NCT03886662 · Lixte Biotechnology Holdings, Inc.
A Study of LB-100 in Patients With Low or Intermediate-1 Risk Myelodysplastic Syndromes (MDS)
What this study is about
The purpose of this study is to test the safety and effectiveness (benefits) of an experimental 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 given through a vein (IV) infusion over 120 minutes. This study will be conducted in 2 phases.
View original scientific 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.
Interventions
DRUG
LB-100
Phase Ib: Two escalating doses of LB-100 in two separate cohorts will be administered intravenously on days 1, 3 and 5 of a 21-day cycle over 120 minutes. Phase 2: Safe dose of LB-100 as determined from phase Ib will be administered intravenously on days 1, 3 and 5 of a 21-day cycle over 120 minutes.
Primary outcome measures
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
Number of patients with treatment-related adverse events as assessed by Common Terminology Criteria for Adverse Events v5.0 (CTCAE v5.0)
For Phase 2 - Best overall response rate of patients to the study treatment as a measure of efficacy of LB-100 study drug
Time frame: At screening and then at the end of Cycle 3 and Cycle 6. (Each cycle is 21 days)
Best overall response rate of the patients to the study treatment as assessed by International Working Group (IWG) 2006 criteria
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Patient has signed the Informed Consent Form (ICF) and is able to comply with protocol requirements.
- 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
- Age ≥18 years at the time of signing the informed consent form.
- 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.
- 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.
- 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.
- An Eastern Cooperative Oncology Group (ECOG) performance status score of 0, 1, or 2.
- 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
- Patient has a known history of HIV infection (testing not mandatory).
- 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
- 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.
- 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.
- 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.
- 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.
Where
- Tampa, Florida
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Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Apr 8, 2019 · Source of record for eligibility and locations