completed

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 12, 2024

Completed

Last Updated

March 19, 2024

Status Verified

March 1, 2024

Enrollment Period

4.6 years

First QC Date

March 7, 2019

Last Update Submit

March 18, 2024

Conditions

Achondroplasia

Keywords

AchondroplasiaDwarfism

Outcome Measures

Primary Outcomes (1)

Annualized height velocity (centimeters/year) in children with achondroplasia
Subjects will undergo a series of height measurements (in centimeters) on Day 1 and then every 6 months over the study period with height velocity reported in cm/year
Up to 5 years

Secondary Outcomes (1)

Collection of natural history of achondroplasia symptoms in children with achondroplasia
Up to 5 years

Eligibility Criteria

Age0 Years - 8 Years

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17)

Sampling MethodProbability Sample

Study Population

Infants and children with achondroplasia from birth to 8 years of age.

You may qualify if:

Legally authorized representative is willing and able to provide written, signed informed consent (with a written assent from the child when appropriate per local requirements)
Willing and able to comply with study protocol per investigator judgement
Clinical diagnosis of achondroplasia (confirmed by the investigator)
Age between 0 to 8 years old at enrollment
Able to stand without assistance (if the child is 24 months or older)

You may not qualify if:

Have received chronic treatment (\> 3 months) of human growth hormone (hGH) or other medicinal products intended to affect stature or body proportionality at any time
Have received any dose of medicinal products intended to affect stature or body proportionality within the previous 6 months of screening
Have received any investigational medicinal product or device intended to affect stature or body proportionality at any time
History or presence of injury or disease of the growth plate(s), other than ACH, that affects growth potential of long bones
History of any bone-related surgery that affects growth potential of long bones, such as orthopedic reconstructive surgery and osteotomy (foramen magnum decompression, and laminectomy with full recovery are allowed with minimum of 6 months of bone healing. Limb-lengthening with full recovery is allowed with a minimum of 12 months of bone healing.)
Have forms of skeletal dysplasias other than achondroplasia or medical conditions that result in short stature or abnormal bone growth \[such as severe achondroplasia with developmental delay and acanthosis nigricans (SADDAN), hypochondroplasia, growth hormone deficiency, Turner syndrome, pseudoachondroplasia. uncontrolled hypothyroidism, uncontrolled diabetes mellitus, autoimmune disease requiring corticosteroid therapy, inflammatory bowel disease, and chronic renal insufficiency\]
History or presence of malignant disease, other than basal cell epithelioma/carcinoma or completely resected squamous skin cancer with no recurrence for 12 months per medical records

Contact the study team to confirm eligibility.