Acthar for Treatment-Resistant or Treatment-Intolerant Proteinuria (PODOCYTE)

Mallinckrodt

Status and phase: Completed; Phase 4

Conditions: Idiopathic Focal Segmental Glomerulosclerosis

Treatments: Drug: Acthar Gel

Study type: Interventional

Funder types: Industry

Identifiers

NCT02633046

MNK14224049

Details and patient eligibility

About

Focal segmental glomerulosclerosis (FSGS) is a condition that harms the kidney "filters" that remove waste from the blood. Proteins are supposed to stay in the blood. Damaged "filters" let protein get into the kidney.

FSGS is a serious condition that can lead to kidney failure. The only treatment for kidney failure is dialysis or kidney transplant.

Proteinuria means too much protein came through the kidneys into the urine.

If the doctor cannot figure out what is causing the problem, it is primary (idiopathic) FSGS. This kind of FSGS is very hard to treat.

This study will test Acthar in patients with this condition who have not responded to other treatments. It primarily investigates how well the therapy is tolerated by the patients and how well they respond to this treatment.

Enrollment: 63 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria

Potential participants must meet the following summary criteria for inclusion in the study:

• Is male or non-pregnant, non-lactating female
• Has primary FSGS, is nephrotic and did not achieve at least partial proteinuria response with prior therapy
• Has been treated with an angiotensin converting enzyme inhibitor or receptor blocker (or have documented intolerance), for at least 4 weeks before screening
• Has blood pressure no higher than 150/90 mmHg
• Meets all other inclusion criteria detailed in the protocol

Exclusion Criteria

Potential participants will not be eligible for the the study if they meet the following summary criteria:

• Has hepatitis B or C, tuberculosis, or other contraindication listed on the United States (US) Prescribing Information for Acthar

• Has Type 1 or Type 2 diabetes mellitus or any clinically significant infection

• Has received specific treatments at exclusionary time points per protocol

• Has been involved in a therapeutic drug/device trial (other than for FSGS) within 4 weeks before screening

• Meets any other exclusion criteria detailed in the protocol

• Has any other condition that might, per protocol or in the opinion of the investigator, compromise:

  1. the safety and well-being of the participant or their offspring
  2. the safety of study staff
  3. analysis of results

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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63 participants in 1 patient group

Acthar Gel

Other group

Description:

Acthar Gel, 1 mL (80 U) by subcutaneous injection (SC) 3x/week will be administered to all participants from Week 0 to 50. Tapering of dose to 1 mL SC 2x/week will be allowed for safety and/ tolerability issues. Once the dose is tapered to 1 mL SC 2x/week it must remain at this level. Participants unable to tolerate 1 mL SC 2x/week will be discontinued. All participants will have an End of Study/Early Termination Visit 4 weeks after discontinuing Investigational Medicinal Product (IMP).

Treatment:

Drug: Acthar Gel