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

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ClinicalTrials.gov Identifier: NCT03875534
Recruitment Status : Recruiting
First Posted : March 14, 2019
Last Update Posted : September 6, 2019
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:   up 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: Medical Affairs Director +1 855-795-2467 ext 4420 achieve-study@ascendispharma.com

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Sponsors and Collaborators
Ascendis Pharma A/S
Investigators
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Study Director: Will Charlton, MD Ascendis Pharma A/S

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Responsible Party: Ascendis Pharma A/S
ClinicalTrials.gov Identifier: NCT03875534     History of Changes
Other Study ID Numbers: TCC-NHS-01
First Posted: March 14, 2019    Key Record Dates
Last Update Posted: September 6, 2019
Last Verified: September 2019
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