A Multi-center, Longitudinal, Observational Study of Children With Achondroplasia
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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
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Condition or disease |
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Achondroplasia |
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 |

- 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
- 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

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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 |
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

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
Contact: Bent Winding, MD | +45 51233590 | bwi@ascendispharma.com |
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 |
Study Director: | Adebola Giwa, MD | Ascendis Pharma |
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 |
Studies a U.S. FDA-regulated Drug Product: | No |
Studies a U.S. FDA-regulated Device Product: | No |
Achondroplasia Dwarfism |
Achondroplasia Dwarfism Bone Diseases, Developmental Bone Diseases |
Musculoskeletal Diseases Osteochondrodysplasias Genetic Diseases, Inborn |