I ran across this information today and wanted to share in case you are interested: New PBC Research Trial / UK and US

NEW PBC RESEARCH

Lumena is an early stage biotech company focused on better treatment options for adults and children living with rare cholestatic liver diseases. Lumena currently has two compounds aimed at not only carrying a reduced risk of systemic toxicities by selectively targeting a transporter in the intestine but is also designed to be minimally absorbed in the body. Phase II studies in children with Alagille syndrome and adults with primary biliary cirrhosis (PBC) have been initiated. In addition, studies in children with progressive familial intrahepatic cholestasis (PFIC) and adults with primary sclerosing cholangitis (PSC) are being planned.

“CLARITY”

PBC Study Newsletter Article

New Hope for Patients with Primary Biliary Cirrhosis

We have exciting news to share for patients with Primary Biliary Cirrhosis (PBC) regarding an investigational study that may help alleviate the symptoms of itching related to PBC. Lumena Pharmaceuticals initiated a new study and is currently enrolling patients with PBC and moderate to severe pruritus (itching). The study is a Phase II international, multi-center trial of LUM001.

LUM001, an investigational oral therapeutic, has been studied in 12 clinical trials in more than 1,400 people. Previous studies have indicated that LUM001 can reduce serum bile acid levels, which may be offer therapeutic benefit to patients with PBC and manage the pruritus that affects patient quality of life. In previous trials, LUM001 was generally tolerated, with the most common side effect being gastrointestinal disturbance.

The trial, known as CLARITY, is a randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of LUM001. The study will enroll approximately 60 patients. The primary endpoint of the study is change from baseline in pruritus compared to placebo. Secondary endpoints include changes in liver enzymes and other biochemical markers of cholestatic liver disease compared to placebo.

As patients know too well, cholestatic liver diseases are characterized by elevated bile acids and severe itching, which can be debilitating for some patients. To date, treatments with anti-pruritics typically provide only modest relief and procedures that remove bile from the circulation can lower serum bile acids and relieve symptoms in some patients, but tend to be invasive in nature. By reducing serum bile acids with an oral, once-daily tablet, the investigational drug LUM001 may offer a novel approach to the treatment of PBC and alleviation of pruritus.

Lumena Pharmaceuticals

lumenapharma.com

NEEDED PATIENTS TO PARTICIPATE in Lumena Pharmaceuticals….Please participate if you can. This is for ALL of US!

Below is a list of the centers where patients can participate in this study.

Sites in the United States

California

Scripps Clinic, La Jolla , California , United States , 92037

Contact: Marjory Simpson 858-652-5420 simpson.marjory@scrippshealth.org

University of California at Davis, Sacramento , California , United States , 95817

Contact: Sandeep Dhaliwal 916-734-8985 sandeep.dhaliwal@ucdmc.ucdavis.edu

Florida

University of Miami, Miami , Florida , United States , 33136

Contact: Marlene McGregory 305-243-2148 MmcGregory@med.miami.edu

Illinois

University of Chicago Medical Center, Chicago , Illinois , United States , 60637

Contact: Monique Williams 773-702-4477 mwillia3@bsd.uchicago.edu

Indiana

Indiana University, Indianapolis , Indiana , United States , 46202

Contact: Rachel Limbach 317-278-9355 rlimbach@iu.edu

Kentucky

University of Louisville, Louisville , Kentucky , United States , 40202

Contact: Lisa Hatter 502-852-3383 lisa.hatter@louisville.edu

Michigan

Henry Ford Health System, Detroit , Michigan , United States , 48202

Contact: Kimberly Baker 313-916-1979 kbaker@hfhs.org

Minnesota

Minnesota Gastroenterology, Plymouth , Minnesota , United States , 55446

Contact: Leon Heller 612-870-5597 Leon.Heller@mngastro.com

Missouri

St. Louis University, St. Louis , Missouri , United States , 63104

Contact: Dorothy Heller 314-977-9389 dorothyheller@slu.edu

New York

Weill Cornell Medical College, New York, New York , United States , 10021

Contact: James Maier 646-962-5394 jam2078@med.cornell.edu

Texas

University of Texas Southwestern Medical Center, Dallas , Texas , United States , 75390

Contact: Rebecca Chason 214-415-0983 Rebecca.Chason@utsouthwestern.edu

Advanced Liver Therapies at St. Lukes Episcopal Hospital, Houston , Texas , United States , 77030

Contact: Beth Dietrich 832-355-8966 bdietrich@stlukeshealth.org

Utah

University of Utah Health Science Center, Salt Lake City , Utah , United States , 84132

Contact: Tiffany Tomkinson 801-585-9155 tiffany.tomkinson@hsc.utah.edu

Virginia

Liver Institute of Virginia, Newport News , Virginia , United States , 23602

Contact: Suzette Tipps 757-947-3190 suzette_tipps@bshsi.org

Hunter Holmes McGuire VA Medical Center, Richmond , Virginia , United States , 23224

Contact: Jennifer Salvatori 804-675-6924 jennifer.salvatori@va.gov

Washington

University of Washington Harborview Medical Center, Seattle , Washington , United States , 98104

Contact: Carly Gintz 206-744-3479 cgintz@medicine.washington.edu

Sites in the United Kingdom

University of Birmingham, Birmingham , England , United Kingdom , B15 2TT

Contact: Darren Barton 44 (0) 121 414 8255 d.barton@bham.ac.uk

Newcastle University, Newcastle , England , United Kingdom , NE1 4LP

Contact: Jennifer Bainbridge 0191 282 0070 j.m.bainbridge@newcastle.ac.uk

Oxford University Hospitals, Oxford , England , United Kingdom

Contact: Madeleine Thyssen 01865 231618 Endoscopy.researchsisters@orh.nhs.uk

Imperial College London St Mary's Hospital, London , United Kingdom , W2 1NY

Secondary link: lumenapharma.com/lumena-pha...

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  • Hiya JennerLayne.

    I read your posting and checked out with regards to this in the UK.

    Now it appears currently that here in the UK you are only eligible currently if you have something called Alagille Syndrome. Here is the UK site with the relevant information.

    clinicaltrials.gov/show/NCT...

  • Hi Peridot --- I hope you are doing well. I pulled this paragraph out and this study is in phase II international, enrolling PBC patients or at least that's what I understand it to say (my come-and-go 'brain fog' may be directing me differently!) From what I can piece together there was the first phase targeted to those with Alagille Syndrome, but the new phase II study is directed to PBC people who have moderate to severe itching. This was announced in October '13 per the website lumenapharma.com/lumena-pha... Here's the paragraph from what I posted yesterday.

    New Hope for Patients with Primary Biliary Cirrhosis

    We have exciting news to share for patients with Primary Biliary Cirrhosis (PBC) regarding an investigational study that may help alleviate the symptoms of itching related to PBC. Lumena Pharmaceuticals initiated a new study and is currently enrolling patients with PBC and moderate to severe pruritus (itching). The study is a Phase II international, multi-center trial of LUM001.

  • Hi JennerLayne.

    I think with this new trial drug being from the US it is more likely that recruitment will take place there for PBC and then extended to other countries. I've noticed this previously with other overseas companies.

    I will keep an eye out for if or when the study is extended to the UK for PBC as at present it appears to just be for this Alagille Syndrome that I have never heard of until now and then don't understand what it is.

    Will be interested in finding out more if you stumble across anything further but on our UK sites that is all I have found so far.

  • Peridot --- I'll keep my eyes open as I am always looking for information that will help support our community of PBCERs. Incidentally, I do plan to attend the US PBC Organization's conference in late July where many renowned hepatologists and doctors will be speaking. I asked about the ability to record sessions and possibly share online (no) but will certainly take notes and forward anything of importance to our group. Take care!

  • Thank you so much JennerLayne for all your efforts to improve the lot of those with PBC, like myself. I look forward to hearing about the conference in the US. I live in the UK

  • That would be wonderful! Thank you as we all will be really interested in anything you report! Have fun at the conference. I am sure it will be really educational.

  • I've heard recent chatter that conference organizers/attendees are asking if the speakers would be open to presentations being taped and/or whether their slides can be posted to the pbcers.org site. I am hopeful this can happen so folks around the world can tap into this vital information. Regardless, I will take detailed notes and report back to this site.

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