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)
A Phase II Randomized, Placebo-Controlled, Double-Blind, Parallel Arms, Pilot Study to Evaluate the Efficacy and Safety of Intravenous Abatacept in Treatment Resistant Nephrotic Syndrome (Focal Segmental Glomerulosclerosis/ Minimal Change Disease)
2 other identifiers
interventional
36
1 country
27
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started Mar 2016
Typical duration for phase_2
27 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
October 29, 2015
CompletedFirst Posted
Study publicly available on registry
October 30, 2015
CompletedStudy Start
First participant enrolled
March 9, 2016
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 28, 2020
CompletedStudy Completion
Last participant's last visit for all outcomes
January 28, 2020
CompletedResults Posted
Study results publicly available
March 5, 2021
CompletedMarch 5, 2021
February 1, 2021
3.9 years
October 29, 2015
January 19, 2021
February 10, 2021
Conditions
Outcome Measures
Primary Outcomes (1)
Percentage of Participants in Renal Response at Day 113
Renal Response is defined as the presence of all the following criteria: PROTEINURIA: Reduction of baseline urine protein/creatinine ratio (UPCR) of \>= 50% and to less than 3. RENAL FUNCTION: No worsening of baseline estimated glomerular filtration rate (eGFR) defined as within normal range if normal at baseline or ≥ 75% baseline value if below normal at baseline.
From first dose to 113 days following first dose of the indicated period (113 days for Double-Blind Period, 226 days for Open Label Period)
Secondary Outcomes (13)
Mean Change From Baseline in Urine Protein/Creatinine Ratio (UPCR) at Day 113
From baseline (measured at day 1 of study) to 113 days following first dose administered in the indicated treatment period (113 days for Double-Blind Period, 226 days for Open Label Period)
Mean Change From Baseline in Serum Albumine at Day 113
From baseline (measured at day 1 of the study) to 113 days following first dose administered in the indicated treatment period (113 days for Double-Blind Period, 226 days for Open Label Period)
Percentage of Participants Achieving Complete Remission at Day 113
From first dose to 113 days following first dose of the indicated period (113 days for Double-Blind Period, 226 days for Open Label Period)
Mean Change From Baseline in Patient Reported Outcomes Measurement Information System (PROMIS) at Day 113 - Adult Participants
From baseline (measured at day 1 of study) to 113 days following first dose administered in the Double-Blind Period
Mean Change From Baseline in Patient Reported Outcomes Measurement Information System (PROMIS) at Day 113 - Pediatric Participants
From baseline (measured at day 1 of study) to 113 days following first dose administered in the Double-Blind Period
- +8 more secondary outcomes
Study Arms (2)
Abatacept
EXPERIMENTAL* 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
Placebo
PLACEBO COMPARATOR* 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
Interventions
Dextrose 5% in Water (D5W) administered on Day 1, 15, 29 and then every 28 days
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (27)
The University Of Alabama At Birmingham
Birmingham, Alabama, 35233, United States
University of Alabama-Birmingham-Parent Account
Birmingham, Alabama, 35294, United States
Los Angeles Biomedical Research Institute
Torrance, California, 90502, United States
University Of Colorado Denver
Aurora, Colorado, 80045, United States
Children's National Health System
Washington D.C., District of Columbia, 20010, United States
University Of Miami Miller School Of Medicine
Miami, Florida, 33136, United States
Nemours Childrens Hospital
Orlando, Florida, 32827, United States
Emory University
Atlanta, Georgia, 30322-1015, United States
Loyola University Medical Center
Maywood, Illinois, 60153, United States
NIH Clinical Center - NIDDK
Bethesda, Maryland, 20892, United States
Boston Childrens Hospital
Boston, Massachusetts, 02115, United States
Brigham And Women'S Hosp Inc.
Boston, Massachusetts, 02115, United States
University of Michigan
Ann Arbor, Michigan, 48109, United States
Mayo Clinic
Rochester, Minnesota, 55905, United States
Childrens Mercy Hospital
Kansas City, Missouri, 64108, United States
New York University Langone Medical Center
New York, New York, 10016, United States
Columbia University Medical Center (Cumc)
New York, New York, 10032, United States
Levine Children's Hospital
Charlotte, North Carolina, 28203, United States
Duke University
Durham, North Carolina, 27701, United States
Cincinnati Children'S Hospital Medical Center
Cincinnati, Ohio, 45229-3039, United States
The Metro Health System
Cleveland, Ohio, 44109, United States
Cleveland Clinic
Cleveland, Ohio, 44195, United States
The Ohio State University Wexner Medical Center
Columbus, Ohio, 43210, United States
University Of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
Local Institution
Pittsburgh, Pennsylvania, 15224, United States
Renal Disease Research Institute
Dallas, Texas, 75235, United States
University Of Texas Southwestern Medical Center
Dallas, Texas, 75390, United States
Related Publications (3)
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.
PMID: 40337980DERIVEDAzukaitis 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.
PMID: 35230699DERIVEDHodson 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.
PMID: 35224732DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Limitations and Caveats
Amendment 02 of the Clinical Protocol (18 April 2018) modified the study design, so that the Double-Blind Period (DB) would not include anymore 2 consecutive periods (DB1 and DB2). However, for transparency and completeness reasons, in the Participant Flow and Adverse Events sections data about DB1 and DB2 are reported separately.
Results Point of Contact
- Title
- Bristol-Myers Squibb Study Director
- Organization
- Bristol-Myers Squibb
Study Officials
- STUDY DIRECTOR
Bristol Myers Squibb
Bristol-Myers Squibb
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 29, 2015
First Posted
October 30, 2015
Study Start
March 9, 2016
Primary Completion
January 28, 2020
Study Completion
January 28, 2020
Last Updated
March 5, 2021
Results First Posted
March 5, 2021
Record last verified: 2021-02