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Trial Results Alpha-1 Augmentation Therapy

The Lancet publishes RAPID trial results showing effectiveness of Alpha-1 augmentation therapy.

The Alpha-1 Foundation on Thursday congratulated the investigators and sponsor of the RAPID Trial, demonstrating the effectiveness of augmentation therapy in slowing emphysema due to Alpha-1 Antitrypsin Deficiency, on the publication of the trial in The Lancet, one of the world’s oldest and most prestigious medical journals.

The Lancet article was posted online this week.

When the results were first announced at the international conference of the American Thoracic Society (ATS) in 2013, lead author Kenneth Chapman, MD, director of the Asthma and Airway Centre of the University Health Network in Toronto, Canada, called the trial “the most rigorous evidence to date that augmentation therapy slows the progression of emphysema in patients with Alpha-1 Antitrypsin Deficiency. The effect of A1-PI seen in this trial was both clinically and statistically significant, finally confirming its benefit in preventing the loss of lung tissue in patients with this potentially debilitating disease.”

The Alpha-1 Foundation hailed the trial results and The Lancet publication. "We congratulate CSL on this landmark clinical trial, which provides the strongest evidence yet that augmentation therapy preserves the lung tissue of individuals with Alpha-1-related lung disease," said John Walsh, Foundation president and CEO. "We hope these results will support Alpha-1 communities around the world in their efforts to win access to therapy.”

Walsh said, “The Foundation has communicated its support for acceptance of the RAPID data as proof of efficacy of augmentation therapy to the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (the European equivalent of the FDA), and has continued a dialogue with both agencies to urge acceptance of these findings.”

CSL Behring sponsored the trial, which randomly assigned 180 Alpha-1 patients to receive either the augmentation product Zemaira or a placebo for a two-year period, followed by a two year of open-label extension study in which all subjects were offered augmentation therapy. “We applaud CSL for its commitment of time and effort to conduct this multinational, multi-year study, and we’re delighted that The Lancet has published the RAPID Trial results,” said Walsh. 

The Lancet article says that an interim analysis of data from the two-year extension trial suggest that early treatment with augmentation therapy shows persistent efficacy in patients with Alpha-1 and emphysema. In addition, when patients who had been receiving a placebo in the original two-year trial switched to treatment with augmentation therapy, their lung density decline (which was more rapid than the treated group in the main study) slowed to the same rate as the treatment group. “These findings should encourage early introduction of augmentation therapy [in patients with emphysema due to Alpha-1] and should stimulate further research into optimum dosing,” says the Lancet article.

Robert Sandhaus, MD, PhD, clinical director of the Alpha-1 Foundation and medical director of AlphaNet, was a co-author of the Lancet article.

The RAPID Trial is the first well-powered randomized, placebo-controlled trial to use CT scan lung density as the primary outcome measure. CT scans are currently considered the most sensitive measure of emphysema detection. The trial was conducted at 28 sites in the United States, Europe, Canada, Australia and Russia.

The multi-center, multi-national trial randomized patients with homozygous Alpha-1 (ZZ) to receive either alpha-1 antitrypsin augmentation therapy intravenously at 60 mg/kg weekly or a placebo over two years. CT scan lung density was measured at baseline, three months, one and two years. Secondary endpoints included spirometry, changes in exercise capacity and the rate of pulmonary exacerbations over two years.

The annual rate of lung density loss was significantly less in augmentation-treated patients (-1.45 +/- 0.24 units vs. -2.19 +/-0.25 units; p = 0.017, one-sided). Secondary outcome variables and adverse events were not significantly different between groups, according to the article. There was one death in the augmentation therapy group and three in the placebo group.

Attached is the full article published in The Lancet, with an accompanying editorial by Ron Crystal, MD, Department of Genetic Medicine, Weill Cornell Medical College.

Source: Alpha-1 Foundation 28/05/2015

 

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