AzTrials
AzTrials

Clonal Cytopenia of Undetermined SignificanceEarly Phase 1NCT06802146

Early Intervention in High Risk CCUS

A Early Phase 1 study testing Inqovi in people with Clonal Cytopenia of Undetermined Significance and Cytopenia, run by Lachelle D. Weeks, MD, PhD at 1 site in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

This research is being done to find out more about the potential risks and benefits of early treatment in participants with high risk Clonal Cytopenia of Unknown Significance (CCUS). This study will give eligible CCUS participants the option of either being observed or taking an oral drug as treatment. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Inqovi
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
108 participants
Where
1 siteUnited States
Study dates
7 Feb 2025to 1 Dec 2028
Phase
Early Phase 1
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

  • Age ≥18 years.
  • Unexplained cytopenia(s) for at least 4 months (at least two separate labs within 4 months including at time of screening must meet this criteria). Cytopenia(s) defined as the presence of ≥ 1 of the following:
  • Hemoglobin (Hgb) <12 g/dL for women and <13g/dL for men
  • Absolute neutrophil count (ANC) < 1.8 × 109/L*
  • Platelet count (Plt) <150 × 109/L *Patients known to have a Duffy-null genotype must have anemia (Hgb < 12g/dL for women, Hgb <13g/dL for men) and/or thrombocytopenia (Plt < 150 × 109/L) to be eligible for this study.
  • 1 pathogenic variant detected in any myeloid driver gene with a VAF of at least 0.02 (2%) identified by local next generation sequencing (NGS) of peripheral blood or bone marrow sample within 3 months from screening bone marrow biopsy.
Show all 15Show fewer
  • Participants must have a high risk score per the Clonal Hematopoiesis Risk Calculator (CHRS). See APPENDIX C for calculation.
  • Screening bone marrow biopsy must not be diagnostic of any overt hematologic malignancy by morphologic assessment and must be consistent with a diagnosis of clonal cytopenia of unknown significance (CCUS) as determined by multi-institutional hematopathology review.
  • ECOG performance status 0-2 (see Appendix A).
  • Participants must meet the following organ function as defined below:
  • Serum aspartate aminotransferase/serum glutamic oxaloacetic transaminase (AST/SGOT) and alanine aminotransferase (ALT/SGPT) ≤3x upper limit of normal (ULN).
  • Serum total bilirubin <1.5x ULN. Higher levels are acceptable if these can be attributed to ineffective erythropoiesis or Gilbert's syndrome. In these cases, approval from the study Sponsor-Investigator is required.
  • Creatinine clearance greater than 40 mL/min based on the Cockcroft-Gault glomerular filtration rate estimation.
  • Ability to understand and the willingness to sign a written informed consent document.
  • For participants of the early pharmacologic intervention cohort: women of childbearing potential must use highly effective contraception during treatment for at least 6 months after the last dose and males with female partners of reproductive potential should use effective contraception during treatment and for 3 months after the last dose.

Who cannot join

  • Concurrent primary malignancy requiring active cytotoxic chemotherapy and/or ionizing radiation therapy.
  • Known inherited bone marrow failure disorder and/or germline predisposition to hematologic malignancy.
  • Receipt of anti-cancer therapy including any cytotoxic chemotherapy, ionizing radiation therapy, immunomodulatory agents such as lenalidomide, and targeted anti-cancer therapies including PARP inhibitors within the last 6 months. Patients with complete surgical resection of a tumor are not excluded from this study.
  • Anti-cancer therapy, including any cytotoxic chemotherapy, ionizing radiation therapy, immunomodulatory agents such as lenalidomide and targeted agents such as PARP inhibitors, planned in the next 6 months. Patients on hormonal adjuvant therapy for nonmetastatic breast and prostate cancer or other minimally-myelosuppressive maintenance therapies for non-metastatic cancer may be eligible at the discretion of the study PI.
  • Diagnosis of MDS, MPN, CMML, AML or any other hematolymphoid malignancy in the patient's lifetime. This includes individuals with MDS-defining chromosomal abnormalities identified via conventional karyotype or FISH.
  • Presence of a concurrent hematologic malignancy precursor state, such as smoldering multiple myeloma (SMM), and smoldering Waldenstrom's macroglobulinemia.
Show all 12Show fewer
  • Presence of an early-stage hematologic precursor state-such as monoclonal gammopathy of undetermined significance (MGUS) and monoclonal B cell lymphocytosis (MBL).
  • Active uncontrolled systemic fungal, bacterial, or viral infection (defined as ongoing signs/symptoms related to the infection without improvement despite appropriate antibiotics, antiviral therapy, and/or other treatment).
  • Recent (within 3 months) vaccination with any live attenuated vaccine or vaccination with live attenuated vaccine planned during the next 15 months. *Live attenuated vaccines include measles, mumps, rubella (MMR combined vaccine), rotavirus, smallpox, chickenpox, and yellow fever.
  • Laboratory evidence indicative of clinically significant red cell hemolysis.
  • Hypersplenism and/or evidence of portal hypertension on physical exam or imaging.
  • Pregnant or lactating.

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

United States1 site, 1 recruiting
  • Dana-Farber Cancer Institute

    Boston, Massachusetts, 02215

    Lachelle Weeks, MD, 617-632-3779, Lachelle_Weeks@DFCI.HARVARD.EDU

    Lachelle Weeks, MD

    Recruiting

Other trials in United States

About this study

Official title: A Multi-Site Break Through Cancer Pilot Study Testing the Feasibility and Safety of Therapeutic Intervention for Patients With High-risk Clonal Cytopenia of Undetermined Significance (CCUS)

This research is being done to find out more about the potential risks and benefits of early treatment in participants with high risk Clonal Cytopenia of Unknown Significance (CCUS). This study will give eligible CCUS participants the option of either being observed or taking an oral drug as treatment.

The names of the study drug involved in this study is:

-Decitabine/cedazuridine (DEC/CED) (a nucleoside metabolic inhibitor and cytidine deaminase inhibitor).

Read the detailed description

This is an open-label, multicenter pilot study testing the feasibility and safety of early pharmacologic intervention, decitabine/cedazuridine, in participants with higher-risk clonal cytopenia of unknown significance (CCUS).

The U.S. Food and Drug Administration (FDA) has not approved DEC/CED for CCUS but it has been approved for other uses.

The research study procedures include screening for eligibility, in-clinic treatment visits, electrocardiograms, echocardiograms, bone marrow biopsies, and blood tests.

Participants who choose to enroll in the intervention cohort will receive the oral drug for 1 year and will continue in a post-treatment observation period for 2 years after treatment. Participants in both groups will each participate in the study for 3 years total.

It is expected that the study will continue to enroll up to 108 participants in total or until there are 30 participants enrolled in the early intervention cohort, whichever occurs first.

Astex Oncology is funding this research study by providing the drug Decitabine/cedazuridine.

Being tested
Inqovi
Keywords
Clonal Cytopenia of Undetermined Significance, CCUS, Cytopenia
Medical subject headings
Cytopenia

What participants receive

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

  • InqoviDrug

    Also called Decitabine and Cedazuridine, ASTX727

    Combination of a nucleoside metabolic inhibitor and cytidine deaminase inhibitor, 10mg DEC / 100mg CED tablet, taken orally per protocol.

Study groups in NCT06802146
Study groupRoleWhat they receive
Inqovi CohortExperimentalParticipants will be enrolled and will complete: * Baseline visit. * In-clinic visits with assessments: Cycle 1 days 8, 15, and 22. * In-clinic visits with assessments and bone marrow biopsies: Cycles 2 - 12 Day 1. * Cycle 1 - 12: • Days 1 - 5 of 28 day cycle: Predetermined dose of Inqovi 1x daily. * End of Treatment visit, Cycle 13 Day 1, with assessments and bone marrow biopsy. * Follow up in-clinic visits with assessments: Cycle 19 Day 21, Cycle 25 Day 1, Cycle 31 Day 1, and Cycle 36 Day 28. Bone marrow biopsy at Cycle 25 Day 1. * End of Study visit, Cycle 36 Day 28, with assessments and bone marrow biopsy.
Receives: Drug: Inqovi
Observational CohortNo interventionParticipants will complete: * Baseline visit. * In-clinic visits with assessments: Cycle 7 Day 1, Cycle 13 Day, Cycle 19 Day, Cycle 25 Day 1, and Cycle 31 Day 1. * Bone marrow biopsies every 12 months at Cycle 13 Day 1 and Cycle 25 Day. * End of study visit, Cycle 36 Day 28, with assessments and bone marrow biopsy.

What is being measured

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

  1. Feasibility Failure Rate (FFR)

    FFR is defined by the proportion of eligible subjects with higher risk CCUS who enter the study and opt to participate in the early intervention as opposed to observation only cohort.

    Measured: Treatment duration up to 12 cycles (28 days per cycle).

Secondary outcomes (8)
  1. Grade 3-5 Treatment-related Toxicity Rate

    All grade 3-5 adverse events (AE) with treatment attribution of possibly, probably or definite based on CTCAEv5 that are not resolved in accordance with treatment guidelines were counted. Rate is the percentage of treated participants experiencing at least one of these adverse events as defined during the time of observation.

    Measured: AE evaluated on treatment at day 1, 8, 15, 22 on cycle 1, then day 1 on cycle 2-12, and EOT. Median treatment duration for this study cohort was 6 months (range T1- T2).

  2. Treatment Tolerability Rate (TTR)

    TTR will be measured by the percentage of participants in the early intervention treatment arm that complete ≥3 cycles of oral DEC/CED.

    Measured: Treatment up to 12 cycles, (28 days per cycle).

  3. Hematologic Response Rate (HRR)

    HRR defined as proportion of participants achieved hematologic response by 2018 World Health Organization (WHO) International Working Group (IWG) MDS response criteria.

    Measured: Response collected every 3 months on treatment. Treatment duration up to 12 cycles, (28 days per cycle).

  4. Median Overt Myeloid Neoplasia (MN)-Free Survival

    MN-free survival based on the Kaplan-Meier method is defined as the duration of time from study entry to overt myeloid neoplasia diagnosed per the 5th edition of the WHO Classification of Haematolymphoid Tumors.

    Measured: Disease evaluated every 3 months on treatment, then every 12 months off treatment, up to 2 years for interventional cohort, up to 3 years for observation cohort.

  5. Somatic Driver Mutation Variant Allele Frequencies (VAFs) Reduction Rate

    Changes in variant allele fraction (VAF) will be assessed as a biomarker for clone size. A clinically significant VAF reduction will be defined as a persistent (evident on 2 sequential measurements 3 or more months apart) VAF reduction of the pathogenic mutation in question of ≥10%, or for initial VAFs <10%, if the mutation becomes undetectable. Rate defines as proportion of participants achieved the VAFs reduction.

    Measured: Clinical next generation sequencing (NGS) is collected every 3 month on treatment, for up to 12 cycles, (28 days per cycle).

  6. Changes in Serum Inflammatory Markers

    Serum Inflammatory Markers measured using established method. Change will be defined as percentage increase/decrease from baseline measurement at screening.

    Measured: High sensitivity CRP will be measured at screening, every 6 months, and at end of study/disease progression, up to 3 years.

  7. Change in Score of the Adult Comorbidity Evaluation 27 (ACE-27)

    Adult Comorbidity Evaluation-27 identify the important medical comorbidities and grade severity using the index. Overall Comorbidity Score is defined according to the highest ranked single ailment, except in the case where two or more Grade 2 ailments occur in different organ systems. In this situation, the overall comorbidity score should be designated Grade 3. Overall score range from 0-None to 3-Serve, and noted 9-Unknown.

    Measured: Survey is evaluated at screening, every 6 months, and at end of study/disease progression, up to 3 years.

  8. Change in Score of the 36-item Short Form Survey (SF-36)

    The Short Form (SF)-36 is a 36-item participant-reported questionnaire that covers eight health domains. Scores for each domain range from 0 to 100%. To score the SF-36, scales are standardized with a scoring algorithm or by SF-36 scoring software to obtain a score ranging from 0 to 100. Higher scores indicate better health status, and a mean score of 50 has been articulated as a normative value for all scales.

    Measured: Survey is evaluated at screening, every 6 months, and at end of study/disease progression, up to 3 years.

Timeline

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

  1. First posted31 Jan 2025
  2. Study start7 Feb 2025
  3. Primary completion1 Dec 2026 (estimated)
  4. Study completion1 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
Early Phase 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
108 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06802146
Sponsor's protocol ID
23-658
Status last verified
Apr 2026
First submitted
27 Jan 2025
Last updated
23 Apr 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: The Dana-Farber / Harvard Cancer Center encourages and supports the responsible and ethical sharing of data from clinical trials. De-identified participant data from the final research dataset used in the published manuscript may only be shared under the terms of a Data Use Agreement. Requests may be directed to: \[contact information for Sponsor Investigator or designee\]. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.

Questions about this trial

What is this clinical trial studying?

A Early Phase 1 study testing Inqovi in people with Clonal Cytopenia of Undetermined Significance and Cytopenia, run by Lachelle D. Weeks, MD, PhD at 1 site 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 15 inclusion and 12 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 23 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site 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 phase mean?

Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.

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 Lachelle Weeks, MD (617-632-3779, Lachelle_Weeks@DFCI.HARVARD.EDU), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Feasibility Failure Rate (FFR) (time frame: Treatment duration up to 12 cycles (28 days per cycle).). It is what the study was designed to measure, not a finding.

Where can I read the original study information?
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 23 Apr 2026.

Free account

Keep searching with a free account

You’ve used your 3 free searches for this session. Log in or create a free AzTrials account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in