- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03875534
A Multi-center, Longitudinal, Observational Study of Children With Achondroplasia
March 18, 2024 updated by: Ascendis Pharma A/S
ACHieve: A Multi-center, Longitudinal, Observational Study of Children With Achondroplasia
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.
Study Overview
Status
Completed
Conditions
Study Type
Observational
Enrollment (Actual)
260
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Bent Winding, MD
- Phone Number: +45 51233590
- Email: bwi@ascendispharma.com
Study Locations
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Victoria
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Parkville, Victoria, Australia, 3052
- Ascendis Pharma Investigational Site
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Linz, Austria, 4020
- Ascendis Pharma Investigational Site
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Alberta
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Edmonton, Alberta, Canada, T6G-1C9
- Ascendis Pharma Investigational Site
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Ontario
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Ottawa, Ontario, Canada, 51H 8L1
- Ascendis Pharma Investigational Site
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Quebec
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Montréal, Quebec, Canada, H3T 1C5
- Ascendis Pharma Investigational Site
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Beijing, China, 100045
- Ascendis Pharma Investigational Site
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Guangzhou, China, 510080
- Ascendis Pharma Investigational Site
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Hangzhou, China, 310053
- Ascendis Pharma Investigational Site
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Shanghai, China, 20082
- Ascendis Pharma Investigational Site
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Shenzhen, China, 518053
- Ascendis Pharma Investigational Site
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Wuhan, China, 430030
- Ascendis Pharma Investigational Site
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Hovedstaden
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Copenhagen, Hovedstaden, Denmark, 2100
- Ascendis Pharma Investigational Site
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Paris, France, 75743
- Ascendis Pharma Investigational Site
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Berlin, Germany, 13353
- Ascendis Pharma Investigational Site
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Dublin, Ireland, 1
- Ascendis Pharma Investigational Site
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Milano, Italy, 20132
- Ascendis Pharma Investigational Site
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Auckland, New Zealand, 1142
- Ascendis Pharma Investigational Site
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Coimbra, Portugal, 3000
- Ascendis Pharma Investigational Site
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Barcelona, Spain, 08035
- Ascendis Pharma Investigational Site
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Esplugues De Llobregat, Spain, 8950
- Ascendis Pharma Investigational Site
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Madrid, Spain, 28046
- Ascendis Pharma Investigational Site
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Vitoria-Gasteiz, Spain, 1008
- Ascendis Pharma Investigational Site
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Lausanne, Switzerland, 1005
- Ascendis Pharma Investigational Site
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Glasgow, United Kingdom, G51 4TF
- Ascendis Pharma Investigational Site
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London, United Kingdom, SE1 7EU
- Ascendis Pharma Investigational Site
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Arkansas
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Little Rock, Arkansas, United States, 72211
- Ascendis Pharma Investigational Site
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California
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Oakland, California, United States, 94609
- Ascendis Pharma Investigational Site
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Colorado
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Aurora, Colorado, United States, 80045
- Ascendis Pharma Investigational Site
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Delaware
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Wilmington, Delaware, United States, 19803
- Ascendis Pharma Investigational Site
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Minnesota
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Saint Paul, Minnesota, United States, 55102
- Ascendis Pharma Investigational Site
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Missouri
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Columbia, Missouri, United States, 65212
- Ascendis Pharma Investigational Site
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New York
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Buffalo, New York, United States, 14203
- Ascendis Pharma Investigational Site
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Texas
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Houston, Texas, United States, 77030
- Ascendis Pharma Investigational Site
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Washington
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Seattle, Washington, United States, 98105
- Ascendis Pharma Investigational Site
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Wisconsin
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Madison, Wisconsin, United States, 53705
- Ascendis Pharma Investigational Site
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
1 year to 6 years (Child)
Accepts Healthy Volunteers
No
Sampling Method
Probability Sample
Study Population
Infants and children with achondroplasia from birth to 8 years of age.
Description
Inclusion Criteria:
- 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)
Exclusion Criteria:
- 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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Observational Models: Case-Only
- Time Perspectives: Prospective
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Annualized height velocity (centimeters/year) in children with achondroplasia
Time Frame: Up to 5 years
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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
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Up to 5 years
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Collection of natural history of achondroplasia symptoms in children with achondroplasia
Time Frame: Up to 5 years
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To characterize achondroplasia symptoms in children with achondroplasia
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Up to 5 years
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Director: Laerke Clement Freiberg, MD, Ascendis Pharma
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
June 19, 2019
Primary Completion (Actual)
January 12, 2024
Study Completion (Actual)
January 12, 2024
Study Registration Dates
First Submitted
March 7, 2019
First Submitted That Met QC Criteria
March 13, 2019
First Posted (Actual)
March 14, 2019
Study Record Updates
Last Update Posted (Actual)
March 19, 2024
Last Update Submitted That Met QC Criteria
March 18, 2024
Last Verified
March 1, 2024
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- TCC-NHS-01
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
No
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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