Blood and Lymph Conditions

All Conditions

Symptoms and General Pathology

Neoplasms

Rare Diseases

Thrombocytopenia

Syndrome

Leukemia

Leukemia, Myeloid

Leukemia, Myeloid, Acute

Preleukemia

Myelodysplastic Syndromes

Neoplasms by Histologic Type

Hematologic Diseases

Bone Marrow Diseases

Precancerous Conditions

Blood Platelet Disorders

Cytopenia

Myeloid Leukemia

Acute Myeloid Leukemia

Acute Non Lymphoblastic Leukemia

Acute Graft Versus Host Disease

Antineoplastic Agents

All Drugs and Chemicals

Azacitidine

Antimetabolites

Enzyme Inhibitors

Vidaza (azacitidine) Revolade (eltrombopag)

Standard: azacitidine D1-5, 8\&9, until loss of clinical benefit. Experimental: eltrombopag at a dose ranging from 50-300mg for 6 months, continuing only in those deriving clinical benefit.

Azacitidine and eltrombopag

Michael Dickinson, Dr

Myelodysplastic Syndrome (MDS) is a disease of the bone marrow characterized by anemia,neutropenia, and thrombocytopenia (low red blood cell, white blood cell, and platelet counts). MDS patients with thrombocytopenia who fail standard therapies require regular platelet transfusions which are expensive and inconvenient, and are a risk for further serious bleeding complications. The new treatment of MDS using azacitidine has shown to increase the survival rate of MDS patients including to improve platelet production over time. However,in the early cycles of treatment with azacitidine,the low platelet counts tend to exacerbate before they provide any clinical benefit.

Eltrombopag is a drug designed to activate the thrombopoietin receptor. Eltrombopag has been able to increase platelet counts in healthy Thrombocytopenia Purpura (ITP), a disease where patients destroy their own platelets very rapidly and thus develop thrombocytopenia.

Eltrombopag is administered orally and is Therapeutic Goods Administration (TGA) approved for the treatment of thrombocytopenia in patients with chronic ITP who failed to respond to standard treatment.

This study is a single arm pilot study to evaluate the safety and tolerability of Eltrombopag in the treatment of low platelet counts in adult subjects with MDS treated using azacitidine This study also incorporates a correlative laboratory component designed to determined the mechanism of action of 5-azacitidine +/- Eltrombopag and to determine a baseline profile which may predict those most responsive. These studies will incorporate gene methylation and expression, and immunoprofiling.

A Single Arm Pilot Study of Azacitidine in Myelodysplastic Syndromes (MDS) / Acute Myeloid Leukaemia (AML), With Eltrombopag Support for Thrombocytopenia

A Single Arm Pilot Study of Azacitidine in Myelodysplastic Syndrome / Acute Myeloid Leukaemia, With Eltrombopag Support for Thrombocytopenia.

The number of events of Grade III/IV non-haematological toxicities will be measured based on the possibly, probably or definitely relatedness to the combination of eltrombopag and azacitidine

GlaxoSmithKline

Celgene Corporation

Peter MacCallum Cancer Centre, Australia

Anti-Infective Agents

Idarubicin

Anti-Bacterial Agents

Antibiotics, Antitubercular

Elacytarabine 1000 mg/m2/d will be administered as a continuous intravenous infusion (CIV) in a d 1-5 q3w cycle.

Idarubicin will be administered IV at a fixed dose of 12 mg/ m2/d IV on d 1-3 q3w.

It is intended that patients receive remission-induction treatment either as two combination courses, elacytarabine 1000 mg/m2/d + idarubicin 12 mg/m2/d or one combination course, elacytarabine 1000 mg/m2/d + idarubicin 12 mg/m2/d followed by one course elacytarabine 2000 mg/m2/d single therapy.

Elacytarabine plus idarubicin

David A Rizzieri, MD

The main objective of this study is to assess the biological activity of elacytarabine in combination with idarubicin in patients with acute myeloid leukaemia who has failed the first course of a remission-induction treatment with cytarabine (ara-C). In addition, the correlation between hENT1 (human equilibrative nucleoside transporter 1) and overall survival will be studied.

Elacytarabine (CP-4055) is a pro-drug of ara-C currently used in the treatment of patients with acute myeloid leukaemia. Patients with nucleoside transporter deficiency (hENT1) seem to have less benefit from cytarabine compared to those with a high expression of the transporter. Preclinical studies indicate that elacytarabine is independent of this transporter. Therefore, patients with low expression of hENT1 and treated with elacytarabine are anticipated to have a better outcome compared to patients treated with ara-C. The main objective of this study is to assess the biological activity of elacytarabine in combination with idarubicin in patients with acute myeloid leukaemia. In addition, the correlation between hENT1 (human equilibrative nucleoside transporter 1) and overall survival will be studied. Patients will be treated with elacytarabine plus idarubicin independent of their hENT1 status. Determination of the patients' hENT1 expression level will be done retrospectively. This study will also explore the safety profile of elacytarabine in combination with idarubicin.

A Study of Elacytarabine (CP-4055) Plus Idarubicin as Second Course Remission-Induction Therapy in Patients With Acute Myeloid Leukaemia

A Phase II Study of Elacytarabine (CP-4055) Plus Idarubicin as Second Course Remission-Induction Therapy in Patients With Acute Myeloid Leukaemia

Theradex

Syneos Health

Clavis Pharma

Panobinostat

Histone Deacetylase Inhibitors

Panobinostat 20 mg oral three times weekly every second week

Panobinostat should be taken orally once on each scheduled day at about the same time, either with or without food. Each dose of panobinostat should be taken with a cup of water.

The capsules should be swallowed as whole. If vomiting occurs during the course of treatment, no re-dosing is allowed before the next scheduled dose. If one dose is forgotten during the morning on a scheduled treatment day then the missed dose should be taken on that same day within 12 hours. After more than 12 hours, that day's dose should be withheld, and the patient should wait to take panobinostat until the next schedule treatment day. The patient should then continue treatment with the original dosing schedule.

Panobinostat will be administered at a dose of 20 mg three times weekly (TIW) every second week and will be dosed on a flat scale of mg/day and not by weight or body surface area.

Panobinostat will be dispensed as a 20 mg capsule or as two 10 mg capsules.

Gesine Bug, PD Dr.

Aim of this prospective randomized trial is to compare maintenance treatment with panobinostat interspersed with donor lymphocyte infusions (DLI) versus the standard approach of pre-emptive DLI alone in patients with poor-risk AML/MDS having favorably received an allogeneic HSCT followed by engraftment, donor chimerism and hematopoietic reconstitution.

Allogeneic hematopoietic stem cell transplantation (HSCT) has been shown to improve the outcome of poor-risk AML and MDS in both younger and older patients. Reduced-intensity conditioning (RIC) regimen have partially abrogated the problem of regimen-related toxicity. However, graft-versus-host disease (GvHD) remains a major cause of non-relapse morbidity and mortality. Despite a strong graft versus leukemia (GvL) effect after allogeneic HSCT, the relapse rate after transplantation in poor-risk leukemia patients is still too high, necessitating new approaches to exploit GvL in a more optimized way. In addition, minimizing the GvHD reaction remains an important goal. One attractive strategy may be the administration of epigenetic therapy early after HSCT in order to optimize the GvL effect, to provide a direct anti-leukemic effect, and to control GvHD. Two preceding phase I/II studies have suggested that post-transplant administration of the histone deacetylase (HDAC) inhibitor panobinostat may be associated with a reduced relapse rate, while allowing for control of GvHD. Based on these two studies, the hypothesis of the present trial is that panobinostat can be an effective drug in preventing relapse by optimizing GvL in MDS and AML patients with high-risk features after HSCT, while at the same time reducing GvHD. It has been designed to test this hypothesis in a prospective randomized trial comparing maintenance with panobinostat interspersed with donor lymphocyte infusions (DLI) versus the standard approach of pre-emptive DLI alone in patients with poor-risk AML/MDS having favorably received an allogeneic HSCT followed by engraftment, donor chimerism and hematopoietic reconstitution.

Panobinostat Maintenance After HSCT fo High-risk AML and MDS

A Randomized, Multicenter Phase III Study to Assess the Efficacy of Panobinostat Maintenance Therapy vs. Standard of Care Following Allogeneic Stem Cell Transplantation in Patients With High-risk AML or MDS (ETAL-4 / HOVON-145)

OS is measured from date of randomization to the date of death or date of last follow up. Observations of patients alive at last follow up will be censored at date of last follow up.

EFS is defined as time interval from randomization until relapse of MDS or AML, any treatment of molecular relapse (except DLI) or death from any cause, whichever occurs first. Observations of patients without any event will be censored at time of last follow up.

DFS is defined as time interval from randomization until relapse of MDS or AML, or death from any cause, whichever occurs first. Observations of patients without any event will be censored at time of last follow up.

Remission duration is defined as time from randomization until relapse (including any treatment (except DLI) of molecular relapse). Death in complete remissions (CR) is considered as competing event for relapse. Patients for whom no relapse nor death in CR was observed will be censored at date of last follow up.

Time to NRM is defined as time from randomization until death in CR from any cause. Relapse is considered as competing event for NRM. Patients for whom no death in CR nor relapse was observed will be censored at date of last follow up.

Cumulative incidence of new onset or aggravation acute GvHD grade III-IV is calculated as number of patients who newly experienced acute GvHD grade III-IV after randomization or whose preexisting lower grade acute GvHD aggravated to grade 3 or 4 divided by all patients in the analysis set. Cumulative incidence is calculated separately for each arm.

Acute and chronic GvHD will be graded according to NIH consensus criteria. In case of chronic GvHD, evaluation forms provided by the NIH Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host-Disease should be used.

Time to chronic GvHD requiring systemic treatment is defined time from randomization until date of first diagnosis of chronic GvHD requiring systemic treatment. Relapse and death in CR without chronic GvHD requiring systemic treatment are considered as competing events. Patients for whom no chronic GvHD, nor relapse, nor death in CR was observed will be censored at date of last follow up.

Percentage of patients who are free of systemic immunosuppressive therapy is calculated as number of patients who are free of systemic immunosuppressive therapy at the respective time point divided by the total number of patients in the analysis set multiplied by 100. The percentage is calculated separately for each arm.

Percentage of patients completing the one year study treatment is calculated as number of patients completing the one year study treatment divided by the total number of patients in the analysis set multiplied by 100. The percentage is calculated for the panobinostat arm only.

The duration of panobinostat administration is calculated from date of first administered dose of study medication until date of last administered dose of study medication.

Percentage of patients with MRD conversion from baseline to 6 months after HSCT is calculated as number of patients who were MRD positive at baseline and are MRD negative at 6 months after HSCT divided by the number of patients who were MRD positive at baseline.

Patients who were MRD negative at baseline will not be considered in this endpoint.

Patient reported health-related quality of life will be assessed using the cancer generic 'European Organization for Research and Treatment of Cancer' quality of life questionnaire.

Stichting Hemato-Oncologie voor Volwassenen Nederland

Polish Adult Leukemia Group

Schweizerische Arbeitsgemeinschaft für klinische Krebsforschung

Goethe University

Director of Transplant Program, Principal Investigator

S64315 will be administered via i.v. infusion from 30 minutes and up to 3 hours once every week (21- day cycle), the starting dose is 50 mg. As data emerge during the study, the infusion duration and alternative dosing regimen may be changed.

S64315 once a week

S64315 will be administered via i.v. infusion from 30 minutes and up to 3 hours twice every week (28- day cycle), the starting dose is 50 mg. As data emerge during the study, the infusion duration and alternative dosing regimen may be changed.

S64315 twice a week

Andrew WEI

The CL1-64315-001 study is a phase I, international, multicentre, open-label, non-randomised, non-comparative study. This study is designed in two parts: one part for dose escalation, one part for dose expansion.

Phase I Study of S64315 Administred Intravenously in Patients With Acute Myeloid Leukaemia or Myelodysplastic Syndrome

Phase I, International, Multicentre, Open-label, Non-randomised, Non-comparative Study of Intravenously Administered S64315, a Mcl-1 Inhibitor, in Patients With Acute Myeloid Leukaemia (AML) or Myelodysplastic Syndrome (MDS)

Qualified scientific and medical researchers can request access to anonymized patient-level and study-level clinical trial data.

Access can be requested for all interventional clinical studies:

* used for Marketing Authorization (MA) of medicines and new indications approved after 1 January 2014 in the European Economic Area (EEA) or the United States (US).
* where Servier is the Marketing Authorization Holder (MAH). The date of the first MA of the new medicine (or the new indication) in one of the EEA Member States will be considered for this scope.

In addition, access can be requested for all interventional clinical studies in patients:

* sponsored by Servier
* with a first patient enrolled as of 1 January 2004 onwards
* for New Chemical Entity or New Biological Entity (new pharmaceutical form excluded) for which development has been terminated before any Marketing authorization (MA) approval.

ADIR, a Servier Group company

Institut de Recherches Internationales Servier

Pharmaceutical Solutions

AZD1152 radiolabelled IV solution. 1.05 mg/ml will be presented as a 15 ml fill in a 20 ml vial.

AZD1152

radiolabelled IV solution, 1.05 mg/ml presented as 15 ml fill in 20ml vial infusion

C14 AZD1152

Mike Dennis, MD

The purpose of the study is to assess how AZD1152 is absorbed or excreted in and out of the body in patients with Acute Myeloid Leukaemia (AML).

An Open Label, Single Centre Mass Balance C14 Study in Patients With Acute Myeloid Leukaemia (AML)

A Phase I Open, Non-randomised, Single-centre Study to Assess the Metabolism, Excretion and Pharmacokinetics of AZD1152 and AZD1152 hQPA Following Intravenous Administration of [14C]-AZD1152 in Patients With Acute Myeloid Leukaemia (AML)

AstraZeneca

Urinary Tract, Sexual Organs, and Pregnancy Conditions

Renal Insufficiency

Fludarabine

Fludarabine phosphate

Treosulfan

Antineoplastic Agents, Alkylating

Alkylating Agents

Participants will receive treosulfan 10 gram per square meter (g/m\^2), intravenous (IV) infusion, given over 2 hours once daily on three consecutive days (day -4 to day -2), followed by fludarabine, 30 milligram per square meter (mg/m\^2) IV infusion once daily on 5 consecutive days (days -6 to -2), preceding allogeneic haematopoietic stem cell transplantation (alloHSCT) on Day 0.

Ute Eckenbach

Chukwuemeka Uzoka

Boglarka Gyurkocza

Sarah Wall

William Clark

This study aim is to assess, if treosulfan pharmacokinetics are influenced by declined renal function and by race/ethnicity of patients. The study also aims to determine an appropriate safe dose of treosulfan, when patient's renal function is impaired. The participants of this study are undergoing allogenic hematopoietic stem cell transplantation for treatment of acute myeloid leukemia or myelodysplastic syndrome.

Effect of Moderate Renal Impairment and Race/Ethnicity on Treosulfan Pharmacokinetics

An Open-label, Non-randomized, Prospective Trial to Evaluate the Effect of Renal Function Impairment and Race/Ethnicity on Treosulfan Pharmacokinetics in Patients With AML or MDS Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

Time to engraftment is defined as the time between Day 0 and neutrophil/leukocyte/platelet engraftment. Engraftment includes Neutrophil engraftment, Leukocyte engraftment and Platelet engraftment. Neutrophil engraftment is defined the first of 3 consecutive days with an absolute neutrophil count (ANC) greater than (\>) 0.5\*10\^9 per liter (/L) in peripheral blood (PB) in the first of 3 consecutive days ;Leukocyte engraftment is defined as the first of 3 consecutive days with a total leukocyte count of more than 1\*10\^9/L in PB. Platelet engraftment is defined as the first of 3 consecutive days with a platelet count of at least 20\*10\^9/L or of at least 50\*10\^9/L in PB in the absence of platelet transfusion. Consecutive days is defined as 3 consecutive blood samples, if these are taken on different days.

A TEAE is defined as any Adverse Event (AE) that has an onset on or after the first dose of investigation medicinal product (IMP) or any pre existing condition that has worsened on or after the first dose of IMP. An SAE or serious adverse reaction (SAR) is any untoward medical occurrence that at any dose: results in death; is life-threatening; requires hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; is a congenital anomaly/birth defect; is another medically important event.

Synteract, Inc.

medac GmbH

Cytarabine

Antiviral Agents

Immunosuppressive Agents

Immunologic Factors

AZD1152 variable dose in combination with 20 mg of LDAC. (The LDAC is given twice daily.)

Variable dose via a 7-day continuous infusion

20 mg subcutaneous injection given twice daily

LDAC (low dose cytosine arabinoside)

Paul Stockman

Hagop Kantarjian

A study to assess the safety and tolerability of AZD1152 in combination with low dose cytosine arabinoside (LDAC) in patients with acute myeloid leukaemia (AML). The first three patients to complete a 28 day cycle in the cohort, before the second three patients start treatment.

Study to Assess the Safety and Tolerability of AZD1152 in Combination With Low Dose Cytosine Arabinoside (LDAC)

A Phase I, Open-label, Multi-centre, Multiple Ascending Dose Study to Assess the Safety and Tolerability of AZD1152 in Combination With Low Dose Cytosine Arabinoside (LDAC) in Patients With Acute Myeloid Leukaemia (AML)

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
A Single Arm Pilot Study of Azacitidine in Myelodysplastic Syndromes (MDS) / Acute Myeloid Leukaemia (AML), With Eltrombopag Support for Thrombocytopenia	NCT01488565	Myelodysplastic... Acute Myeloid L...	Azacitidine and...	18 Years -	Peter MacCallum Cancer Centre, Australia
A Study of Elacytarabine (CP-4055) Plus Idarubicin as Second Course Remission-Induction Therapy in Patients With Acute Myeloid Leukaemia	NCT01035502	Acute Myeloid L...	Elacytarabine p...	18 Years -	Clavis Pharma
Panobinostat Maintenance After HSCT fo High-risk AML and MDS	NCT04326764	Acute Myeloid L... Myelodysplastic...	Panobinostat	18 Years - 70 Years	Goethe University
Phase I Study of S64315 Administred Intravenously in Patients With Acute Myeloid Leukaemia or Myelodysplastic Syndrome	NCT02979366	Acute Myeloid L... Myelodysplastic...	S64315 once a w... S64315 twice a ...	18 Years -	Servier
An Open Label, Single Centre Mass Balance C14 Study in Patients With Acute Myeloid Leukaemia (AML)	NCT01019161	Acute Myeloid L...	AZD1152 C14 AZD1152	18 Years -	AstraZeneca
Effect of Moderate Renal Impairment and Race/Ethnicity on Treosulfan Pharmacokinetics	NCT05534620	Acute Myeloid L... Myelodysplastic... Allogeneic Hema...	Treosulfan Fludarabine	18 Years - 80 Years	medac GmbH
Study to Assess the Safety and Tolerability of AZD1152 in Combination With Low Dose Cytosine Arabinoside (LDAC)	NCT00926731	Acute Myeloid L...	AZD1152 LDAC (low dose ...	60 Years -	AstraZeneca

Spots Global Cancer Trial Database for acute myeloid leukaemia (aml)