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Monoclonal Antibodies

Safety of Fresolimumab in the Treatment of 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

No Placebo-Only Group

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.

Eligible Conditions

Osteogenesis Imperfecta

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 6 months in single dose study and 12 months in repeat dose study

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

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0

Secondary study objectives

Percentage Change in Bone Turnover Markers or P1NP, Osteocalcin or Ocn, and C-terminal Telopeptide or CTX

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

Spinal Edema

Hepatic Encephalopathy

Elevated Lipase

Hyperglycemia

Hypoalbuminemia

Hypocalcemia

Nausea

Oliguria

Cord compression

alanine aminotransferase

Neuropathy- Lower Extremities

Anemia

Lower Sacral Pain

Abdominal Pain- Severe

Pain (under R breast)

Hypercalcemia

Dehydration

Elevated Amylase

Neuropathy

Bilaterial Effusion

INR

Elevated Bilirubin

Cellulitis

hypercalcemia

Hypokalemia

100%

80%

60%

40%

20%

Study treatment Arm

Arm 1 (Fresolimumab 1 mg/kg)

Arm 2 (Fresolimumab 10 mg/kg)

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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 (Total anticipated 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 (Total Anticipated 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

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