A Phase 2 Extension Study of Study GCS-100-CS-4002

The primary objective of this study is to determine the safety and tolerability of extended dosing of GCS-100 in patients with CKD.

Study Overview

• Status: Completed
• Conditions: Chronic Kidney Disease
• Intervention / Treatment: Drug: GCS-100

Detailed Description

Galectin-3 contributes to fibrosis, is elevated in patients with ESRD, and correlates with adverse outcomes. Animal models with genetic knockout of galectin-3 demonstrate a reduction in structural and functional deficits in the kidney. GCS-100 is a galectin-3 antagonist that has been shown to reduce fibrosis pre-clinically. Based on the role of galectin-3 and fibrosis in kidney disease, the Sponsor believes GCS-100 may be effective at treating patients with CKD.

Study GCS-100-CS-4002 is an ongoing randomized, placebo controlled study of GCS-100 in Stage 3b and 4 CKD. The objective of the study is to determine the safety and efficacy of 8 weekly injections of GCS-100 on eGFR. Patients who have completed that study without adverse safety events, may be asked to participate in this study to test the safety of prolonged administration of study drug.

• Study Type: Interventional
• Enrollment (Actual): 92
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Tempe, Arizona, United States, 85284
      • Southwest Clinical Research Institute, LLC
  • California
    • La Mesa, California, United States, 91942
      • California Institute of Renal Research
  • Colorado
    • Denver, Colorado, United States, 80230
      • Denver Nephrology
  • North Carolina
    • Asheville, North Carolina, United States, 28801
      • Mountain Kidney and Hypertension Associates, PA
  • Texas
    • San Antonio, Texas, United States, 78215
      • Clinical Advancement Center, PLLC

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 80 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Subject is capable of understanding the purpose and risks of the study and is able to provide written informed consent
2. Subject was enrolled in and completed the End of Study (Day 85) of GCS-100-CS-4002

Exclusion Criteria:

1. Subject experienced a Grade 3 or worse adverse event related to GCS-100 in clinical study GCS-100-CS-4002
2. Systolic blood pressure ≤90 mmHg and ≥160 mmHg and diastolic blood pressure ≤40 mmHg and ≤100 mmHg at screening

3. Subject has clinical laboratory results of:

   1. Hemoglobin: ≤9g/dL
   2. Total bilirubin: >1.5X the upper limit of normal (ULN)
   3. ALT and/or AST: >2.5X ULN
4. Subject has a concomitant disease or condition, including laboratory abnormalities, which, in the opinion of the investigator, could interfere with the conduct of the study or put the subject at unacceptable risk
5. Subject who may require renal replacement therapy within the next 2 months, at the discretion of the investigator

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Single

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Low Dose GCS-100; Low dose of GCS-100	Drug: GCS-100; 1.5 mg/m2 or 30 mg/m2 GCS-100. GCS-100 will be administered as IV infusions once weekly for 8 weeks with a 1 week and 4 week follow-up period and then at least every 30 days for up to a total of 1 year.
Experimental: High Dose GCS-100; GCS-100 High dose	Drug: GCS-100; 1.5 mg/m2 or 30 mg/m2 GCS-100. GCS-100 will be administered as IV infusions once weekly for 8 weeks with a 1 week and 4 week follow-up period and then at least every 30 days for up to a total of 1 year.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Evaluation of Safety	Evaluation of safety measures for long-term dosing including collection of adverse events in clinically significant changes in lab values	Participants will be followed for the duration of the study, up to 1 year with a four week additional follow-up period.

Collaborators and Investigators

• Sponsor: La Jolla Pharmaceutical Company
• Investigators: Study Director: George Tidmarsh, MD, PhD

Study record dates

Study Major Dates

• Study Start: January 1, 2014
• Primary Completion (Actual): May 1, 2016
• Study Completion (Actual): June 1, 2016

Study Registration Dates

• First Submitted: April 9, 2014
• First Submitted That Met QC Criteria: June 2, 2014
• First Posted (Estimate): June 4, 2014

Study Record Updates

• Last Update Posted (Estimate): December 26, 2016
• Last Update Submitted That Met QC Criteria: December 22, 2016
• Last Verified: December 1, 2016

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Urologic Diseases; Renal Insufficiency; Kidney Diseases; Renal Insufficiency, Chronic
• Other Study ID Numbers: GCS-100-CS-4003