A Multi-center, Longitudinal, Observational Study of Children With Achondroplasia

Achondroplasia Clinical Trial

Official title:

ACHieve: A Multi-center, Longitudinal, Observational Study of Children With Achondroplasia

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03875534
• Other study ID #: TCC-NHS-01
• Status: Completed
• Start date: June 19, 2019
• Est. completion date: January 12, 2024

Study information

• Verified date: March 2024
• Source: Ascendis Pharma A/S
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 260
• Est. completion date: January 12, 2024
• Est. primary completion date: January 12, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 0 Years to 8 Years

Eligibility

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

Related Conditions & MeSH terms

• Achondroplasia

Locations

Country	Name	City	State
Australia	Ascendis Pharma Investigational Site	Parkville	Victoria
Austria	Ascendis Pharma Investigational Site	Linz
Canada	Ascendis Pharma Investigational Site	Edmonton	Alberta
Canada	Ascendis Pharma Investigational Site	Montréal	Quebec
Canada	Ascendis Pharma Investigational Site	Ottawa	Ontario
China	Ascendis Pharma Investigational Site	Beijing
China	Ascendis Pharma Investigational Site	Guangzhou
China	Ascendis Pharma Investigational Site	Hangzhou
China	Ascendis Pharma Investigational Site	Shanghai
China	Ascendis Pharma Investigational Site	Shenzhen
China	Ascendis Pharma Investigational Site	Wuhan
Denmark	Ascendis Pharma Investigational Site	Copenhagen	Hovedstaden
France	Ascendis Pharma Investigational Site	Paris
Germany	Ascendis Pharma Investigational Site	Berlin
Ireland	Ascendis Pharma Investigational Site	Dublin
Italy	Ascendis Pharma Investigational Site	Milano
New Zealand	Ascendis Pharma Investigational Site	Auckland
Portugal	Ascendis Pharma Investigational Site	Coimbra
Spain	Ascendis Pharma Investigational Site	Barcelona
Spain	Ascendis Pharma Investigational Site	Esplugues De Llobregat
Spain	Ascendis Pharma Investigational Site	Madrid
Spain	Ascendis Pharma Investigational Site	Vitoria-Gasteiz
Switzerland	Ascendis Pharma Investigational Site	Lausanne
United Kingdom	Ascendis Pharma Investigational Site	Glasgow
United Kingdom	Ascendis Pharma Investigational Site	London
United States	Ascendis Pharma Investigational Site	Aurora	Colorado
United States	Ascendis Pharma Investigational Site	Buffalo	New York
United States	Ascendis Pharma Investigational Site	Columbia	Missouri
United States	Ascendis Pharma Investigational Site	Houston	Texas
United States	Ascendis Pharma Investigational Site	Little Rock	Arkansas
United States	Ascendis Pharma Investigational Site	Madison	Wisconsin
United States	Ascendis Pharma Investigational Site	Oakland	California
United States	Ascendis Pharma Investigational Site	Saint Paul	Minnesota
United States	Ascendis Pharma Investigational Site	Seattle	Washington
United States	Ascendis Pharma Investigational Site	Wilmington	Delaware

Sponsors (1)

Lead Sponsor	Collaborator
Ascendis Pharma A/S

Countries where clinical trial is conducted

United States,  Australia,  Austria,  Canada,  China,  Denmark,  France,  Germany,  Ireland,  Italy,  New Zealand,  Portugal,  Spain,  Switzerland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Collection of natural history of achondroplasia symptoms in children with achondroplasia	To characterize achondroplasia symptoms in children with achondroplasia	Up to 5 years