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Multicenter,Single-arm Study to Evaluate Efficacy, Safety, & Pharmacokinetics of Denosumab in Children w/ OI (OI)

Primary Purpose

Osteogenesis Imperfecta

Status

Terminated

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

Denosumab

About this trial

This is an interventional treatment trial for Osteogenesis Imperfecta focused on measuring Amgen, OI, Bone

Eligibility Criteria

2 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

• Eligibility criteria relates to initial enrollment into this study (6-Month Dosing Regimen). Subjects reconsenting to a 3-Month Dosing Regimen will not repeat eligibility assessments

Inclusion Criteria:

• Clinical diagnosis of OI defined as a clinical history consistent with type I-IV OI Clinical severity of OI as defined by 2 or more prevalent vertebral compression fractures; OR1 prevalent vertebral compression fracture and 1 or more nonvertebral fractures within the previous 2 years; OR 3 or more fractures within the previous 2 years.

Exclusion Criteria:

Inability or unwillingness to comply with the requirements for frequent calcium and phosphorus monitoring for 14 days after the first dose of denosumab (only applies to the first 5 subjects age 11 to17 enrolled in the study and the first 5 subjects of any age meeting the criteria for increased bone turnover
Currently unhealed fracture or osteotomy as defined by orthopedic opinion
Osteotomy within 5 months of screening
Evidence of untreated oral cavities or oral infections
Recent or planned invasive dental procedure
Surgical tooth extraction which has not healed by screening
History of an electrophoresis pattern inconsistent with type I to IV OI
History of genetic testing results inconsistent with type I to IV OI
Abnormalities of the following per central laboratory reference ranges at screening: Serum albumin corrected calcium < lower limit of normal (LLN) Serum vitamin D < 20 ng/mL; re-screening for Vitamin D level < 20 ng/mL will be allowed, after adequate supplementation
Aspartate aminotransferase (AST), alanine aminotransferase (ALT) > 1.5 x upper limit of normal (ULN)
Total bilirubin (TBL) > 1.5 x ULN (subjects with Gilbert syndrome are eligible)
Serum phosphorus < LLN
Serum alkaline phosphatase > 20% above the ULN or > 20% below the LLN
Estimated glomerular filtration rate (eGFR) < 60 mL/min/1.73 m2 (calculated bythe Schwartz equation at screening) Evidence of any of the following: Current hyperthyroidism (unless well-controlled on stable antithyroid therapy)
Current clinical hypothyroidism (unless well-controlled on stable thyroid replacement therapy)
History of hyperparathyroidism
Current hypoparathyroidism
Current, uncontrolled hypercalcemia (albumin-corrected serum Ca >10% ULN)
History of osteomalacia or rickets (chart review)
Other bone diseases that affect bone metabolism (eg, osteoporosis pseudoglioma syndrome, idiopathic juvenile osteoporosis, osteopetrosis, hypophosphatasia)
History of autoimmune disease
History of rare hereditary problems of fructose intolerance
Positive blood screen for human immunodeficiency virus -1 or -2 antibody
Positive blood screen for hepatitis B surface antigen or hepatitis C antibody
Received other osteoporosis treatment or bone active treatment with the following guidelines:
Prior treatment with
- denosumab
- fluoride or strontium for bone disease (fluoride taken for routine dental care is permitted)
- parathyroid hormone (PTH) or PTH derivatives within 12 months prior to screening
- zoledronic acid within 6 months prior to screening
- oral bisphosphonates or intravenous bisphosphonates other than zoledronic acid if the first dose of denosumab would be before their next scheduled bisphosphonate dose would have been given
Administration of systemic glucocorticoids (≥ 5.0 mg prednisone equivalents/day for more than 10 days) within 3 months of screening.
Topical and inhaled glucocorticoids will be allowed
Administration of any of the following treatment within 3 months of screening:
- Growth hormone (subjects on stable dose of growth hormone for at least 3 months prior to screening will be allowed)
Currently receiving treatment in another investigational drug study, or less than 30 days since ending treatment on another investigational drugstudy(s), or current or planned participation in a clinical trial that would preclude compliance with study requirements Other inclusion/exclusion criteria may apply.

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Denosumab

Arm Description

Participants received denosumab 1 mg/kg (up to a maximum of 60 mg) subcutaneously every 6 months (Q6M) for up to 36 months. Early efficacy and PK data from Q6M dosing supported adjustment of the dosing regimen from Q6M to every 3 months (Q3M). Participants enrolled and still receiving denosumab were transitioned from Q6M to Q3M dosing schedule. Participants could transition to Q3M dosing schedule up to and including the date they attended for their month 36 visit under the Q6M dosing regimen. Those participants received denosumab during the Q3M dosing regimen for 12 months. Participants who transition to Q3M at month 18 of the Q6M dosing regimen received denosumab Q3M for up to 18 months.

Outcomes

Primary Outcome Measures

Change From Baseline in Lumbar Spine Bone Mineral Density (BMD) Z-Score at 12 Months

Lumbar spine BMD was measured by dual-energy X-ray absorptiometry (DXA) adjusted for age, sex, and race/ethnicity. 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.

Secondary Outcome Measures

Change From Baseline in Lumbar Spine BMD Z-score at 6 Months

Lumbar spine BMD was measured by DXA adjusted for age, sex, and race/ethnicity. 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.

Change From Baseline in Proximal Femur BMD Z-score at 6 and 12 Months

Proximal femur (total hip and femoral neck) BMD Z-score was measured by DXA adjusted for age, sex, and race/ethnicity. 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.

Percentage of Participants With at Least 1 X-ray Confirmed Long Bone or New and Worsening Vertebral Fracture

Percentage of Participants With at Least 1 X-ray Confirmed New and Worsening Vertebral Fracture

Percentage of Participants With at Least 1 X-ray Confirmed New Vertebral Fracture

Percentage of Participants Wth at Least 1 X-ray Confirmed Improving Vertebral Fracture

Percentage of Participants With at Least 1 Vertebral and Nonvertebral Fracture

Change From Baseline in Child Health Questionnaire-Parent Form Physical Summary Score (CHQ-PF-50) at 12 Months

The CHQ-PF-50 was a 50-item questionnaire completed by the parents or guardians of children between 5 and 18 years of age. The 50 questions measure 14 domains which were summarized as the physical and psychological summary scores. Each summary score was transformed and could range from 0 to 100, with higher score indicating better physical and psychosocial health. A negative change from Baseline indicates decreased well-being.

Change From Baseline in CHQ-PF-50 Psychological Summary Score at 12 Months

The CHQ-PF-50 was a 50-item questionnaire completed by the parents or guardians of children between 5 and 18 years of age. The 50 questions measure 14 domains which were summarized as the physical and psychological summary scores. Each summary score was transformed and could range from 0 to 100, with higher score indicating better physical and psychosocial health. A positive change from Baseline indicates improved well-being.

Change From Baseline in Childhood Health Assessment Questionnaire (CHAQ) Disability Index Score at 12 Months

The disability domain (questions 1-54) of the CHAQ was used to measure the participant's assessment of physical functioning or the parent's assessment of the child's physical functioning. The disability index comprised of 8 categories (dressing and grooming, arising, eating, walking, hygiene, reaching, gripping, and activities). Scoring ranged from 1 to 5; 1 was "without any difficulty," 2 was "with some difficulty," 3 was "with much difficulty," and 4 was "unable to do." An answer of "not applicable" was scored as a 5, but was not counted. If a child required assistance from another person or used an aid or other device for any of the 8 categories, the minimum score for that category was recorded as a 3. The CHAQ questions were scored and converted to a total index score ranging from 0 to 3. Negative change from Baseline indicates an improvement.

Change From Baseline in Wong-Baker Faces Pain Rating Scale (WBFPRS) at 12 Months

Participants were asked to report their level of pain by choosing a face that best described their own pain (the corresponding number: 0, 2, 4, 6, 8, 10) were then recorded. The WBFPRS ranged from 0, "no hurt," to 10, "hurts worst". A negative change from baseline indicates an improvement.

Serum Concentration of Denosumab

Serum Bone Turnover Marker (BTM) - Serum Type I Collagen C Telopeptide

BTM - Bone-specific Alkaline Phosphatase (BSAP)

Change From Baseline in Growth Velocity at 12 Months

Change from baseline in growth velocity was determined by calculating age-adjusted Z-scores for height, weight and body mass index (BMI). Height-for-age Z-score was defined as the difference between the participant's height and the median height for the population with the same age and gender, divided by the population standard deviation. The definitions of growth velocity based on weight and BMI were analogously calculated. To programmatically calculate the Z-scores, the National Center for Health Statistics percentiles growth charts, based on the 2000 Center for Disease Control and Prevention (CDC) (http://www.cdc.gov/growthcharts/c c_charts.htm), and the CDC Anthropometric Software Package 3.0 Z-scores were used. During normal growth, the change in z-score for each of the three should equal 0. A positive change in any of the three indicates growth acceleration, whereas a negative change indicates deceleration.

Full Information

NCT ID

NCT02352753

First Posted

January 28, 2015

Last Updated

November 28, 2022

Sponsor

Amgen

1. Study Identification

Unique Protocol Identification Number

NCT02352753

Brief Title

Multicenter,Single-arm Study to Evaluate Efficacy, Safety, & Pharmacokinetics of Denosumab in Children w/ OI

Acronym

Official Title

To Evaluate the Effect of Denosumab in Lumbar Spine Bone Mineral Density (BMD) Z-score at 12 Months, as Assessed by Dual-energy X-ray Absorptiometry (DXA), in Children 2 to 17 Years of Age (at the Time of Screening) on a 3-Month Dosing Regimen With OI

Study Type

Interventional

2. Study Status

Record Verification Date

November 2022

Overall Recruitment Status

Terminated

Why Stopped

The study was stopped earlier than planned due to safety concerns about high levels of calcium in the blood of the participants

Study Start Date

June 24, 2015 (Actual)

Primary Completion Date

March 26, 2022 (Actual)

Study Completion Date

March 26, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Amgen

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

This is a prospective, multicenter, single-arm study in children 2 to 17 years of age with OI to evaluate efficacy and safety of denosumab.

Detailed Description

To evaluate the effect of denosumab in lumbar spine bone mineral density (BMD) Z-score at 12 months, as assessed by dual-energy X-ray absorptiometry (DXA), in children 2 to 17 years of age (at the time of screening) on a 3-Month Dosing Regimen with osteogenesis imperfecta (OI)

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Osteogenesis Imperfecta

Keywords

Amgen, OI, Bone

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Sequential Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

153 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Denosumab

Arm Type

Experimental

Arm Description

Intervention Type

Drug

Intervention Name(s)

Denosumab

Intervention Description

Subcutaneous (SC) injection.

Primary Outcome Measure Information:

Title

Change From Baseline in Lumbar Spine Bone Mineral Density (BMD) Z-Score at 12 Months

Description

Time Frame

Baseline and 12 months

Secondary Outcome Measure Information:

Title

Change From Baseline in Lumbar Spine BMD Z-score at 6 Months

Description

Time Frame

Baseline and 6 months

Title

Change From Baseline in Proximal Femur BMD Z-score at 6 and 12 Months

Description

Time Frame

Baseline, 6 and 12 months

Title

Percentage of Participants With at Least 1 X-ray Confirmed Long Bone or New and Worsening Vertebral Fracture

Time Frame

Q6M Dosing Regimen: Last 12 months of treatment (median treatment duration was 730.0 days); Q3M Dosing Regimen: Day 1 up to 12 months

Title

Percentage of Participants With at Least 1 X-ray Confirmed New and Worsening Vertebral Fracture

Time Frame

Q6M Dosing Regimen: Last 12 months of treatment (median treatment duration was 730.0 days); Q3M Dosing Regimen: Day 1 up to 12 months

Title

Percentage of Participants With at Least 1 X-ray Confirmed New Vertebral Fracture

Time Frame

Q6M Dosing Regimen: Last 12 months of treatment (median treatment duration was 730.0 days); Q3M Dosing Regimen: Day 1 up to 12 months

Title

Percentage of Participants Wth at Least 1 X-ray Confirmed Improving Vertebral Fracture

Time Frame

Q3M Dosing Regimen: Baseline up to 12 months

Title

Percentage of Participants With at Least 1 Vertebral and Nonvertebral Fracture

Time Frame

Q6M Dosing Regimen: Last 12 months of treatment (median treatment duration was 730.0 days); Q3M Dosing Regimen: Day 1 up to 12 months

Title

Change From Baseline in Child Health Questionnaire-Parent Form Physical Summary Score (CHQ-PF-50) at 12 Months

Description

Time Frame

Baseline and 12 months

Title

Change From Baseline in CHQ-PF-50 Psychological Summary Score at 12 Months

Description

The CHQ-PF-50 was a 50-item questionnaire completed by the parents or guardians of children between 5 and 18 years of age. The 50 questions measure 14 domains which were summarized as the physical and psychological summary scores. Each summary score was transformed and could range from 0 to 100, with higher score indicating better physical and psychosocial health. A positive change from Baseline indicates improved well-being.

Time Frame

Baseline and 12 months

Title

Change From Baseline in Childhood Health Assessment Questionnaire (CHAQ) Disability Index Score at 12 Months

Description

Time Frame

Baseline and 12 months

Title

Change From Baseline in Wong-Baker Faces Pain Rating Scale (WBFPRS) at 12 Months

Description

Time Frame

Baseline and 12 months

Title

Serum Concentration of Denosumab

Time Frame

Days 1 (predose), 10, 30, and 60, & weeks 12, 24, 36, 48, 60, 72 (end of study visit), early termination visit, & follow-up visit 12 weeks after last dose (average duration of treatment: 231 days)

Title

Serum Bone Turnover Marker (BTM) - Serum Type I Collagen C Telopeptide

Time Frame

Baseline and Days 10 and 30, and Months 3, 6, 9, 12, 15 and 18

Title

BTM - Bone-specific Alkaline Phosphatase (BSAP)

Time Frame

Baseline and Days 10 and 30, and Months 3, 6, 9, 12, and 15

Title

Change From Baseline in Growth Velocity at 12 Months

Description

Time Frame

Baseline and 12 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

2 Years

Maximum Age & Unit of Time

17 Years

Accepts Healthy Volunteers

Eligibility Criteria

• Eligibility criteria relates to initial enrollment into this study (6-Month Dosing Regimen). Subjects reconsenting to a 3-Month Dosing Regimen will not repeat eligibility assessments Inclusion Criteria: • Clinical diagnosis of OI defined as a clinical history consistent with type I-IV OI Clinical severity of OI as defined by 2 or more prevalent vertebral compression fractures; OR1 prevalent vertebral compression fracture and 1 or more nonvertebral fractures within the previous 2 years; OR 3 or more fractures within the previous 2 years. Exclusion Criteria: Inability or unwillingness to comply with the requirements for frequent calcium and phosphorus monitoring for 14 days after the first dose of denosumab (only applies to the first 5 subjects age 11 to17 enrolled in the study and the first 5 subjects of any age meeting the criteria for increased bone turnover Currently unhealed fracture or osteotomy as defined by orthopedic opinion Osteotomy within 5 months of screening Evidence of untreated oral cavities or oral infections Recent or planned invasive dental procedure Surgical tooth extraction which has not healed by screening History of an electrophoresis pattern inconsistent with type I to IV OI History of genetic testing results inconsistent with type I to IV OI Abnormalities of the following per central laboratory reference ranges at screening: Serum albumin corrected calcium < lower limit of normal (LLN) Serum vitamin D < 20 ng/mL; re-screening for Vitamin D level < 20 ng/mL will be allowed, after adequate supplementation Aspartate aminotransferase (AST), alanine aminotransferase (ALT) > 1.5 x upper limit of normal (ULN) Total bilirubin (TBL) > 1.5 x ULN (subjects with Gilbert syndrome are eligible) Serum phosphorus < LLN Serum alkaline phosphatase > 20% above the ULN or > 20% below the LLN Estimated glomerular filtration rate (eGFR) < 60 mL/min/1.73 m2 (calculated bythe Schwartz equation at screening) Evidence of any of the following: Current hyperthyroidism (unless well-controlled on stable antithyroid therapy) Current clinical hypothyroidism (unless well-controlled on stable thyroid replacement therapy) History of hyperparathyroidism Current hypoparathyroidism Current, uncontrolled hypercalcemia (albumin-corrected serum Ca >10% ULN) History of osteomalacia or rickets (chart review) Other bone diseases that affect bone metabolism (eg, osteoporosis pseudoglioma syndrome, idiopathic juvenile osteoporosis, osteopetrosis, hypophosphatasia) History of autoimmune disease History of rare hereditary problems of fructose intolerance Positive blood screen for human immunodeficiency virus -1 or -2 antibody Positive blood screen for hepatitis B surface antigen or hepatitis C antibody Received other osteoporosis treatment or bone active treatment with the following guidelines: Prior treatment with denosumab fluoride or strontium for bone disease (fluoride taken for routine dental care is permitted) parathyroid hormone (PTH) or PTH derivatives within 12 months prior to screening zoledronic acid within 6 months prior to screening oral bisphosphonates or intravenous bisphosphonates other than zoledronic acid if the first dose of denosumab would be before their next scheduled bisphosphonate dose would have been given Administration of systemic glucocorticoids (≥ 5.0 mg prednisone equivalents/day for more than 10 days) within 3 months of screening. Topical and inhaled glucocorticoids will be allowed Administration of any of the following treatment within 3 months of screening: Growth hormone (subjects on stable dose of growth hormone for at least 3 months prior to screening will be allowed) Currently receiving treatment in another investigational drug study, or less than 30 days since ending treatment on another investigational drugstudy(s), or current or planned participation in a clinical trial that would preclude compliance with study requirements Other inclusion/exclusion criteria may apply.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Organizational Affiliation

Amgen

Official's Role

Study Director

Facility Information:

Facility Name

Research Site

City

Los Angeles

State/Province

California

ZIP/Postal Code

90027

Country

United States

Facility Name

Research Site

City

Torrance

State/Province

California

ZIP/Postal Code

90502

Country

United States

Facility Name

Research Site

City

Aurora

State/Province

Colorado

ZIP/Postal Code

80045

Country

United States

Facility Name

Research Site

City

New Haven

State/Province

Connecticut

ZIP/Postal Code

06510

Country

United States

Facility Name

Research Site

City

Wilmington

State/Province

Delaware

ZIP/Postal Code

19803

Country

United States

Facility Name

Research Site

City

Decatur

State/Province

Georgia

ZIP/Postal Code

30033

Country

United States

Facility Name

Research Site

City

Indianapolis

State/Province

Indiana

ZIP/Postal Code

46202

Country

United States

Facility Name

Research Site

City

Iowa City

State/Province

Iowa

ZIP/Postal Code

52242

Country

United States

Facility Name

Research Site

City

Baltimore

State/Province

Maryland

ZIP/Postal Code

21205

Country

United States

Facility Name

Research Site

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02115

Country

United States

Facility Name

Research Site

City

Omaha

State/Province

Nebraska

ZIP/Postal Code

68114

Country

United States

Facility Name

Research Site

City

Nashville

State/Province

Tennessee

ZIP/Postal Code

37232

Country

United States

Facility Name

Research Site

City

Houston

State/Province

Texas

ZIP/Postal Code

77030

Country

United States

Facility Name

Research Site

City

Westmead

State/Province

New South Wales

ZIP/Postal Code

2145

Country

Australia

Facility Name

Research Site

City

Subiaco

State/Province

Western Australia

ZIP/Postal Code

6008

Country

Australia

Facility Name

Research Site

City

Bruxelles

ZIP/Postal Code

1200

Country

Belgium

Facility Name

Research Site

City

Gent

ZIP/Postal Code

9000

Country

Belgium

Facility Name

Research Site

City

Leuven

ZIP/Postal Code

3000

Country

Belgium

Facility Name

Research Site

City

Sofia

ZIP/Postal Code

1606

Country

Bulgaria

Facility Name

Research Site

City

Sofia

ZIP/Postal Code

1784

Country

Bulgaria

Facility Name

Research Site

City

Varna

ZIP/Postal Code

9010

Country

Bulgaria

Facility Name

Research Site

City

Ottawa

State/Province

Ontario

ZIP/Postal Code

K1H 8L1

Country

Canada

Facility Name

Research Site

City

Toronto

State/Province

Ontario

ZIP/Postal Code

M5G 1X8

Country

Canada

Facility Name

Research Site

City

Montreal

State/Province

Quebec

ZIP/Postal Code

H4A 0A9

Country

Canada

Facility Name

Research Site

City

Hradec Kralove

ZIP/Postal Code

500 05

Country

Czechia

Facility Name

Research Site

City

Pardubice

ZIP/Postal Code

532 03

Country

Czechia

Facility Name

Research Site

City

Plzen

ZIP/Postal Code

305 99

Country

Czechia

Facility Name

Research Site

City

Praha 4

ZIP/Postal Code

140 59

Country

Czechia

Facility Name

Research Site

City

Zlin

ZIP/Postal Code

762 75

Country

Czechia

Facility Name

Research Site

City

Bordeaux Cedex

ZIP/Postal Code

33076

Country

France

Facility Name

Research Site

City

Paris Cedex 15

ZIP/Postal Code

75743

Country

France

Facility Name

Research Site

City

Paris

ZIP/Postal Code

75012

Country

France

Facility Name

Research Site

City

Saint Priest en Jarez

ZIP/Postal Code

42270

Country

France

Facility Name

Research Site

City

Toulouse Cedex 9

ZIP/Postal Code

31059

Country

France

Facility Name

Research Site

City

Köln

ZIP/Postal Code

50931

Country

Germany

Facility Name

Research Site

City

Budapest

ZIP/Postal Code

1094

Country

Hungary

Facility Name

Research Site

City

Roma

ZIP/Postal Code

00161

Country

Italy

Facility Name

Research Site

City

Verona

ZIP/Postal Code

37126

Country

Italy

Facility Name

Research Site

City

Bialystok

ZIP/Postal Code

15-274

Country

Poland

Facility Name

Research Site

City

Lodz

ZIP/Postal Code

91-738

Country

Poland

Facility Name

Research Site

City

Rzeszow

ZIP/Postal Code

35-301

Country

Poland

Facility Name

Research Site

City

Warszawa

ZIP/Postal Code

04-730

Country

Poland

Facility Name

Research Site

City

Esplugues de Llobregat

State/Province

Cataluña

ZIP/Postal Code

08950

Country

Spain

Facility Name

Research Site

City

Valencia

State/Province

Comunidad Valenciana

ZIP/Postal Code

46026

Country

Spain

Facility Name

Research Site

City

Getafe

State/Province

Madrid

ZIP/Postal Code

28905

Country

Spain

Facility Name

Research Site

City

Birmingham

ZIP/Postal Code

B4 6NH

Country

United Kingdom

Facility Name

Research Site

City

Bristol

ZIP/Postal Code

BS2 8AE

Country

United Kingdom

Facility Name

Research Site

City

Glasgow

ZIP/Postal Code

G51 4TF

Country

United Kingdom

Facility Name

Research Site

City

Sheffield

ZIP/Postal Code

S10 2TH

Country

United Kingdom

12. IPD Sharing Statement

Plan to Share IPD

Yes

IPD Sharing Plan Description

De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request

IPD Sharing Time Frame

Data sharing requests relating to this study will be considered beginning 18 months after the study has ended and either 1) the product and indication (or other new use) have been granted marketing authorization in both the US and Europe or 2) clinical development for the product and/or indication discontinues and the data will not be submitted to regulatory authorities. There is no end date for eligibility to submit a data sharing request for this study.

IPD Sharing Access Criteria

Qualified researchers may submit a request containing the research objectives, the Amgen product(s) and Amgen study/studies in scope, endpoints/outcomes of interest, statistical analysis plan, data requirements, publication plan, and qualifications of the researcher(s). In general, Amgen does not grant external requests for individual patient data for the purpose of re-evaluating safety and efficacy issues already addressed in the product labelling. Requests are reviewed by a committee of internal advisors, and if not approved, may be further arbitrated by a Data Sharing Independent Review Panel. Upon approval, information necessary to address the research question will be provided under the terms of a data sharing agreement. This may include anonymized individual patient data and/or available supporting documents, containing fragments of analysis code where provided in analysis specifications. Further details are available at the URL below.

IPD Sharing URL

https://www.amgen.com/datasharing

Links:

URL

http://www.amgentrials.com

Description

AmgenTrials clinical trials website

Learn more about this trial

Multicenter,Single-arm Study to Evaluate Efficacy, Safety, & Pharmacokinetics of Denosumab in Children w/ OI

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Multicenter,Single-arm Study to Evaluate Efficacy, Safety, & Pharmacokinetics of Denosumab in Children w/ OI (OI)

Osteogenesis Imperfecta

About this trial

Eligibility Criteria

Sites / Locations

Arms of the Study

Arm 1

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

1. Study Identification

2. Study Status

3. Sponsor/Collaborators

4. Oversight

5. Study Description

6. Conditions and Keywords

7. Study Design

8. Arms, Groups, and Interventions

10. Eligibility

12. IPD Sharing Statement

Learn more about this trial