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Monoclonal Antibodies

Fresolimumab for Osteogenesis Imperfecta

Phase 1
Waitlist Available
Led By Brendan Lee, M.D., Ph.D.
Research Sponsored by Baylor College of Medicine
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 6 months in single dose study and 12 months in repeat dose study
Awards & highlights

Study Summary

This trial is testing if an antibody that silences a protein important in bone formation is safe in the treatment of Osteogenesis Imperfecta, a rare disorder that causes bones to break easily.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~6 months in single dose study and 12 months in repeat dose study
This trial's timeline: 3 weeks for screening, Varies for treatment, and 6 months in single dose study and 12 months in repeat dose study for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Secondary outcome measures
Change in ml in FEV1
Change in ml in FVC
Difference in score of numeric rating scale for pain
+4 more

Side effects data

From 2014 Phase 2 trial • 23 Patients • NCT01401062
27%
Fatigue
27%
Aspartate Aminotransferase Increased
18%
Alkaline Phosphate Increased
18%
Disease Progression
18%
Disease progression
18%
Thrombocytopenia
18%
atrial fibrillation
9%
Hypoalbuminemia
9%
Hepatic Encephalopathy
9%
Nausea
9%
Hypocalcemia
9%
Oliguria
9%
Elevated Lipase
9%
Hyperglycemia
9%
Spinal Edema
9%
Cord compression
9%
alanine aminotransferase
9%
Neuropathy- Lower Extremities
9%
Anemia
9%
Lower Sacral Pain
9%
Abdominal Pain- Severe
9%
Pain (under R breast)
9%
Hypercalcemia
9%
Dehydration
9%
Elevated Amylase
9%
Neuropathy
9%
Bilaterial Effusion
9%
INR
9%
Elevated Bilirubin
9%
Cellulitis
9%
hypercalcemia
9%
Hypokalemia
100%
80%
60%
40%
20%
0%
Study treatment Arm
Arm 1 (Fresolimumab 1 mg/kg)
Arm 2 (Fresolimumab 10 mg/kg)

Trial Design

4Treatment groups
Experimental Treatment
Group I: Stage 2 Repeat doses every 3 monthsExperimental Treatment1 Intervention
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
Group II: Stage 2 Repeat dose every 6 monthsExperimental Treatment1 Intervention
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
Group III: Stage 2 High doseExperimental Treatment1 Intervention
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
Group IV: Stage 1 Low doseExperimental Treatment1 Intervention
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
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Fresolimumab
2011
Completed Phase 2
~80

Find a Location

Who is running the clinical trial?

Oregon Health and Science UniversityOTHER
973 Previous Clinical Trials
7,385,801 Total Patients Enrolled
3 Trials studying Osteogenesis Imperfecta
1,154 Patients Enrolled for Osteogenesis Imperfecta
Baylor College of MedicineLead Sponsor
1,001 Previous Clinical Trials
6,002,241 Total Patients Enrolled
5 Trials studying Osteogenesis Imperfecta
1,175 Patients Enrolled for Osteogenesis Imperfecta
Genzyme, a Sanofi CompanyIndustry Sponsor
524 Previous Clinical Trials
85,618 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
clinicaltrials.govStudy
Safety of Fresolimumab in the Treatment of Osteogenesis Imperfecta
Brendan Lee 2017. "Safety of Fresolimumab in the Treatment of Osteogenesis Imperfecta". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03064074.
~1 spots leftby Apr 2025
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