New Novel Medication for Genetic Kidney Disease Explored

A multi-site clinical trial is exploring a new medicine to treat Genetic Kidney Disease known as inherited gene-damaged chronic kidney disease (CKD) at the University of Kansas School of Medicine.

Healthy kidneys filter blood waste and fluid. Urine removes these wastes. CKD disrupts this mechanism, causing proteinuria and waste buildup. CKD can cause high blood pressure, cardiac problems, nerve damage, and more. Patients need dialysis or a transplant after renal failure.

According to the National Kidney Foundation, about 1 in 7 Americans have genetic kidney disease, CKD. CKD is categorized. An hereditary mutation in the APOL1 (apolipoprotein L1) gene produces a rogue protein that damages the kidneys, causing a particularly severe type. This clinical trial is testing Vertex Pharmaceuticals’ novel medication ingredient inaxaplin to determine if it can suppress this damaging protein and reduce or stop disease development.

“Right now, there are no treatments specifically for APOL1-mediated kidney disease,” said Kelly Liang, M.D., associate professor of nephrology and hypertension and principal investigator for the clinical trial site at KU School of Medicine. “Currently, treatment may consist of general blood pressure medications that are used for other proteinuric kidney diseases. However, the agent being investigated is specifically targeted toward this APOL1 mutation, the underlying cause, to see if it may be more effective.”

Moving science
African Americans, Blacks, Caribbeans, Sub-Saharan Africans, and Latinos (Cuban, Mexican, Puerto Rican, or South or Central Americans) are more likely to have a mutant APOL1 gene.

“With this trial, the science moves beyond just saying, ‘among people from African descent, there is increased risk,’” noted Reem Mustafa, M.D., Ph.D., MPH, professor of nephrology and hypertension at KU School of Medicine and director of the Evidence-based Practice and Impact Center (EPIC). “We’re trying to understand why this mutation specifically increases the risk, and (we want) to identify and evaluate targeted therapies for this specific form of CKD, and that’s what we’re investigating.”

The study

AMPLITUDE is a clinical trial testing inaxaplin at different doses in 18–60-year-olds with APOL1-mediated CKD.

Participants are genetically screened for the mutation. The AMPLITUDE research randomly assigns participants with the mutation to one of three groups: 15 mg, 45 mg, or no medication.

How much protein is in the urine and how well the kidneys filter waste from the blood will determine the drug’s efficacy. The study teams plan to try the most beneficial dosage in more subjects after determining it. The four-year experiment is expected.

“Most of these patients have progressive disease, so we really need a better treatment option,” said Liang.

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