A Study of Cemdisiran in Adults With Immunoglobulin A Nephropathy (IgAN)

Alnylam Pharmaceuticals

Status and phase: Terminated; Phase 2

Conditions: Glomerulonephritis, IgA; IgA Nephropathy (IgAN); Berger Disease

Treatments: Drug: Placebo; Drug: Cemdisiran

Study type: Interventional

Funder types: Industry

Identifiers

NCT03841448

ALN-CC5-005

2018-002716-27 (EudraCT Number)

Details and patient eligibility

About

The purpose of this study is to evaluate the effect of cemdisiran on proteinuria in adults with immunoglobulin A nephropathy (IgAN), who excrete >1 gram (gm) of protein per day despite standard of care, which includes treatment with angiotensin-converting enzyme (ACE) inhibitors or angiotensin receptor blockers (ARB). These participants are at high risk for progression of kidney disease, which can result in end-stage renal failure.

Enrollment: 31 patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Diagnosed with primary IgAN
• Currently being treated for IgAN with stable, optimal therapy, including an ACE inhibitor or ARB.
• Has urine protein greater than or equal to 1 gram/24-hour
• Has hematuria (blood cells present in urine)

Exclusion criteria

• Has renal disease other than IgAN
• Has a diagnosis of rapidly progressive glomerulonephritis
• Has a diagnosis of Henoch-Schonlein Purpura (IgA Vasculitis)
• Has poor kidney function with estimated glomerular filtration rate (eGFR) <30 milliliters per minute per 1.73 meters square (mL/min/1.73 m^2)
• Has known human immunodeficiency virus (HIV) infection, hepatitis C virus (HCV) infection or hepatitis B virus (HBV) infection
• Has on-going high blood pressure
• Treated with systemic corticosteroids for more than 7 days, or other immunosuppressant agents in the past 6 months
• Received an organ transplant

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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31 participants in 4 patient groups, including a placebo group

Double-Blind Treatment (DBT) Period: Cemdisiran

Experimental group

Description:

Participants received cemdisiran, 600 milligrams (mg), subcutaneous (SC) injection, once every 4 weeks (Q4W) in combination with standard of care treatment up to a maximum of 38 weeks in the DBT period.

Treatment:

Drug: Cemdisiran

DBT Period: Placebo

Placebo Comparator group

Description:

Participants received cemdisiran matching placebo, SC injection, Q4W in combination with standard of care treatment up to a maximum of 38 weeks in the DBT period.

Treatment:

Drug: Placebo

DBT Period: Cemdisiran to Open-Label Extension (OLE) Period: Cemdisiran

Experimental group

Description:

Participants who were randomized to receive cemdisiran in the DBT period continued receiving cemdisiran, 600 mg, SC injection, Q4W in combination with standard of care treatment up to a maximum of 88 weeks in the OLE treatment period.

Treatment:

Drug: Cemdisiran

DBT Period: Placebo to OLE Period: Cemdisiran

Placebo Comparator group

Description:

Participants who were randomized to receive cemdisiran matching placebo in the DBT period started receiving cemdisiran, 600 mg, SC injection, Q4W in combination with standard of care treatment up to a maximum of 88 weeks in the OLE treatment period.

Treatment:

Drug: Cemdisiran