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

• ClinicalTrials.gov Identifier: NCT03875534
• Recruitment Status: Recruiting
• First Posted: March 14, 2019
• Last Update Posted: February 8, 2021
• Sponsor: Ascendis Pharma A/S
• 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: February 8, 2021
• 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

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

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, New Zealand, Portugal, Spain, Switzerland, United Kingdom, United States

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: Marie-Louise Hartoft-Nielsen, MD, PhD; Ascendis Pharma A/S

• PRS Account: Ascendis Pharma A/S
• Verification Date: February 2021