Trial Outcomes & Findings for Belinostat in Treating Patients With Myelodysplastic Syndromes (NCT NCT00357162)
NCT ID: NCT00357162
Last Updated: 2014-05-20
Results Overview
Complete Response (CR) A CR is defined as a participant with bone marrow showing less than 5% myeloblasts with no evidence of dysplasia and with adequate peripheral blood counts for at least 2 months (hemoglobin \> 11 g/dl, neutrophils ≥ 1500/mm3, platelets ≥ 100,000/mm3) and with no blasts in the peripheral. Partial Response (PR) All the CR criteria except bone marrow blasts decreased by ≥ 50% over pretreatment, or a less advanced WHO classification than pretreatment. Hematologic Improvement (HI) A 2g/dl increase in hemoglobin for participants with \<11g/dl hemoglobin at pretreatment, or an increase of \>30,000/mm\^3 platelets for participants with \<100,000/mm\^3 at pretreatment, or a 100% increase in neutrophil counts for participants with \<1500/mm\^3 at pretreatment
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
21 participants
Primary outcome timeframe
12 weeks
Results posted on
2014-05-20
Participant Flow
Twenty-one patients were accrued, and all were eligible and\> evaluable.
Participant milestones
| Measure |
Treatment (Enzyme Inhibitor Therapy)
Patients receive belinostat IV over 30 minutes on days 1-5. Treatment repeats every 21 days for 4 courses in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Overall Study
STARTED
|
21
|
|
Overall Study
COMPLETED
|
21
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Belinostat in Treating Patients With Myelodysplastic Syndromes
Baseline characteristics by cohort
| Measure |
Treatment (Enzyme Inhibitor Therapy)
n=21 Participants
Patients receive belinostat IV over 30 minutes on days 1-5. Treatment repeats every 21 days for 4 courses in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Age, Continuous
|
67 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
17 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
21 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 12 weeksComplete Response (CR) A CR is defined as a participant with bone marrow showing less than 5% myeloblasts with no evidence of dysplasia and with adequate peripheral blood counts for at least 2 months (hemoglobin \> 11 g/dl, neutrophils ≥ 1500/mm3, platelets ≥ 100,000/mm3) and with no blasts in the peripheral. Partial Response (PR) All the CR criteria except bone marrow blasts decreased by ≥ 50% over pretreatment, or a less advanced WHO classification than pretreatment. Hematologic Improvement (HI) A 2g/dl increase in hemoglobin for participants with \<11g/dl hemoglobin at pretreatment, or an increase of \>30,000/mm\^3 platelets for participants with \<100,000/mm\^3 at pretreatment, or a 100% increase in neutrophil counts for participants with \<1500/mm\^3 at pretreatment
Outcome measures
| Measure |
Treatment (Enzyme Inhibitor Therapy)
n=21 Participants
Patients receive belinostat IV over 30 minutes on days 1-5. Treatment repeats every 21 days for 4 courses in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Number of Confirmed Responses (Complete Response, Partial Response, or Hematologic Improvement) Noted on 2 Consecutive Evaluations at Least 4 Weeks Apart
Confirmed Hematologic Improvement (HI)
|
1 participants
|
|
Number of Confirmed Responses (Complete Response, Partial Response, or Hematologic Improvement) Noted on 2 Consecutive Evaluations at Least 4 Weeks Apart
Confirmed Partial Response (PR)
|
0 participants
|
|
Number of Confirmed Responses (Complete Response, Partial Response, or Hematologic Improvement) Noted on 2 Consecutive Evaluations at Least 4 Weeks Apart
COnfirmed Complete Response (CR)
|
0 participants
|
SECONDARY outcome
Timeframe: Time from registration to the date of progression or last follow-up, assessed up to 3 yearsEstimated using the method of Kaplan-Meier.
Outcome measures
| Measure |
Treatment (Enzyme Inhibitor Therapy)
n=21 Participants
Patients receive belinostat IV over 30 minutes on days 1-5. Treatment repeats every 21 days for 4 courses in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Time to Progression
|
14.9 months
Interval 5.8 to 30.0
|
SECONDARY outcome
Timeframe: From date of registration to the date of last follow-up or death due to any cause, assessed up to 3 yearsEstimated using the method of Kaplan-Meier.
Outcome measures
| Measure |
Treatment (Enzyme Inhibitor Therapy)
n=13 Participants
Patients receive belinostat IV over 30 minutes on days 1-5. Treatment repeats every 21 days for 4 courses in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Overall Survival
|
17.9 months
Interval 13.2 to 30.0
|
SECONDARY outcome
Timeframe: From the date of documented response until the date of progression or last follow-up, assessed up to 3 yearsPopulation: One participant had a confirmed Hematologica Improvement. For patient confidentiality, we are not reporting response data.
Estimated using the method of Kaplan-Meier.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Prior to each course (every 21 days), and every 3 months for up to 3 years after completion of study treatmentGraded using the Common Terminology Criteria for Adverse Events (CTCAE) version 3.0. Reporting events deemed at least possibly related to study treatment.
Outcome measures
| Measure |
Treatment (Enzyme Inhibitor Therapy)
n=21 Participants
Patients receive belinostat IV over 30 minutes on days 1-5. Treatment repeats every 21 days for 4 courses in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Toxicity of Belinostat in Patients With Myelodysplastic Syndrome
Grade 3 Hematologic Adverse Event
|
13 participants
|
|
Toxicity of Belinostat in Patients With Myelodysplastic Syndrome
Grade 4 Hematologic Adverse Events
|
11 participants
|
|
Toxicity of Belinostat in Patients With Myelodysplastic Syndrome
Grade 3 Non-Hemeatologic Adverse Events
|
5 participants
|
|
Toxicity of Belinostat in Patients With Myelodysplastic Syndrome
Grade 4 Non-hematologic Adverse Events
|
1 participants
|
Adverse Events
Treatment (Enzyme Inhibitor Therapy)
Serious events: 10 serious events
Other events: 21 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Treatment (Enzyme Inhibitor Therapy)
n=21 participants at risk
Patients receive belinostat IV over 30 minutes on days 1-5. Treatment repeats every 21 days for 4 courses in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
4.8%
1/21 • Number of events 1
|
|
Blood and lymphatic system disorders
Hemoglobin decreased
|
9.5%
2/21 • Number of events 2
|
|
Gastrointestinal disorders
Constipation
|
4.8%
1/21 • Number of events 1
|
|
Gastrointestinal disorders
Diarrhea
|
9.5%
2/21 • Number of events 2
|
|
Gastrointestinal disorders
Dyspepsia
|
4.8%
1/21 • Number of events 1
|
|
Gastrointestinal disorders
Nausea
|
4.8%
1/21 • Number of events 1
|
|
Gastrointestinal disorders
Oral hemorrhage
|
4.8%
1/21 • Number of events 1
|
|
Gastrointestinal disorders
Rectal hemorrhage
|
4.8%
1/21 • Number of events 1
|
|
Gastrointestinal disorders
Vomiting
|
4.8%
1/21 • Number of events 1
|
|
General disorders
Disease progression
|
4.8%
1/21 • Number of events 1
|
|
General disorders
Fever
|
4.8%
1/21 • Number of events 1
|
|
Immune system disorders
Cytokine release syndrome
|
4.8%
1/21 • Number of events 1
|
|
Infections and infestations
Pneumonia
|
9.5%
2/21 • Number of events 2
|
|
Infections and infestations
Urinary tract infection
|
4.8%
1/21 • Number of events 1
|
|
Investigations
Creatinine increased
|
4.8%
1/21 • Number of events 1
|
|
Investigations
Leukocyte count decreased
|
14.3%
3/21 • Number of events 3
|
|
Investigations
Neutrophil count decreased
|
23.8%
5/21 • Number of events 6
|
|
Investigations
Platelet count decreased
|
19.0%
4/21 • Number of events 7
|
|
Nervous system disorders
Syncope
|
4.8%
1/21 • Number of events 1
|
|
Renal and urinary disorders
Renal failure
|
4.8%
1/21 • Number of events 1
|
Other adverse events
| Measure |
Treatment (Enzyme Inhibitor Therapy)
n=21 participants at risk
Patients receive belinostat IV over 30 minutes on days 1-5. Treatment repeats every 21 days for 4 courses in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Blood and lymphatic system disorders
Hemoglobin decreased
|
95.2%
20/21 • Number of events 100
|
|
Cardiac disorders
Left ventricular failure
|
4.8%
1/21 • Number of events 1
|
|
Cardiac disorders
Palpitations
|
4.8%
1/21 • Number of events 1
|
|
Eye disorders
Vision blurred
|
4.8%
1/21 • Number of events 1
|
|
Gastrointestinal disorders
Constipation
|
33.3%
7/21 • Number of events 15
|
|
Gastrointestinal disorders
Diarrhea
|
28.6%
6/21 • Number of events 18
|
|
Gastrointestinal disorders
Mucositis oral
|
4.8%
1/21 • Number of events 1
|
|
Gastrointestinal disorders
Nausea
|
81.0%
17/21 • Number of events 34
|
|
Gastrointestinal disorders
Vomiting
|
33.3%
7/21 • Number of events 10
|
|
General disorders
Edema limbs
|
14.3%
3/21 • Number of events 5
|
|
General disorders
Fatigue
|
90.5%
19/21 • Number of events 64
|
|
General disorders
Fever
|
4.8%
1/21 • Number of events 1
|
|
General disorders
Injection site reaction
|
9.5%
2/21 • Number of events 5
|
|
Immune system disorders
Cytokine release syndrome
|
4.8%
1/21 • Number of events 1
|
|
Immune system disorders
Hypersensitivity
|
9.5%
2/21 • Number of events 3
|
|
Infections and infestations
Infection
|
9.5%
2/21 • Number of events 2
|
|
Infections and infestations
Pneumonia
|
4.8%
1/21 • Number of events 1
|
|
Infections and infestations
Skin infection
|
4.8%
1/21 • Number of events 1
|
|
Investigations
Alanine aminotransferase increased
|
23.8%
5/21 • Number of events 17
|
|
Investigations
Alkaline phosphatase increased
|
4.8%
1/21 • Number of events 3
|
|
Investigations
Aspartate aminotransferase increased
|
19.0%
4/21 • Number of events 6
|
|
Investigations
Blood bilirubin increased
|
9.5%
2/21 • Number of events 4
|
|
Investigations
Creatinine increased
|
4.8%
1/21 • Number of events 1
|
|
Investigations
Electrocardiogram QTc interval prolonged
|
9.5%
2/21 • Number of events 2
|
|
Investigations
Leukocyte count decreased
|
90.5%
19/21 • Number of events 71
|
|
Investigations
Lymphocyte count decreased
|
4.8%
1/21 • Number of events 1
|
|
Investigations
Neutrophil count decreased
|
85.7%
18/21 • Number of events 62
|
|
Investigations
Platelet count decreased
|
85.7%
18/21 • Number of events 73
|
|
Metabolism and nutrition disorders
Anorexia
|
14.3%
3/21 • Number of events 3
|
|
Metabolism and nutrition disorders
Blood glucose increased
|
19.0%
4/21 • Number of events 7
|
|
Metabolism and nutrition disorders
Iron overload
|
4.8%
1/21 • Number of events 1
|
|
Metabolism and nutrition disorders
Serum albumin decreased
|
4.8%
1/21 • Number of events 1
|
|
Metabolism and nutrition disorders
Serum calcium decreased
|
4.8%
1/21 • Number of events 1
|
|
Metabolism and nutrition disorders
Serum glucose decreased
|
4.8%
1/21 • Number of events 3
|
|
Metabolism and nutrition disorders
Serum magnesium decreased
|
4.8%
1/21 • Number of events 2
|
|
Metabolism and nutrition disorders
Serum potassium decreased
|
4.8%
1/21 • Number of events 1
|
|
Metabolism and nutrition disorders
Serum sodium decreased
|
4.8%
1/21 • Number of events 1
|
|
Metabolism and nutrition disorders
Serum sodium increased
|
4.8%
1/21 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
9.5%
2/21 • Number of events 2
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
4.8%
1/21 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
14.3%
3/21 • Number of events 3
|
|
Nervous system disorders
Dizziness
|
19.0%
4/21 • Number of events 6
|
|
Nervous system disorders
Dysgeusia
|
4.8%
1/21 • Number of events 1
|
|
Nervous system disorders
Extrapyramidal disorder
|
4.8%
1/21 • Number of events 1
|
|
Nervous system disorders
Headache
|
23.8%
5/21 • Number of events 9
|
|
Psychiatric disorders
Anxiety
|
4.8%
1/21 • Number of events 1
|
|
Psychiatric disorders
Confusion
|
9.5%
2/21 • Number of events 2
|
|
Psychiatric disorders
Insomnia
|
9.5%
2/21 • Number of events 3
|
|
Renal and urinary disorders
Kidney pain
|
4.8%
1/21 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
14.3%
3/21 • Number of events 3
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
47.6%
10/21 • Number of events 15
|
|
Respiratory, thoracic and mediastinal disorders
Hiccups
|
4.8%
1/21 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
4.8%
1/21 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Petechiae
|
4.8%
1/21 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
4.8%
1/21 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Rash desquamating
|
4.8%
1/21 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Skin disorder
|
4.8%
1/21 • Number of events 2
|
|
Skin and subcutaneous tissue disorders
Sweating
|
4.8%
1/21 • Number of events 2
|
|
Vascular disorders
Flushing
|
4.8%
1/21 • Number of events 1
|
|
Vascular disorders
Hypotension
|
9.5%
2/21 • Number of events 2
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60