A Phase 2a Study of Weekly Doses of GCS-100 in Patients With Chronic Kidney Disease

NCT01843790 | Completed Phase 2

• 1 other identifier: GCS-100-CS-4002
• Study Type: interventional
• Target: 120
• Locations: 1 country
• Sites: 4

Brief Summary

The primary objective of this study is to compare the change in estimated glomerular filtration rate (eGFR) from baseline to Week 8 between placebo and GCS-100 treatment. The secondary objective is to determine the safety and tolerability of GCS-100 administered for 8 weeks relative to placebo. In addition, the study will measure the effect of GCS-100 on circulating galectin-3 and other markers of disease activity.

Conditions

Chronic Kidney Disease

Outcome Measures

Primary Outcomes (1)

• Change in estimated glomerular filtration rate (eGFR) from baseline relative to placebo after administration of GCS-100 for 8 weeks in patients with chronic kidney disease (CKD) and baseline eGFR of 15 - 44 mL/min/1.73m2

  12 weeks

Secondary Outcomes (1)

• Number of adverse events as a measure of safety and tolerability of GCS-100 administered for 8 weeks relative to placebo in patients with CKD

  12 weeks

Study Arms (3)

Placebo, saline

PLACEBO COMPARATOR

Saline, dosed weekly for 8 weeks

Drug: Placebo, Saline

GCS-100 low dose

EXPERIMENTAL

Low dose of GCS-100 given IV once per week for 8 weeks

Drug: GCS-100

GCS-100 high dose

EXPERIMENTAL

High dose of GCS-100 given IV once per week for 8 weeks

Drug: GCS-100

Interventions

GCS-100 DRUG

The amount (in mg) of GCS-100 to be administered will be determined based on body surface area, which will be calculated based on body weight and height. The study drug dose should be calculated on Day 1. Drug should be added to an infusion bag or syringe containing 0.9% Sodium Chloride Injection, USP. Drug be infused into a large vein via a large bore catheter to avoid local irritation and administered at no more than 2 mg/mL in no less than 60 mL.

GCS-100 high dose; GCS-100 low dose

Placebo, Saline DRUG

The amount (in mg) of drug to be administered will be determined based on body surface area, which will be calculated based on body weight and height. The drug dose should be calculated on Day 1. Drug should be added to an infusion bag or syringe containing 0.9% Sodium Chloride Injection, USP. Drug be infused into a large vein via a large bore catheter to avoid local irritation and administered at no more than 2 mg/mL in no less than 60 mL.

Also known as: Saline solution, 0.9% Sodium Chloride Injection

Placebo, saline

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Subject is capable of understanding the purpose and risks of the study and is able to provide written informed consent
• Subject is ≥18 and ≤75 years of age; patients \>75 years old may be included at the request of the investigator and discretion of the Medical Monitor
• Subject has an eGFR of 15 - 44 mL/min/1.73m2 determined using the 4-variable Modification of Diet in Renal Disease (MDRD) equation (see Section 10.1)
• Patients with CKD diagnosis \>12 months and stable, in the opinion of the investigator, within the past 3 months
• Subject is willing and able to comply with all protocol requirements
• Female subjects of childbearing potential (i.e., women who have not been surgically sterilized or have not been post-menopausal for at least 1 year) and male subjects with partners of childbearing potential must agree to use medically acceptable methods of contraception throughout the study period

You may not qualify if:

• Treatment with an experimental (unlicensed) drug within 4 weeks or ≤5 half-lives prior to screening
• Kidney disease due to systemic lupus erythematosus (regardless of whether active or in remission), any form of vasculitis (regardless of whether active or in remission), IgA nephropathy, multiple myeloma, polycystic kidney disease, untreated obstructed nephropathy or any other causes that, in the opinion of the investigator, may put the subject at an increased risk
• Planned renal replacement therapy of any kind within 6 months of randomization
• Previous solid organ transplant
• Systolic blood pressure ≤90 mmHg and ≥160 mmHg and diastolic blood pressure ≤40 mmHg and ≥100 mmHg at screening
• Subject has clinical laboratory values of:
• Hemoglobin: ≤9 g/dL
• Total bilirubin: \>1.5X the upper limit of normal (ULN)
• ALT and/or AST: \>2.5X ULN
• Current treatment with immunosuppressive agents, except for topical agents or inhaled steroids when conditions are chronic and stable
• Treatment with any form of IV iron therapy within 4 weeks prior to screening
• Known history of cancer (excluding non-melanoma skin cancer that is not being actively treated) within 5 years of screening
• Known history of human immunodeficiency virus, active hepatitis C virus (HCV), active hepatitis B virus (HBV), or prior history of infection with HBV (HBcAb positive); if adequate hepatic function has been documented for patients with HCV or prior history of hepatitis B without evidence of cirrhosis, the Medical Monitor may approve their enrollment
• Clinically relevant active infection and/or a serious co-morbid medical condition, such as recent myocardial infarction (within the last 6 months), unstable angina, difficult-to-control congestive heart failure, uncontrolled hypertension, difficult-to-control cardiac arrhythmias, severe or uncontrolled chronic obstructive or chronic restrictive pulmonary disease, and/or cirrhosis.
• Subject had major surgery within 4 weeks of randomization

Sponsors & Collaborators

• La Jolla Pharmaceutical Company: lead

Study Sites (4)

Southwest Clinical Research Institute, LLC

Tempe, Arizona, 85284, United States

Location

California Institute of Renal Research

La Mesa, California, 91942, United States

Location

Denver Nephrology

Denver, Colorado, 80230, United States

Location

Clinical Advancement Center, PLLC

San Antonio, Texas, 78215, United States

Location

MeSH Terms

Conditions

Renal Insufficiency, Chronic

Interventions

GCS-100; Sodium Chloride; Saline Solution

Condition Hierarchy (Ancestors)

Renal Insufficiency; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Chlorides; Hydrochloric Acid; Chlorine Compounds; Inorganic Chemicals; Sodium Compounds; Crystalloid Solutions; Isotonic Solutions; Solutions; Pharmaceutical Preparations

Study Officials

• George Tidmarsh, MD, PhD

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: PARTICIPANT
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 25, 2013
• First Posted: May 1, 2013
• Study Start: June 1, 2013
• Primary Completion: February 1, 2014
• Study Completion: September 1, 2014
• Last Updated: September 1, 2015

Record last verified: 2015-08

Locations

US (4)