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)

Bristol-Myers Squibb (BMS)

Status and phase: Completed; Phase 2

Conditions: Minimal Change Disease; Focal Segmental Glomerulosclerosis; Nephrotic Syndrome

Treatments: Other: Normal Saline; Drug: Abatacept; Other: D5W

Study type: Interventional

Funder types: Industry

Identifiers

NCT02592798

2015-005450-36 (EudraCT Number)

IM101-566

Details and patient eligibility

About

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.

Enrollment: 36 patients

Sex: All

Ages: 6+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

---

36 participants in 2 patient groups, including a placebo group

Abatacept

Experimental group

Description:

* 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

Treatment:

Drug: Abatacept

Placebo

Placebo Comparator group

Description:

* 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

Treatment:

Other: Normal Saline

Other: D5W