Trial Outcomes & Findings for Luspatercept With or Without Hydroxyurea for the Treatment of Myelodysplastic/Myeloproliferative Neoplasms With Ring Sideroblasts and Thrombocytosis or Unclassifiable With Ring Sideroblasts (NCT NCT05005182)
NCT ID: NCT05005182
Last Updated: 2024-09-19
Results Overview
Defined as the proportion of patients who achieve an erythroid response out of the total number of evaluable patients (i.e. eligible patients who received at least one dose of treatment on study).
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
3 participants
Primary outcome timeframe
8 weeks
Results posted on
2024-09-19
Participant Flow
Participant milestones
| Measure |
Cohort A (Luspatercept)
Patients receive luspatercept SC on day 1. Cycles repeat every 21 days for 6 months in the absence of disease progression or unacceptable toxicity.
|
Cohort B (Luspatercept, Hydroxyurea)
Patients receive luspatercept SC on day 1 and hydroxyurea PO on days 1-21. Cycles repeat every 21 days for 6 months in the absence of disease progression or unacceptable toxicity.
|
|---|---|---|
|
Overall Study
STARTED
|
1
|
2
|
|
Overall Study
COMPLETED
|
1
|
2
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Luspatercept With or Without Hydroxyurea for the Treatment of Myelodysplastic/Myeloproliferative Neoplasms With Ring Sideroblasts and Thrombocytosis or Unclassifiable With Ring Sideroblasts
Baseline characteristics by cohort
| Measure |
All Patients
n=3 Participants
Cohort A (luspatercept) patients receive luspatercept SC on day 1. Cycles repeat every 21 days for 6 months in the absence of disease progression or unacceptable toxicity.\>
\> Cohort B (luspatercept, hydroxyurea) patients receive luspatercept SC on day 1 and hydroxyurea PO on days 1-21. Cycles repeat every 21 days for 6 months in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Age, Continuous
|
69 years
STANDARD_DEVIATION 6.08 • n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 8 weeksDefined as the proportion of patients who achieve an erythroid response out of the total number of evaluable patients (i.e. eligible patients who received at least one dose of treatment on study).
Outcome measures
| Measure |
Cohort A (Luspatercept)
n=1 Participants
Patients receive luspatercept SC on day 1. Cycles repeat every 21 days for 6 months in the absence of disease progression or unacceptable toxicity.
|
Cohort B (Luspatercept, Hydroxyurea)
n=2 Participants
Patients receive luspatercept SC on day 1 and hydroxyurea PO on days 1-21. Cycles repeat every 21 days for 6 months in the absence of disease progression or unacceptable toxicity.
|
|---|---|---|
|
Erythroid Response Rate
|
0 proportion of participants
|
0.5 proportion of participants
|
SECONDARY outcome
Timeframe: 3 months, 29 daysAEs as well as toxicities (AEs felt to be at least possibly related to study treatment) will be reported in the adverse events section of the report. The count of participants by cohort that were evaluated for address events is reported here.
Outcome measures
| Measure |
Cohort A (Luspatercept)
n=1 Participants
Patients receive luspatercept SC on day 1. Cycles repeat every 21 days for 6 months in the absence of disease progression or unacceptable toxicity.
|
Cohort B (Luspatercept, Hydroxyurea)
n=2 Participants
Patients receive luspatercept SC on day 1 and hydroxyurea PO on days 1-21. Cycles repeat every 21 days for 6 months in the absence of disease progression or unacceptable toxicity.
|
|---|---|---|
|
Incidence of Adverse Events (AEs)
|
1 Participants
|
2 Participants
|
SECONDARY outcome
Timeframe: 69 daysPopulation: All patients that achieved Erythroid response.
Time from erythroid response to progression or death.
Outcome measures
| Measure |
Cohort A (Luspatercept)
n=1 Participants
Patients receive luspatercept SC on day 1. Cycles repeat every 21 days for 6 months in the absence of disease progression or unacceptable toxicity.
|
Cohort B (Luspatercept, Hydroxyurea)
Patients receive luspatercept SC on day 1 and hydroxyurea PO on days 1-21. Cycles repeat every 21 days for 6 months in the absence of disease progression or unacceptable toxicity.
|
|---|---|---|
|
Duration of Response
|
69 Days
|
—
|
SECONDARY outcome
Timeframe: 6 monthsPopulation: No AML transformations were reported among the three patients on trial.
Will be evaluated and characterized within each cohort using the methods of Kaplan and Meier.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From time of treatment to progression to acute myeloid leukemia or death from any cause, assessed up to 6 monthsPopulation: No AML transformations were reported among the three patients on trial.
Will be evaluated and characterized within each cohort using the methods of Kaplan and Meier.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 117 117 days [was intended to be 'Up to 6 months' (duration of treatment), but study terminated early due to slow accrual]Will be evaluated and characterized within each cohort using the methods of Kaplan and Meier.
Outcome measures
| Measure |
Cohort A (Luspatercept)
n=1 Participants
Patients receive luspatercept SC on day 1. Cycles repeat every 21 days for 6 months in the absence of disease progression or unacceptable toxicity.
|
Cohort B (Luspatercept, Hydroxyurea)
n=2 Participants
Patients receive luspatercept SC on day 1 and hydroxyurea PO on days 1-21. Cycles repeat every 21 days for 6 months in the absence of disease progression or unacceptable toxicity.
|
|---|---|---|
|
Overall Survival
|
87.0 days
Not enough events or patients to calculate the confidence intervals
|
NA days
Not enough events to calculate the median or confidence intervals
|
Adverse Events
Cohort A (Luspatercept)
Serious events: 1 serious events
Other events: 1 other events
Deaths: 1 deaths
Cohort B (Luspatercept, Hydroxyurea)
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Cohort A (Luspatercept)
n=1 participants at risk
Patients receive luspatercept SC on day 1. Cycles repeat every 21 days for 6 months in the absence of disease progression or unacceptable toxicity.
|
Cohort B (Luspatercept, Hydroxyurea)
n=2 participants at risk
Patients receive luspatercept SC on day 1 and hydroxyurea PO on days 1-21. Cycles repeat every 21 days for 6 months in the absence of disease progression or unacceptable toxicity.
|
|---|---|---|
|
Infections and infestations
Lung infection
|
100.0%
1/1 • Number of events 1 • 3 months, 29 days
|
0.00%
0/2 • 3 months, 29 days
|
Other adverse events
| Measure |
Cohort A (Luspatercept)
n=1 participants at risk
Patients receive luspatercept SC on day 1. Cycles repeat every 21 days for 6 months in the absence of disease progression or unacceptable toxicity.
|
Cohort B (Luspatercept, Hydroxyurea)
n=2 participants at risk
Patients receive luspatercept SC on day 1 and hydroxyurea PO on days 1-21. Cycles repeat every 21 days for 6 months in the absence of disease progression or unacceptable toxicity.
|
|---|---|---|
|
Blood and lymphatic system disorders
Anemia
|
100.0%
1/1 • Number of events 2 • 3 months, 29 days
|
50.0%
1/2 • Number of events 2 • 3 months, 29 days
|
|
Blood and lymphatic system disorders
Blood and lymph sys disorders - Oth Spec
|
100.0%
1/1 • Number of events 1 • 3 months, 29 days
|
0.00%
0/2 • 3 months, 29 days
|
|
Cardiac disorders
Sinus tachycardia
|
100.0%
1/1 • Number of events 1 • 3 months, 29 days
|
0.00%
0/2 • 3 months, 29 days
|
|
Eye disorders
Eye disorders - Other, specify
|
100.0%
1/1 • Number of events 2 • 3 months, 29 days
|
0.00%
0/2 • 3 months, 29 days
|
|
Gastrointestinal disorders
Abdominal pain
|
100.0%
1/1 • Number of events 1 • 3 months, 29 days
|
0.00%
0/2 • 3 months, 29 days
|
|
Gastrointestinal disorders
Diarrhea
|
100.0%
1/1 • Number of events 1 • 3 months, 29 days
|
0.00%
0/2 • 3 months, 29 days
|
|
Gastrointestinal disorders
Nausea
|
100.0%
1/1 • Number of events 1 • 3 months, 29 days
|
0.00%
0/2 • 3 months, 29 days
|
|
General disorders
Fatigue
|
100.0%
1/1 • Number of events 2 • 3 months, 29 days
|
50.0%
1/2 • Number of events 1 • 3 months, 29 days
|
|
General disorders
Fever
|
100.0%
1/1 • Number of events 2 • 3 months, 29 days
|
0.00%
0/2 • 3 months, 29 days
|
|
General disorders
Flu like symptoms
|
100.0%
1/1 • Number of events 1 • 3 months, 29 days
|
0.00%
0/2 • 3 months, 29 days
|
|
Immune system disorders
Immune system disorders - Other, specify
|
100.0%
1/1 • Number of events 1 • 3 months, 29 days
|
0.00%
0/2 • 3 months, 29 days
|
|
Infections and infestations
Sepsis
|
100.0%
1/1 • Number of events 1 • 3 months, 29 days
|
0.00%
0/2 • 3 months, 29 days
|
|
Infections and infestations
Upper respiratory infection
|
100.0%
1/1 • Number of events 1 • 3 months, 29 days
|
0.00%
0/2 • 3 months, 29 days
|
|
Investigations
Blood bilirubin increased
|
100.0%
1/1 • Number of events 4 • 3 months, 29 days
|
0.00%
0/2 • 3 months, 29 days
|
|
Investigations
Neutrophil count decreased
|
100.0%
1/1 • Number of events 2 • 3 months, 29 days
|
0.00%
0/2 • 3 months, 29 days
|
|
Investigations
Platelet count decreased
|
100.0%
1/1 • Number of events 7 • 3 months, 29 days
|
0.00%
0/2 • 3 months, 29 days
|
|
Investigations
White blood cell decreased
|
100.0%
1/1 • Number of events 5 • 3 months, 29 days
|
0.00%
0/2 • 3 months, 29 days
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
100.0%
1/1 • Number of events 1 • 3 months, 29 days
|
0.00%
0/2 • 3 months, 29 days
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
100.0%
1/1 • Number of events 2 • 3 months, 29 days
|
0.00%
0/2 • 3 months, 29 days
|
|
Musculoskeletal and connective tissue disorders
Flank pain
|
100.0%
1/1 • Number of events 1 • 3 months, 29 days
|
0.00%
0/2 • 3 months, 29 days
|
|
Musculoskeletal and connective tissue disorders
Joint range of motion decr lumbar spine
|
100.0%
1/1 • Number of events 1 • 3 months, 29 days
|
0.00%
0/2 • 3 months, 29 days
|
|
Nervous system disorders
Dizziness
|
100.0%
1/1 • Number of events 2 • 3 months, 29 days
|
0.00%
0/2 • 3 months, 29 days
|
|
Nervous system disorders
Extrapyramidal disorder
|
0.00%
0/1 • 3 months, 29 days
|
50.0%
1/2 • Number of events 1 • 3 months, 29 days
|
|
Nervous system disorders
Paresthesia
|
0.00%
0/1 • 3 months, 29 days
|
50.0%
1/2 • Number of events 1 • 3 months, 29 days
|
|
Nervous system disorders
Presyncope
|
100.0%
1/1 • Number of events 2 • 3 months, 29 days
|
0.00%
0/2 • 3 months, 29 days
|
|
Renal and urinary disorders
Hematuria
|
100.0%
1/1 • Number of events 1 • 3 months, 29 days
|
0.00%
0/2 • 3 months, 29 days
|
|
Renal and urinary disorders
Renal and urinary disorders - Oth spec
|
100.0%
1/1 • Number of events 1 • 3 months, 29 days
|
0.00%
0/2 • 3 months, 29 days
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
100.0%
1/1 • Number of events 2 • 3 months, 29 days
|
0.00%
0/2 • 3 months, 29 days
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
100.0%
1/1 • Number of events 1 • 3 months, 29 days
|
0.00%
0/2 • 3 months, 29 days
|
|
Respiratory, thoracic and mediastinal disorders
Resp, thoracic, mediastinal - Oth spec
|
100.0%
1/1 • Number of events 1 • 3 months, 29 days
|
0.00%
0/2 • 3 months, 29 days
|
|
Vascular disorders
Arterial thromboembolism
|
0.00%
0/1 • 3 months, 29 days
|
50.0%
1/2 • Number of events 1 • 3 months, 29 days
|
|
Vascular disorders
Hypertension
|
100.0%
1/1 • Number of events 1 • 3 months, 29 days
|
0.00%
0/2 • 3 months, 29 days
|
|
Vascular disorders
Hypotension
|
100.0%
1/1 • Number of events 1 • 3 months, 29 days
|
0.00%
0/2 • 3 months, 29 days
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place