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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03875534




Registration number
NCT03875534
Ethics application status
Date submitted
7/03/2019
Date registered
14/03/2019
Date last updated
27/06/2019

Titles & IDs
Public title
A Multi-center, Longitudinal, Observational Study of Children With Achondroplasia
Scientific title
ACHieve: A Multi-center, Longitudinal, Observational Study of Children With Achondroplasia
Secondary ID [1] 0 0
TCC-NHS-01
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Achondroplasia 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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
Timepoint [1] 0 0
Up to 5 years
Secondary outcome [1] 0 0
Collection of natural history of achondroplasia symptoms in children with achondroplasia - To characterize achondroplasia symptoms in children with achondroplasia
Timepoint [1] 0 0
Up to 5 years

Eligibility
Key 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)
Minimum age
No limit
Maximum age
8 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Ascendis Pharma Investigational Site - Parkville
Recruitment postcode(s) [1] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arkansas
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Delaware
Country [4] 0 0
United States of America
State/province [4] 0 0
Georgia
Country [5] 0 0
United States of America
State/province [5] 0 0
Massachusetts
Country [6] 0 0
United States of America
State/province [6] 0 0
Minnesota
Country [7] 0 0
United States of America
State/province [7] 0 0
Missouri
Country [8] 0 0
United States of America
State/province [8] 0 0
Texas
Country [9] 0 0
United States of America
State/province [9] 0 0
Virginia
Country [10] 0 0
United States of America
State/province [10] 0 0
Washington
Country [11] 0 0
United States of America
State/province [11] 0 0
Wisconsin
Country [12] 0 0
Austria
State/province [12] 0 0
Linz
Country [13] 0 0
Canada
State/province [13] 0 0
Montréal
Country [14] 0 0
Germany
State/province [14] 0 0
Berlin
Country [15] 0 0
Ireland
State/province [15] 0 0
Dublin
Country [16] 0 0
Italy
State/province [16] 0 0
Rome
Country [17] 0 0
Portugal
State/province [17] 0 0
Coimbra
Country [18] 0 0
Spain
State/province [18] 0 0
Barcelona
Country [19] 0 0
Spain
State/province [19] 0 0
Madrid
Country [20] 0 0
Switzerland
State/province [20] 0 0
Lausanne
Country [21] 0 0
United Kingdom
State/province [21] 0 0
Glasgow
Country [22] 0 0
United Kingdom
State/province [22] 0 0
Liverpool
Country [23] 0 0
United Kingdom
State/province [23] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Ascendis Pharma A/S
Address
Country

Ethics approval
Ethics application status

Summary
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 website
https://clinicaltrials.gov/show/NCT03875534
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Will Charlton, MD
Address 0 0
Ascendis Pharma A/S
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Medical Affairs Director
Address 0 0
Country 0 0
Phone 0 0
+1 855-795-2467
Fax 0 0
Email 0 0
achieve-study@ascendispharma.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT03875534