A type of research study that tests how a drug, medical device, or treatment approach works in people. There are several types of clinical trials. Treatment trials test new treatment options. Diagnostic trials test new ways to diagnose a disease. Screening trials test the best way to detect a disease or health problem. Quality of life (supportive care) trials study ways to improve the comfort of people with chronic illness. Prevention trials look for better ways to prevent disease in people who have never had the disease.
Tests a new drug or treatment in a small group to see if it is safe.
Expands the study to a larger group of people to find out if it works.
Expands the study to an even larger group of people to compare it to the standard treatment for the disease.
Takes place after the drug or treatment has been licensed and marketed to find out the long-term impact of the new treatment.
INternational Study of Phase III Intravenous RigosErtib
A Phase III, international, randomized, controlled study of Rigosertib + best supportive care versus physician’s choice of treatment + best supportive care in patients with myelodysplastic syndrome (MDS) after failure of a hypomethylating agent (HMA).
Overall survival in the intention-to-treat population and in patients with very high risk per the Revised International Prognostic Scoring System (Greenberg et al, Blood 2012).
More than 200 trial sites, globally.
For additional information on this study, please call the INSPIRE help line at 1-267-759-3676 or visit www.clinicaltrials.gov, identifier: NCT02562443.
Rigosertib is an investigational agent and is not approved by the FDA or other regulatory agencies worldwide as a treatment for any indication.
A Phase 2, Randomized, Controlled, Open-Label, Clinical Study of the Efficacy and Safety of Pevonedistat Plus Azacitidine Versus Single-Agent Azacitidine in Patients with Higher-Risk Myelodysplastic Syndromes (MDS), Chronic Myelomonocytic Leukemia (CMML), and Low-Blast Acute Myelogenous Leukemia (AML).
Takeda Pharmaceuticals International Co. is currently enrolling patients for a Phase 2 clinical trial of the study drug pevonedistat. The purpose of this study is to evaluate the efficacy and safety of pevonedistat plus azacitidine versus single-agent azacitidine in participants with higher-risk myelodysplastic syndromes, chronic myelomonocytic leukemia and low-blast acute myelogeneous leukemia.
This study will look at the overall survival, event-free survival and response to treatment in people who take pevonedistat and azacitidine when compared to people who take single-agent azacitidine.
The study will enroll approximately 117 participants. Once enrolled, participants will be randomly assigned (by chance, like flipping a coin) to one of the two treatment groups in a 28 day treatment cycle:
All participants will receive azacitidine via the intravenous or subcutaneous route. Participants randomized to the combination arm will also receive pevonedistat intravenous infusion. This multi-center trial will be conducted worldwide. Patients may qualify for this study if:
In order to refer a patient with MDS, CMML, or low-blast AML for enrollment to this study and review eligibility criteria, physicians/health care providers should visit www.clinicaltrials.gov(NCT02610777)
Contact: 1-877-674-3784; firstname.lastname@example.org
Pevonedistat is an investigational agent and is not approved by the FDA or other regulatory agencies worldwide as a treatment for any indication.
Takeda Oncology is a trademark of Takeda Pharmaceutical Company Limited. Millennium Pharmaceuticals, Inc. is a wholly owned subsidiary of Takeda Pharmaceutical Company Limited. ©2016 Millennium Pharmaceuticals, Inc.
MEDALIST is an international clinical trial for people who have myelodysplastic syndrome (MDS). MEDALIST will determine whether an investigational drug called luspatercept* can help
of very low–, low-, or intermediate risk and require regular blood transfusions, you may be eligible to take part in an international clinical trial called the MEDALIST Trial.
Ask your doctor about the MEDALIST Trial. Together you can decide if this clinical trial is right for you.
*The safety and efficacy of luspatercept has not been established. There is no guarantee that luspatercept will become commercially available.
San Diego-based La Jolla Pharmaceutical Company is currently seeking qualified patients for consideration in their trial in Patients at Risk for Iron Overload using its novel formulation of hepcidin (LJPC-401).
Hepcidin, an endogenous peptide hormone, is a naturally occurring regulator of iron absorption and distribution. Manipulation of hepcidin has the potential to prevent excessive iron accumulation in vital organs, such as the liver and heart, where it can cause significant damage and even result in death. La Jolla is developing LJPC-401 for the potential treatment of iron overload, which occurs as a result of diseases such as myelodysplastic syndrome (MDS), beta thalassemia, hereditary hemochromatosis, and sickle cell disease (SCD).
LJPC-401 has been shown to be effective in reducing serum iron in preclinical testing involving animals. La Jolla’s Phase 1 clinical trial of LJPC-401 in patients at risk of iron overload is currently ongoing. Phase 1 trials are intended to evaluate safety and potential dosing requirements and are not intended to evaluate efficacy.
La Jolla is currently seeking patients with conditions putting them at risk for iron overload for a Phase 1 clinical trial at study centers in the United States.
Enrollment criteria include the following:
Patients with any of the following disorders:
1. Refractory or hemolytic anemia of any type. Examples include, but are not limited to, MDS, hemoglobinopathies, sideroblastic anemia, and congential anemias
Who have been transfused with at least 2 units of blood over the past two (2) months, OR 4 units of blood over the past six (6) months, OR received any form of iron chelation therapy over the past six (6) months, OR have a serum ferritin > 1,000 μg/L.
2. Patients with hemochromatosis that require phlebotomy at least once every two months OR have received iron chelation therapy in the past six (6) months.
Patients 18-85 years of age, inclusive.
Anyone wishing more information about this study may contact the following clinical trial sites:
Cincinnati, OH @ Jennifer Palmer – (513) 721-3868 http://www.ctifacts.com/research-center.aspx
San Diego, CA @ Ruth Garcia – (877) 500-3788 http://estudysite.com/studies
Myelodysplastic Syndromes (MDS) remain difficult to treat, in part because these blood disorders are rare and researchers don’t have enough samples from patients to study. The National Myelodysplastic Syndromes Natural History Study is a chance to help improve MDS treatment in the future. For the first time ever in the U.S., this study will collect information and samples from patients with MDS, and follow those patients for years to answer questions about what causes MDS, how it gets worse over time, and what treatments are most likely to work. This National Institutes of Health (NIH) sponsored study will create a public resource by collecting clinical information and blood, bone marrow, and tissue samples from up to 2,000 people with, or suspected to have, MDS.
You may be eligible for this study if you are at least 18 years old and either:
If you participate in this study, you will receive your normal care and treatment from your doctor but will not receive additional treatment as part of this study. Instead, your normal care and treatment will be followed. This will also include following your quality of life during your time on the study.
For more information on the study, including how to find a participating hospital near you, please visit the study website at https://thenationalmdsstudy.net/ or https://clinicaltrials.gov/ (NCT02775383).
Current Status: Part 1 recruitment complete; Part 2 is not yet open for recruitment. For additional details, refer to the Geron press release (12Sep2016) at http://ir.geron.com/phoenix.zhtml?c=67323&p=irol-newsArticle&ID=2201055.
Janssen Research & Development, LLC is conducting a Phase 2/3 clinical study referred to as “IMerge”, with the study drug Imetelstat, which is a first-in-class telomerase inhibitor. With its novel mechanism of action, Imetelstat may provide clinical benefit to MDS patients. In this study, Imetelstat is administered as a 2-hour intravenous infusion every 28 days.
IMerge is a study for people with MDS who need blood transfusions due to anemia (low red blood cell counts). People with low or intermediate-1 risk MDS that has relapsed or is refractory to Erythropoiesis-Stimulating Agents (ESAs) treatment are enrolled in the study. This study is being conducted at multiple hospitals and institutions around the world, in approximately 80 sites globally.
For more information about this clinical study, please visit www.clinicaltrials.gov (NCT02598661).
This is a phase 2 study of selinexor for MDS patients (with up to 30% blasts) and CMML who have received prior decitabine or 5-azacytidine. The primary endpoint is to determine the response rate of this novel drug in patients who have refractory or relapsed disease after receiving either decitabine or 5-azacytidine.
Secondary Objectives of the study are to determine if selinexor improves the survival of people with MDS and CMML after receiving 5-azacytidine and CMML. Additional secondary endpoints include an assessment of response duration and tolerability of selinexor, and to perform research studies on blood and bone marrow samples to better understand the mechanism of selinexor in MDS and CMML.
Pfizer is conducting a clinical study to determine the safety, efficacy, PK and PD of Glasdegib (PF-04449913) or placebo when combined with azacitidine in patients with previously untreated Intermediate-2 or High Risk Myelodysplastic Syndrome (MDS), Acute Myeloid Leukemia (AML) with 20-30% blasts and multi-lineage dysplasia, or Chronic Myelomonocytic Leukemia (CMML).
This study is being conducted at multiple hospitals and institutions around the world and includes two components: (a) a Phase 1b open -label safety lead in component and (b) a Phase 2 randomized double-blind component. In the Phase 1b component, approximately 10 patients will be enrolled and will receive Glasdegib (PF-04449913) and azacitidine and in the Phase 2 component, 160 patients will be randomized to receive to receive either Glasdegib (PF-04449913) (Arm A) or placebo (Arm B) in combination with azacitidine.
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, AML, or CMML patient please see additional information and contact details at the following websites: