NCT05659719

A Study to Learn About Recifercept in Patients With Achondroplasia

Completed Results posted Last updated 20 September 2024

What this trial tests

trial testing Recifercept in Achondroplasia in 248 participants. Completed in 16 May 2023.

Timeline

19 October 2022

Primary endpoint
16 May 2023

16 May 2023

Quick facts

Lead sponsor	Pfizer
Status	Completed
Study type	OBSERVATIONAL
Enrollment	248
Start date	19 October 2022
Primary completion	16 May 2023
Estimated completion	16 May 2023
Sites	1 location across United States

Drugs / interventions tested

• Recifercept — full drug profile →

Conditions studied

• Achondroplasia — all drugs for Achondroplasia →

Sponsor

Pfizer — full company profile →

Who can join

Adults 3 Months to 10, any sex, with Achondroplasia. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Mean Height Growth at Month 3 Primary · 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.

Group	Value	95% CI
C4181005 Cohort: Recifercept 1 mg/kg QW	1.11	± 0.487
C4181005 Cohort: Recifercept 2 mg/kg BIW	0.84	± 0.548
C4181005 Cohort: Recifercept 1.5 mg/kg QD	0.75	± 0.707
C4181001 Cohort	1.07	± 0.820

Mean Height Growth at Month 6 Primary · 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.

Group	Value	95% CI
C4181005 Cohort: Recifercept 1 mg/kg QW	0.94	± 0.429
C4181005 Cohort: Recifercept 2 mg/kg BIW	1.06	± 0.440
C4181005 Cohort: Recifercept 1.5 mg/kg QD	1.01	± 0.453
C4181001 Cohort	1.11	± 0.567

Mean Height Growth at Month 9 Primary · 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.

Group	Value	95% CI
C4181005 Cohort: Recifercept 1 mg/kg QW	1.01	± 0.374
C4181005 Cohort: Recifercept 2 mg/kg BIW	0.97	± 0.242
C4181005 Cohort: Recifercept 1.5 mg/kg QD	1.03	± 0.285
C4181001 Cohort	1.10	± 0.419

Mean Height Growth at Month 12 Primary · 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.

Group	Value	95% CI
C4181005 Cohort: Recifercept 1 mg/kg QW	0.94	± 0.298
C4181005 Cohort: Recifercept 2 mg/kg BIW	1.06	± 0.217
C4181005 Cohort: Recifercept 1.5 mg/kg QD	0.84	± 0.036
C4181001 Cohort	1.10	± 0.376

Mean Change From Baseline in Arm Span to Height/Length Difference at Months 3, 6, 9 and 12 Secondary · 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)]

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.

Month 3

Group	Value	95% CI
C4181005 Cohort: Recifercept 1 mg/kg QW	0.46	± 0.956
C4181005 Cohort: Recifercept 2 mg/kg BIW	0.02	± 0.879
C4181005 Cohort: Recifercept 1.5 mg/kg QD	-0.61	± 1.705
C4181001 Cohort	0.09	± 1.180

Month 6

Group	Value	95% CI
C4181005 Cohort: Recifercept 1 mg/kg QW	0.19	± 1.863
C4181005 Cohort: Recifercept 2 mg/kg BIW	0.72	± 1.028
C4181005 Cohort: Recifercept 1.5 mg/kg QD	-0.47	± 1.927
C4181001 Cohort	0.31	± 1.706

Month 9

Group	Value	95% CI
C4181005 Cohort: Recifercept 1 mg/kg QW	0.64	± 0.890
C4181005 Cohort: Recifercept 2 mg/kg BIW	0.69	± 1.394
C4181005 Cohort: Recifercept 1.5 mg/kg QD	0.15	± 0.781
C4181001 Cohort	0.53	± 1.705

Month 12

Group	Value	95% CI
C4181005 Cohort: Recifercept 1 mg/kg QW	0.47	± 1.220
C4181005 Cohort: Recifercept 2 mg/kg BIW	1.12	± 1.754
C4181005 Cohort: Recifercept 1.5 mg/kg QD	0.20	± 0.707
C4181001 Cohort	1.29	± 3.745

Mean Change From Baseline in Knee Height: Lower Segment Ratio at Months 3, 6, 9 and 12 Secondary · 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)]

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.

Month 3

Group	Value	95% CI
C4181005 Cohort: Recifercept 1 mg/kg QW	-0.00	± 0.020
C4181005 Cohort: Recifercept 2 mg/kg BIW	-0.01	± 0.052
C4181005 Cohort: Recifercept 1.5 mg/kg QD	0.02	± 0.037
C4181001 Cohort	-0.09	± 0.864

Month 6

Group	Value	95% CI
C4181005 Cohort: Recifercept 1 mg/kg QW	-0.00	± 0.028
C4181005 Cohort: Recifercept 2 mg/kg BIW	-0.02	± 0.062
C4181005 Cohort: Recifercept 1.5 mg/kg QD	0.00	± 0.026
C4181001 Cohort	-0.01	± 0.048

Month 9

Group	Value	95% CI
C4181005 Cohort: Recifercept 1 mg/kg QW	-0.00	± 0.027
C4181005 Cohort: Recifercept 2 mg/kg BIW	-0.02	± 0.065
C4181005 Cohort: Recifercept 1.5 mg/kg QD	0.01	± 0.026
C4181001 Cohort	-0.01	± 0.050

Month 12

Group	Value	95% CI
C4181005 Cohort: Recifercept 1 mg/kg QW	0.00	± 0.028
C4181005 Cohort: Recifercept 2 mg/kg BIW	-0.02	± 0.081
C4181005 Cohort: Recifercept 1.5 mg/kg QD	0.02	± 0.023
C4181001 Cohort	-0.01	± 0.049

Mean Change From Baseline in Height Standard Deviation Score (Z-Score) at Months 3, 6, 9, 12 Secondary · 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)]

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 va

Month 3

Group	Value	95% CI
C4181005 Cohort: Recifercept 1 mg/kg QW	0.03	± 0.134
C4181005 Cohort: Recifercept 2 mg/kg BIW	-0.04	± 0.173
C4181005 Cohort: Recifercept 1.5 mg/kg QD	-0.02	± 0.266
C4181001 Cohort	0.03	± 0.289

Month 6

Group	Value	95% CI
C4181005 Cohort: Recifercept 1 mg/kg QW	-0.03	± 0.205
C4181005 Cohort: Recifercept 2 mg/kg BIW	0.03	± 0.211
C4181005 Cohort: Recifercept 1.5 mg/kg QD	-0.00	± 0.236
C4181001 Cohort	0.06	± 0.355

Month 9

Group	Value	95% CI
C4181005 Cohort: Recifercept 1 mg/kg QW	0.00	± 0.258
C4181005 Cohort: Recifercept 2 mg/kg BIW	-0.02	± 0.214
C4181005 Cohort: Recifercept 1.5 mg/kg QD	0.02	± 0.205
C4181001 Cohort	0.07	± 0.412

Month 12

Group	Value	95% CI
C4181005 Cohort: Recifercept 1 mg/kg QW	-0.03	± 0.327
C4181005 Cohort: Recifercept 2 mg/kg BIW	0.07	± 0.257
C4181005 Cohort: Recifercept 1.5 mg/kg QD	-0.24	± 0.169
C4181001 Cohort	0.12	± 0.439

Number of Participants With Achondroplasia Related Orthopedic Complications Secondary · 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)]

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 o

Lordosis

Group	Value	95% CI
C4181005 Cohort: Recifercept 1 mg/kg QW	0
C4181005 Cohort: Recifercept 2 mg/kg BIW	0
C4181005 Cohort: Recifercept 1.5 mg/kg QD	0
C4181001 Cohort	79

Kyphosis

Group	Value	95% CI
C4181005 Cohort: Recifercept 1 mg/kg QW	0
C4181005 Cohort: Recifercept 2 mg/kg BIW	0
C4181005 Cohort: Recifercept 1.5 mg/kg QD	0
C4181001 Cohort	1

Foramen magnum is slightly narrow

Group	Value	95% CI
C4181005 Cohort: Recifercept 1 mg/kg QW	0
C4181005 Cohort: Recifercept 2 mg/kg BIW	0
C4181005 Cohort: Recifercept 1.5 mg/kg QD	0
C4181001 Cohort	1

Foramen magnum stenosis

Group	Value	95% CI
C4181005 Cohort: Recifercept 1 mg/kg QW	0
C4181005 Cohort: Recifercept 2 mg/kg BIW	0
C4181005 Cohort: Recifercept 1.5 mg/kg QD	0
C4181001 Cohort	3

Foramen magnum stenosis with associated c1-c2 secondary myelopathy

Group	Value	95% CI
C4181005 Cohort: Recifercept 1 mg/kg QW	0
C4181005 Cohort: Recifercept 2 mg/kg BIW	0
C4181005 Cohort: Recifercept 1.5 mg/kg QD	0
C4181001 Cohort	1

Foramen magnum stenosis with mild posterior deformity of bulbospinal union

Group	Value	95% CI
C4181005 Cohort: Recifercept 1 mg/kg QW	0
C4181005 Cohort: Recifercept 2 mg/kg BIW	0
C4181005 Cohort: Recifercept 1.5 mg/kg QD	0
C4181001 Cohort	1

Foramen magnum stenosis without spinal cord alteration

Group	Value	95% CI
C4181005 Cohort: Recifercept 1 mg/kg QW	0
C4181005 Cohort: Recifercept 2 mg/kg BIW	0
C4181005 Cohort: Recifercept 1.5 mg/kg QD	0
C4181001 Cohort	1

Foramen magnum stenosis; cervical myelomalacia; bulbar compression

Group	Value	95% CI
C4181005 Cohort: Recifercept 1 mg/kg QW	0
C4181005 Cohort: Recifercept 2 mg/kg BIW	0
C4181005 Cohort: Recifercept 1.5 mg/kg QD	0
C4181001 Cohort	1

Number of Participants With Other Achondroplasia Related Orthopedic Complications Secondary · 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)]

Other achondroplasia related orthopedic complications included otitis media, otitis media acute, hydrocephalus and obstructive sleep apnea syndrome.

Otitis media

Group	Value	95% CI
C4181005 Cohort: Recifercept 1 mg/kg QW	3
C4181005 Cohort: Recifercept 2 mg/kg BIW	1
C4181005 Cohort: Recifercept 1.5 mg/kg QD	1
C4181001 Cohort	0

Otitis media acute

Group	Value	95% CI
C4181005 Cohort: Recifercept 1 mg/kg QW	0
C4181005 Cohort: Recifercept 2 mg/kg BIW	1
C4181005 Cohort: Recifercept 1.5 mg/kg QD	0
C4181001 Cohort	0

Hydrocephalus

Group	Value	95% CI
C4181005 Cohort: Recifercept 1 mg/kg QW	0
C4181005 Cohort: Recifercept 2 mg/kg BIW	0
C4181005 Cohort: Recifercept 1.5 mg/kg QD	0
C4181001 Cohort	2

Obstructive sleep apnea syndrome

Group	Value	95% CI
C4181005 Cohort: Recifercept 1 mg/kg QW	0
C4181005 Cohort: Recifercept 2 mg/kg BIW	1
C4181005 Cohort: Recifercept 1.5 mg/kg QD	0
C4181001 Cohort	0

Sponsor's own description

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.

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

1. Natural disease course modeling of achondroplasia to evaluate the efficacy of recifercept in the absence of a placebo control arm in phase II study.
   Qiao W, Boucher M, Slade A, Dawra VK. · · 2024 · cited 3× · PMID 38685585 · DOI 10.1002/psp4.13143