AchondroplasiaNCT03875534
A Multi-center, Longitudinal, Observational Study of Children With Achondroplasia
An observational study of Achondroplasia, run by Ascendis Pharma A/S at 35 sites in 15 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 0 to 8 years
- Conditions the study is for
- Achondroplasia
- How many people
- 260 participants
- Where
- 35 sitesin 15 countries
- Study dates
- 19 Jun 2019to 12 Jan 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 to 8 years
- All sexes
- No healthy volunteers
- Child
Study population: Infants and children with achondroplasia from birth to 8 years of age.
Who may be able to join
- 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)
Who cannot join
- 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
Trial locations and contacts
35 sites in 15 countries.
35 sites in 15 countries
United States10 sites
Ascendis Pharma Investigational Site
Aurora, Colorado, 80045
Ascendis Pharma Investigational Site
Buffalo, New York, 14203
Ascendis Pharma Investigational Site
Columbia, Missouri, 65212
Ascendis Pharma Investigational Site
Houston, Texas, 77030
Ascendis Pharma Investigational Site
Little Rock, Arkansas, 72211
Ascendis Pharma Investigational Site
Madison, Wisconsin, 53705
Ascendis Pharma Investigational Site
Oakland, California, 94609
Ascendis Pharma Investigational Site
Saint Paul, Minnesota, 55102
Ascendis Pharma Investigational Site
Seattle, Washington, 98105
Ascendis Pharma Investigational Site
Wilmington, Delaware, 19803
China6 sites
Ascendis Pharma Investigational Site
Beijing, 100045
Ascendis Pharma Investigational Site
Guangzhou, 510080
Ascendis Pharma Investigational Site
Hangzhou, 310053
Ascendis Pharma Investigational Site
Shanghai, 20082
Ascendis Pharma Investigational Site
Shenzhen, 518053
Ascendis Pharma Investigational Site
Wuhan, 430030
Spain4 sites
Ascendis Pharma Investigational Site
Barcelona, 08035
Ascendis Pharma Investigational Site
Esplugues de Llobregat, 8950
Ascendis Pharma Investigational Site
Madrid, 28046
Ascendis Pharma Investigational Site
Vitoria-Gasteiz, 1008
Canada3 sites
Ascendis Pharma Investigational Site
Edmonton, Alberta, T6G-1C9
Ascendis Pharma Investigational Site
Montreal, Quebec, H3T 1C5
Ascendis Pharma Investigational Site
Ottawa, Ontario, 51H 8L1
United Kingdom2 sites
Ascendis Pharma Investigational Site
Glasgow, G51 4TF
Ascendis Pharma Investigational Site
London, SE1 7EU
Australia1 site
Ascendis Pharma Investigational Site
Parkville, Victoria, 3052
Denmark1 site
Ascendis Pharma Investigational Site
Copenhagen, Capital Region, 2100
About this study
Official title: 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.
- Conditions
- Achondroplasia
- Keywords
- Achondroplasia, Dwarfism
- Medical subject headings
- Achondroplasia, Dwarfism
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized height velocity (centimeters/year) in children with achondroplasia
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
Measured: Up to 5 years
Secondary outcomes (1)
Collection of natural history of achondroplasia symptoms in children with achondroplasia
To characterize achondroplasia symptoms in children with achondroplasia
Measured: Up to 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Mar 2019
- Study start19 Jun 2019
- Primary completion12 Jan 2024
- Study completion12 Jan 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AchondroplasiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 260 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ascendis Pharma A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03875534
- Sponsor's protocol ID
- TCC-NHS-01
- Status last verified
- Mar 2024
- First submitted
- 7 Mar 2019
- Last updated
- 19 Mar 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- ACHieve: A Multi-center, Longitudinal, Observational Study of Children With Achondroplasia
- ClinicalTrials.gov ID
- NCT03875534
- Acronym / sponsor ID
- TCC-NHS-01
- Conditions
- Achondroplasia
- Keywords
- Achondroplasia, Dwarfism
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 260 participants (actual)
- Lead sponsor
- Ascendis Pharma A/SIndustry (pharma, biotech, devices)
- Study start
- 19 Jun 2019 (actual)
- Primary completion
- 12 Jan 2024 (actual)
- Study completion
- 12 Jan 2024 (actual)
- First posted
- 14 Mar 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Ages
- 0 to 8 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Infants and children with achondroplasia from birth to 8 years of age.
Key inclusion criteria (5 listed)
- 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)
Key exclusion criteria (7 listed)
- 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.)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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
1 secondary outcome
Collection of natural history of achondroplasia symptoms in children with achondroplasia
Time frame: Up to 5 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Mar 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
35 sites in 15 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Achondroplasia, run by Ascendis Pharma A/S at 35 sites in 15 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 35 sites in United States, China, Spain, Canada, United Kingdom and 10 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Annualized height velocity (centimeters/year) in children with achondroplasia (time frame: Up to 5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Mar 2024.