Part A: RG7388 as a single agent; given at a starting dose of 100 mg each day for five days for the first cycle which will be 56 days.
Part B: combination of RG7388 and Pegasys if subject does not achieve at least a PR by the end of 3 cycles of single agent RG7388

Polycythemia Vera

Essential Thrombocythemia

RG7388 is supplied as film-coated tablets at dosage strengths of 50 mg, 200 mg, 300mg and 400 mg.

RG7388

PEGASYS is supplied as a clear, sterile solution for subcutaneous (SQ) injection available in prefilled syringes. weekly doses at 45ug subcutaneously

Pegasys

Georgetown University Medical Center

Icahn School of Medicine at Mount Sinai

Wake Forest University Baptist Medical Center

Open Label Study of Single Agent Oral RG7388 in Patients With Polycythemia Vera and Essential Thrombocythemia

Open Label Phase I Study of Single Agent Oral RG7388 in Patients With Polycythemia Vera and Essential Thrombocythemia (With Pilot Feasibility Study in Combination With Pegylated Interferon Alfa 2a for Patients Who do Not Respond to the Single Agent at Each Dose Level)

John Mascarenhas

Associate Professor

Roche Pharma AG

The Leukemia and Lymphoma Society

Day-to-Day Care:
Advanced Practice Providers (APP) will travel to subjects' homes in the morning, where they will perform the same daily assessment as standard care. They will draw labs and bring them back to the hospital for processing. When results are available, a second home visit is made to deliver necessary interventions. Subjects will have internet access through cellular-networked iPads and have daily videoconferences with their physicians. Daily follow up at home will continue until discharge as per above criteria.
Caregivers: identified by the subject as the person taking primary care of them will answer surveys and also collect stool samples to analyze in conjunction with those from subjects.

These subjects will begin as inpatients or outpatients, where advanced practice providers (APPs) (nurse practitioners and physician assistants) will perform histories and physical exams. Nurses will collect labs and providers will enter orders in our electronic health record (EHR). All steps will be repeated daily until discharge to home. Suitability for discharge is determined by standard clinical criteria including stable blood counts, freedom from active or severe complications (e.g. active infection, severe GVHD), and ability to care for self.
Caregivers: identified by the subject as the person taking primary care of them will answer surveys and also collect stool samples to analyze in conjunction with those from subjects.

Blood Disorders

Subjects will receive their conditioning chemotherapy +/- radiation and stem cells in the hospital or day hospital, after which they will receive care during the neutropenic/recovery period at home. Advanced Practice Providers (APP) will travel to subjects' homes in the morning, where they will perform the same daily assessment as standard care. They will draw labs and bring them back to the hospital for processing. When results are available, a second home visit is made to deliver necessary interventions. Subjects will have internet access through cellular-networked iPads and have daily videoconferences with their physicians. Daily follow up at home will continue until discharge as per above criteria.

Patient-Centered Medical Home (PCMH)

Subjects will receive their conditioning chemotherapy +/- radiation and stem cells in the hospital or day hospital, after which they will receive care during the neutropenic/recovery period inpatient or outpatient. Advanced practice providers (APPs) will perform histories and physical exams. Nurses will collect labs and inpatient rounds occur twice daily, or for outpatients receiving care through our outpatient clinics, a healthcare provider typically sees them daily for the first month following transplant or discharge from the day hospital.

Standard Care

Duke University Adult Bone Marrow Transplant Clinic

Medical Home Care for Hematopoietic Stem Cell Transplantation Phase 2

Patient-Centered Medical Home Care for Hematopoietic Stem Cell Transplantation: A Randomized Phase 2 Study

The primary endpoint is the incidence (number of events) of grade II-IV acute GVHD at 6 months. GVHD will be evaluated daily by advanced practice providers (APPs) according to standard criteria. The investigators will compare GVHD incidence rates between treatment arms using survival type regression modeling that takes competing risks into account. They will examine cause-specific Cox proportional hazards models as well as the proportional subdistribution hazard regression model developed by Fine and Gray. While the primary treatment comparison will be unadjusted, adjustment for covariates to explore the impact of various risk factors will also be performed.

Rates of bacterial, fungal, and viral infections as well as overall infections at one year.

Shannon diversity index will be compared using unpaired two-sided Student's t tests with a more stringent cut-off of 0.0125 given multiple comparisons, by the Bonferroni correction for 4 time periods of independent comparisons. Comparisons of bacterial populations will be made by using the Adonis non-parametric method from within the Qiime package using UnifFrac distance matrices from the populations being compared. P values will be calculated with α= 0.05. Metastatistics will be used for further statistical analyses of population structure, membership, and diversity with metadata such as time from transplant and dietary measurements such as caloric intake. Changes in specific bacterial families of interest will be compared using a two-sided Student's t test, with normality confirmed by D'Agostino and Pearson omnibus test with p≤0.05. All other comparisons will be done using two-sided Mann-Whitney tests.

Duke University

Total of 24 cycles of lenalidomide. Subjects received a starting daily dose of 10mg lenalidomide on days 1 through 28 of each 28 day cycle. Subjects initiated lenalidomide 28-100 days post-ASCT.

Total of twenty-four (24) 28-day cycles of Lenalidomide, 10mg, oral tablets, daily.

Maintenance Lenalidomide in Lymphoma

Sex: Female, Male

Ethnicity (NIH/OMB)

Race (NIH/OMB)

Region of Enrollment

Lymphoma sub-type

Dose-limiting toxicity (DLT) is defined as any grade 3 toxicity or higher that occurs during the first 28 days of therapy and is possibly, probably, or definitely related to lenalidomide maintenance.

Total of twenty-four (24) 28-day cycles of Lenalidomide, 10mg, oral tablets, daily

Number of Subjects With Dose-limiting Toxicities

Progression free survival (PFS) is defined as days from start of high dose chemotherapy to first documented progression of disease, death due to any cause or last patient contact.

Progression Free Survival

Overall survival (OS) is defined as days from start of high dose chemotherapy to death due to any cause or last patient contact.

Overall Survival

Overall Study

Jakub Svoboda, MD. Study Principal Investigator

Total of twenty-four (24) 28-day cycles of lenalidomide, 10mg, oral tablets, daily
Lenalidomide: Lenalidomide, 10mg, oral tablets, daily

pancytopenia/braken

Open Label Study of Single Agent Oral RG7388 in Patients With Polycythemia Vera and Essential Thrombocythemia

Medical Home Care for Hematopoietic Stem Cell Transplantation Phase 2

Maintenance Lenalidomide in Lymphoma

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