Did you know?

The IWMF raised more than five million dollars for WM medical research

Did you know?

The IWMF raised more than 60 support groups in North America and nearly 20 others world-wide

Did you know?

The IWMF has a current worldwide membership of nearly 4,000

Did you know?

IWMF-TALK is a wonderful place to share information and learn from other survivors

Did you know?

Each year 1,500 new cases of Waldenstrom's macroglobulinemia are diagnosed in the USA

Did you know?

Volunteers at the IWMF Telephone Lifeline are waiting to share their experiences with you

Did you know?

You can order DVD recordings of past WM Educational Forums online or by mail

Did you know?

All IWMF programs and services are funded by WM survivors, caregivers, friends, and family

Clinical Trials




The IWMF supports the promotion of clinical trials because it recognizes that major advances in treatment or for a cure for Waldenstrom’s macroglobulinemia (WM) cannot occur without the participation of WM patients in clinical trials.


So-called “orphan” (rare) diseases like WM are at a disadvantage when it comes to competing for research dollars simply because of the low number of patients with WM and the consequent lack of interest by the pharmaceutical industry. For example, in the United States, with a population of over 300 million, there are approximately 1,500 newly diagnosed WM patients every year; in contrast, approximately 195,000 patients are newly diagnosed with invasive breast cancer every year.


It is a rather startling fact that over 80% of pediatric patients participate in clinical trials, whereas only 5% of adults do. Ninety percent of pediatric blood cancers are now curable. We seek the same results in adult blood cancers!


The folks at cancercare.org developed a helpful document, called "Clinical Trials: Improving the Care of People Living with Cancer," which presents the basics of clinical trials and is designed to help a patient feel more at ease with the decision to enter a clinical trial.  The document is no longer in print, but they have allowed us to provide it on our website (click the link above).


The sections presented below will also introduce you to basic information about clinical trials and provide links to additional information on clinical trials, as well as a listing of specific clinical trials available for WM patients.



What are clinical trials?

Clinical trials are research studies in human volunteers that answer specific health questions, with the goal of developing better ways to prevent, screen for, diagnose, or treat a disease. Cancer clinical trials may include new drugs, new surgical or radiation therapy techniques, new combinations of treatments, and revolutionary methods such as gene therapy.


Why are clinical trials necessary?

Most of today’s most effective cancer treatments are the direct result of clinical trials. Important scientific questions may be answered by clinical trials about the safety and effectiveness of new therapies, for example questions about optimal dosage, frequency of dosage, side effects, duration of remission, and comparison with other therapies already in use. Results from clinical trials may also suggest future research directions.


Why should I enter a clinical trial?

People who take part in cancer clinical trials have an opportunity to contribute to knowledge of, and progress against, cancer. They receive up-to-date care from cancer experts and undergo either a new promising treatment or the best available standard treatment for their cancer. Many patients now choose to receive their first treatment in a clinical trial because of the increasing evidence that clinical trials (particularly in rare diseases such as WM) provide excellent care and better overall results. Whether you should take part in a clinical trial is a personal decision. Consult those who are close to you and discuss it with your health care team. Educate yourself about clinical trials by visiting the websites listed in this section.


How are clinical trials carried out?

Research with humans is conducted according to strict scientific and ethical principles. Patients who join clinical trials are given either some of the best treatment currently available or the chance to receive a new treatment under consideration. Placebos (so-called sugar pills) are rarely used in cancer clinical trials and are never used in place of treatment. Every clinical trial has a “protocol,” a detailed action plan that acts as a recipe for conducting the trial. The same protocol is used by every doctor or research center taking part in the trial. The “outcome” or “endpoint” of the study is its specific goal or purpose.


Each trial enrolls a certain number of eligible volunteer patients who may be alike in key ways – type of cancer, age, gender, history of prior treatments, current health status, etc. – these are called “inclusion criteria.” Even though patients may meet inclusion criteria, they may not be eligible for a particular trial because they have "exclusion criteria," namely other medical conditions or prior treatments that exclude them from participation in the trial. Clinical trials take place in doctors’ offices, cancer centers, other medical centers, community hospitals and clinics, and veterans' and military hospitals in cities and towns across the U.S. and in other countries. Participants may be at one or two highly specialized centers, or they may be spread out in several locations at the same time.


What is informed consent?

Informed consent is a process by which patients learn the important facts about a clinical trial to help them decide whether to participate. This information includes details about the purpose of the study, the tests and other procedures used in the study, and the possible risks and benefits. Patients receive a written consent form explaining the study. The informed consent process continues for the duration of the study. If new benefits, risks, or side effects are discovered during the study, researchers must notify the participants. Signing the written consent form does not mean that patients are obligated to remain in the study for its duration – they may withdraw from the study at any time.


Who pays for the cost of clinical trials?

The trial sponsor, which may be a government entity, a research institution, a business such as a pharmaceutical company, or any combination thereof, usually pays for the cost of the treatment being studied and for costs associated with any special testing or extra doctor visits that may be required. Travel costs and routine costs (which are the usual costs of medical care, such as doctor visits, hospital stay, laboratory tests, X-rays, etc., that you would receive whether or not you were participating in a clinical trial), may not be covered by your health plan while you are in a trial.


In the United States, certain states now have laws that require insurers to pay for the costs of routine care that occur doing the course of a clinical trial; if you are eligible, Medicare may also cover some of the costs connected with a clinical trial. Contact your health insurer or the clinical trial research coordinator, research nurse, or social worker for assistance in determining what costs will be covered and what your financial responsibility will be. Because a lack of coverage for some of these costs can keep people from enrolling in trials, the National Cancer Institute is working with major health plans to find solutions.


What are the different “phases” of clinical trials?

Clinical trials are usually conducted in a series of steps, called phases.

Phase I trials are the first step in testing a new treatment in humans. Researchers evaluate what dose is safe, how a new agent should be given (by mouth, injection into a vein, injection into a muscle) and how often. Researchers watch for harmful side effects. The dose of the new therapy or technique is increased a little at a time. The highest dose with an acceptable level of side effects is determined to be appropriate for further testing. Phase I trials usually include only a limited number of patients and are often carried out at a few large academic medical centers.

Phase II trials attempt to determine whether the new agent or technique works for a specific type of cancer and continue to study its safety and effectiveness.

Phase III trials compare the treatment outcomes of patients taking the new therapy with results of people taking standard treatment. Participants are randomly assigned to the standard (also called control) group or to the new treatment group. This method, called randomization, helps to avoid bias and ensures that human choices or other factors do not affect the study’s results. Randomized trials are sometimes set up as “single-blind” or “double-blind” studies – in single-blind studies, patients do not know whether they are receiving the new treatment or the standard treatment; in double-blind studies, research coordinators know which treatment is being administered, but neither the patient nor the clinical physicians and nurses know. In most cases, studies move into Phase III testing only after they have shown promise in Phases I and II. Phase III trials often include large numbers of participants.

Phase IV trials occur after a treatment has been approved and is being marketed. The drug’s maker may study it further to evaluate the side effects, risks, and benefits over a longer period of time and in a larger number of people than in Phase III clinical trials.



Additional information on clinical trials can be found at the organizations listed below:


American Cancer Society  phone 1-800-303-5691


Australia and New Zealand Clinical Trials Registry (ANZCTR)


Canadian Cancer Trials


Cedars-Sinai Clinical Trials


Coalition of Cancer Cooperative Groups


Dana-Farber Cancer Institute (DFCI) Clinical Trials   phone 1-866-790-4500


Education Network to Advance Cancer Clinical Trials (ENACCT)


Fred Hutchinson Cancer Research Center


Herbert Irving Comprehensive Cancer Center of Columbia University  phone 1-212-305-8615


Lymphoma Research Foundation (LRF)   phone 1-800-500-9976


Mayo Clinic Clinical Trials  phone 1-800-664-4542


National Cancer Institute (NCI)  phone 1-888-624-1937


National Institutes of Health (NIH)


Scripps Cancer Center Clinical Trials  phone 1-858-345-7018


The Center for Information & Study on Clinical Research Participation (CISCRP)


The Leukemia & Lymphoma Society (LLS)  phone 1-800-955-4572


The University of Texas MD Anderson Cancer Center  phone 1-877-632-6789


New York-Presbyterian Hospital/Weill Cornell Medical College Clinical Trials  phone 646-962-2064


World Health Organization International Clinical Trials Registry Platform (WHO ICTRP)





Below you will find several resources around the world that are designed specifically to provide patients, their caregivers and family members, health care professionals, researchers, and the public with easy access to information on publicly and privately supported clinical studies on a wide range of diseases and conditions.  Once you access one of these online resources, you will then have to enter the specific search term you are researching (such as “waldenstrom”, immunoglobulin, neuropathy, hypogammaglobulinemia, etc.).  For the ClinicalTrials.gov, IWMF volunteers have already done the searching for you for WM-specific trials (see below).


United States  ClinicalTrials.gov Registry



ClinicalTrials.gov is maintained by the National Library of Medicine (NLM) at the National Institutes of Health (NIH).  Studies listed in the database are conducted in all 50 States and in 184 countries.


ClinicalTrials.gov - WM Specific Listing:

List of clinical trials for WM, currently active and listed on ClinicalTrials.gov.  This information will be periodically updated, so keep checking here for clinical trials that might be of interest to you.  Basic information is included for each trial – if you desire additional information regarding eligibility criteria, location, etc., please click on each trial name in the listing to view the complete trial summary.  Click here also to view the list in PDF format.


Canada Clinical Trials Registries


Canadian Cancer Trials



This cancer trial website was created by the Candian Partnership Against Cancer and its partners to help people learn about lcinical trials for cancer.  It includes trials for all the provinces in Canada.


Health Canada Clinical Trials Database



Health Canada develped this database to provide to the public a listing of specific informaiton relating to phase I, II, and III clinical trials in patients.  It is managed by Health Canada and provides a source of informaiton about Canadian clinical trials involving human pharmaceutical and biological drugs.


European Union Clinical Trials Register



The EU Clinical Trials Register website allows you to search for information on clinical trials in European Union (EU) member states and the European Economic Area (EEA) and clinical trials which are conducted outside the EU/EEA if they form part of a paediatric investigation plan (PIP).  The website is hosted by the European Medicines Agency (EMA).


Australian New Zealand Clinical Trials Registry



The Australian New Zealand Clinical Trials Registry (ANZCTR) is an online register of clinical trials being undertaken in Australia, New Zealand and elsewhere. The ANZCTR includes trials from the full spectrum of therapeutic areas of pharmaceuticals, surgical procedures, preventive measures, lifestyle, devices, treatment and rehabilitation strategies and complementary therapies.


World Health Organization (WHO) International Clinical Trials Registry Platform



The main aim of the WHO ICTRP is to facilitate the prospective registration of the WHO Trial Registration Data Set on all clinical trials, and the public accessibility of that information.   The intent is to “establish a voluntary platform to link clinical trials registers in order to ensure a single point of access and the unambiguous identification of trials with a view to enhancing access to information by patients, families, patient groups and others".

Brazilian Clinical Trials Registry / Registro Brasileiro de Ensaios Clinicos (ReBEC)



Chinese Clinical Trial Registry (ChiCTR)


Cuban Public Registry of Clinical Trials (RPCEC)



German Clinical Trials Register (DRKS)



India Clinical Trials Registry



Iranian Registry of Clinical Trials



Japan Primary Registries Network (JPRN)



Korea  Clinical Resarch Information Service (CRIS)



The Netherlands National Trial Register (NTR)



Pan African Clinical Trials Registry (PACTR)



Sri Lanka Clinical Trials Registry (SLCTR)