Safety and Efficacy of Mesenchymal Precursor Cells in Diabetic Nephropathy

Sponsor

Mesoblast, Ltd. (Industry)

Overall Status

Completed

CT.gov ID

NCT01843387

Collaborator

(none)

Enrollment

Locations

Arms

Duration (Months)

Patients Per Site

0.6

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The study investigates the safety, tolerability and efficacy of a single intravenous infusion of two doses of mesenchymal precursor cells versus placebo in subjects with diabetic nephropathy and type 2 diabetes.

Condition or Disease	Intervention/Treatment	Phase
Diabetic Nephropathy Type 2 Diabetes	Biological: Mesenchymal Precursor Cells (MPCs) Biological: Mesenchymal Precursor Cells (MPCs)	Phase 1/Phase 2

Detailed Description

This study is taking place in Melbourne, Australia.

Study Design

Study Type:

Interventional

Actual Enrollment :

30 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Triple (Participant, Investigator, Outcomes Assessor)

Primary Purpose:

Treatment

Official Title:

A Randomized, Controlled, Dose-Escalation Pilot Study to Assess the Safety and Efficacy of a Single Intravenous Infusion of Allogeneic Mesenchymal Precursor Cells (MPCs) in Subjects With Diabetic Nephropathy and Type 2 Diabetes

Study Start Date :

Jul 1, 2013

Actual Primary Completion Date :

Sep 1, 2014

Actual Study Completion Date :

Sep 1, 2015

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Cohort 1 Mesenchymal Precursor Cells (MPCs) - Dose 1 or Placebo	Biological: Mesenchymal Precursor Cells (MPCs) Single Intravenous Infusion of MPCs Dose 1 or Placebo
Experimental: Cohort 2 Mesenchymal Precursor Cells (MPCs) - Dose 2 or Placebo	Biological: Mesenchymal Precursor Cells (MPCs) Single Intravenous Infusion of MPCs Dose 2 or Placebo

Arm

Intervention/Treatment

Experimental: Cohort 1

Mesenchymal Precursor Cells (MPCs) - Dose 1 or Placebo

Biological: Mesenchymal Precursor Cells (MPCs)

Single Intravenous Infusion of MPCs Dose 1 or Placebo

Experimental: Cohort 2

Mesenchymal Precursor Cells (MPCs) - Dose 2 or Placebo

Biological: Mesenchymal Precursor Cells (MPCs)

Single Intravenous Infusion of MPCs Dose 2 or Placebo

Outcome Measures

Primary Outcome Measures

The primary objective of the study is to assess the safety and tolerability of MPC therapy [60 Weeks]
Outcomes include the following safety parameters: Number of and percent of subject with adverse events and serious adverse events Clinically significant values and shifts from baseline in vital signs, physical examinations and electrocardiograms Clinical laboratory tests (hematology, chemistry and urinalysis, flow cytometry with Class I and Class II PRA % with specificity)

Secondary Outcome Measures

Exploratory assessment of the efficacy of MPC therapy [12 Weeks]
Outcomes include changes from baseline at 12 weeks in the following parameters: Renal function (glomerular filtration rate, renal blood flow) Serum creatinine Urinary albumin and protein excretion Glycemic control Biomarkers

Eligibility Criteria

Criteria

Ages Eligible for Study:

50 Years to 85 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Men and women who are ≥ 50 and ≤ 85 years old
Subjects diagnosed with type 2 diabetes at least 2 years prior to Screening
Subjects with diabetic nephropathy and CKD stage 3b-4
Albumin-to-creatinine ratio (ACR) from a spot urine sample >30 and < 3000 mg/g at Screening
Subjects must be receiving standard of care treatment for their diabetic nephropathy with an angiotensin converting enzyme inhibitor (ACEi) and/or an angiotensin II receptor blocker (ARB) for at least 12 weeks prior to Screening.
HbA1c < 10.0% at Screening

Exclusion Criteria:

Prior participation in any stem cell study
Women of childbearing potential
Potentially unreliable subjects and those judged by the Investigator to be unsuitable for the study
History of active substance abuse (including alcohol) within the past 2 years. Current alcohol abuse is defined as daily consumption of >3 alcoholic beverages (i.e. > 21 alcoholic beverages per week)
Body weight >150 kg
Subjects with non-diabetic renal disease e.g. known polycystic kidney disease
Subjects with a history of a renal transplant or who have had prior dialysis within 3 months of Screening and/or have not maintained a stable level of kidney function within 3 months of Screening
Current or history within 6 months of Screening of NYHA Class III or IV heart failure
Myocardial infarction or stroke within 6 months prior to Screening
Any concurrent medical condition/disorder or clinically symptomatic cardiovascular, gastrointestinal, hematological, pulmonary, acute or chronic infectious disease, active retinal disease or other disorder which in the Investigator's opinion would interfere with the subjects ability to complete the trial, would require administration of treatment that could affect the interpretation of the efficacy and safety variables or would preclude safe involvement in the study