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NCT02995655 · ClinicalTrials.gov registry record · Phase 1
CX-01 Combined With Azacitidine in the Treatment of Relapsed or Refractory Myelodysplastic Syndrome and Acute Myeloid Leukemia
A Phase 1 study, sponsored by Washington University School of Medicine.
- Completed
- Registry status
- Phase 1
- Development phase
- 20
- Enrollment target
NCT02995655: Completed Phase 1 study, sponsored by Washington University School of Medicine.
NCT02995655 is a Phase 1 study that has completed, run by Washington University School of Medicine. The registered enrollment target is 20 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (67% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02995655, a Phase 1 study, has completed, sponsored by Washington University School of Medicine.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 20 participants
- Enrollment target
Study Summary
The investigators hypothesize that CX-01 will disrupt the bone marrow microenvironment and increase the cytotoxic effects of azacitidine on myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) hematopoietic stem cells by disrupting the High-mobility group box protein 1 (HMGB1) interaction with toll-like receptor 4 (TLR4) and receptors for advanced glycation end products (RAGE), the CXC chemokine CXCL12/chemokine receptor 4 (CXCR4) axis, and by disrupting other leukocyte and vascular adhesion molecules. In addition, CX-01 may also help promote count recovery after treatment given its affinity for platelet factor-4 (PF4). The selection of CX-01 dose for study in relapsed or refractory MDS and AML has been based upon the dual requirements to have sufficient drug administered to have potential activity but without clinically significant anticoagulation. The study dose chosen (4 mg/kg bolus followed by 0.25 mg/kg/hour) fulfills both of these criteria. In addition, this dose is expected to result in serum levels of CX-01 which are significantly higher than the IC90 identified in preclinical studies for inhibition of HMGB1-RAGE, toll-like receptor 2 (TLR2) and TLR4 interaction. Therefore, the chosen dose represents a rational balance between effective dosing and safety in thrombocytopenic patients with MDS and AML. This dose was previously established to be safe and tolerable when combined with cytarabine and idarubicin in patients with AML.
Primary Outcome
* Overall response rate = the percentage of patients obtaining partial response or higher * Patients will be assessed for response according to modified International Working Group (IWG) criteria
Interventions
- DRUG Azacitidine
- PROCEDURE Peripheral blood draw
- PROCEDURE Bone marrow biopsy
- DRUG CX-01
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 20 participants |
| Start Date | 2017-04-07 |
| Est. Completion | 2019-04-29 |
| Phase | Phase 1 |
What the finished NCT02995655 record still lists
NCT02995655 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 20 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (67% lower).
The record links to 0 conditions, and to 4 interventions - of which Azacitidine is the first listed.
NCT02995655 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT02995655 about?
NCT02995655 is a clinical study titled "CX-01 Combined With Azacitidine in the Treatment of Relapsed or Refractory Myelodysplastic Syndrome and Acute Myeloid Leukemia". The investigators hypothesize that CX-01 will disrupt the bone marrow microenvironment and increase the cytotoxic effects of azacitidine on myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) hematopoietic stem cells by disrupting the High-mobility group box protein 1 (HMGB1) interaction...
What is the current status of trial NCT02995655?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 20 participants. The study started on 2017-04-07. Estimated completion is 2019-04-29.
What interventions are being tested in trial NCT02995655?
The interventions under investigation include: Azacitidine (DRUG), Peripheral blood draw (PROCEDURE), Bone marrow biopsy (PROCEDURE), CX-01 (DRUG).
Who is sponsoring clinical trial NCT02995655?
This trial is sponsored by Washington University School of Medicine, which has 1,502 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT02995655's enrollment target sits among peer trials
20 1629th of 2000 higher than 285 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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