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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 : June 27, 2019
Sponsor:
Information provided by (Responsible Party):
Ascendis Pharma A/S

Tracking Information
First Submitted Date March 7, 2019
First Posted Date March 14, 2019
Last Update Posted Date June 27, 2019
Actual Study Start Date June 19, 2019
Estimated Primary Completion Date June 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: March 13, 2019)
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
Original Primary Outcome Measures Same as current
Change History Complete list of historical versions of study NCT03875534 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures
 (submitted: March 13, 2019)
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
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title A Multi-center, Longitudinal, Observational Study of Children With Achondroplasia
Official Title ACHieve: A Multi-center, Longitudinal, Observational Study of Children With Achondroplasia
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.
Detailed Description Not Provided
Study Type Observational
Study Design Observational Model: Case-Only
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Probability Sample
Study Population Infants and children with achondroplasia from birth to 8 years of age.
Condition Achondroplasia
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: March 13, 2019)
200
Original Estimated Enrollment Same as current
Estimated Study Completion Date June 2024
Estimated Primary Completion Date June 2024   (Final data collection date for primary outcome measure)
Eligibility 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
Sex/Gender
Sexes Eligible for Study: All
Ages up to 8 Years   (Child)
Accepts Healthy Volunteers No
Contacts
Contact: Medical Affairs Director +1 855-795-2467 ext 4420 achieve-study@ascendispharma.com
Listed Location Countries Australia,   Austria,   Canada,   Germany,   Ireland,   Italy,   Portugal,   Spain,   Switzerland,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT03875534
Other Study ID Numbers TCC-NHS-01
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: No
Responsible Party Ascendis Pharma A/S
Study Sponsor Ascendis Pharma A/S
Collaborators Not Provided
Investigators
Study Director: Will Charlton, MD Ascendis Pharma A/S
PRS Account Ascendis Pharma A/S
Verification Date June 2019