Safety Study of GC1008 in Patients With Focal Segmental Glomerulosclerosis (FSGS) of Single Doses of GC1008 in Patients With Treatment Resistant Idiopathic FSGS

March 17, 2014 updated by: Genzyme, a Sanofi Company

A Phase I, Multicentre, Open-label, Dose-escalating Study of Single Doses of GC1008 in Patients With Treatment Resistant Idiopathic Focal Segmental Glomerulosclerosis (FSGS)

This study will investigate whether GC1008, an antibody which neutralizes TGF-beta, is safe in treating patients with the disease called focal segmental glomerulosclerosis (FSGS). The highest dose without excessive side effects will be investigated. Tests will determine how long GC1008 is in the body and how it is excreted.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

Patients in each cohort will receive a single dose of GC1008 infusion at 1, 2, 4 or 0.3 mg/kg body weight. The higher dose cohort will not start until the first 28 days safety data for the lower dose cohort have been reviewed by the independent Data Monitoring Committee (DMC). Cohort C and D will run concurrently with patients randomised to receive either a 4 or 0.3 mg/kg body weight dose, respectively. After receiving the infusion of GC1008 on Day 0, patients will be monitored for the 24 hours following the infusion. Patients will return periodically over the following 112 days for safety evaluations and clinical outcome assessments. Blood samples will be collected to evaluate the pharmacokinetics of single dose administration of GC1008 as well as for evaluation of markers of clinical efficacy

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
16

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Germany
      • Berlin, Germany
      • Duesseldorf, Germany
      • Solingen, Germany
  • Italy
      • Bergamo, Italy
  • United Kingdom
      • Cambridge, United Kingdom
  • United States
    • California
      • San Francisco, California, United States
    • Minnesota
      • Rochester, Minnesota, United States
    • New York
      • New Hyde Park, New York, United States, 11042-1433
    • North Carolina
      • Chapel Hill, North Carolina, United States

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

14 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • GFR≥25ml/min/1.73m2 calculated by the MDRD equation
  • Urinary total protein: creatinine ratios >200mg/mmol derived from the average of 2 first morning voids taken during screening period
  • Biopsy confirmed as idiopathic FSGS by a central reviewer
  • Treatment resistance. NOTE:Patients to have received minimum 6 week course of steroids or immunosuppressant
  • If receiving treatment with an ACEi and/or ARB dose to be stable for a minimum of 4 weeks prior to randomization
  • Influenza vaccine (according to season)
  • Negative screening per American Cancer Society (ACS) 2003 guidelines, as appropriate to patient demographics and clinical status

Exclusion Criteria:

  • Secondary FSGS
  • steroid resistant patients who are unable to reduce their steroid dose to <10mg/day of prednisolone or equivalent 4 weeks prior to study dosing day
  • Positive serology for serious infections (including but not limited to infection with Hep B or C, HIV)
  • Concomitant illnesses:Diabetes Type I; Cardiac or Hepatic disease, HIV; Cancer, precancerous state (eg familial adenomatous polyposis; Any condition requiring treatment with other immunosuppressant drugs within 4 weeks prior to dosing day or during the course of the study
  • Pre-existing oral-pharyngeal disease (dental carries and other minor dental disease are acceptable)
  • Haemoglobin level of <9.0g/dL prior to dosing
  • Treatment with coumadin, anti-vitamin K analogues or low molecular weight heparins. Patients must have stopped treatment a minimum of four weeks prior to receiving study medication.
  • Patients requiring ongoing treatment with non-steroidal anti-inflammatory drugs (NSAIDs). Patients must have stopped treatment a minimum of four weeks prior to receiving study medication.
  • Patients who have had surgery/fracture within 3 months prior to dosing day
  • History of cancer unresolved within 5 years prior to screening or a known precancerous state; or any form of skin cancer either current or past history
  • Women who are pregnant, lactating or who plan to become pregnant within 4 months of infusion
  • Women of childbearing potential unless taking medically acceptable contraceptive
  • Men with female partners of childbearing potential unless they are taking medically acceptable contraceptive precautions
  • Use of any investigation drug administered as part of a clinical trial within 4 weeks prior to commencing screening
  • Other clinically significant, uncontrolled medical condition that in the investigator's opinion may interfere with the assessment or follow-up
  • Active ethanol or drug abuse, excluding tobacco use
  • Electrocardiogram (ECG) abnormalities considered to be clinically significant at screening
  • Unable to comply with the requirements of the study
  • Active thrombophlebitis, thromboembolism, hypercoagulability states, bleeding, or use of anticoagulation therapy (including anti-platelet agents). Patients with a history of deep venous thrombosis may participate if successfully treated, completely resolved, and no treatment has been given for >4 months.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Cohort A
Dose Group
Biological: GC1008
1 mg/kg, IV infusion on Day 0 and monitored over 24 hours. Post infusion for safety up to 112 days.
Biological: GC1008
2 mg/kg, IV infusion on Day 0 and monitored over 24 hours. Post infusion for safety up to 112 days.
Biological: GC1008
4 mg/kg, IV infusion on Day 0 and monitored over 24 hours. Post infusion for safety up to 112 days.
Biological: GC1008
0.3 mg/kg, IV infusion on Day 0 and monitored over 24 hours. Post infusion for safety up to 112 days.
Experimental: Cohort B
Dose Group
Biological: GC1008
1 mg/kg, IV infusion on Day 0 and monitored over 24 hours. Post infusion for safety up to 112 days.
Biological: GC1008
2 mg/kg, IV infusion on Day 0 and monitored over 24 hours. Post infusion for safety up to 112 days.
Biological: GC1008
4 mg/kg, IV infusion on Day 0 and monitored over 24 hours. Post infusion for safety up to 112 days.
Biological: GC1008
0.3 mg/kg, IV infusion on Day 0 and monitored over 24 hours. Post infusion for safety up to 112 days.
Experimental: Cohort C
Dose Group
Biological: GC1008
1 mg/kg, IV infusion on Day 0 and monitored over 24 hours. Post infusion for safety up to 112 days.
Biological: GC1008
2 mg/kg, IV infusion on Day 0 and monitored over 24 hours. Post infusion for safety up to 112 days.
Biological: GC1008
4 mg/kg, IV infusion on Day 0 and monitored over 24 hours. Post infusion for safety up to 112 days.
Biological: GC1008
0.3 mg/kg, IV infusion on Day 0 and monitored over 24 hours. Post infusion for safety up to 112 days.
Experimental: Cohort D
Dose Group
Biological: GC1008
1 mg/kg, IV infusion on Day 0 and monitored over 24 hours. Post infusion for safety up to 112 days.
Biological: GC1008
2 mg/kg, IV infusion on Day 0 and monitored over 24 hours. Post infusion for safety up to 112 days.
Biological: GC1008
4 mg/kg, IV infusion on Day 0 and monitored over 24 hours. Post infusion for safety up to 112 days.
Biological: GC1008
0.3 mg/kg, IV infusion on Day 0 and monitored over 24 hours. Post infusion for safety up to 112 days.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
To determine Safety and tolerability of single dose infusions of GC1008 in patients with treatment resistant idiopathic FSGS and nephrotic range proteinuria
Time Frame: up to 2 years
up to 2 years
Pharmacokinetics of GC1008 following a single dose infusion
Time Frame: up to 2 years
up to 2 years

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
To investigate Effect of single dose infusions of GC1008 on biomarkers of clinical efficacy.
Time Frame: up to 2 years
up to 2 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Genzyme, a Sanofi Company

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
May 1, 2007

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
January 1, 2010

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
February 1, 2010

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
April 20, 2007

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 20, 2007

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
April 23, 2007

Study Record Updates

Last Update Posted (Estimate)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 19, 2014

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 17, 2014

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
March 1, 2014

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • GC1008FSGS00505

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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