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

Last updated: March 18, 2024
Sponsor: Ascendis Pharma A/S
Overall Status: Completed

Phase

N/A

Condition

Severe Short Stature

Treatment

N/A

Clinical Study ID

NCT03875534
TCC-NHS-01
  • Ages < 8
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Legally authorized representative is willing and able to provide written, signedinformed consent (with a written assent from the child when appropriate per localrequirements)
  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

Exclusion Criteria:

  1. Have received chronic treatment (> 3 months) of human growth hormone (hGH) or othermedicinal products intended to affect stature or body proportionality at any time
  2. Have received any dose of medicinal products intended to affect stature or bodyproportionality within the previous 6 months of screening
  3. Have received any investigational medicinal product or device intended to affectstature or body proportionality at any time
  4. History or presence of injury or disease of the growth plate(s), other than ACH, thataffects growth potential of long bones
  5. History of any bone-related surgery that affects growth potential of long bones, suchas orthopedic reconstructive surgery and osteotomy (foramen magnum decompression, andlaminectomy 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 bonehealing.)
  6. Have forms of skeletal dysplasias other than achondroplasia or medical conditions thatresult in short stature or abnormal bone growth [such as severe achondroplasia withdevelopmental delay and acanthosis nigricans (SADDAN), hypochondroplasia, growthhormone deficiency, Turner syndrome, pseudoachondroplasia. uncontrolledhypothyroidism, uncontrolled diabetes mellitus, autoimmune disease requiringcorticosteroid therapy, inflammatory bowel disease, and chronic renal insufficiency]
  7. History or presence of malignant disease, other than basal cell epithelioma/carcinomaor completely resected squamous skin cancer with no recurrence for 12 months permedical records

Study Design

Total Participants: 260
Study Start date:
June 19, 2019
Estimated Completion Date:
January 12, 2024

Connect with a study center

  • Ascendis Pharma Investigational Site

    Parkville, Victoria 3052
    Australia

    Site Not Available

  • Ascendis Pharma Investigational Site

    Linz, 4020
    Austria

    Site Not Available

  • Ascendis Pharma Investigational Site

    Edmonton, Alberta T6G-1C9
    Canada

    Site Not Available

  • Ascendis Pharma Investigational Site

    Ottawa, Ontario 51H 8L1
    Canada

    Site Not Available

  • Ascendis Pharma Investigational Site

    Montréal, Quebec H3T 1C5
    Canada

    Site Not Available

  • Ascendis Pharma Investigational Site

    Beijing, 100045
    China

    Site Not Available

  • Ascendis Pharma Investigational Site

    Guangzhou, 510080
    China

    Site Not Available

  • Ascendis Pharma Investigational Site

    Hangzhou, 310053
    China

    Site Not Available

  • Ascendis Pharma Investigational Site

    Shanghai, 20082
    China

    Site Not Available

  • Ascendis Pharma Investigational Site

    Shenzhen, 518053
    China

    Site Not Available

  • Ascendis Pharma Investigational Site

    Wuhan, 430030
    China

    Site Not Available

  • Ascendis Pharma Investigational Site

    Copenhagen, Hovedstaden 2100
    Denmark

    Site Not Available

  • Ascendis Pharma Investigational Site

    Paris, 75743
    France

    Site Not Available

  • Ascendis Pharma Investigational Site

    Berlin, 13353
    Germany

    Site Not Available

  • Ascendis Pharma Investigational Site

    Dublin, 1
    Ireland

    Site Not Available

  • Ascendis Pharma Investigational Site

    Milano, 20132
    Italy

    Site Not Available

  • Ascendis Pharma Investigational Site

    Rome, 00165
    Italy

    Site Not Available

  • Ascendis Pharma Investigational Site

    Auckland, 1142
    New Zealand

    Site Not Available

  • Ascendis Pharma Investigational Site

    Coimbra, 3000
    Portugal

    Site Not Available

  • Ascendis Pharma Investigational Site

    Barcelona, 08035
    Spain

    Site Not Available

  • Ascendis Pharma Investigational Site

    Esplugues De Llobregat, 8950
    Spain

    Site Not Available

  • Ascendis Pharma Investigational Site

    Madrid, 28046
    Spain

    Site Not Available

  • Ascendis Pharma Investigational Site

    Vitoria-Gasteiz, 1008
    Spain

    Site Not Available

  • Ascendis Pharma Investigational Site

    Lausanne, 1005
    Switzerland

    Site Not Available

  • Ascendis Pharma Investigational Site

    Glasgow, G51 4TF
    United Kingdom

    Site Not Available

  • Ascendis Pharma Investigational Site

    Liverpool, LA12 2AP
    United Kingdom

    Site Not Available

  • Ascendis Pharma Investigational Site

    London, SE1 7EU
    United Kingdom

    Site Not Available

  • Ascendis Pharma Investigational Site

    Little Rock, Arkansas 72211
    United States

    Site Not Available

  • Ascendis Pharma Investigational Site

    Oakland, California 94609
    United States

    Site Not Available

  • Ascendis Pharma Investigational Site

    Aurora, Colorado 80045
    United States

    Site Not Available

  • Ascendis Pharma Investigational Site

    Wilmington, Delaware 19803
    United States

    Site Not Available

  • Ascendis Pharma Investigational Site

    Atlanta, Georgia 30322
    United States

    Site Not Available

  • Ascendis Pharma Investigational Site

    Boston, Massachusetts 02115
    United States

    Site Not Available

  • Ascendis Pharma Investigational Site

    Saint Paul, Minnesota 55102
    United States

    Site Not Available

  • Ascendis Pharma Investigational Site

    Columbia, Missouri 65212
    United States

    Site Not Available

  • Ascendis Pharma Investigational Site

    Buffalo, New York 14203
    United States

    Site Not Available

  • Ascendis Pharma Investigational Site

    Houston, Texas 77030
    United States

    Site Not Available

  • Ascendis Pharma Investigational Site

    Norfolk, Virginia 23507
    United States

    Site Not Available

  • Ascendis Pharma Investigational Site

    Seattle, Washington 98105
    United States

    Site Not Available

  • Ascendis Pharma Investigational Site

    Madison, Wisconsin 53705
    United States

    Site Not Available

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