Trial Outcomes & Findings for Lenalidomide and Prednisone in Low and Int-1 Myelodysplastic Syndrome (MDS) Non 5q MDS (NCT NCT01133275)
NCT ID: NCT01133275
Last Updated: 2019-12-17
Results Overview
The rate of erythroid response to treatment with the lenalidomide/prednisone combination in non-del (5q) low and int-1 risk Myelodysplastic Syndrome (MDS) with symptomatic anemia. Hematological improvement erythroid response (HI-E) according to International Working Group (IWG) 2006 criteria.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
28 participants
Primary outcome timeframe
Up to 7 months
Results posted on
2019-12-17
Participant Flow
Participants were recruited at Moffitt Cancer Center and Shands Cancer Hospital at the University of Florida from 4/28/2010 through 8/13/2013.
Participant milestones
| Measure |
Lenalidomide and Prednisone Therapy
Lenalidomide and prednisone therapy for 6 cycles (24 weeks). Each cycle is 28 days (4 weeks).
|
|---|---|
|
Overall Study
STARTED
|
28
|
|
Overall Study
COMPLETED
|
26
|
|
Overall Study
NOT COMPLETED
|
2
|
Reasons for withdrawal
| Measure |
Lenalidomide and Prednisone Therapy
Lenalidomide and prednisone therapy for 6 cycles (24 weeks). Each cycle is 28 days (4 weeks).
|
|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
|
Overall Study
Cerebrovascular accident (CVA)
|
1
|
Baseline Characteristics
Lenalidomide and Prednisone in Low and Int-1 Myelodysplastic Syndrome (MDS) Non 5q MDS
Baseline characteristics by cohort
| Measure |
Lenalidomide and Prednisone Therapy
n=28 Participants
Lenalidomide and prednisone therapy for 6 cycles (24 weeks). Each cycle is 28 days (4 weeks).
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
8 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
20 Participants
n=5 Participants
|
|
Age, Continuous
|
68.64 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
18 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
28 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to 7 monthsPopulation: All evaluable participants
The rate of erythroid response to treatment with the lenalidomide/prednisone combination in non-del (5q) low and int-1 risk Myelodysplastic Syndrome (MDS) with symptomatic anemia. Hematological improvement erythroid response (HI-E) according to International Working Group (IWG) 2006 criteria.
Outcome measures
| Measure |
Lenalidomide and Prednisone Therapy
n=26 Participants
Lenalidomide and prednisone therapy for 6 cycles (24 weeks). Each cycle is 28 days (4 weeks).
|
|---|---|
|
Number of Participants With Erythroid Response
|
5 participants
|
SECONDARY outcome
Timeframe: Up to 54 monthsPopulation: All participants
Grade 3 or 4 events Related/Possibly Related/Probably Related to study treatment. Number of participants with events specified in the study protocol: Neutropenia, Thrombocytopenia, Febrile Neutropenia, Infection, Sepsis, Venous Thromboembolic Events. Evaluations according to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) V4.0.
Outcome measures
| Measure |
Lenalidomide and Prednisone Therapy
n=28 Participants
Lenalidomide and prednisone therapy for 6 cycles (24 weeks). Each cycle is 28 days (4 weeks).
|
|---|---|
|
Number of Participants With Grade 3 or 4 Adverse Events Possibly Related to Treatment
Neutropenia
|
8 participants
|
|
Number of Participants With Grade 3 or 4 Adverse Events Possibly Related to Treatment
Thrombocytopenia
|
4 participants
|
|
Number of Participants With Grade 3 or 4 Adverse Events Possibly Related to Treatment
Febrile Neutropenia
|
0 participants
|
|
Number of Participants With Grade 3 or 4 Adverse Events Possibly Related to Treatment
Infection and Sepsis
|
0 participants
|
|
Number of Participants With Grade 3 or 4 Adverse Events Possibly Related to Treatment
Venous Thromboembolic Events
|
0 participants
|
Adverse Events
Lenalidomide and Prednisone Therapy
Serious events: 8 serious events
Other events: 27 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Lenalidomide and Prednisone Therapy
n=28 participants at risk
Lenalidomide and prednisone therapy for 6 cycles (24 weeks). Each cycle is 28 days (4 weeks).
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
3.6%
1/28 • Number of events 1 • 4 years, 6 months
|
|
Blood and lymphatic system disorders
Blood clot
|
3.6%
1/28 • Number of events 1 • 4 years, 6 months
|
|
Cardiac disorders
Myocardial infarction
|
3.6%
1/28 • Number of events 1 • 4 years, 6 months
|
|
Cardiac disorders
Chest pain
|
3.6%
1/28 • Number of events 1 • 4 years, 6 months
|
|
Cardiac disorders
Heart arrhythmia
|
3.6%
1/28 • Number of events 1 • 4 years, 6 months
|
|
Gastrointestinal disorders
Diarrhea
|
3.6%
1/28 • Number of events 1 • 4 years, 6 months
|
|
Gastrointestinal disorders
Rectal bleeding
|
3.6%
1/28 • Number of events 1 • 4 years, 6 months
|
|
Gastrointestinal disorders
Severe abdominal cramping
|
3.6%
1/28 • Number of events 1 • 4 years, 6 months
|
|
Gastrointestinal disorders
Severe nausea
|
3.6%
1/28 • Number of events 1 • 4 years, 6 months
|
|
Gastrointestinal disorders
Uncontrolled vomiting
|
3.6%
1/28 • Number of events 1 • 4 years, 6 months
|
|
Gastrointestinal disorders
Small intestinal blockage
|
3.6%
1/28 • Number of events 1 • 4 years, 6 months
|
|
General disorders
Edema limbs
|
3.6%
1/28 • Number of events 1 • 4 years, 6 months
|
|
General disorders
Sudden death NOS
|
3.6%
1/28 • Number of events 1 • 4 years, 6 months
|
|
General disorders
Fever
|
3.6%
1/28 • Number of events 1 • 4 years, 6 months
|
|
General disorders
Pain - sore throat
|
3.6%
1/28 • Number of events 1 • 4 years, 6 months
|
|
Infections and infestations
Soft tissue infection
|
3.6%
1/28 • Number of events 1 • 4 years, 6 months
|
|
Metabolism and nutrition disorders
Hyponatremia
|
3.6%
1/28 • Number of events 1 • 4 years, 6 months
|
|
Nervous system disorders
Stroke / Cerebrovascular Accident (CVA)
|
3.6%
1/28 • Number of events 1 • 4 years, 6 months
|
|
Psychiatric disorders
Confusion
|
3.6%
1/28 • Number of events 1 • 4 years, 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
3.6%
1/28 • Number of events 1 • 4 years, 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary hypertension
|
3.6%
1/28 • Number of events 1 • 4 years, 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
3.6%
1/28 • Number of events 1 • 4 years, 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia
|
3.6%
1/28 • Number of events 1 • 4 years, 6 months
|
Other adverse events
| Measure |
Lenalidomide and Prednisone Therapy
n=28 participants at risk
Lenalidomide and prednisone therapy for 6 cycles (24 weeks). Each cycle is 28 days (4 weeks).
|
|---|---|
|
Gastrointestinal disorders
Diarrhea
|
46.4%
13/28 • Number of events 18 • 4 years, 6 months
|
|
Gastrointestinal disorders
Constipation
|
21.4%
6/28 • Number of events 7 • 4 years, 6 months
|
|
Gastrointestinal disorders
Nausea
|
17.9%
5/28 • Number of events 7 • 4 years, 6 months
|
|
Gastrointestinal disorders
Vomiting
|
17.9%
5/28 • Number of events 5 • 4 years, 6 months
|
|
Gastrointestinal disorders
Abdominal pain
|
10.7%
3/28 • Number of events 3 • 4 years, 6 months
|
|
Gastrointestinal disorders
Gastrointestinal disorders - Other
|
10.7%
3/28 • Number of events 3 • 4 years, 6 months
|
|
Gastrointestinal disorders
Dry mouth
|
7.1%
2/28 • Number of events 3 • 4 years, 6 months
|
|
Gastrointestinal disorders
Dysphagia
|
7.1%
2/28 • Number of events 2 • 4 years, 6 months
|
|
Gastrointestinal disorders
Mucositis oral
|
7.1%
2/28 • Number of events 2 • 4 years, 6 months
|
|
General disorders
Fatigue
|
46.4%
13/28 • Number of events 14 • 4 years, 6 months
|
|
General disorders
Edema limbs
|
25.0%
7/28 • Number of events 10 • 4 years, 6 months
|
|
General disorders
Paul
|
25.0%
7/28 • Number of events 9 • 4 years, 6 months
|
|
General disorders
Fever
|
17.9%
5/28 • Number of events 5 • 4 years, 6 months
|
|
General disorders
Chills
|
14.3%
4/28 • Number of events 4 • 4 years, 6 months
|
|
Investigations
Platelet count decreased
|
57.1%
16/28 • Number of events 37 • 4 years, 6 months
|
|
Investigations
Neutrophil count decreased
|
46.4%
13/28 • Number of events 49 • 4 years, 6 months
|
|
Investigations
White blood cell decreased
|
46.4%
13/28 • Number of events 40 • 4 years, 6 months
|
|
Investigations
Creatinine increased
|
7.1%
2/28 • Number of events 2 • 4 years, 6 months
|
|
Investigations
Investigations - Other
|
7.1%
2/28 • Number of events 4 • 4 years, 6 months
|
|
Investigations
Lymphocyte count decreased
|
7.1%
2/28 • Number of events 5 • 4 years, 6 months
|
|
Investigations
Weight gain
|
7.1%
2/28 • Number of events 3 • 4 years, 6 months
|
|
Blood and lymphatic system disorders
Anemia
|
53.6%
15/28 • Number of events 36 • 4 years, 6 months
|
|
Blood and lymphatic system disorders
Leukocytosis
|
17.9%
5/28 • Number of events 5 • 4 years, 6 months
|
|
Nervous system disorders
Movements involuntary
|
21.4%
6/28 • Number of events 6 • 4 years, 6 months
|
|
Nervous system disorders
Dizziness
|
14.3%
4/28 • Number of events 5 • 4 years, 6 months
|
|
Nervous system disorders
Headache
|
14.3%
4/28 • Number of events 4 • 4 years, 6 months
|
|
Nervous system disorders
Memory impairment
|
7.1%
2/28 • Number of events 2 • 4 years, 6 months
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
7.1%
2/28 • Number of events 3 • 4 years, 6 months
|
|
Nervous system disorders
Sinus pain
|
7.1%
2/28 • Number of events 3 • 4 years, 6 months
|
|
Skin and subcutaneous tissue disorders
Hyperhidrosis
|
17.9%
5/28 • Number of events 5 • 4 years, 6 months
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
17.9%
5/28 • Number of events 7 • 4 years, 6 months
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
10.7%
3/28 • Number of events 3 • 4 years, 6 months
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other
|
10.7%
3/28 • Number of events 4 • 4 years, 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
21.4%
6/28 • Number of events 7 • 4 years, 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
10.7%
3/28 • Number of events 3 • 4 years, 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Hoarseness
|
10.7%
3/28 • Number of events 3 • 4 years, 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
10.7%
3/28 • Number of events 3 • 4 years, 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Allergic rhinitis
|
7.1%
2/28 • Number of events 2 • 4 years, 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
7.1%
2/28 • Number of events 2 • 4 years, 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders - Other
|
7.1%
2/28 • Number of events 2 • 4 years, 6 months
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
14.3%
4/28 • Number of events 4 • 4 years, 6 months
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal and connective tissue disorder - Other
|
14.3%
4/28 • Number of events 5 • 4 years, 6 months
|
|
Injury, poisoning and procedural complications
Bruising
|
21.4%
6/28 • Number of events 6 • 4 years, 6 months
|
|
Injury, poisoning and procedural complications
Fall
|
14.3%
4/28 • Number of events 4 • 4 years, 6 months
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
14.3%
4/28 • Number of events 4 • 4 years, 6 months
|
|
Metabolism and nutrition disorders
Hypokalemia
|
10.7%
3/28 • Number of events 4 • 4 years, 6 months
|
|
Metabolism and nutrition disorders
Metabolism and nutrition disorders - Other
|
7.1%
2/28 • Number of events 6 • 4 years, 6 months
|
|
Infections and infestations
Sinusitis
|
7.1%
2/28 • Number of events 2 • 4 years, 6 months
|
|
Psychiatric disorders
Insomnia
|
21.4%
6/28 • Number of events 6 • 4 years, 6 months
|
|
Eye disorders
Blurred vision
|
14.3%
4/28 • Number of events 4 • 4 years, 6 months
|
|
Eye disorders
Eye disorders - Other
|
7.1%
2/28 • Number of events 2 • 4 years, 6 months
|
Additional Information
Dr. Rami Komrokji
H. Lee Moffitt Cancer Center and Research Institute
Phone: 813-745-4692
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place