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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03875534
Recruitment Status : Recruiting
First Posted : March 14, 2019
Last Update Posted : November 29, 2022
Sponsor:
Information provided by (Responsible Party):
Ascendis Pharma A/S

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.

Condition or disease
Achondroplasia

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Study Type : Observational
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

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Ages Eligible for Study:   0 Years 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03875534


Contacts
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Contact: Bent Winding, MD +45 51233590 bwi@ascendispharma.com

Locations
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United States, Arkansas
Ascendis Pharma Investigational Site Recruiting
Little Rock, Arkansas, United States, 72211
United States, California
Ascendis Pharma Investigational Site Recruiting
Oakland, California, United States, 94609
United States, Colorado
Ascendis Pharma Investigational Site Recruiting
Aurora, Colorado, United States, 80045
United States, Delaware
Ascendis Pharma Investigational Site 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, New York
Ascendis Pharma Investigational Site Recruiting
Buffalo, New York, United States, 14203
United States, Texas
Ascendis Pharma Investigational Site 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 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, Alberta
Ascendis Pharma Investigational Site Recruiting
Edmonton, Alberta, Canada, T6G-1C9
Canada, Ontario
Ascendis Pharma Investigational Site Recruiting
Ottawa, Ontario, Canada, 51H 8L1
Canada, Quebec
Ascendis Pharma Investigational Site Recruiting
Montréal, Quebec, Canada, H3T 1C5
China
Ascendis Pharma Investigational Site Recruiting
Beijing, China, 100045
Ascendis Pharma Investigational Site Recruiting
Guangzhou, China, 510080
Contact: Ascendis Pharma I Site         
Ascendis Pharma Investigational Site Recruiting
Hangzhou, China, 310053
Ascendis Pharma Investigational Site Recruiting
Shanghai, China, 20082
Ascendis Pharma Investigational Site Recruiting
Shenzhen, China, 518053
Contact: Ascendis Pharma I Site         
Ascendis Pharma Investigational Site Recruiting
Wuhan, China, 430030
Denmark
Ascendis Pharma Investigational Site Recruiting
Copenhagen, Hovedstaden, Denmark, 2100
France
Ascendis Pharma Investigational Site Recruiting
Paris, France, 75743
Germany
Ascendis Pharma Investigational Site Recruiting
Berlin, Germany, 13353
Ireland
Ascendis Pharma Investigational Site Recruiting
Dublin, Ireland, 1
Italy
Ascendis Pharma Investigational Site Recruiting
Milano, Italy, 20132
Ascendis Pharma Investigational Site Not yet recruiting
Rome, Italy, 00165
New Zealand
Ascendis Pharma Investigational Site Recruiting
Auckland, New Zealand, 1142
Portugal
Ascendis Pharma Investigational Site Recruiting
Coimbra, Portugal, 3000
Spain
Ascendis Pharma Investigational Site Not yet recruiting
Barcelona, Spain, 08035
Ascendis Pharma Investigational Site Recruiting
Esplugues De Llobregat, Spain, 8950
Ascendis Pharma Investigational Site Active, not recruiting
Madrid, Spain, 28046
Ascendis Pharma Investigational Site Recruiting
Vitoria-Gasteiz, Spain, 1008
Switzerland
Ascendis Pharma Investigational Site Recruiting
Lausanne, Switzerland, 1005
United Kingdom
Ascendis Pharma Investigational Site Recruiting
Glasgow, United Kingdom, G51 4TF
Ascendis Pharma Investigational Site Withdrawn
Liverpool, United Kingdom, LA12 2AP
Ascendis Pharma Investigational Site Recruiting
London, United Kingdom, SE1 7EU
Sponsors and Collaborators
Ascendis Pharma A/S
Investigators
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Study Director: Adebola Giwa, MD Ascendis Pharma
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Responsible Party: Ascendis Pharma A/S
ClinicalTrials.gov Identifier: NCT03875534    
Other Study ID Numbers: TCC-NHS-01
First Posted: March 14, 2019    Key Record Dates
Last Update Posted: November 29, 2022
Last Verified: November 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Ascendis Pharma A/S:
Achondroplasia
Dwarfism
Additional relevant MeSH terms:
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Achondroplasia
Dwarfism
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Osteochondrodysplasias
Genetic Diseases, Inborn