Menu

Top Stories

Published on Thursday, May 28, 2015

The Lancet publishes RAPID trial results showing effectiveness of 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 Ronald Crystal, MD, Department of Genetic Medicine, Weill Cornell Medical College.

Photo at top: Ken Chapman, MD. Below: John Walsh, left, and Robert Sandhaus, MD.


Print

CSL Behring to extend "compassionate use" access for Irish Alphas

Monday, August 14, 2017
Pharmaceutical manufacturer CSL Behring has decided to extend a compassionate use programme for its Respreeza drug in the State after the company came under fire over the timing of its closure. The Respreeza drug had been provided on...
Divider

FDA to expand public education to prevent youth e-cigarette use

Wednesday, August 9, 2017
The U.S. Food and Drug Administration announced it would pursue a strategic, new public health education campaign aimed at discouraging the use of e-cigarettes and other electronic nicotine delivery systems (ENDS) by kids. The agency plans to...
Divider

Bob Campbell passes away, Foundation Communications Director, former AlphaNet Coordinator

Friday, July 28, 2017
Our dear friend and colleague, Bob Campbell passed Thursday evening with his wife Elizabeth by his side. Campbell began his Alpha-1 journey when he was diagnosed by his allergist. In 2002, he took the leap and became an AlphaNet...
Divider

Kamada Submits Proposed Phase 3 Protocol to FDA for Inhaled Alpha-1 Antitrypsin Treatment

Friday, July 21, 2017
Kamada Ltd., a plasma-derived protein therapeutics company focused on orphan indications, today announced that the Company has submitted to the U.S. Food and Drug Administration (FDA) for review a proposed pivotal Phase 3 protocol for its...
Divider

Nicotine addict finds hope after Alpha-1 Antitrypsin Deficiency diagnosis

Wednesday, July 19, 2017
Diagnosed with the lungs of a 126-year-old man from years of smoking and a rare genetic lung disorder, Ryan Taylor moved home from Alberta this past Christmas to die. But a news report his father saw on Memorial’s School of...
Divider

5-year old Alpha becomes the face of Australia’s DonateLife Week

Monday, July 17, 2017
If you still aren't sure about whether or not to become an organ donor, look into the eyes of Archer Irwin. The five-year-old from Wodonga has become the face of DonateLife Week, the federal government's push to have more...
Divider

Alpha Emma Lou Carr Glenn passes away

Monday, July 17, 2017
Emma Louise (Lou) Glenn passed away Tuesday, July 11. Many in the Alpha-1 community will remember Lou as Patti Brown's mom.  Patti is an AlphaNet Coordinator in Texas.  Lou became involved in the Alpha-1 community after Patti's...
Divider

Foundation president and CEO urges Alphas to find their place in the Alpha-1 Community

Thursday, June 29, 2017
CHICAGO, Illinois – Henry Moehring, president and CEO of the Alpha–1 Foundation, presided over the 26th and largest National Education Conference of the Alpha–1 community since its inception, he said in his Foundation update...
Divider

Alpha-1 Foundation Accepting Proposals for the John W. Walsh Translational Research Award in Alpha-1 Antitrypsin Deficiency

Wednesday, June 28, 2017
The Alpha-1 Foundation is committed to finding a cure for Alpha-1 Antitrypsin Deficiency and to improving the lives of people affected by Alpha-1 worldwide. This award is named after John W. Walsh, who dedicated his life’s work to finding...
Divider

Barbara Pusey honored in memoriam at Alpha–1 awards and recognition night

Monday, June 26, 2017
Barbara Pusey, who was the first AlphaNet Coordinator for the augmentation therapy marketed in the United States as Zemaira and was a major influence in building the AlphaNet Zemaira program into what it is today, was recognized posthumously, with...
Divider

Grifols announces $1 million grant to support Alpha-1 Foundation’s John W. Walsh Research Fund

Monday, June 26, 2017
Washington, DC  — Grifols announced that it will award a million-dollar grant to support the John. W. Walsh Research Fund, the Foundation’s peer-reviewed research program named in honor of Walsh, the Foundation’s co-founder...
Divider

Alpha-1, the "genetic component" in COPD, featured in national news magazine

Thursday, June 8, 2017
The following article on Alpha-1 Antitrypsin Deficiency is from U.S. News Health. It contains extensive quotes from Robert Sandhaus, MD, PhD, clinical director of the Alpha-1 Foundation and medical director of AlphaNet. You might not realize...
Divider
12345678910Last

Article Search

Enter keywords in text box and click Search button for results.

Archive