Blood and Lymph Conditions

All Conditions

Neoplasms

Rare Diseases

Primary Myelofibrosis

Bone Marrow Neoplasms

Myeloproliferative Disorders

Bone Marrow Diseases

Hematologic Diseases

Hematologic Neoplasms

Chronic Myeloproliferative Disorders

Antineoplastic Agents

Antisickling Agents

All Drugs and Chemicals

Hydroxyurea

Antimetabolites

Enzyme Inhibitors

Standard of care hematopoietic stem cell transplant with a matched donor.

Standard of care treatment with a janus kinase (JAK) inhibitor drug called ruxolitinib or treatment with an antimetabolite drug called hydroxyurea.

Intravenous infusion of hematopoietic stem cells from a donor.

Hematopoietic stem cell transplant

Ruxolitinib is type of drug called a janus kinase (JAK) inhibitor. Ruxolitinib is taken orally (by mouth).

Ruxolitinib

Hydroxyurea is a type of drug called an antimetabolite. Hydroxyurea is taken orally (by mouth).

Vikas Gupta, M.D.

Sonia Cerquozzi, M.D.

Elena Liew, M.D.

Lynda Foltz, M.D.

Mahmoud Elsawy, M.D.

The purpose of this research study is to see how effective hematopoietic stem cell transplantation (HCT) is compared to best available non-transplant therapies (BAT) in patients with high risk myelofibrosis. This will be done by asking participants to choose the treatment that they prefer to receive (HCT or BAT) and then comparing the outcomes of the participants in both treatment groups.

There is currently little information regarding which treatments are best for patients with myelofibrosis. On one hand, hematopoietic stem cell transplantation (HCT) is potentially curative treatment but is associated with significant risk of complications related to graft failure (the new donor cells does not grow properly after the transplant), side effects such as graft versus host disease (the patient's cells attack the new donor cells), and risk of infections. Non-transplant therapies such as ruxolitinib provide effective symptom control for few months to few years, but are not curative in nature. As such, this study will compare the effectiveness of HCT versus best available non-transplant therapies (BAT) in patients with high risk myelofibrosis.

This is an observational study, meaning that participants will be followed to assess the effects of their treatment, but no intervention (treatments) will be given as a part of this study.

Blood samples for future research related to hematological (blood) cancers and their treatments.

Study of Stem Cell Transplant vs. Non-Transplant Therapies in High-Risk Myelofibrosis

A Patient Preferences-Controlled Study of Allogeneic Hematopoietic Cell Transplantation Versus Best Available Non-Transplant Therapies in Patients With High-Risk Myelofibrosis (ALLO-BAT Study)

Time from study allocation to death or last follow up.

Range from -3 to 3. Positive number equals increase in quality of life.

Range from 0 to 10. Increase equals worsening of symptoms.

Range from 1 to 4. Increase equals increase in quality of life.

Time from allocation to study arm to death/acute myeloid leukemia transformation or last follow up.

University Health Network, Toronto

Heart and Blood Diseases

Immune System Diseases

Multiple Myeloma

Neoplasms, Plasma Cell

Neoplasms by Histologic Type

Hemostatic Disorders

Blood Coagulation Disorders

Vascular Diseases

Paraproteinemias

Blood Protein Disorders

Hemorrhagic Disorders

Lymphoproliferative Disorders

Immunoproliferative Disorders

Anti-Inflammatory Agents

Antiemetics

Gastrointestinal Agents

Dexamethasone

Lenalidomide

Dexamethasone acetate

Glucocorticoids

Hormones

Hormone Antagonists

Antineoplastic Agents, Hormonal

Immunologic Factors

Angiogenesis Inhibitors

Growth Inhibitors

Carfilzomib, lenalidomide, dexamethasone (KRd) regimen using once-weekly carfilzomib 56 mg/m2

Carfilzomib, lenalidomide, dexamethasone (KRd) regimen using twice-weekly carfilzomib 27 mg/m2

Once weekly IV over 30 minutes on day 1, 8 and 15 of each 28 day cycle. The dose will be 20 mg/m2 on cycle 1 day 1 and 56 mg/m2 beginning with cycle 1 day 8 and thereafter. 12 cycles or until progression, unacceptable toxicity, death, loss to follow up or withdrawal of consent.

Carfilzomib

Twice weekly IV over 10 minutes on day 1, 2, 8, 9, 15 and 16 of each 28 day cycle. The dose will be 20 mg/m2 on cycle 1 days 1 and 2 and 27 mg/m2 beginning with cycle 1 day 8 and thereafter. 12 cycles or until progression, unacceptable toxicity, death, loss to follow up or withdrawal of consent.

Once daily orally 25 mg days 1 to 21 of each cycle. 12 cycles or until progression, unacceptable toxicity, death, loss to follow up or withdrawal of consent

Once daily orally or by IV 40 mg days 1, 8 and 15 of each cycle. Also day 22 of cycles 1 to 9. 12 cycles or until progression, unacceptable toxicity, death, loss to follow up or withdrawal of consent

Compare efficacy of 56 mg/m2 carfilzomib administered once-weekly in combination with lenalidomide and dexamethasone (KRd 56 mg/m2) to 27 mg/m2 carfilzomib administered twice-weekly in combination with lenalidomide and dexamethasone (KRd 27 mg/m2) in subjects with relapsed or refractory multiple myeloma (RRMM) with 1 to 3 prior lines of therapy.

A Study Comparing Once-weekly vs Twice-weekly Carfilzomib in Combination With Lenalidomide and Dexamethasone in Subjects With Relapsed or Refractory Multiple Myeloma

A Randomized, Open-label, Phase 3 Study Comparing Once-weekly vs Twice-weekly Carfilzomib in Combination With Lenalidomide and Dexamethasone in Subjects With Relapsed or Refractory Multiple Myeloma (A.R.R.O.W.2)

De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request.

ORR was defined as the percentage of participants with a best overall response of stringent complete response (sCR), complete response (CR), very good partial response (VGPR), or partial response (PR) per IMWG-URC. CR: negative immunofixation on serum and urine, soft tissue plasmacytomas disappearance, \< 5% plasma cells in bone marrow (BM). sCR: CR and normal serum free light chain ratio and no clonal cells in BM by immunohistochemistry. VGPR: Serum and urine M-protein detectable by immunofixation or ≥ 90% reduction in serum M-protein (urine M-protein level \< 100 mg/24-hours). PR: ≥ 50% reduction of serum M-protein and reduction in 24-hours urinary M-protein by ≥ 90% or to \< 200 mg/24-hours. The ORR 95% confidence intervals (CIs) were estimated using the Clopper-Pearson method.

PFS rate was defined as the percentage of participants without disease progression or death due to any cause at 12 months. The PFS rate at 12 months was estimated using the Kaplan-Meier method by Klein and Moeschberger (1997). 95% CIs were estimated using the method by Kalbfleisch and Prentice (1980). PFS data was censored for participants who met any one of the following: 1. no baseline/no post-baseline disease assessments; 2. starting new anti-myeloma therapy before documentation of progressive disease (PD) or death; 3. PD or death immediately after more than 1 consecutively missed disease assessment visit (PD or death immediately after \> 63 days without disease assessment visit); 4. alive without documentation of PD; 5. lost to follow-up or withdrawn consent.

Patient-reported convenience was measured by the Patient-reported Convenience with Carfilzomib-dosing Schedule Question. The items in the questionnaire were categorized as 'Convenient', which included responses of 'Very Convenient' and 'Convenient', and 'Inconvenient' which included responses of 'Inconvenient' and 'Very Inconvenient'. The 95% CIs were estimated using the Clopper-Pearson method.

An adverse event (AE) was any untoward medical occurrence in a clinical study participant irrespective of a causal relationship with the study treatment. TEAEs were AEs starting on or after the first dose of any study drug, and up to 30 days of the last dose of any study drug, excluding AEs reported after End of Study date.

TTR was defined as the time from randomization to the earliest date when confirmed sCR, CR, VGPR, or PR per IMWG-URC was first achieved.

For participants who achieved a PR or better, i.e., sCR, CR, VGPR, or PR per IMWG-URC, the DOR was defined as the time from the earliest date when a PR or better was first achieved, and subsequently confirmed, to the earliest date of confirmed PD or death due to any cause. Median DOR was estimated using the Kaplan-Meier method. 95% CIs were estimated using the method by Klein and Moeschberger (1997) with log-log transformation. For those alive and who had not experienced PD by analysis time, DOR was censored for participants who met any one of the following: 1. no baseline/no post-baseline disease assessments; 2. starting new anti-myeloma therapy before documentation of PD or death; 3. PD or death immediately after more than 1 consecutively missed disease assessment visit (PD or death immediately after \> 63 days without disease assessment visit); 4. alive without documentation of PD; 5. lost to follow-up or withdrawn consent.

TTP was defined as the duration from randomization for the first documented disease progression per IMWG-UCR. Median TTP was estimated using the Kaplan-Meier method. 95% CIs were estimated using the method by Klein and Moeschberger (1997) with log-log transformation. TTP was censored for participants who had no confirmed PD at the last non non-evaluable (non-NE), post-baseline disease assessment or the earlier of the following, where applicable: 1. the last non-NE, post-baseline disease assessment prior to start of a new anti-myeloma treatment, or 2. the last non-NE, post-baseline assessment followed \> 63 days later by disease progression; otherwise, at randomization.

OS was defined as the time from randomization to the date of death due to any cause. Median OS was estimated using the Kaplan-Meier method. 95% CIs were estimated using the method by Klein and Moeschberger (1997) with log-log transformation. Participants still alive or lost to follow-up or withdrawn consent from study by the analysis time were censored at the date on which the participant was last known to be alive.

MRD\[-\]CR was defined as achievement of CR or better by IRC per IMWG-URC and achievement of MRD negativity as assessed by next generation sequencing method at a 10\^-5 threshold over the duration of the study. The 95% CIs for percentages were estimated using the Clopper-Pearson method.

The percentage of participants with achievement of MRD\[-\] at 12 months (± 4 weeks) from randomization, as assessed by next generation sequencing method at a 10\^-5 threshold. MRD negativity results from BM samples obtained at 8 to 13 months from randomization and prior to new anti-myeloma therapy or disease progression were considered in the calculation. The 95% CIs for percentages were estimated using the Clopper-Pearson method.

The QLQ-C30 physical function is one of the functional domains of the EORTC QLQ-C30 self-reported instrument and the physical functioning score score ranges from 0-100 points, with 100 points indicating the best possible functioning. A positive change from baseline indicated an improvement in functioning.

The QLQ-C30 role function is one of the functional domains of the EORTC QLQ-C30 self-reported instrument and the role functioning score ranges from 0-100 points, with 100 points indicating the best possible functioning. A positive change from baseline indicated an improvement in functioning.

The CTSQ measures treatment satisfaction in individuals with cancer and includes a domain for satisfaction with therapy. The satisfaction with therapy scores ranges from 0 to 100 points, with 100 points indicating greatest satisfaction.

Analysis was based on ANCOVA model. The dependent variable of the models were the scale scores measured at each visit. The model included effects of intercept, scale score measured at cycle 2 day 1 visit, treatment arm, and randomization stratification factors.

Amgen

All-cause mortality was collected for all participants enrolled/randomized in the study and is presented according to the randomized treatment arm. Serious AEs and other AEs were collected for all participants in the safety population who received at least one dose of study drug. Participants in the safety population were analyzed according to the treatment arm corresponding to the actual treatment received.

Carfilzomib was administered twice-weekly intravenously as a 10 ± 5 minute infusion using an infusion pump on Days 1, 2, 8, 9, 15, and 16 of each 28-day cycle. The dose was 20 mg/m\^2 on Days 1 and 2 of Cycle 1 and 27 mg/m\^2 beginning on Day 8 of Cycle 1 and thereafter. Participants also received lenalidomide 25 mg daily orally on Days 1 to 21 of each cycle and dexamethasone 40 mg weekly orally or intravenously. Participants were treated for up to 12 28-day cycles.

Twice-weekly KRd 20/27 mg/m^2

Carfilzomib was administered once-weekly intravenously as a 30 ± 5 minute infusion using an infusion pump on Days 1, 8, and 15 of each 28-day cycle. The dose was 20 mg/m\^2 on Day 1 of Cycle 1 and 56 mg/m\^2 beginning on Day 8 of Cycle 1 and thereafter. Participants also received lenalidomide 25 mg daily orally on Days 1 to 21 of each cycle and dexamethasone 40 mg weekly orally or intravenously. Participants were treated for up to 12 28-day cycles.

Once-weekly KRd 20/56 mg/m^2

Total

Age, Continuous

Sex: Female, Male

Ethnicity (NIH/OMB)

Race/Ethnicity, Customized

ISS stage classification at study entry per interactive voice/web response system for randomization. Stage 1: serum beta-2 microglobulin \< 3.5 mg/L and serum albumin ≥ 3.5 g/dL; Stage 2: serum beta-2 microglobulin \< 3.5 mg/L and serum albumin \< 3.5 g/dL or serum beta-2 microglobulin 3.5 - \< 5.5 mg/L irrespective of the serum albumin; Stage 3: serum beta-2 microglobulin ≥ 5.5 mg/L.

Original ISS Stage at Study Entry

Prior Lenalidomide Treatment

Prior Proteasome Inhibitor Treatment

Prior Anti-CD38 Treatment

Study Director

The intent-to-treat (ITT) population included all randomized participants.

Overall Response Rate (ORR) Per International Myeloma Working Group Uniform Response Criteria (IMWG-URC)

The ITT analysis set included all randomized participants.

Kaplan-Meier Estimate of Progression-free Survival (PFS) Rate at 12 Months

The ITT population included all randomized participants.

Percentage of Participants Who Reported Convenience as Measured by the Patient-reported Convenience With Carfilzomib-dosing Schedule Question

The safety population included all randomized participants who received at least 1 dose of any study treatment (carfilzomib, lenalidomide, or dexamethasone). Participants were analyzed according to the treatment arm corresponding to the actual treatment received.

Number of Participants With Treatment-emergent Adverse Events (TEAEs)

The ITT population included all randomized participants. Data is presented for participants in the ITT population who achieved a confirmed response of PR or better.

Time to Response (TTR)

Kaplan-Meier Estimate of Duration of Response (DOR)

Kaplan-Meier Estimate of Time to Progression (TTP)

Kaplan-Meier Estimate of Overall Survival (OS)

Percentage of Participants Who Achieved Minimal Residual Disease Negative Complete Response (MRD[-]CR) by Independent Review Committee (IRC) Per IMWG-URC

Percentage of Participants With Minimal Residual Disease Negativity (MRD[-]) by IRC Per IMWG-URC at 12 Months

Cycle 3 Day 1

Cycle 5 Day 1

Cycle 7 Day 1

Cycle 9 Day 1

Cycle 12 Day 1

Safety Follow-up

The ITT population included all randomized participants. Participants with data available at each time point are presented. All participants in the overall number of participants analyzed contributed to the data in the endpoint.

Change From Baseline in European Organization for Research and Treatment of Cancer Quality-of-Life Questionnaire (EORTC QLQ)-Core 30 (C30) Physical Functioning Scale

Change From Baseline in EORTC QLQ-C30 Role Functioning Scale

Safety follow-up

The ITT population included all randomized participants. Participants with data available at each time point are presented.

Patient-reported Treatment Satisfaction as Measured by the Cancer Therapy Satisfaction Questionnaire (CTSQ)

Twice-weekly Kyprolis (Carfilzomib) + Lenalidomide + Dexamethsone (KRd) 20/27 mg/m^2

Overall Study

Leukemia

Leukemia, Myeloid

Leukemia, Myeloid, Acute

Myeloid Leukemia

Acute Myeloid Leukemia

Acute Non Lymphoblastic Leukemia

Acute Graft Versus Host Disease

TBX-2400

Vivienne Margolis, B.Sc

Yosef Refaeli, Ph.D

Nadira Durakovic, MD, PhD

Simona Sica, MD

Andrea Bacigalupo, MD

This is a study of allogeneic stem cell transplantation with TBX-2400 in adult subjects with Acute Myelogenous Leukemia (AML) or Myelofibrosis (MF).

The donor cells are exposed to a protein that has been shown in the laboratory to improve the ability of the donor cells to make blood and immune cells after transplant. Exposure of the donor cells to this protein does not modify the genes in the cells in any way.

This study has two goals. The first goal is to find out if transplant with TBX-2400 is safe. The second goal is to find out what effects TBX-2400 stem cells have on time to engraftment in adult subjects with AML or MF.

The study hypothesis is that TBX-2400 cells will shorten the time to immune reconstitution after transplant.

Safety and Early Efficacy Study of TBX-2400 in Patients With AML or Myelofibrosis

A Phase I Study to Assess the Safety and Early Efficacy of TBX-2400 in Enhancing Engraftment in Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplant for the Treatment of Acute Myelogenous Leukemia or Myelofibrosis

Adverse events from subject reporting or other assessments

Assessment of transplant engraftment will include absolute neutrophil count, untransfused platelet count and donor chimerism

Immune reconstitution as measured by CD3+ cell count \> 300/μL

CD45 RA versus RO at 3, 6, 9 and 12 months

QoL assessed at 3, 6, 9 and 12 months (World Health Organization \[WHO\] Five Wellbeing Index).

Taiga Biotechnologies, Inc.

Musculoskeletal Diseases

Bone Diseases

Bortezomib

bortezomib (Velcade) 1.6 mg/m² bolus injection on Days 1, 8, 15 and 22 every 5 weeks for 4 cycles

Each cycle will consist of 5 weeks treatment. Subjects in the treatment group will receive: Velcade® 1.6 mg/m2 as an intravenous bolus injection on Days 1, 8, 15, and 22 of each cycle followed by a 13-day rest period (Days 23 to 35) Cycle will be repeated on Day 36. Subjects in the treatment group will receive up to 4 treatment cycles, unless they experience either unacceptable toxicity or if the subject requests to withdraw from the study.

bortezomib

Janssen-Cilag International NV Clinical Trial

The purpose of this study is to assess the effect of bortezomib on myeloma-related bone disease, analyzing bone mineral density (BMD) in patients with Multiple Myeloma (MMY) who have received high dose chemotherapy and autologous stem cell transplantation for primary treatment of MMY (single- or double-transplant). Eligible patients will be randomized (study treatment assigned by chance like flipping a coin) to either bortezomib or observation alone. Patients in the bortezomib arm will receive treatment of bortezomib for a total of 4 cycles. All subjects will be followed for a total of 24 months after randomization.

This is a randomized, open-label (all people involved know the identity of the intervention), multicenter, multi-country parallel group study (each group of patients will be treated at the same time) in patients with Multiple Myeloma (MMY) who have received high dose chemotherapy and autologous stem cell transplantation for primary treatment of MMY (single- or double-transplant). The purpose of the study is to assess the effect of bortezomib on myeloma-related bone disease by analyzing bone mineral density. Approximately 80% of patients with MMY may experience myeloma-related bone disease. Myeloma-related bone disease may cause skeletal complications, including bone pain, bone lesions, abnormal fractures and super-elevated calcium concentrations in the blood. This study is investigating the safety and efficacy of an experimental drug, bortezomib, to consolidate the response to the primary treatment compared to no such consolidation treatment. The analyses in this study will be exploratory in nature, since the study will not address any pre-defined statements but is designed to generate valid hypotheses on safety and efficacy issues. Bortezomib is currently marketed for the treatment of different types of cancer including MMY. Bortezomib has been shown to promote bone formation and increase the number of bone-regenerating cells in preliminary studies. In the present study, the effect of bortezomib on bone formation will be assessed by dual energy x-ray absorptiometry (DXA) measuring bone mineral density, which is a measure for the quality of the bone structures. Several bone markers will be measured on serum samples. Bone markers are indicators of the bone activity. Patients will be evaluated for eligibility within a 14-day screening period, after providing written informed consent. Eligible patients will then be randomised to either bortezomib or observation alone. Baseline efficacy and safety assessments should be available on Cycle 1 Day 1 prior to administration of study medication. Randomization will be stratified by bisphosphonate treatment (use or not) at baseline and age (65 years or more versus less than 65 years). The treatment period is defined as the time the patient is actively receiving study treatment. Patients in the bortezomib arm will receive treatment of bortezomib for a total of 4 cycles or until start of alternative MMY treatment if earlier. Each cycle will consist of 5 weeks treatment. Subjects in the treatment group will receive bortezomib 1.6 mg/m2 on Days 1, 8, 15, and 22 of each cycle followed by a 13-day rest period (Days 23 to 35). Bisphosphonate therapy can be administered as medically indicated and according to local practice. Patients are to attend study center visits on Days 1, 8, 15, and 22 of each treatment cycle or at Days 1, 36, 71, and 106 in the observation arm and at end of treatment (EOT) Visit. During these visits, different evaluations will be conducted to follow treatment safety: symptom directed physical examination, vital signs and laboratory tests. In addition, at Day 1 of Cycle 3 (prior to dosing) or Day 71 in the observation arm, patients will be assessed for disease progression or relapse by measurements of M-protein in serum and urine. If needed, absence of M-protein in serum and urine will be confirmed by immunofixation. Additional evaluations may be performed to document patient's clinical status and ability to tolerate additional therapy as clinically indicated. All patients will be followed for a total of 24 months since baseline. After end of the treatment phase, there will be a long-term follow-up period with visits at 4, 6, 12 and 18 months after the EOT Visit. In case the patient started alternative MMY treatment before completing the 18 months of follow-up, study assessments will stop, except for QoL assessments, which are to be continued up to 18 months of follow-up, and for a long-term follow-up for survival, which will be collected every 6 months by either a telephone call or a visit to the study site. The follow-up for survival will continue for all patients until the last patient has completed follow-up. In addition to scheduled visits, patients may have additional visits as clinically indicated. Every patient who was randomized should enter the long-term follow-up phase. Assessment of progressive disease (PD) or relapse from complete response (CR) should be performed according to the International Myeloma Working Group (IMWG) 2009 criteria. Changes in serum and urine M-protein (monoclonal paraprotein) levels will be confirmed by immunofixation in patients without measurable serum and urine M-protein levels. Karnofsky performance status will also be assessed. Safety will be assessed by monitoring of adverse events, physical examination (including neurological/peripheral neurological examinations), vital signs measurements and clinical laboratory tests. A total of two exploratory analyses will be performed for the present study: The first analysis (main analysis) will be presented after completion of the EOT Visit (24-weeks data). The second analysis will be the analysis at the end of the study on 18 months follow-up data. Patients in the bortezomib arm will receive treatment of bortezomib 1.6 mg/m² as a bolus infusion on Days 1, 8, 15, and 22 every 5 weeks for a total of 4 cycles

Velcade Consolidation Bone Study

A Phase 2, Multicentre, Randomised, Open-Label, Parallel Group Study to Evaluate the Effect of VELCADE on Myeloma Related Bone Disease

Change from baseline in bone mineral density (BMD) will be assessed by dual energy x-ray absorptiometry scans at baseline and the EOT visit

Change from baseline in bone mineral density (BMD) will be assessed by dual energy x-ray absorptiometry scans at baseline and the end of treatment EOT visit

The Progression-Free Survival (PFS) was assessed as median number of months from baseline until the first documented sign of disease progression (increase in disease; radiographic, clinical, or both) or death due to any cause, whichever occurred earlier.

Bone markers (carboxyterminal telopeptide of type I collagen (ICTP), osteocalcin (Oc) and bone-specific alkaline phosphatase (BAP) was measured on serum samples.

Change from Baseline in Biochemical Bone Markers: CTX-I was assessed

Bone markers Dickkopf homolog 1 (DKK-1) was measured on serum samples.

Number of patients with skeletal-related events (i.e. pathological fracture (vertebral, non-vertebral, combined), radiotherapy, spinal cord compression, orthopaedic surgery, hypercalcaemia) occurring over 24 months study period

Appearance of new bone lesions assessed by skeletal survey compared to baseline

T score is used to calculate bone mineral density (calcium and other types of minerals) in an area of the bone. T score is the number of standard deviations above or below the mean for a healthy 30 year old adult of the same sex and ethnicity as a participant. This score is calculated from participant's age, gender and race and skeletal site. T score has a mean of '50' and a standard deviation of '10'. T score lower than its mean indicate low bone mineral density.

The Karnofsky performance status is a way to quantify cancer patients' general well-being and activities of daily life and runs from 100 to 0, where 100 is "perfect" health and 0 is death.

Overall survival defined as time from first treatment of MMY, i.e. day of first dose of induction therapy for MMY to date of death

Subjects were asked to rate their general state of health on a Visual analog scale (in millimeter \[mm\]) ranging from 0 (worst state of health) to 100 (best conceivable state of health) mm.

Tumor response was assessed as VGPR based on IMWG response criteria if, a) serum/urine M protein detectable by immunofixation but not on electrophoresis or; b) greater than or equal to 90% reduction in serum M protein plus urine M protein level less than 100 milligram/24 hour. CR=normal free light chain (FLC) ratio and absence of phenotypically aberrant plasma cells (PC) in bone marrow with a minimum of 3000 total PC analyzed by multiparametric flow cytometry; Complete response (CR) negative immunofixation on the serum and urine and, disappearance of any soft tissue plasmocytomas and \<5% plasma cells in bone marrow.

Tumor response was assessed as SD based on IMWG response criteria as not meeting criteria for CR, VGPR, PR, or progressive disease; PD as Increase of \>=25% from lowest response level in any one or more of the following: serum M protein (absolute increase \>=0.5 g/dl)c or urine M protein (absolute increase \>=200 mg/24 h); or serum/urine M protein unmeasurable: difference between involved and uninvolved free light chain (FLC) levels; absolute increase \>10 mg/dL; or % bone marrow plasma cells: absolute value \>=10% or definite development of new bone lesions or soft tissue plasmocytomas or definite increase in the size of existing bone lesions or soft tissue plasmocytomas; or development of hypercalcemia attributed solely to the plasma cell proliferative disorder.

Tumor response was assessed as PR based on IMWG response criteria as \>=50% reduction of serum and reduction in 24-h urinary M protein by \>=90% or to \<200 mg/24 h; or serum/urine M protein unmeasurable:\>=50% decrease in the difference between involved and uninvolved FLC levels; or serum/urine M protein and FLC assay unmeasurable: \>=50% reduction in plasma cells provided baseline bone marrow plasma cell percentage was \>=30%; or plus if present at baseline: \>=50% reduction in size of soft tissue plasmocytomas.

Janssen-Cilag International NV

Non-treated Control

EMEA Medical Affairs Director

ITT analysis:13 patients in the bortezomib group and 14 patients in the non-treated control group did not have values at the 2 timepoints to allow calculation of the parameter

Change From Baseline in Bone Mineral Density (BMD) in the Spine at End of Treatment (EOT)

Femur neck

Femur total

ITT analysis:8 patients in the bortezomib group and 8 patients in the non-treated control group did not have values at the 2 timepoints to allow calculation of the parameter

Change From Baseline in Bone Mineral Density (BMD) in the Femur at End of Treatment

Full Analysis Set (FAS) included all the randomized subjects who were eligible for efficacy analysis, that is, excluding subjects who did not have baseline and/or post-baseline data.

Progression Free Survival

ICTP (n= 44, 42)

Osteocalcin (44, 42)

BAP (42, 40)

FAS was used for analysis. "N" (number of subjects analyzed) signifies the subjects evaluable this measure and n=number pf participants analysed for this outcome measure at specific time point. Missing data was imputed by last observation carried forward (LOCF) method.

Change From Baseline in Biochemical Bone Markers:Carboxyterminal Telopeptide of Type I Collagen (ICTP), Osteocalcin, Bone-specific Alkaline Phosphatase (BAP)

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Study of Stem Cell Transplant vs. Non-Transplant Therapies in High-Risk Myelofibrosis	NCT04217356	Myelofibrosis High-Risk Cance... Bone Marrow Can...	Hematopoietic s... Ruxolitinib Hydroxyurea	18 Years - 70 Years	University Health Network, Toronto
A Study Comparing Once-weekly vs Twice-weekly Carfilzomib in Combination With Lenalidomide and Dexamethasone in Subjects With Relapsed or Refractory Multiple Myeloma	NCT03859427	Relapsed or Ref...	Carfilzomib Carfilzomib Lenalidomide Dexamethasone	18 Years - 99 Years	Amgen
Safety and Early Efficacy Study of TBX-2400 in Patients With AML or Myelofibrosis	NCT04709458	Myelofibrosis Acute Myelogeno...	TBX-2400	18 Years -	Taiga Biotechnologies, Inc.
Velcade Consolidation Bone Study	NCT01286077	Multiple Myelom...	bortezomib	18 Years -	Janssen-Cilag International NV
A Phase II Study of Belinostat in Combination With Bortezomib in Patients With Relapsed, Refractory Multiple Myeloma	NCT00431340	Multiple Myelom...	PXD101	18 Years -	Valerio Therapeutics
A Study Comparing Once-weekly vs Twice-weekly Carfilzomib in Combination With Lenalidomide and Dexamethasone in Subjects With Relapsed or Refractory Multiple Myeloma	NCT03859427	Relapsed or Ref...	Carfilzomib Carfilzomib Lenalidomide Dexamethasone	18 Years - 99 Years	Amgen
A Phase II Study of Belinostat in Combination With Bortezomib in Patients With Relapsed, Refractory Multiple Myeloma	NCT00431340	Multiple Myelom...	PXD101	18 Years -	Valerio Therapeutics
The Effect of Relaxing Breathing Exercise Applied to Patients Before Bone Marrow Biopsy on Vital Signs, Pain and Anxiety Levels	NCT05632874	Bone Marrow Can...	relaxing breath...	18 Years - 65 Years	Uskudar University
Improving Patient Experience: BMBA	NCT05690230	Bone Marrow Can... Leukemia Lymphoma Multiple Myelom... Hematologic Can...	Virtual reality Environmental c...	18 Years -	Thomas Jefferson University
Safety and Early Efficacy Study of TBX-2400 in Patients With AML or Myelofibrosis	NCT04709458	Myelofibrosis Acute Myelogeno...	TBX-2400	18 Years -	Taiga Biotechnologies, Inc.
Treatment With Velcade (Bortezomib) Plus Dexamethasone (VD) or VD Plus Cyclophosphamide or VD Plus Lenalidomide in Patients With Multiple Myeloma Stabilized After 4 Cycles of VD	NCT00908232	Multiple Myelom...	Cyclophosphamid... Bortezomib Dexamethasone Lenalidomide	18 Years -	Janssen-Cilag International NV

Spots Global Cancer Trial Database for bone marrow cancer