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

 
Update - 21th Oct 2019

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.
All content in ClinicalTrials.gov identifier (NCT number): NCT03875534

 

Study Type  : Observational (no drug is administered)
Estimated Enrollment  : 200 participants
Observational Model: Case-Only
Time Perspective: Prospective
Official Title: ACHieve: A Multi-center, Longitudinal, Observational Study of Children With Achondroplasia
Actual Study Start Date  : June 19, 2019
Estimated Primary Completion Date  : June 2024
Estimated Study Completion Date  : June 2024

 

 

Primary Outcome Measures  :
  1. Annualized height velocity (centimeters/year) in children with achondroplasia [ Time Frame: Up to 5 years ]
    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

Secondary Outcome Measures  :
  1. Collection of natural history of achondroplasia symptoms in children with achondroplasia [ Time Frame: Up to 5 years ]
    To characterize achondroplasia symptoms in children with achondroplasia

 

Information from the National Library of Medicine

 

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   up to 8 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
Infants and children with achondroplasia from birth to 8 years of age.
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

Contacts

Study Director: Will Charlton, MD Ascendis Pharma A/S  


Locations

United States, Arkansas
Ascendis Pharma Investigational Site Recruiting
Little Rock, Arkansas, United States, 72211
United States, California
Ascendis Pharma Investigational Site Not yet recruiting
Oakland, California, United States, 94609
United States, Delaware
Ascendis Pharma Investigational Site Not yet recruiting
Wilmington, Delaware, United States, 19803
United States, Massachusetts
Ascendis Pharma Investigational Site Not yet recruiting
Boston, Massachusetts, United States, 02115
United States, Minnesota
Ascendis Pharma Investigational Site Recruiting
Saint Paul, Minnesota, United States, 55102
United States, Missouri
Ascendis Pharma Investigational Site Recruiting
Columbia, Missouri, United States, 65212
United States, Texas
Ascendis Pharma Investigational Site Not yet recruiting
Houston, Texas, United States, 77030
United States, Washington
Ascendis Pharma Investigational Site Recruiting
Seattle, Washington, United States, 98105
United States, Wisconsin
Ascendis Pharma Investigational Site Not yet recruiting
Madison, Wisconsin, United States, 53705
Australia, Victoria
Ascendis Pharma Investigational Site Recruiting
Parkville, Victoria, Australia, 3052
Austria
Ascendis Pharma Investigational Site Recruiting
Linz, Austria, 4020
Canada
Ascendis Pharma Investigational Site Not yet recruiting
Montréal, Canada, H3T 1C5
Germany
Ascendis Pharma Investigational Site Not yet recruiting
Berlin, Germany, 13353
Ireland
Ascendis Pharma Investigational Site Not yet recruiting
Dublin, Ireland, 1
Italy
Ascendis Pharma Investigational Site Not yet recruiting
Rome, Italy, 00165
Portugal
Ascendis Pharma Investigational Site Not yet recruiting
Coimbra, Portugal, 3000
Spain
Ascendis Pharma Investigational Site Not yet recruiting
Barcelona, Spain, 08035
Ascendis Pharma Investigational Site Not yet recruiting
Madrid, Spain, 28046
Switzerland
Ascendis Pharma Investigational Site Not yet recruiting
Lausanne, Switzerland, 1005
United Kingdom
Ascendis Pharma Investigational Site Not yet recruiting
Glasgow, United Kingdom, G51 4TF
Ascendis Pharma Investigational Site Not yet recruiting
Liverpool, United Kingdom, LA12 2AP
Ascendis Pharma Investigational Site Not yet recruiting
London, United Kingdom, SE1 7EU
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