A Study of H3B-8800 (RVT-2001) in Participants With Lower Risk Myelodysplastic Syndromes (Encore-MDS)

  • End date
    Dec 1, 2024
  • participants needed
  • sponsor
    Hemavant Sciences GmbH
Updated on 4 April 2023
myeloid leukemia
myelodysplastic syndromes
chronic myelomonocytic leukemia
bone marrow procedure
induction chemotherapy
blood transfusion
myelomonocytic leukemia


A Phase 1, an Open-label, Multicenter Phase 1 Trial to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of Splicing Modulator H3B-8800 (RVT-2001) for Subjects With Myelodysplastic Syndromes, Acute Myeloid Leukemia, and Chronic Myelomonocytic Leukemia


This study is a Phase 1, open-label, first-in-human (FIH) study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary antitumor activity of H3B-8800 (RVT-2001) in subset of participants with Myelodysplastic Syndromes (MDS), Acute Myeloid Leukemia (AML), or Chronic Myelomonocytic Leukemia (CMML). The study consists of three parts, the dose escalation part (Part 1) exploring multiple once daily (QD) schedules and MDS Expansion part (Part 2) and Dose Optimization part (Part 3) exploring dosing schedules in lower-risk MDS.

Condition Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Leukemia, Myelomonocytic, Chronic
Treatment H3B-8800, H3B-8800 (RVT-2001)
Clinical Study IdentifierNCT02841540
SponsorHemavant Sciences GmbH
Last Modified on4 April 2023


Yes No Not Sure

Inclusion Criteria

Confirmed diagnosis of MDS, CMML, or AML
For the MDS Expansion cohort, participants must be lower-risk MDS, defined as
Participants with higher-risk MDS/CMML must be intolerant of hypomethylating agents (HMAs) or not have responded to 4 treatment cycles of decitabine or 6 treatment cycles of azacitidine, or must have progressed at any point after initiation of an HMA
low or intermediate-1 risk categorization per International Prognostic Scoring
System (IPSS) criteria that carries a missense SF3B1 mutation
For the Dose Optimization cohort, participants must be transfusion-dependent
lower-risk MDS, defined as very-low to intermediate risk categorization per
Eastern Cooperative Oncology Group (ECOG) performance score of 0-2
IPSS-R criteria that carries a missense SF3B1 mutation
Participants must meet the following criteria relevant to their specific diagnosis
Adequate baseline organ function
For the Dose Escalation portion, participants with lower-risk MDS/CMML must be transfusion-dependent for red blood cells or platelets
For the MDS expansion cohort, lower risk MDS participants must be RBC transfusion
dependent according to IWG 2006 criteria and must also have failed erythropoiesis
stimulating agents (ESA) or have serum erythropoietin (EPO) levels greater than (>)
units per liter (U/L)
C. For the Dose Optimization cohort, lower-risk MDS participants must be RBC
transfusion-dependent at baseline defined as ≥3 RBC units (concentrates) in 16-weeks
in at least 2 transfusion episodes prior to the first dose of H3B-8800 (RVT-2001) and
must also have failed ESA or have serum EPO levels > 500 U/L. Any ESA use should be
discontinued ≥6 weeks prior to enrollment
D. Participants with AML must either refuse or not be considered candidates for
intensive induction chemotherapy using consensus criteria for defining such
E. Participants with CMML must have been treated with at least one prior therapy
(hydroxyurea or a hypomethylating agent [HMA])
For MDS expansion and Dose optimization cohorts - absolute neutrophil count (ANC)
greater than or equal to (>=) 500/ microliter (mcL) (0.510^9/L)
For expansion and Dose optimization cohorts- platelet count >50,000/mcL (5010^9/L)
For Dose-optimization cohort: No prior HMA or lenalidomide in participants with
lower-risk MDS

Exclusion Criteria

Diagnosis of a core binding factor leukemia (t(8;21), t(16;16) or inv(16)). Diagnosis
of acute promyelocytic leukemia (t(15;17))
Participants are deemed candidate for hematopoietic stem cell transplants at the time
of enrollment (for AML participants only)
Known prior or current retinal or optic nerve disease (example, Retinitis Pigmentosa
diabetic retinopathy, optic neuritis) not stable for at least 6 months
History of clinically significant, uncorrected vitamin B12 or folate deficiency
Clear my responses

How to participate?

Step 1 Connect with a study center
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer  to help accelerate the development of new treatments and to get notified about similar trials.

You are contacting

Investigator Avatar

Primary Contact


Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

Learn more

If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

Learn more

Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

Learn more

Similar trials to consider


Browse trials for

Not finding what you're looking for?

Every year hundreds of thousands of volunteers step forward to participate in research. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.

Sign up as volunteer

user name

Added by • 



Reply by • Private

Lorem ipsum dolor sit amet consectetur, adipisicing elit. Ipsa vel nobis alias. Quae eveniet velit voluptate quo doloribus maxime et dicta in sequi, corporis quod. Ea, dolor eius? Dolore, vel!

  The passcode will expire in None.

No annotations made yet

Add a private note
  • abc Select a piece of text from the left.
  • Add notes visible only to you.
  • Send it to people through a passcode protected link.
Add a private note