Trial · NCT04638153
A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia
NCT04638153, Phase 2, Terminated, Pfizer; results first posted 2024-02-15
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia. Trials, record NCT04638153. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04638153
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia
- NCT number
- NCT04638153
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 60 participants, actual
- Lead sponsor
- Pfizer
- Conditions
- Achondroplasia
- Interventions
- Recifercept
- First posted
- 2020-11-20
- Last updated
- 2024-02-15
- Other registries' IDs
- eudract:2020-001189-13
- Results first posted
- 2024-02-15
- Limitations and caveats
- The PK study cohort was not enrolled due to early study termination, therefore, the data of the PK cohort were not collected.
Source ClinicalTrials.gov
Registered outcomes23
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Treatment Emergent Treatment-Related Adverse Events (AEs) | The first dose up to 28 to 35 days after the last dose of study intervention (13 months) | Treatment-related AE was any untoward medical occurrence attributed to study intervention in a participant who received study intervention. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent AE is defined as an AE with onset date occurring during the on-treatment period. Relatedness to recifercept was assessed by the investigator (Yes/No). |
| Primary | Least Square Mean of Change From Baseline Height Growth at Month 3, Month 6, Month 9, and Month 12 | Baseline, Month 3, Month 6, Month 9, and Month 12 | Height growth was defined as the ratio of observed change from baseline in standing height to the expected change from baseline in the reference population. |
| Secondary | Change From Baseline in Pulse Rate at Month 3, Month 6, Month 9, and Month 12 | Baseline, Month 3, Month 6, Month 9, and Month 12 | Pulse rate measurements were preceded by at least 5 minutes of rest for the participant in a quiet setting without distractions (eg, television, cell phones) where possible (with consideration of the age of the child). Pulse rate was summarized by treatment in accordance with the sponsor reporting standards. |
| Secondary | Change From Baseline in Respiratory Rate at Month 3, Month 6, Month 9, and Month 12 | Baseline, Month 3, Month 6, Month 9, and Month 12 | Respiratory rate was obtained with participant in the seated position, after having sat calmly for at least 5 minutes. Respiratory rate was summarized by treatment in accordance with the sponsor reporting standards. |
| Secondary | Change From Baseline in Blood Pressure at Month 3, Month 6, Month 9, and Month 12 | Baseline, Month 3, Month 6, Month 9, and Month 12 | Blood pressure measurements were preceded by at least 5 minutes of rest for the participant in a quiet setting without distractions (eg, television, cell phones) where possible (with consideration of the age of the child). Supine systolic blood pressure (SBP) and diastolic blood pressure (DBP) were summarized by treatment in accordance with the sponsor reporting standards. |
| Secondary | Change From Baseline in Temperature at Month 3, Month 6, Month 9, and Month 12 | Baseline, Month 3, Month 6, Month 9, and Month 12 | Temperature was obtained with participant in the seated position, after having sat calmly for at least 5 minutes. Temperature measurements were summarized by treatment in accordance with the sponsor reporting standards. |
| Secondary | Number of Participants With Abnormal Physical Examination Findings at Month 3, Month 6, Month 9, and Month 12 | Month 3, Month 6, Month 9, and Month 12 | A physical examination included, at a minimum, assessments of the cardiovascular, respiratory, gastrointestinal systems and skin. Physical examination assessments were summarized by treatment in accordance with the sponsor reporting standards. |
| Secondary | Number of Participants With Laboratory Abnormalities (Without Regard to Baseline Abnormality) | Baseline to Month 12 | Participants with laboratory abnormalities that met pre-specified criteria included following parameters: hematology (corpuscular volume, corpuscular hemoglobin, corpuscular hemoglobin concentration, platelet, leukocytes, lymphocytes, neutrophils, eosinophils, and monocytes), and chemistry (bilirubin, alkaline phosphatase, albumin, urea nitrogen, urate, potassium, phosphate, bicarbonate). |
| Secondary | Pre-Dose Serum Concentration (Ctrough) of Recifercept | Pre-dose on Day(s) 4, 8, 15, 29, 61, 91, 183, 273, 365 | Ctrough was defined as pre-dose serum concentration during dosing and observed directly from data. |
| Secondary | Number of Participants With Positive Anti-Drug Antibodies (ADA) and Neutralizing Antibody (NAb) of Recifercept | The first dose up to 28 to 35 days after the last dose of study intervention (13 months) | The immunogenicity was measured by presence of ADA and NAb in participants treated with recifercept and summarized by dose regimen. |
1 to 10 of 23Source ClinicalTrials.gov
See all 23 registered outcomesOutcomes23
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 Treatment-Related Adverse Events (AEs) | The first dose up to 28 to 35 days after the last dose of study intervention (13 months) | Analysis population included all participants who received at least 1 dose of recifercept. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Least Square Mean of Change From Baseline Height Growth at Month 3, Month 6, Month 9, and Month 12 | Baseline, Month 3, Month 6, Month 9, and Month 12 | Analysis population included all participants who received at least 1 dose of recifercept. | Ratio | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in Pulse Rate at Month 3, Month 6, Month 9, and Month 12 | Baseline, Month 3, Month 6, Month 9, and Month 12 | Analysis population included all participants who received at least 1 dose of recifercept. The Number Analyzed in a row was based on actual analyzed participants at each visit. | beats per minute (BPM) | MEAN |
| SECONDARY | Change From Baseline in Respiratory Rate at Month 3, Month 6, Month 9, and Month 12 | Baseline, Month 3, Month 6, Month 9, and Month 12 | Analysis population included all participants who received at least 1 dose of recifercept. The Number Analyzed in a row was based on actual analyzed participants at each visit. | breaths/min | MEAN |
| SECONDARY | Change From Baseline in Blood Pressure at Month 3, Month 6, Month 9, and Month 12 | Baseline, Month 3, Month 6, Month 9, and Month 12 | Analysis population included all participants who received at least 1 dose of recifercept. The Number Analyzed in a row was based on actual analyzed participants at each visit. | millimeters of mercury (mm Hg) | MEAN |
| SECONDARY | Change From Baseline in Temperature at Month 3, Month 6, Month 9, and Month 12 | Baseline, Month 3, Month 6, Month 9, and Month 12 | Analysis population included all participants who received at least 1 dose of recifercept. The Number Analyzed in a row was based on actual analyzed participants at each visit. | Degree Celsius (°C) | MEAN |
| SECONDARY | Number of Participants With Abnormal Physical Examination Findings at Month 3, Month 6, Month 9, and Month 12 | Month 3, Month 6, Month 9, and Month 12 | Analysis population included all participants who received at least 1 dose of recifercept. The Number Analyzed in a row was based on actual analyzed participants at each visit. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Laboratory Abnormalities (Without Regard to Baseline Abnormality) | Baseline to Month 12 | Analysis population included all participants who received at least 1 dose of recifercept. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Pre-Dose Serum Concentration (Ctrough) of Recifercept | Pre-dose on Day(s) 4, 8, 15, 29, 61, 91, 183, 273, 365 | Analysis population included all participants who received at least 1 dose of recifercept and had at least one evaluable concentration. The Number Analyzed in a row was based on actual analyzed participants at each visit. | nanograms per millilitre (ng/mL) | MEAN |
| SECONDARY | Number of Participants With Positive Anti-Drug Antibodies (ADA) and Neutralizing Antibody (NAb) of Recifercept | The first dose up to 28 to 35 days after the last dose of study intervention (13 months) | Analysis population included all participants who received at least 1 dose of recifercept. | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 23Source ClinicalTrials.gov
See all 23 outcomesResults by group387
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 Treatment-Related Adverse Events (AEs) | Recifercept 1 mg/kg QW | Treatment-related AE | 7 | Participants | |
| Number of Participants With Treatment Emergent Treatment-Related Adverse Events (AEs) | Recifercept 2 mg/kg BIW | Treatment-related AE | 15 | Participants | |
| Number of Participants With Treatment Emergent Treatment-Related Adverse Events (AEs) | Recifercept 1.5 mg/kg QD | Treatment-related AE | 14 | Participants | |
| Number of Participants With Treatment Emergent Treatment-Related Adverse Events (AEs) | Recifercept 1 mg/kg QW | Treatment-related SAE | 0 | Participants | |
| Number of Participants With Treatment Emergent Treatment-Related Adverse Events (AEs) | Recifercept 2 mg/kg BIW | Treatment-related SAE | 0 | Participants | |
| Number of Participants With Treatment Emergent Treatment-Related Adverse Events (AEs) | Recifercept 1.5 mg/kg QD | Treatment-related SAE | 0 | Participants | |
| Least Square Mean of Change From Baseline Height Growth at Month 3, Month 6, Month 9, and Month 12 | Recifercept 1 mg/kg QW | Month 3 | 1.1 | 95% Confidence Interval 0.8 to 1.4 | Ratio |
| Least Square Mean of Change From Baseline Height Growth at Month 3, Month 6, Month 9, and Month 12 | Recifercept 2 mg/kg BIW | Month 3 | 0.9 | 95% Confidence Interval 0.6 to 1.2 | Ratio |
| Least Square Mean of Change From Baseline Height Growth at Month 3, Month 6, Month 9, and Month 12 | Recifercept 1.5 mg/kg QD | Month 3 | 0.6 | 95% Confidence Interval 0.3 to 0.9 | Ratio |
| Least Square Mean of Change From Baseline Height Growth at Month 3, Month 6, Month 9, and Month 12 | Recifercept 1 mg/kg QW | Month 6 | 0.9 | 95% Confidence Interval 0.7 to 1.1 | Ratio |
1 to 10 of 387Source ClinicalTrials.gov
See all 387 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 mg/kg QW | 20 | |
| Overall Study | STARTED | Recifercept 2 mg/kg BIW | 19 | |
| Overall Study | STARTED | Recifercept 1.5 mg/kg QD | 18 | |
| Overall Study | COMPLETED | Recifercept 1 mg/kg QW | 16 | |
| Overall Study | COMPLETED | Recifercept 2 mg/kg BIW | 17 | |
| Overall Study | COMPLETED | Recifercept 1.5 mg/kg QD | 3 | |
| Overall Study | NOT COMPLETED | Recifercept 1 mg/kg QW | 4 | |
| Overall Study | NOT COMPLETED | Recifercept 2 mg/kg BIW | 2 | |
| Overall Study | NOT COMPLETED | Recifercept 1.5 mg/kg QD | 15 |
Source ClinicalTrials.gov
Adverse events378
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 | Anaemia | Blood and lymphatic system disorders | Recifercept 1 mg/kg QW | 1 | 20 | 1 |
| other | Anaemia | Blood and lymphatic system disorders | Recifercept 2 mg/kg BIW | 1 | 19 | 1 |
| other | Anaemia | Blood and lymphatic system disorders | Recifercept 1.5 mg/kg QD | 1 | 18 | 1 |
| other | Eosinophilia | Blood and lymphatic system disorders | Recifercept 1 mg/kg QW | 0 | 20 | 0 |
| other | Eosinophilia | Blood and lymphatic system disorders | Recifercept 2 mg/kg BIW | 1 | 19 | 2 |
| other | Eosinophilia | Blood and lymphatic system disorders | Recifercept 1.5 mg/kg QD | 0 | 18 | 0 |
| other | Monocytosis | Blood and lymphatic system disorders | Recifercept 1 mg/kg QW | 0 | 20 | 0 |
| other | Monocytosis | Blood and lymphatic system disorders | Recifercept 2 mg/kg BIW | 1 | 19 | 1 |
| other | Monocytosis | Blood and lymphatic system disorders | Recifercept 1.5 mg/kg QD | 0 | 18 | 0 |
| other | Thrombocytosis | Blood and lymphatic system disorders | Recifercept 1 mg/kg QW | 0 | 20 | 0 |
1 to 10 of 378Source ClinicalTrials.gov
See all 378 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 QW | 0 | 20 |
| Total, all-cause mortality | Recifercept 2 mg/kg BIW | 0 | 19 |
| Total, all-cause mortality | Recifercept 1.5 mg/kg QD | 0 | 18 |
| Total, other adverse events | Recifercept 1 mg/kg QW | 16 | 20 |
| Total, other adverse events | Recifercept 2 mg/kg BIW | 19 | 19 |
| Total, other adverse events | Recifercept 1.5 mg/kg QD | 16 | 18 |
| Total, serious adverse events | Recifercept 1 mg/kg QW | 1 | 20 |
| Total, serious adverse events | Recifercept 2 mg/kg BIW | 0 | 19 |
| Total, serious adverse events | Recifercept 1.5 mg/kg QD | 0 | 18 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 38685585 | DERIVED |
Source PubMed
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/NCT04638153
GET https://conductscience.org/api/trials/v1/NCT04638153/registered?page=2