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Sclerostin Inhibitor

Romosozumab for Osteogenesis Imperfecta

Phase 1
Waitlist Available
Research Sponsored by Amgen
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 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
Awards & highlights

Study Summary

This trial will study how a new drug affects the bones of children and adolescents with Osteogenesis Imperfecta.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~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
This trial's timeline: 3 weeks for screening, Varies for treatment, and 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 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Accumulation Ratio of Romosozumab
Area Under the Serum Concentration Time Curve (AUC) From Time 0 to Day 28 (AUC[0-28]) of Romosozumab
Maximum Observed Serum Concentration (Cmax) of Romosozumab
+2 more
Secondary outcome measures
Mean Change From Baseline in Lumbar Spine BMD Z-Score
Number of Participants With Anti-romosozumab Antibodies
Number of Participants With Changes From Baseline in Cranial Nerve VII Examination Findings at Day 57, Day 85, and Day 169
+6 more

Side effects data

From 2016 Phase 3 trial • 245 Patients • NCT02186171
21%
Nasopharyngitis
9%
Back pain
9%
Hypertension
6%
Arthralgia
6%
Constipation
6%
Headache
5%
Procedural pain
2%
Muscle spasms
2%
Myalgia
1%
Cerebral ischaemia
1%
Urinary tract infection
1%
Subdural haematoma
1%
Laceration
1%
Rib fracture
1%
Anaemia postoperative
1%
Carotid artery stenosis
1%
Vascular encephalopathy
1%
Depressed mood
1%
Thoracic vertebral fracture
1%
Oropharyngeal cancer
1%
Atrial flutter
1%
Death
1%
Device related infection
1%
Escherichia sepsis
1%
Coronary artery stenosis
1%
Cardio-respiratory arrest
1%
Carotid arteriosclerosis
1%
Cerebrovascular accident
1%
Angina unstable
1%
Implant site haematoma
1%
Non-cardiac chest pain
1%
Appendicitis perforated
1%
Cardiac failure
1%
Cholecystitis
1%
Appendicitis
1%
Pneumonia
1%
Haemorrhagic stroke
1%
Depression
1%
Benign prostatic hyperplasia
1%
Myocardial ischaemia
1%
Wolff-Parkinson-White syndrome
1%
Gastrooesophageal reflux disease
1%
Osteoarthritis
1%
Basal cell carcinoma
100%
80%
60%
40%
20%
0%
Study treatment Arm
Placebo
Romosozumab 210 mg

Trial Design

2Treatment groups
Experimental Treatment
Group I: Romosozumab: 5 - < 12 Years of AgeExperimental Treatment3 Interventions
Participants will receive 1 of 3 dose levels of romosozumab. All participants also received calcium and vitamin D.
Group II: Romosozumab: 12 - < 18 Years of AgeExperimental Treatment3 Interventions
Participants will receive 1 of 3 dose levels of romosozumab. All participants also received calcium and vitamin D.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Calcium
2014
Completed Phase 4
~4660
Vitamin D
2013
Completed Phase 4
~3470
Romosozumab
2021
Completed Phase 4
~13910

Find a Location

Who is running the clinical trial?

AmgenLead Sponsor
1,371 Previous Clinical Trials
1,378,081 Total Patients Enrolled
3 Trials studying Osteogenesis Imperfecta
334 Patients Enrolled for Osteogenesis Imperfecta
MDStudy DirectorAmgen
914 Previous Clinical Trials
924,554 Total Patients Enrolled
3 Trials studying Osteogenesis Imperfecta
334 Patients Enrolled for Osteogenesis Imperfecta

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Who meets the criteria for inclusion in this experiment?

"To be eligible for this medical trial, applicants must have Lobstein's Syndrome and should fall between the ages of 5 to 17. The researchers are looking to enrol 25 individuals in total."

Answered by AI

Is the patient pool for this research limited to individuals of a certain age?

"This study has an age requirement of 5-17years, so individuals that fit into this bracket are eligible to apply."

Answered by AI

Has Romosozumab been subject to any other scientific investigations?

"The initial clinical trials for romosozumab were launched in 2020 at the Kessler Institute for Rehabilitation. To date, 217 such studies have been finished and 12 more are presently enrolling patients from Indianapolis, Indiana and its surrounding area."

Answered by AI

Is this research endeavor the inaugural trial of its kind?

"To date, 12 active research trials with Romosozumab are taking place in 13 cities of 9 nations. The initial investigation into the drug's efficacy began at Kessler Institute for Rehabilitation back in 2020 and involved 39 participants; it concluded its Phase 2 approval stage successfully. Moreover, a total of 217 studies have been finished since then."

Answered by AI

How many participants are engaged in this clinical research study?

"This trial is no longer open for recruitment, having been posted on January 21st 2021 and last updated October 25th 2022. If you are looking to participate in other studies, there are currently 10 trials involving Lobstein's Disease that require volunteers and 12 research initiatives recruiting participants who could benefit from Romosozumab treatments."

Answered by AI

What adverse effects are linked to the utilization of Romosozumab?

"Our evaluation at Power concluded that Romosozumab has a relatively low safety score of 1, as this is only in the early stages of clinical trials and there are limited data points on efficacy."

Answered by AI

Have participants been actively sought for this trial?

"The trial, which had its inception on the 21st of January 2021 and was last revised on October 25th 2022 is not currently enrolling. Nevertheless, there are 22 other clinical trials seeking participants."

Answered by AI
~6 spots leftby Apr 2025