AchondroplasiaNCT05659719
A Study to Learn About Recifercept in Patients With Achondroplasia
An observational study of Achondroplasia, run by Pfizer at 1 site in United States.
Trial summary
The purpose of this study is to learn about the study medicine (called recifercept) in people with achondroplasia. Achondroplasia is a very rare disease and patients of achondroplasia have short arms and legs. Read more about this study
- Who is it for?
- Ages 3 months to 10 years
- Conditions the study is for
- Achondroplasia
- What is being tested?
- Recifercept
- How many people
- 248 participants
- Where
- 1 siteUnited States
- Study dates
- 19 Oct 2022to 16 May 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 3 months to 10 years
- All sexes
- No healthy volunteers
- Child
Study population: Achondroplasia patients, aged 3 months to 11 years old with documented and confirmed genetic diagnosis of achondroplasia, who have completed at least 2 valid height and length measurement in the natural history study and have at least 6 months of follow-up data in either the natural history study or the phase 2 recifercept trial.
Who may be able to join
- All patients from Study C4181005 who have completed Visits 1 through 11 (at D183) will be included in this project.
- To construct a concurrent external control, patients from Study C4181001 will need to meet the following inclusion criteria from Study C4181005 to be eligible for inclusion:
- Documented, confirmed genetic diagnosis of achondroplasia from historical medical records (test must have been performed at a laboratory fully accredited for genetic testing under local regulations)
- Aged ≥ 3 months to <11 years (up to the day before 11th birthday inclusive) at time of enrollment into the observational natural history study.
- Havecompleted at least 2 valid height/length measurements (at least 3 months apart)
- Assessed for Tanner stage 1 during physical examination before or at enrollment (must include assessment of breast development for females, testicular stage for males)
- Able to stand independently for height measurements (if ≥ 2 years of age at enrollment); If aged <2 years at enrollment, has a documented historical MRI brain/cervical spine performed in the previous 12 months.
- Have at least 6 months of available follow-up data after enrollment into the natural history study
Who cannot join
- Patients meeting any of the following criteria will not be included in the study:
- Presence of severe obesity (BMI>95% percentile on Hoover-Fong BMI charts);
- Body weight <7kg or >30kg
- History of chronic kidney disease (CKD) or renal impairment
- History of receipt of any treatment that are known to potentially affect growth (including oral steroids > 5 days in the last 6 months before enrollment, high dose inhaled corticosteroids (>800 mcg/day beclametasone equivalent) and medication for attention deficient hyperactivity disorder.
- Less than 6 months since fracture or surgical procedure of any bone determined from the baseline visit date.
- Presence of any internal guided growth plates/devices
- History of removal of internal guided growth plates/devices within 6 months prior to enrollment
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Pilot Project to Evaluate the Feasibility of Constructing a Concurrent External Control for Recifercept
The purpose of this study is to learn about the study medicine (called recifercept) in people with achondroplasia. Achondroplasia is a very rare disease and patients of achondroplasia have short arms and legs. The study will include data already collected from a recifercept clinical trial and data collected from a separate study of achondroplasia. This study will compare patient experiences and will help the investigators determine if the study medicine, recifercept, is effective.
- Conditions
- Achondroplasia
- Being tested
- Recifercept
- Medical subject headings
- Achondroplasia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ReciferceptOther
Patients received recifercept intervention in the phase 2 clinical trial
| Study group | Role | What they receive |
|---|---|---|
| Recifercept cohort | Achondroplasia patients enrolled in the recifercept phase 2 clinical trial Receives: Other: Recifercept | |
| Natural history cohort | Achondroplasia patients enrolled in the achondroplasia natural history study |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean Height Growth at Month 3
Height growth was defined as ratio of observed change from baseline in standing height (centimeter) to expected change from baseline (centimeter) in reference population. For C4181001 cohort, baseline value was defined as the value recorded at the date of informed consent, whereas for C4181005 cohort the baseline value was the value taken at Day 1 before dosing.
Measured: Baseline, Month 3 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]
Mean Height Growth at Month 6
Height growth was defined as ratio of observed change from baseline in standing height (centimeter) to expected change from baseline (centimeter) in reference population. For C4181001 cohort, baseline value was defined as the value recorded at the date of informed consent, whereas for C4181005 cohort the baseline value was the value taken at Day 1 before dosing.
Measured: Baseline, Month 6 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]
Mean Height Growth at Month 9
Height growth was defined as ratio of observed change from baseline in standing height (centimeter) to expected change from baseline (centimeter) in reference population. For C4181001 cohort, baseline value was defined as the value recorded at the date of informed consent, whereas for C4181005 cohort the baseline value was the value taken at Day 1 before dosing.
Measured: Baseline, Month 9 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]
Mean Height Growth at Month 12
Height growth was defined as ratio of observed change from baseline in standing height (centimeter) to expected change from baseline (centimeter) in reference population. For C4181001 cohort, baseline value was defined as the value recorded at the date of informed consent, whereas for C4181005 cohort the baseline value was the value taken at Day 1 before dosing.
Measured: Baseline, Month 12 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]
Secondary outcomes (5)
Mean Change From Baseline in Arm Span to Height/Length Difference at Months 3, 6, 9 and 12
Arm span to standing height or length difference was defined as absolute value (arm length [centimeter] - standing height [centimeter]). For C4181001 cohort, baseline value was defined as the value recorded at the date of informed consent, whereas for C4181005 cohort the baseline value was the value taken at Day 1 before dosing.
Measured: Baseline, Months 3, 6, 9, 12 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]
Mean Change From Baseline in Knee Height: Lower Segment Ratio at Months 3, 6, 9 and 12
Knee height: lower segment ratio was the ratio of knee to heel length to the difference between standing height and sitting height. For C4181001 cohort, baseline value was defined as the value recorded at the date of informed consent, whereas for C4181005 cohort the baseline value was the value taken at Day 1 before dosing.
Measured: Baseline, Months 3, 6, 9, 12 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]
Mean Change From Baseline in Height Standard Deviation Score (Z-Score) at Months 3, 6, 9, 12
Z-score was calculated as the difference between mean observed standing height at specified visit and mean value of reference population divided by standard deviation of reference population. Z score indicated how similar the participant was to the reference population. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean. For C4181001 cohort, baseline value was defined as the value recorded at the date of informed consent, whereas for C4181005 cohort the baseline value was the value taken at Day 1 before dosing.
Measured: Baseline, Months 3, 6, 9, 12 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]
Number of Participants With Achondroplasia Related Orthopedic Complications
Achondroplasia related orthopedic complications included lordosis, kyphosis, small foramen magnum: foramen magnum is slightly narrow and the spinal cord is slightly compressed at the level, foramen magnum stenosis, foramen magnum stenosis with associated c1-c2 secondary myelopathy, foramen magnum stenosis with mild posterior deformity of bulbospinal union, foramen magnum stenosis without spinal cord alteration, cervical myelomalacia; bulbar compression, marked stenosis of the foramen magnum with evident compression of bulb and bulb-medulla passage, mild foramen magnus stenosis, mild stenosis of foramen magnum, moderate to severe narrowing of the foramen magnum, severe stenosis at foramen magnum and stenosis foramen magnum.
Measured: C4181005 cohorts: 12 months; C4181001 cohort: 5 years [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]
Number of Participants With Other Achondroplasia Related Orthopedic Complications
Other achondroplasia related orthopedic complications included otitis media, otitis media acute, hydrocephalus and obstructive sleep apnea syndrome.
Measured: C4181005 cohorts: 12 months; C4181001 cohort: 5 years [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Dec 2022
- Study start19 Oct 2022
- Primary completion16 May 2023
- Study completion16 May 2023
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.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 248 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pfizer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Pfizer CT.gov Call Center, Study director, Pfizer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05659719
- Sponsor's protocol ID
- C4181010
- Status last verified
- May 2024
- First submitted
- 12 Oct 2022
- Last updated
- 20 Sep 2024
- Results posted
- Yes, on ClinicalTrials.gov
- 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.
Publications
Qiao W, Boucher M, Slade A, Dawra VK. Natural disease course modeling of achondroplasia to evaluate the efficacy of recifercept in the absence of a placebo control arm in phase II study. CPT Pharmacometrics Syst Pharmacol. 2024 Jul;13(7):1103-1116. doi: 10.1002/psp4.13143. Epub 2024 Apr 29.
PubMed 38685585
Study at a glance
From the registry record, last updated by the sponsor on 20 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Project to Evaluate the Feasibility of Constructing a Concurrent External Control for Recifercept
- ClinicalTrials.gov ID
- NCT05659719
- Acronym / sponsor ID
- C4181010
- Conditions
- Achondroplasia
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 248 participants (actual)
- Lead sponsor
- PfizerIndustry (pharma, biotech, devices)
- Study start
- 19 Oct 2022 (actual)
- Primary completion
- 16 May 2023 (actual)
- Study completion
- 16 May 2023 (actual)
- First posted
- 21 Dec 2022 (actual)
- Results
- Posted 20 Sep 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to learn about the study medicine (called recifercept) in people with achondroplasia. Achondroplasia is a very rare disease and patients of achondroplasia have short arms and legs. The study will include data already collected from a recifercept clinical trial and data collected from a separate study of achondroplasia. This study will compare patient experiences and will help the investigators determine if the study medicine, recifercept, is effective.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Recifercept cohort | – | Other: ReciferceptAchondroplasia patients enrolled in the recifercept phase 2 clinical trial |
| Natural history cohort | – | –Achondroplasia patients enrolled in the achondroplasia natural history study |
Who may qualify
- Ages
- 3 months to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Achondroplasia patients, aged 3 months to 11 years old with documented and confirmed genetic diagnosis of achondroplasia, who have completed at least 2 valid height and length measurement in the natural history study and have at least 6 months of follow-up data in either the natural history study or the phase 2 recifercept trial.
Key inclusion criteria (8 listed)
- All patients from Study C4181005 who have completed Visits 1 through 11 (at D183) will be included in this project.
- To construct a concurrent external control, patients from Study C4181001 will need to meet the following inclusion criteria from Study C4181005 to be eligible for inclusion:
- Documented, confirmed genetic diagnosis of achondroplasia from historical medical records (test must have been performed at a laboratory fully accredited for genetic testing under local regulations)
- Aged ≥ 3 months to <11 years (up to the day before 11th birthday inclusive) at time of enrollment into the observational natural history study.
- Havecompleted at least 2 valid height/length measurements (at least 3 months apart)
Key exclusion criteria (8 listed)
- Patients meeting any of the following criteria will not be included in the study:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ReciferceptPatients received recifercept intervention in the phase 2 clinical trial | Other | Recifercept cohort |
Primary outcome
Mean Height Growth at Month 3
Time frame: Baseline, Month 3 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]
Height growth was defined as ratio of observed change from baseline in standing height (centimeter) to expected change from baseline (centimeter) in reference population. For C4181001 cohort, baseline value was defined as the value recorded at the date of informed consent, whereas for C4181005 cohort the baseline value was the value taken at Day 1 before dosing.
Mean Height Growth at Month 6
Time frame: Baseline, Month 6 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]
Height growth was defined as ratio of observed change from baseline in standing height (centimeter) to expected change from baseline (centimeter) in reference population. For C4181001 cohort, baseline value was defined as the value recorded at the date of informed consent, whereas for C4181005 cohort the baseline value was the value taken at Day 1 before dosing.
Mean Height Growth at Month 9
Time frame: Baseline, Month 9 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]
Height growth was defined as ratio of observed change from baseline in standing height (centimeter) to expected change from baseline (centimeter) in reference population. For C4181001 cohort, baseline value was defined as the value recorded at the date of informed consent, whereas for C4181005 cohort the baseline value was the value taken at Day 1 before dosing.
Mean Height Growth at Month 12
Time frame: Baseline, Month 12 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]
Height growth was defined as ratio of observed change from baseline in standing height (centimeter) to expected change from baseline (centimeter) in reference population. For C4181001 cohort, baseline value was defined as the value recorded at the date of informed consent, whereas for C4181005 cohort the baseline value was the value taken at Day 1 before dosing.
5 secondary outcomes
Mean Change From Baseline in Arm Span to Height/Length Difference at Months 3, 6, 9 and 12
Time frame: Baseline, Months 3, 6, 9, 12 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]
Mean Change From Baseline in Knee Height: Lower Segment Ratio at Months 3, 6, 9 and 12
Time frame: Baseline, Months 3, 6, 9, 12 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]
Mean Change From Baseline in Height Standard Deviation Score (Z-Score) at Months 3, 6, 9, 12
Time frame: Baseline, Months 3, 6, 9, 12 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]
Number of Participants With Achondroplasia Related Orthopedic Complications
Time frame: C4181005 cohorts: 12 months; C4181001 cohort: 5 years [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]
Number of Participants With Other Achondroplasia Related Orthopedic Complications
Time frame: C4181005 cohorts: 12 months; C4181001 cohort: 5 years [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Sep 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 38685585Qiao W, Boucher M, Slade A, Dawra VK. Natural disease course modeling of achondroplasia to evaluate the efficacy of recifercept in the absence of a placebo control arm in phase II study. CPT Pharmacometrics Syst Pharmacol. 2024 Jul;13(7):1103-1116. doi: 10.1002/psp4.13143. Epub 2024 Apr 29.
- 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.
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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.
Pfizer CT.gov Call Center study director
Pfizer
1 site in 1 country: 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 1 site in United States.
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 8 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: finished, as of the sponsor's last update on 20 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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 20 Sep 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?
Mean Height Growth at Month 3 (time frame: Baseline, Month 3 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05659719, which also lists 1 linked publication.
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 20 Sep 2024; AzTrials last refreshed its copy on 6 Nov 2026.