A Pilot, Open-Label Study of Luspatercept for Patients With Lower Risk Myelodysplastic Syndromes (MDS)

March 1, 2024 updated by: M.D. Anderson Cancer Center
To learn if luspatercept is more effective in helping to reduce the number of blood transfusions needed by patients with LR-MDS.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

Objectives:

Primary Objectives:

  • To assess the clinical activity and safety of luspatercept in patients with transfusion independent lower risk MDS
  • To assess the clinical activity and safety of luspatercept in patients with transfusion dependent lower risk MDS
  • To study the effects on hematopoeisis of luspatercept in patients treated on this study

Secondary Objectives:

  • To assess the duration of response of patients with treated with luspatercept in this study
  • To assess the overall survival and time to transformation of patients with treated with luspatercept in this study
  • To assess transfusion free survival period in patients that were transfusion independent treated with luspatercept in this study
  • To measure trends in neutrophil and platelet counts in patients treated in this study

Study Type

Interventional

Enrollment (Estimated)

40

Phase

  • Early Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • Texas
      • Houston, Texas, United States, 77030
        • Recruiting
        • M D Anderson Cancer Center
        • Contact:
        • Principal Investigator:
          • Guillermo Garcia-Manero, M D

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Age ≥ 18 years; as MDS there is no significant experience with luspatercept in pediatric patients
  2. Cohorts #1 and #2: Diagnosis of MDS according to WHO 2023 criteria (5) and with low or int-1 risk by IPSS or a score of ≤ 3.5 by IPSS-R.(3, 22)
  3. Cohort #2: Patient defined as transfusion dependent by documentation of receiving at least 2 units of packed red blood cells (PRBCs) for a hemoglobin of less than 8.0 g/dL during an 8-week period prior to study enrollment.
  4. Cohort #1: Patients with symptomatic anemia that are transfusion independent defined as not requiring a transfusion for a hemoglobin of less than 8.0 g/dL during an 8-week period prior to study enrollment.
  5. MDS patients with either a platelet count of ≤100 K/uL and/or ANC of ≤1.8 K/uL
  6. Patient must have signed an informed consent and is willing to participate in the study.
  7. Adequate hepatic function with total bilirubin ≤3 x ULN, AST or ALT ≤3xULN.
  8. Serum creatinine clearance ≥40mL/min and no end/stage renal disease (using Cockcroft-Gault).
  9. ECOG performance status </=2.

Exclusion Criteria:

  1. Active infection not adequately responding to appropriate antibiotics.
  2. Prior treatment with luspatercept or sotarcetept
  3. Female patients who are pregnant or lactating.
  4. Patients with reproductive potential who are unwilling to following contraception requirements (including condom use for males with sexual partners, and for females: prescription oral contraceptives [birth control pills], contraceptive injections, intrauterine devices [IUD], double-barrier method [spermicidal jelly or foam with condoms or diaphragm], contraceptive patch, or surgical sterilization) throughout the study. Reproductive potential is defined as no previous surgical sterilization or females that are not post-menopausal for 12 months.
  5. Female patients with reproductive potential who do not have a negative urine or blood beta-human chorionic gonadotropin (beta HCG) pregnancy test at screening.
  6. History of an active malignancy within the past 2 years prior to study entry, with the exception of: a. Adequately treated in situ carcinoma of the cervix uteri b. Adequately treated basal cell carcinoma or localized squamous cell carcinoma of the skin or any other malignancy with a life expectancy of more than 2 years.
  7. Patients receiving any other concurrent investigational agent or chemotherapy, radiotherapy, or immunotherapy (within 14 days of initiating study treatment).

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Cohort 1
Participants with lower risk MDS patients who have symptomatic anemia that are transfusion independent (TI). Participants will be asked to come to the study clinic weekly for the first 3 weeks and then 1 time every 3 weeks after that to have tests and procedures (such as physical exams and blood draws). Luspatercept will be administered once every 3 weeks.
Drug: Luspatercept
Given by SC or (Injection)
Other Names:
  • ACE-536
Experimental: Cohort 2
Participants with lower risk MDS that are transfusion dependent (TD). Participants will be asked to come to the study clinic weekly for the first 3 weeks and then 1 time every 3 weeks after that to have tests and procedures (such as physical exams and blood draws). Luspatercept will be administered once every 3 weeks.
Drug: Luspatercept
Given by SC or (Injection)
Other Names:
  • ACE-536

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0
Time Frame: through study completion; an average 1 year.
through study completion; an average 1 year.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

M.D. Anderson Cancer Center

Investigators

  • Principal Investigator: Guillermo Garcia-Manero, M D, M.D. Anderson Cancer Center

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 1, 2024

Primary Completion (Estimated)

June 2, 2026

Study Completion (Estimated)

June 2, 2026

Study Registration Dates

First Submitted

October 27, 2023

First Submitted That Met QC Criteria

October 27, 2023

First Posted (Actual)

November 2, 2023

Study Record Updates

Last Update Posted (Estimated)

March 5, 2024

Last Update Submitted That Met QC Criteria

March 1, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2022-1080
  • NCI-2023-09312 (Other Identifier: NCI-CTRP Clinical Trials Registry)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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