Trial Outcomes & Findings for Safety of Fresolimumab in the Treatment of Osteogenesis Imperfecta (NCT NCT03064074)
NCT ID: NCT03064074
Last Updated: 2024-12-04
Results Overview
Safety of single and repeat doses of fresolimumab will be assessed in adult patients with moderate to severe osteogenesis imperfecta
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
11 participants
Primary outcome timeframe
6 months for single dose study and 12 months for repeat dose study
Results posted on
2024-12-04
Participant Flow
Participant milestones
| Measure |
Stage 1 Low Dose
There are a total of 6 study visits within approximately a 6 month timespan. Investigators will evaluate the safety of a single administration of fresolimumab in adult patients with OI. Subjects will receive a single-dose of 1 mg/kg of fresolimumab (n=4).
At each study visit, the participant may have the following testing done:
* Physical exam
* Vitals
* Blood draw for safety labs, pharmacokinetics, etc
* If the participant is female, she will have a pregnancy test
* EKG
* DXA
* Infusion of the study drug
Fresolimumab: The purpose of this study is to determine if fresolimumab is safe as a treatment for OI. We will evaluate the safety of a single dose of fresolimumab in the 1st stage of the study. We will evaluate the safety of multiple doses of fresolimumab in the 2nd stage of the study. The Investigators will evaluate the effect of the two doses of fresolimumab in Stage 1 on markers of bone turnover and determine the dose that shows the greatest reduction in bone turnover markers compared to no treatment. This dose will be chosen for the repeat dose studies. If there were no significant changes between the bone turnover markers with either dose, the 4 mg/kg dose will be chosen for the repeat dose study.
|
Stage 2 High Dose
There are a total of 6 study visits within approximately a 6 month timespan. Investigators will evaluate the safety of a single administration of fresolimumab in adult patients with OI. Subjects will receive a single-dose of 4 mg/kg of fresolimumab (n=4).
At each study visit, the participant may have the following testing done:
* Physical exam
* Vitals
* Blood draw for safety labs, pharmacokinetics, etc
* If the participant is female, she will have a pregnancy test
* EKG
* DXA
* Infusion of the study drug
Fresolimumab: The purpose of this study is to determine if fresolimumab is safe as a treatment for OI. We will evaluate the safety of a single dose of fresolimumab in the 1st stage of the study. We will evaluate the safety of multiple doses of fresolimumab in the 2nd stage of the study. The Investigators will evaluate the effect of the two doses of fresolimumab in Stage 1 on markers of bone turnover and determine the dose that shows the greatest reduction in bone turnover markers compared to no treatment. This dose will be chosen for the repeat dose studies. If there were no significant changes between the bone turnover markers with either dose, the 4 mg/kg dose will be chosen for the repeat dose study.
|
Stage 2 Repeat Dose Every 6 Months
Fresolimumab will be administered every six months for a total treatment period of 12 months (n=4). The dose to be administered (1 or 4 mg/kg) will be chosen after completion of Stage 1. The primary Stage 2 endpoint will be safety measures assessed over 12 months. The secondary endpoints will be changes in markers of bone remodeling, bone mineral density, estimated strength.
At each study visit, participants may have the following testing done:
* Physical exam
* Vitals
* Blood draw for safety labs, pharmacokinetics, etc
* If the participant is female, she will have a pregnancy test
* EKG
* DXA
* Infusion of the study drug
* Skeletal survey
* Peripheral quantitative CT (pQCT) of the forearm
* Quality of Life Surveys
* Pulmonary function test
* Walk test
Fresolimumab: The purpose of this study is to determine if fresolimumab is safe as a treatment for OI. We will evaluate the safety of a single dose of fresolimumab in the 1st stage of the study. We will evaluate the safety of multiple doses of fresolimumab in the 2nd stage of the study. The Investigators will evaluate the effect of the two doses of fresolimumab in Stage 1 on markers of bone turnover and determine the dose that shows the greatest reduction in bone turnover markers compared to no treatment. This dose will be chosen for the repeat dose studies. If there were no significant changes between the bone turnover markers with either dose, the 4 mg/kg dose will be chosen for the repeat dose study.
|
Stage 2 Repeat Doses Every 3 Months
Fresolimumab will be administered every three months for a total treatment period of 12 months (n=4). The dose to be administered (1 or 4 mg/kg) will be chosen after completion of Stage 1. The primary Stage 2 endpoint will be safety measures assessed over 12 months. The secondary endpoints will be changes in markers of bone remodeling, bone mineral density, estimated strength.
At each study visit, participants may have the following testing done:
* Physical exam
* Vitals
* Blood draw for safety labs, pharmacokinetics, etc
* If the participant is female, she will have a pregnancy test
* EKG
* DXA
* Infusion of the study drug
* Skeletal survey
* Peripheral quantitative CT (pQCT) of the forearm
* Quality of Life Surveys
* Pulmonary function test
* Walk test
Fresolimumab: The purpose of this study is to determine if fresolimumab is safe as a treatment for OI. We will evaluate the safety of a single dose of fresolimumab in the 1st stage of the study. We will evaluate the safety of multiple doses of fresolimumab in the 2nd stage of the study. The Investigators will evaluate the effect of the two doses of fresolimumab in Stage 1 on markers of bone turnover and determine the dose that shows the greatest reduction in bone turnover markers compared to no treatment. This dose will be chosen for the repeat dose studies. If there were no significant changes between the bone turnover markers with either dose, the 4 mg/kg dose will be chosen for the repeat dose study.
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
4
|
4
|
2
|
1
|
|
Overall Study
COMPLETED
|
4
|
4
|
2
|
0
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
1
|
Reasons for withdrawal
| Measure |
Stage 1 Low Dose
There are a total of 6 study visits within approximately a 6 month timespan. Investigators will evaluate the safety of a single administration of fresolimumab in adult patients with OI. Subjects will receive a single-dose of 1 mg/kg of fresolimumab (n=4).
At each study visit, the participant may have the following testing done:
* Physical exam
* Vitals
* Blood draw for safety labs, pharmacokinetics, etc
* If the participant is female, she will have a pregnancy test
* EKG
* DXA
* Infusion of the study drug
Fresolimumab: The purpose of this study is to determine if fresolimumab is safe as a treatment for OI. We will evaluate the safety of a single dose of fresolimumab in the 1st stage of the study. We will evaluate the safety of multiple doses of fresolimumab in the 2nd stage of the study. The Investigators will evaluate the effect of the two doses of fresolimumab in Stage 1 on markers of bone turnover and determine the dose that shows the greatest reduction in bone turnover markers compared to no treatment. This dose will be chosen for the repeat dose studies. If there were no significant changes between the bone turnover markers with either dose, the 4 mg/kg dose will be chosen for the repeat dose study.
|
Stage 2 High Dose
There are a total of 6 study visits within approximately a 6 month timespan. Investigators will evaluate the safety of a single administration of fresolimumab in adult patients with OI. Subjects will receive a single-dose of 4 mg/kg of fresolimumab (n=4).
At each study visit, the participant may have the following testing done:
* Physical exam
* Vitals
* Blood draw for safety labs, pharmacokinetics, etc
* If the participant is female, she will have a pregnancy test
* EKG
* DXA
* Infusion of the study drug
Fresolimumab: The purpose of this study is to determine if fresolimumab is safe as a treatment for OI. We will evaluate the safety of a single dose of fresolimumab in the 1st stage of the study. We will evaluate the safety of multiple doses of fresolimumab in the 2nd stage of the study. The Investigators will evaluate the effect of the two doses of fresolimumab in Stage 1 on markers of bone turnover and determine the dose that shows the greatest reduction in bone turnover markers compared to no treatment. This dose will be chosen for the repeat dose studies. If there were no significant changes between the bone turnover markers with either dose, the 4 mg/kg dose will be chosen for the repeat dose study.
|
Stage 2 Repeat Dose Every 6 Months
Fresolimumab will be administered every six months for a total treatment period of 12 months (n=4). The dose to be administered (1 or 4 mg/kg) will be chosen after completion of Stage 1. The primary Stage 2 endpoint will be safety measures assessed over 12 months. The secondary endpoints will be changes in markers of bone remodeling, bone mineral density, estimated strength.
At each study visit, participants may have the following testing done:
* Physical exam
* Vitals
* Blood draw for safety labs, pharmacokinetics, etc
* If the participant is female, she will have a pregnancy test
* EKG
* DXA
* Infusion of the study drug
* Skeletal survey
* Peripheral quantitative CT (pQCT) of the forearm
* Quality of Life Surveys
* Pulmonary function test
* Walk test
Fresolimumab: The purpose of this study is to determine if fresolimumab is safe as a treatment for OI. We will evaluate the safety of a single dose of fresolimumab in the 1st stage of the study. We will evaluate the safety of multiple doses of fresolimumab in the 2nd stage of the study. The Investigators will evaluate the effect of the two doses of fresolimumab in Stage 1 on markers of bone turnover and determine the dose that shows the greatest reduction in bone turnover markers compared to no treatment. This dose will be chosen for the repeat dose studies. If there were no significant changes between the bone turnover markers with either dose, the 4 mg/kg dose will be chosen for the repeat dose study.
|
Stage 2 Repeat Doses Every 3 Months
Fresolimumab will be administered every three months for a total treatment period of 12 months (n=4). The dose to be administered (1 or 4 mg/kg) will be chosen after completion of Stage 1. The primary Stage 2 endpoint will be safety measures assessed over 12 months. The secondary endpoints will be changes in markers of bone remodeling, bone mineral density, estimated strength.
At each study visit, participants may have the following testing done:
* Physical exam
* Vitals
* Blood draw for safety labs, pharmacokinetics, etc
* If the participant is female, she will have a pregnancy test
* EKG
* DXA
* Infusion of the study drug
* Skeletal survey
* Peripheral quantitative CT (pQCT) of the forearm
* Quality of Life Surveys
* Pulmonary function test
* Walk test
Fresolimumab: The purpose of this study is to determine if fresolimumab is safe as a treatment for OI. We will evaluate the safety of a single dose of fresolimumab in the 1st stage of the study. We will evaluate the safety of multiple doses of fresolimumab in the 2nd stage of the study. The Investigators will evaluate the effect of the two doses of fresolimumab in Stage 1 on markers of bone turnover and determine the dose that shows the greatest reduction in bone turnover markers compared to no treatment. This dose will be chosen for the repeat dose studies. If there were no significant changes between the bone turnover markers with either dose, the 4 mg/kg dose will be chosen for the repeat dose study.
|
|---|---|---|---|---|
|
Overall Study
Physician Decision
|
0
|
0
|
0
|
1
|
Baseline Characteristics
Safety of Fresolimumab in the Treatment of Osteogenesis Imperfecta
Baseline characteristics by cohort
| Measure |
Stage 1 Low Dose
n=4 Participants
There are a total of 6 study visits within approximately a 6 month timespan. Investigators will evaluate the safety of a single administration of fresolimumab in adult patients with OI. Subjects will receive a single-dose of 1 mg/kg of fresolimumab (n=4).
At each study visit, the participant may have the following testing done:
* Physical exam
* Vitals
* Blood draw for safety labs, pharmacokinetics, etc
* If the participant is female, she will have a pregnancy test
* EKG
* DXA
* Infusion of the study drug
Fresolimumab: The purpose of this study is to determine if fresolimumab is safe as a treatment for OI. We will evaluate the safety of a single dose of fresolimumab in the 1st stage of the study. We will evaluate the safety of multiple doses of fresolimumab in the 2nd stage of the study. The Investigators will evaluate the effect of the two doses of fresolimumab in Stage 1 on markers of bone turnover and determine the dose that shows the greatest reduction in bone turnover markers compared to no treatment. This dose will be chosen for the repeat dose studies. If there were no significant changes between the bone turnover markers with either dose, the 4 mg/kg dose will be chosen for the repeat dose study.
|
Stage 2 High Dose
n=4 Participants
There are a total of 6 study visits within approximately a 6 month timespan. Investigators will evaluate the safety of a single administration of fresolimumab in adult patients with OI. Subjects will receive a single-dose of 4 mg/kg of fresolimumab (n=4).
At each study visit, the participant may have the following testing done:
* Physical exam
* Vitals
* Blood draw for safety labs, pharmacokinetics, etc
* If the participant is female, she will have a pregnancy test
* EKG
* DXA
* Infusion of the study drug
Fresolimumab: The purpose of this study is to determine if fresolimumab is safe as a treatment for OI. We will evaluate the safety of a single dose of fresolimumab in the 1st stage of the study. We will evaluate the safety of multiple doses of fresolimumab in the 2nd stage of the study. The Investigators will evaluate the effect of the two doses of fresolimumab in Stage 1 on markers of bone turnover and determine the dose that shows the greatest reduction in bone turnover markers compared to no treatment. This dose will be chosen for the repeat dose studies. If there were no significant changes between the bone turnover markers with either dose, the 4 mg/kg dose will be chosen for the repeat dose study.
|
Stage 2 Repeat Dose Every 6 Months
n=2 Participants
Fresolimumab will be administered every six months for a total treatment period of 12 months (n=4). The dose to be administered (1 or 4 mg/kg) will be chosen after completion of Stage 1. The primary Stage 2 endpoint will be safety measures assessed over 12 months. The secondary endpoints will be changes in markers of bone remodeling, bone mineral density, estimated strength.
At each study visit, participants may have the following testing done:
* Physical exam
* Vitals
* Blood draw for safety labs, pharmacokinetics, etc
* If the participant is female, she will have a pregnancy test
* EKG
* DXA
* Infusion of the study drug
* Skeletal survey
* Peripheral quantitative CT (pQCT) of the forearm
* Quality of Life Surveys
* Pulmonary function test
* Walk test
Fresolimumab: The purpose of this study is to determine if fresolimumab is safe as a treatment for OI. We will evaluate the safety of a single dose of fresolimumab in the 1st stage of the study. We will evaluate the safety of multiple doses of fresolimumab in the 2nd stage of the study. The Investigators will evaluate the effect of the two doses of fresolimumab in Stage 1 on markers of bone turnover and determine the dose that shows the greatest reduction in bone turnover markers compared to no treatment. This dose will be chosen for the repeat dose studies. If there were no significant changes between the bone turnover markers with either dose, the 4 mg/kg dose will be chosen for the repeat dose study.
|
Stage 2 Repeat Doses Every 3 Months
n=1 Participants
Fresolimumab will be administered every three months for a total treatment period of 12 months (n=4). The dose to be administered (1 or 4 mg/kg) will be chosen after completion of Stage 1. The primary Stage 2 endpoint will be safety measures assessed over 12 months. The secondary endpoints will be changes in markers of bone remodeling, bone mineral density, estimated strength.
At each study visit, participants may have the following testing done:
* Physical exam
* Vitals
* Blood draw for safety labs, pharmacokinetics, etc
* If the participant is female, she will have a pregnancy test
* EKG
* DXA
* Infusion of the study drug
* Skeletal survey
* Peripheral quantitative CT (pQCT) of the forearm
* Quality of Life Surveys
* Pulmonary function test
* Walk test
Fresolimumab: The purpose of this study is to determine if fresolimumab is safe as a treatment for OI. We will evaluate the safety of a single dose of fresolimumab in the 1st stage of the study. We will evaluate the safety of multiple doses of fresolimumab in the 2nd stage of the study. The Investigators will evaluate the effect of the two doses of fresolimumab in Stage 1 on markers of bone turnover and determine the dose that shows the greatest reduction in bone turnover markers compared to no treatment. This dose will be chosen for the repeat dose studies. If there were no significant changes between the bone turnover markers with either dose, the 4 mg/kg dose will be chosen for the repeat dose study.
|
Total
n=11 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
4 Participants
n=21 Participants
|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
4 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
11 Participants
n=21 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
7 Participants
n=21 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
White
|
3 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
8 Participants
n=21 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
2 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Region of Enrollment
United States
|
4 participants
n=5 Participants
|
4 participants
n=7 Participants
|
2 participants
n=5 Participants
|
1 participants
n=4 Participants
|
11 participants
n=21 Participants
|
PRIMARY outcome
Timeframe: 6 months for single dose study and 12 months for repeat dose studySafety of single and repeat doses of fresolimumab will be assessed in adult patients with moderate to severe osteogenesis imperfecta
Outcome measures
| Measure |
Stage 1 Low Dose
n=4 Participants
There are a total of 6 study visits within approximately a 6 month timespan. Investigators will evaluate the safety of a single administration of fresolimumab in adult patients with OI. Subjects will receive a single-dose of 1 mg/kg of fresolimumab (n=4).
At each study visit, the participant may have the following testing done:
* Physical exam
* Vitals
* Blood draw for safety labs, pharmacokinetics, etc
* If the participant is female, she will have a pregnancy test
* EKG
* DXA
* Infusion of the study drug
Fresolimumab: The purpose of this study is to determine if fresolimumab is safe as a treatment for OI. We will evaluate the safety of a single dose of fresolimumab in the 1st stage of the study. We will evaluate the safety of multiple doses of fresolimumab in the 2nd stage of the study. The Investigators will evaluate the effect of the two doses of fresolimumab in Stage 1 on markers of bone turnover and determine the dose that shows the greatest reduction in bone turnover markers compared to no treatment. This dose will be chosen for the repeat dose studies. If there were no significant changes between the bone turnover markers with either dose, the 4 mg/kg dose will be chosen for the repeat dose study.
|
Stage 2 High Dose
n=4 Participants
There are a total of 6 study visits within approximately a 6 month timespan. Investigators will evaluate the safety of a single administration of fresolimumab in adult patients with OI. Subjects will receive a single-dose of 4 mg/kg of fresolimumab (n=4).
At each study visit, the participant may have the following testing done:
* Physical exam
* Vitals
* Blood draw for safety labs, pharmacokinetics, etc
* If the participant is female, she will have a pregnancy test
* EKG
* DXA
* Infusion of the study drug
Fresolimumab: The purpose of this study is to determine if fresolimumab is safe as a treatment for OI. We will evaluate the safety of a single dose of fresolimumab in the 1st stage of the study. We will evaluate the safety of multiple doses of fresolimumab in the 2nd stage of the study. The Investigators will evaluate the effect of the two doses of fresolimumab in Stage 1 on markers of bone turnover and determine the dose that shows the greatest reduction in bone turnover markers compared to no treatment. This dose will be chosen for the repeat dose studies. If there were no significant changes between the bone turnover markers with either dose, the 4 mg/kg dose will be chosen for the repeat dose study.
|
Stage 2 Repeat Dose Every 6 Months
n=2 Participants
Fresolimumab will be administered every six months for a total treatment period of 12 months (n=4). The dose to be administered (1 or 4 mg/kg) will be chosen after completion of Stage 1. The primary Stage 2 endpoint will be safety measures assessed over 12 months. The secondary endpoints will be changes in markers of bone remodeling, bone mineral density, estimated strength.
At each study visit, participants may have the following testing done:
* Physical exam
* Vitals
* Blood draw for safety labs, pharmacokinetics, etc
* If the participant is female, she will have a pregnancy test
* EKG
* DXA
* Infusion of the study drug
* Skeletal survey
* Peripheral quantitative CT (pQCT) of the forearm
* Quality of Life Surveys
* Pulmonary function test
* Walk test
Fresolimumab: The purpose of this study is to determine if fresolimumab is safe as a treatment for OI. We will evaluate the safety of a single dose of fresolimumab in the 1st stage of the study. We will evaluate the safety of multiple doses of fresolimumab in the 2nd stage of the study. The Investigators will evaluate the effect of the two doses of fresolimumab in Stage 1 on markers of bone turnover and determine the dose that shows the greatest reduction in bone turnover markers compared to no treatment. This dose will be chosen for the repeat dose studies. If there were no significant changes between the bone turnover markers with either dose, the 4 mg/kg dose will be chosen for the repeat dose study.
|
Stage 2 Repeat Doses Every 3 Months
n=1 Participants
Fresolimumab will be administered every three months for a total treatment period of 12 months (n=4). The dose to be administered (1 or 4 mg/kg) will be chosen after completion of Stage 1. The primary Stage 2 endpoint will be safety measures assessed over 12 months. The secondary endpoints will be changes in markers of bone remodeling, bone mineral density, estimated strength.
At each study visit, participants may have the following testing done:
* Physical exam
* Vitals
* Blood draw for safety labs, pharmacokinetics, etc
* If the participant is female, she will have a pregnancy test
* EKG
* DXA
* Infusion of the study drug
* Skeletal survey
* Peripheral quantitative CT (pQCT) of the forearm
* Quality of Life Surveys
* Pulmonary function test
* Walk test
Fresolimumab: The purpose of this study is to determine if fresolimumab is safe as a treatment for OI. We will evaluate the safety of a single dose of fresolimumab in the 1st stage of the study. We will evaluate the safety of multiple doses of fresolimumab in the 2nd stage of the study. The Investigators will evaluate the effect of the two doses of fresolimumab in Stage 1 on markers of bone turnover and determine the dose that shows the greatest reduction in bone turnover markers compared to no treatment. This dose will be chosen for the repeat dose studies. If there were no significant changes between the bone turnover markers with either dose, the 4 mg/kg dose will be chosen for the repeat dose study.
|
|---|---|---|---|---|
|
Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0
|
2 Participants
|
4 Participants
|
2 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: 6 months in single dose study and 12 months in repeat dose studyPercentage change in bone turnover markers from baseline to Day 180
Outcome measures
| Measure |
Stage 1 Low Dose
n=4 Participants
There are a total of 6 study visits within approximately a 6 month timespan. Investigators will evaluate the safety of a single administration of fresolimumab in adult patients with OI. Subjects will receive a single-dose of 1 mg/kg of fresolimumab (n=4).
At each study visit, the participant may have the following testing done:
* Physical exam
* Vitals
* Blood draw for safety labs, pharmacokinetics, etc
* If the participant is female, she will have a pregnancy test
* EKG
* DXA
* Infusion of the study drug
Fresolimumab: The purpose of this study is to determine if fresolimumab is safe as a treatment for OI. We will evaluate the safety of a single dose of fresolimumab in the 1st stage of the study. We will evaluate the safety of multiple doses of fresolimumab in the 2nd stage of the study. The Investigators will evaluate the effect of the two doses of fresolimumab in Stage 1 on markers of bone turnover and determine the dose that shows the greatest reduction in bone turnover markers compared to no treatment. This dose will be chosen for the repeat dose studies. If there were no significant changes between the bone turnover markers with either dose, the 4 mg/kg dose will be chosen for the repeat dose study.
|
Stage 2 High Dose
n=4 Participants
There are a total of 6 study visits within approximately a 6 month timespan. Investigators will evaluate the safety of a single administration of fresolimumab in adult patients with OI. Subjects will receive a single-dose of 4 mg/kg of fresolimumab (n=4).
At each study visit, the participant may have the following testing done:
* Physical exam
* Vitals
* Blood draw for safety labs, pharmacokinetics, etc
* If the participant is female, she will have a pregnancy test
* EKG
* DXA
* Infusion of the study drug
Fresolimumab: The purpose of this study is to determine if fresolimumab is safe as a treatment for OI. We will evaluate the safety of a single dose of fresolimumab in the 1st stage of the study. We will evaluate the safety of multiple doses of fresolimumab in the 2nd stage of the study. The Investigators will evaluate the effect of the two doses of fresolimumab in Stage 1 on markers of bone turnover and determine the dose that shows the greatest reduction in bone turnover markers compared to no treatment. This dose will be chosen for the repeat dose studies. If there were no significant changes between the bone turnover markers with either dose, the 4 mg/kg dose will be chosen for the repeat dose study.
|
Stage 2 Repeat Dose Every 6 Months
n=2 Participants
Fresolimumab will be administered every six months for a total treatment period of 12 months (n=4). The dose to be administered (1 or 4 mg/kg) will be chosen after completion of Stage 1. The primary Stage 2 endpoint will be safety measures assessed over 12 months. The secondary endpoints will be changes in markers of bone remodeling, bone mineral density, estimated strength.
At each study visit, participants may have the following testing done:
* Physical exam
* Vitals
* Blood draw for safety labs, pharmacokinetics, etc
* If the participant is female, she will have a pregnancy test
* EKG
* DXA
* Infusion of the study drug
* Skeletal survey
* Peripheral quantitative CT (pQCT) of the forearm
* Quality of Life Surveys
* Pulmonary function test
* Walk test
Fresolimumab: The purpose of this study is to determine if fresolimumab is safe as a treatment for OI. We will evaluate the safety of a single dose of fresolimumab in the 1st stage of the study. We will evaluate the safety of multiple doses of fresolimumab in the 2nd stage of the study. The Investigators will evaluate the effect of the two doses of fresolimumab in Stage 1 on markers of bone turnover and determine the dose that shows the greatest reduction in bone turnover markers compared to no treatment. This dose will be chosen for the repeat dose studies. If there were no significant changes between the bone turnover markers with either dose, the 4 mg/kg dose will be chosen for the repeat dose study.
|
Stage 2 Repeat Doses Every 3 Months
n=1 Participants
Fresolimumab will be administered every three months for a total treatment period of 12 months (n=4). The dose to be administered (1 or 4 mg/kg) will be chosen after completion of Stage 1. The primary Stage 2 endpoint will be safety measures assessed over 12 months. The secondary endpoints will be changes in markers of bone remodeling, bone mineral density, estimated strength.
At each study visit, participants may have the following testing done:
* Physical exam
* Vitals
* Blood draw for safety labs, pharmacokinetics, etc
* If the participant is female, she will have a pregnancy test
* EKG
* DXA
* Infusion of the study drug
* Skeletal survey
* Peripheral quantitative CT (pQCT) of the forearm
* Quality of Life Surveys
* Pulmonary function test
* Walk test
Fresolimumab: The purpose of this study is to determine if fresolimumab is safe as a treatment for OI. We will evaluate the safety of a single dose of fresolimumab in the 1st stage of the study. We will evaluate the safety of multiple doses of fresolimumab in the 2nd stage of the study. The Investigators will evaluate the effect of the two doses of fresolimumab in Stage 1 on markers of bone turnover and determine the dose that shows the greatest reduction in bone turnover markers compared to no treatment. This dose will be chosen for the repeat dose studies. If there were no significant changes between the bone turnover markers with either dose, the 4 mg/kg dose will be chosen for the repeat dose study.
|
|---|---|---|---|---|
|
Percentage Change in Bone Turnover Markers or P1NP, Osteocalcin or Ocn, and C-terminal Telopeptide or CTX
Percentage Change in CTX
|
49.89 Percent change
Standard Error 29.29
|
-4.02 Percent change
Standard Error 33.82
|
51.83 Percent change
Standard Error 34.56
|
NA Percent change
Standard Error NA
Only one participant was enrolled. This participant had to be withdrawn due to fracture because of OI and could not complete the study procedures. Thus, the percentage change of CTX could not be calculated.
|
|
Percentage Change in Bone Turnover Markers or P1NP, Osteocalcin or Ocn, and C-terminal Telopeptide or CTX
Percentage change in Osteocalcin
|
76.4 Percent change
Standard Error 39.1
|
-18.6 Percent change
Standard Error 39.13
|
83.9 Percent change
Standard Error 17.56
|
NA Percent change
Standard Error NA
Only one participant was enrolled. This participant had to be withdrawn due to fracture because of OI and could not complete the study procedures. Thus, the percentage change in Ocn could not be calculated.
|
|
Percentage Change in Bone Turnover Markers or P1NP, Osteocalcin or Ocn, and C-terminal Telopeptide or CTX
Percentage Change in P1NP
|
28.31 Percent change
Standard Error 17.44
|
24.95 Percent change
Standard Error 17.44
|
118.63 Percent change
Standard Error 45.40
|
NA Percent change
Standard Error NA
Only one participant was enrolled. This participant had to be withdrawn due to fracture because of OI and could not complete the study procedures. Thus, the percentage change of P1NP could not be calculated.
|
Adverse Events
Stage 1 Low Dose
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Stage 2 High Dose
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Stage 2 Repeat Dose Every 6 Months
Serious events: 1 serious events
Other events: 2 other events
Deaths: 0 deaths
Stage 2 Repeat Doses Every 3 Months
Serious events: 1 serious events
Other events: 1 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Stage 1 Low Dose
n=4 participants at risk
There are a total of 6 study visits within approximately a 6 month timespan. Investigators will evaluate the safety of a single administration of fresolimumab in adult patients with OI. Subjects will receive a single-dose of 1 mg/kg of fresolimumab (n=4).
At each study visit, the participant may have the following testing done:
* Physical exam
* Vitals
* Blood draw for safety labs, pharmacokinetics, etc
* If the participant is female, she will have a pregnancy test
* EKG
* DXA
* Infusion of the study drug
Fresolimumab: The purpose of this study is to determine if fresolimumab is safe as a treatment for OI. We will evaluate the safety of a single dose of fresolimumab in the 1st stage of the study. We will evaluate the safety of multiple doses of fresolimumab in the 2nd stage of the study. The Investigators will evaluate the effect of the two doses of fresolimumab in Stage 1 on markers of bone turnover and determine the dose that shows the greatest reduction in bone turnover markers compared to no treatment. This dose will be chosen for the repeat dose studies. If there were no significant changes between the bone turnover markers with either dose, the 4 mg/kg dose will be chosen for the repeat dose study.
|
Stage 2 High Dose
n=4 participants at risk
There are a total of 6 study visits within approximately a 6 month timespan. Investigators will evaluate the safety of a single administration of fresolimumab in adult patients with OI. Subjects will receive a single-dose of 4 mg/kg of fresolimumab (n=4).
At each study visit, the participant may have the following testing done:
* Physical exam
* Vitals
* Blood draw for safety labs, pharmacokinetics, etc
* If the participant is female, she will have a pregnancy test
* EKG
* DXA
* Infusion of the study drug
Fresolimumab: The purpose of this study is to determine if fresolimumab is safe as a treatment for OI. We will evaluate the safety of a single dose of fresolimumab in the 1st stage of the study. We will evaluate the safety of multiple doses of fresolimumab in the 2nd stage of the study. The Investigators will evaluate the effect of the two doses of fresolimumab in Stage 1 on markers of bone turnover and determine the dose that shows the greatest reduction in bone turnover markers compared to no treatment. This dose will be chosen for the repeat dose studies. If there were no significant changes between the bone turnover markers with either dose, the 4 mg/kg dose will be chosen for the repeat dose study.
|
Stage 2 Repeat Dose Every 6 Months
n=2 participants at risk
Fresolimumab will be administered every six months for a total treatment period of 12 months (n=4). The dose to be administered (1 or 4 mg/kg) will be chosen after completion of Stage 1. The primary Stage 2 endpoint will be safety measures assessed over 12 months. The secondary endpoints will be changes in markers of bone remodeling, bone mineral density, estimated strength.
At each study visit, participants may have the following testing done:
* Physical exam
* Vitals
* Blood draw for safety labs, pharmacokinetics, etc
* If the participant is female, she will have a pregnancy test
* EKG
* DXA
* Infusion of the study drug
* Skeletal survey
* Peripheral quantitative CT (pQCT) of the forearm
* Quality of Life Surveys
* Pulmonary function test
* Walk test
Fresolimumab: The purpose of this study is to determine if fresolimumab is safe as a treatment for OI. We will evaluate the safety of a single dose of fresolimumab in the 1st stage of the study. We will evaluate the safety of multiple doses of fresolimumab in the 2nd stage of the study. The Investigators will evaluate the effect of the two doses of fresolimumab in Stage 1 on markers of bone turnover and determine the dose that shows the greatest reduction in bone turnover markers compared to no treatment. This dose will be chosen for the repeat dose studies. If there were no significant changes between the bone turnover markers with either dose, the 4 mg/kg dose will be chosen for the repeat dose study.
|
Stage 2 Repeat Doses Every 3 Months
n=1 participants at risk
Fresolimumab will be administered every three months for a total treatment period of 12 months (n=4). The dose to be administered (1 or 4 mg/kg) will be chosen after completion of Stage 1. The primary Stage 2 endpoint will be safety measures assessed over 12 months. The secondary endpoints will be changes in markers of bone remodeling, bone mineral density, estimated strength.
At each study visit, participants may have the following testing done:
* Physical exam
* Vitals
* Blood draw for safety labs, pharmacokinetics, etc
* If the participant is female, she will have a pregnancy test
* EKG
* DXA
* Infusion of the study drug
* Skeletal survey
* Peripheral quantitative CT (pQCT) of the forearm
* Quality of Life Surveys
* Pulmonary function test
* Walk test
Fresolimumab: The purpose of this study is to determine if fresolimumab is safe as a treatment for OI. We will evaluate the safety of a single dose of fresolimumab in the 1st stage of the study. We will evaluate the safety of multiple doses of fresolimumab in the 2nd stage of the study. The Investigators will evaluate the effect of the two doses of fresolimumab in Stage 1 on markers of bone turnover and determine the dose that shows the greatest reduction in bone turnover markers compared to no treatment. This dose will be chosen for the repeat dose studies. If there were no significant changes between the bone turnover markers with either dose, the 4 mg/kg dose will be chosen for the repeat dose study.
|
|---|---|---|---|---|
|
Musculoskeletal and connective tissue disorders
Fracture
|
0.00%
0/4 • 6 Months for single dose study and 1 year for the multi dose study
|
0.00%
0/4 • 6 Months for single dose study and 1 year for the multi dose study
|
50.0%
1/2 • Number of events 1 • 6 Months for single dose study and 1 year for the multi dose study
|
100.0%
1/1 • Number of events 2 • 6 Months for single dose study and 1 year for the multi dose study
|
Other adverse events
| Measure |
Stage 1 Low Dose
n=4 participants at risk
There are a total of 6 study visits within approximately a 6 month timespan. Investigators will evaluate the safety of a single administration of fresolimumab in adult patients with OI. Subjects will receive a single-dose of 1 mg/kg of fresolimumab (n=4).
At each study visit, the participant may have the following testing done:
* Physical exam
* Vitals
* Blood draw for safety labs, pharmacokinetics, etc
* If the participant is female, she will have a pregnancy test
* EKG
* DXA
* Infusion of the study drug
Fresolimumab: The purpose of this study is to determine if fresolimumab is safe as a treatment for OI. We will evaluate the safety of a single dose of fresolimumab in the 1st stage of the study. We will evaluate the safety of multiple doses of fresolimumab in the 2nd stage of the study. The Investigators will evaluate the effect of the two doses of fresolimumab in Stage 1 on markers of bone turnover and determine the dose that shows the greatest reduction in bone turnover markers compared to no treatment. This dose will be chosen for the repeat dose studies. If there were no significant changes between the bone turnover markers with either dose, the 4 mg/kg dose will be chosen for the repeat dose study.
|
Stage 2 High Dose
n=4 participants at risk
There are a total of 6 study visits within approximately a 6 month timespan. Investigators will evaluate the safety of a single administration of fresolimumab in adult patients with OI. Subjects will receive a single-dose of 4 mg/kg of fresolimumab (n=4).
At each study visit, the participant may have the following testing done:
* Physical exam
* Vitals
* Blood draw for safety labs, pharmacokinetics, etc
* If the participant is female, she will have a pregnancy test
* EKG
* DXA
* Infusion of the study drug
Fresolimumab: The purpose of this study is to determine if fresolimumab is safe as a treatment for OI. We will evaluate the safety of a single dose of fresolimumab in the 1st stage of the study. We will evaluate the safety of multiple doses of fresolimumab in the 2nd stage of the study. The Investigators will evaluate the effect of the two doses of fresolimumab in Stage 1 on markers of bone turnover and determine the dose that shows the greatest reduction in bone turnover markers compared to no treatment. This dose will be chosen for the repeat dose studies. If there were no significant changes between the bone turnover markers with either dose, the 4 mg/kg dose will be chosen for the repeat dose study.
|
Stage 2 Repeat Dose Every 6 Months
n=2 participants at risk
Fresolimumab will be administered every six months for a total treatment period of 12 months (n=4). The dose to be administered (1 or 4 mg/kg) will be chosen after completion of Stage 1. The primary Stage 2 endpoint will be safety measures assessed over 12 months. The secondary endpoints will be changes in markers of bone remodeling, bone mineral density, estimated strength.
At each study visit, participants may have the following testing done:
* Physical exam
* Vitals
* Blood draw for safety labs, pharmacokinetics, etc
* If the participant is female, she will have a pregnancy test
* EKG
* DXA
* Infusion of the study drug
* Skeletal survey
* Peripheral quantitative CT (pQCT) of the forearm
* Quality of Life Surveys
* Pulmonary function test
* Walk test
Fresolimumab: The purpose of this study is to determine if fresolimumab is safe as a treatment for OI. We will evaluate the safety of a single dose of fresolimumab in the 1st stage of the study. We will evaluate the safety of multiple doses of fresolimumab in the 2nd stage of the study. The Investigators will evaluate the effect of the two doses of fresolimumab in Stage 1 on markers of bone turnover and determine the dose that shows the greatest reduction in bone turnover markers compared to no treatment. This dose will be chosen for the repeat dose studies. If there were no significant changes between the bone turnover markers with either dose, the 4 mg/kg dose will be chosen for the repeat dose study.
|
Stage 2 Repeat Doses Every 3 Months
n=1 participants at risk
Fresolimumab will be administered every three months for a total treatment period of 12 months (n=4). The dose to be administered (1 or 4 mg/kg) will be chosen after completion of Stage 1. The primary Stage 2 endpoint will be safety measures assessed over 12 months. The secondary endpoints will be changes in markers of bone remodeling, bone mineral density, estimated strength.
At each study visit, participants may have the following testing done:
* Physical exam
* Vitals
* Blood draw for safety labs, pharmacokinetics, etc
* If the participant is female, she will have a pregnancy test
* EKG
* DXA
* Infusion of the study drug
* Skeletal survey
* Peripheral quantitative CT (pQCT) of the forearm
* Quality of Life Surveys
* Pulmonary function test
* Walk test
Fresolimumab: The purpose of this study is to determine if fresolimumab is safe as a treatment for OI. We will evaluate the safety of a single dose of fresolimumab in the 1st stage of the study. We will evaluate the safety of multiple doses of fresolimumab in the 2nd stage of the study. The Investigators will evaluate the effect of the two doses of fresolimumab in Stage 1 on markers of bone turnover and determine the dose that shows the greatest reduction in bone turnover markers compared to no treatment. This dose will be chosen for the repeat dose studies. If there were no significant changes between the bone turnover markers with either dose, the 4 mg/kg dose will be chosen for the repeat dose study.
|
|---|---|---|---|---|
|
Investigations
General Adverse events
|
100.0%
4/4 • Number of events 38 • 6 Months for single dose study and 1 year for the multi dose study
|
100.0%
4/4 • Number of events 24 • 6 Months for single dose study and 1 year for the multi dose study
|
100.0%
2/2 • Number of events 13 • 6 Months for single dose study and 1 year for the multi dose study
|
100.0%
1/1 • Number of events 21 • 6 Months for single dose study and 1 year for the multi dose study
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place