Focal Segmental GlomerulosclerosisPhase 1NCT00464321
Safety Study of GC1008 in Patients With Focal Segmental Glomerulosclerosis (FSGS) of Single Doses of GC1008 in Patients With Treatment Resistant Idiopathic FSGS
A Phase 1 study testing GC1008 in people with Focal Segmental Glomerulosclerosis, run by Genzyme, a Sanofi Company at 9 sites in 4 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Focal Segmental Glomerulosclerosis
- What is being tested?
- GC1008
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 9 sitesin 4 countries
- Study dates
- May 2007to Feb 2010
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
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- 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.
Trial locations and contacts
9 sites in 4 countries.
9 sites in 4 countries
United States4 sites
Study site
Chapel Hill, North Carolina
Study site
New Hyde Park, New York, 11042-1433
Study site
Rochester, Minnesota
Study site
San Francisco, California
About this study
Official title: 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.
Read the 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
- Conditions
- Focal Segmental Glomerulosclerosis
- Being tested
- GC1008
- Medical subject headings
- Glomerulosclerosis, Focal Segmental
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GC1008Biological
1 mg/kg, IV infusion on Day 0 and monitored over 24 hours. Post infusion for safety up to 112 days.
GC1008Biological
2 mg/kg, IV infusion on Day 0 and monitored over 24 hours. Post infusion for safety up to 112 days.
GC1008Biological
4 mg/kg, IV infusion on Day 0 and monitored over 24 hours. Post infusion for safety up to 112 days.
GC1008Biological
0.3 mg/kg, IV infusion on Day 0 and monitored over 24 hours. Post infusion for safety up to 112 days.
| Study group | Role | What they receive |
|---|---|---|
| Cohort A | Experimental | Dose Group Receives: Biological: GC1008 |
| Cohort B | Experimental | Dose Group Receives: Biological: GC1008 |
| Cohort C | Experimental | Dose Group Receives: Biological: GC1008 |
| Cohort D | Experimental | Dose Group Receives: Biological: GC1008 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine Safety and tolerability of single dose infusions of GC1008 in patients with treatment resistant idiopathic FSGS and nephrotic range proteinuria
Measured: up to 2 years
Pharmacokinetics of GC1008 following a single dose infusion
Measured: up to 2 years
Secondary outcomes (1)
To investigate Effect of single dose infusions of GC1008 on biomarkers of clinical efficacy.
Measured: up to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Apr 2007 (estimated)
- Study startMay 2007
- Primary completionJan 2010
- Study completionFeb 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Focal Segmental GlomerulosclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genzyme, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, Study director, Genzyme, a Sanofi Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00464321
- Sponsor's protocol ID
- GC1008FSGS00505
- Status last verified
- Mar 2014
- First submitted
- 20 Apr 2007
- Last updated
- 19 Mar 2014
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 19 Mar 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I, Multicentre, Open-label, Dose-escalating Study of Single Doses of GC1008 in Patients With Treatment Resistant Idiopathic Focal Segmental Glomerulosclerosis (FSGS)
- ClinicalTrials.gov ID
- NCT00464321
- Acronym / sponsor ID
- GC1008FSGS00505
- Conditions
- Focal Segmental Glomerulosclerosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Study start
- May 2007
- Primary completion
- Jan 2010 (actual)
- Study completion
- Feb 2010 (actual)
- First posted
- 23 Apr 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Cohort A | Experimental | Biological: GC1008Dose Group |
| Cohort B | Experimental | Biological: GC1008Dose Group |
| Cohort C | Experimental | Biological: GC1008Dose Group |
| Cohort D | Experimental | Biological: GC1008Dose Group |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (19 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GC10081 mg/kg, IV infusion on Day 0 and monitored over 24 hours. Post infusion for safety up to 112 days. | Biological | Cohort A |
| GC10082 mg/kg, IV infusion on Day 0 and monitored over 24 hours. Post infusion for safety up to 112 days. | Biological | Cohort B |
| GC10084 mg/kg, IV infusion on Day 0 and monitored over 24 hours. Post infusion for safety up to 112 days. | Biological | Cohort C |
| GC10080.3 mg/kg, IV infusion on Day 0 and monitored over 24 hours. Post infusion for safety up to 112 days. | Biological | Cohort D |
Primary outcome
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
Pharmacokinetics of GC1008 following a single dose infusion
Time frame: up to 2 years
1 secondary outcome
To investigate Effect of single dose infusions of GC1008 on biomarkers of clinical efficacy.
Time frame: up to 2 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Mar 2014.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Monitor study director
Genzyme, a Sanofi Company
9 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing GC1008 in people with Focal Segmental Glomerulosclerosis, run by Genzyme, a Sanofi Company at 9 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Mar 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States, Germany, Italy, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Mar 2014.