Clinical Trials for ITP

IMPORTANT!
This summary of clinical trials for ITP is intended to be used as a starting point for discussion with a health care professional. Be sure you understand all the implications of the proposed treatment before enrolling in a clinical trial.

What are Clinical Trials?

Clinical Trials are controlled patient studies that must be performed before a new drug or treatment is approved by the FDA. They are also done after a drug or treatment is approved to document the effectiveness of the treatment or to gain FDA approval to use the drug or therapy to treat a different disease.

There are three main phases to clinical trials, done in sequence. A Phase I study tests the safety and side effects of the drug. A Phase II study is initiated to determine the efficacy of the drug, to see if it works as anticipated. A Phase III study tests the results on a large population of volunteers.

Pharmaceutical and other companies who are doing the trials seek volunteers to participate. If you are considering participating in any of these trials be sure to read the fine print.

Go to the centerwatch page for answers to the following questions.

• How are experimental drugs tested in humans?
• Who pays for clinical research?
• Should you participate in clinical research?
• Where can you get more information about clinical research?

“Should I Enter a Clinical Trial? A Patient Reference Guide for Adults with a Serious or Life-Threatening Illness” explains the objectives, risks, benefits, and implications of a clinical trial.   Read it.  Discuss with your doctor.  Make more informed decisions.

The guide is available FREE at - http://www.ecri.org/

Transportation Assistance

Clinical trials are not always conducted in locations convenient to the patients who may contribute and/or benefit the most. That’s a problem. One solution is Angel Flight of America. This organization provides air transportation for people to obtain a medical procedure or to participate in a clinical trial. For more information go to http://www.angelflightamerica.org/ or call 800-446-1231.

Clinical Trials List

Check www.clinicaltrials.gov for additional information.

LGD4665 – Ligand Pharmaceuticals has initiated a phase IIA clinical study of LGD4665, a potent, oral drug that is a highly specific for increasing platelet count.  It acts like the natural platelet regulator, thrombopoietin and induces platelet increases in humans within 3 to 5 days.  The study is being conducted in the US and is designed to demonstrate the activity of LGD4665 at safe doses in patients with immune thrombocytopenic purpura (ITP). 

This trial has two parts: Part 1 is a 6 week randomized, double-blind treatment and Part 2 is a 3-month open label (active LGD4665 only) study extension.  The double blind part of the study is placebo controlled for 6 weeks where every patient has a 2/3 chance of receiving LGD4665.  The extension part of the study examines the optimal doses of LGD4665 for individual patients and its durability in maintaining increased platelet levels. 

To enter the study, patients must have received one prior therapy for ITP and must have low platelet counts of <30,000/uL (or < 50,000/uL if patient is on a stable regimen of oral corticosteroid).    For more information contact Victor Stevens,  858-550-7739, vstevens@ligand.com or see the www.clinicaltrials.gov website.

PRYX-100 - Protalex Inc. has initiated a phase Ib therapeutic trial of study of PRTX-100, which is a highly-purified native bacterial protein called staphylococcal protein A.  This protein was the active-ingredient in the PROSORBA plasma-adsorption device, which was approved for treatment of ITP and rheumatoid arthritis in the 1990's.  Studies later determined that treatment with this device actually dosed patients with small amounts of staph protein A. 

This is an open-label (no placebo) sequential dose escalation trial, being conducted at hematology referral centers in Australia and New Zealand.   The study is designed to obtain data on the safety profile, the pharmacokinetics, the immunogenicity, and the effects on platelet counts and fatigue in patients with chronic ITP.   Treatment will be given on 4 weekly visits, with a follow up period of up of at least 1 month afterwards, up to 2 months in responding patients.   Three cohorts of 7 patients each will be studied in sequence, receiving 0.075, 0.15, and 0.3 µh/kg of PRTX-100.  Responding patients who desire eventual re-treatment will be offered treatment in an extension study protocol.

Patients must have failed to have obtained an adequate or sustained response to at least one standard of care therapy in order to be eligible.  Patients may be on certain concurrent ITP medications such as oral prednisone.  To be eligible, patients must have a platelet count of <50,000/µl, or if on daily oral prednisone, a platelet count of < 90,000. For more information contact Edward W. Bernton, MD, 215-862-9720, ebernton@protalex.com

Low Platelets and Clotting Problems - Researchers are looking for the genetic patters in people with this combination. You can help. Read the brochure or call Laurel of the Rare Thrombotic Disease Consortium (RTDC) at (919) 681-2668 to discuss.

AMG 531

AMG 531 stimulates the body to make more platelets. There is an on-going trial for adult patients refractory to many treatments and also a trial of AMG 531 for pediatric patients. See. www.clinicaltrials.gov or at www.amgentrials.com

GMA161

Genzyme Corporation in collaboration with MacroGenics, Inc. is currently conducting a Phase I research study for people over the age of 17 years who have been diagnosed with chronic idiopathic thrombocytopenic purpura (ITP). The study is designed to investigate the safety of a single infusion of GMA161, a monoclonal antibody, as well as the way the drug enters and leaves the body. In addition, throughout the study, platelet counts and other blood cell numbers will be measured.  

For more details about eligibility criteria and contact information for participating medical centers,  please visit:  http://www.clinicaltrials.gov/ct/show/NCT00244257

Combination of WinRho and Danazol - If you are 18 years of age or older and have been diagnosed with Immune thrombocytopenic Purpura (ITP), you may qualify for a clinical research study at St Luke’s-Roosevelt Hospital center and Beth Israel Medical Center (New York City). Hematologists are testing a combination of WinRho and Danazol that may help increase platelet counts and/or reduce bleeding symptoms. Please call 212-844-6286 to check if you qualify.

Various Platelet Disorders

This study is being conducted by the National Institutes of Health.   The purpose of the study is to determine how platelet sacs are formed and what happens to cause bleeding disorders.  They are looking for people diagnosed with Gray Platelet Syndrome (GPS), Hermansky-Pudlak Syndrome (HPS), Storage Pool Deficiency (SPD), Chediak-Higashi Syndrome (CHS), Griscelli Syndrome (GS), Wiscott-Aldrich Syndrome (WAS), Thrombocytopenia Absent Radius Syndrome (TAR)  Call 1-800-411-1222 for more information.  Click to see flyer.

See http://clinicalstudies.info.nih.gov/detail/A_2004-HG-0226.html

Rituximab in Children

The purpose is to formally evaluate the safety and effectiveness of Rituximab in children and adolescents with severe or refractory ITP. For very general information or reference for parents, see  http://www.itpkids.org.

More specific inquiries (from MDs) may be addressed to Pamela Boardman, Study Coordinator, Children's Hospital Boston 617-355-2901; pamela.boardman@tch.harvard.edu (Her info is also on the w.itpkids.org site)

Bone Marrow Transplantation

A phase I trial underway at the NIH has been extended.  Initial results show some positive response in 57% of the patients. NOTE: THIS IS A VERY SEVERE TREATMENT!  Also note that the NIH funds participation in this trial.

Contact Dr. Cynthia Dunbar at (301) 496-5093 or dunbarc@nhlbi.nih.gov

See Abstract for the latest results.