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

NCT ID: NCT03875534

Last Updated: 2024-03-19

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

260 participants

Study Classification

OBSERVATIONAL

Study Start Date

2019-06-19

Study Completion Date

2024-01-12

Brief Summary

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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

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Conditions

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Achondroplasia

Study Design

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Observational Model Type

CASE_ONLY

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

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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
Minimum Eligible Age

0 Years

Maximum Eligible Age

8 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Ascendis Pharma A/S

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Ascendis Pharma Investigational Site

Little Rock, Arkansas, United States

Site Status

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Oakland, California, United States

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Aurora, Colorado, United States

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Wilmington, Delaware, United States

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Saint Paul, Minnesota, United States

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Columbia, Missouri, United States

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Buffalo, New York, United States

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Houston, Texas, United States

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Seattle, Washington, United States

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Madison, Wisconsin, United States

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Parkville, Victoria, Australia

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Linz, , Austria

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Edmonton, Alberta, Canada

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Ottawa, Ontario, Canada

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Montreal, Quebec, Canada

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Beijing, , China

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Guangzhou, , China

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Hangzhou, , China

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Shanghai, , China

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Shenzhen, , China

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Wuhan, , China

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Copenhagen, Capital Region, Denmark

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Paris, , France

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Berlin, , Germany

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Dublin, , Ireland

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Milan, , Italy

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Auckland, , New Zealand

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Coimbra, , Portugal

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Barcelona, , Spain

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Esplugues de Llobregat, , Spain

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Madrid, , Spain

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Vitoria-Gasteiz, , Spain

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Lausanne, , Switzerland

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Glasgow, , United Kingdom

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London, , United Kingdom

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Countries

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United States Australia Austria Canada China Denmark France Germany Ireland Italy New Zealand Portugal Spain Switzerland United Kingdom

Other Identifiers

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TCC-NHS-01

Identifier Type: -

Identifier Source: org_study_id

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