Clinical Trials /

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

NCT03886662

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.

Related Conditions:
  • Myelodysplastic Syndromes
  • Myelodysplastic/Myeloproliferative Neoplasm
Recruiting Status:

Recruiting

Phase:

Phase 1/Phase 2

Trial Eligibility

Document

Title

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

Clinical Trial IDs

  • ORG STUDY ID: MCC-19635
  • NCT ID: NCT03886662

Conditions

  • Myelodysplastic Syndromes

Interventions

DrugSynonymsArms
LB-100LB-100 for Intravenous administration

Purpose

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.

Trial Arms

NameTypeDescriptionInterventions
LB-100 for Intravenous administrationExperimentalPhase Ib: Escalating doses of LB-100 administered. Phase 2: Safe dose of LB-100 from phase Ib administered.
  • LB-100

Eligibility Criteria

        Inclusion 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.

        Exclusion Criteria:

          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.
      
Maximum Eligible Age:N/A
Minimum Eligible Age:18 Years
Eligible Gender:All
Healthy Volunteers:No

Primary Outcome Measures

Measure:For Phase Ib - Number of patients with adverse events related to the study treatment as a measure of safety and tolerability of LB-100 study drug
Time Frame:From the first dose of the study drug to 30-days following last dose of the study drug
Safety Issue:
Description:Number of patients with treatment-related adverse events as assessed by Common Terminology Criteria for Adverse Events v5.0 (CTCAE v5.0)

Details

Phase:Phase 1/Phase 2
Primary Purpose:Interventional
Overall Status:Not yet recruiting
Lead Sponsor:Lixte Biotechnology Holdings, Inc.

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