Gene Therapy Clinical Trial for Alpha-1 Patients

[gerald]

A treatment for A1AT deficiency is going into clinical trials!!!!


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Gene Therapy Clinical Trial for Alpha-1 Patients
Safety Dose Finding Study of ADVM-043 Gene Therapy to Treat Alpha-1 Antitrypsin (A1AT) Deficiency (ADVANCE)

Detailed Description:

Alpha-1 Antitrypsin (A1AT) is a major inhibitor of serine proteases and plays an important role in the lung as an inhibitor of neutrophil elastase. A1AT deficiency is associated with decreases in plasma A1AT levels and is associated with a increased risk for developing asthma, emphysema/COPD, and bronchiectasis. Much of the lung damage is thought to be caused by proteolytic damage from neutrophil elastase and other proteases.

ADVM-043 is an investigational gene therapy product (serotype AAVrh.10 vector) expressing human A1AT that is intended to deliver a functional gene to the liver of patients with A1AT deficiency. Study ADVM-043-01 will study up to 4 dose levels in up to 20 patients and assess the hypothesis that a single administration of an AAV vector expressing the human M-type A1AT (i.e., ADVM-043) to patients with A1AT deficiency is safe and results in persistent therapeutic levels of A1AT in blood and alveolar epithelial lining fluid. The primary endpoint is safety, and changes in plasma A1AT levels at multiple time points up to 52 weeks after dosing. A prophylactic tapering corticosteroid regimen will be used to protect against potential vector induced transaminitis. Subjects will be followed for up to 52 weeks after dosing. After completion of this study, subjects will be asked to enroll in a Long Term Follow Up study.

Actual Study Start Date : November 28, 2017
Estimated Primary Completion Date : December 2018
Estimated Study Completion Date : August 2019

clinicaltrials.gov/ct2/show/NCT02168686?term=Gene+therapy&recrs=ab&cond=COPD&age=12&rank=1

[Wendall]

I wish I had seen this sooner. I'm very sick in oxygen 24/7 now. Had no clue till 2 years ago I had Apha 1 deficiency. My lung collapsed when I was 42. They said it was spontaneous. No lung disease or COPD. Doctor error it ended being collapsed three days. Since then I've no longer worker and have plummeted quickly. Have six children all over 18 this year but grandchildren coming. Would love to last longer then the rate I'm going now. Any help out there. I'm in Edmonton Alberta. Thank you for readinge.

[jim]

Hopefully, one of our Canadian members can give you some info on what is available near you in Alberta.

[gerald]

Wendall, my knowledge of Edmonton is a bit dated.  However, if you need good doctors, I used to use the  Links clinic on 11th Ave (around 119th).  My doctor was Dr Doer (however I suspect he will have retired).   They have an expert pulmonary specialist in the  same building.  There maybe a doctor there taking new patients.

University of Alberta, at Edmonton used to be one of the foremost research universities, it might be worth seeing if they have a reasonable to see if they have a Research program into respiratory issues.

As Alberta allows private medical as well as government funded there are some good private clinics as well.

Also there is lots of info in these various topics to help answer many questions you  may have.