Trial Outcomes & Findings for Perifosine in Relapsed or Refractory Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (NCT NCT00873457)

Last Updated: 2013-05-27

Results Overview

Per International Workshop on Chronic Lymphocytic Leukemia, Complete Response (CR):normal CBC; absence of the following: clonal lymphocytes in blood and marrow, lymphadenopathy, hepatomegaly or splenomegaly, and constitutional symptoms; and bone marrow has \<30% lymphocytes, is normocellular, and is without B-lymphoid nodules. Partial Response(PR): one of the following: decrease lymphadenopathy ≥ 50%; decrease of liver and/or spleen size ≥ 50%, any constitutional symptoms, Polymorphonuclear leukocytes ≥ 1,500/µl or a 50% improvement, or decrease of circulating clonal B lymphocytes ≥ 50% AND one of the following: Platelets ≥ 100,000/µl or a 50% improvement, Hemoglobin ≥ 11.0 g/dl or a 50% improvement, Bone marrow has ≥ 30% lymphocytes, or B-lymphoid nodules, or not done. Overall Response (OR)= CR+PR

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

16 participants

Primary outcome timeframe

after 3 months of treatment

Results posted on

2013-05-27

Participant Flow

All patients were recruited from Duke University Medical Center between 8/2009 and 7/2011

One patient was a screen failure and did not go on to participate in the study.

Participant milestones

Participant milestones
Measure	Perifosine Perifosine 50 mg twice a day for a total of six 28-day cycles.
Overall Study STARTED	16
Overall Study COMPLETED	16
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Perifosine in Relapsed or Refractory Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Perifosine n=16 Participants Perifosine 50 mg twice a day for a total of six 28-day cycles.
Age Continuous	69.5 years n=99 Participants
Age, Customized <=18 years	0 participants n=99 Participants
Age, Customized Between 18 and 65 years	3 participants n=99 Participants
Age, Customized >=65 years	13 participants n=99 Participants
Sex: Female, Male Female	5 Participants n=99 Participants
Sex: Female, Male Male	11 Participants n=99 Participants
Region of Enrollment United States	16 participants n=99 Participants

PRIMARY outcome

Timeframe: after 3 months of treatment

Population: Patients who completed 3 months of therapy.

Outcome measures

Outcome measures
Measure	Perifosine n=8 Participants Perifosine 50 mg twice a day for a total of six 28-day cycles.
Overall Response	1 participants

PRIMARY outcome

Timeframe: after 6 months of treatment

Population: Patients who completed 6 months of therapy

Outcome measures

Outcome measures
Measure	Perifosine n=1 Participants Perifosine 50 mg twice a day for a total of six 28-day cycles.
Overall Response	0 participants

SECONDARY outcome

Timeframe: up to a maximum of 2 years

Population: All treated patients

Overall survival is defined as the length of time between discontinuation of perifosine until death or 2 year's followup, whichever comes first.

Outcome measures

Outcome measures
Measure	Perifosine n=16 Participants Perifosine 50 mg twice a day for a total of six 28-day cycles.
Overall Survival	289.5 Days Interval 19.0 to 730.0

SECONDARY outcome

Timeframe: up to a maximum of 2 years

Population: All treated patients

Event-free survival will be defined as the length of time between the discontinuation of study treatment and disease progression, next therapy, or death,whichever comes first, up to a maximum of 2 years.

Outcome measures

Outcome measures
Measure	Perifosine n=16 Participants Perifosine 50 mg twice a day for a total of six 28-day cycles.
Event-free Survival	118 Days Interval 19.0 to 350.0

Adverse Events

Perifosine

Serious events: 16 serious events

Other events: 16 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Perifosine n=16 participants at risk Perifosine 50 mg twice a day for a total of six 28-day cycles.
Investigations Platelets	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
General disorders Fever (in the absence of neutropenia)	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Skin and subcutaneous tissue disorders Rash / desquamation	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Metabolism and nutrition disorders Anorexia	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Infections and infestations Infection with unknown ANC	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Infections and infestations Infection without febrile neutropenia	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
General disorders Edema: limb	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Investigations Bilirubin (hyperbilirubinemia)	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Metabolism and nutrition disorders Dehydration	12.5% 2/16 • Number of events 3 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Gastrointestinal disorders Hemorrhage, GI	12.5% 2/16 • Number of events 3 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Respiratory, thoracic and mediastinal disorders Dyspnea (shortness of breath)	12.5% 2/16 • Number of events 2 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Blood and lymphatic system disorders Hemoglobin	18.8% 3/16 • Number of events 3 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Blood and lymphatic system disorders Febrile neutropenia (fever of unknown origin without clinically or microbiologically documented infe	18.8% 3/16 • Number of events 3 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Infections and infestations Infection - Other	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Nervous system disorders Syncope (fainting)	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose

Other adverse events

Other adverse events
Measure	Perifosine n=16 participants at risk Perifosine 50 mg twice a day for a total of six 28-day cycles.
Investigations Platelets	50.0% 8/16 • Number of events 8 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Skin and subcutaneous tissue disorders Rash / desquamation	25.0% 4/16 • Number of events 7 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Metabolism and nutrition disorders Anorexia	31.2% 5/16 • Number of events 5 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Infections and infestations Infection - Other	18.8% 3/16 • Number of events 3 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
General disorders Edema: limb	12.5% 2/16 • Number of events 2 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Investigations Bilirubin (hyperbilirubinemia)	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Metabolism and nutrition disorders Dehydration	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Respiratory, thoracic and mediastinal disorders Dyspnea (shortness of breath)	18.8% 3/16 • Number of events 3 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Blood and lymphatic system disorders Hemoglobin	62.5% 10/16 • Number of events 10 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Blood and lymphatic system disorders Febrile neutropenia (fever of unknown origin without clinically or microbiologically documented infe	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Respiratory, thoracic and mediastinal disorders Allergic rhinitis (including sneezing, nasal stuffiness, postnasal drip)	12.5% 2/16 • Number of events 3 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Ear and labyrinth disorders Hearing Loss	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Investigations Leukocytes (total WBC)	25.0% 4/16 • Number of events 4 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Investigations Neutrophils / granulocytes (ANC / AGC)	31.2% 5/16 • Number of events 7 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
General disorders Fatigue (asthenia, lethargy, malaise)	75.0% 12/16 • Number of events 14 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Psychiatric disorders Insomnia	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Skin and subcutaneous tissue disorders Sweating (diaphoresis)	12.5% 2/16 • Number of events 2 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Skin and subcutaneous tissue disorders Dermatology / Skin - Other	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Skin and subcutaneous tissue disorders Dry Skin	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Skin and subcutaneous tissue disorders Hair Loss / Alopecia (scalp or body)	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Skin and subcutaneous tissue disorders Pruritus / itching	12.5% 2/16 • Number of events 2 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Vascular disorders Hot flashes / flushes	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Gastrointestinal disorders Constipation	12.5% 2/16 • Number of events 2 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Gastrointestinal disorders Diarrhea	31.2% 5/16 • Number of events 6 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Gastrointestinal disorders Flatulence	12.5% 2/16 • Number of events 2 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Gastrointestinal disorders Gastrointestinal - Other	12.5% 2/16 • Number of events 2 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Gastrointestinal disorders Heartburn / dyspepsia	12.5% 2/16 • Number of events 2 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Gastrointestinal disorders Mucositis / Stomatitis	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Gastrointestinal disorders Nausea	12.5% 2/16 • Number of events 3 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Nervous system disorders Taste Alteration (dysgeusia)	18.8% 3/16 • Number of events 3 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Gastrointestinal disorders Vomiting	12.5% 2/16 • Number of events 2 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Respiratory, thoracic and mediastinal disorders Hemorrhage, pulmonary / upper respiratory	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
General disorders Edema: Other	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Investigations Alkaline phosphatase	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Metabolism and nutrition disorders Calcium, serum-high (hypercalcemia)	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Metabolism and nutrition disorders Calcium, serum-low (hypocalcemia)	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Investigations Creatinine	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Metabolism and nutrition disorders Glucose, serum-high (hyperglycemia)	18.8% 3/16 • Number of events 4 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Metabolism and nutrition disorders Sodium, serum-low (hyponatremia)	6.2% 1/16 • Number of events 2 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Musculoskeletal and connective tissue disorders Muscle weakness, generalized or specific area (not due to neuropathy)	18.8% 3/16 • Number of events 3 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Psychiatric disorders Confusion	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Nervous system disorders Dizziness	18.8% 3/16 • Number of events 4 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Psychiatric disorders Mood Alteration - Agitation	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Nervous system disorders Neurology - Other	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Nervous system disorders Neuropathy: sensory	12.5% 2/16 • Number of events 2 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Nervous system disorders Syncope (fainting)	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Eye disorders Ocular / Visual - Other	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Eye disorders Vision - blurred vision	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Eye disorders Vision - flashing lights / floaters	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Gastrointestinal disorders Pain, Gastrointestinal	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Musculoskeletal and connective tissue disorders Pain, Musculoskeletal	37.5% 6/16 • Number of events 10 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Nervous system disorders Pain, Neurology	18.8% 3/16 • Number of events 3 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Musculoskeletal and connective tissue disorders Pain, Neck	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
General disorders Pain, Other	12.5% 2/16 • Number of events 2 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Respiratory, thoracic and mediastinal disorders Pain, Pulmonary / Upper Respiratory	12.5% 2/16 • Number of events 2 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Respiratory, thoracic and mediastinal disorders Cough	18.8% 3/16 • Number of events 3 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Respiratory, thoracic and mediastinal disorders Pulmonary / Upper Respiratory - Other	12.5% 2/16 • Number of events 2 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Respiratory, thoracic and mediastinal disorders Voice changes / dysarthria (e.g., hoarseness, loss or alteration in voice, laryngitis)	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Renal and urinary disorders Urinary frequency / urgency	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Infections and infestations Infection with unknown ANC - Ungual (nails)	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
Respiratory, thoracic and mediastinal disorders Pain, chest/thorax NOS	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose
General disorders Extremity-lower (gait / walking)	6.2% 1/16 • Number of events 1 • Adverse events were recorded from the initiation of study drug until 30 days after the last dose

Additional Information

Daphne Friedman, MD

Duke University Medical Center

Phone: 919-684-9220

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place