CLINICAL TRIAL UPDATES
Clinical Trials for Rare Diseases
Below , we take a look at ongoing clinical trials for rare diseases . In addition to searching for new therapeutic targets and developing novel drugs for rare conditions , many researchers are investigating combinations of currently available therapies to treat diseases for which there are no or limited therapeutic options .
BONE MARROW FAILURE DISEASES
A Multicenter , Open-Label , Pilot Study of Alisertib ( MLN8237 ), a Novel Inhibitor of Aurora Kinase A , in Adult Patients With Relapsed / Refractory Acute Megakaryoblastic Leukemia or Myelofibrosis ( Including Primary and Post-Essential / Post- Polycythemic Myelofibrosis ) ( NCT02530619 )
study design : Multicenter , open-label , pilot safety / efficacy study study start date : October 2015 estimated study completion date : July 2017 study status : Currently recruiting participants
estimated enrollment : 24 sponsor : Northwestern University This study will evaluate the efficacy and safety of the investigational drug alisertib in patients with acute megakaryoblastic leukemia ( AMKL ) or myelofibrosis ( MF ). Alisertib has been shown preclinically to have an effect on megakaryocytes , which produce platelets and is known to be defective in both AMKL and MF .
Ruxolitinib Versus Best Available Therapy in Patients With High-risk Polycythemia Vera or High-risk Essential Thrombocythemia - The Ruxo-BEAT Trial ( NCT02577926 )
study design : Randomized , parallel-group , open-label safety / efficacy study
study start date : October 2015 estimated study completion date : December 2020 study status : Currently recruiting participants
estimated enrollment : 380 sponsor : RWTH Aachen University Ruxolitinib is a JAK1 / 2-specific tyrosine kinase inhibitor that has been approved for the treatment of symptomatic MF and polycythemia vera ( PV ) in those who have had an inadequate response to or are intolerant of hydroxyurea . Ruxolitinib is being studied in phase II / III clinical trials for high-risk hydroxyurearesistant or -intolerant PV and essential thrombocythemnia ( ET ). This study will assess the feasibility , efficacy , and safety of ruxolitinib treatment compared with best available treatment in patients with high-risk PV or ET .
Polycythemia Vera , Myelofibrosis and Essential Thrombocythemia : Identification of PV , MF & ET Genes ( NCT00715247 )
study design : Observational , case-controlled , prospective study study start date : July 2006 estimated study completion date : July 2020 study status : Currently recruiting participants
estimated enrollment : 700 sponsor : University of Utah Researchers are hoping to identify genes whose mutations cause these myeloproliferative disorders . Finding these genes would aid in developing better diagnostic measures for these diseases and new therapies .
Extended Dosing With Eltrombopag in Refractory Severe Aplastic Anemia ( NCT01891994 )
study design : Open-label efficacy study study start date : June 2013 estimated study completion date : May 2020 study status : Currently recruiting participants
estimated enrollment : 60 sponsor : National Heart , Lung , and Blood Institute Severe aplastic anemia ( SAA ) is a life-threatening blood disease that can be successfully treated with immunosuppressive drug regimens or allogeneic hematopoietic cell transplantation ( HCT ). However , 20 to 40 percent of patients are ineligible for transplant due to the lack of an appropriate donor , age , or comorbidities . Eltrombopag , a second generation oral small molecule TPOagonist , is being investigated for the treatment of SAA because it has been shown to increase platelets in healthy patients and thrombocytopenic patients .
A Two-Arm Phase II Clinical Study of the Clinical Efficacy and Safety of Tosedostat in Patients With Myelodysplastic Syndromes ( MDS ) After Failure of Hypomethylating Agent- Based Therapy ( NCT02452346 )
study design : Non-randomized , open-label , single-group assignment safety / efficacy study
study start date : February 2015 estimated study completion date : September 2018 study status : Currently recruiting participants
estimated enrollment : 80 sponsor : Weill Medical College of Cornell University Tosedostat , an oral inhibitor of aminopeptidase that influences cellular protein clearance , has demonstrated activity in relapsed / refractory acute myeloid leukemia . This investigator-sponsored study will examine its efficacy in myelodysplastic syndromes .
Controlled Study of Rigosertib Versus Physician ’ s Choice of Treatment in MDS Patients After Failure of an HMA ( INSPIRE ) ( NCT02562443 )
study design : Randomized , parallel-assignment , open-label safety / efficacy study
study start date : October 2015
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