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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00464321
• Date of registration: 20/04/2007
• Prospective Registration: Yes
• Primary sponsor: Genzyme, a Sanofi Company
• Public title: Safety Study of GC1008 in Patients With Focal Segmental Glomerulosclerosis (FSGS) of Single Doses of GC1008 in Patients With Treatment Resistant Idiopathic FSGS
• Scientific 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)
• Date of first enrolment: May 2007
• Target sample size: 16
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00464321
• Study type: Interventional
• Study design: Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 1

Countries of recruitment

Germany, Italy, United Kingdom, United States

Contacts

• Name: Medical Monitor
• Affiliation: Genzyme, a Sanofi Company

Key inclusion & exclusion criteria

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.



Age minimum: 18 Years
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Focal Segmental Glomerulosclerosis

Intervention(s)

Biological: GC1008

Primary Outcome(s)

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]

Secondary Outcome(s)

To investigate Effect of single dose infusions of GC1008 on biomarkers of clinical efficacy. [Time Frame: up to 2 years]

Secondary ID(s)

GC1008FSGS00505

Source(s) of Monetary Support

Please refer to primary and secondary sponsors