Alba Clinical Trials Via Celiac Central

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BEYOND RICE CAKES

A Celiac Pill: Alba Calls for Participants to Test Effectiveness of AT-1001

By Vanessa Maltin

NFCA Director of Outreach & Programming

Do you dream about being able to eat pizza, pasta, cookies, cake and all of your favorite gluten-containing products again? The first year after being diagnosed with celiac disease, I remember wandering up and down the aisles of the grocery store just wishing I could buy the same old products as before I had to be on a gluten-free diet. They were cheaper, tasted better and were easier to find. Even though I've been gluten-free for over four years and am a pro at managing my lifestyle, I still find myself having moments where I wish I weren't on the diet...especially those times when I am inadvertently exposed to gluten.

Over the last year, the National Foundation for Celiac Awareness has surveyed thousands of celiac disease patients about items that are important to them in order to determine what new developments would make their gluten-free lifestyle easier to manage. As I'm sure you can guess, the most common response was: a pill that would allow the body to safely digest gluten.

For most of us with celiac disease, a pill would represent a dream come true. At this time, there are no drugs in the pipeline to allow patients to eat a normal gluten-containing diet. However, a dynamic group of researchers at Baltimore-based Alba Therapeutics have developed a pill that is expected to protect celiac patients against a small amount of gluten. How much you ask? Only about 2.5 grams, which is equal to roughly one slice of bread. Essentially, it will protect against inadvertent cross-contamination.

Right now Alba Therapeutics is recruiting participants for a Phase II clinical trial of AT-1001. The study is examining three doses of the drug for efficacy and safety for treating celiac disease. The goal of the study is to show that AT-1001 can prevent intestinal damage when a patient undergoes a six-week gluten-challenge.

BUT...just because you're on the drug while participating in the study, doesn't mean you can eat all of the gluten you want for six weeks! The researchers will provide you with a kit that contains gluten capsules for you to swallow. This allows the researchers to control the exact amount of gluten intake and determine appropriate dosages of AT-1001.

Are you confused? The first time I heard all of this, I was, so lets take a step back and understand the basic science.

The Science of Celiac:

Before you can understand what goes wrong in celiac patients, you have to understand the normal digestion process. When a healthy person eats food, it travels to the small intestines where it is absorbed into cells and broken down. Once the food particles are processed in the small intestine cells, the cells release the nutrients into the blood stream where they are absorbed and used by the body to function. However, between the cells we have what are known as "tight junctions" These are actually dynamic gates that open and close. When a person with celiac disease eats gluten, the food travels into the small intestines, but accidentally slips past the cells through the gates. We commonly call this "leaky gut" syndrome because the gut is leaking out the gluten protein before it has a chance to be processed and digested. This is toxic for a person with celiac disease.

When the gluten particles leak through these gates, they directly enter the gut wall. In celiac patients, this causes an immune system response, which produces antibodies. (NOTE: These antibodies are what are measured when a patient has a celiac blood test, and are called anti-tissue transglutaminase or anti-tTG.)

How can AT-1001 Help?

Last week I spent the day with Alba's Senior Vice President for Clinical Development and Medical Affairs Dr. Betsy Van Parijs. She very eloquently explained the theory of how this investigational drug works and helped convince me that data so far have shown that the drug is well-tolerated in celiac patients.

AT-1001 is a peptide, which means that it consists of natural amino acids that are protein fragments). When you ingest it, the gut sees it as if it was a natural matter and breaks it down. This means that it gets broken down rather than absorbed into the gut wall. This is good news and explains why there have been no reported serious or severe side effects of the drug.

AT-1001 works like a topical ointment in the gut that coats the inside of a patient's small intestine lining. This prevents a small amount of gluten from passing through the "leaky gates" and prevents the gluten toxins from entering the gut wall.

Why Can't You Eat All the Gluten In the World?

Even while taking AT-1001, patients can only eat 2.5 grams milligrams of gluten per day. According to Dr. Van Parijs, celiac patients might never be able to eat a piece of pizza or a plate of glutinous pasta, largely because scientists can't change genetics. All they can do is try to control the effects.

Try thinking of it in terms of high cholesterol. Patients with high cholesterol are commonly prescribed a drug called Lipitor. They are told to take the drug in conjunction with a low-fat diet and exercise. Lipitor helps reduce the cholesterol, but can't control it without the added change in diet and exercise.

So, for celiac patients, the same theory applies. AT-1001 will help digest a SMALL amount of gluten—about the amount in accidental cross contamination, but in order to remain healthy, you must still attempt to adhere to a gluten-free diet.

The bottom line is that if AT-1001 works as Alba expects it to, the drug will help prevent long-term complications for sure and provide short-term relief if you accidentally consume gluten. However, the clinical trials to investigate this and other effects of AT-1001 are ongoing, and no conclusions about the therapeutic effects of the drug can be made in advance of the results of the trials

What Does the Clinical Trial Involve?

Alba is looking for biopsy-confirmed celiac disease patients to participate in a phase II clinical trial to test the effectiveness of AT-1001. To participate in the study, patients must be 18-72 years old, diagnosed with celiac disease for more than six months and have been on a strict gluten-free diet for at least six months. In addition, patients must have a negative anti-tissue transglutaminase (anti-tTG) test.

If you visit a study site, you will speak with a nurse who will walk you through the process of the trial. The nurse will explain that you will need to submit a urine sample and undergo other types of testing during the six-week study. You will be asked to sign a consent form.

Once you've given your consent to participate in the study, you will have a screening visit to be physically examined. All of your vital signs will be checked and then the blood test for anti-tTG will be done, and the biopsy report from a previous exam will be reviewed. If all of your test results are within the acceptable ranges, you will be enrolled in the study.

At this point you will be given a kit that contains either AT-1001 or the placebo. It is a double blind, randomized study, so you will not know which kit you have. You will also receive tablets that contain gluten. You will be instructed to take both the study drug and gluten pills three times per day, one pill approximately 15 minutes before you eat a meal and two pills along with your meal.

Once you begin taking the drugs, you will need to return to the clinic for further testing and procedures and fill out daily diary on an electronic device like a PDA monitoring how many pills you take each day and if you experience any form of discomfort.

The entire duration of treatment for the study is six weeks. After the six week period, you will return to the study clinic one week later for follow-up to determine if you had any unforeseen side effects.

Are there Dangers to Participating?

The AT-1001 study does involve a placebo group. This means that it is completely random whether you receive the active medication or a placebo pill. Both patient groups ingest gluten tablets, but according to Dr. Van Parijs, the risk of long-term complications is low, because the amount of gluten is so small and it is for a very short period of time compared to a lifetime of ingesting gluten.

She cites supportive medical literature suggesting that ingesting 2.5 grams of gluten per day "over a period of six weeks is not sufficient enough to cause long-term damage." She notes that it will produce mild or moderate side effects such as diarrhea, constipation or bloating related to gluten ingestion, but generally "not to an intolerable degree."

Dr. Van Parijs says the researchers at Alba think daily about the Hippocratic oath they took to become doctors and can say confidently that they are "not harming our patients " by asking them to eat gluten during the study. However, as in all clinical trials, there may be unforeseen side effects. Please seek additional information about potential risks before participating.

How to Participate in the Trial:

To participate in the trial, you must be:

Age between 18 and 72 years

Diagnosed with celiac disease for more than 6 months

Negative anti-tissue Transglutaminase

On a gluten-free diet for at least six months

BMI between 18.5 and 38

Exclusion criteria—You may NOT participate if you are any of the following:

Current smoker

Has chronic active GI disease other than celiac disease (ex. Crohn's, Colitis)

Has Diabetes

Unable to abstain from alcohol consumption for 48 hours prior to each intestinal permeability collection

Unable to refrain from consuming non-steroidal anti-inflammatory agents for 48 hours prior to intestinal permeability collection

Participated in any clinical drug study within the past 30 days or has had previous exposure to AT-1001

Presents with or has a history of dermatitis herpetiformis.

If you are interested in participating, please contact: clintrials@albatherapeutics.com or call Alba Therapeutics directly at 1-877-415-3282. There are currently 23 study locations nationwide.

One last note...

The work that Alba is doing is absolutely fascinating, so take a few minutes to watch our interview with Dr. Van Parijs and listen to all she has to stay about the drug and the study. Then take a look at their website for even more information! www.albatherapeutics.com.