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Splicing Modulator

H3B-8800 for Myelodysplastic Syndrome (Encore-MDS Trial)

Phase 1
Waitlist Available
Research Sponsored by H3 Biomedicine Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Confirmed diagnosis of MDS, CMML, or AML.
For the MDS Expansion cohort, participants must be lower-risk MDS, defined as low or intermediate-1 risk categorization per International Prognostic Scoring System (IPSS) criteria that carries a missense SF3B1 mutation.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to approximately 50 months
Awards & highlights

Encore-MDS Trial Summary

This trial is testing a new drug, H3B-8800, for people with myelodysplastic syndromes, acute myeloid leukemia, and chronic myelomonocytic leukemia. The goal is to see if it is safe and to learn how it works in the body.

Who is the study for?
This trial is for adults with certain blood disorders like lower-risk Myelodysplastic Syndromes (MDS), Chronic Myelomonocytic Leukemia (CMML), or Acute Myeloid Leukemia (AML) who haven't responded well to other treatments. They should be able to perform daily activities with ease to moderate difficulty and have a specific mutation in their cells. People can't join if they're set for stem cell transplants, have acute promyelocytic leukemia, certain genetic abnormalities, or unstable eye diseases.Check my eligibility
What is being tested?
The study tests H3B-8800 (RVT-2001), a drug designed to modify how genes are read in cells of patients with MDS, AML, and CMML. It's an early-phase trial looking at the safety of the drug and how it affects the body when taken by people with these conditions.See study design
What are the potential side effects?
Since this is an early-stage trial for H3B-8800 (RVT-2001), detailed side effects aren't fully known yet. However, participants will be closely monitored for any adverse reactions that could include typical chemotherapy-related issues such as fatigue, nausea, or more serious complications.

Encore-MDS Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have been diagnosed with MDS, CMML, or AML.
Select...
My MDS is low-risk and has a specific SF3B1 mutation.

Encore-MDS Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to approximately 50 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to approximately 50 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of Participants with Dose-limiting Toxicities (DLTs)
Number of Participants with Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Secondary outcome measures
Area Under the Plasma Concentration-time Curve From Time 0 Through the Last Measurable Point (AUC0-t)
Duration of Response (DOR)
Maximum Observed Plasma Concentration (Cmax)
+7 more

Encore-MDS Trial Design

3Treatment groups
Experimental Treatment
Group I: H3B-8800 (RVT-2001) MDS ExpansionExperimental Treatment1 Intervention
Transfusion-dependent, lower-risk MDS subjects (very-low to intermediate risk categorization per IPSS-R) with missense mutations in SF3B1.
Group II: H3B-8800 (RVT-2001) Dose OptimizationExperimental Treatment1 Intervention
Transfusion-dependent, lower-risk MDS subjects (very-low to intermediate risk categorization per IPSS-R) with missense mutations in SF3B1, and who have not been exposed to HMA's or lenalidomide in a prior line of therapy.
Group III: H3B-8800 (RVT-2001) Dose EscalationExperimental Treatment1 Intervention
H3B-8800 Acute Myeloid Leukemia or High Risk Myelodysplastic Syndromes/ Low Risk Myelodysplastic Syndromes/ Chronic Myelomonocytic Leukemia.

Find a Location

Who is running the clinical trial?

H3 Biomedicine Inc.Lead Sponsor
6 Previous Clinical Trials
406 Total Patients Enrolled
Hemavant Sciences GmbHLead Sponsor
Keisuke Kuida, MD, PhDStudy DirectorHemavant Sciences

Media Library

H3B-8800 (RVT-2001) (Splicing Modulator) Clinical Trial Eligibility Overview. Trial Name: NCT02841540 — Phase 1
Acute Myeloid Leukemia Research Study Groups: H3B-8800 (RVT-2001) Dose Escalation, H3B-8800 (RVT-2001) MDS Expansion, H3B-8800 (RVT-2001) Dose Optimization
Acute Myeloid Leukemia Clinical Trial 2023: H3B-8800 (RVT-2001) Highlights & Side Effects. Trial Name: NCT02841540 — Phase 1
H3B-8800 (RVT-2001) (Splicing Modulator) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02841540 — Phase 1

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What is the approximate participant threshold for this research study?

"This study necessitates 200 participants who meet the prespecified inclusion criteria. Hemavant Sciences GmbH are spearheading this trial from two locations: Massachusetts General Hospital in Detroit, Michigan and Karmanos Cancer Institute in Miami, Florida."

Answered by AI

Are there any additional investigations involving H3B-8800?

"Currently, research into H3B-8800 is present in the form of 1 trial globally. None of these are currently in Phase 3 trials. The majority of locations conducting this study are based around Paris and Navarra with a total 27 sites running clinical assessments for this drug."

Answered by AI

Is this a pioneering research study?

"The only extant trial for H3B-8800 spans 4 nations and 21 cities. This investigation, funded by Hemavant Sciences GmbH, began in 2016 with 200 patients being enrolled; it has since moved onto Phase 1 drug approval. No additional trials have been launched since the first one was concluded."

Answered by AI

What potential hazards has H3B-8800 been associated with?

"Our team at Power has rated H3B-8800 as a 1 out of 3 for safety due to the fact that this Phase 1 trial only possesses limited evidence regarding efficacy and security."

Answered by AI

Are there vacancies available in this experimental trial?

"Yes, that is correct. Per the information found on clinicaltrials.gov, this medical trial which was originally posted on October 6th 2016 is currently searching for participants. The study requires 200 patients to be recruited from 7 different sites."

Answered by AI

What number of hospitals in this locale are running the experiment?

"Several medical establishments are currently involved in this trial, such as Massachusetts General Hospital located in Detroit, the Karmanos Cancer Institute based in Miami and University of Miami situated in Rochester. Additionally 4 other sites have been included."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
A Study of H3B-8800 (RVT-2001) in Participants With Lower Risk Myelodysplastic Syndromes
2016. "A Study of H3B-8800 (RVT-2001) in Participants With Lower Risk Myelodysplastic Syndromes". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02841540.
~15 spots leftby Apr 2025
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