Trial Outcomes & Findings for Pilot Lenalidomide in Adult Diamond-Blackfan Anemia Patients w/ RBC Transfusion-Dependent Anemia (NCT NCT01034592)

Last Updated: 2017-08-02

Results Overview

Red blood cell (RBC) transfusion independence is reported as the number of subjects who achieve a continuous absence of the intravenous infusion of any RBC transfusion during any consecutive "rolling" 56 days during the treatment period.

Recruitment status

TERMINATED

Study phase

PHASE1

Target enrollment

2 participants

Primary outcome timeframe

6 months

Results posted on

2017-08-02

Participant Flow

Participant milestones

Participant milestones
Measure	Lenalidomide Subjects will initially receive lenalidomide 2.5 mg, and may escalate up to 2.5 mg/wk up to 5 mg 3x/wk, depending toxicity and response.
Overall Study STARTED	2
Overall Study COMPLETED	2
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Pilot Lenalidomide in Adult Diamond-Blackfan Anemia Patients w/ RBC Transfusion-Dependent Anemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Lenalidomide n=2 Participants Subjects will initially receive lenalidomide 2.5 mg, and may escalate up to 2.5 mg/wk up to 5 mg 3x/wk, depending toxicity and response.
Race (NIH/OMB) Black or African American	0 Participants n=93 Participants
Race (NIH/OMB) White	2 Participants n=93 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants
Region of Enrollment United States	2 participants n=93 Participants
Age, Categorical <=18 years	0 Participants n=93 Participants
Age, Categorical Between 18 and 65 years	2 Participants n=93 Participants
Age, Categorical >=65 years	0 Participants n=93 Participants
Sex: Female, Male Female	1 Participants n=93 Participants
Sex: Female, Male Male	1 Participants n=93 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=93 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	2 Participants n=93 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants
Race (NIH/OMB) Asian	0 Participants n=93 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants

PRIMARY outcome

Timeframe: 6 months

Population: All treated subjects were analyzed.

Outcome measures

Outcome measures
Measure	Lenalidomide n=2 Participants Subjects will initially receive lenalidomide 2.5 mg, and may escalate up to 2.5 mg/wk up to 5 mg 3x/wk, depending toxicity and response.
Red Blood Cell (RBC) Transfusion Independence	0 participants

SECONDARY outcome

Timeframe: 6 months

Population: Both participants were assessed and contributed to the analysis.

The effect on red blood cell (RBC) transfusions was assessed as the number of participants that achieved a greater than 50% decrease in RBC transfusion requirements.

Outcome measures

Outcome measures
Measure	Lenalidomide n=2 Participants Subjects will initially receive lenalidomide 2.5 mg, and may escalate up to 2.5 mg/wk up to 5 mg 3x/wk, depending toxicity and response.
Red Blood Cell (RBC) Transfusions	0 Participants

SECONDARY outcome

Timeframe: 6 months

Population: Both participants were assessed and contributed to the analysis.

The effect on hemoglobin concentration was assessed as the change from baseline, measured in g/dL.

Outcome measures

Outcome measures
Measure	Lenalidomide n=2 Participants Subjects will initially receive lenalidomide 2.5 mg, and may escalate up to 2.5 mg/wk up to 5 mg 3x/wk, depending toxicity and response.
Hemoglobin Concentration	0.4 g/dL Interval 0.4 to 0.4

SECONDARY outcome

Timeframe: 6 months

Population: Both participants were assessed and contributed to the analysis.

The effect on neutrophil levels was assessed as the change in neutrophil count from baseline.

Outcome measures

Outcome measures
Measure	Lenalidomide n=2 Participants Subjects will initially receive lenalidomide 2.5 mg, and may escalate up to 2.5 mg/wk up to 5 mg 3x/wk, depending toxicity and response.
Neutrophil Response	0.50 1000/uL Interval 0.1 to 0.91

SECONDARY outcome

Timeframe: 6 months

Population: Both participants were assessed and contributed to the analysis.

The effect on platelet levels as assessed as the change in platelet count from baseline.

Outcome measures

Outcome measures
Measure	Lenalidomide n=2 Participants Subjects will initially receive lenalidomide 2.5 mg, and may escalate up to 2.5 mg/wk up to 5 mg 3x/wk, depending toxicity and response.
Platelet Response	25.5 1000/uL Interval -20.0 to 71.0

SECONDARY outcome

Timeframe: 6 months

Population: Both participants were assessed and contributed to the analysis, but never demonstrated any therapeutic response.

The response duration was measured from the last of the consecutive 56 days during which the subject was free of red blood cells (RBC) transfusions to the date of the first RBC transfusion after the 56-day RBC-transfusion-free period.

Outcome measures

Outcome measures
Measure	Lenalidomide n=2 Participants Subjects will initially receive lenalidomide 2.5 mg, and may escalate up to 2.5 mg/wk up to 5 mg 3x/wk, depending toxicity and response.
Duration of Response	0 days Interval 0.0 to 0.0

SECONDARY outcome

Timeframe: 6 months

Population: Both participants were assessed and contributed to the analysis.

Toxicity was assessed as the number of adverse events related to lenalidomide.

Outcome measures

Outcome measures
Measure	Lenalidomide n=2 Participants Subjects will initially receive lenalidomide 2.5 mg, and may escalate up to 2.5 mg/wk up to 5 mg 3x/wk, depending toxicity and response.
Toxicity	1 Related Adverse Events

Adverse Events

Serious Adverse Events

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Serious Adverse Events n=2 participants at risk Serious Adverse Events include: adverse events that result in death, require either inpatient hospitalization or the prolongation of hospitalization, are life-threatening, result in a persistent or significant disability/incapacity or result in a congenital anomaly/birth defect. Other important medical events, based upon appropriate medical judgment, may also be considered Serious Adverse Events if a trial participant's health is at risk and intervention is required to prevent an outcome mentioned.
General disorders Fatigue	100.0% 2/2 • Number of events 2 • Adverse event data was collected over the duration of the treatment and maintenance phase of the study, for a total of 2 years.
Blood and lymphatic system disorders Neutropenia	50.0% 1/2 • Number of events 1 • Adverse event data was collected over the duration of the treatment and maintenance phase of the study, for a total of 2 years.
Infections and infestations Ear Infection	50.0% 1/2 • Number of events 1 • Adverse event data was collected over the duration of the treatment and maintenance phase of the study, for a total of 2 years.
Investigations Aspartate aminotransferase, increased	50.0% 1/2 • Number of events 1 • Adverse event data was collected over the duration of the treatment and maintenance phase of the study, for a total of 2 years.
Investigations Alanine aminotransferase, increased	50.0% 1/2 • Number of events 1 • Adverse event data was collected over the duration of the treatment and maintenance phase of the study, for a total of 2 years.
Respiratory, thoracic and mediastinal disorders Dry cough	50.0% 1/2 • Number of events 1 • Adverse event data was collected over the duration of the treatment and maintenance phase of the study, for a total of 2 years.

Additional Information

Jason R Gotlib, MD, Professor of Medicine (Hematology)

Stanford University Medical Center

Phone: 650-867-2823

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place