AchondroplasiaNCT03794609
Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia.
An observational study of Achondroplasia, run by Pfizer at 57 sites in 14 countries.
Trial summary
This is a registry study in children with achondroplasia, age 0-10 years, to be conducted at multiple clinical centers in several countries. Information collected will include in anthropometric characteristics, related symptoms, tests, \& treatments Children's information will be collected in the registry for a… Read more about this study
- Who is it for?
- Ages 0 to 15 years
- Conditions the study is for
- Achondroplasia
- How many people
- 315 participants
- Where
- 57 sitesin 14 countries
- Study dates
- 15 Jun 2018to 13 Jan 2023
- Phase
- Observational
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 15 years
- All sexes
- No healthy volunteers
- Child
Study population: It is assumed that each of the study sites will enroll approximately 10-15 children of both genders and of various ages (0-10 years old). The total number of children planned to be enrolled across all sites is approximately 200.
Who may be able to join
- Written informed consent is obtained from the children's parent(s) / legal guardian(s) before any study-related activity is carried out
- The child is able to provide written informed assent, where this is required according to national legislation, before any study related activity is carried out
- The child has been diagnosed as having achondroplasia documented by clinical diagnosis
- The child is between 0 years and 10 years of age, inclusive, on the date of consent / assent
- The investigator has considered the family and prospective participating child being able to comply with the study procedures
Who cannot join
- The child has a diagnosis of hypochondroplasia or any short stature condition other than achondroplasia (eg, spondyloepiphyseal dysplasia congenital [SEDC], pseudoachondroplasia, trisomy 21)
- The child has any medical condition that may impact growth or where the treatment is known to impact growth, such as but not limited to hypothyroidism or hyperthyroidism, insulin-requiring diabetes mellitus, autoimmune inflammatory disease (including celiac disease, systemic lupus erythematosus [SLE], juvenile dermatomyositis, scleroderma, and others), autonomic neuropathy, or inflammatory bowel disease
- Treatment in the previous 12 months prior to consent and assent with growth hormone, insulin-like growth factor 1 (IGF-1), anabolic steroids, or any other drug expected to affect growth velocity
- Any surgery that affects the growth plate of the long bones that is planned, or has occurred in the past 18 months
- Participation in any interventional study (investigational product or device) for treatment of achondroplasia or short stature
- Has had bone-related surgery impacting assessment of anthropometric measurements or is expected to have it during the study period. Children with previous limb-lengthening surgery may enroll if surgery occurred at least 18 months prior to the date of consent/assent and healing is complete without sequelae as determined by the investigator
- Has any condition that in the view of the investigator places the child at high risk of poor compliance with the visit schedule or of not completing the study.
- Any concurrent disease or condition that in the view of the investigator would interfere with study participation
Trial locations and contacts
57 sites in 14 countries.
57 sites in 14 countries
United States14 sites
The John Hopkins
Baltimore, Maryland, 21287
The Johns Hopkins Hospital
Baltimore, Maryland, 21287
Boston Children's Hospital
Boston, Massachusetts, 02115
Boston Childrens Hospital
Boston, Massachusetts, 02115
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Univesity of Missouri - Columbia
Columbia, Missouri, 65203
Texas Children Hospital
Houston, Texas, 77030
Texas Children'S Hospital
Houston, Texas, 77030
Los Angeles Biomedical Research Institute At Harbour-UCLA Medical Centre
Los Angeles, California, 90502
University of Utah Health
Salt Lake City, Utah, 84132
Seattle Children's Hospital
Seattle, Washington, 98105
Los Angeles BioMedical Research Institute at Harbor-UCLA Medical Center
Torrance, California, 90502
Alfred I. duPont Hospital for Children - Wilmington
Wilmington, Delaware, 419803-3607
Alfred I. duPont Hospital for Children
Wilmington, Delaware, 419803
United Kingdom8 sites
Bristol Royal Childrens Hospital
Bristol, BS2 8BJ
University Hospitals Bristol NHS Foundation Trust
Bristol, BS2 8BJ
Guy's & St Thomas NHS Trust, St Thomas' Hospital
London, SE1 7EH
Guys & St Thomas NHS Trust
London, SE1 7EH
Newcastle Hospital NHS Foundation Trust
Newcastle, NE1 3BZ
Newcastle Hospitals NHS Foundation Trust
Newcastle upon Tyne, NE1 3BZ
Sheffield Children's NHS Foundation Trust
Sheffield, S10 2TH
Sheffield Childrens NHS Foundation Trust
Sheffield, S10 2TH
Italy7 sites
ASST Lariana Como
Como, 22042
IRCCS Istituto Giannina Gaslini
Genova, Genoa, 16147
IRCCS Istituto Giannina Gaslini
Genova, 6147
San Raffaele Hospital
Milan, 20132
Center for Rare Diseases, Department of Pediatrics, Polo Salute Donna e Bambino
Rome, 00168
Fondazione Policlinico Universitario A.Gemelli
Rome, 00168
ASST Lariana Como
San Fermo della Battaglia, COMO, 22042
Germany4 sites
Center for Rare Skeletal Diseases in Childhood and Adolescence
Cologne, 50937
University Hospital of Cologne
Cologne, 50937
Otto-von-Guericke- Universitat Magdeburg
Magdeburg, 39120
Otto-von-Guericke-Universitat Magdeburg
Magdeburg, 39120
Canada3 sites
CHU Sainte-Justine
Montreal, Quebec, H3T 1C5
The Hospital for Sick Children
Toronto, Ontario, M5G 1X8
The Hospital for Sick Children
Toronto
France3 sites
Centre Hospitalier Universitaire La Timone
Marseille, 13385
Centre Hospitalier Univesitaire La Timone
Marseille, 13385
Hopital Necker-Enfants Malades
Paris, 75015
Japan3 sites
Osaka Women's and Children's Hospital
Izumi, Osaka, 594-1101
Okayama University Hospital
Okayama, 700-8558
Osaka University Hospital
Suita, Osaka, 565-0871
Spain3 sites
Vall d'Hebron University Hospital
Barcelona, 08035
Vall D'Hebron Univeristy Hospital
Barcelona
Hospital Quironsalud Malaga
Málaga
Australia2 sites
Childrens Hospital Melbourne
Melbourne
Murdoch Childrens Research Institute
Parkville, Victoria, 3052
Belgium2 sites
Antwerp University Hospital
Antwerp, 2650
Universitair Ziekenhuis Antwerpen (UZA)
Edegem, 2650
China2 sites
Beijing Children's Hospital, Capital Medical University
Beijing, 100045
The First Affiliated Hospital, Sun Yat-sen University
Guangzhou, Guangdong, 510080
Denmark2 sites
Righospitalet
Copenhagen, 2100
Bispebjerg Hospital
Copenhagen, 2400
Portugal2 sites
Hospital Pediatrico de Coimbra
Coimbra, 3000-602
Hospital Pediátrico de Coimbra
Coimbra, 3000-602
Switzerland2 sites
CENTRE HOSPITALIER UNIVERSITAIRE VAUDOIS -CHUV Hôpital Neslé
Lausanne, 1011
Centre Hospitalier Universitaire Vaudois -CHUV
Lausanne, 1011
About this study
Official title: An International, Prospective Registry Investigating the Natural History of Participants With Achondroplasia
This is a registry study in children with achondroplasia, age 0-10 years, to be conducted at multiple clinical centers in several countries. Information collected will include in anthropometric characteristics, related symptoms, tests, \& treatments
Children's information will be collected in the registry for a maximum of 5 years.
- Conditions
- Achondroplasia
- Medical subject headings
- Achondroplasia
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Standing Height
Standing height (for participants aged ≥2 years) is an assessment of maximum vertical size. Standing height was measured using a stadiometer with a fixed vertical backboard and an adjustable head piece. Height in supine position (or length for participants aged <2 years) was measured using measuring board. Three valid measurements were performed at each visit and the average of the 3 valid measurements was reported and used in descriptive summaries. Data is reported according to age group (<2 years and ≥2 years) and gender of the participants.
Measured: Baseline, 1 year, 2 years, 3 years, 4 years
Change From Baseline in Sitting Height
Sitting height (for participants aged ≥2 years) is a measure of the trunk of the body from the buttocks to the top of the head when the participant is sitting upright. Sitting height was measured using a stadiometer and sitting height table. Crown-rump length (for participants aged <2 years) was measured using measuring board. Three valid measurements were performed at each visit and the average of the 3 valid measurements was reported and used in descriptive summaries. Data is reported according to age group (<2 years and ≥2 years) and gender of the participants.
Measured: Baseline, 1 year, 2 years, 3 years, 4 years
Change From Baseline in Knee Height
Knee height means lower segment length (lower segment=standing height - sitting height). Knee height was measured using knee height caliper. Three valid measurements were performed at each visit and the average of the 3 valid measurements was reported and used in descriptive summaries. Data is reported according to age group (<2 years, 2-10 years, and ≥10 years) and gender of the participants.
Measured: Baseline, 1 year, 2 years, 3 years, 4 years
Change From Baseline in Head Circumference
Head circumference is a measurement of the circumference of the child's head at its largest area, above the eyebrows and ears and around the back of the head. Head circumfernce was measured using measuring tape. Three valid measurements were performed at each visit and the average of the 3 valid measurements was reported and used in descriptive summaries. Data is reported according to age group (<2 years, 2-10 years, and ≥10 years) and gender of the participants.
Measured: Baseline, 1 year, 2 years, 3 years, 4 years
Change From Baseline in Arm Span
Arm span measurement is the distance between fingertips when the arms are outstretched. All participants should be measured supine lying on the measurement grid. Three valid measurements were performed at each visit and the average of the 3 valid measurements was reported and used in descriptive summaries. Data is reported according to age group (<2 years, 2-10 years, and ≥10 years) and gender of the participants.
Measured: Baseline, 1 year, 2 years, 3 years, 4 years
Number of Participants With Achondroplasia-Related Treatments
Achondroplasia-related treatments were collected in a prospective and standardized fashion to allow characterization of the natural history of the disease course in the youngest participants in relation to the level of actual disease burden over time due to treatments, as well as data to support identification of possible risk factors. Data is reported according to the gender of the participants.
Measured: Baseline to end of study visit (up to 55 months)
Secondary outcomes (1)
Baseline and Post-baseline Measurements of Serum Collagen X Marker
Collagen X Marker (CXM), is a degradation by-product of endochondral ossification that is released into the circulation in proportion to overall growth plate activity. This marker corresponds to the rate of linear bone growth at time of measurement as recently demonstrated in children of normal average growth. Data is reported according to age group (<2 years, 2-10 years, and ≥10 years) and gender of the participants.
Measured: Baseline, 1 year, 2 years, 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jan 2019
- Study start15 Jun 2018
- Primary completion13 Jan 2023
- Study completion13 Jan 2023
Why it stopped: The study was terminated on 18th November 2022. The decision to terminate the trial was not based on any safety and/or efficacy concerns.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for AchondroplasiaStudies of the same condition, open and under way.
- More trials run by PfizerThe sponsor's other registered studies.
- 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
- 315 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pfizer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Pfizer Pfizer CT.gov Call Center, Study director, Pfizer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03794609
- Sponsor's protocol ID
- TA46-002
- Other IDs
- C4181001
- Status last verified
- Jul 2024
- First submitted
- 19 Nov 2018
- Last updated
- 17 Oct 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical\_trials/trial\_data\_and\_results/data\_requests.
Study at a glance
From the registry record, last updated by the sponsor on 17 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An International, Prospective Registry Investigating the Natural History of Participants With Achondroplasia
- ClinicalTrials.gov ID
- NCT03794609
- Acronym / sponsor ID
- TA46-002
- Conditions
- Achondroplasia
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: The study was terminated on 18th November 2022. The decision to terminate the trial was not based on any safety and/or efficacy concerns.
- Enrolment
- 315 participants (actual)
- Lead sponsor
- PfizerIndustry (pharma, biotech, devices)
- Study start
- 15 Jun 2018 (actual)
- Primary completion
- 13 Jan 2023 (actual)
- Study completion
- 13 Jan 2023 (actual)
- First posted
- 7 Jan 2019 (actual)
- Results
- Posted 17 Oct 2024
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 registry study in children with achondroplasia, age 0-10 years, to be conducted at multiple clinical centers in several countries. Information collected will include in anthropometric characteristics, related symptoms, tests, \& treatments
Children's information will be collected in the registry for a maximum of 5 years.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Ages
- 0 to 15 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- It is assumed that each of the study sites will enroll approximately 10-15 children of both genders and of various ages (0-10 years old). The total number of children planned to be enrolled across all sites is approximately 200.
Key inclusion criteria (5 listed)
- Written informed consent is obtained from the children's parent(s) / legal guardian(s) before any study-related activity is carried out
- The child is able to provide written informed assent, where this is required according to national legislation, before any study related activity is carried out
- The child has been diagnosed as having achondroplasia documented by clinical diagnosis
- The child is between 0 years and 10 years of age, inclusive, on the date of consent / assent
- The investigator has considered the family and prospective participating child being able to comply with the study procedures
Key exclusion criteria (8 listed)
- The child has a diagnosis of hypochondroplasia or any short stature condition other than achondroplasia (eg, spondyloepiphyseal dysplasia congenital [SEDC], pseudoachondroplasia, trisomy 21)
- The child has any medical condition that may impact growth or where the treatment is known to impact growth, such as but not limited to hypothyroidism or hyperthyroidism, insulin-requiring diabetes mellitus, autoimmune inflammatory disease (including celiac disease, systemic lupus erythematosus [SLE], juvenile dermatomyositis, scleroderma, and others), autonomic neuropathy, or inflammatory bowel disease
- Treatment in the previous 12 months prior to consent and assent with growth hormone, insulin-like growth factor 1 (IGF-1), anabolic steroids, or any other drug expected to affect growth velocity
- Any surgery that affects the growth plate of the long bones that is planned, or has occurred in the past 18 months
- Participation in any interventional study (investigational product or device) for treatment of achondroplasia or short stature
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
Change From Baseline in Standing Height
Time frame: Baseline, 1 year, 2 years, 3 years, 4 years
Standing height (for participants aged ≥2 years) is an assessment of maximum vertical size. Standing height was measured using a stadiometer with a fixed vertical backboard and an adjustable head piece. Height in supine position (or length for participants aged <2 years) was measured using measuring board. Three valid measurements were performed at each visit and the average of the 3 valid measurements was reported and used in descriptive summaries. Data is reported according to age group (<2 years and ≥2 years) and gender of the participants.
Change From Baseline in Sitting Height
Time frame: Baseline, 1 year, 2 years, 3 years, 4 years
Sitting height (for participants aged ≥2 years) is a measure of the trunk of the body from the buttocks to the top of the head when the participant is sitting upright. Sitting height was measured using a stadiometer and sitting height table. Crown-rump length (for participants aged <2 years) was measured using measuring board. Three valid measurements were performed at each visit and the average of the 3 valid measurements was reported and used in descriptive summaries. Data is reported according to age group (<2 years and ≥2 years) and gender of the participants.
Change From Baseline in Knee Height
Time frame: Baseline, 1 year, 2 years, 3 years, 4 years
Knee height means lower segment length (lower segment=standing height - sitting height). Knee height was measured using knee height caliper. Three valid measurements were performed at each visit and the average of the 3 valid measurements was reported and used in descriptive summaries. Data is reported according to age group (<2 years, 2-10 years, and ≥10 years) and gender of the participants.
Change From Baseline in Head Circumference
Time frame: Baseline, 1 year, 2 years, 3 years, 4 years
Head circumference is a measurement of the circumference of the child's head at its largest area, above the eyebrows and ears and around the back of the head. Head circumfernce was measured using measuring tape. Three valid measurements were performed at each visit and the average of the 3 valid measurements was reported and used in descriptive summaries. Data is reported according to age group (<2 years, 2-10 years, and ≥10 years) and gender of the participants.
Change From Baseline in Arm Span
Time frame: Baseline, 1 year, 2 years, 3 years, 4 years
Arm span measurement is the distance between fingertips when the arms are outstretched. All participants should be measured supine lying on the measurement grid. Three valid measurements were performed at each visit and the average of the 3 valid measurements was reported and used in descriptive summaries. Data is reported according to age group (<2 years, 2-10 years, and ≥10 years) and gender of the participants.
Number of Participants With Achondroplasia-Related Treatments
Time frame: Baseline to end of study visit (up to 55 months)
Achondroplasia-related treatments were collected in a prospective and standardized fashion to allow characterization of the natural history of the disease course in the youngest participants in relation to the level of actual disease burden over time due to treatments, as well as data to support identification of possible risk factors. Data is reported according to the gender of the participants.
1 secondary outcome
Baseline and Post-baseline Measurements of Serum Collagen X Marker
Time frame: Baseline, 1 year, 2 years, 3 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Oct 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- To obtain contact information for a study center near you, click here.Link provided by the sponsor.
- Individual participant dataSharing planned: No. Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
Related trials for Achondroplasia
Enrolling by invitationPhase 2
Extension Study of Infigratinib in Children With Achondroplasia (ACH)
Achondroplasia
RecruitingPhase 2/3
Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
Achondroplasia
Active, not recruitingPhase 2
A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia.
Achondroplasia
Active, not recruitingPhase 3
Evaluate the Efficacy and Safety of KK8398 in Patients With Achondroplasia（AOBA Study）
Achondroplasia
RecruitingPhase 2
A Study of TYRA-300 in Children With Achondroplasia: BEACH301
Achondroplasia
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.
Pfizer Pfizer CT.gov Call Center study director
Pfizer
57 sites in 14 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Achondroplasia, run by Pfizer at 57 sites in 14 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 8 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: stopped early, as of the sponsor's last update on 17 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 57 sites in United States, United Kingdom, Italy, Germany, Canada and 9 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?
Yes. Results are posted in the registry (first posted 17 Oct 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Change From Baseline in Standing Height (time frame: Baseline, 1 year, 2 years, 3 years, 4 years). The study lists 6 primary outcomes in all. 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 17 Oct 2024.