Ascendis - ACHieve study (21st Oct 2019 update)
A Multi-center, Longitudinal, Observational Study of Children With Achondroplasia
Update - 21th Oct 2019
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.
All content in ClinicalTrials.gov identifier (NCT number): NCT03875534
All content in ClinicalTrials.gov identifier (NCT number): NCT03875534
Study Type : | Observational (no drug is administered) |
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 :
- 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 :
- 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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
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:
- 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
Contacts
Contact: Medical Affairs Director | +1 855-795-2467 ext 4420 | This email address is being protected from spambots. You need JavaScript enabled to view it. |
Study Director: | Will Charlton, MD | Ascendis Pharma A/S |
Locations
United States, Arkansas | |
Ascendis Pharma Investigational Site | Recruiting |
Little Rock, Arkansas, United States, 72211 | |
United States, California | |
Ascendis Pharma Investigational Site | Not yet recruiting |
Oakland, California, United States, 94609 | |
United States, Delaware | |
Ascendis Pharma Investigational Site | Not yet recruiting |
Wilmington, Delaware, United States, 19803 | |
United States, Massachusetts | |
Ascendis Pharma Investigational Site | Not yet recruiting |
Boston, Massachusetts, United States, 02115 | |
United States, Minnesota | |
Ascendis Pharma Investigational Site | Recruiting |
Saint Paul, Minnesota, United States, 55102 | |
United States, Missouri | |
Ascendis Pharma Investigational Site | Recruiting |
Columbia, Missouri, United States, 65212 | |
United States, Texas | |
Ascendis Pharma Investigational Site | Not yet recruiting |
Houston, Texas, United States, 77030 | |
United States, Washington | |
Ascendis Pharma Investigational Site | Recruiting |
Seattle, Washington, United States, 98105 | |
United States, Wisconsin | |
Ascendis Pharma Investigational Site | Not yet recruiting |
Madison, Wisconsin, United States, 53705 | |
Australia, Victoria | |
Ascendis Pharma Investigational Site | Recruiting |
Parkville, Victoria, Australia, 3052 | |
Austria | |
Ascendis Pharma Investigational Site | Recruiting |
Linz, Austria, 4020 | |
Canada | |
Ascendis Pharma Investigational Site | Not yet recruiting |
Montréal, Canada, H3T 1C5 | |
Germany | |
Ascendis Pharma Investigational Site | Not yet recruiting |
Berlin, Germany, 13353 | |
Ireland | |
Ascendis Pharma Investigational Site | Not yet recruiting |
Dublin, Ireland, 1 | |
Italy | |
Ascendis Pharma Investigational Site | Not yet recruiting |
Rome, Italy, 00165 | |
Portugal | |
Ascendis Pharma Investigational Site | Not yet recruiting |
Coimbra, Portugal, 3000 | |
Spain | |
Ascendis Pharma Investigational Site | Not yet recruiting |
Barcelona, Spain, 08035 | |
Ascendis Pharma Investigational Site | Not yet recruiting |
Madrid, Spain, 28046 | |
Switzerland | |
Ascendis Pharma Investigational Site | Not yet recruiting |
Lausanne, Switzerland, 1005 | |
United Kingdom | |
Ascendis Pharma Investigational Site | Not yet recruiting |
Glasgow, United Kingdom, G51 4TF | |
Ascendis Pharma Investigational Site | Not yet recruiting |
Liverpool, United Kingdom, LA12 2AP | |
Ascendis Pharma Investigational Site | Not yet recruiting |
London, United Kingdom, SE1 7EU |