Trials Sponsors Conditions Drugs Pricing

Conditions

Low Risk Myelodysplastic Syndromes

Keywords

MDSLR MDSMyelodysplastic SyndromesHematology OncologyHem/ Onc

Outcome Measures

Primary Outcomes (1)

Safety and Tolerability
* Incidence of adverse events (AEs) * Incidence of discontinuation or interruptions of R289 due to AEs * Incidence of dose limiting toxicities (DLTs)
2 Year

Secondary Outcomes (2)

Outcome Measure: Preliminary Efficacy
24 Weeks for Primary Efficacy; 8 Weeks for Characterized PK
Characterize pharmacokinetics (PK)
8 Weeks

Other Outcomes (1)

Characterize pharmacodynamic (PD)
1 year

Study Arms (1)

Experimental

EXPERIMENTAL

ESCALATION PHASE: Dose Level 1: 250mg PO qd Dose Level 2: 500mg PO qd Dose Level 3: 750 mg PO qd Dose Level 4: 250 mg PO bid Dose Level 5: 500 mg PO AM/250 mg PO PM Dose Level 6: 500 mg PO bid EXPANSION PHASE (randomized 1:1): Dose Level 1: 250 mg PO qd Dose Level 2: 250 mg PO bid

Drug: R906289 Monosodium (R289 Na)

Interventions

R906289 Monosodium (R289 Na)Drug

Drug: R906289 Monosodium (R289 Na) R906289 Monosodium (250mg PO qd, 250mg PO bid, 500 mg PO qd, 500 mg PO bid, 750 mg PO qd, split dose - 500 mg PO AM/250 mg PO PM)

Also known as: R906289 Monosodium

Experimental

Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.

Eligibility Criteria

Age18 Years+

Sexall

Healthy VolunteersNo

Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

Patient must be ≥ 18 years of age at the time of signing the informed consent.
Must have definitive diagnosis of MDS with very low, low, or intermediate-1 risk (International Prognostic Scoring System (IPSS)-R ≤ 3.5) and ≤5% bone marrow myeloblasts.

Sponsors & Collaborators

Rigel Pharmaceuticals
Industrylead

Study Sites (15)

University of California, Los Angeles

Los Angeles, California, 90095, United States

RECRUITING

University of California, Irvine

Orange, California, 92868, United States

RECRUITING

Stanford Cancer Institute

Palo Alto, California, 94304, United States

RECRUITING

University of Miami

MeSH Terms

Conditions

Myelodysplastic Syndromes

Condition Hierarchy (Ancestors)

Bone Marrow DiseasesHematologic DiseasesHemic and Lymphatic Diseases

Central Study Contacts

Strait Hicklin

CONTACT

(650) 624-1100 shicklin@rigel.com

Study Design

Study Type: interventional
Phase: phase 1 2
Allocation: NA
Masking: NONE
Purpose: TREATMENT
Intervention Model: SEQUENTIAL
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

First Submitted

March 2, 2022

First Posted

April 4, 2022

Study Start

September 12, 2022

Actual (reported)

Primary Completion

August 2026

Estimated (date passed)

This estimate has passed. The registry has not confirmed an actual date for this milestone.

You may not qualify if:

Prior treatment for MDS (i.e., TPOs, EPOs, luspatercept, HMAs) concluded \< 4 weeks prior to study treatment
Clinically significant anemia resulting from iron, B12 or folate deficiencies, autoimmune or hereditary hemolysis, or GI bleeding.
MDS secondary to treatment with radiotherapy, chemotherapy, and/or immunotherapy for malignant or autoimmune diseases.
Diagnosis of chronic myelomonocytic leukemia.
History of uncontrolled seizures.
Uncontrolled bacterial or viral infection (i.e., documented HIV, hepatitis B or hepatitis C).
History of other malignancy that could affect compliance or interpretation of results. Patients with an malignancy other than leukemia appropriately treated with curative intent and the malignancy has been in remission without treatment for ≥ 2 years prior to study entry are eligible as are:
Adequately treated in situ carcinoma of the cervix uteri
Adequately treated basal cell carcinoma or localized squamous cell carcinoma of the skin, or
Low grade, early-stage prostate cancer (Gleason score 6 or below, stage 1 or 2) with no requirement for therapy at any time prior to the study, or previously resected.
History of or active, clinically significant, cardiovascular, respiratory, GI, renal, hepatic, neurological, psychiatric, musculoskeletal, genitourinary, dermatological, or other disorder that, in the Investigator's opinion (or following review by the Sponsor), could affect the conduct of the study or the absorption, metabolism or excretion of the study treatment.
Prior history of autologous or allogeneic stem cell transplantation
Marked baseline prolongation of QT/QTc interval (e.g., repeated demonstration of a QTc interval \> 480 milliseconds \[msec\]) (Common Terminology Criteria for Adverse Events \[CTCAE\] Grade 1) using Fridericia's QT correction formula.
History of additional risk factors for TdP (e.g., symptomatic heart failure with left ventricular ejection fraction \[LVEF\] \<40%, hypokalemia, family history of Long QT Syndrome).
Receiving any other concurrent chemotherapy, radiotherapy, or immunotherapy (within 2 weeks of initiating study treatment), or the toxicity of the relevant prior treatment has not been resolved yet. For any long-acting systemic agent such as a monoclonal antibody, study treatment should not begin within two half-lives of the agent.
+2 more criteria

Study of R289 in Patients With Lower-risk Myelodysplastic Syndromes (LR MDS)

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (2)

Other Outcomes (1)

Study Arms (1)

Experimental

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (15)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Central Study Contacts

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (2)

Other Outcomes (1)

Study Arms (1)

Experimental

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (15)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Central Study Contacts

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations