MDS/MPNPhase 1/2ABNL-MARRONCT04061421
Active Myeloid Target Compound Combinations in MDS/MPN Overlap Syndromes Overlap Syndromes (ABNL-MARRO)
A Phase 1/2 study testing ASTX727, Itacitinib and Ruxolitinib in people with MDS/MPN, run by Michael Savona at 4 sites in United States.
Trial summary
ABNL-MARRO (A Basket study of Novel therapy for untreated MDS/MPN and Relapsed/Refractory Overlap Syndromes) is an international European-American cooperation providing the framework for collaborative studies to advance treatment of myelodysplastic/myeloproliferative neoplasms (MDS/MPN) and explore clinical-pathologic… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- MDS/MPN
- What is being tested?
- ASTX727, Itacitinib and Ruxolitinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 94 participants
- Where
- 4 sitesUnited States
- Study dates
- 24 Nov 2021to Dec 2027
- Phase
- Phase 1/2
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Must be ≥ 18 years of age at the time of signing the Informed Consent Form (ICF); must voluntarily sign an ICF; and must be willing and able to meet all study requirements.
- Must have morphologically confirmed diagnosis of MDS/MPN, excluding JMML, in accordance with WHO (2016) diagnostic criteria (Appendix 1, Section 12.1).
- Treatment-naïve patients (patients who have had no prior disease-modifying therapy) may enroll in any AM-001 Arm that is open to accrual in phase 1 or phase 2. Treatment-naïve patients may have received recombinant erythropoietin, danazol, hydroxyurea or anagrelide, which are not considered to be disease-modifying therapy for the purpose of this study.
- After an appropriate wash-out period, patients who have failed (or were intolerant to) prior therapy with a regimen(s) containing a DNMTi may enroll in any Arm in phase 1b or any Arm which has met the criterion of the first Simon's Stage and are open to accrual in the second Simon's Stage in phase 2 (Error! Reference source not found.). Except in the first stage of the phase 2, there are no limits on number of prior therapies if the patient meets all other eligibility criteria. Previously treated patients include:
- Patients treated with DNMTi therapy prior to enrollment in AM-001 who failed to achieve a complete remission, per the MDS/MPN IWG response criteria, after at least 4 cycles of DNMTi therapy
- Patients enrolled in AM-001, or patients treated off-study with a regimen containing a DNMTi, who have definitive disease progression as defined in the protocol after at least 2 cycles of the prior therapy-this includes patients who fail to achieve a response with clearly progressive disease and patients who achieve an initial response who then lose that response;
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- Patients enrolled in AM-001 who have stable disease as best response at the second response evaluation after 6 cycles of the prior AM-001 therapy;
- Patients treated on AM-001 who had and recovered from an adverse event that precludes further therapy on that Arm; after recovery from a toxicity that is likely to be related to ASTX727, enrollment in another AM-001 may occur provided that dose modifications are made as appropriate.
- Must be willing to undergo bone marrow biopsy with aspiration during screening and bone marrow aspiration with tissue collection for disease assessment and correlative studies periodically throughout the trial.
- Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2.
- Life expectancy of at least 3 months, as assessed by the treating physician.
- For previously treated patients, recovery to ≤ Grade 1 or baseline of any toxicities due to prior systemic treatments, excluding alopecia.
- Must have adequate hepatic and renal function during screening as demonstrated by:
- ALT (SGPT) and AST (SGOT) ≤ 3x the institutional upper limit of normal (ULN);
- Total bilirubin ≤ 1.5x ULN or ≤ 2x ULN, if upon judgment of the treating investigator the elevated bilirubin is due to extramedullary hematopoiesis related to the underlying MDS/MPN or to Gilbert's disease;
- Calculated creatinine clearance (CrCl) ≥60 mL/min. For dose modification purposes in Arm B, CrCl should be calculated using the Cockcroft-Gault equation. For patients with renal impairment entering Arm B (ASTX727 + ruxolitinib), specific dose modifications per the section 6.5.4 will apply.
Who cannot join
- Patients should be excluded from any treatment Arm that includes a novel targeted agent to which they have had previous exposure. Novel targeted agents in this study currently include itacitinib (INCB039110) and ruxolitinib (RUX) only, currently. Patients who have had prior exposure to ASTX727 therapy are not excluded, provided they meet all other eligibility criteria.
- Prior receipt of any investigational study drug, including treatment on any prior AM-001 Arm, within 30 days or 5 half-lives (whichever is shorter) before receiving the first dose of study drug in an Arm of AM-001, except if approved by the medical monitor.
- Prior receipt of any systemic antineoplastic therapy, including but not limited to prior DNMTi therapy, standard induction or cytotoxic chemotherapy (excluding hydroxyurea), or approved targeted agent within 21 days or 5 half-lives (whichever is shorter) before receiving the first dose of study drug in an Arm of AM-001.
- Known hypersensitivity to decitabine and ruxolitinib.
- Transformation to acute myeloid leukemia (e.g., >20% myeloid blasts in bone marrow or >20% circulating blasts in peripheral blood).
- Organ transplant recipients including allogeneic hematopoietic stem cell transplant.
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- History of clinically significant or uncontrolled cardiac disease, including recent history (within 6 months) of unstable angina, acute myocardial infarction, New York Heart Association Class III or IV congestive heart failure, or clinically significant uncontrolled arrhythmia. Patients with history of atrial tachycardia and/or bradycardia that is well-controlled with medical management and/or pacemaker for at least 1 month before the first dose of study drug will be allowed.
- History of thromboemobolic events (such as deep vein thrombosis, pulmonary embolism, stroke, myocardial infraction) in the 6 months prior to study entry.
- Active HBV or HCV. Participants with positive total HBc antibody or positive HCV antibody must have negative viral load for HBV and HCV at screening.
- Known HIV seropositive status. For participants with unknown HIV status, HIV testing will be performed at screening.
- History of abnormal electrocardiogram (ECG) or presence of abnormal screening ECG that, in the investigator's opinion, is clinically significant and contraindicated for clinical study. Corrected QT interval (QTc), as corrected by Fredericia, on screening ECG >500 milliseconds is excluded, unless there is concomitant right bundle branch block (RBBB) or concomitant left bundle branch block (LBBB) with a pacemaker.
- Any known contraindications to the use of ASTX727.
- Any sign of active and clinically significant bleeding.
- Other active malignancy, not including localized non-melanoma skin cancer, cervical carcinoma in situ, breast ductal carcinoma in situ of the breast, or localized prostate cancer controlled with hormone therapy. Patients with history of other cancers should be free of disease without ongoing anti-neoplastic therapy for at least 2 years.
- Receipt of wide-field radiotherapy (including therapeutic radioisotopes) ≤ 28 days or limited field radiation for palliation ≤ 14 days prior to starting study medications; or has not recovered from side effects of such therapy.
- Patients who require continuation of a prohibited concomitant medication (Section 6.6) for which no alternative therapy or allowable substitute is available.
- Active, uncontrolled infection. Patients with infection that is under control with active treatment are eligible.
- Major surgery requiring general anesthesia within 4 weeks prior to starting study treatment. (Placement of a central line or port-a-catheter is acceptable within this time frame and does not exclude the patient.)
- Women who are pregnant or lactating.
- Subjects who expect to conceive or father children within the projected duration of the study and/or who are unwilling to use highly effective methods of contraception throughout the duration of the study, starting with the screening visit through the end of treatment visit. For women of child-bearing potential (WOCBP), a negative urine pregnancy test at screening and immediately prior to initiating treatment on any AM-001 treatment Arm (Cycle 1 Day 1) is required.
- Any concurrent serious or unstable medical or psychiatric condition that in the investigator's opinion would jeopardize the patient's ability to provide informed consent or to comply with the protocol.
- Any psychological, familial, geographical or sociological condition that in the investigator's opinion would jeopardize the patient's ability to comply with the protocol.
Trial locations and contacts
4 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States4 sites, 4 recruiting
- Recruiting
Vanderbilt-Ingram Cancer Center
Nashville, Tennessee, 37232
Vanderbilt-Ingram Service Information Program, 800-811-8480, cip@vumc.org
Michael Savona, MD
- Recruiting
Oregon Health Sciences University
Portland, Oregon, 97239
Ted Braun, braunt@ohsu.edu
- Recruiting
University of Rochester Wilmot Cancer Institute
Rochester, New York, 14609
Jason Mendler, jason_mendler@urmc.rochester.edu
- Recruiting
Moffitt Cancer Center
Tampa, Florida, 33612
Zhuoer Xie, zhuoer.xie@moffitt.org
About this study
Official title: The ABNL-MARRO 001 Study: A Phase 1/2 Study of Active Myeloid Target Compound Combinations in MDS/MPN Overlap Syndromes.
ABNL-MARRO (A Basket study of Novel therapy for untreated MDS/MPN and Relapsed/Refractory Overlap Syndromes) is an international European-American cooperation providing the framework for collaborative studies to advance treatment of myelodysplastic/myeloproliferative neoplasms (MDS/MPN) and explore clinical-pathologic markers of disease severity, prognosis and treatment response.
ABNL MARRO 001 (AM-001) is an Open label, phase 1/2 study within the framework of the ABNL-MARRO that will test novel treatment combinations in MDS/MPN. Each Arm of AM-001 will test an active myeloid target compound in combination with ASTX727, an oral drug combining fixed doses of the DNA methyltransferase inhibitor (DNMTi) decitabine and the cytidine deaminase inhibitor E7727, also known as cedazuridine in a single tablet.
Read the detailed description
Current Arm: ASTX727 + ruxolitinib (JAK1/JAK2 inhibitor)
Primary Objective Phase 2:
- To test whether the overall response to each novel ASTX727 combination therapy in MDS/MPN patients is sufficiently high to warrant further investigation in more definitive trials.
Secondary Objectives:
- To expand the safety analysis of each treatment combination in MDS/MPN patients.
- To assess the morphologic bone marrow response in MDS/MPN patients treated on each Arm of the study.
- To estimate the effect of each treatment combination on patient survival
- To test the applicability of the proposed MDS/MPN IWG response criteria across multiple Arms of this study.
Tertiary/Exploratory objectives:
- To investigate genetic biomarkers of response in MDS/MPN.
- To characterize molecular responses to individual treatments.
- To evaluate synergistic effects of hypomethylation by ASTX727 and specific pathway blockade by study compounds.
- To explore the use of automated quantification of spleen volume from CT exams as a measure of clinical benefit
- To test and/or validate diagnostic algorithms and prognostic indices for MDS/MPN patients
- To investigate the correlation of patient reported outcomes with disease severity and/or treatment response
- Conditions
- MDS/MPN
- Being tested
- ASTX727, Itacitinib, Ruxolitinib
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ASTX727Drug
Also called Fixed dose combination of cedazuridine (100mg) + oral decitabine (35mg)
Taken by mouth daily during days 1-5 of every 28-day cycle.
ItacitinibDrug
Also called INCB039110
Taken by mouth daily during each 28-day cycle
RuxolitinibDrug
Also called Jakafi for oral
Dosage will be 5,15, or 20mg Taken by mouth BID for 28-day cycle
| Study group | Role | What they receive |
|---|---|---|
| ASTX727 + itacitinib | Experimental | ASTX727 and itacitinib will be taken by mouth Receives: Drug: ASTX727; Drug: Itacitinib |
| ASTX727 + ruxolitinib | Experimental | ASTX727 and ruxolitinib will be taken by mouth Receives: Drug: ASTX727; Drug: Ruxolitinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Phase 1: Determination of dose
Per CTCAE 5.0
Measured: Up to 28 days
Phase 2: Overall response rate (ORR). Overall response includes patients who achieve best response of CR, PR, MR, or CB as defined by the MDS/MPN IWG proposed response criteria
Measured: Up to 2 years
Secondary outcomes (4)
Adverse Event/DLT severity and frequency
Measured: Up to 2 years
CR/MR rate defined as the proportion of patients who achieve either complete remission or marrow response as a best response to treatment.
Measured: Up to 2 years
Overall survival (OS)
Defined as the time from first protocol-indicated treatment to death for any reason
Measured: Up to 2 years
Progression free survival (PFS)
Defined as the time from first protocol-indicated treatment to disease progression or death for any reason
Measured: Up to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Aug 2019
- Study start24 Nov 2021
- Primary completionAug 2027 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for MDS/MPNStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 94 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Michael Savona
- Sponsor type
- University, hospital or other
- Collaborators
- Incyte Corporation, Theradex, Astex Pharmaceuticals, Inc.
- Responsible party
- Sponsor-investigator, Michael Savona, Vanderbilt-Ingram Cancer Center
- Investigators
- Michael Savona, MD, Study chair, Vanderbilt-Ingram Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04061421
- Sponsor's protocol ID
- VICC HEMP 1977
- Acronym
- ABNL-MARRO
- Status last verified
- Feb 2026
- First submitted
- 9 Aug 2019
- Last updated
- 29 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Moyo TK, Mendler JH, Itzykson R, Kishtagari A, Solary E, Seegmiller AC, Gerds AT, Ayers GD, Dezern AE, Nazha A, Valent P, van de Loosdrecht AA, Onida F, Pleyer L, Cirici BX, Tibes R, Geissler K, Komrokji RS, Zhang J, Germing U, Steensma DP, Wiseman DH, Pfeilstoecker M, Elena C, Cross NCP, Kiladjian JJ, Luebbert M, Mesa RA, Montalban-Bravo G, Sanz GF, Platzbecker U, Patnaik MM, Padron E, Santini V, Fenaux P, Savona MR; MDS/MPN International Working Group. The ABNL-MARRO 001 study: a phase 1-2 study of randomly allocated active myeloid target compound combinations in MDS/MPN overlap syndromes. BMC Cancer. 2022 Sep 24;22(1):1013. doi: 10.1186/s12885-022-10073-w.
PubMed 36153475
Study at a glance
From the registry record, last updated by the sponsor on 29 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The ABNL-MARRO 001 Study: A Phase 1/2 Study of Active Myeloid Target Compound Combinations in MDS/MPN Overlap Syndromes.
- ClinicalTrials.gov ID
- NCT04061421
- Acronym / sponsor ID
- ABNL-MARRO / VICC HEMP 1977
- Conditions
- MDS/MPN
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 94 participants (estimated)
- Lead sponsor
- Michael SavonaUniversity, hospital or other
- Collaborators
- Incyte Corporation, Theradex, Astex Pharmaceuticals, Inc.
- Study start
- 24 Nov 2021 (actual)
- Primary completion
- Aug 2027 (estimated)
- Study completion
- Dec 2027 (estimated)
- First posted
- 19 Aug 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
ABNL-MARRO (A Basket study of Novel therapy for untreated MDS/MPN and Relapsed/Refractory Overlap Syndromes) is an international European-American cooperation providing the framework for collaborative studies to advance treatment of myelodysplastic/myeloproliferative neoplasms (MDS/MPN) and explore clinical-pathologic markers of disease severity, prognosis and treatment response.
ABNL MARRO 001 (AM-001) is an Open label, phase 1/2 study within the framework of the ABNL-MARRO that will test novel treatment combinations in MDS/MPN. Each Arm of AM-001 will test an active myeloid target compound in combination with ASTX727, an oral drug combining fixed doses of the DNA methyltransferase inhibitor (DNMTi) decitabine and the cytidine deaminase inhibitor E7727, also known as cedazuridine in a single tablet.
Detailed description
Current Arm: ASTX727 + ruxolitinib (JAK1/JAK2 inhibitor)
Primary Objective Phase 2:
- To test whether the overall response to each novel ASTX727 combination therapy in MDS/MPN patients is sufficiently high to warrant further investigation in more definitive trials.
Secondary Objectives:
- To expand the safety analysis of each treatment combination in MDS/MPN patients.
- To assess the morphologic bone marrow response in MDS/MPN patients treated on each Arm of the study.
- To estimate the effect of each treatment combination on patient survival
- To test the applicability of the proposed MDS/MPN IWG response criteria across multiple Arms of this study.
Tertiary/Exploratory objectives:
- To investigate genetic biomarkers of response in MDS/MPN.
- To characterize molecular responses to individual treatments.
- To evaluate synergistic effects of hypomethylation by ASTX727 and specific pathway blockade by study compounds.
- To explore the use of automated quantification of spleen volume from CT exams as a measure of clinical benefit
- To test and/or validate diagnostic algorithms and prognostic indices for MDS/MPN patients
- To investigate the correlation of patient reported outcomes with disease severity and/or treatment response
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ASTX727 + itacitinib | Experimental | Drug: ASTX727, Drug: ItacitinibASTX727 and itacitinib will be taken by mouth |
| ASTX727 + ruxolitinib | Experimental | Drug: ASTX727, Drug: RuxolitinibASTX727 and ruxolitinib will be taken by mouth |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- Must be ≥ 18 years of age at the time of signing the Informed Consent Form (ICF); must voluntarily sign an ICF; and must be willing and able to meet all study requirements.
- Must have morphologically confirmed diagnosis of MDS/MPN, excluding JMML, in accordance with WHO (2016) diagnostic criteria (Appendix 1, Section 12.1).
- Treatment-naïve patients (patients who have had no prior disease-modifying therapy) may enroll in any AM-001 Arm that is open to accrual in phase 1 or phase 2. Treatment-naïve patients may have received recombinant erythropoietin, danazol, hydroxyurea or anagrelide, which are not considered to be disease-modifying therapy for the purpose of this study.
- After an appropriate wash-out period, patients who have failed (or were intolerant to) prior therapy with a regimen(s) containing a DNMTi may enroll in any Arm in phase 1b or any Arm which has met the criterion of the first Simon's Stage and are open to accrual in the second Simon's Stage in phase 2 (Error! Reference source not found.). Except in the first stage of the phase 2, there are no limits on number of prior therapies if the patient meets all other eligibility criteria. Previously treated patients include:
- Must be willing to undergo bone marrow biopsy with aspiration during screening and bone marrow aspiration with tissue collection for disease assessment and correlative studies periodically throughout the trial.
Key exclusion criteria (22 listed)
- Patients should be excluded from any treatment Arm that includes a novel targeted agent to which they have had previous exposure. Novel targeted agents in this study currently include itacitinib (INCB039110) and ruxolitinib (RUX) only, currently. Patients who have had prior exposure to ASTX727 therapy are not excluded, provided they meet all other eligibility criteria.
- Prior receipt of any investigational study drug, including treatment on any prior AM-001 Arm, within 30 days or 5 half-lives (whichever is shorter) before receiving the first dose of study drug in an Arm of AM-001, except if approved by the medical monitor.
- Prior receipt of any systemic antineoplastic therapy, including but not limited to prior DNMTi therapy, standard induction or cytotoxic chemotherapy (excluding hydroxyurea), or approved targeted agent within 21 days or 5 half-lives (whichever is shorter) before receiving the first dose of study drug in an Arm of AM-001.
- Known hypersensitivity to decitabine and ruxolitinib.
- Transformation to acute myeloid leukemia (e.g., >20% myeloid blasts in bone marrow or >20% circulating blasts in peripheral blood).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ASTX727Also: Fixed dose combination of cedazuridine (100mg) + oral decitabine (35mg)Taken by mouth daily during days 1-5 of every 28-day cycle. | Drug | ASTX727 + itacitinib, ASTX727 + ruxolitinib |
| ItacitinibAlso: INCB039110Taken by mouth daily during each 28-day cycle | Drug | ASTX727 + itacitinib |
| RuxolitinibAlso: Jakafi for oralDosage will be 5,15, or 20mg Taken by mouth BID for 28-day cycle | Drug | ASTX727 + ruxolitinib |
Primary outcome
Phase 1: Determination of dose
Time frame: Up to 28 days
Per CTCAE 5.0
Phase 2: Overall response rate (ORR). Overall response includes patients who achieve best response of CR, PR, MR, or CB as defined by the MDS/MPN IWG proposed response criteria
Time frame: Up to 2 years
4 secondary outcomes
Adverse Event/DLT severity and frequency
Time frame: Up to 2 years
CR/MR rate defined as the proportion of patients who achieve either complete remission or marrow response as a best response to treatment.
Time frame: Up to 2 years
Overall survival (OS)
Time frame: Up to 2 years
Progression free survival (PFS)
Time frame: Up to 2 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Oct 2026.
- Background: PubMed 36153475Moyo TK, Mendler JH, Itzykson R, Kishtagari A, Solary E, Seegmiller AC, Gerds AT, Ayers GD, Dezern AE, Nazha A, Valent P, van de Loosdrecht AA, Onida F, Pleyer L, Cirici BX, Tibes R, Geissler K, Komrokji RS, Zhang J, Germing U, Steensma DP, Wiseman DH, Pfeilstoecker M, Elena C, Cross NCP, Kiladjian JJ, Luebbert M, Mesa RA, Montalban-Bravo G, Sanz GF, Platzbecker U, Patnaik MM, Padron E, Santini V, Fenaux P, Savona MR; MDS/MPN International Working Group. The ABNL-MARRO 001 study: a phase 1-2 study of randomly allocated active myeloid target compound combinations in MDS/MPN overlap syndromes. BMC Cancer. 2022 Sep 24;22(1):1013. doi: 10.1186/s12885-022-10073-w.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Michael Savona, MD study chair
Vanderbilt-Ingram Cancer Center
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ASTX727, Itacitinib and Ruxolitinib in people with MDS/MPN, run by Michael Savona at 4 sites in United States.
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 16 inclusion and 22 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: looking for participants now, as of the sponsor's last update on 29 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 Theradex Oncology US Inquiry (609.799.7580, info@theradex.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Phase 1: Determination of dose (time frame: Up to 28 days). 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 NCT04061421, which also lists 1 linked publication.
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 29 Oct 2026; AzTrials last refreshed its copy on 6 Nov 2026.