Phase 2
N=36
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)
Nephrotic Syndrome · Focal Segmental Glomerulosclerosis · Minimal Change Disease
Bottom Line
View on ClinicalTrials.gov: NCT02592798 ↗Enrolled (actual)
36
Serious AEs
21.0%
Results posted
Mar 2021
Primary outcome: Primary: Percentage of Participants in Renal Response at Day 113 — 0.0; 7.7; 12.5; 33.3 Percent of Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Abatacept (Drug); Normal Saline (Other); D5W (Other)
- Age
- Pediatric, Adult, Older Adult · 6+ yrs
- Sex
- All
- Sponsor
- Bristol-Myers Squibb
- Primary completion
- Jan 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants in Renal Response at Day 113 |
0.0; 7.7; 12.5; 33.3 | — |
| SECONDARY Mean Change From Baseline in Urine Protein/Creatinine Ratio (UPCR) at Day 113 |
0.12; -0.25; 1.98; 0.02 | — |
| SECONDARY Mean Change From Baseline in Serum Albumine at Day 113 |
0.08; -0.05; 0.21; 0.09 | — |
| SECONDARY Percentage of Participants Achieving Complete Remission at Day 113 |
0.0; 0.0; 12.5; 11.1 | — |
| SECONDARY Mean Change From Baseline in Patient Reported Outcomes Measurement Information System (PROMIS) at Day 113 - Adult Participants |
-5.56; -4.87; -4.88; -1.18; 0.78; 1.77 | — |
| SECONDARY Mean Change From Baseline in Patient Reported Outcomes Measurement Information System (PROMIS) at Day 113 - Pediatric Participants |
6.43; -6.02; 5.70; -1.44; 0.80; 1.68 | — |
| SECONDARY Number of Participants Experiencing Adverse Events |
13; 15; 26; 5; 4; 10 | — |
| SECONDARY Number of Participants Experiencing Adverse Events of Special Interest |
6; 3; 8; 9; 0; 0 | — |
| SECONDARY Percentage of Participants With Positive Antibody Response Relative to Baseline |
7.1; 0.0; 40.0; 66.7; 28.6; 66.7 | — |
| SECONDARY Minimum Blood Plasma Concentration (Cmin) of Abatacept - Adult Participants |
7.613; 12.274; 3.641; 4.101; 2.786 | — |
| SECONDARY Minimum Blood Plasma Concentration (Cmin) of Abatacept - Pediatric Participants |
2.829; 2.588; 0.504; 1.617; 0.990 | — |
| SECONDARY Maximum Observed Serum Concentration (Cmax) of Abatacept |
200.46; 174.65 | — |
| SECONDARY Area Under the Serum Concentration Time Curve Over a Dosing Interval (AUC(TAU)) of Abatacept |
20394.83; 18282.50 | — |
| SECONDARY Time to Reach Peak Serum Concentration (Tmax(h)) of Abatacept |
0.883; 0.589 | — |
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.
Eligibility Criteria
For more information regarding BMS clinical trial participation, please visit www.BMSStudyConnect.com
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
Other protocol defined inclusion/exclusion criteria may apply
Data sourced from ClinicalTrials.gov (NCT02592798). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.