Amgen readies for Phase 3 study of Tavneos in children with AAV
Trial will test oral therapy in 20 pediatric patients with GPA, MPA
Amgen is planning to launch a Phase 3 clinical trial testing Tavneos (avacopan) — its approved therapy for adults with ANCA-associated vasculitis (AAV) — in combination with a standard regimen of rituximab or cyclophosphamide in children and adolescents with active AAV.
That’s according to a company press release announcing Amgen‘s early-year financial results, which noted that the oral therapy generated $51 million in sales in the first quarter of 2024.
The new trial in pediatric patients (NCT06321601) is expected to enroll up to 20 boys and girls with a diagnosis of granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA), the two most common types of AAV. Eligible patients will be those ages 6 to 17 who weigh more than 15 kilograms (about 33 pounds).
While no information has been provided as yet on where the clinical trial will take place, Amgen anticipates it to launch at the end of July, with its completion expected in April 2030, according to the company’s clinical trials website.
Tavneos is approved in the U.S. and Canada, and in the European Union and the U.K., as well as in other countries, as an add-on to standard induction treatment in adults with severe and active GPA or MPA.
Investigating the safety, effectiveness of Tavneos in children 6-17
The treatment works by blocking the receptor for C5a, a complement protein. In AAV, the complement system — a group of proteins that normally operate as a network to help clear harmful microbes from the body — goes haywire and contributes to the small blood vessel damage that marks the condition.
Tavneos’ approvals were based on the results of ADVOCATE (NCT02994927), a Phase 3 trial that tested the therapy against high-dose glucocorticoids. It involved 330 patients who were receiving immunosuppressive treatment with either cyclophosphamide followed by azathioprine or rituximab.
The proportion of patients who reached remission after six months was similar in the two groups, but higher after one year in those treated with Tavneos. The Amgen therapy also helped improve kidney function and reduce reliance on glucocorticoids, which may have serious side effects when taken at high doses or for a long duration.
As of now, Tavneos’s safety and efficacy in pediatric GPA and MPA patients remain unclear. Thus, Amgen is launching this trial to test the medication in children.
There will be no control group. Instead, all participants will receive Tavneos, taken by mouth twice daily as an oral or a liquid medication. The medication will be given for up to about a year, in combination with a rituximab- or cyclophosphamide-containing standard treatment regimen.
The main goal will be to assess the proportion of patients reaching and staying in disease remission, where no symptoms of AAV are experienced, after 26 weeks, or about six months, and at 52 weeks, or about one year.
Disease remission will be evaluated through changes in the Pediatric Vasculitis Activity Score, which measures disease activity on a scale from 0 to 63 points, with higher scores indicating more severe disease.
Secondary goals will include changes in other efficacy measures, including disease activity and kidney function, glucocorticoid dosage, and its pharmacokinetics, or the therapy’s movement into, through, and out of the body. Safety also will be assessed as a secondary goal.
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