- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02944955
A Study of BLEX 404 Oral Liquid in Patients With International Prognostic Scoring System (IPSS) Intermediate-1, Intermediate-2 or High-Risk Myelodysplastic Syndrome (MDS) and Chronic Myelomonocytic Leukemia (CMML)
Study Overview
Status
Conditions
Intervention / Treatment
Study Type
Enrollment (Estimated)
Phase
- Phase 2
Contacts and Locations
Study Contact
- Name: Hsien-Ming Wu, M.S.
- Phone Number: +886-3-6685386
- Email: sonnywu@bioliteinc.com
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
Inclusion Criteria:
- Age 20 or older, able to sign informed consent, understand and comply with protocol requirements and instructions.
- Body weight between 30 to 120 kg
- International Prognostic Scoring System (IPSS) intermediate-1 (int-1), intermediate-2 (int-2) or high-risk myelodysplastic syndrome (MDS) by World Health Organization (WHO) or Chronic Myelomonocytic Leukemia (CMML) patients.
- Not previously treated with MDS and CMML therapy and require treatment with azacitidine* for the first time at 75 mg/m2 subcutaneously or intravenously, daily for 7 days (7 nonconsecutive day of 6-1-1 or 5-2-2 treatment schedule is acceptable in which the drug is administered Monday-Saturday and then Monday of the following week for a 6-1-1 schedule, and in which the drug is administered Monday-Friday and then Monday-Tuesday of the following week for 5-2-2 schedule) per cycle. Treatment of the study drug, BLEX 404 Oral Liquid will begin at the same time receiving the first dose of azacitidine in this study.
- Eastern Cooperative Oncology Group (ECOG) Performance Status 0-3.
- Adequate baseline organ function defined by the criteria below: total bilirubin ≤ 1.5x the upper limit of normal (ULN) except for Gilbert's syndrome or indirect hyperbilirubinemia due to hemolysis; ALT ≤ 2.5x ULN; AST ≤ 2.5x ULN; creatinine clearance ≥ 60 mL/min/1.73 m2.
- Women must be either of non-child bearing potential, or women with child-bearing potential and men with reproductive potential and a female partner of childbearing potential must either have had a prior vasectomy or agree to use effective contraception from time of Screening Visit until the Follow-Up Visit.
Exclusion Criteria:
- Historical allergic events caused by mushroom.
- Previous treatment with hypomethylating agent or known immediate or delayed hypersensitivity reaction to drugs chemically related to azacitidine that contraindicates the subjects' participation.
- Patients with active infections or require treatments with immunosuppressive drugs at screening visit. However, patients require ongoing treatments with corticosteroids may be recruited.
- Treatment with an investigational drug within 30 days or 5 half-lives (whichever is longer) preceding the first dose of BLEX 404 Oral Liquid.
- Any serious and/or unstable pre-existing medical condition (including any advanced malignancy other than the disease under study), psychiatric disorder, or other conditions that could interfere with subject's safety, obtaining informed consent or compliance with the study procedures.
- Any clinically significant pulmonary, cardiovascular, endocrine, neurologic, gastrointestinal or genitourinary disease unrelated to underlying hematologic disorder.
- Active and uncontrolled infections of the following: hepatitis B (HBV), hepatitis C (HCV), and human immunodeficiency virus (HIV) infection. However, HBV and HCV carriers with viral load test of "not detected" or "negative" results may be recruited in this study.
- Lactating female and women with a positive serum or urine pregnancy test at screening assessments.
- (For Part II Interventional Study subjects only) No indication, in the opinion of the treating physician, for antibiotic prophylaxis within 2 weeks prior to study treatment.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: BLEX 404 Oral Liquid
Part I: Part I-1: Low Dose BLEX 404 Oral Liquid (3 mg/kg, BID) is administered in combination with 2 cycles of azacitidine. Part I-2: High Dose BLEX 404 Oral Liquid (4.5 mg/kg, BID) is administered in combination with 2 cycles of azacitidine. Part II: Recommended Dose Level (RDL) of BLEX 404 Oral Liquid will be determined by results from Part I. BLEX 404 Oral Liquid at RDL is administered in combination with 6 cycles of azacitidine. |
BLEX 404 Oral Liquid is orally administered twice daily in combination with azacitdine treatment cycles (2 cycles in Part I and 6 cycles in Part II). Azacitidine treatment: SC or IV injections at 75 mg/m2, QD for 7 days each cycle. 28 days/cycle, and repeat cycles every 4 weeks. |
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Part I: Recommended dose level (RDL) of BLEX 404 Oral Liquid
Time Frame: 8 weeks (2 cycles of azacitidine)
|
RDL is defined by BLEX 404 Oral Liquid-related prohibited toxicity in subjects undergoing 2 cycles of azacitidine treatment.
When one of the stopping criteria is met during the study treatment period, RDL will be set at the lower dose level.
|
8 weeks (2 cycles of azacitidine)
|
Part II: Incidence of infections
Time Frame: 24 weeks (6 cycles of azacitidine)
|
Number of subjects experience infection events and inpatient hospitalization due to infections.
|
24 weeks (6 cycles of azacitidine)
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Part I: (A) Number of subjects experience infections events and inpatient hospitalization due to infections
Time Frame: 8 weeks (2 cycles of azacitidine)
|
8 weeks (2 cycles of azacitidine)
|
|
Part I: (B) Treatments and duration of each infections
Time Frame: 8 weeks (2 cycles of azacitidine)
|
8 weeks (2 cycles of azacitidine)
|
|
Part I: (C) Complete remission (CR) or partial remission (PR) of bone marrow, peripheral blood, cytogenetic response (defined by 2006 IWG Criteria)
Time Frame: 8 weeks (2 cycles of azacitidine)
|
Hematological Response in subjects undergone BLEX 404 Oral Liquid in combination with 2 cycles of azacitidine.
|
8 weeks (2 cycles of azacitidine)
|
Part I: (D) Hematological improvement of erythroid (hemoglobin and RBC-transfusion independence), neutrophil (Absolute Neutrophil Count), and platelet (platelet count) response (defined by 2006 IWG Criteria)
Time Frame: 8 weeks (2 cycles of azacitidine)
|
Hematological Response in subjects undergone BLEX 404 Oral Liquid in combination with 2 cycles of azacitidine.
|
8 weeks (2 cycles of azacitidine)
|
Part I: (E) Frequency and requirement of red blood cell transfusion (defined by 2006 IWG Criteria)
Time Frame: 8 weeks (2 cycles of azacitidine)
|
Hematological Response in subjects undergone BLEX 404 Oral Liquid in combination with 2 cycles of azacitidine.
|
8 weeks (2 cycles of azacitidine)
|
Part I: (F) Disease Progression (defined by 2006 IWG Criteria)
Time Frame: 8 weeks (2 cycles of azacitidine)
|
Disease progression of bone marrow, erythroid (hemoglobin and RBC-transfusion independence), and platelet (platelet count) in subjects undergone BLEX 404 Oral Liquid treatment in combination with 2 cycles of azacitidine.
|
8 weeks (2 cycles of azacitidine)
|
Part II: (A) Time-to-first infection
Time Frame: 24 weeks (6 cycles of azacitidine)
|
24 weeks (6 cycles of azacitidine)
|
|
Part II: (B) Duration (days) of each infection event
Time Frame: 24 weeks (6 cycles of azacitidine)
|
24 weeks (6 cycles of azacitidine)
|
|
Part II: (C) Number of infections requiring supportive care or antimicrobial agents
Time Frame: 24 weeks (6 cycles of azacitidine)
|
24 weeks (6 cycles of azacitidine)
|
|
Part II: (D) Duration (days) of treatments for infections
Time Frame: 24 weeks (6 cycles of azacitidine)
|
24 weeks (6 cycles of azacitidine)
|
|
Part II: (E) Complete remission (CR) or partial remission (PR) of bone marrow, peripheral blood, cytogenetic response (defined by 2006 IWG Criteria)
Time Frame: 24 weeks (6 cycles of azacitidine)
|
Hematological Response in subjects undergone BLEX 404 Oral Liquid in combination with 6 cycles of azacitidine.
|
24 weeks (6 cycles of azacitidine)
|
Part II: (F) Hematological improvement of erythroid (hemoglobin and RBC-transfusion independence), neutrophil (Absolute Neutrophil Count), and platelet (platelet count) response (defined by 2006 IWG Criteria)
Time Frame: 24 weeks (6 cycles of azacitidine)
|
Hematological Response in subjects undergone BLEX 404 Oral Liquid in combination with 6 cycles of azacitidine.
|
24 weeks (6 cycles of azacitidine)
|
Part II: (G) Frequency and requirement of red blood cell transfusion (defined by 2006 IWG Criteria)
Time Frame: 24 weeks (6 cycles of azacitidine)
|
Hematological Response in subjects undergone BLEX 404 Oral Liquid in combination with 6 cycles of azacitidine.
|
24 weeks (6 cycles of azacitidine)
|
Part II: (H) Disease Progression (defined by 2006 IWG Criteria)
Time Frame: 24 weeks (6 cycles of azacitidine)
|
Disease progression of bone marrow, erythroid (hemoglobin and RBC-transfusion independence), and platelet (platelet count) in subjects undergone BLEX 404 Oral Liquid treatment in combination with 2 cycles of azacitidine.
|
24 weeks (6 cycles of azacitidine)
|
Part II: (I) Six-month overall survival rate (defined by 2006 IWG Criteria)
Time Frame: 24 weeks (6 cycles of azacitidine)
|
defined as time from beginning of treatment till death from any cause occurs or last follow-up.
|
24 weeks (6 cycles of azacitidine)
|
Part II: (J) Six-month progression-free survival (defined by 2006 IWG Criteria)
Time Frame: 24 weeks (6 cycles of azacitidine)
|
defined as time from beginning of treatment till disease progression, death from MDS or CMML, or last follow-up.
|
24 weeks (6 cycles of azacitidine)
|
Part II: (K) Immune Response
Time Frame: 24 weeks (6 cycles of azacitidine)
|
Neutrophil and monocyte function by respiratory burst assay
|
24 weeks (6 cycles of azacitidine)
|
Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start (Estimated)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimated)
Study Record Updates
Last Update Posted (Estimated)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Pathologic Processes
- Neoplasms by Histologic Type
- Neoplasms
- Disease Attributes
- Disease
- Bone Marrow Diseases
- Hematologic Diseases
- Precancerous Conditions
- Myelodysplastic-Myeloproliferative Diseases
- Leukemia
- Leukemia, Myeloid
- Chronic Disease
- Syndrome
- Myelodysplastic Syndromes
- Preleukemia
- Leukemia, Myelomonocytic, Chronic
- Leukemia, Myelomonocytic, Juvenile
Other Study ID Numbers
- BLI-1301-01
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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