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 Locations

  • Australia
    • Victoria
      • Parkville, Victoria, Australia, 3052
        • Ascendis Pharma Investigational Site
  • Austria
      • Linz, Austria, 4020
        • Ascendis Pharma Investigational Site
  • Canada
    • Alberta
      • Edmonton, Alberta, Canada, T6G-1C9
        • Ascendis Pharma Investigational Site
    • Ontario
      • Ottawa, Ontario, Canada, 51H 8L1
        • Ascendis Pharma Investigational Site
    • Quebec
      • Montréal, Quebec, Canada, H3T 1C5
        • Ascendis Pharma Investigational Site
  • China
      • Beijing, China, 100045
        • Ascendis Pharma Investigational Site
      • Guangzhou, China, 510080
        • Ascendis Pharma Investigational Site
      • Hangzhou, China, 310053
        • Ascendis Pharma Investigational Site
      • Shanghai, China, 20082
        • Ascendis Pharma Investigational Site
      • Shenzhen, China, 518053
        • Ascendis Pharma Investigational Site
      • Wuhan, China, 430030
        • Ascendis Pharma Investigational Site
  • Denmark
    • Hovedstaden
      • Copenhagen, Hovedstaden, Denmark, 2100
        • Ascendis Pharma Investigational Site
  • France
      • Paris, France, 75743
        • Ascendis Pharma Investigational Site
  • Germany
      • Berlin, Germany, 13353
        • Ascendis Pharma Investigational Site
  • Ireland
      • Dublin, Ireland, 1
        • Ascendis Pharma Investigational Site
  • Italy
      • Milano, Italy, 20132
        • Ascendis Pharma Investigational Site
  • New Zealand
      • Auckland, New Zealand, 1142
        • Ascendis Pharma Investigational Site
  • Portugal
      • Coimbra, Portugal, 3000
        • Ascendis Pharma Investigational Site
  • Spain
      • Barcelona, Spain, 08035
        • Ascendis Pharma Investigational Site
      • Esplugues De Llobregat, Spain, 8950
        • Ascendis Pharma Investigational Site
      • Madrid, Spain, 28046
        • Ascendis Pharma Investigational Site
      • Vitoria-Gasteiz, Spain, 1008
        • Ascendis Pharma Investigational Site
  • Switzerland
      • Lausanne, Switzerland, 1005
        • Ascendis Pharma Investigational Site
  • United Kingdom
      • Glasgow, United Kingdom, G51 4TF
        • Ascendis Pharma Investigational Site
      • London, United Kingdom, SE1 7EU
        • Ascendis Pharma Investigational Site
  • United States
    • Arkansas
      • Little Rock, Arkansas, United States, 72211
        • Ascendis Pharma Investigational Site
    • California
      • Oakland, California, United States, 94609
        • Ascendis Pharma Investigational Site
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Ascendis Pharma Investigational Site
    • Delaware
      • Wilmington, Delaware, United States, 19803
        • Ascendis Pharma Investigational Site
    • Minnesota
      • Saint Paul, Minnesota, United States, 55102
        • Ascendis Pharma Investigational Site
    • Missouri
      • Columbia, Missouri, United States, 65212
        • Ascendis Pharma Investigational Site
    • New York
      • Buffalo, New York, United States, 14203
        • Ascendis Pharma Investigational Site
    • Texas
      • Houston, Texas, United States, 77030
        • Ascendis Pharma Investigational Site
    • Washington
      • Seattle, Washington, United States, 98105
        • Ascendis Pharma Investigational Site
    • Wisconsin
      • Madison, Wisconsin, United States, 53705
        • Ascendis Pharma Investigational Site

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

3 years to 4 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:

  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

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
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
Up to 5 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
Up to 5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Ascendis Pharma A/S

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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