Clinical trial NCT03875534: A Multi-center, Longitudinal, Observational Study of Children With Achondroplasia
Official title: ACHieve: A Multi-center, Longitudinal, Observational Study of Children With Achondroplasia
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Achondroplasia
Keywords: Achondroplasia
Dwarfism
Lead sponsor: Ascendis Pharma A/S
Sponsor class: INDUSTRY
First posted: 2019-03-14
Enrollment: 260
Sex: ALL
Minimum age (years): 0
Maximum age (years): 8
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Infants and children with achondroplasia from birth to 8 years of age.

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.


Eligibility criteria: Inclusion Criteria:

1. 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)
2. Willing and able to comply with study protocol per investigator judgement
3. Clinical diagnosis of achondroplasia (confirmed by the investigator)
4. Age between 0 to 8 years old at enrollment
5. Able to stand without assistance (if the child is 24 months or older)

Exclusion Criteria:

1. 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
2. Have received any dose of medicinal products intended to affect stature or body proportionality within the previous 6 months of screening
3. Have received any investigational medicinal product or device intended to affect stature or body proportionality at any time
4. History or presence of injury or disease of the growth plate(s), other than ACH, that affects growth potential of long bones
5. 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.)
6. Have forms of skeletal dysplasias other than achondroplasia or medical conditions that result in short stature or abnormal bone growth \[such as severe achondroplasia with developmental delay and acanthosis nigricans (SADDAN), hypochondroplasia, growth hormone deficiency, Turner syndrome, pseudoachondroplasia. uncontrolled hypothyroidism, uncontrolled diabetes mellitus, autoimmune disease requiring corticosteroid therapy, inflammatory bowel disease, and chronic renal insufficiency\]
7. 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

Primary outcomes:
measure: Annualized height velocity (centimeters/year) in children with achondroplasia
timeFrame: Up to 5 years
Secondary outcomes:
measure: Collection of natural history of achondroplasia symptoms in children with achondroplasia
timeFrame: Up to 5 years
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: Ascendis Pharma Investigational Site
state: Arkansas
city: Oakland
country: United States
facility: Ascendis Pharma Investigational Site
state: California
city: Aurora
country: United States
facility: Ascendis Pharma Investigational Site
state: Colorado
city: Wilmington
country: United States
facility: Ascendis Pharma Investigational Site
state: Delaware
city: Saint Paul
country: United States
facility: Ascendis Pharma Investigational Site
state: Minnesota
city: Columbia
country: United States
facility: Ascendis Pharma Investigational Site
state: Missouri
city: Buffalo
country: United States
facility: Ascendis Pharma Investigational Site
state: New York
city: Houston
country: United States
facility: Ascendis Pharma Investigational Site
state: Texas
city: Seattle
country: United States
facility: Ascendis Pharma Investigational Site
state: Washington
city: Madison
country: United States
facility: Ascendis Pharma Investigational Site
state: Wisconsin
city: Parkville
country: Australia
facility: Ascendis Pharma Investigational Site
state: Victoria
city: Linz
country: Austria
facility: Ascendis Pharma Investigational Site
city: Edmonton
country: Canada
facility: Ascendis Pharma Investigational Site
state: Alberta
city: Ottawa
country: Canada
facility: Ascendis Pharma Investigational Site
state: Ontario
city: Montréal
country: Canada
facility: Ascendis Pharma Investigational Site
state: Quebec
city: Beijing
country: China
facility: Ascendis Pharma Investigational Site
city: Guangzhou
country: China
facility: Ascendis Pharma Investigational Site
city: Hangzhou
country: China
facility: Ascendis Pharma Investigational Site
city: Shanghai
country: China
facility: Ascendis Pharma Investigational Site
city: Shenzhen
country: China
facility: Ascendis Pharma Investigational Site
city: Wuhan
country: China
facility: Ascendis Pharma Investigational Site
city: Copenhagen
country: Denmark
facility: Ascendis Pharma Investigational Site
state: Hovedstaden
city: Paris
country: France
facility: Ascendis Pharma Investigational Site
city: Berlin
country: Germany
facility: Ascendis Pharma Investigational Site
city: Dublin
country: Ireland
facility: Ascendis Pharma Investigational Site
city: Milano
country: Italy
facility: Ascendis Pharma Investigational Site
city: Auckland
country: New Zealand
facility: Ascendis Pharma Investigational Site
city: Coimbra
country: Portugal
facility: Ascendis Pharma Investigational Site
city: Barcelona
country: Spain
facility: Ascendis Pharma Investigational Site
city: Esplugues De Llobregat
country: Spain
facility: Ascendis Pharma Investigational Site
city: Madrid
country: Spain
facility: Ascendis Pharma Investigational Site
city: Vitoria-Gasteiz
country: Spain
facility: Ascendis Pharma Investigational Site
city: Lausanne
country: Switzerland
facility: Ascendis Pharma Investigational Site
city: Glasgow
country: United Kingdom
facility: Ascendis Pharma Investigational Site
city: London
country: United Kingdom
facility: Ascendis Pharma Investigational Site
Study officials:
References: