Osteogenesis ImperfectaPhase 1NCT04545554

Study to Evaluate Romosozumab in Children and Adolescents With Osteogenesis Imperfecta

A Phase 1 study testing Romosozumab, Calcium and Vitamin D in people with Osteogenesis Imperfecta, run by Amgen at 15 sites in 8 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

The primary objective of this study is to evaluate the pharmacokinetics (PK) profile following multiple subcutaneous (SC) doses of romosozumab in children and adolescents with Osteogenesis Imperfecta (OI). Read more about this study

Who is it for?: Ages 5 to 17 years
Conditions the study is for: Osteogenesis Imperfecta
What is being tested?: Romosozumab, Calcium and Vitamin D
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 25 participants
Where: 15 sitesin 8 countries
Study dates: 21 Jan 2021to 30 Mar 2023
Phase: Phase 1
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 5 to 17 years
All sexes
No healthy volunteers
Child

Who may be able to join

Ambulatory male or female children 5 to less than 18 years of age upon entry into screening
Clinical diagnosis of OI defined as a clinical history consistent with type I-IV OI as determined by presence of expected phenotype and lack of additional features unrelated to type I-IV OI

Who cannot join

History of an electrophoresis pattern inconsistent with type I to type IV OI
History of known mutation in a gene other than collagen type I alpha 1/collagen type I alpha 2 (COL1AI/COL1A2) causing OI or other metabolic bone disease
History of other bone diseases that affect bone metabolism (eg, osteoporosis pseudoglioma syndrome, idiopathic juvenile osteoporosis, osteopetrosis, hypophosphatasia)
History of Kawasaki disease, rheumatic myocarditis, ischemic cardiomyopathy, inherited cardiomyopathies, nephrotic syndrome, familial hypercholesterolemia, stroke, or any thromboembolic disorder
Unhealed fracture as defined by orthopedic opinion
Symptoms associated with skull abnormalities such as basilar invagination, basilar impression or Chiari malformation

Show all 9Show fewer

Prior treatment with anti-sclerostin antibody, fluoride or strontium, parathyroid hormone (PTH) within 12 months prior to screening, denosumab within 12 months or zoledronic acid within 6 months prior to first dose
Less than 2 evaluable vertebrae by DXA evaluation in the region of interest, L1 - L4, as confirmed by the central imaging laboratory.
Clinically significant valvular heart disease based on local echocardiogram (ECHO) results.

Trial locations and contacts

15 sites in 8 countries.

Country

City, postcode or hospital

15 sites in 8 countries

Spain4 sites

Hospital de Cruces
Barakaldo, Basque Country, 48903
Hospital Sant Joan de Deu
Esplugues de Llobregat, Catalonia, 08950
Hospital Universitario de Getafe
Getafe, Madrid, 28905
Hospital Universitari i Politecnic La Fe
Valencia, Valencia, 46026

Other trials in Spain

United States3 sites

Riley Hospital for Children
Indianapolis, Indiana, 46202
The Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
Vanderbilt University Medical Center
Nashville, Tennessee, 37212-3157

Other trials in United States

Turkey (Türkiye)3 sites

Gazi Universitesi Tip Fakultesi
Ankara, 06500
Koc Universitesi Hastanesi
Istanbul, 34010
Ege Universitesi Ilac Gelistirme ve Farmakokinetik Arastirma Uygulama Merkezi (ARGEFAR)
Izmir, 35100

Other trials in Turkey (Türkiye)

Austria1 site

Kepler Universitaetsklinikum GmbH
Linz, 4020

Other trials in Austria

Germany1 site

Uniklinik Köln
Cologne, 50937

Other trials in Germany

Greece1 site

General Children Hospital Panagioti and Aglaias Kyriakou
Athens, 11527

Other trials in Greece

Hungary1 site

Semmelweis Egyetem
Budapest, 1094

Other trials in Hungary

Italy1 site

IRCCS Ospedale Pediatrico Bambino Gesu
Roma, 00165

Other trials in Italy

About this study

Official title: An Open-label, Ascending Multiple-dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Romosozumab in Children and Adolescents With Osteogenesis Imperfecta

Conditions: Osteogenesis Imperfecta
Being tested: Romosozumab, Calcium, Vitamin D
Medical subject headings: Osteogenesis Imperfecta

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

RomosozumabDrug
Participants will receive multiple doses of romosozumab via a SC injection.
CalciumDietary supplement
All participants will receive daily supplements of elemental calcium.
Vitamin DDietary supplement
All participants will receive daily supplementation with vitamin D.

Study groups in NCT04545554
Study group	Role	What they receive
Romosozumab: 12 - < 18 Years of Age	Experimental	Participants will receive 1 of 3 dose levels of romosozumab. All participants also received calcium and vitamin D. Receives: Drug: Romosozumab; Dietary Supplement: Calcium; Dietary Supplement: Vitamin D
Romosozumab: 5 - < 12 Years of Age	Experimental	Participants will receive 1 of 3 dose levels of romosozumab. All participants also received calcium and vitamin D. Receives: Drug: Romosozumab; Dietary Supplement: Calcium; Dietary Supplement: Vitamin D

What is being measured

The primary outcome is the main question the study is designed to answer.

Maximum Observed Serum Concentration (Cmax) of Romosozumab
Mean Cmax values following Days 1 and 57 are presented.
Measured: 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
Time to Cmax (Tmax) of Romosozumab
Median tmax values following Days 1 and 57 are presented.
Measured: 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
Area Under the Serum Concentration Time Curve (AUC) From Time 0 to Day 28 (AUC[0-28]) of Romosozumab
Mean AUC(0-28) values following Days 1 and 57 are presented.
Measured: 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
Accumulation Ratio of Romosozumab
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.
Measured: 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
Terminal Half-life of Romosozumab
Median terminal half-life values at Day 57 are presented.
Measured: 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

Secondary outcomes (9)

Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
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.
Measured: Day 1 to end of study (up to Day 169); median duration on study was 5.55 months
Number of Participants With Changes From Baseline in Cranial Nerve VII Examination Findings at 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.
Measured: Baseline (Day 1), Day 57, Day 85, and Day 169
Number of Participants With Anti-romosozumab Antibodies
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.
Measured: Blood samples for anti-romosozumab antibodies were taken Day 1, Day 15, Day 29, Day 85, and Day 169
Percentage Change From Baseline in Serum Concentrations of Serum Type 1 Collagen C-Telopeptide (CTX)
Serum concentrations of the bone turnover marker CTX were determined at pre-specified time points.
Measured: Blood samples were taken Days 1 (baseline), 8, 15, 29, 57, 64, 71, 85, 113, and 169
Percentage Change From Baseline in Serum Concentrations of Procollagen Type 1 N-terminal Propeptide (P1NP)
Serum concentrations of the bone turnover marker P1NP were determined at pre-specified time points.
Measured: Blood samples were taken Days 1 (baseline), 8, 15, 29, 57, 64, 71, 85, 113, and 169
Percentage Change From Baseline in Bone Mineral Density (BMD) of the Lumbar Spine
BMD was assessed by dual energy X-ray absorptiometry (DXA) scans of the anteroposterior lumbar spine (L1 through L4) and analyzed by a central imaging laboratory. At least 2 lumbar vertebrae from L1 - L4 must be evaluable by DXA.
Measured: DXA scans were during screening (baseline) and at Day 85 and Day 169
Percentage Change From Baseline in Bone Mineral Content (BMC) of the Lumbar Spine
BMC was assessed by DXA scans of the anteroposterior lumbar spine (L1 through L4) and analyzed by a central imaging laboratory. At least 2 lumbar vertebrae from L1 - L4 must be evaluable by DXA.
Measured: DXA scans were during screening (baseline) and at Day 85 and Day 169
Percentage Change From Baseline in Lumbar Spine Bone Area
Bone area was assessed by DXA scans of the anteroposterior lumbar spine (L1 through L4) and analyzed by a central imaging laboratory. At least 2 lumbar vertebrae from L1 - L4 must be evaluable by DXA.
Measured: DXA scans were during screening (baseline) and at Day 85 and Day 169
Mean Change From Baseline in Lumbar Spine BMD Z-Score
Lumbar spine BMD was assessed by DXA scans. The results were then converted to Z-scores. The Z-score indicated the number of standard deviations away from the reference population and a score of 0 is equal to the mean. Positive changes from baseline indicated an improvement in lumbar spine BMD.
Measured: DXA scans were during screening (baseline) and at Day 85 and Day 169

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

21 Jan 202130 Mar 2023

First posted11 Sep 2020
Study start21 Jan 2021
Primary completion30 Mar 2023
Study completion30 Mar 2023

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (2)Papers the sponsor has linked to this study.
Other trials for Osteogenesis ImperfectaStudies of the same condition, open and under way.
More trials run by AmgenThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 1
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Sequential
Masking: None (open label)
Enrolment: 25 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04545554
Sponsor's protocol ID: 20160227
Other IDs: 2017-004972-74, 2017
Status last verified: Oct 2023
First submitted: 4 Sep 2020
Last updated: 15 Apr 2024
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: Yes: De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request

Publications

Bartosik M, Windels O, von Brackel FN, Amling M, Oheim R. Romosozumab in postmenopausal women with classical Osteogenesis imperfecta. Arch Osteoporos. 2026 Jul 18;21(1):100. doi: 10.1007/s11657-026-01744-3.
PubMed 42470570
Tabaie SA, O'Mara AE, Sheppard ED, Tosi LL. A Comprehensive Review of Bone Health in a Child: From Birth to Adulthood. J Am Acad Orthop Surg. 2024 May 1;32(9):363-372. doi: 10.5435/JAAOS-D-23-00853. Epub 2024 Jan 23.
PubMed 38261781

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 2
Osteogenesis Imperfecta Trial of AGA2115 for ADUlts With COL1A1 and/or COL1A2 GeNetic Variations (IDUN)
Osteogenesis Imperfecta (OI)
Active, not recruitingObservational study
Cardiopulmonary Outcomes in Osteogenesis Imperfecta: BBD7708
Osteogenesis Imperfecta
Enrolling by invitationObservational study
GRACE II (General Retrospective Analysis of Commercial Experience With AGN1 LOEP)
Osteoporosis
Active, not recruitingPhase 3
Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta
Osteogenesis Imperfecta
Active, not recruitingPhase 2/3
Setrusumab vs Placebo for Osteogenesis Imperfecta
Osteogenesis Imperfecta

All Osteogenesis Imperfecta trials

Study at a glance

From the registry record, last updated by the sponsor on 15 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: An Open-label, Ascending Multiple-dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Romosozumab in Children and Adolescents With Osteogenesis Imperfecta
ClinicalTrials.gov ID: NCT04545554
Acronym / sponsor ID: 20160227
Conditions: Osteogenesis Imperfecta
Phase: Phase 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 25 participants (actual)
Lead sponsor: AmgenIndustry (pharma, biotech, devices)
Study start: 21 Jan 2021 (actual)
Primary completion: 30 Mar 2023 (actual)
Study completion: 30 Mar 2023 (actual)
First posted: 11 Sep 2020 (actual)
Results: Posted 15 Apr 2024

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Sequential
Masking: None (open label)
Number of arms: 2

Arm	Type	Receives
Romosozumab: 12 - < 18 Years of Age	Experimental	Drug: Romosozumab, Dietary Supplement: Calcium, Dietary Supplement: Vitamin DParticipants will receive 1 of 3 dose levels of romosozumab. All participants also received calcium and vitamin D.
Romosozumab: 5 - < 12 Years of Age	Experimental	Drug: Romosozumab, Dietary Supplement: Calcium, Dietary Supplement: Vitamin DParticipants will receive 1 of 3 dose levels of romosozumab. All participants also received calcium and vitamin D.

Who may qualify

Ages: 5 to 17 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (2 listed)

Ambulatory male or female children 5 to less than 18 years of age upon entry into screening
Clinical diagnosis of OI defined as a clinical history consistent with type I-IV OI as determined by presence of expected phenotype and lack of additional features unrelated to type I-IV OI

Key exclusion criteria (9 listed)

History of an electrophoresis pattern inconsistent with type I to type IV OI
History of known mutation in a gene other than collagen type I alpha 1/collagen type I alpha 2 (COL1AI/COL1A2) causing OI or other metabolic bone disease
History of other bone diseases that affect bone metabolism (eg, osteoporosis pseudoglioma syndrome, idiopathic juvenile osteoporosis, osteopetrosis, hypophosphatasia)
History of Kawasaki disease, rheumatic myocarditis, ischemic cardiomyopathy, inherited cardiomyopathies, nephrotic syndrome, familial hypercholesterolemia, stroke, or any thromboembolic disorder
Unhealed fracture as defined by orthopedic opinion

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
RomosozumabParticipants will receive multiple doses of romosozumab via a SC injection.	Drug	Romosozumab: 12 - < 18 Years of Age, Romosozumab: 5 - < 12 Years of Age
CalciumAll participants will receive daily supplements of elemental calcium.	Dietary supplement	Romosozumab: 12 - < 18 Years of Age, Romosozumab: 5 - < 12 Years of Age
Vitamin DAll participants will receive daily supplementation with vitamin D.	Dietary supplement	Romosozumab: 12 - < 18 Years of Age, Romosozumab: 5 - < 12 Years of Age

Primary outcome

Maximum Observed Serum Concentration (Cmax) of Romosozumab
Time frame: 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.
Time to Cmax (Tmax) of Romosozumab
Time frame: 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.
Area Under the Serum Concentration Time Curve (AUC) From Time 0 to Day 28 (AUC[0-28]) of Romosozumab
Time frame: 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.
Accumulation Ratio of Romosozumab
Time frame: 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.
Terminal Half-life of Romosozumab
Time frame: 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.

9 secondary outcomes

Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Time frame: Day 1 to end of study (up to Day 169); median duration on study was 5.55 months
Number of Participants With Changes From Baseline in Cranial Nerve VII Examination Findings at Day 57, Day 85, and Day 169
Time frame: Baseline (Day 1), Day 57, Day 85, and Day 169
Number of Participants With Anti-romosozumab Antibodies
Time frame: Blood samples for anti-romosozumab antibodies were taken Day 1, Day 15, Day 29, Day 85, and Day 169
Percentage Change From Baseline in Serum Concentrations of Serum Type 1 Collagen C-Telopeptide (CTX)
Time frame: Blood samples were taken Days 1 (baseline), 8, 15, 29, 57, 64, 71, 85, 113, and 169
Percentage Change From Baseline in Serum Concentrations of Procollagen Type 1 N-terminal Propeptide (P1NP)
Time frame: Blood samples were taken Days 1 (baseline), 8, 15, 29, 57, 64, 71, 85, 113, and 169
Percentage Change From Baseline in Bone Mineral Density (BMD) of the Lumbar Spine
Time frame: DXA scans were during screening (baseline) and at Day 85 and Day 169
Percentage Change From Baseline in Bone Mineral Content (BMC) of the Lumbar Spine
Time frame: DXA scans were during screening (baseline) and at Day 85 and Day 169
Percentage Change From Baseline in Lumbar Spine Bone Area
Time frame: DXA scans were during screening (baseline) and at Day 85 and Day 169
Mean Change From Baseline in Lumbar Spine BMD Z-Score
Time frame: DXA scans were during screening (baseline) and at Day 85 and Day 169

Results and safety

Results posted to the registry on 15 Apr 2024, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Cohort 1: Romosozumab Dose A (12 to < 18 Years of Age)	4	4
Cohort 2: Romosozumab Dose A (5 to < 12 Years of Age)	4	4
Cohort 3: Romosozumab Dose B (12 to < 18 Years of Age)	4	4
Cohort 4: Romosozumab Dose B (5 to < 12 Years of Age)	5	4
Cohort 5: Romosozumab Dose C (12 to < 18 Years of Age)	4	4
Cohort 6: Romosozumab Dose C (5 to < 12 Years of Age)	4	4

Primary outcome: Maximum Observed Serum Concentration (Cmax) of Romosozumab

Time frame: 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 (Standard Deviation). Unit: μg/mL

Measure	Cohort 1: Romosozumab Dose A (12 to < 18 Years of Age) n = 4	Cohort 2: Romosozumab Dose A (5 to < 12 Years of Age) n = 4	Cohort 3: Romosozumab Dose B (12 to < 18 Years of Age) n = 4	Cohort 4: Romosozumab Dose B (5 to < 12 Years of Age) n = 4	Cohort 5: Romosozumab Dose C (12 to < 18 Years of Age) n = 4	Cohort 6: Romosozumab Dose C (5 to < 12 Years of Age) n = 4
Day 1	2.64 (1.06)	1.74 (0.825)	13.8 (6.32)	10.3 (1.70)	25.7 (3.02)	21.3 (6.45)
Day 57	2.43 (1.36)	5.55 (2.11)	12.8 (6.31)	9.14 (7.50)	22.8 (10.5)	19.4 (2.14)

Population analysed: Pharmacokinetic (PK) Analysis Set: all participants for whom at least 1 PK parameter or endpoint could be adequately tested.

Primary outcome: Time to Cmax (Tmax) of Romosozumab

Time frame: 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 (Full Range). Unit: day

Measure	Cohort 1: Romosozumab Dose A (12 to < 18 Years of Age) n = 4	Cohort 2: Romosozumab Dose A (5 to < 12 Years of Age) n = 4	Cohort 3: Romosozumab Dose B (12 to < 18 Years of Age) n = 4	Cohort 4: Romosozumab Dose B (5 to < 12 Years of Age) n = 4	Cohort 5: Romosozumab Dose C (12 to < 18 Years of Age) n = 4	Cohort 6: Romosozumab Dose C (5 to < 12 Years of Age) n = 4
Day 1	6.5 (5.9 to 7.9)	7.9 (7.9 to 15)	6.9 (6.9 to 7.0)	6.9 (4.9 to 7.1)	7.0 (4.9 to 8.0)	7.5 (6.9 to 8.9)
Day 57	7.0 (7.0 to 12)	7.0 (6.9 to 7.0)	7.0 (6.0 to 7.9)	8.0 (6.0 to 11)	7.0 (4.0 to 12)	7.5 (6.0 to 8.8)

Population analysed: PK Analysis Set: all participants for whom at least 1 PK parameter or endpoint could be adequately tested.

Primary outcome: Area Under the Serum Concentration Time Curve (AUC) From Time 0 to Day 28 (AUC[0-28]) of Romosozumab

Time frame: 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 (Standard Deviation). Unit: day*μg/mL

Measure	Cohort 1: Romosozumab Dose A (12 to < 18 Years of Age) n = 4	Cohort 2: Romosozumab Dose A (5 to < 12 Years of Age) n = 4	Cohort 3: Romosozumab Dose B (12 to < 18 Years of Age) n = 4	Cohort 4: Romosozumab Dose B (5 to < 12 Years of Age) n = 4	Cohort 5: Romosozumab Dose C (12 to < 18 Years of Age) n = 4	Cohort 6: Romosozumab Dose C (5 to < 12 Years of Age) n = 4
Day 1	30.9 (13.5)	16.9 (5.78)	163 (89.1)	113 (50.3)	344 (106)	234 (93.7)
Day 57	27.4 (15.1)	50.5 (20.7)	153 (86.7)	95.1 (76.1)	293 (134)	203 (40.7)

Population analysed: PK Analysis Set: all participants for whom at least 1 PK parameter or endpoint could be adequately tested.

Primary outcome: Accumulation Ratio of Romosozumab

Time frame: 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 (Standard Deviation). Unit: ratio

Measure	Cohort 1: Romosozumab Dose A (12 to < 18 Years of Age) n = 4	Cohort 2: Romosozumab Dose A (5 to < 12 Years of Age) n = 4	Cohort 3: Romosozumab Dose B (12 to < 18 Years of Age) n = 4	Cohort 4: Romosozumab Dose B (5 to < 12 Years of Age) n = 4	Cohort 5: Romosozumab Dose C (12 to < 18 Years of Age) n = 4	Cohort 6: Romosozumab Dose C (5 to < 12 Years of Age) n = 4
Value	0.885 (0.487)	3.6 (2.56)	0.922 (0.0673)	0.724 (0.392)	0.843 (0.260)	0.935 (0.283)

Population analysed: PK Analysis Set: all participants for whom at least 1 PK parameter or endpoint could be adequately tested.

9 secondary outcomes are reported in the full results.

Adverse events

Time frame: All-cause mortality was collected from enrollment to the end of study visit (Day 169); median time on study was 5.55 months. Adverse events were collected from first dose of study drug until the end of study visit (Day 169); median duration was 5.55 months.. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Cohort 1: Romosozumab Dose A (12 to < 18 Years of Age)	0 of 4 (0%)	1 of 4 (25%)	0 of 4
Cohort 2: Romosozumab Dose A (5 to < 12 Years of Age)	1 of 4 (25%)	0 of 4 (0%)	0 of 4
Cohort 3: Romosozumab Dose B (12 to < 18 Years of Age)	0 of 4 (0%)	2 of 4 (50%)	0 of 4
Cohort 4: Romosozumab Dose B (5 to < 12 Years of Age)	1 of 5 (20%)	4 of 5 (80%)	0 of 5
Cohort 5: Romosozumab Dose C (12 to < 18 Years of Age)	0 of 4 (0%)	1 of 4 (25%)	0 of 4
Cohort 6: Romosozumab Dose C (5 to < 12 Years of Age)	0 of 4 (0%)	3 of 4 (75%)	0 of 4

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 15 Apr 2024.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 42470570Bartosik M, Windels O, von Brackel FN, Amling M, Oheim R. Romosozumab in postmenopausal women with classical Osteogenesis imperfecta. Arch Osteoporos. 2026 Jul 18;21(1):100. doi: 10.1007/s11657-026-01744-3.
Derived from this study: PubMed 38261781Tabaie SA, O'Mara AE, Sheppard ED, Tosi LL. A Comprehensive Review of Bone Health in a Child: From Birth to Adulthood. J Am Acad Orthop Surg. 2024 May 1;32(9):363-372. doi: 10.5435/JAAOS-D-23-00853. Epub 2024 Jan 23.
AmgenTrials clinical trials websiteLink provided by the sponsor.
Individual participant dataSharing planned: Yes. De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request

Related trials for Osteogenesis Imperfecta

RecruitingPhase 2
Osteogenesis Imperfecta Trial of AGA2115 for ADUlts With COL1A1 and/or COL1A2 GeNetic Variations (IDUN)
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Cardiopulmonary Outcomes in Osteogenesis Imperfecta: BBD7708
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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

MD study director

Amgen

15 sites in 8 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Romosozumab, Calcium and Vitamin D in people with Osteogenesis Imperfecta, run by Amgen at 15 sites in 8 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 2 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 15 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 15 sites in Spain, United States, Turkey (Türkiye), Austria, Germany and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 15 Apr 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Maximum Observed Serum Concentration (Cmax) of Romosozumab (time frame: 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). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04545554, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Apr 2024.