Open-label Extension of Study 20130173 of Denosumab in Children and Young Adults With Osteogenesis Imperfecta

NCT ID: NCT03638128

Last Updated: 2022-12-20

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE3

Total Enrollment

75 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-07-26

Study Completion Date

2022-03-28

Brief Summary

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To evaluate long-term safety of denosumab in children/young adults with pediatric osteogenesis imperfecta (OI) who completed the prior study 20130173 (NCT02352753).

Detailed Description

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All participants who completed the prior denosumab study 20130173 (NCT02352753) were offered participation in this study (20170534). Participants could continue to receive denosumab once every 3 months (Q3M) or could receive denosumab once every 6 months (Q6M) or off-treatment observation only at the investigator's discretion. The study design allowed subjects to discontinue denosumab, resume denosumab, initiate alternative osteoporosis medication, discontinue alternative osteoporosis medication, or receive no treatment (observation only) at any time. Therefore results of this study were analyzed according to both baseline treatment and subsequent treatment trajectories.

Conditions

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Osteogenesis Imperfecta (OI)

Keywords

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OI Bone.

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Alternative Medications / Observational

Participants who received non-denosumab alternative therapy during the study or who were not receiving any medication at baseline.

Alternative osteoporosis medication(s) were determined at the investigator's discretion and per standard of care and local guidelines.

Group Type OTHER

Alternative osteoporosis medications

Intervention Type DRUG

Alternative osteoporosis medication/s at the discretion of the investigator.

Denosumab 1 mg/kg Q6M

Participants who received at least 1 dose of 1 mg/kg denosumab administered once every 6 months (Q6M) by subcutaneous injection, but no Q3M denosumab during this study.

Group Type EXPERIMENTAL

Denosumab

Intervention Type DRUG

Solution for injection

Denosumab 1 mg/kg Q3M

Participants who received at least one dose of 1 mg/kg denosumab administered once every 3 months (Q3M) by subcutaneous injection during this study.

Group Type EXPERIMENTAL

Denosumab

Intervention Type DRUG

Solution for injection

Interventions

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Denosumab

Solution for injection

Intervention Type DRUG

Alternative osteoporosis medications

Alternative osteoporosis medication/s at the discretion of the investigator.

Intervention Type DRUG

Other Intervention Names

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Prolia

Eligibility Criteria

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Inclusion Criteria

* Subject has provided informed consent/assent prior to initiation of any Study 20170534 specific activities/procedures. Subject's legally acceptable representative has provided informed consent when the subject is legally too young to provide informed consent and the subject has provided written assent based on local regulations and/or guidelines prior to any study-specific activities/procedures being initiated.
* Subject is currently/was enrolled in Study 20130173 and

* completed the 20130173 End of Study (EOS) visit (regardless of completing or ending investigational product early) OR
* did not reconsent/reassent to transition to 3-month dosing regimen on Study 20130173 OR
* early terminated from Study 20130173 as a result of meeting bone mineral density (BMD) Z-score investigational product stopping criteria.

Exclusion Criteria

* Treatment with any prohibited proscribed medications while receiving denosumab. Eligibility into study treatment with alternative osteoporosis medication/s of investigator's choice, follow guidelines per the specific alternative osteoporosis medication/s selected. For subjects off-treatment (observation only), no prohibited medications apply.
* Subjects currently receiving treatment in another investigational device or drug study other than Study 20130173. Other investigational procedures while participating in this study are excluded.
* For subjects expected to receive investigational product (denosumab) at study day 1: Female subject is pregnant or breastfeeding or planning to become pregnant or breastfeed during treatment and for an additional 5 months after the last dose of denosumab. Females of childbearing potential (Tanner Stage greater than or equal to 2) should only be included in the study after a negative highly sensitive urine or serum pregnancy test. For study treatment with alternative osteoporosis medication/s of investigator's choice, follow guidelines per the specific alternative osteoporosis medication/s selected. For Subjects off-treatment (observation only), no exclusion applies.
* For subjects expected to receive investigational product (denosumab) at study day 1: Female subjects of childbearing potential unwilling to practice true sexual abstinence (refrain from heterosexual intercourse) or use 1 highly effective method of contraception during treatment and for an additional 5 months after the last dose of investigational product (denosumab). For study treatment with alternative osteoporosis medication/s of investigator's choice, follow contraception guidelines per the specific alternative osteoporosis medication/s selected. For subjects not receiving any investigational product (observation only), no contraception required.
* History or evidence of any other clinically significant disorder, condition or disease (with the exception of those outlined above) that, in the opinion of the investigator or Amgen physician, if consulted, would pose a risk to subject safety or interfere with the study evaluation, procedures or completion.
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Amgen

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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MD

Role: STUDY_DIRECTOR

Amgen

Locations

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Childrens Hospital of Los Angeles

Los Angeles, California, United States

Site Status

Indiana University - Riley Hospital for Children

Indianapolis, Indiana, United States

Site Status

The Childrens Hospital at Westmead

Westmead, New South Wales, Australia

Site Status

Perth Childrens Hospital

Nedlands, Western Australia, Australia

Site Status

Universite Catholique de Louvain Cliniques Universitaires Saint Luc

Brussels, , Belgium

Site Status

Childrens Hospital of Eastern Ontario

Ottawa, Ontario, Canada

Site Status

The Hospital for Sick Children

Toronto, Ontario, Canada

Site Status

Shriners Hospital for Children

Montreal, Quebec, Canada

Site Status

Fakultni nemocnice Plzen

Pilsen, , Czechia

Site Status

Thomayerova nemocnice

Prague, , Czechia

Site Status

Centre Hospitalier Universitaire de Bordeaux - Hopital Pellegrin

Bordeaux, , France

Site Status

Hopital Necker Enfants Malades

Paris, , France

Site Status

Uniklinik Köln

Cologne, , Germany

Site Status

Semmelweis Egyetem

Budapest, , Hungary

Site Status

Azienda Ospedaliera Policlinico Umberto I

Roma, , Italy

Site Status

SPZOZ Centralny Szpital Kliniczny Uniwersytetu Medycznego w Lodzi

Lodz, , Poland

Site Status

Hospital Sant Joan de Deu

Esplugues de Llobregat, Catalonia, Spain

Site Status

Hospital Universitari i Politecnic La Fe

Valencia, Valencia, Spain

Site Status

Birmingham Childrens Hospital

Birmingham, , United Kingdom

Site Status

Bristol Royal Hospital for Children

Bristol, , United Kingdom

Site Status

Royal Hospital for Children

Glasgow, , United Kingdom

Site Status

Sheffield Childrens Hospital

Sheffield, , United Kingdom

Site Status

Countries

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United States Australia Belgium Canada Czechia France Germany Hungary Italy Poland Spain United Kingdom

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

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http://www.amgentrials.com

AmgenTrials clinical trials website

Other Identifiers

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2018-000550-21

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

20170534

Identifier Type: -

Identifier Source: org_study_id