Announcing NEW Clinical Trials:
CLINICAL RESEARCH TRIAL WITH BIRINAPANT (TL32711) AND 5-AZACITIDINE IN HIGH RISK MDS NAÏVE, RELAPSED OR REFRACTORY PATIENTS
NOW ENROLLING IN DOSE EXPANSION PHASE: NCT01828346
We would like to announce a clinical trial study for high risk naïve, relapsed or refractory Myelodysplastic Syndrome (MDS) patients.
Primary Objective of this study is to determine the maximum tolerated dose (MTD), recommended Phase 2 dose, and pharmacodynamics (PD) of birinapant (TL32711) when administered in combination with 5-azacitidine (5 AZA) in subjects with myelodysplastic syndrome (MDS) who are naïve, refractory or have relapsed to 5-AZA therapy.
Secondary Objectives of the study are to determine the clinical activity using the International Working Group (IWG) (Cheson, 2006) Response Criteria for MDS during the Phase 1b dose escalation stage of the study and in the Phase 2a expansion cohort, to determine the pharmacokinetics (PK) of birinapant when administered with 5-AZA in plasma and to assess exploratory translational biomarkers of anti-tumor activity of birinapant in combination therapy.
In an effort to accelerate the clinical development of birinapant for myelodysplastic syndromes the MDS Foundation will be assisting the sponsor and would appreciate hearing from you.
If you are a physician or health care provider and would like to refer a patient for enrollment into this clinical trial OR if you are an MDS patient who is newly diagnosed with MDS or has failed, become intolerant, or relapsed after Vidaza® (azacitidine) or Dacogen® (decitabine) treatment, please see additional information and contact details at http://clinicaltrials.gov/ct2/show/NCT01828346.
CLINICAL RESEARCH TRIAL WITH 5-AZACITIDINE WITH/WITHOUT BIRINAPANT
(TL32711) IN HIGHER RISK MDS/CMML PATIENTS
NOW ENROLLING: NCT02147873
We would like to announce a clinical trial randomized double blind, placebo controlled, multicentric study for higher risk newly diagnosed Myelodysplastic Syndrome (MDS)/Chronic myelomonocytic leukemia (CMML) patients.
Primary Objective of this study is to compare the relative effect of azacitidine plus birinapant versus azacitidine plus placebo on response rate [RR=complete response (CR) + partial response (PR)] using the modified International Working Group (IWG) Response Criteria for MDS, in patients with higher-risk MDS or CMML.
Secondary Objectives of the study are to compare the effect of azacitidine plus birinapant relative to azacitidine plus placebo, on the hematologic improvement (HI), relapse-free survival according to modified IWG 2006 criteria, time to response, change in transfusion requirements, duration of response according to modified IWG 2006 criteria, overall
urvival and adverse event (AE) profile.
The exploratory objective of the study is to assess exploratory translational biomarkers for antitumor effects.
Study description: Approximately 158 patients (10 patients in initial open label single arm safety run in portion and 148 subjects in the double blinded placebo controlled randomized portion) with higher risk Myelodysplastic syndrome
yelomonocytic leukemia (CMMoL) who are naïve, to azacitidine therapy will be enrolled.
In an effort to accelerate the clinical development of birinapant for myelodysplastic syndrome and CMML the MDS
oundation will be assisting the sponsor and would appreciate hearing from you.
If you are a physician or health care provider and would like to refer a patient for enrollment into this clinical trial OR if
you are an MDS patient who is newly diagnosed with MDS please see additional information and contact details on
NICE Proposes to Recommend Lenalidomide for MDS
NICE has issued final draft guidance recommending lenalidomide (also known as Revlimid and marketed by Celgene) as an option, for treating myelodysplastic syndromes.
Myelodysplastic syndromes (MDS), which are diagnosed in around 2000 people each year in England, are a group of bone marrow disorders characterised by the underproduction of one or more types of blood cells due to dysfunction of the marrow. MDS can lead to life threatening disease including acute myeloid leukaemia (AML), as well as anaemia and increased risk of bleeding and infections.
This appraisal focused on the use of lenalidomide for treating people with a specific type of MDS that is characterised by a chromosomal abnormality called an isolated deletion 5q cytogenetic abnormality. At the moment the main treatment option for people with the particular kind of MDS considered in this appraisal is best supportive care including regular blood transfusions.
Commenting on the final draft guidance, Sir Andrew Dillon, NICE chief executive, said: “The committee heard from clinical experts that lenalidomide is an effective therapy. Celgene – who market lenalidomide – worked with us to provide enough evidence to make it possible for us to recommend it for this group of people.
“Celgene provided a revised analyses and further information on their proposal for a reduction in the cost of the drug to the NHS (patient access scheme).”
Celgene’s patient access scheme involves the NHS paying for lenalidomide treatment for up to 26 monthly cycles. The company will provide the drug free of charge for those people who receive more than 26 monthly cycles.
The draft guidance is now with consultees, who have the opportunity to appeal against it. Until NICE issues final guidance, NHS bodies should make decisions locally on the funding of specific treatments.
Important new survey started by EHA – the European Haematology Association. EHA is interested in finding out if certain medicines are not available in some parts of the country – or in some parts of Europe. Patients, Caregivers, and Healthcare professionals are encouraged to complete this survey. The information from this survey will educate European policy makers to take measures that will help provide effective medicines that are not affordable or are not marketed in specific countries.
GREAT NEWS Italian Study Supports Use Of Revised International Prognostic Scoring! Under the aegis of the MDS Foundation, the International Working Group for Prognosis in MDS generated an improved method analyzing MDS patient prognosis more precisely than the initial IPSS. ▼ Read More
Revlimid® Approved In Europe For Use in MDS
The European Medicines Agency (EMA) has approved Revlimid® for the treatment of MDS patients with transfusion-dependent anemia associated with the del(5q) chromosomal abnormality.
REVISED INTERNATIONAL PROGNOSTIC SCORING SYSTEM FOR MDS
The manuscript describing the Revised IPSS (IPSS-R) for MDS is available now in the September 23, 2012 issue of Blood (vol.120, p2454. Under the aegis of the MDS Foundation, the International Working Group for Prognosis in MDS (IWG-PM) analyzed clinical features and outcome data from over 7000 patients and generated a method analyzing MDS patient prognosis more precisely than the initial IPSS.
Novel components of this prognostic system include: five rather than three cytogenetic prognostic subgroups with specific and new classifications of a number of less common cytogenetic subsets, splitting the low marrow blast percentage value, and depth of cytopenias. In addition to the major prognostic variables of marrow blasts, cytogenetics and peripheral cytopenias, additive features for survival include patient age, performance status, serum ferritin and LDH.
The IPSS-R calculator tool is accessible through the following URLs : http://www.mds-foundation.org/ipss-r-calculator/index.php or http://www.ipss-r.com. An iPhone App for the IPSS-R calculator tool is also accessible through the Apple Store (enter MDS IPSS-R).
This IPSS-R should prove beneficial for predicting the clinical outcomes of untreated MDS patients and aiding design and analysis of clinical trials in this disease. Copies of this manuscript are available upon request from the MDS Foundation and can also be accessed online at: http://www.mds-foundation.org/revised-prognostic-system-for-mds/
MDSF Goes to the New Jersey State House
The MDS Foundation, Inc. will be joining the National Organization for Rare Disorders (NORD) and others around the world in observing World Rare Disease Day on February 28, 2013.
To raise MDS awareness at the state level and Rare Disease Day we will be going to the New Jersey State House with other patient groups on March 4th, 2013. Our message is to ensure the needs of all rare disease patients are considered in future legislative policy and making sure “Every Patient Counts” is a NJ State imperative.
Senator Bucco’s office is creating 2 Resolutions for us for Rare Disease Day. The first one will be honoring all the Rare Disease Organizations in New Jersey. The 2nd Resolution will be stating that the last day of February is acknowledged as Rare Disease Day in New Jersey.
MDS is a puzzling, life-threatening group of diseases of the bone marrow for which there are no easy cures or quick remedies. In MDS, the bone marrow is abnormal because of a variety of malignant changes. The result is ineffective production of normal mature blood cells, resulting in low blood counts (cytopenias). Various subtypes of the disease exist with variable prognoses, treatment options, and risk of developing leukemia.
A rare disease is one that affects fewer than 200,000 Americans. There are nearly 7,000 such diseases affecting nearly 30 million Americans.
On Rare Disease Day, people with rare diseases around the world promote awareness of the challenges of living with a rare disease. The global theme for 2013 is “Rare Disorders Without Borders.”
World Rare Disease Day was launched in Europe four years ago and last year was observed in more than 60 nations. It is always observed on the last day of February. On that day, patients and patient organizations will post stories, videos and blogs online and host events to raise awareness of these diseases, which are often called “orphans”.
This year, the observance has special significance in the U.S. because 1983 is also the 30th anniversary of the Orphan Drug Act, which provides incentives to encourage companies to develop treatments for rare diseases, and of NORD, which was established by patient advocates in 1983.
In 1983, the Orphan Drug Act was passed by Congress to create financial incentives for companies to develop treatments for rare diseases. Since then, more than 400 orphan drugs and biologics have been approved by the Food and Drug Administration (FDA). It is estimated that approximately 15 million Americans benefit from these products, but that still leaves millions more with diseases for which there is no approved treatment.
MDS World Awareness Day Film Clip
This video was made to mark the first MDS World Awareness Day – on 25th October – and to illustrate the Life Beyond Limits campaign run in several countries already.
This campaign highlights the fact that all MDS patients deserve to have access to treatment – regardless of their age – so that patients are able to enjoy a better quality of life and a Life Beyond Limits.
This brilliant clip was made by the spanish patient group AEAL www.aeal.es – who cover MDS, Lymphoma, Myeloma and Leukaemia, as part of the Life Beyond Limits campaign. We thank them for their great creative work and cooperation. At a later stage they will adapt the video to have english voice-over – but for now we hope you will like the international cooperation.
MDS FOUNDATION A VALUED PARTNER OF GUIDESTAR
We are proud to be a valued partner of GuideStar, America’s first information service publicizing information about nonprofit organizations. Access the GuideStar link for additional financial information or to learn more about ways to give an annual or planned gift to fund MDS programs and services.
Launch of Fast Track for First Prescription™ Service through Celgene Patient Support®
Fast Track for First Prescription Program™
Fast Track for First PrescriptionTM is an optional Celgene Patient Support® service which helps patients on an oral Celgene product receive their first prescription faster.
Additional details about eligibility and enrollment are addressed below.
For more information, or for any questions, call Celgene Patient Support® at 1-800-931-8691.
Which patients are eligible for this program?
• Patients on an oral Celgene product receiving their first prescription
• Patients with documented proof of insurance
• Patients who are registered in a Celgene risk management (REMS) program and have a valid authorization number
How are eligible patients enrolled in this program?
• Prescriber must fax the Fast Track Cover Sheet and the Patient Prescription Form with the authorization number and the patient risk category
(i.e., adult female of childbearing potential, adult male, etc) to 1-800-822-2496
• Patient’s contact information and insurance information should be included
• Prescriber must ensure patient has completed their Celgene risk management (REMS) survey
What can a patient expect after being enrolled in this program?
The specialty pharmacy will contact the patient to:
• Review their insurance benefits for the prescribed product (i.e., co-pay responsibilities and potential financial co-pay assistance options)
• Provide appropriate counseling (if applicable)
• Set up a delivery date for shipment of the medication
Am J Med MDS Supplement - View the MDS Supplement in the American Journal of Medicine. This supplement is supported by Celgene Corporation. These articles are available on the internet through open access, allowing interested parties to download the entire supplement for free.
MDS Trends and Treatments was the focus of a recent Leukaemia Foundation education session in Melbourne, Australia. The two hour presentation, featuring Dr. Michael Dickinson, Haematologist from Peter Mac Callum Centre, and Haematology Oncology Nurse Sandra Kurtin from the MDS Foundation Board of Directors and Nurse Leadership Board, was recorded and is available to view here.
Myelodysplastic Syndromes (MDS) Event Free Survival With Iron Chelation Therapy Study (TELESTO)
Currently Recruiting Participants. The primary purpose of this study is to prospectively assess the efficacy and safety of iron chelation therapy with deferasirox compared to placebo in patients with myelodysplastic syndromes (low/int-1 risk) and transfusional iron overload. Contact the Novartis Clinical Trials Hotline at 800-340-6843 or click here for additional information and to view the active sites.