Trial Outcomes & Findings for Lenalidomide for Adult Histiocyte Disorders (NCT NCT02523040)
NCT ID: NCT02523040
Last Updated: 2025-12-10
Results Overview
Proportion of participants achieving either complete resolution of all signs or symptoms which is non-active disease (NAD) status or regression of signs or symptoms along with no new lesions which is better active-disease (AD) status per Histiocyte Society criteria any time on treatment.
Recruitment status
ACTIVE_NOT_RECRUITING
Study phase
PHASE2
Target enrollment
12 participants
Primary outcome timeframe
Disease assessed every 3 cycles on treatment up do 12 cycles (approximately 12 months).
Results posted on
2025-12-10
Participant Flow
Participant milestones
| Measure |
Lenalidomide
Single agent lenalidomide (len) orally days 1-21 of a 28-day cycle up to 12 cycles. During cycle 1 patients receive 10mg len. If tolerated well, dose increases to 25mg and, if not, dose is reduced to 5mg. Dose also dependent on creatinine clearance (CrCl) level. For patients not already receiving systemic anticoagulation, a minimum aspirin dose of 81mg daily or for one month following treatment discontinuation is required.
|
|---|---|
|
Overall Study
STARTED
|
12
|
|
Overall Study
Evaluable for TNF-Alpha Evaluable for TNF Alpha
|
6
|
|
Overall Study
COMPLETED
|
12
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Lenalidomide for Adult Histiocyte Disorders
Baseline characteristics by cohort
| Measure |
Lenalidomide
n=12 Participants
Single agent lenalidomide (len) orally days 1-21 of a 28-day cycle up to 12 cycles. During cycle 1 patients receive 10mg len. If tolerated well, dose increases to 25mg and, if not, dose is reduced to 5mg. Dose also dependent on creatinine clearance (CrCl) level. For patients not already receiving systemic anticoagulation, a minimum aspirin dose of 81mg daily or for one month following treatment discontinuation is required.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=4 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
11 Participants
n=4 Participants
|
|
Age, Categorical
>=65 years
|
1 Participants
n=4 Participants
|
|
Age, Continuous
|
44 years
n=4 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
11 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
7 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
2 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=4 Participants
|
|
Region of Enrollment
United States
|
12 participants
n=4 Participants
|
|
ECOG performance status
00 - Fully Active
|
4 Participants
n=4 Participants
|
|
ECOG performance status
01 - Restricted
|
7 Participants
n=4 Participants
|
|
ECOG performance status
02 - Ambulatory and Capable of Self Care
|
1 Participants
n=4 Participants
|
PRIMARY outcome
Timeframe: Disease assessed every 3 cycles on treatment up do 12 cycles (approximately 12 months).Proportion of participants achieving either complete resolution of all signs or symptoms which is non-active disease (NAD) status or regression of signs or symptoms along with no new lesions which is better active-disease (AD) status per Histiocyte Society criteria any time on treatment.
Outcome measures
| Measure |
Lenalidomide
n=12 Participants
Single agent lenalidomide (len) orally days 1-21 of a 28-day cycle up to 12 cycles. During cycle 1 patients receive 10mg len. If tolerated well, dose increases to 25mg and, if not, dose is reduced to 5mg. Dose also dependent on creatinine clearance (CrCl) level. For patients not already receiving systemic anticoagulation, a minimum aspirin dose of 81mg daily or for one month following treatment discontinuation is required.
|
|---|---|
|
Overall Response Rate (ORR)
|
0.67 proportion of participants
Interval 0.39 to 0.88
|
SECONDARY outcome
Timeframe: Disease assessed on treatment every 3 cycles and in long-term follow-up up to the earlier of 36 months (every 3 months for 2 years then every 6 months) or start of new anti-cancer therapy. Relevant for this endpoint was12-months estimate.Progression-free survival (PFS) based on the Kaplan-Meier method is defined as time from registration to progression (PD) or death, censored for patients alive and progression-free at last disease assessment. PD is defined by Histiocyte criteria. Percent PFS is the percent of patients who are alive and progression-free at 12 months.
Outcome measures
| Measure |
Lenalidomide
n=12 Participants
Single agent lenalidomide (len) orally days 1-21 of a 28-day cycle up to 12 cycles. During cycle 1 patients receive 10mg len. If tolerated well, dose increases to 25mg and, if not, dose is reduced to 5mg. Dose also dependent on creatinine clearance (CrCl) level. For patients not already receiving systemic anticoagulation, a minimum aspirin dose of 81mg daily or for one month following treatment discontinuation is required.
|
|---|---|
|
12-months Progression-free Survival (PFS)
|
62 percentage probability
Interval 39.0 to 100.0
|
SECONDARY outcome
Timeframe: 12 MonthsPopulation: Survival followed up 36 months from start of protocol treatment. Relevant for this endpoint was12-months estimate.
Overall survival (OS) based on the Kaplan-Meier method is defined as the time from registration to death, censored for patients alive at last contact. Percent OS is the percent of patients who are alive at 12 months.
Outcome measures
| Measure |
Lenalidomide
n=12 Participants
Single agent lenalidomide (len) orally days 1-21 of a 28-day cycle up to 12 cycles. During cycle 1 patients receive 10mg len. If tolerated well, dose increases to 25mg and, if not, dose is reduced to 5mg. Dose also dependent on creatinine clearance (CrCl) level. For patients not already receiving systemic anticoagulation, a minimum aspirin dose of 81mg daily or for one month following treatment discontinuation is required.
|
|---|---|
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12-months Overall Survival (OS)
|
92 percentage probability
Interval 77.0 to 100.0
|
SECONDARY outcome
Timeframe: Up to 12 months on treatment.Grade 3-4 adverse events (AE) with treatment attribution of possibly, probably or definite (treatment-related) based on NCI Common Toxicity Criteria for Adverse Events version 3 (CTCAEv3) as reported on case report forms were tabulated by maximum grade. Incidence is the proportion of participants experiencing at least one treatment-related grade 3-4 AE of any type during the time of observation.
Outcome measures
| Measure |
Lenalidomide
n=12 Participants
Single agent lenalidomide (len) orally days 1-21 of a 28-day cycle up to 12 cycles. During cycle 1 patients receive 10mg len. If tolerated well, dose increases to 25mg and, if not, dose is reduced to 5mg. Dose also dependent on creatinine clearance (CrCl) level. For patients not already receiving systemic anticoagulation, a minimum aspirin dose of 81mg daily or for one month following treatment discontinuation is required.
|
|---|---|
|
Incidence of Grade 3-4 Toxicity
|
0.25 proportion of participants
Interval 0.07 to 0.53
|
SECONDARY outcome
Timeframe: 12 MonthsQuantitative serial measurements of urine cell free DNA for BRAF mutation as a biomarker of response
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Measured at baseline, day 1 of cycles 3, 6 ad 12 cycles and within 28 days post-treatment end (up to 13 months).Population: Participants with baseline and at least one on-treatment cycle TNF-alpha measurement.
Serum TNF-alpha levels were quantified per established methods and maximum percent change in level from baseline derived.
Outcome measures
| Measure |
Lenalidomide
n=6 Participants
Single agent lenalidomide (len) orally days 1-21 of a 28-day cycle up to 12 cycles. During cycle 1 patients receive 10mg len. If tolerated well, dose increases to 25mg and, if not, dose is reduced to 5mg. Dose also dependent on creatinine clearance (CrCl) level. For patients not already receiving systemic anticoagulation, a minimum aspirin dose of 81mg daily or for one month following treatment discontinuation is required.
|
|---|---|
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Percent Change in Serum TNF-alpha Levels on Therapy From Baseline up to 12 Cycles
|
-14.4 percent change from baseline
Interval -67.3 to 8.3
|
SECONDARY outcome
Timeframe: 12 monthsQuantitative serial measurements of plasma cell free DNA for BRAF mutation as a biomarker of response
Outcome measures
Outcome data not reported
Adverse Events
Lenalidomide
Serious events: 5 serious events
Other events: 11 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Lenalidomide
n=12 participants at risk
After the screening procedures confirm participation in the research study.
\- Lenalidomide Oral, Daily for 21 days of each cycle
Lenalidomide
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Metabolism and nutrition disorders
Hyponatremia
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
General disorders
Neck edema
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, specify
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Investigations
Neutrophil count decreased
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Vascular disorders
Superficial thrombophlebitis
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Vascular disorders
Thromboembolic event
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Injury, poisoning and procedural complications
Vascular access complication
|
8.3%
1/12 • Number of events 1 • 12 months
|
Other adverse events
| Measure |
Lenalidomide
n=12 participants at risk
After the screening procedures confirm participation in the research study.
\- Lenalidomide Oral, Daily for 21 days of each cycle
Lenalidomide
|
|---|---|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Gastrointestinal disorders
Enterocolitis
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Eye disorders
Eye disorders - Other, specify
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Eye disorders
Eye pain
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
General disorders
General disorders and administration site conditions - Other, specify
|
8.3%
1/12 • Number of events 2 • 12 months
|
|
Ear and labyrinth disorders
Hearing impaired
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Hoarseness
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Metabolism and nutrition disorders
Hypernatremia
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Musculoskeletal and connective tissue disorders
Joint effusion
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Investigations
Lymphocyte count decreased
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Metabolism and nutrition disorders
Metabolism and nutrition disorders - Other, specify
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness lower limb
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal and connective tissue disorder - Other, specify
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Nervous system disorders
Nervous system disorders - Other, specify
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
General disorders
Non-cardiac chest pain
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Gastrointestinal disorders
Oral pain
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
General disorders
Pain
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Skin and subcutaneous tissue disorders
Scalp pain
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Cardiac disorders
Sinus tachycardia
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Gastrointestinal disorders
Small intestinal obstruction
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Gastrointestinal disorders
Stomach pain
|
8.3%
1/12 • Number of events 2 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Stridor
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Nervous system disorders
Tremor
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Infections and infestations
Upper respiratory infection
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Renal and urinary disorders
Urinary frequency
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Infections and infestations
Urinary tract infection
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Renal and urinary disorders
Urinary tract pain
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Eye disorders
Vitreous hemorrhage
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Investigations
Weight gain
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Investigations
Platelet count decreased
|
33.3%
4/12 • Number of events 6 • 12 months
|
|
Nervous system disorders
Dizziness
|
25.0%
3/12 • Number of events 4 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
25.0%
3/12 • Number of events 3 • 12 months
|
|
General disorders
Edema limbs
|
25.0%
3/12 • Number of events 3 • 12 months
|
|
Investigations
White blood cell decreased
|
25.0%
3/12 • Number of events 6 • 12 months
|
|
Investigations
Alanine aminotransferase increased
|
16.7%
2/12 • Number of events 3 • 12 months
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
16.7%
2/12 • Number of events 2 • 12 months
|
|
Investigations
Aspartate aminotransferase increased
|
16.7%
2/12 • Number of events 6 • 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
16.7%
2/12 • Number of events 2 • 12 months
|
|
Gastrointestinal disorders
Dysphagia
|
16.7%
2/12 • Number of events 2 • 12 months
|
|
General disorders
Edema face
|
16.7%
2/12 • Number of events 2 • 12 months
|
|
Metabolism and nutrition disorders
Hypokalemia
|
16.7%
2/12 • Number of events 2 • 12 months
|
|
Metabolism and nutrition disorders
Hyponatremia
|
16.7%
2/12 • Number of events 2 • 12 months
|
|
Nervous system disorders
Paresthesia
|
16.7%
2/12 • Number of events 2 • 12 months
|
|
Gastrointestinal disorders
Vomiting
|
16.7%
2/12 • Number of events 3 • 12 months
|
|
Gastrointestinal disorders
Abdominal distension
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Gastrointestinal disorders
Abdominal pain
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Metabolism and nutrition disorders
Anorexia
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
8.3%
1/12 • Number of events 1 • 12 months
|
|
Investigations
Blood bilirubin increased
|
8.3%
1/12 • Number of events 3 • 12 months
|
|
General disorders
Fatigue
|
83.3%
10/12 • Number of events 12 • 12 months
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
75.0%
9/12 • Number of events 19 • 12 months
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
50.0%
6/12 • Number of events 7 • 12 months
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
41.7%
5/12 • Number of events 5 • 12 months
|
|
Investigations
Neutrophil count decreased
|
41.7%
5/12 • Number of events 16 • 12 months
|
|
Blood and lymphatic system disorders
Anemia
|
33.3%
4/12 • Number of events 6 • 12 months
|
|
Gastrointestinal disorders
Constipation
|
33.3%
4/12 • Number of events 4 • 12 months
|
|
Gastrointestinal disorders
Diarrhea
|
33.3%
4/12 • Number of events 5 • 12 months
|
|
Nervous system disorders
Headache
|
33.3%
4/12 • Number of events 4 • 12 months
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
33.3%
4/12 • Number of events 4 • 12 months
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
33.3%
4/12 • Number of events 5 • 12 months
|
|
Gastrointestinal disorders
Nausea
|
33.3%
4/12 • Number of events 6 • 12 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place