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Completed

NCT Number: NCT03875534

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.

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Key information

Age range

0 year–8 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Ascendis Pharma Investigational Site, Parkville, Victoria, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Primary outcomes

  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 outcomes

  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

Sponsors and collaborators

Lead sponsor

Ascendis Pharma A/S

Industry

Registry information

Official study title

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

Important dates

Study start
2019
Primary completion
2024
Study completion
2024
First posted
Mar 14, 2019
Registry last updated
Mar 19, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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