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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Recifercept cohort | Achondroplasia patients enrolled in the recifercept phase 2 clinical trial |
| |
| Natural history cohort | Achondroplasia patients enrolled in the achondroplasia natural history study |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Recifercept | Other | Patients received recifercept intervention in the phase 2 clinical trial |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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. | 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. | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Inclusion Criteria:
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:
Exclusion Criteria:
Patients meeting any of the following criteria will not be included in the study:
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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.
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| Name | Affiliation | Role |
|---|---|---|
| Pfizer CT.gov Call Center | Pfizer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Pfizer | New York | New York | 10017 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 38685585 | Derived | 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. |
| Label | URL |
|---|---|
| To obtain contact information for a study center near you, click here. | View source |
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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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Data from eligible participants from C4181005 (NCT04638153) study and from C4181001 (NCT03794609) study were extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months).
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| ID | Title | Description |
|---|---|---|
| FG000 | C4181005 Cohort: Recifercept 1 mg/kg QW | Eligible participants who received recifercept 1 milligram per kilogram (mg/kg) once weekly (QW) subcutaneously for 12 months in study C4181005 were included in this arm. Data from these participants were observed in this retrospective observational study. |
| FG001 | C4181005 Cohort: Recifercept 2 mg/kg BIW | Eligible participants who received recifercept 2 mg/kg twice weekly (BIW) subcutaneously for 12 months in study C4181005 were included in this arm. Data from these participants were observed in this retrospective observational study. |
| FG002 | C4181005 Cohort: Recifercept 1.5 mg/kg QD | Eligible participants who received recifercept 1.5 mg/kg once daily (QD) subcutaneously for 12 months in study C4181005 were included in this arm. Data from these participants were observed in this retrospective observational study. |
| FG003 | C4181001 Cohort | Eligible participants from study C4181001 who met the eligibility criteria for study C4181005 and had at least 6 months and up to 5 years follow-up data were included in this arm. Data from these participants were observed in this retrospective observational study. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
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Full Analysis set (FAS) included all enrolled participants who met the inclusion and exclusion criteria for the study and whose data was retrieved and observed in this study.
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| ID | Title | Description |
|---|---|---|
| BG000 | C4181005 Cohort: Recifercept 1 mg/kg QW | Eligible participants who received recifercept 1 mg/kg QW subcutaneously for 12 months in study C4181005 were included in this arm. Data from these participants were observed in this retrospective observational study. |
| BG001 | C4181005 Cohort: Recifercept 2 mg/kg BIW |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | FAS included all enrolled participants who met the inclusion and exclusion criteria for the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | Ratio | Baseline, Month 3 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)] |
|
Not applicable as adverse events were not planned to be collected during this study.
This observational retrospective study involved data that existed as structured data by the time of study start. In these data sources, individual participants data were not retrieved or validated. The minimum criteria for reporting an adverse event (AE) (i.e., identifiable participants, identifiable reporter, a suspect product, and event) was not met. Hence, AEs were not planned to be collected during the study and not reported.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | C4181005 Cohort: Recifercept 1 mg/kg QW | Eligible participants who received recifercept 1 mg/kg QW subcutaneously for 12 months in study C4181005 were included in this arm. Data from these participants were observed in this retrospective observational study. |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Pfizer ClinicalTrials.gov Call Center | Pfizer Inc. | 1-800-718-1021 | ClinicalTrials.gov_Inquiries@pfizer.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Nov 9, 2023 | May 10, 2024 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 15, 2024 | May 10, 2024 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D000130 | Achondroplasia |
| ID | Term |
|---|---|
| D004392 | Dwarfism |
| D001848 | Bone Diseases, Developmental |
| D001847 | Bone Diseases |
| D009140 | Musculoskeletal Diseases |
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| Baseline, Month 12 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)] |
| 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. | 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. | 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. | 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. | 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)] |
| Increased age moving to different baseline study with prospects of moving to intervention in future |
|
| Inclusion/Exclusion criteria |
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| Wish of the child, or of the child's parent(S)/legal guardian(S), to withdrew from the study |
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| Lost to Follow-up |
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| Moved to another observational study |
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| The child was enrolled in an interventional study |
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| The child went to lower limbs elongation surgery in few weeks' time |
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| The family wished to pursue a prescription a voxzogo instead of waiting for phase 3 |
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Eligible participants who received recifercept 2 mg/kg BIW subcutaneously for 12 months in study C4181005 were included in this arm. Data from these participants were observed in this retrospective observational study. |
| BG002 | C4181005 Cohort: Recifercept 1.5 mg/kg QD | Eligible participants who received recifercept 1.5 mg/kg QD subcutaneously for 12 months in study C4181005 were included in this arm. Data from these participants were observed in this retrospective observational study. |
| BG003 | C4181001 Cohort | Eligible participants from study C4181001 who met the eligibility criteria for study C4181005 and had at least 6 months and up to 5 years follow-up data were included in this arm. Data from these participants were observed in this retrospective observational study. |
| BG004 | Total | Total of all reporting groups |
| Years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| OG001 | C4181005 Cohort: Recifercept 2 mg/kg BIW | Eligible participants who received recifercept 2 mg/kg BIW subcutaneously for 12 months in study C4181005 were included in this arm. Data from these participants were observed in this retrospective observational study. |
| OG002 | C4181005 Cohort: Recifercept 1.5 mg/kg QD | Eligible participants who received recifercept 1.5 mg/kg QD subcutaneously for 12 months in study C4181005 were included in this arm. Data from these participants were observed in this retrospective observational study. |
| OG003 | C4181001 Cohort | Eligible participants from study C4181001 who met the eligibility criteria for study C4181005 and had at least 6 months and up to 5 years follow-up data were included in this arm. Data from these participants were observed in this retrospective observational study. |
|
|
| Primary | 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. | FAS included all enrolled participants who met the inclusion and exclusion criteria for the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | Ratio | Baseline, Month 6 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)] |
|
|
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| Primary | 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. | FAS included all enrolled participants who met the inclusion and exclusion criteria for the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | Ratio | Baseline, Month 9 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)] |
|
|
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| Primary | 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. | FAS included all enrolled participants who met the inclusion and exclusion criteria for the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | Ratio | Baseline, Month 12 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)] |
|
|
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| Secondary | 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. | FAS included all enrolled participants who met the inclusion and exclusion criteria for the study. Here, "Number Analyzed" signifies participants evaluable for specific timepoints. | Posted | Mean | Standard Deviation | Centimeter | 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)] |
|
|
|
| Secondary | 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. | FAS included all enrolled participants who met the inclusion and exclusion criteria for the study. Here, "Number Analyzed" signifies participants evaluable for specific timepoints. | Posted | Mean | Standard Deviation | Ratio | 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)] |
|
|
|
| Secondary | 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. | FAS included all enrolled participants who met the inclusion and exclusion criteria for the study. Here, "Number Analyzed" signifies participants evaluable for specific timepoints. | Posted | Mean | Standard Deviation | Z-score | 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)] |
|
|
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| Secondary | 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. | FAS included all enrolled participants who met the inclusion and exclusion criteria for the study. | Posted | Count of Participants | Participants | 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)] |
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|
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| Secondary | 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. | FAS included all enrolled participants who met the inclusion and exclusion criteria for the study. | Posted | Count of Participants | Participants | 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)] |
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| 0 |
| 0 |
| 0 |
| 0 |
| 0 |
| 0 |
| EG001 | C4181005 Cohort: Recifercept 2 mg/kg BIW | Eligible participants who received recifercept 2 mg/kg BIW subcutaneously for 12 months in study C4181005 were included in this arm. Data from these participants were observed in this retrospective observational study. | 0 | 0 | 0 | 0 | 0 | 0 |
| EG002 | C4181005 Cohort: Recifercept 1.5 mg/kg QD | Eligible participants who received recifercept 1.5 mg/kg QD subcutaneously for 12 months in study C4181005 were included in this arm. Data from these participants were observed in this retrospective observational study. | 0 | 0 | 0 | 0 | 0 | 0 |
| EG003 | C4181001 Cohort | Eligible participants from study C4181001 who met the eligibility criteria for study C4181005 and had at least 6 months and up to 5 years follow-up data were included in this arm. Data from these participants were observed in this retrospective observational study. | 0 | 0 | 0 | 0 | 0 | 0 |
Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publication until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
| D010009 |
| Osteochondrodysplasias |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| Month 6 |
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| Month 9 |
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| Month 12 |
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| Month 6 |
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| Month 9 |
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| Month 12 |
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| Month 6 |
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| Month 9 |
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| Month 12 |
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| Kyphosis |
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| Foramen magnum is slightly narrow |
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| Foramen magnum stenosis |
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| Foramen magnum stenosis with associated c1-c2 secondary myelopathy |
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| Foramen magnum stenosis with mild posterior deformity of bulbospinal union |
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| Foramen magnum stenosis without spinal cord alteration |
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| Foramen magnum stenosis; cervical myelomalacia; bulbar compression |
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| Marked stenosis of the foramen magnum with evident compression of Bulb and bulb-medulla passage |
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| Mild foramen magnus stenosis |
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| Moderate to severe narrowing of the foramen magnum |
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| Severe stenosis at foramen magnum |
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| Stenosis foramen magnum |
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| Otitis media acute |
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| Hydrocephalus |
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| Obstructive sleep apnea syndrome |
|