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



Status:Recruiting
Healthy:No
Age Range:Any - 8
Updated:3/21/2019
Start Date:March 2019
End Date:June 2024
Contact:Medical Affairs Director
Email:achieve-study@ascendispharma.com
Phone:+1 650-352-8389

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ACHieve: 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.


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
We found this trial at
7
sites
Atlanta, Georgia 30322
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Atlanta, GA
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Columbia, Missouri 65212
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Columbia, MO
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Little Rock, Arkansas 72202
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Little Rock, AR
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Oakland, California 94609
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Oakland, CA
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Parkville,
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Seattle, Washington 98105
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Seattle, WA
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Wilmington, Delaware 19803
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Wilmington, DE
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