Trial · NCT04545554
Study to Evaluate Romosozumab in Children and Adolescents With Osteogenesis Imperfecta
NCT04545554, Phase 1, Completed, Amgen; results first posted 2024-04-15
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study to Evaluate Romosozumab in Children and Adolescents With Osteogenesis Imperfecta. Trials, record NCT04545554. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04545554
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Evaluate Romosozumab in Children and Adolescents With Osteogenesis Imperfecta
- NCT number
- NCT04545554
- Status
- Completed
- Phases
- Phase 1
- Enrollment
- 25 participants, actual
- Lead sponsor
- Amgen
- Conditions
- Osteogenesis Imperfecta
- Interventions
- Romosozumab, Calcium, Vitamin D
- First posted
- 2020-09-11
- Last updated
- 2024-04-15
- Other registries' IDs
- eudract:2017-004972-74
- Results first posted
- 2024-04-15
Source ClinicalTrials.gov
Registered outcomes14
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Maximum Observed Serum Concentration (Cmax) of Romosozumab | Single blood samples were taken on days 1, 8, 15, 29, 57, 64, 71, 85, 113, and 169 (end of study); pre-specified PK analysis took place on Days 1 and 57 | Mean Cmax values following Days 1 and 57 are presented. |
| Primary | Time to Cmax (Tmax) of Romosozumab | Single blood samples were taken on days 1, 8, 15, 29, 57, 64, 71, 85, 113, and 169 (end of study); pre-specified PK analysis took place on Days 1 and 57 | Median tmax values following Days 1 and 57 are presented. |
| Primary | Area Under the Serum Concentration Time Curve (AUC) From Time 0 to Day 28 (AUC[0-28]) of Romosozumab | Single blood samples were taken on days 1, 8, 15, 29, 57, 64, 71, and 85; pre-specified PK analysis took place on Days 1 and 57 | Mean AUC(0-28) values following Days 1 and 57 are presented. |
| Primary | Accumulation Ratio of Romosozumab | Single blood samples were taken on days 1, 8, 15, 29, 57, 64, 71, and 85; pre-specified PK analysis took place on Days 1 and 57 | The accumulation ratio was calculated as AUC(0-28) at Day 57/AUC(0-28) at Day 1. Mean accumulation ratio values based on analysis at Days 1 and 57 are presented, as pre-specified. |
| Primary | Terminal Half-life of Romosozumab | Single blood samples were taken on days 1, 8, 15, 29, 57, 64, 71, 85, 113, and 169 (end of study); pre-specified PK analysis took place on Day 57 | Median terminal half-life values at Day 57 are presented. |
| Secondary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Day 1 to end of study (up to Day 169); median duration on study was 5.55 months | TEAEs were adverse events (AEs) that started on or after first dose of investigational product up to the end of study (up to Day 169). Any clinically significant changes in vital signs, electrocardiogram parameters, physical exam findings, and clinical laboratory parameters were reported as TEAEs. Injection site reactions were events of interest (EOI) for this study. |
| Secondary | Number of Participants With Changes From Baseline in Cranial Nerve VII Examination Findings at Day 57, Day 85, and Day 169 | Baseline (Day 1), Day 57, Day 85, and Day 169 | Facial nerve (cranial nerve VII) function was assessed clinically by facial symmetry inspection at rest, followed by assessment of the symmetry of specific facial movements: raising eyebrows, closing the eyes, blowing out the cheeks, smiling, pursing and closing the lips. Results of the cranial nerve examination were classed as 0 = Normal; 1 = Abnormal not clinically significant; and 2 = Abnormal clinically significant. An increase from baseline indicates an increase in abnormal clinical findings on the cranial nerve VII examination. |
| Secondary | Number of Participants With Anti-romosozumab Antibodies | Blood samples for anti-romosozumab antibodies were taken Day 1, Day 15, Day 29, Day 85, and Day 169 | Treatment-boosted anti-romosozumab antibody was defined as binding antibody positive at baseline with a \>4 x increase in magnitude post-baseline. Transient results were defined as negative results at the participant's last time point tested within the study period. |
| Secondary | Percentage Change From Baseline in Serum Concentrations of Serum Type 1 Collagen C-Telopeptide (CTX) | Blood samples were taken Days 1 (baseline), 8, 15, 29, 57, 64, 71, 85, 113, and 169 | Serum concentrations of the bone turnover marker CTX were determined at pre-specified time points. |
| Secondary | Percentage Change From Baseline in Serum Concentrations of Procollagen Type 1 N-terminal Propeptide (P1NP) | Blood samples were taken Days 1 (baseline), 8, 15, 29, 57, 64, 71, 85, 113, and 169 | Serum concentrations of the bone turnover marker P1NP were determined at pre-specified time points. |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 registered outcomesOutcomes14
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Maximum Observed Serum Concentration (Cmax) of Romosozumab | Single blood samples were taken on days 1, 8, 15, 29, 57, 64, 71, 85, 113, and 169 (end of study); pre-specified PK analysis took place on Days 1 and 57 | Pharmacokinetic (PK) Analysis Set: all participants for whom at least 1 PK parameter or endpoint could be adequately tested. | μg/mL | MEAN |
| PRIMARY | Time to Cmax (Tmax) of Romosozumab | Single blood samples were taken on days 1, 8, 15, 29, 57, 64, 71, 85, 113, and 169 (end of study); pre-specified PK analysis took place on Days 1 and 57 | PK Analysis Set: all participants for whom at least 1 PK parameter or endpoint could be adequately tested. | day | MEDIAN |
| PRIMARY | Area Under the Serum Concentration Time Curve (AUC) From Time 0 to Day 28 (AUC[0-28]) of Romosozumab | Single blood samples were taken on days 1, 8, 15, 29, 57, 64, 71, and 85; pre-specified PK analysis took place on Days 1 and 57 | PK Analysis Set: all participants for whom at least 1 PK parameter or endpoint could be adequately tested. | day*μg/mL | MEAN |
| PRIMARY | Accumulation Ratio of Romosozumab | Single blood samples were taken on days 1, 8, 15, 29, 57, 64, 71, and 85; pre-specified PK analysis took place on Days 1 and 57 | PK Analysis Set: all participants for whom at least 1 PK parameter or endpoint could be adequately tested. | ratio | MEAN |
| PRIMARY | Terminal Half-life of Romosozumab | Single blood samples were taken on days 1, 8, 15, 29, 57, 64, 71, 85, 113, and 169 (end of study); pre-specified PK analysis took place on Day 57 | PK Analysis Set: all participants for whom at least 1 PK parameter or endpoint could be adequately tested. Data is presented for participants with evaluable data. | day | MEDIAN |
| SECONDARY | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Day 1 to end of study (up to Day 169); median duration on study was 5.55 months | Safety Analysis Set: all participants enrolled in the study who received at least 1 dose of investigational product. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Changes From Baseline in Cranial Nerve VII Examination Findings at Day 57, Day 85, and Day 169 | Baseline (Day 1), Day 57, Day 85, and Day 169 | Safety Analysis Set: all participants enrolled in the study who received at least 1 dose of investigational product. Participants with data available at each time point are presented. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Anti-romosozumab Antibodies | Blood samples for anti-romosozumab antibodies were taken Day 1, Day 15, Day 29, Day 85, and Day 169 | Safety Analysis Set: all participants enrolled in the study who received at least 1 dose of investigational product. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Percentage Change From Baseline in Serum Concentrations of Serum Type 1 Collagen C-Telopeptide (CTX) | Blood samples were taken Days 1 (baseline), 8, 15, 29, 57, 64, 71, 85, 113, and 169 | PD Analysis Set: all participants for whom at least 1 PD parameter or endpoint could be adequately estimated. Participants with data available at each time point are presented. | percentage change | MEAN |
| SECONDARY | Percentage Change From Baseline in Serum Concentrations of Procollagen Type 1 N-terminal Propeptide (P1NP) | Blood samples were taken Days 1 (baseline), 8, 15, 29, 57, 64, 71, 85, 113, and 169 | PD Analysis Set: all participants for whom at least 1 PD parameter or endpoint could be adequately estimated. Participants with data available at each time point are presented. | percentage change | MEAN |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 outcomesResults by group489
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Maximum Observed Serum Concentration (Cmax) of Romosozumab | Cohort 1: Romosozumab Dose A (12 to < 18 Years of Age) | Day 1 | 2.64 | Standard Deviation 1.06 | μg/mL |
| Maximum Observed Serum Concentration (Cmax) of Romosozumab | Cohort 2: Romosozumab Dose A (5 to < 12 Years of Age) | Day 1 | 1.74 | Standard Deviation 0.825 | μg/mL |
| Maximum Observed Serum Concentration (Cmax) of Romosozumab | Cohort 3: Romosozumab Dose B (12 to < 18 Years of Age) | Day 1 | 13.8 | Standard Deviation 6.32 | μg/mL |
| Maximum Observed Serum Concentration (Cmax) of Romosozumab | Cohort 4: Romosozumab Dose B (5 to < 12 Years of Age) | Day 1 | 10.3 | Standard Deviation 1.70 | μg/mL |
| Maximum Observed Serum Concentration (Cmax) of Romosozumab | Cohort 5: Romosozumab Dose C (12 to < 18 Years of Age) | Day 1 | 25.7 | Standard Deviation 3.02 | μg/mL |
| Maximum Observed Serum Concentration (Cmax) of Romosozumab | Cohort 6: Romosozumab Dose C (5 to < 12 Years of Age) | Day 1 | 21.3 | Standard Deviation 6.45 | μg/mL |
| Maximum Observed Serum Concentration (Cmax) of Romosozumab | Cohort 1: Romosozumab Dose A (12 to < 18 Years of Age) | Day 57 | 2.43 | Standard Deviation 1.36 | μg/mL |
| Maximum Observed Serum Concentration (Cmax) of Romosozumab | Cohort 2: Romosozumab Dose A (5 to < 12 Years of Age) | Day 57 | 5.55 | Standard Deviation 2.11 | μg/mL |
| Maximum Observed Serum Concentration (Cmax) of Romosozumab | Cohort 3: Romosozumab Dose B (12 to < 18 Years of Age) | Day 57 | 12.8 | Standard Deviation 6.31 | μg/mL |
| Maximum Observed Serum Concentration (Cmax) of Romosozumab | Cohort 4: Romosozumab Dose B (5 to < 12 Years of Age) | Day 57 | 9.14 | Standard Deviation 7.50 | μg/mL |
1 to 10 of 489Source ClinicalTrials.gov
See all 489 results by groupParticipant flow30
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cohort 1: Romosozumab Dose A (12 to < 18 Years of Age) | 4 | |
| Overall Study | STARTED | Cohort 2: Romosozumab Dose A (5 to < 12 Years of Age) | 4 | |
| Overall Study | STARTED | Cohort 3: Romosozumab Dose B (12 to < 18 Years of Age) | 4 | |
| Overall Study | STARTED | Cohort 4: Romosozumab Dose B (5 to < 12 Years of Age) | 5 | |
| Overall Study | STARTED | Cohort 5: Romosozumab Dose C (12 to < 18 Years of Age) | 4 | |
| Overall Study | STARTED | Cohort 6: Romosozumab Dose C (5 to < 12 Years of Age) | 4 | |
| Overall Study | Received at Least 1 Dose Romosozumab | Cohort 1: Romosozumab Dose A (12 to < 18 Years of Age) | 4 | |
| Overall Study | Received at Least 1 Dose Romosozumab | Cohort 2: Romosozumab Dose A (5 to < 12 Years of Age) | 4 | |
| Overall Study | Received at Least 1 Dose Romosozumab | Cohort 3: Romosozumab Dose B (12 to < 18 Years of Age) | 4 | |
| Overall Study | Received at Least 1 Dose Romosozumab | Cohort 4: Romosozumab Dose B (5 to < 12 Years of Age) | 5 |
1 to 10 of 30Source ClinicalTrials.gov
See all 30 participant flowAdverse events216
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 | Ocular hyperaemia | Eye disorders | Cohort 1: Romosozumab Dose A (12 to < 18 Years of Age) | 0 | 4 | |
| other | Ocular hyperaemia | Eye disorders | Cohort 2: Romosozumab Dose A (5 to < 12 Years of Age) | 0 | 4 | |
| other | Ocular hyperaemia | Eye disorders | Cohort 3: Romosozumab Dose B (12 to < 18 Years of Age) | 0 | 4 | |
| other | Ocular hyperaemia | Eye disorders | Cohort 4: Romosozumab Dose B (5 to < 12 Years of Age) | 1 | 5 | |
| other | Ocular hyperaemia | Eye disorders | Cohort 5: Romosozumab Dose C (12 to < 18 Years of Age) | 0 | 4 | |
| other | Ocular hyperaemia | Eye disorders | Cohort 6: Romosozumab Dose C (5 to < 12 Years of Age) | 0 | 4 | |
| other | Abdominal pain | Gastrointestinal disorders | Cohort 1: Romosozumab Dose A (12 to < 18 Years of Age) | 0 | 4 | |
| other | Abdominal pain | Gastrointestinal disorders | Cohort 2: Romosozumab Dose A (5 to < 12 Years of Age) | 0 | 4 | |
| other | Abdominal pain | Gastrointestinal disorders | Cohort 3: Romosozumab Dose B (12 to < 18 Years of Age) | 0 | 4 | |
| other | Abdominal pain | Gastrointestinal disorders | Cohort 4: Romosozumab Dose B (5 to < 12 Years of Age) | 0 | 5 |
1 to 10 of 216Source ClinicalTrials.gov
See all 216 adverse eventsAdverse event totals18
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cohort 1: Romosozumab Dose A (12 to < 18 Years of Age) | 0 | 4 |
| Total, all-cause mortality | Cohort 2: Romosozumab Dose A (5 to < 12 Years of Age) | 0 | 4 |
| Total, all-cause mortality | Cohort 3: Romosozumab Dose B (12 to < 18 Years of Age) | 0 | 4 |
| Total, all-cause mortality | Cohort 4: Romosozumab Dose B (5 to < 12 Years of Age) | 0 | 5 |
| Total, all-cause mortality | Cohort 5: Romosozumab Dose C (12 to < 18 Years of Age) | 0 | 4 |
| Total, all-cause mortality | Cohort 6: Romosozumab Dose C (5 to < 12 Years of Age) | 0 | 4 |
| Total, other adverse events | Cohort 1: Romosozumab Dose A (12 to < 18 Years of Age) | 1 | 4 |
| Total, other adverse events | Cohort 2: Romosozumab Dose A (5 to < 12 Years of Age) | 0 | 4 |
| Total, other adverse events | Cohort 3: Romosozumab Dose B (12 to < 18 Years of Age) | 2 | 4 |
| Total, other adverse events | Cohort 4: Romosozumab Dose B (5 to < 12 Years of Age) | 4 | 5 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 adverse event totalsPapers2
The papers the trial lists on ClinicalTrials.gov.
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/NCT04545554
GET https://conductscience.org/api/trials/v1/NCT04545554/results?page=2