Symptoms and General Pathology

All Conditions

Neoplasms

Blood and Lymph Conditions

Rare Diseases

Syndrome

Preleukemia

Myelodysplastic Syndromes

Bone Marrow Diseases

Hematologic Diseases

Precancerous Conditions

Hematinics

All Drugs and Chemicals

Darbepoetin alfa

Participants received darbepoetin alfa 500 µg every three weeks (Q3W) for 24 weeks in the double-blind treatment period, and continued to receive darbepoetin alfa 500 µg Q3W during the active treatment period for an additional 48 weeks.

Participants received placebo subcutaneous injection every 3 weeks (Q3W) for 24 weeks during the double-blind treatment period. From week 25 participants received darbepoetin alfa 500 µg Q3W during the active treatment period for 48 weeks.

Administered by subcutaneous injection every 3 weeks

Placebo

The primary objective was to assess the superiority of darbepoetin alfa versus placebo on the incidence of red blood cell transfusions during the 24-week double-blind treatment period in anemic patients with low or intermediate-1 risk MDS.

This study consists of a 3-week screening period, a 24-week double-blind treatment period, and a 48-week active treatment period and the long-term follow-up period.

An end of treatment period (EOTP) visit occurs at week 25, or 3 weeks after last dose of investigational product (IP) for participants who withdraw from the study. After entering the active treatment period, an end of active treatment period (EOATP) visit occurs at week 72 / 73, or 3 weeks after the last dose of darbepoetin alfa.

Long-term follow-up (LTFU) will occur every 26 weeks (± 4 weeks) from the EOATP visit (or EOTP visit if the participant does not enter the active treatment period) and will continue for a minimum of 3 years from the first dose of IP. Follow-up may occur through clinic visit or telephone contacts. Information on the participant's survival and progression to AML status will be collected during LTFU.

Darbepoetin Alfa in Patients With Anemic Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)

A Multicenter, Randomised, Double-blind, Placebo-controlled Study of Darbepoetin Alfa for the Treatment of Anaemic Subjects With Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)

International Working Group 2006 erythroid response was defined as achieving an initial ≥ 1.5 g/dL increase in hemoglobin from baseline and sustaining an average rise of ≥ 1.5 g/dL in a rolling 56-consecutive day period in the absence of RBC transfusion.

Participants with no hemoglobin collected to the minimum time required to observe an IWG erythroid response (Week 13) were considered non-responders.

The severity of each adverse event was graded using the the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 grading scale, where grade 1 = mild, grade 2 = moderate, grade 3 = severe, grade 4 = life-threatening and grade 5 = death.

Prespecified adverse events of interest for darbepoetin alfa, based on clinical data in anemic patients with cancer, included the following categories: hypersensitivity, cardiac failure, hypertension, malignancies, embolic and thrombolic events, venous thromboembolic events (VTEs), central nervous system vascular disorders, and ischemic heart disease.

Transformation to AML was assessed according to WHO guidelines in the absence of IP and any haematopoietic growth factors (2 weeks off dosing). Bone marrow and/or cytogenetic report confirmation of AML was required (marrow or peripheral blast cells ≥ 20%, presence of pathognomic AML cytogenetic change, or evidence of marrow blast criteria for erythroleukemia). A pathology report confirming other leukemias such as chloroma (granulocytic sarcoma, myeloid sarcoma) or leukemia cutis also constituted transformation to AML.

Two validated assays were used to detect the presence of anti-darbepoetin alfa antibodies.

Samples were first tested in an immunoassay to detect antibodies capable of binding to darbepoetin alfa. Samples confirmed to be positive for binding antibodies were subsequently tested in a cell-based assay to determine neutralizing activity against darbepoetin alfa. If a sample was positive for binding antibodies and demonstrated neutralizing activity at the same time point, the sample was defined as positive for neutralizing antibodies.

The number of participants who developed antibodies to darbepoetin alfa is defined as participants who were neutralizing antibody positive post-baseline with a negative or no result at baseline.

The FACIT-Fatigue scale was a 13-item self-administered questionnaire that assesses both the physical and functional consequences of fatigue. Each question is answered on a 5-point scale, where 0 means "not at all," and 4 means "very much." The FACIT-Fatigue scale score ranges from 0 to 52, with higher scores denoting lower levels of fatigue.

A positive change from baseline score indicates an improvement. End of treatment period (EOTP) analysis includes last available values.

The EQ-5D visual analog scale (VAS) is a global evaluation of overall health state with scores ranging from 0 (worse health state a participant can imagine) to 100 (best health state a participant can imagine).

End of treatment period (EOTP) analysis includes last available values.

The FACIT-Fatigue scale was a 13-item self-administered questionnaire that assesses both the physical and functional consequences of fatigue. Each question is answered on a 5-point scale, where 0 means "not at all," and 4 means "very much." The FACIT-Fatigue scale score ranges from 0 to 52, with higher scores denoting lower levels of fatigue.

Clinically meaningful improvement in fatigue is defined as an increase of ≥ 3 points in the FACIT-Fatigue subscale score, from baseline to EOTP.

Amgen

One participant randomized to the Placebo arm inadvertently received one 200 µg dose of darbepoetin alfa and is reported in the Darbepoetin alfa group for adverse events.

Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.

Participants received placebo subcutaneous injection every 3 weeks (Q3W) for 24 weeks.

Participants received darbepoetin alfa 500 µg Q3W for 24 weeks.

Darbepoetin Alfa

Total

Age, Continuous

Sex: Female, Male

French clinical sites, per regulation, did not complete the ethnicity question.

Ethnicity (NIH/OMB)

White

Race/Ethnicity, Customized

Intermediate-1

The Myelodysplastic Syndrome (MDS) IPSS score assesses the severity of MDS based on 3 prognostic factors each assigned a score: the percentage of bone marrow blasts, chromosome changes in the marrow cells (karyotype) and the presence of one or more low blood cell counts (cytopenias). The IPSS score is the sum of the bone marrow blast + karyotype + cytopenia score and ranges from 0 (low risk) to 3.5 (high risk). Prognosis is categorized as Low risk (score = 0), Intermediate-1 (score 0.5 to 1.0), Intermediate-2 (score 1.5 to 2.0) or High risk (score ≥ 2.5).

IPSS Risk Category

Refractory anemia (RA)

RA with ringed sideroblasts (RARS)

Refractory cytopenia multilineage dysplasia (RCMD)

MDS, unclassified (MDS-U)

MDS associated with isolated del(5q)

Refractory anemia with excess blasts-1 (RAEB-1)

Refractory anemia with excess blasts-2 (RAEB-2)

Unknown

The World Health Organization (WHO) classification recognizes eight subtypes of MDS that are distinguished by the percentage of myeloblasts, presence or absence of ringed sideroblasts (i.e., erythroid precursors with iron deposits surrounding the nucleus), presence of a monocytosis or a deletion 5q.

World Health Organization (WHO) Classification of MDS

Data available for 43 and 88 participants in each treatment group respectively

Time since MDS Diagnosis

Data available for 35 and 75 participants in each treatment group respectively

Hemoglobin

Primary Analysis Set includes all randomized and consented participants who received at least 1 dose of investigational product.

Study Director

The primary hypothesis to be tested was that the percentage of participants with at least

1 RBC transfusion from week 5 to the EOTP was lower in the darbepoetin alfa group than in the placebo group. This hypothesis was confirmed if the incidence of RBC transfusion in the darbepoetin alfa group was lower and had a p-value \< 0.05 from a 2-sided Chi-square test.

Transfusion Primary Analysis Set which includes all randomized and consented participants who received at least 1 dose of study drug and who had an end of treatment period (EOTP) visit ≥ day 29 (ie, start of week 5).

Percentage of Participants With at Least One Red Blood Cell (RBC) Transfusion During the Double-blind Treatment Period

If the primary hypothesis was confirmed, the secondary hypothesis to be tested was that the percentage of participants achieving an IWG erythroid response during the 24-week double-blind treatment period was greater in the darbepoetin alfa group than in the placebo group. This hypothesis was confirmed if erythroid response was higher in the darbepoetin alfa group and the p-value was \< 0.05 from a 2-sided Cochran-Mantel-Haenszel test using the IPSS as a stratification factor.

Primary analysis set participants with a central laboratory baseline hemoglobin value

Percentage of Participants Who Achieved an Erythroid Response Based on International Working Group (IWG) 2006 Criteria in the Double-blind Treatment Period

Any adverse event (AE)

AE Grade ≥ 2

AE Grade ≥ 3

AE Grade ≥ 4

Serious adverse events (SAE)

AE leading to discontinuation of study drug

Fatal adverse events

Adverse events of special interest

Treatment-related adverse events (TRAE)

Treatment-related serious adverse events

TRAE leading to discontinuation of study drug

Treatment-related fatal adverse events

Safety Analysis Set, including all participants who received at least 1 dose of study drug. One participant in the placebo arm inadvertently received a dose of darbepoetin alfa and is counted in the darbepoetin alfa group for safety analyses.

Number of Participants With Adverse Events

Number of Participants With Disease Progression to Acute Myeloid Leukemia (AML)

Number of Participants With Malignancies Other Than AML, Basal Cell Carcinoma, or Squamous Cell Carcinoma of the Skin

Safety analysis set participants with post-baseline antibody results

Number of Participants Who Developed Neutralizing Antibodies to Darbepoetin Alfa

Baseline (N = 42, 90)

Change from Baseline to Week 13 (n = 41, 85)

Change from Baseline to Week 25 (n = 39, 85)

Change from Baseline to EOTP (n = 42, 90)

FACIT-Fatigue Analysis Set, includes all participants in the primary analysis set who completed or partially completed both the baseline and at least 1 subsequent FACIT-F questionnaire.

Change From Baseline in Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F)

Change from Baseline to Week 13 (n = 41, 83)

Change from Baseline to Week 25 (n = 39, 81)

Change from Baseline to EOTP (n = 42, 89)

The EQ-5D visual analog analysis set includes all participants in the primary analysis set who completed both the baseline and at least 1 subsequent visual analog scale.

Change From Baseline in EuroQol-5D (EQ-5D) Visual Analog Scale (VAS)

Percentage of Participants With a Clinically Meaningful Improvement in Fatigue

Overall Study

Enzyme Inhibitors

Dose Schedule 1: Two weeks treatment with Telintra 3000 mg per day in two divided doses followed by one week with no treatment per three week cycle.

Dose Schedule 2: Three weeks treatment with Telintra 2000 mg per day in two divided doses followed by one week with no treatment per four week cycle.

Dose Schedule 1-3000 mg orally per day in two divided doses for two weeks followed by 1 week with no treatment per three week cycle.

Dose Schedule 2-2000 mg orally per day in two divided doses for three weeks followed by 1 week with no treatment per four week cycle.

Ezatiostat Hydrochloride

Gail Brown, MD

This is a randomized, open label, multicenter, Phase 2 study comparing two dose schedules of Telintra in patients with Low or Intermediate-1 risk MDS. Patients at least 18 years of age presenting with histologically confirmed Low to Intermediate-1 risk MDS with documented significant cytopenia for at least two months by the IWG criteria are eligible.

Phase 2 Study Comparing Two Dose Schedules of Telintra™ in Myelodysplastic Syndrome (MDS)

Phase 2 Randomized Study Comparing Two Dose Schedules of Ezatiostat Hydrochloride (Telintra™, TLK199 Tablets) in Low to Intermediate-1 Risk Myelodysplastic Syndrome (MDS)

Telik

Three weeks of treatment with ezatiostat at 2000 mg per day in divided doses followed by a one week rest period in four-week treatment cycles.

ezatiostat hydrochloride (Telintra®)

Gail L Brown, MD

This is a multicenter, single arm open label Phase 2b Study of oral ezatiostat (Telintra®) in Patients who are RBC tranfusion dependent, Low to INT-1 IPSS risk, non-del (5q) Myelodysplastic Syndrome (MDS).

Study of (Telintra®) in Non-Del(5q) Myelodysplastic Syndrome

Phase 2b Study of Oral Ezatiostat Hydrochloride (Telintra®) in Patients With Low to Intermediate-1 Risk, Non-Deletion 5q Myelodysplastic Syndrome

Hematologic Improvement response will be assessed per the IWG MDS response criteria (2006)

Recording and grading of AEs using NCI-CTCAE v4.03

Immune System Diseases

Lymphoma

Lymphosarcoma

Amino Acids

JNJ-64619178

Arginine

Participants with solid tumors or non-Hodgkin lymphoma (NHL) will receive JNJ-64619178 orally as per the assigned sequential cohorts and doses will be escalated based on the review of all available data including, but not limited to, pharmacokinetic, pharmacodynamic, safety, and clinical activity. One or more recommended Phase 2 dose(s) (RP2Ds) may be determined for further exploration.

Participants with myelodysplastic syndromes (MDS) will receive JNJ-64619178 at a dose less than or equal to the RP2D selected in Part 1 for 24 weeks, or longer if there is evidence of clinical benefit. The dose level of JNJ-64619178 may be adjusted based on observed toxicities.

JNJ-64619178 capsules to be administered orally.

Janssen Research & Development, LLC Clinical Trial

The purpose of the study is to identify the maximum tolerated dose (MTD) of JNJ-64619178 in participants with relapsed/refractory B cell non-Hodgkin lymphoma (NHL) or advanced solid tumors and also to identify the recommended Phase 2 dose(s) (RP2Ds) of JNJ-64619178 for NHL and advanced solid tumors (Part 1) and to confirm the tolerability of JNJ-64619178 in participants with lower risk myelodysplastic syndromes (MDS) (Part 2).

The study is designed to determine the maximum tolerated dose (MTD) of JNJ-64619178, and to select a dose(s) and regimen(s) that may be used in future clinical development. Study evaluations will include safety, pharmacokinetics, biomarkers and efficacy evaluations (Disease Assessments). Adverse events will be evaluated throughout the study. The study is divided into 4 periods: a screening phase, a pharmacokinetic run-in phase, a treatment phase, and a post treatment follow-up phase. An end-of-treatment visit will be completed less than or equal (\<=) 30 days (+7 days) after the last dose of study drug or prior to the start of a new anticancer therapy, whichever comes first.

A Study of JNJ-64619178, an Inhibitor of PRMT5 in Participants With Advanced Solid Tumors, NHL, and Lower Risk MDS

A Phase 1, First-in-Human, Open-Label Study of the Safety, Pharmacokinetics, and Pharmacodynamics of JNJ-64619178, an Inhibitor of Protein Arginine Methyltransferase 5 (PRMT5) in Subjects With Advanced Cancers

DLTs are defined as certain non-hematologic and hematologic toxicities of Grade 3 or higher.

AE is any untoward medical occurrence in participant who received study drug without regard to possibility of causal relationship.

Severity will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE). Severity scale ranges from Grade 1 (Mild) to Grade 4 (Life-threatening). Grade 1= Mild, Grade 2= Moderate, Grade 3= Severe, Grade 4= Life-threatening and Grade 5= Death related to adverse event.

Number of participants with abnormality in vital signs (temperature, pulse/heart rate, and blood pressure) will be reported.

Number of participants with laboratory abnormalities (serum chemistry, hematology, and coagulation, and disease-related laboratory abnormalities) will be reported.

Number of participants with electrocardiogram(ECG) abnormalities will be reported.

Cmax is the maximum observed plasma concentration.

AUCtau is the measure of the plasma drug concentration from time zero to end of dosing interval.

Cmin is the minimum observed plasma concentration.

Plasma decay half-life is the time measured for the plasma concentration to decrease by one half.

Volume of distribution is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired blood concentration of a drug. Steady state volume of distribution (Vss) is the apparent volume of distribution at steady state which is estimated by (D/AUC\[0-infinity\])\* (AUMC\[0-infinity\])/(AUC\[0-infinity\]) where D is the dose of study drug, AUMC(0-infinity) is the area under the first moment curve extrapolated to infinity and AUC (0-infinity) is the area under the plasma concentration-time curve from time zero to infinite time. All these parameters will be assessed as influenced by the fraction absorbed.

Drug clearance is a quantitative measure of the rate at which a drug substance is removed from the blood.

Accumulation Index (RA) is calculated as AUC (0-24) value at steady state divided by AUC (0-24) value after first dose.

Plasma concentration of symmetric dimethyl-arginine (SDMA) will be evaluated.

Overall response rate (ORR) is defined as the percentage of participants who have a PR or better. Per Lugano classification, PR is defined as greater than or equal (\>=) 50 percent (%) decrease in size of target lesions.

Overall response rate (ORR) is defined as the percentage of participants who have a PR or better. PR criteria in solid tumors (RECIST) is \>= 30 percent (%) decrease in the sum of the diameters of all index lesions compared with baseline in 2 observations at least 4 weeks apart, in absence of new lesions or unequivocal progression of non-index lesions.

Duration of response (DOR) will be calculated from the date of initial documentation of a response (PR or better) to the date of first documented evidence of progressive disease (PD), as defined in the disease-specific response criteria, or death due to any cause, whichever occurs first. PD (per RECIST 1.1) defined as at least 20% increase in the sum of the longest diameters of index lesions or unequivocal progression of non-index lesions. Per Lugano classification, PD: target lesions larger; clear progression of non-target lesions; or new tumor lesions; new or recurrent bone marrow involvement; splenomegaly + 2 centimeter (cm) or +50%.

Clinical benefit rate is defined as percentage of participants who have a complete response (CR), PR and sustained stable disease of 12 weeks or more. Sustained stable disease of 12 weeks or more is defined as participants with stable disease assessment maintained for about 12 weeks or longer from baseline. Per Lugano classification CR is defined as no detectable disease by computed tomography (CT) or positron emission tomography (PET)/CT scan. Evaluation criteria as per RECIST 1.1 for CR is defined as disappearance of all lesions in 2 consecutive observations not less than 4 weeks apart.

Percentage of participants with TI, defined as being without any transfusion during any consecutive 8 weeks (56 days) between the first dose of study drug and treatment discontinuation.

Overall improvement rate is defined as the percentage of participants achieving complete remission (bone marrow: less than or equal to (\<=)5% myeloblasts with normal maturation of all cell lines; Peripheral blood: hemoglobin \>=11 gram per deciliter (g/dL); platelets \>=100\*109/liter(L); neutrophils \>=1.0\*109/L; blasts, 0%), partial remission (All complete remission criteria if abnormal before treatment except: Bone marrow blasts decreased by \>=50% over pretreatment but still \>5% Cellularity and morphology not relevant), or hematologic improvement (HI) (Erythroid response \[pretreatment, \<11 g/dL\]; Platelet response (pretreatment, \<100\*10\^9/L); Neutrophil response (pretreatment, \<1\*10\^9/L); Progression or relapse after HI) according to modified International Working Group (IWG) criteria.

Janssen Research & Development, LLC

Inflammation

Anemia

200 mg canakinumab subcutaneously every three weeks

Administration for a duration of 6 months for all patients and in case of response further treatment for up to three years

Canakinumab Injection

Anne Sophie Kubasch, Dr.

Hematologic improvement of erythrocytes after 6 months of canakinumab treatment.

To study the erythroid response rate (HI-E) of canakinumab in patients with IPSS-R very low, low, or intermediate risk MDS or MDS/MPN after 6 months of treatment.

Efficacy and Safety of Canakinumab for the Treatment of Anemia in LR-MDS Patients

A Phase II, Single-Arm, Open-Label Study to Assess the Efficacy and Safety of Canakinumab for the Treatment of Anemia in Patients With IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes or MDS/MPN

Erythroid response rate (HI-E) of canakinumab

Duration of erythroid response rate will be measured up to loss of response or reaching end of study (after max. 3 years of treatment)

The safety profile of canakinumab will be described by collecting AE (adverse event) and SAE (serious adverse event) information up to the start of new treatment or reaching end of study (after max. 3 years of treatment). Special consideration will be laid on events that lead to treatment discontinuation.

Proportion of disease progression (after reaching PD at any time during the trial after primary end-point visit)

QoL assessment using the QUALMS questionnaire up to end of treatment:

38-item assessment tool for patients with Myelodysplastic Syndromes (MDS) QUALMS scores ranged from 24 to 99, with higher scores for better outcome

QoL assessment using the EORTC-C30 questionnaire up to end of treatment:

To assess patient-reported quality of life during canakinumab treatment: 30 questions assessing the quality of life of oncology patients across 10 subscales will be analyzed. All subscales have a score range from 0 to 100 points.

Function subscales: a higher score represents a higher quality of life. Symptoms subscales: higher score represents higher level of symptoms/problems, i.e., represents lower quality of life

Novartis Pharmaceuticals

University of Leipzig

Coordinating Investigator

Thrombocytopenia

Blood Platelet Disorders

Cytopenia

Weekly subcutaneous dosing based on platelet count for 26 weeks during the Test Treatment Period and for 24 weeks during the Extended Treatment Period, separated by a 4-week interim washout period. Starting dose is at 750 μg, up to a maximum dose of 1000 μg, or reduced to a minimum of 250 μg.

Weekly subcutaneous dosing with blinded matching placebo dose level for 26 weeks during the Test Treatment Period and for 24 weeks during the Extended Treatment Period, separated by a 4-week interim washout period.

Placebo is supplied in a 5 mL single use glass vial as a sterile, white, preservative-free, lyophilized powder.

Romiplostim is supplied in a 5 mL single use glass vial as a sterile, white, preservative-free, lyophilized powder.

Romiplostim

The Data Monitoring Committee (DMC) for study 20060198 recommended that all subjects discontinue treatment of study drug and continue to be followed for long term follow-up. Amgen adopted the DMC recommendation.

This is a Phase 2, multicenter, randomized, double blind, placebo controlled study designed to assess the efficacy and safety of romiplostim (formerly, AMG 531) treatment in thrombocytopenic MDS patients. The study is composed of a 26-week placebo controlled test treatment period (romiplostim versus Placebo), a 4 week interim wash-out period, a 24-week placebo controlled extended treatment period, and a 4-week follow-up period followed by an End of Study (EOS) visit. During the interim wash-out period, a bone marrow biopsy will be performed in the absence of growth factor to assess changes in the marrow. In the extended treatment period, safety assessments will continue and participants will be allowed to receive any standard of care treatments for MDS. Patients will be followed for survival for an additional 60 months following the End of Study (EOS) visit.

Romiplostim Treatment of Thrombocytopenia in Subjects With Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)

A Randomized, Double Blind, Placebo Controlled Study Evaluating the Efficacy and Safety of Romiplostim Treatment of Thrombocytopenia in Subjects With Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)

A clinically significant bleeding event is defined as any bleeding event of grade ≥ 2 per the modified World Health Organization (WHO) bleeding scale: • Grade 0 = no bleeding • Grade 1 = petechia or mucosal or retinal bleeding not requiring intervention • Grade 2 = melena, hematemesis, hematuria, hemoptysis • Grade 3 = bleeding required red cell transfusion • Grade 4 = retinal bleeding with visual impairment • Grade 5 = non-fatal cerebral bleeding • Grade 6 = fatal cerebral bleeding • Grade 7 = fatal non-cerebral bleeding. Bleeding events that continue for more than 7 days were counted as separate events every eighth day. Multiple events that arose from one organ system on one day were collapsed into one single event. Bleeding events with a start date between the first dose date and the last dose date of the test treatment period+7 days are included.

A discrete platelet transfusion is any number of platelet transfusion administered within a 3-day period. Transfusions administered more than 3 days apart are counted as separate events. Transfusion given in the absence of any bleeding, when platelet count is \>10x10\^9/L, is not counted as a platelet transfusion event. Events with start date between the first dose date and the last dose date of the test treatment period +7 days are included. Exposure adjusted event rate per 100 patient-years = (events / patient-years \* 100). Patient Year = total patient years of exposure to investigational product during 26 weeks test treatment period.

The time from first dose of study drug to the last dose of 26-week test treatment period. A bleeding event is defined as any bleeding event reported during the test treatment period. Bleeding events that continue for more than 7 days are counted as separate events every eighth day. Multiple events that arose from one organ system on one day are collapsed into one single event. Exposure adjusted event rate per 100 patient-years = events / patient-year \* 100).

The time from first dose of study drug to the last dose of 26-week test treatment period. A unit of platelets is defined as a single pack of pooled platelet-rich plasma comprised of 6 to 8 individual platelet concentrate packs (200 to 400 mL), a single pack of pooled buffy-coat concentrate, or 1 apheresis (single donor) concentrate. Exposure adjusted event rate per 100 patient-years = events / patient-years \* 100.

Platelet Hematologic Improvemen defined by the international working group (IWG) as: an absolute increase in platelet count of ≥ 30 x 10\^9/L for a patient starting with a platelet count of ≥ 20 x 10\^9/L or an increase in platelet count from \< 20 x 10\^9/L to ≥ 20 x 10\^9/L and by at least 100% in a patient that started with a platelet count \< 20 x 10\^9/L. To account for any possible contribution from platelet transfusions, platelet counts within 3 days following administration of platelet transfusion is not counted towards the platelet hematologic improvement endpoint. If no platelet measurements are available on the weekly scheduled dose day, then that week is not counted towards the platelet hematologic improvement endpoint.

Duration for participants who did not report HI-P during the period is 0. A platelet hematologic improvement (HI-P) is defined by an MDS International Working criteria as patients with a baseline platelet count of ≥ 20 x 10\^9/L achieving an absolute increase of ≥ 30 x 10\^9/L or increasing the platelet count to above 20 x 10\^9/L and by at least 100% in patients with a baseline of \< 20 x 10\^9/L for at least 8 consecutive weeks. To account for any possible contribution from platelet transfusions, platelet counts within 3 days following administration of platelet transfusion is not counted towards the platelet hematologic improvement endpoint. If no platelet measurements are available on the weekly scheduled dose day, then that week is not counted towards the platelet hematologic improvement endpoint. The durations of HI-P are cumulative if more than one incidence occurred. Exposure adjusted event rate per 100 patient-weeks = total number of weeks / patient-weeks \* 100.

Overall survival was calculated using Kaplan-Meier methods. For patients who discontinued early, additional information from the long term follow-up are added (closest available follow-up information up to 58 weeks).

Overall survival was calculated using Kaplan-Meier methods.

The number of bleeding events was obtained from the thrombocytopenia symptoms (Th-symptoms) survey. Patients reported spontaneous bleeding to have occurred 0, 1 or 2, 3 or 4, 5 or 6, or 7 or more times in the past week. The lower threshold of bleeding counts is used for conservative purposes (i.e., the "3" is used for the response option of "3 or 4 times"). Exposure adjusted event rate per 100 patient-years = number of events / patient-year \* 100.

The safety analysis set consisted of 250 patients including 168 in the romiplostim group and 82 in the placebo group. One patient enrolled in the placebo group received 1 dose of romiplostim at week 4 of the extended treatment period. Results for this patient were analyzed as part of the romiplostim group in the Safety Analysis Set.

White or Caucasian

Black or African American

Hispanic or Latino

Asian

Other

RARS

RAEB-1

RAEB-2

RCMD

RCMD-RS

RA: Refractory Anemia RAEB-1: Refractory Anemia with Excess Blasts-1 RAEB-2: Refractory Anemia with Excess Blasts-2 RARS: Refractory Anemia with Ringed Sideroblasts RCMD: Refractory cytopenia with multilineage dysplasia RCMD-RS: Refractory cytopenia with multilineage dysplasia and ringed sideroblasts MDS-U: Myelodysplastic syndrome - unclassified MDS associated with isolated del(5q).

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Darbepoetin Alfa in Patients With Anemic Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)	NCT01362140	MDS	Darbepoetin alf... Placebo	18 Years -	Amgen
Phase 2 Study Comparing Two Dose Schedules of Telintra™ in Myelodysplastic Syndrome (MDS)	NCT00700206	Myelodysplastic...	Ezatiostat Hydr...	18 Years -	Telik
Study of (Telintra®) in Non-Del(5q) Myelodysplastic Syndrome	NCT01459159	Myelodysplastic...	ezatiostat hydr...	18 Years -	Telik
A Study of JNJ-64619178, an Inhibitor of PRMT5 in Participants With Advanced Solid Tumors, NHL, and Lower Risk MDS	NCT03573310	Neoplasms Solid Tumor, Ad... Non-Hodgkin Lym... Myelodysplastic...	JNJ-64619178	18 Years -	Janssen Research & Development, LLC
Darbepoetin Alfa in Patients With Anemic Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)	NCT01362140	MDS	Darbepoetin alf... Placebo	18 Years -	Amgen
Efficacy and Safety of Canakinumab for the Treatment of Anemia in LR-MDS Patients	NCT05237713	Anemia Myelodysplastic...	Canakinumab Inj...	18 Years -	University of Leipzig
Romiplostim Treatment of Thrombocytopenia in Subjects With Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)	NCT00614523	MDS Myelodysplastic... Thrombocytopeni...	Placebo Romiplostim	18 Years - 90 Years	Amgen
Allogeneic Hematopoietic Stem Cell Transplantation in Patients With Myelodysplastic Syndrome Low Risk	NCT02757989	MDS	transplantation	18 Years - 69 Years	Groupe Francophone des Myelodysplasies
Darbepoetin Alfa MDS Companion Protocol	NCT02175277	Myelodysplastic...	Darbepoetin Alf...	18 Years -	Amgen
Allogeneic Hematopoietic Stem Cell Transplantation in Patients With Myelodysplastic Syndrome Low Risk	NCT02757989	MDS	transplantation	18 Years - 69 Years	Groupe Francophone des Myelodysplasies
Phase 2 Study Comparing Two Dose Schedules of Telintra™ in Myelodysplastic Syndrome (MDS)	NCT00700206	Myelodysplastic...	Ezatiostat Hydr...	18 Years -	Telik
Darbepoetin Alfa in Patients With Anemic Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)	NCT01362140	MDS	Darbepoetin alf... Placebo	18 Years -	Amgen
Allogeneic Hematopoietic Stem Cell Transplantation in Patients With Myelodysplastic Syndrome Low Risk	NCT02757989	MDS	transplantation	18 Years - 69 Years	Groupe Francophone des Myelodysplasies

Spots Global Cancer Trial Database for low risk mds

Every month we try and update this database with for low risk mds cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.