Trial Roundup
ASH Clinical News ’ Associate Editors select clinical trials to keep an eye on .
THE BEAT AML MASTER TRIAL
Study Design : Observational , prospective cohort study Study Start Date : November 2016 Estimated Study Completion Date : November 2023 Study Status : Currently recruiting participants Estimated Enrollment : 500 Sponsor : The Leukemia & Lymphoma Society
The American Society of Hematology and The Leukemia & Lymphoma Society ( LLS ) have teamed up to raise awareness and provide education about the need for new treatments for acute myeloid leukemia ( AML ).
After launching the Beat AML initiative in 2013 to bring together researchers , patients , pharmaceutical companies , and physicians to analyze the genomic causes of AML and identify new treatment options , in mid-October LLS announced the Beat AML Master Trial , in which older AML patients will undergo genomic testing to assign them to a treatment strategy . The study is led by principal investigators John C . Byrd , MD , from The Ohio State University Comprehensive Cancer Center – Arthur G . James Cancer Hospital and Richard J . Solove Research Institute ; Brian Druker , MD , of OSHU Knight Cancer Institute ; and Ross Levine , MD , from the Memorial Sloan Kettering Cancer Center .
“ We have seen too few drugs achieve approval for AML treatment , and , even when drugs are approved , getting them into the clinic takes too long and these agents generally are not tailored to a patient ’ s genotype ,” Dr . Levine told ASH Clinical News , adding that “ This trial builds on recent successes in AML to rapidly test AML drugs in the populations most likely to respond . We hope this trial increases the speed in which we can bring new drugs to AML patients and provides a template for new master trials and drug development in a wider spectrum of human cancers .” The Beat AML Master Trial will enroll more than 500 adult patients ( ≥60 years ) with newly diagnosed , untreated AML , over the course of three to five years , and comprises three phases :
1 . Patients will undergo a bone marrow biopsy , followed by genomic screening within a sevenday period .
2 . Based on their genetic profile , patients will be assigned to receive personalized investigational therapy . Unlike other clinical trials where one drug or drug combination is studied , this trial will begin with four different treatments . Combination regimens may also be studied during the trial . If a patient does not have a genetic marker that would place him or her in a specific treatment cohort , patients will be offered a different investigational broad-acting AML agent .
3 . Follow-up period of ≥28 days after treatment is complete . The total duration may last from one to three years .
The trial will also include patient-reported outcomes . The first patients are expected to be enrolled by
December from the five participating centers :
• Memorial Sloan Kettering Cancer Center in New York
• The Ohio State University Comprehensive Cancer Center in Ohio
• OHSU Knight Cancer Institute in Oregon
• Dana-Farber Cancer Institute in Massachusetts
• Massachusetts General Hospital Cancer Center in Massachusetts
Six additional clinical sites are prepared to begin enrolling patients next year , and the trial will eventually expand to between 15 and 20 sites , with up to 10 different treatment arms . Trial participants will be treated with one of four investigational drugs ( samalizumab , BI 836858 , enasidenib , entospletinib ) provided by participating pharmaceutical companies . “ This precision medicine approach is aimed to bring the best targeted drugs to AML patients at the time of diagnosis and to show how genomic profiling can be used to inform clinical trial development and to accelerate drug discovery ,” Dr . Levine said .
Visit lls . org / beat-aml for more information on the trial .
LEUKEMIA
Mikkael Sekeres , MD , MS Cleveland Clinic
The National Myelodysplastic Syndromes ( MDS ) Study ( NCT02775383 )
Study Design : Prospective registry , natural history study Study Start Date : April 2016 Estimated Study Completion Date : September 2021 Study Status : Currently recruiting participants Estimated Enrollment : 3,500 Sponsor : National Heart , Lung , and Blood Institute
The goal of the National MDS Study , the largest MDS natural history study ever conducted in the United States , is to establish a publicly available resource to facilitate the study of MDS . This will be accomplished through : 1 ) creation of a multi-institutional , longitudinal collection of consistently processed and clinically well-annotated blood and tissue specimens collected prospectively from participants with MDS and participants with idiopathic cytopenia of undetermined significance ( ICUS ); and 2 ) support for investigator-initiated studies of MDS that will have high-impact for MDS patients , including basic science , clinical , health outcomes and epidemiological research . This multicenter , prospective cohort study is enrolling patients from centers in the National Cancer Institute ’ s ( NCI ) National Clinical Trials Network and NCI Community Oncology Research Program . The accrual period is more than five years . After a central pathology review of registered participants , approximately 2,000 cases of MDS or MDS / myeloproliferative neoplam overlap disorders , and 500 cases of ICUS will be identified for the longitudinal study cohort and up to 1,000 cases will be identified for the cross-sectional cohort . No more than 3,500 total participants will be registered . Participants in the longitudinal cohort may be followed for life .
LYMPHOMA & MYELOMA
Keith Stewart , MBChB , MBA Mayo Clinic , Arizona
Elevate CLL R / R : A Randomized , Multicenter , Open-Label , Non-Inferiority , Phase 3 Study of ACP-196 Versus Ibrutinib in Previously Treated Subjects With High Risk Chronic Lymphocytic
Leukemia ( NCT02477696 )
Study Design : Randomized , parallel-assignment , open-label safety / efficacy study Study Start Date : June 2015 Estimated Study Completion Date : June 2019 Study Status : Currently recruiting participants Estimated Enrollment : 500 Sponsor : Acerta Pharma BV
This phase III trial of acalabrutinib , a novel Bruton tyrosine kinase inhibitor , will investigate the agent in patients with “ high-risk ” chronic lymphocytic leukemia . The goal of the new treatment is to retain efficacy – with more “ on-target ” effects – while lowering the associated toxicity .
The VITAL Amyloidosis Study , a Global Phase 3 , Efficacy and Safety Study of NEOD001 in Patients With AL Amyloidosis ( VITAL ) ( NCT02312206 )
Study Design : Randomized , parallel-assignment , double-blind safety / efficacy study Study Start Date : February 2015 Estimated Study Completion Date : August 2018 Study Status : Currently recruiting participants Estimated Enrollment : 236 Sponsor : Prothena Therapeutics , Ltd .
NEOD001 , is a humanized murine monoclonal antibody , which is directed against a cryptic epitope on amyloid fibrils . NE0D001 specifically targets misfolded light chain aggregates and amyloid deposits . This trial will evaluate the efficacy of NEOD001 versus placebo ( administered intravenously ) added to standard of care in patients with amyloid light-chain amyloidosis by assessing time to all-cause mortality or cardiac hospitalization . ●
30 ASH Clinical News November 2016