Trial · NCT05116046
Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia
NCT05116046, Phase 2, Terminated, Pfizer; results first posted 2024-02-08
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia. Trials, record NCT05116046. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05116046
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia
- NCT number
- NCT05116046
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 35 participants, actual
- Lead sponsor
- Pfizer
- Conditions
- Achondroplasia
- Interventions
- Recifercept
- First posted
- 2021-11-10
- Last updated
- 2024-02-08
- Other registries' IDs
- eudract:2021-003149-39
- Results first posted
- 2024-02-08
- Limitations and caveats
Data was not collected for efficacy endpoints as study was terminated early based on sponsor discretion due to lack of efficacy at any of the tested doses. The decision to terminate the study was not related to a safety concern.
Source ClinicalTrials.gov
Registered outcomes16
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Severe AEs | From first dose of study drug up to 28 days after last dose of study drug (maximum up to 11 months) | An AE was any untoward medical occurrence that did not necessarily have a causal relationship with study treatment. TEAE was an AE that occurred after initiation of study treatment that was not present at the time of treatment start or an AE that increased in severity after the initiation of medication, if the event was present at the time of treatment start emerges. SAE was an AE resulting in any of the following outcomes or considered medically significant: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly or birth defect. Severe AEs were AEs that were medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated disabling, limiting self-care activities of daily living. |
| Primary | Change From Baseline in Height at Month 24 | Baseline, Month 24 | Height was measured using anthropometric measurements. Anthropometric data was collected by appropriately trained individuals at the trial site and in accordance with the anthropometric measurement manual. |
| Secondary | Clearance (CL/F) of Recifercept | Predose on Day 91, 181, 271, 361 and 451. | Clearance of a drug was a measure of the rate at which a drug is metabolised or eliminated by normal biological processes. |
| Secondary | Change From Baseline in Sitting Height to Standing Height Ratio at Months 3, 6, 9 | Baseline and Months 3, 6, 9 | Sitting height to standing height ratio was calculated based upon the anthropometric measurements. Anthropometric data was collected by appropriately trained individuals at the trial site and in accordance with the anthropometric measurement manual. |
| Secondary | Change From Baseline in Arm Span to Height/Length Difference at Months 3, 6, 9 | Baseline and Months 3, 6, 9 | Height and length difference was calculated with anthropometric measurements. Anthropometric data was collected by appropriately trained individuals at the trial site and in accordance with the anthropometric measurement manual. |
| Secondary | Change From Baseline in Knee Height to Lower Segment Ratio at Months 3, 6, 9 | Baseline and Months 3, 6, 9 | Knee height was defined as the distance from the sole of the foot to the most anterior surface of the femoral condyles of the thigh (medial being more anterior), with the ankle and knee each flexed to a 90-degree angle. Lower segment of the leg included tibia and foot height |
| Secondary | Change From Baseline in Occipito-Frontal Circumference at Months 3, 6, 9 | Baseline and Months 3, 6, 9 | Occipito-frontal circumference was measured by anthropometric measurements. It was measured over the most prominent part on the back of the head (occiput) and just above the eyebrows (supraorbital ridges). |
| Secondary | Change From Baseline in Occipito-Frontal Distance to Occipito-mid-Face Measurements Ratio at Months 3, 6, 9 | Baseline, Months 3, 6, 9 | Occipito-frontal circumference was measured by anthropometric measurements. Anthropometric data was collected by appropriately trained individuals at the trial site and in accordance with the anthropometric measurement manual. |
| Secondary | Change From Baseline in Z-Score for Occipito-frontal Circumference, Arm Span, Sitting Height and Skull Morphology at Months 3, 6, 9 | Baseline, Months 3, 6, 9 | The Z-score described how many standard deviations a given measurement lies above or below a size or age-specific population mean. A Z-score above the population mean indicates a positive value, whereas a Z-score below the population mean indicates a negative value. The greater the deviation of the Z-score from zero (in a positive or negative direction), the greater the magnitude of deviation from the mean. |
| Secondary | Change From Baseline in Fixed Flexion Angles at Elbow at Months 3, 6, 9 | Baseline, Months 3, 6, 9 | Fixed Flexion Angles was measured by anthropometric measurements. Anthropometric data was collected by appropriately trained individuals at the trial site and in accordance with the anthropometric measurement manual. |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 registered outcomesOutcomes16
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Severe AEs | From first dose of study drug up to 28 days after last dose of study drug (maximum up to 11 months) | FAS consisted of all participants who received at least one dose of recifercept. Participants were analyzed according to the dose they actually received. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Change From Baseline in Height at Month 24 | Baseline, Month 24 | Data was not collected as study was terminated based on sponsor discretion due to lack of efficacy at any of the tested doses. The decision to terminate the study was not related to a safety concern. | ||
| SECONDARY | Clearance (CL/F) of Recifercept | Predose on Day 91, 181, 271, 361 and 451. | Data was not collected as study was terminated based on sponsor discretion due to lack of efficacy at any of the tested doses. The decision to terminate the study was not related to a safety concern. | ||
| SECONDARY | Change From Baseline in Sitting Height to Standing Height Ratio at Months 3, 6, 9 | Baseline and Months 3, 6, 9 | Data was not collected as study was terminated based on sponsor discretion due to lack of efficacy at any of the tested doses. The decision to terminate the study was not related to a safety concern. | ||
| SECONDARY | Change From Baseline in Arm Span to Height/Length Difference at Months 3, 6, 9 | Baseline and Months 3, 6, 9 | Data was not collected as study was terminated based on sponsor discretion due to lack of efficacy at any of the tested doses. The decision to terminate the study was not related to a safety concern. | ||
| SECONDARY | Change From Baseline in Knee Height to Lower Segment Ratio at Months 3, 6, 9 | Baseline and Months 3, 6, 9 | Data was not collected as study was terminated based on sponsor discretion due to lack of efficacy at any of the tested doses. The decision to terminate the study was not related to a safety concern. | ||
| SECONDARY | Change From Baseline in Occipito-Frontal Circumference at Months 3, 6, 9 | Baseline and Months 3, 6, 9 | Data was not collected as study was terminated based on sponsor discretion due to lack of efficacy at any of the tested doses. The decision to terminate the study was not related to a safety concern. | ||
| SECONDARY | Change From Baseline in Occipito-Frontal Distance to Occipito-mid-Face Measurements Ratio at Months 3, 6, 9 | Baseline, Months 3, 6, 9 | Data was not collected as study was terminated based on sponsor discretion due to lack of efficacy at any of the tested doses. The decision to terminate the study was not related to a safety concern. | ||
| SECONDARY | Change From Baseline in Z-Score for Occipito-frontal Circumference, Arm Span, Sitting Height and Skull Morphology at Months 3, 6, 9 | Baseline, Months 3, 6, 9 | Data was not collected as study was terminated based on sponsor discretion due to lack of efficacy at any of the tested doses. The decision to terminate the study was not related to a safety concern. | ||
| SECONDARY | Change From Baseline in Fixed Flexion Angles at Elbow at Months 3, 6, 9 | Baseline, Months 3, 6, 9 | Data was not collected as study was terminated based on sponsor discretion due to lack of efficacy at any of the tested doses. The decision to terminate the study was not related to a safety concern. |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 outcomesResults by group30
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Severe AEs | Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly | AEs | 9 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Severe AEs | Recifercept 2 mg/kg Twice Weekly | AEs | 11 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Severe AEs | Recifercept 1.5 mg/kg Once Daily | AEs | 0 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Severe AEs | Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly | SAEs | 0 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Severe AEs | Recifercept 2 mg/kg Twice Weekly | SAEs | 0 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Severe AEs | Recifercept 1.5 mg/kg Once Daily | SAEs | 0 | Participants | |
| Number of Participants With Clinically Meaningful Findings in Laboratory Test Parameters Through the Study | Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly | Hematology: Lymphocytes | 0 | Participants | |
| Number of Participants With Clinically Meaningful Findings in Laboratory Test Parameters Through the Study | Recifercept 2 mg/kg Twice Weekly | Hematology: Lymphocytes | 1 | Participants | |
| Number of Participants With Clinically Meaningful Findings in Laboratory Test Parameters Through the Study | Recifercept 1.5 mg/kg Once Daily | Hematology: Lymphocytes | 0 | Participants | |
| Number of Participants With Clinically Meaningful Findings in Laboratory Test Parameters Through the Study | Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly | Hematology: Neutrophils | 0 | Participants |
1 to 10 of 30Source ClinicalTrials.gov
See all 30 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly | 16 | |
| Overall Study | STARTED | Recifercept 2 mg/kg Twice Weekly | 17 | |
| Overall Study | STARTED | Recifercept 1.5 mg/kg Once Daily | 2 | |
| Overall Study | COMPLETED | Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly | 0 | |
| Overall Study | COMPLETED | Recifercept 2 mg/kg Twice Weekly | 0 | |
| Overall Study | COMPLETED | Recifercept 1.5 mg/kg Once Daily | 0 | |
| Overall Study | NOT COMPLETED | Recifercept 1 Milligram Per Kilogram (mg/kg) Once Weekly | 16 | |
| Overall Study | NOT COMPLETED | Recifercept 2 mg/kg Twice Weekly | 17 | |
| Overall Study | NOT COMPLETED | Recifercept 1.5 mg/kg Once Daily | 2 |
Source ClinicalTrials.gov
Adverse events123
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Lymphopenia | Blood and lymphatic system disorders | Recifercept 1 mg/kg Once Weekly | 0 | 16 | |
| other | Lymphopenia | Blood and lymphatic system disorders | Recifercept 2 mg/kg Twice Weekly | 1 | 17 | |
| other | Lymphopenia | Blood and lymphatic system disorders | Recifercept 1.5 mg/kg Once Daily | 0 | 2 | |
| other | Ear pain | Ear and labyrinth disorders | Recifercept 1 mg/kg Once Weekly | 0 | 16 | |
| other | Ear pain | Ear and labyrinth disorders | Recifercept 2 mg/kg Twice Weekly | 1 | 17 | |
| other | Ear pain | Ear and labyrinth disorders | Recifercept 1.5 mg/kg Once Daily | 0 | 2 | |
| other | Eustachian tube dysfunction | Ear and labyrinth disorders | Recifercept 1 mg/kg Once Weekly | 0 | 16 | |
| other | Eustachian tube dysfunction | Ear and labyrinth disorders | Recifercept 2 mg/kg Twice Weekly | 1 | 17 | |
| other | Eustachian tube dysfunction | Ear and labyrinth disorders | Recifercept 1.5 mg/kg Once Daily | 0 | 2 | |
| other | Tympanic membrane perforation | Ear and labyrinth disorders | Recifercept 1 mg/kg Once Weekly | 0 | 16 |
1 to 10 of 123Source ClinicalTrials.gov
See all 123 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Recifercept 1 mg/kg Once Weekly | 0 | 16 |
| Total, all-cause mortality | Recifercept 2 mg/kg Twice Weekly | 0 | 17 |
| Total, all-cause mortality | Recifercept 1.5 mg/kg Once Daily | 0 | 2 |
| Total, other adverse events | Recifercept 1 mg/kg Once Weekly | 9 | 16 |
| Total, other adverse events | Recifercept 2 mg/kg Twice Weekly | 11 | 17 |
| Total, other adverse events | Recifercept 1.5 mg/kg Once Daily | 0 | 2 |
| Total, serious adverse events | Recifercept 1 mg/kg Once Weekly | 0 | 16 |
| Total, serious adverse events | Recifercept 2 mg/kg Twice Weekly | 0 | 17 |
| Total, serious adverse events | Recifercept 1.5 mg/kg Once Daily | 0 | 2 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT05116046
GET https://conductscience.org/api/trials/v1/NCT05116046/results?page=2