Trial Outcomes & Findings for A Study to Learn About Recifercept in Patients With Achondroplasia (NCT NCT05659719)
NCT ID: NCT05659719
Last Updated: 2024-09-20
Results Overview
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.
Recruitment status
COMPLETED
Target enrollment
248 participants
Primary outcome timeframe
Baseline, Month 3 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]
Results posted on
2024-09-20
Participant Flow
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).
Participant milestones
| Measure |
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.
|
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.
|
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.
|
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.
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
20
|
19
|
18
|
191
|
|
Overall Study
COMPLETED
|
20
|
19
|
18
|
2
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
189
|
Reasons for withdrawal
| Measure |
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.
|
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.
|
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.
|
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.
|
|---|---|---|---|---|
|
Overall Study
At sponsor's request
|
0
|
0
|
0
|
95
|
|
Overall Study
Increased age moving to different baseline study with prospects of moving to intervention in future
|
0
|
0
|
0
|
1
|
|
Overall Study
Inclusion/Exclusion criteria
|
0
|
0
|
0
|
5
|
|
Overall Study
Wish of the child, or of the child's parent(S)/legal guardian(S), to withdrew from the study
|
0
|
0
|
0
|
27
|
|
Overall Study
Lost to Follow-up
|
0
|
0
|
0
|
2
|
|
Overall Study
Moved to another observational study
|
0
|
0
|
0
|
1
|
|
Overall Study
The child was enrolled in an interventional study
|
0
|
0
|
0
|
55
|
|
Overall Study
The child went to lower limbs elongation surgery in few weeks' time
|
0
|
0
|
0
|
1
|
|
Overall Study
The family wished to pursue a prescription a voxzogo instead of waiting for phase 3
|
0
|
0
|
0
|
2
|
Baseline Characteristics
A Study to Learn About Recifercept in Patients With Achondroplasia
Baseline characteristics by cohort
| Measure |
C4181005 Cohort: Recifercept 1 mg/kg QW
n=20 Participants
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.
|
C4181005 Cohort: Recifercept 2 mg/kg BIW
n=19 Participants
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.
|
C4181005 Cohort: Recifercept 1.5 mg/kg QD
n=18 Participants
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.
|
C4181001 Cohort
n=191 Participants
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.
|
Total
n=248 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Continuous
|
5.75 Years
STANDARD_DEVIATION 2.84 • n=5 Participants
|
5.16 Years
STANDARD_DEVIATION 2.59 • n=7 Participants
|
5.83 Years
STANDARD_DEVIATION 2.31 • n=5 Participants
|
4.65 Years
STANDARD_DEVIATION 2.59 • n=4 Participants
|
4.86 Years
STANDARD_DEVIATION 2.61 • n=21 Participants
|
|
Sex: Female, Male
Female
|
9 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
79 Participants
n=4 Participants
|
103 Participants
n=21 Participants
|
|
Sex: Female, Male
Male
|
11 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
112 Participants
n=4 Participants
|
145 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
10 Participants
n=4 Participants
|
15 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
16 Participants
n=5 Participants
|
19 Participants
n=7 Participants
|
15 Participants
n=5 Participants
|
181 Participants
n=4 Participants
|
231 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
2 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
Race · White
|
15 Participants
n=5 Participants
|
19 Participants
n=7 Participants
|
14 Participants
n=5 Participants
|
143 Participants
n=4 Participants
|
191 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
Race · Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
2 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
Race · Asian
|
3 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
41 Participants
n=4 Participants
|
46 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
Race · Other
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
5 Participants
n=4 Participants
|
5 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
Race · Multiracial
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
2 Participants
n=21 Participants
|
|
Race/Ethnicity, Customized
Race · Not reported
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
2 Participants
n=21 Participants
|
PRIMARY outcome
Timeframe: Baseline, Month 3 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]Population: 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.
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.
Outcome measures
| Measure |
C4181005 Cohort: Recifercept 1 mg/kg QW
n=15 Participants
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.
|
C4181005 Cohort: Recifercept 2 mg/kg BIW
n=17 Participants
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.
|
C4181005 Cohort: Recifercept 1.5 mg/kg QD
n=15 Participants
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.
|
C4181001 Cohort
n=95 Participants
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.
|
|---|---|---|---|---|
|
Mean Height Growth at Month 3
|
1.11 Ratio
Standard Deviation 0.487
|
0.84 Ratio
Standard Deviation 0.548
|
0.75 Ratio
Standard Deviation 0.707
|
1.07 Ratio
Standard Deviation 0.820
|
PRIMARY outcome
Timeframe: Baseline, Month 6 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]Population: 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.
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.
Outcome measures
| Measure |
C4181005 Cohort: Recifercept 1 mg/kg QW
n=16 Participants
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.
|
C4181005 Cohort: Recifercept 2 mg/kg BIW
n=16 Participants
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.
|
C4181005 Cohort: Recifercept 1.5 mg/kg QD
n=15 Participants
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.
|
C4181001 Cohort
n=72 Participants
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.
|
|---|---|---|---|---|
|
Mean Height Growth at Month 6
|
0.94 Ratio
Standard Deviation 0.429
|
1.06 Ratio
Standard Deviation 0.440
|
1.01 Ratio
Standard Deviation 0.453
|
1.11 Ratio
Standard Deviation 0.567
|
PRIMARY outcome
Timeframe: Baseline, Month 9 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]Population: 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.
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.
Outcome measures
| Measure |
C4181005 Cohort: Recifercept 1 mg/kg QW
n=16 Participants
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.
|
C4181005 Cohort: Recifercept 2 mg/kg BIW
n=16 Participants
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.
|
C4181005 Cohort: Recifercept 1.5 mg/kg QD
n=11 Participants
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.
|
C4181001 Cohort
n=62 Participants
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.
|
|---|---|---|---|---|
|
Mean Height Growth at Month 9
|
1.01 Ratio
Standard Deviation 0.374
|
0.97 Ratio
Standard Deviation 0.242
|
1.03 Ratio
Standard Deviation 0.285
|
1.10 Ratio
Standard Deviation 0.419
|
PRIMARY outcome
Timeframe: Baseline, Month 12 [Eligible data extracted and observed in this retrospective cohort study from 19-October-2022 to 16-May-2023 (approximately 7 months)]Population: 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.
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.
Outcome measures
| Measure |
C4181005 Cohort: Recifercept 1 mg/kg QW
n=7 Participants
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.
|
C4181005 Cohort: Recifercept 2 mg/kg BIW
n=9 Participants
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.
|
C4181005 Cohort: Recifercept 1.5 mg/kg QD
n=2 Participants
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.
|
C4181001 Cohort
n=62 Participants
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.
|
|---|---|---|---|---|
|
Mean Height Growth at Month 12
|
0.94 Ratio
Standard Deviation 0.298
|
1.06 Ratio
Standard Deviation 0.217
|
0.84 Ratio
Standard Deviation 0.036
|
1.10 Ratio
Standard Deviation 0.376
|
SECONDARY outcome
Timeframe: 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)]Population: FAS included all enrolled participants who met the inclusion and exclusion criteria for the study. Here, "Number Analyzed" signifies participants evaluable for specific timepoints.
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.
Outcome measures
| Measure |
C4181005 Cohort: Recifercept 1 mg/kg QW
n=20 Participants
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.
|
C4181005 Cohort: Recifercept 2 mg/kg BIW
n=19 Participants
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.
|
C4181005 Cohort: Recifercept 1.5 mg/kg QD
n=18 Participants
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.
|
C4181001 Cohort
n=191 Participants
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.
|
|---|---|---|---|---|
|
Mean Change From Baseline in Arm Span to Height/Length Difference at Months 3, 6, 9 and 12
Month 3
|
0.46 Centimeter
Standard Deviation 0.956
|
0.02 Centimeter
Standard Deviation 0.879
|
-0.61 Centimeter
Standard Deviation 1.705
|
0.09 Centimeter
Standard Deviation 1.180
|
|
Mean Change From Baseline in Arm Span to Height/Length Difference at Months 3, 6, 9 and 12
Month 6
|
0.19 Centimeter
Standard Deviation 1.863
|
0.72 Centimeter
Standard Deviation 1.028
|
-0.47 Centimeter
Standard Deviation 1.927
|
0.31 Centimeter
Standard Deviation 1.706
|
|
Mean Change From Baseline in Arm Span to Height/Length Difference at Months 3, 6, 9 and 12
Month 9
|
0.64 Centimeter
Standard Deviation 0.890
|
0.69 Centimeter
Standard Deviation 1.394
|
0.15 Centimeter
Standard Deviation 0.781
|
0.53 Centimeter
Standard Deviation 1.705
|
|
Mean Change From Baseline in Arm Span to Height/Length Difference at Months 3, 6, 9 and 12
Month 12
|
0.47 Centimeter
Standard Deviation 1.220
|
1.12 Centimeter
Standard Deviation 1.754
|
0.20 Centimeter
Standard Deviation 0.707
|
1.29 Centimeter
Standard Deviation 3.745
|
SECONDARY outcome
Timeframe: 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)]Population: FAS included all enrolled participants who met the inclusion and exclusion criteria for the study. Here, "Number Analyzed" signifies participants evaluable for specific timepoints.
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.
Outcome measures
| Measure |
C4181005 Cohort: Recifercept 1 mg/kg QW
n=20 Participants
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.
|
C4181005 Cohort: Recifercept 2 mg/kg BIW
n=19 Participants
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.
|
C4181005 Cohort: Recifercept 1.5 mg/kg QD
n=18 Participants
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.
|
C4181001 Cohort
n=191 Participants
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.
|
|---|---|---|---|---|
|
Mean Change From Baseline in Knee Height: Lower Segment Ratio at Months 3, 6, 9 and 12
Month 3
|
-0.00 Ratio
Standard Deviation 0.020
|
-0.01 Ratio
Standard Deviation 0.052
|
0.02 Ratio
Standard Deviation 0.037
|
-0.09 Ratio
Standard Deviation 0.864
|
|
Mean Change From Baseline in Knee Height: Lower Segment Ratio at Months 3, 6, 9 and 12
Month 6
|
-0.00 Ratio
Standard Deviation 0.028
|
-0.02 Ratio
Standard Deviation 0.062
|
0.00 Ratio
Standard Deviation 0.026
|
-0.01 Ratio
Standard Deviation 0.048
|
|
Mean Change From Baseline in Knee Height: Lower Segment Ratio at Months 3, 6, 9 and 12
Month 9
|
-0.00 Ratio
Standard Deviation 0.027
|
-0.02 Ratio
Standard Deviation 0.065
|
0.01 Ratio
Standard Deviation 0.026
|
-0.01 Ratio
Standard Deviation 0.050
|
|
Mean Change From Baseline in Knee Height: Lower Segment Ratio at Months 3, 6, 9 and 12
Month 12
|
0.00 Ratio
Standard Deviation 0.028
|
-0.02 Ratio
Standard Deviation 0.081
|
0.02 Ratio
Standard Deviation 0.023
|
-0.01 Ratio
Standard Deviation 0.049
|
SECONDARY outcome
Timeframe: 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)]Population: FAS included all enrolled participants who met the inclusion and exclusion criteria for the study. Here, "Number Analyzed" signifies participants evaluable for specific timepoints.
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.
Outcome measures
| Measure |
C4181005 Cohort: Recifercept 1 mg/kg QW
n=20 Participants
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.
|
C4181005 Cohort: Recifercept 2 mg/kg BIW
n=19 Participants
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.
|
C4181005 Cohort: Recifercept 1.5 mg/kg QD
n=18 Participants
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.
|
C4181001 Cohort
n=191 Participants
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.
|
|---|---|---|---|---|
|
Mean Change From Baseline in Height Standard Deviation Score (Z-Score) at Months 3, 6, 9, 12
Month 12
|
-0.03 Z-score
Standard Deviation 0.327
|
0.07 Z-score
Standard Deviation 0.257
|
-0.24 Z-score
Standard Deviation 0.169
|
0.12 Z-score
Standard Deviation 0.439
|
|
Mean Change From Baseline in Height Standard Deviation Score (Z-Score) at Months 3, 6, 9, 12
Month 6
|
-0.03 Z-score
Standard Deviation 0.205
|
0.03 Z-score
Standard Deviation 0.211
|
-0.00 Z-score
Standard Deviation 0.236
|
0.06 Z-score
Standard Deviation 0.355
|
|
Mean Change From Baseline in Height Standard Deviation Score (Z-Score) at Months 3, 6, 9, 12
Month 3
|
0.03 Z-score
Standard Deviation 0.134
|
-0.04 Z-score
Standard Deviation 0.173
|
-0.02 Z-score
Standard Deviation 0.266
|
0.03 Z-score
Standard Deviation 0.289
|
|
Mean Change From Baseline in Height Standard Deviation Score (Z-Score) at Months 3, 6, 9, 12
Month 9
|
0.00 Z-score
Standard Deviation 0.258
|
-0.02 Z-score
Standard Deviation 0.214
|
0.02 Z-score
Standard Deviation 0.205
|
0.07 Z-score
Standard Deviation 0.412
|
SECONDARY outcome
Timeframe: 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)]Population: FAS included all enrolled participants who met the inclusion and exclusion criteria for the study.
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.
Outcome measures
| Measure |
C4181005 Cohort: Recifercept 1 mg/kg QW
n=20 Participants
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.
|
C4181005 Cohort: Recifercept 2 mg/kg BIW
n=19 Participants
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.
|
C4181005 Cohort: Recifercept 1.5 mg/kg QD
n=18 Participants
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.
|
C4181001 Cohort
n=191 Participants
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.
|
|---|---|---|---|---|
|
Number of Participants With Achondroplasia Related Orthopedic Complications
Severe stenosis at foramen magnum
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Achondroplasia Related Orthopedic Complications
Marked stenosis of the foramen magnum with evident compression of Bulb and bulb-medulla passage
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Achondroplasia Related Orthopedic Complications
Lordosis
|
0 Participants
|
0 Participants
|
0 Participants
|
79 Participants
|
|
Number of Participants With Achondroplasia Related Orthopedic Complications
Kyphosis
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Achondroplasia Related Orthopedic Complications
Foramen magnum is slightly narrow
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Achondroplasia Related Orthopedic Complications
Foramen magnum stenosis
|
0 Participants
|
0 Participants
|
0 Participants
|
3 Participants
|
|
Number of Participants With Achondroplasia Related Orthopedic Complications
Foramen magnum stenosis with associated c1-c2 secondary myelopathy
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Achondroplasia Related Orthopedic Complications
Foramen magnum stenosis with mild posterior deformity of bulbospinal union
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Achondroplasia Related Orthopedic Complications
Foramen magnum stenosis without spinal cord alteration
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Achondroplasia Related Orthopedic Complications
Foramen magnum stenosis; cervical myelomalacia; bulbar compression
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Achondroplasia Related Orthopedic Complications
Mild foramen magnus stenosis
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Achondroplasia Related Orthopedic Complications
Moderate to severe narrowing of the foramen magnum
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Achondroplasia Related Orthopedic Complications
Stenosis foramen magnum
|
0 Participants
|
0 Participants
|
0 Participants
|
3 Participants
|
SECONDARY outcome
Timeframe: 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)]Population: FAS included all enrolled participants who met the inclusion and exclusion criteria for the study.
Other achondroplasia related orthopedic complications included otitis media, otitis media acute, hydrocephalus and obstructive sleep apnea syndrome.
Outcome measures
| Measure |
C4181005 Cohort: Recifercept 1 mg/kg QW
n=20 Participants
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.
|
C4181005 Cohort: Recifercept 2 mg/kg BIW
n=19 Participants
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.
|
C4181005 Cohort: Recifercept 1.5 mg/kg QD
n=18 Participants
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.
|
C4181001 Cohort
n=191 Participants
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.
|
|---|---|---|---|---|
|
Number of Participants With Other Achondroplasia Related Orthopedic Complications
Obstructive sleep apnea syndrome
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Other Achondroplasia Related Orthopedic Complications
Otitis media
|
3 Participants
|
1 Participants
|
1 Participants
|
0 Participants
|
|
Number of Participants With Other Achondroplasia Related Orthopedic Complications
Otitis media acute
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Other Achondroplasia Related Orthopedic Complications
Hydrocephalus
|
0 Participants
|
0 Participants
|
0 Participants
|
2 Participants
|
Adverse Events
C4181005 Cohort: Recifercept 1 mg/kg QW
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
C4181005 Cohort: Recifercept 2 mg/kg BIW
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
C4181005 Cohort: Recifercept 1.5 mg/kg QD
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
C4181001 Cohort
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee 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.
- Publication restrictions are in place
Restriction type: OTHER