RECRUITING

A Study of LB-100 in Patients With Low or Intermediate-1 Risk Myelodysplastic Syndromes (MDS)

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

The purpose of this study is to test the safety and efficacy (benefits) of an investigational drug LB-100, for treatment of myelodysplastic syndromes. LB-100 has previously been administered to patients with various solid tumors. In this study, LB-100 will be administered as an intravenous infusion over 120 minutes. This study will be conducted in 2 phases. In phase Ib, escalating doses of LB-100 will be administered to patients to study the safety and to determine a safe dose of LB-100. In phase 2, patients will be administered LB-100 at the dose that was found to be safe in phase Ib. The efficacy (benefits) and safety of LB-100 will be determined in this phase of the study.

Official Title

A Phase 1b/2 Study Evaluating the Safety and Efficacy of Intravenous LB-100 in Patients With Low or Intermediate-1 Risk Myelodysplastic Syndromes (MDS) Who Had Disease Progression or Are Intolerant to Prior Therapy

Quick Facts

Study Start:2019-04

Study Completion:2021-07

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT03886662

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
1. Patient has signed the Informed Consent Form (ICF) and is able to comply with protocol requirements. 2. Patient has adequate organ function as defined by the following laboratory values: * Creatinine clearance (CrCl) ≥ 60ml/min * Total serum bilirubin \< 1.5 x Upper Limit of Normal (ULN) or total bilirubin ≤ 3.0 x ULN with direct bilirubin within normal range only in patients with well documented Gilbert's syndrome or hemolysis or who required regular blood transfusions * Alanine aminotransferase (AST) and aspartate aminotransferase (ALT) \< 3.0 x ULN 3. Age ≥18 years at the time of signing the informed consent form. 4. Documented diagnosis of MDS or MDS/myeloproliferative neoplasm (MPN) by World Health Organization (WHO) criteria that require treatment due to cytopenias and meet the International Prognostic Scoring System (IPSS) criteria for low or int-1 risk. 5. For non-del(5q) patients, failed prior treatment with at least 2 cycles started of azacitidine or decitabine or lenalidomide defined as no response to treatment, loss of response at any time point while on treatment or within 6 months of treatment discontinuation, or progressive disease/intolerance to therapy. 6. For del(5q) patients, failed prior treatment with at least 2 cycles started of lenalidomide defined as no response to treatment, loss of response at any time point, or progressive disease/intolerance to therapy. 7. An Eastern Cooperative Oncology Group (ECOG) performance status score of 0, 1, or 2. 8. Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence; tubal ligation, partner's vasectomy) prior to Cycle 1 Day 1 (C1D1) and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.	1. Patient has a known history of HIV infection (testing not mandatory). 2. Patient has any of the following cardiac abnormalities: * symptomatic congestive heart failure * myocardial infarction ≤ 6 months prior to enrollment * unstable angina pectoris as designated by the treating physician * serious uncontrolled cardiac arrhythmia as designated by the treating physician * QTcF (Fridericia's correction formula) ≥ 450 msec 3. Concomitant malignancies or previous malignancies with less than a 1-year disease free interval at the time of enrollment. Patients with adequately resected basal or squamous cell carcinoma of the skin, or adequately resected carcinoma in situ (i.e. cervix) may enroll irrespective of the time of diagnosis. 4. Use of chemotherapeutic agents or experimental agents (agents that are not commercially available) for the treatment of MDS within 14 days of the first day of study drug treatment. 5. No concurrent use of erythroid stimulating agents, Granulocyte-colony stimulating factor (G-CSF), Granulocyte-macrophage colony-stimulating factor (GM-CSF) is allowed during study except in cases of febrile neutropenia where G-CSF can be used for short term. Growth factors must be stopped two weeks prior to study. 6. Pregnant women are excluded from this study because LB-100 has not been studied in pregnant subjects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with LB-100, breastfeeding should be discontinued if the mother is treated with LB-100.

Inclusion Criteria

Exclusion Criteria

1. Patient has signed the Informed Consent Form (ICF) and is able to comply with protocol requirements.
2. Patient has adequate organ function as defined by the following laboratory values:
* Creatinine clearance (CrCl) ≥ 60ml/min
* Total serum bilirubin \< 1.5 x Upper Limit of Normal (ULN) or total bilirubin ≤ 3.0 x ULN with direct bilirubin within normal range only in patients with well documented Gilbert's syndrome or hemolysis or who required regular blood transfusions
* Alanine aminotransferase (AST) and aspartate aminotransferase (ALT) \< 3.0 x ULN
3. Age ≥18 years at the time of signing the informed consent form.
4. Documented diagnosis of MDS or MDS/myeloproliferative neoplasm (MPN) by World Health Organization (WHO) criteria that require treatment due to cytopenias and meet the International Prognostic Scoring System (IPSS) criteria for low or int-1 risk.
5. For non-del(5q) patients, failed prior treatment with at least 2 cycles started of azacitidine or decitabine or lenalidomide defined as no response to treatment, loss of response at any time point while on treatment or within 6 months of treatment discontinuation, or progressive disease/intolerance to therapy.
6. For del(5q) patients, failed prior treatment with at least 2 cycles started of lenalidomide defined as no response to treatment, loss of response at any time point, or progressive disease/intolerance to therapy.
7. An Eastern Cooperative Oncology Group (ECOG) performance status score of 0, 1, or 2.
8. Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence; tubal ligation, partner's vasectomy) prior to Cycle 1 Day 1 (C1D1) and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.

1. Patient has a known history of HIV infection (testing not mandatory).
2. Patient has any of the following cardiac abnormalities:
* symptomatic congestive heart failure
* myocardial infarction ≤ 6 months prior to enrollment
* unstable angina pectoris as designated by the treating physician
* serious uncontrolled cardiac arrhythmia as designated by the treating physician
* QTcF (Fridericia's correction formula) ≥ 450 msec
3. Concomitant malignancies or previous malignancies with less than a 1-year disease free interval at the time of enrollment. Patients with adequately resected basal or squamous cell carcinoma of the skin, or adequately resected carcinoma in situ (i.e. cervix) may enroll irrespective of the time of diagnosis.
4. Use of chemotherapeutic agents or experimental agents (agents that are not commercially available) for the treatment of MDS within 14 days of the first day of study drug treatment.
5. No concurrent use of erythroid stimulating agents, Granulocyte-colony stimulating factor (G-CSF), Granulocyte-macrophage colony-stimulating factor (GM-CSF) is allowed during study except in cases of febrile neutropenia where G-CSF can be used for short term. Growth factors must be stopped two weeks prior to study.
6. Pregnant women are excluded from this study because LB-100 has not been studied in pregnant subjects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with LB-100, breastfeeding should be discontinued if the mother is treated with LB-100.

Contacts and Locations

Principal Investigator

Rami Komrokji, MD

PRINCIPAL_INVESTIGATOR

H. Lee Moffitt Cancer Center and Research Institute

Study Locations (Sites)

H. Lee Moffitt Cancer Center & Research Institute

Tampa, Florida, 33612

United States

Collaborators and Investigators

Sponsor: Lixte Biotechnology Holdings, Inc.

Rami Komrokji, MD, PRINCIPAL_INVESTIGATOR, H. Lee Moffitt Cancer Center and Research Institute

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2019-04

Study Completion Date2021-07

Study Record Updates

Study Start Date2019-04

Study Completion Date2021-07

Terms related to this study

Additional Relevant MeSH Terms

Myelodysplastic Syndromes