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A Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804 (Asteroid)

Primary Purpose

Osteogenesis Imperfecta, Type I, Osteogenesis Imperfecta Type III, Osteogenesis Imperfecta Type IV

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
setrusumab
Calcium
Vitamin D
zoledronic acid (optional)
Sponsored by
Ultragenyx Pharmaceutical Inc
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Osteogenesis Imperfecta, Type I focused on measuring Osteogenesis Imperfecta, Brittle Bone Disease

Eligibility Criteria

18 Years - 75 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Patients with a clinical diagnosis of OI Type I, III or IV with a confirmed defect in the COL1A1/COL1A2 genes, as confirmed by genetic testing
  • One or more fractures in the past 5 years
  • Capable of giving signed consent

Exclusion Criteria:

  • History of skeletal malignancies or other bone diseases (other than OI)
  • History of neural foraminal stenosis (except if due to scoliosis)
  • History of myocardial infarction, angina pectoris, ischaemic stroke or transient ischaemic attack
  • History of endocrine or thyroid/parathyroid conditions that could affect bone metabolism
  • Treatment with bisphosphonates within 3 months of randomisation
  • Treatment with teraparatide, denosumab or other anabolic/anti-reabsorptive medications within 6 months of randomisation

Sites / Locations

  • Mereo Investigator Site
  • Mereo Investigator Site
  • Mereo Investigator Site
  • Mereo Investigator Site
  • Mereo Investigator Site
  • Mereo Investigator Site
  • Mereo Investigator Site
  • Mereo Investigator Site
  • Mereo Investigator Site
  • Mereo Investigator Site
  • Mereo Investigator Site
  • Mereo Investigator Site
  • Mereo Investigator Site
  • Mereo Investigator Site
  • Mereo Investigator Site
  • Mereo Investigator Site
  • Mereo Investigator Site
  • Mereo Investigator Site
  • Mereo Investigator Site
  • Mereo Investigator Site
  • Mereo Investigator Site
  • Mereo Investigator Site
  • Mereo Investigator Site
  • Mereo Investigator Site
  • Mereo Investigator Site

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm Type

Experimental

Experimental

Experimental

Experimental

Placebo Comparator

Arm Label

Setrusumab 20 mg/kg (Blinded)

Setrusumab 8 mg/kg (Blinded)

Setrusumab 2 mg/kg (Blinded)

Setrusumab 20 mg/kg (Open-Label)

Placebo

Arm Description

Setrusumab 20 mg/kg intravenous (IV) infusion once a month for 12 months plus 500 mg calcium oral tablets and 800 IU vitamin D capsules.

Setrusumab 8 mg/kg IV infusion once a month for 12 months plus 500 mg calcium oral tablets and 800 IU vitamin D capsules.

Setrusumab 2 mg/kg IV infusion once a month for 12 months plus 500 mg calcium oral tablets and 800 IU vitamin D capsules.

Setrusumab 20 mg/kg IV infusion once a month for 12 months plus 500 mg calcium oral tablets and 800 IU vitamin D capsules.

Placebo IV infusion once a month for 12 months plus 500 mg calcium oral tablets and 800 IU vitamin D capsules.

Outcomes

Primary Outcome Measures

Change From Baseline in Radial Trabecular Volumetric Bone Mineral Density (Tr vBMD) at Month 12
Assessed by high resolution peripheral quantitative computed tomography (HRpQCT). HRpQCT scans were performed on the participant's distal non-dominant arm. In cases of an arm that had been supported with rods or had significant deformity, the dominant limb was selected. Data presents the ratio of the means between the visit and Baseline from analysis of covariance (ANCOVA).
Change From Baseline in Radial Bone Strength (Failure Load) at Month 12
Assessed by finite element analysis (FEA) of models generated from HRpQCT images of the distal radius.
Change From Baseline in Radial Bone Strength (Stiffness) at Month 12
Assessed by FEA of models generated from HRpQCT images of the distal radius.

Secondary Outcome Measures

Change From Baseline in Radial and Tibial Tr VBMD Over Time: Full Analysis Set
Assessed by HRpQCT. HRpQCT scans were performed on the participant's distal non-dominant arm. In cases of an arm that had been supported with rods or had significant deformity, the dominant limb was selected. Data presented is the ratio of the means between the Visit and Baseline from ANCOVA.
Changes From Baseline in Radial and Tibial Tr VBMD at Months 6 and 12: Open-Label Arm
Assessed by HRpQCT. HRpQCT scans were performed on the participant's distal non-dominant arm. In cases of an arm that had been supported with rods or had significant deformity, the dominant limb was selected. Data presented is the ratio of the means between the Visit and Baseline from ANCOVA.
Changes From Baseline in Radial and Tibial Bone Strength (Failure Load) Over Time: Full Analysis Set
Assessed by FEA of models generated from HRpQCT images of the distal radius.
Changes From Baseline in Radial and Tibial Bone Strength (Failure Load) at Months 6 and 12: Open-Label Arm
Assessed by FEA of models generated from HRpQCT images of the distal radius.
Changes From Baseline in Radial and Tibial Bone Strength (Stiffness) Over Time: Full Analysis Set
Assessed by FEA of models generated from HRpQCT images of the distal radius.
Changes From Baseline in Radial and Tibial Bone Strength (Stiffness) at Months 6 and 12: Open-Label Arm
Assessed by FEA of models generated from HRpQCT images of the distal radius.
Percentage of Participants With at Least 1 New Fracture (Peripheral, Vertebral, Long-Bone, Any) at Month 12
Fracture assessment, confirmed by central radiographic reading, was carried out for peripheral including all major long bones, minor bone (digits, ribs) and vertebral fractures. Fractures without clinical symptoms, detected only by means of radiographic investigations, were not included in the analysis.
Change From Baseline in Lumbar, Total Body, and Femoral Neck Bone Mineral Density (BMD) T-score at Month 6
BMD was evaluated by dual-energy x-ray absorptiometry (DXA). T-Score was calculated based on actual measured bone density value. T-scores are standardized scores that reflect the standard deviations (SDs) above/below the normal mean for young adults. A score of 50 indicates the population mean with a standard deviation of 10. A positive change in DXA T-score indicates an improvement in BMD.
Change From Baseline in Lumbar, Total Body, and Femoral Neck BMD at Month 6
BMD was evaluated by DXA.
Change From Baseline in Lumbar, Total Body, and Femoral Neck BMD T-score at Month 12
BMD was evaluated by DXA. T-Score was calculated based on actual measured bone density value. T-scores are standardized scores that reflect the standard deviations (SDs) above/below the normal mean for young adults. A score of 50 indicates the population mean with a standard deviation of 10. A positive change in DXA T-score indicates an improvement in BMD.
Change From Baseline in Lumbar, Total Body, and Femoral Neck BMD at Month 12
BMD was evaluated by DXA.
Change From Baseline in Total vBMD (Radial and Tibial) Over Time
Assessed by HRpQCT. HRpQCT scans were performed on the participant's distal non-dominant arm. In cases of an arm that had been supported with rods or had significant deformity, the dominant limb was selected. Data presented is the ratio of the means between the Visit and Baseline from ANCOVA.
Change From Baseline in Cortical vBMD (Radial and Tibial) Over Time
Assessed by HRpQCT. HRpQCT scans were performed on the participant's distal non-dominant arm. In cases of an arm that had been supported with rods or had significant deformity, the dominant limb was selected. Data presented is the ratio of the means between the Visit and Baseline from ANCOVA.
Number of Participants With Clinically Significant Changes From Baseline in Body Height, Weight and Body Mass Index (BMI) at 6 and 12 Months: Full Analysis Set
Change From Baseline in Lean and Fat Body Mass From Whole Body at Months 6 and 12
Lean and fat body mass was evaluated using whole body DXA (including the head).
Change From Baseline in Amino-Terminal Propeptide of Type 1 Procollagen (P1NP) up to Month 12
Change From Baseline in Carboxy-Terminal Telo-Peptide [CTX-1] up to Month 12
Change From Baseline in Short Form 12 Health Survey (SF-12) Physical Component Summary Score at Months 6 and 12
The SF-12 is a generic, 12-item survey that measures 8 domains of health: physical functioning, role limitations due to physical health, bodily pain, general health perceptions, vitality, social functioning, role limitations due to emotional problems, and mental health. It yields scale scores for each of these 8 domains and 2 summary measures of physical and mental health: The Physical Component Summary and the Mental Component Summary. The total score for the Physical Component Summary ranges from 0 to 100, where higher scores reflect better physical functioning.
Change From Baseline in SF-12 Mental Component Summary Score at Months 6 and 12
The SF-12 is a generic, 12-item survey that measures 8 domains of health: physical functioning, role limitations due to physical health, bodily pain, general health perceptions, vitality, social functioning, role limitations due to emotional problems, and mental health. It yields scale scores for each of these 8 domains and 2 summary measures of physical and mental health: The Physical Component Summary and the Mental Component Summary. The total score for the Mental Component Summary ranges from 0 to 100, where higher scores reflect better mental health functioning.
Change From Baseline in Index (Utility) Score on EuroQol 5-Dimension 5-Level Descriptive System (EQ-5D-5L) Score at Months 6 and 12
The EQ-5D-5L is a standardised measure of health status comprised of a descriptive system of 5 health-related quality of life states (i.e., mobility, self-care, usual activities, pain/discomfort, and anxiety/depression) and a Visual Analogue Scale (VAS) of overall health. Each dimension is rated on a 5-point response scale indicating severity of problems, where 1 is "no problems" and 5 is "extreme problems". The 5 questions are scored and together contribute to the EQ-5D index (utility) score between 0 and 1 (1 being perfect health).
Change From Baseline in Osteogenesis Imperfecta Specific Quality of Life Questionnaire for Adults (OIQoL-A) Total Score at Months 6 and 12
The OIQoL-A measures 5 areas of quality of life related to OI (Physical Function, Pain, Hearing Loss, Taking Care/Concerns, Social and Family Life and Activities). The total score is calculated on a 0-100 scale, where higher scores indicate a greater (negative) impact on quality of life.
Change From Baseline in OIQoL-A Pain Subscale Score at Months 6 and 12
The OIQoL-A measures 5 areas of quality of life related to OI (Physical Function, Pain, Hearing Loss, Taking Care/Concerns, Social and Family Life and Activities). The Pain subscale ranges from 0 to 10, with higher value representing worse pain.
Change From Baseline in OIQoL-A Activity Subscale Score at Months 6 and 12
The OIQoL-A measures 5 areas of quality of life related to OI (Physical Function, Pain, Hearing Loss, Taking Care/Concerns, Social and Family Life and Activities). The Activities subscale ranges from 0 to 100, with higher value representing increased difficulty.
Percentage of Participants Who Were Positive for Anti-Setrusumab Antibodies at Any Time During the Study up to Month 14
Serum samples were screened for antibodies binding to setrusumab using a validated assay method by or under the supervision of the sponsor.
Percentage of Participants With Adverse Events (AEs), Treatment-Emergent AEs (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation or Death
An AE is any untoward medical occurrence, which does not necessarily have a causal relationship with treatment. A serious AE (SAE) is defined as any untoward medical occurrence that, at any dose: results in death; is life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent disability/incapacity; is a congenital anomaly/birth defect; is another important medical event. The intensity for each AE was graded as mild, moderate or severe, according to the investigator's judgement. An event was considered related to study drug if there were a "reasonable possibility" of a relationship, according to the investigator's clinical judgment. A TEAE was defined as an event occurring or worsening on or after the first dose of study medication.

Full Information

First Posted
April 3, 2017
Last Updated
June 29, 2023
Sponsor
Ultragenyx Pharmaceutical Inc
Collaborators
Mereo BioPharma
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1. Study Identification

Unique Protocol Identification Number
NCT03118570
Brief Title
A Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804
Acronym
Asteroid
Official Title
A Phase 2b, Multicentre, Multinational, Double-blind, Dose-finding Study, Incorporating an Open Label Substudy, in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With Setrusumab (BPS804)
Study Type
Interventional

2. Study Status

Record Verification Date
February 2022
Overall Recruitment Status
Completed
Study Start Date
September 11, 2017 (Actual)
Primary Completion Date
October 1, 2019 (Actual)
Study Completion Date
November 12, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Ultragenyx Pharmaceutical Inc
Collaborators
Mereo BioPharma

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The purpose of this study is to select a suitable dose of BPS804 by measuring the strength/quality of bone using a special type of CT scanner. Participants will be treated for 12 months and followed up for a further 12 months.
Detailed Description
This study was previously posted by Mereo Biopharma and was transferred to Ultragenyx in February 2021.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Osteogenesis Imperfecta, Type I, Osteogenesis Imperfecta Type III, Osteogenesis Imperfecta Type IV
Keywords
Osteogenesis Imperfecta, Brittle Bone Disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Model Description
Double-blind, Dose-finding Study, incorporating an open-label substudy
Masking
ParticipantInvestigator
Masking Description
Sponsor will be masked until the primary analysis of the study except for open-label substudy treatment arm. The study site pharmacist will be unmasked to treatment allocation throughout. Study treatment will be monitored by a separate unmasked monitoring team.
Allocation
Randomized
Enrollment
112 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Setrusumab 20 mg/kg (Blinded)
Arm Type
Experimental
Arm Description
Setrusumab 20 mg/kg intravenous (IV) infusion once a month for 12 months plus 500 mg calcium oral tablets and 800 IU vitamin D capsules.
Arm Title
Setrusumab 8 mg/kg (Blinded)
Arm Type
Experimental
Arm Description
Setrusumab 8 mg/kg IV infusion once a month for 12 months plus 500 mg calcium oral tablets and 800 IU vitamin D capsules.
Arm Title
Setrusumab 2 mg/kg (Blinded)
Arm Type
Experimental
Arm Description
Setrusumab 2 mg/kg IV infusion once a month for 12 months plus 500 mg calcium oral tablets and 800 IU vitamin D capsules.
Arm Title
Setrusumab 20 mg/kg (Open-Label)
Arm Type
Experimental
Arm Description
Setrusumab 20 mg/kg IV infusion once a month for 12 months plus 500 mg calcium oral tablets and 800 IU vitamin D capsules.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo IV infusion once a month for 12 months plus 500 mg calcium oral tablets and 800 IU vitamin D capsules.
Intervention Type
Drug
Intervention Name(s)
setrusumab
Other Intervention Name(s)
BPS804
Intervention Description
Intravenous infusion
Intervention Type
Dietary Supplement
Intervention Name(s)
Calcium
Intervention Description
tablets
Intervention Type
Dietary Supplement
Intervention Name(s)
Vitamin D
Intervention Description
capsules
Intervention Type
Drug
Intervention Name(s)
zoledronic acid (optional)
Intervention Description
Following completion of the study treatment (Month 12) participants can receive an optional single dose of zoledronic acid. Participants can receive an optional further dose of zoledronic acid at Month 18 at the discretion of their treating physician.
Primary Outcome Measure Information:
Title
Change From Baseline in Radial Trabecular Volumetric Bone Mineral Density (Tr vBMD) at Month 12
Description
Assessed by high resolution peripheral quantitative computed tomography (HRpQCT). HRpQCT scans were performed on the participant's distal non-dominant arm. In cases of an arm that had been supported with rods or had significant deformity, the dominant limb was selected. Data presents the ratio of the means between the visit and Baseline from analysis of covariance (ANCOVA).
Time Frame
Baseline, Month 12 (end of treatment [EOT])
Title
Change From Baseline in Radial Bone Strength (Failure Load) at Month 12
Description
Assessed by finite element analysis (FEA) of models generated from HRpQCT images of the distal radius.
Time Frame
Baseline, Month 12 (EOT)
Title
Change From Baseline in Radial Bone Strength (Stiffness) at Month 12
Description
Assessed by FEA of models generated from HRpQCT images of the distal radius.
Time Frame
Baseline, Month 12 (EOT)
Secondary Outcome Measure Information:
Title
Change From Baseline in Radial and Tibial Tr VBMD Over Time: Full Analysis Set
Description
Assessed by HRpQCT. HRpQCT scans were performed on the participant's distal non-dominant arm. In cases of an arm that had been supported with rods or had significant deformity, the dominant limb was selected. Data presented is the ratio of the means between the Visit and Baseline from ANCOVA.
Time Frame
Baseline, Months 6, 12 (EOT), 18, 24
Title
Changes From Baseline in Radial and Tibial Tr VBMD at Months 6 and 12: Open-Label Arm
Description
Assessed by HRpQCT. HRpQCT scans were performed on the participant's distal non-dominant arm. In cases of an arm that had been supported with rods or had significant deformity, the dominant limb was selected. Data presented is the ratio of the means between the Visit and Baseline from ANCOVA.
Time Frame
Baseline, Months 6, 12 (EOT)
Title
Changes From Baseline in Radial and Tibial Bone Strength (Failure Load) Over Time: Full Analysis Set
Description
Assessed by FEA of models generated from HRpQCT images of the distal radius.
Time Frame
Baseline, Months 6, 12 (EOT), 18, 24
Title
Changes From Baseline in Radial and Tibial Bone Strength (Failure Load) at Months 6 and 12: Open-Label Arm
Description
Assessed by FEA of models generated from HRpQCT images of the distal radius.
Time Frame
Baseline, Months 6, 12 (EOT)
Title
Changes From Baseline in Radial and Tibial Bone Strength (Stiffness) Over Time: Full Analysis Set
Description
Assessed by FEA of models generated from HRpQCT images of the distal radius.
Time Frame
Baseline, Months 6, 12 (EOT), 18, 24
Title
Changes From Baseline in Radial and Tibial Bone Strength (Stiffness) at Months 6 and 12: Open-Label Arm
Description
Assessed by FEA of models generated from HRpQCT images of the distal radius.
Time Frame
Baseline, Months 6, 12 (EOT)
Title
Percentage of Participants With at Least 1 New Fracture (Peripheral, Vertebral, Long-Bone, Any) at Month 12
Description
Fracture assessment, confirmed by central radiographic reading, was carried out for peripheral including all major long bones, minor bone (digits, ribs) and vertebral fractures. Fractures without clinical symptoms, detected only by means of radiographic investigations, were not included in the analysis.
Time Frame
Month 12 (EOT)
Title
Change From Baseline in Lumbar, Total Body, and Femoral Neck Bone Mineral Density (BMD) T-score at Month 6
Description
BMD was evaluated by dual-energy x-ray absorptiometry (DXA). T-Score was calculated based on actual measured bone density value. T-scores are standardized scores that reflect the standard deviations (SDs) above/below the normal mean for young adults. A score of 50 indicates the population mean with a standard deviation of 10. A positive change in DXA T-score indicates an improvement in BMD.
Time Frame
Baseline, Month 6
Title
Change From Baseline in Lumbar, Total Body, and Femoral Neck BMD at Month 6
Description
BMD was evaluated by DXA.
Time Frame
Baseline, Month 6
Title
Change From Baseline in Lumbar, Total Body, and Femoral Neck BMD T-score at Month 12
Description
BMD was evaluated by DXA. T-Score was calculated based on actual measured bone density value. T-scores are standardized scores that reflect the standard deviations (SDs) above/below the normal mean for young adults. A score of 50 indicates the population mean with a standard deviation of 10. A positive change in DXA T-score indicates an improvement in BMD.
Time Frame
Baseline, Month 12 (EOT)
Title
Change From Baseline in Lumbar, Total Body, and Femoral Neck BMD at Month 12
Description
BMD was evaluated by DXA.
Time Frame
Baseline, Month 12 (EOT)
Title
Change From Baseline in Total vBMD (Radial and Tibial) Over Time
Description
Assessed by HRpQCT. HRpQCT scans were performed on the participant's distal non-dominant arm. In cases of an arm that had been supported with rods or had significant deformity, the dominant limb was selected. Data presented is the ratio of the means between the Visit and Baseline from ANCOVA.
Time Frame
Baseline, Months 6, 12 (EOT), 18, and 24
Title
Change From Baseline in Cortical vBMD (Radial and Tibial) Over Time
Description
Assessed by HRpQCT. HRpQCT scans were performed on the participant's distal non-dominant arm. In cases of an arm that had been supported with rods or had significant deformity, the dominant limb was selected. Data presented is the ratio of the means between the Visit and Baseline from ANCOVA.
Time Frame
Baseline, Months 6, 12 (EOT), 18, and 24
Title
Number of Participants With Clinically Significant Changes From Baseline in Body Height, Weight and Body Mass Index (BMI) at 6 and 12 Months: Full Analysis Set
Time Frame
Baseline, Month 6, Month 12 (EOT)
Title
Change From Baseline in Lean and Fat Body Mass From Whole Body at Months 6 and 12
Description
Lean and fat body mass was evaluated using whole body DXA (including the head).
Time Frame
Baseline, Months 6, 12 (EOT)
Title
Change From Baseline in Amino-Terminal Propeptide of Type 1 Procollagen (P1NP) up to Month 12
Time Frame
Baseline, Months 1, 3, 6, 9, 12 (EOT)
Title
Change From Baseline in Carboxy-Terminal Telo-Peptide [CTX-1] up to Month 12
Time Frame
Baseline, Months 1, 3, 6, 9, 12 (EOT)
Title
Change From Baseline in Short Form 12 Health Survey (SF-12) Physical Component Summary Score at Months 6 and 12
Description
The SF-12 is a generic, 12-item survey that measures 8 domains of health: physical functioning, role limitations due to physical health, bodily pain, general health perceptions, vitality, social functioning, role limitations due to emotional problems, and mental health. It yields scale scores for each of these 8 domains and 2 summary measures of physical and mental health: The Physical Component Summary and the Mental Component Summary. The total score for the Physical Component Summary ranges from 0 to 100, where higher scores reflect better physical functioning.
Time Frame
Baseline, Months 6, 12 (EOT)
Title
Change From Baseline in SF-12 Mental Component Summary Score at Months 6 and 12
Description
The SF-12 is a generic, 12-item survey that measures 8 domains of health: physical functioning, role limitations due to physical health, bodily pain, general health perceptions, vitality, social functioning, role limitations due to emotional problems, and mental health. It yields scale scores for each of these 8 domains and 2 summary measures of physical and mental health: The Physical Component Summary and the Mental Component Summary. The total score for the Mental Component Summary ranges from 0 to 100, where higher scores reflect better mental health functioning.
Time Frame
Baseline, Months 6, 12 (EOT)
Title
Change From Baseline in Index (Utility) Score on EuroQol 5-Dimension 5-Level Descriptive System (EQ-5D-5L) Score at Months 6 and 12
Description
The EQ-5D-5L is a standardised measure of health status comprised of a descriptive system of 5 health-related quality of life states (i.e., mobility, self-care, usual activities, pain/discomfort, and anxiety/depression) and a Visual Analogue Scale (VAS) of overall health. Each dimension is rated on a 5-point response scale indicating severity of problems, where 1 is "no problems" and 5 is "extreme problems". The 5 questions are scored and together contribute to the EQ-5D index (utility) score between 0 and 1 (1 being perfect health).
Time Frame
Baseline, Months 6 and 12 (EOT)
Title
Change From Baseline in Osteogenesis Imperfecta Specific Quality of Life Questionnaire for Adults (OIQoL-A) Total Score at Months 6 and 12
Description
The OIQoL-A measures 5 areas of quality of life related to OI (Physical Function, Pain, Hearing Loss, Taking Care/Concerns, Social and Family Life and Activities). The total score is calculated on a 0-100 scale, where higher scores indicate a greater (negative) impact on quality of life.
Time Frame
Baseline, Months 6, 12 (EOT)
Title
Change From Baseline in OIQoL-A Pain Subscale Score at Months 6 and 12
Description
The OIQoL-A measures 5 areas of quality of life related to OI (Physical Function, Pain, Hearing Loss, Taking Care/Concerns, Social and Family Life and Activities). The Pain subscale ranges from 0 to 10, with higher value representing worse pain.
Time Frame
Baseline, Months 6, 12 (EOT)
Title
Change From Baseline in OIQoL-A Activity Subscale Score at Months 6 and 12
Description
The OIQoL-A measures 5 areas of quality of life related to OI (Physical Function, Pain, Hearing Loss, Taking Care/Concerns, Social and Family Life and Activities). The Activities subscale ranges from 0 to 100, with higher value representing increased difficulty.
Time Frame
Baseline, Months 6, 12 (EOT)
Title
Percentage of Participants Who Were Positive for Anti-Setrusumab Antibodies at Any Time During the Study up to Month 14
Description
Serum samples were screened for antibodies binding to setrusumab using a validated assay method by or under the supervision of the sponsor.
Time Frame
up to Month 14
Title
Percentage of Participants With Adverse Events (AEs), Treatment-Emergent AEs (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation or Death
Description
An AE is any untoward medical occurrence, which does not necessarily have a causal relationship with treatment. A serious AE (SAE) is defined as any untoward medical occurrence that, at any dose: results in death; is life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent disability/incapacity; is a congenital anomaly/birth defect; is another important medical event. The intensity for each AE was graded as mild, moderate or severe, according to the investigator's judgement. An event was considered related to study drug if there were a "reasonable possibility" of a relationship, according to the investigator's clinical judgment. A TEAE was defined as an event occurring or worsening on or after the first dose of study medication.
Time Frame
Non-serious AEs: up to Month 14; Serious AEs: up to Month 24. (Average duration of exposure to placebo was 5 months and for setrusumab was 11 month plus follow-up to 24 months.)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Patients with a clinical diagnosis of OI Type I, III or IV with a confirmed defect in the COL1A1/COL1A2 genes, as confirmed by genetic testing One or more fractures in the past 5 years Capable of giving signed consent Exclusion Criteria: History of skeletal malignancies or other bone diseases (other than OI) History of neural foraminal stenosis (except if due to scoliosis) History of myocardial infarction, angina pectoris, ischaemic stroke or transient ischaemic attack History of endocrine or thyroid/parathyroid conditions that could affect bone metabolism Treatment with bisphosphonates within 3 months of randomisation Treatment with teraparatide, denosumab or other anabolic/anti-reabsorptive medications within 6 months of randomisation
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director
Organizational Affiliation
Mereo BioPharma
Official's Role
Study Director
Facility Information:
Facility Name
Mereo Investigator Site
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35294
Country
United States
Facility Name
Mereo Investigator Site
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32207
Country
United States
Facility Name
Mereo Investigator Site
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Facility Name
Mereo Investigator Site
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
012115
Country
United States
Facility Name
Mereo Investigator Site
City
Saint Paul
State/Province
Minnesota
ZIP/Postal Code
55101
Country
United States
Facility Name
Mereo Investigator Site
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Mereo Investigator Site
City
Albuquerque
State/Province
New Mexico
ZIP/Postal Code
87106
Country
United States
Facility Name
Mereo Investigator Site
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Mereo Investigator Site
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Mereo Investigator Site
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15225
Country
United States
Facility Name
Mereo Investigator Site
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
Mereo Investigator Site
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Mereo Investigator Site
City
Toronto
State/Province
Ontario
Country
Canada
Facility Name
Mereo Investigator Site
City
Montreal
State/Province
Quebec
Country
Canada
Facility Name
Mereo Investigator Site
City
Quebec City
State/Province
Quebec
Country
Canada
Facility Name
Mereo Investigator Site
City
Aarhus
Country
Denmark
Facility Name
Mereo Investigator Site
City
Odense
Country
Denmark
Facility Name
Mereo Investigator Site
City
Paris
State/Province
Paris Cedex 14
Country
France
Facility Name
Mereo Investigator Site
City
Lyon
Country
France
Facility Name
Mereo Investigator Site
City
Paris
Country
France
Facility Name
Mereo Investigator Site
City
Cambridge
State/Province
Cambridgeshire
Country
United Kingdom
Facility Name
Mereo Investigator Site
City
Newcastle upon Tyne
State/Province
Newcastle
Country
United Kingdom
Facility Name
Mereo Investigator Site
City
Oxford
State/Province
Oxfordshire
Country
United Kingdom
Facility Name
Mereo Investigator Site
City
Bristol
Country
United Kingdom
Facility Name
Mereo Investigator Site
City
London
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

A Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804

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