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Myelodysplastic Syndrome (MDS)Phase 2NCT02944955

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)

A Phase 2 study testing BLEX 404 Oral Liquid in people with Myelodysplastic Syndrome (MDS), run by BioLite, Inc..

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Sites and contacts
Official record
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Trial summary

This is a clinical trial to determine the safety, recommended dose level (RDL), and infection control of BLEX 404 Oral Liquid in combination with azacitidine in patients with International Prognostic Scoring System (IPSS) intermediate-1 (int-1), intermediate-2 (int-2) or high-risk myelodysplastic syndrome (MDS) and… Read more about this study

Who is it for?
Ages 20 years and older
Conditions the study is for
Myelodysplastic Syndrome (MDS)
What is being tested?
BLEX 404 Oral Liquid
Could I receive a placebo?
No, everyone receives an active treatment
How many people
52 participants
Where
No sites listed yet
Study dates
1 Jan 2027to 31 Dec 2028
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 20 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.

Who cannot join

  • 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.
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  • 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.

Trial locations and contacts

No sites are listed yet. Contact the study team or a site near you to ask about taking part.

Hsien-Ming Wu, M.S. contact

+886-3-6685386

sonnywu@bioliteinc.com

About this study

This is a clinical trial to determine the safety, recommended dose level (RDL), and infection control of BLEX 404 Oral Liquid in combination with azacitidine in patients with International Prognostic Scoring System (IPSS) intermediate-1 (int-1), intermediate-2 (int-2) or high-risk myelodysplastic syndrome (MDS) and Chronic Myelomonocytic Leukemia (CMML).

Being tested
BLEX 404 Oral Liquid
Medical subject headings
Myelodysplastic Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • BLEX 404 Oral LiquidDrug

    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.

Study groups in NCT02944955
Study groupRoleWhat they receive
BLEX 404 Oral LiquidExperimentalPart 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.
Receives: Drug: BLEX 404 Oral Liquid

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Part I: Recommended dose level (RDL) of BLEX 404 Oral Liquid

    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.

    Measured: 8 weeks (2 cycles of azacitidine)

  2. Part II: Incidence of infections

    Number of subjects experience infection events and inpatient hospitalization due to infections.

    Measured: 24 weeks (6 cycles of azacitidine)

Secondary outcomes (17)

  1. Part I: (A) Number of subjects experience infections events and inpatient hospitalization due to infections

    Measured: 8 weeks (2 cycles of azacitidine)

  2. Part I: (B) Treatments and duration of each infections

    Measured: 8 weeks (2 cycles of azacitidine)

  3. Part I: (C) Complete remission (CR) or partial remission (PR) of bone marrow, peripheral blood, cytogenetic response (defined by 2006 IWG Criteria)

    Hematological Response in subjects undergone BLEX 404 Oral Liquid in combination with 2 cycles of azacitidine.

    Measured: 8 weeks (2 cycles of azacitidine)

  4. 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)

    Hematological Response in subjects undergone BLEX 404 Oral Liquid in combination with 2 cycles of azacitidine.

    Measured: 8 weeks (2 cycles of azacitidine)

  5. Part I: (E) Frequency and requirement of red blood cell transfusion (defined by 2006 IWG Criteria)

    Hematological Response in subjects undergone BLEX 404 Oral Liquid in combination with 2 cycles of azacitidine.

    Measured: 8 weeks (2 cycles of azacitidine)

  6. Part I: (F) Disease Progression (defined by 2006 IWG Criteria)

    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.

    Measured: 8 weeks (2 cycles of azacitidine)

  7. Part II: (A) Time-to-first infection

    Measured: 24 weeks (6 cycles of azacitidine)

  8. Part II: (B) Duration (days) of each infection event

    Measured: 24 weeks (6 cycles of azacitidine)

  9. Part II: (C) Number of infections requiring supportive care or antimicrobial agents

    Measured: 24 weeks (6 cycles of azacitidine)

  10. Part II: (D) Duration (days) of treatments for infections

    Measured: 24 weeks (6 cycles of azacitidine)

  11. Part II: (E) Complete remission (CR) or partial remission (PR) of bone marrow, peripheral blood, cytogenetic response (defined by 2006 IWG Criteria)

    Hematological Response in subjects undergone BLEX 404 Oral Liquid in combination with 6 cycles of azacitidine.

    Measured: 24 weeks (6 cycles of azacitidine)

  12. 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)

    Hematological Response in subjects undergone BLEX 404 Oral Liquid in combination with 6 cycles of azacitidine.

    Measured: 24 weeks (6 cycles of azacitidine)

  13. Part II: (G) Frequency and requirement of red blood cell transfusion (defined by 2006 IWG Criteria)

    Hematological Response in subjects undergone BLEX 404 Oral Liquid in combination with 6 cycles of azacitidine.

    Measured: 24 weeks (6 cycles of azacitidine)

  14. Part II: (H) Disease Progression (defined by 2006 IWG Criteria)

    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.

    Measured: 24 weeks (6 cycles of azacitidine)

  15. Part II: (I) Six-month overall survival rate (defined by 2006 IWG Criteria)

    defined as time from beginning of treatment till death from any cause occurs or last follow-up.

    Measured: 24 weeks (6 cycles of azacitidine)

  16. Part II: (J) Six-month progression-free survival (defined by 2006 IWG Criteria)

    defined as time from beginning of treatment till disease progression, death from MDS or CMML, or last follow-up.

    Measured: 24 weeks (6 cycles of azacitidine)

  17. Part II: (K) Immune Response

    Neutrophil and monocyte function by respiratory burst assay

    Measured: 24 weeks (6 cycles of azacitidine)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted26 Oct 2016 (estimated)
  2. Study start1 Jan 2027 (estimated)
  3. Primary completion31 Dec 2028 (estimated)
  4. Study completion31 Dec 2028 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
52 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02944955
Sponsor's protocol ID
BLI-1301-01
Status last verified
Apr 2026
First submitted
4 Oct 2016
Last updated
13 Apr 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing BLEX 404 Oral Liquid in people with Myelodysplastic Syndrome (MDS), run by BioLite, Inc..

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: opening soon, as of the sponsor's last update on 13 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Hsien-Ming Wu, M.S. (+886-3-6685386, sonnywu@bioliteinc.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Part I: Recommended dose level (RDL) of BLEX 404 Oral Liquid (time frame: 8 weeks (2 cycles of azacitidine)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02944955.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Apr 2026.

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