Pilot Study to Evaluate the Safety and Efficacy of Abatacept in Adults and Children 6 Years and Older With Excessive Loss of Protein in the Urine Due to Either Focal Segmental Glomerulosclerosis (FSGS) or Minimal Change Disease (MCD)

NCT ID: NCT02592798

Last Updated: 2021-03-05

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

36 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-03-09

Study Completion Date

2020-01-28

Brief Summary

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The purpose of this study is evaluate if abatacept is effective and safe in decreasing the level of protein loss in the urine in patients with excessive loss of protein in the urine (nephrotic syndrome) due to either focal segmental glomerulosclerosis (FSGS) or minimal change disease (MCD). Candidates must have a prior kidney biopsy with either diagnosis. Another kidney biopsy will not be required as part of the study. Candidates must have failed or be intolerant of prior therapy for their kidney disease. The failed or intolerant therapy must include corticosteroids and at least one other drug. Candidates can be adults and children over the age of 6. Abatacept will be administered by venous infusion every 4 weeks.

Detailed Description

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Conditions

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Nephrotic Syndrome Focal Segmental Glomerulosclerosis Minimal Change Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Abatacept

* Double Blind Periods 1 and 2 (DB1 and DB2): Abatacept IV administered on Day 1, 15, 29 and then every 28 days until the end of the Double Blind Period.
* Open Label Period (OLE): Abatacept IV administered every 28 days

Group Type EXPERIMENTAL

Abatacept

Intervention Type DRUG

Abatacept IV administered on Day 1, 15, 29 and then every 28 days

Placebo

* Double Blind Periods 1 and 2 (DB1 and DB2): Normal Saline or Dextrose 5% in Water (D5W) administer on Day 1, 15, 29 and then every 28 days until the end of the Double Blind Period.
* Open Label Period (OLE): Abatacept IV administered every 28 days

Group Type PLACEBO_COMPARATOR

Normal Saline

Intervention Type OTHER

Normal Saline administer on Day 1, 15, 29 and then every 28 days

D5W

Intervention Type OTHER

Dextrose 5% in Water (D5W) administered on Day 1, 15, 29 and then every 28 days

Interventions

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Abatacept

Abatacept IV administered on Day 1, 15, 29 and then every 28 days

Intervention Type DRUG

Normal Saline

Normal Saline administer on Day 1, 15, 29 and then every 28 days

Intervention Type OTHER

D5W

Dextrose 5% in Water (D5W) administered on Day 1, 15, 29 and then every 28 days

Intervention Type OTHER

Other Intervention Names

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5% Dextrose in Water

Eligibility Criteria

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Inclusion Criteria

* Male and female subjects ages ≥ 6 years
* Subjects resistant to corticosteroids, calcineurin inhibitors (cyclosporine and tacrolimus), sirolimus, mycophenolate mofetil (MMF), mycophenolic acid (MPA), or cyclophosphamide or intolerant to at least 2 of these
* UPCR ≥ 3 at screening
* FSGS or MCD confirmed by renal biopsy
* eGFR ≥ 45 for children and adults
* Concomitant use of angiotensin-converting-enzyme inhibitor (ACEi) or angiotensin receptor blocker (ARB) at stable doses for at least 2 weeks or have intolerance documented in the source documents maintained at the site

Exclusion Criteria

* Kidney diseases other than FSGS or MCD
* Collapsing FSGS
* Systemic lupus erythematosus
* Diabetes mellitus, both type 1 and type 2
* Clinically significant congestive heart failure
* Post renal transplantation, including relapsing post-transplant FSGS
* Body mass index (BMI): \> 40 in subjects ≥ 18 years of age and ≥ 99% percentile for subjects \< 18 years of age
Minimum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Bristol-Myers Squibb

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Bristol Myers Squibb

Role: STUDY_DIRECTOR

Bristol-Myers Squibb

Locations

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The University Of Alabama At Birmingham

Birmingham, Alabama, United States

Site Status

University of Alabama-Birmingham-Parent Account

Birmingham, Alabama, United States

Site Status

Los Angeles Biomedical Research Institute

Torrance, California, United States

Site Status

University Of Colorado Denver

Aurora, Colorado, United States

Site Status

Children's National Health System

Washington D.C., District of Columbia, United States

Site Status

University Of Miami Miller School Of Medicine

Miami, Florida, United States

Site Status

Nemours Childrens Hospital

Orlando, Florida, United States

Site Status

Emory University

Atlanta, Georgia, United States

Site Status

Loyola University Medical Center

Maywood, Illinois, United States

Site Status

NIH Clinical Center - NIDDK

Bethesda, Maryland, United States

Site Status

Boston Childrens Hospital

Boston, Massachusetts, United States

Site Status

Brigham And Women'S Hosp Inc.

Boston, Massachusetts, United States

Site Status

University of Michigan

Ann Arbor, Michigan, United States

Site Status

Mayo Clinic

Rochester, Minnesota, United States

Site Status

Childrens Mercy Hospital

Kansas City, Missouri, United States

Site Status

New York University Langone Medical Center

New York, New York, United States

Site Status

Columbia University Medical Center (Cumc)

New York, New York, United States

Site Status

Levine Children's Hospital

Charlotte, North Carolina, United States

Site Status

Duke University

Durham, North Carolina, United States

Site Status

Cincinnati Children'S Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

The Metro Health System

Cleveland, Ohio, United States

Site Status

Cleveland Clinic

Cleveland, Ohio, United States

Site Status

The Ohio State University Wexner Medical Center

Columbus, Ohio, United States

Site Status

University Of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status

Local Institution

Pittsburgh, Pennsylvania, United States

Site Status

Renal Disease Research Institute

Dallas, Texas, United States

Site Status

University Of Texas Southwestern Medical Center

Dallas, Texas, United States

Site Status

Countries

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United States

References

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Liu ID, Willis NS, Craig JC, Hodson EM. Interventions for idiopathic steroid-resistant nephrotic syndrome in children. Cochrane Database Syst Rev. 2025 May 8;5(5):CD003594. doi: 10.1002/14651858.CD003594.pub7.

Reference Type DERIVED
PMID: 40337980 (View on PubMed)

Azukaitis K, Palmer SC, Strippoli GF, Hodson EM. Interventions for minimal change disease in adults with nephrotic syndrome. Cochrane Database Syst Rev. 2022 Mar 1;3(3):CD001537. doi: 10.1002/14651858.CD001537.pub5.

Reference Type DERIVED
PMID: 35230699 (View on PubMed)

Hodson EM, Sinha A, Cooper TE. Interventions for focal segmental glomerulosclerosis in adults. Cochrane Database Syst Rev. 2022 Feb 28;2(2):CD003233. doi: 10.1002/14651858.CD003233.pub3.

Reference Type DERIVED
PMID: 35224732 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

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Other Identifiers

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2015-005450-36

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

IM101-566

Identifier Type: -

Identifier Source: org_study_id

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