“Our mission is to improve the health and well being of those affected by Alpha-1 through support to patients, educating healthcare professionals and advancing public policy for the Alpha-1 Community.”

Identification of Individuals with Alpha-1 Antitrypsin Deficiency by a Targeted Screening Program

An article in Respiratory Medicine (Volume 101, Issue 8, August 2007, Pages 1708-1714) details a screening program conducted in Germany because “Alpha-1-antitrypsin deficiency (AATD) is significantly underdiagnosed. The early detection of AATD would enable affected persons to make lifestyle changes such as quitting smoking. It was the aim of the study to determine whether the combination of an awareness program with the offer of a cost-free diagnostic test results in the identification of a significant number of individuals with severe AATD.”
The screening combined a series of measures to promote awareness with the offer of a diagnostic test at no charge. Test blood was applied to a filter paper and sent to a laboratory. The level of AAT was measured by nephelometry, the presence of the S- or Z-allele was determined by PCR, and phenotyping was performed by isoelectric focusing.
During 37 months, 17,688 testing kits were distributed and 2722 were sent back to a laboratory and 335 patients with severe AATD including 16 individuals with rare genotypes were identified. Prescreening by determining the AAT serum levels by the submitting physician increased the detection rate as compared to similar programs that screened unselected individuals.
These data show that the combination of an awareness program with the offer of free diagnostic testing results in the identification of a large number individuals with severe AATD.

New Inhalation Product

Kamada, a research and development company from Israel is currently enrolling patients in clinical trials for their new inhalation product for Alpha-1 lung disease. The trials for an inhaled A1PI product are in Phase 1, while trials for an intravenous product are at Phase II–III level. The intravenous trials are being conducted at the University of Florida Gainesville, Denver’s National Jewish Medical Center and the University of Texas, Tyler. The research will be looking for non-inferiority to Prolastin, efficacy of the Kamada product, safety of the product compared to Prolastin, and at the levels of A1PI in the lungs before beginning the study and at the end of the study. For more information you can email Kamada at Clinicaltrials@kamada.com.

Sibling Study
Alpha-1 International Registry (AIR), Utah

The Alpha-1 International Registry (AIR), Utah, under the direction of Dr. Ed. Campbell is currently undergoing a sibling study. This involves blood draws and information gathering and of course, a signed informed consent. You can contact Dr. Campbell by visiting the web site at
http://www.aatregistry.org/usa.html

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Genetic Modifiers of Alpha 1-Antitrypsin Deficiency

We are looking for men and women that are 30 years old or older that have been told they have Alpha 1-Antitrypsin deficiency. We would like them to participate in a research study. The study is trying to find out the genetic reasons why some people who have been told they have Alpha 1-Antitrypsin deficiency develop problems with breathing and others don't. Participation in the study includes a single visit to the subject's home at their convenience that lasts about 1.5 hours. During this visit the participant would perform some breathing tests, complete a series of questionnaires, and give a blood sample. Participants are sent $50 for completing the tests.

Patient Inclusion/Exclusion Criteria:
To be eligible subjects must be
- over the age of 30
- Diagnosed with Alpha 1-Antitrypsin deficiency
- have one or more siblings that are also diagnosed with Alpha 1-Antitrypsin deficiency or are of unknown status.

Contact: James Keary Project Manager
Brigham and Womens Hospital
75 Francis Street Boston, MA 02115
Telephone: 617-525-2290
Email: rejmk@channing.harvard.edu

Go to the following website to fill out the form or call the above number.
http://www.centerwatch.com/patient/studies/stu47583.html

For additional studies, not specifically about Alpha 1, but dealing with pulmonary issues, go to: http://www.centerwatch.com/patient/studies/area18.html

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University of Florida
Scientists combine experimental therapies
to fight gene disorders.
University of Florida scientists have combined stem cell therapy and gene therapy in a new strategy to correct inherited diseases, according to a study published this week in the online edition of the journal Hepatology. Researchers removed a group of mouse liver cells that has the ability of stem cells to rebuild damaged organs and equipped them with a healthy human gene. The researchers then put the cells back into the mice, where they successfully began to rebuild the liver with cells that expressed the healthy gene. Complete information:
http://opi.health.ufl.edu/stories/2004/Sept/092304_Pastor.shtml

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Liver Research:

Ex vivo transduced liver progenitor cells
as a platform for gene therapy in mice.
Department of Pharmaceutics, University of Florida

Allogeneic stem cell-based transplants may be limited by allograft rejection, as is seen with conventional organ transplantation. One way to avert such a response is to use autologous stem cells, but that may carry the risk of recurrence of the original disease, particularly in the context of a genetic defect. More information: http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=pubmed&dopt=Abstract&list_uids=15382177

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Gene Therapy:

Gene therapy for human alpha1-antitrypsin deficiency
in an animal model using SV40-derived vectors.

Transplant Research Institute, University of California, Davis Medical Center, Sacramento 95817, USA.

In most genetic diseases, the goal of gene therapy is to deliver a particular transgene; however, sometimes a deleterious gene product must be eliminated. Because of the promise of recombinant simian virus 40 (rSV40) vectors, we tested their ability to deliver a transgene and to target a transcript for destruction by direct administration of the vectors to the liver of an animal model for human alpha1-antitrypsin (alpha1-AT) deficiency. For more information:
http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=pubmed&dopt=Abstract&list_uids=15480999

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Aralast Phase I in New Zealand and Austalia

There is a new clinical trial, Phase I on Aralast available in New Zealand and Austalia. Go to this web site for further details: http://www.clinicaltrials.gov/show/NCT00242385

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New clinical trial of efficacy of Zemaira..
Zemaira in Subjects With Emphysema Due to API Deficiency

This study is currently recruiting patients.
Verified by ZLB Behring March 2006
Sponsored by: ZLB Behring
Information provided by: ZLB Behring
ClinicalTrials.gov Identifier: NCT00261833

Purpose
Randomized, placebo-controlled, double-blind, multicenter phase III/IV study to compare the efficacy and safety of Zemaira® with placebo in subjects with emphysema due to alpha1-proteinase inhibitor deficiency. The effect of Zemaira® on the progression of emphysema will be assessed by the decline of lung density, measured by computed tomography (CT).

Condition Intervention Phase : Alpha1-Proteinase Inhibitor Deficiency Drug: Alpha1-proteinase inhibitor Phase IV

MedlinePlus consumer health information
Study Type: Interventional Study Design: Treatment, Randomized, Double-Blind, Placebo Control, Parallel Assignment, Safety/Efficacy Study

Official Title: A Randomized, Placebo-Controlled, Double-Blind, Multicenter Phase III/IV Study to Compare the Efficacy and Safety of 60 Mg/Kg Body Weight of Zemaira® Weekly i.v. Administration With Placebo Weekly i.v. Administration in Chronic Augmentation and Maintenance Therapy in Subjects With Emphysema Due to Alpha1-Proteinase Inhibitor Deficiency Further study details as provided by ZLB Behring:

Primary Outcomes: Lung density as measured by CT
Secondary Outcomes: Number, severity and duration of exacerbations; Lung function as measured by FEV1 and DLco

Expected Total Enrollment: 100

Study start: January 2006
Eligibility
Ages Eligible for Study: 18 Years - 60 Years, Genders Eligible for Study: Both Criteria

Inclusion Criteria:
* Alpha1-proteinase inhibitor deficiency
Exclusion Criteria:
* Current tobacco smoker (smoking has to be ceased at least 6 months prior to study inclusion).

Location and Contact Information
Please refer to this study by ClinicalTrials.gov identifier NCT00261833

Otto-Erich Girgsdies, Dr. +49 6421 39 Ext. 4019
Germany

For more information on sites participating in this study, please
contact our clinical research team in, Marburg, 35002, Germany; Recruiting Otto-Erich Girgsdies, Dr. +49 642139 Ext. 3019
Otto-Erich.Girgsdies@zlbbehring.com

More Information
Study ID Numbers: CE1226_4001
Last Updated: March 13, 2006
Record first received: December 2, 2005
ClinicalTrials.gov Identifier: NCT00261833
Health Authority: United States: Food and Drug Administration
ClinicalTrials.gov processed this record on 2006-05-08 http://www.clinicaltrials.gov/ct/show/NCT00261833

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New clinical trial why some people who have A1AD
develop problems, while other do not.

This study needs men and women who have been diagnosed with Alpha-1 Antitrypsin deficiency.

The National Jewish Medical and Research Center, in partnership with Brigham and Women's Hospital, is conducting a research study to find out the genetic reasons why some people who have Alpha-1 Antitrypsin deficiency develop breathing problems, while others with the diagnosis do not.

To be eligible, you must be 30 years of age or older, have a diagnosis of Alpha 1-Antitrypsin deficiency, and have one or more siblings also diagnosed with Alpha 1-Antitrypsin deficiency or are of unknown status.

The research site is in Denver, Colorado.

More Information
Please see http://www.centerwatch.com/patient/studies/cat258.htm

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Disclaimer: The Alpha 1 Advocacy Alliance supplies the reader with the above information and does not endorse or receive any goods or services for reporting this information.

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