Trial · NCT02415608

Ibrutinib in Treating Patients With Advanced Systemic Mastocytosis

NCT02415608, Phase 2, Terminated, Jason Robert Gotlib; results first posted 2018-09-20

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Ibrutinib in Treating Patients With Advanced Systemic Mastocytosis. Trials, record NCT02415608. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02415608

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Ibrutinib in Treating Patients With Advanced Systemic Mastocytosis
NCT number: NCT02415608
Status: Terminated
Phases: Phase 2
Enrollment: 4 participants, actual
Lead sponsor: Jason Robert Gotlib
Conditions: Aggressive Systemic Mastocytosis, Mast Cell Leukemia, Systemic Mastocytosis
Interventions: Ibrutinib
First posted: 2015-04-14
Last updated: 2018-09-20
Results first posted: 2018-09-20

Source ClinicalTrials.gov

Registered outcomes11

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Overall Response Rate (ORR)	Up to 6 months	Overall response rate (ORR) is reported as the sum of the rates of participants achieving complete remission (CR), partial remission (PR), \& clinical improvement (CI). A clinical response is a response with duration of ≥ 12 weeks. CR is defined as all 4 criteria: * No presence of compact neoplastic mast cell aggregates * Serum tryptase level \< 20 ng/mL * Peripheral blood count remission defined as absolute neutrophil count (ANC) ≥1 x 10e9/L + normal differential, Hb ≥11 g/dL, \& platelet count ≥100x10e9/L * Complete resolution of palpable hepatosplenomegaly \& all biopsy-proven or suspected SM-related organ damage PR is defined as all 3 criteria with response duration ≥12 weeks, that is not CR or progressive disease: * ≥ 50% reduction in neoplastic mast cells * Serum tryptase level reduced ≥50% * Resolution of 1+ biopsy-proven or suspected systemic mastocytosis (SM)-related organ damage findings CI is defined as any improvement in any of the above measures.
Secondary	Number of Participants With Adverse Events	30 days	Adverse events will be graded according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.03, and reported as the number and percentage of participants having any adverse event; by each grade of adverse event; and by affected body systems.
Secondary	Ibrutinib Pharmacokinetics (PK)	28 days	Plasma concentration-time profiles for each subject and mean plasma concentration-time profiles for each dose level will be plotted, plasma concentration data for ibrutinib at each time point will be summarized by descriptive statistics, and PK parameters such as maximum concentration (Cmax), minimum concentration, time at which the Cmax is reached, and area under the curve will be summarized with mean, geometric mean, medium, minimum, maximum, standard deviation, and coefficient of variation.
Secondary	Change of Mast Cell Burden	2 years	The change in the number of neoplastic mast cells in tissues (blood and/or bone marrow), ie, a measure of mast cell burden, will be assessed by immunophenotyping and/or immunohistochemistry (depending on patient and disease specifics) using mast cell markers, eg, CD25, CD30, CD117, tryptase, reticulin, Wright-Giemsa staining, and/or hematoxylin-eosin staining, in peripheral blood smears or bone marrow samples. For each participant, the data are used to collectively determine a single assessment for the number of mast cells present at baseline and after treatment. The outcome is reported as the median change in that level of mast cells, with full range, from baseline up to 2 years.
Secondary	Serum Tryptase Levels	2 years	Serum tryptase level is a surrogate marker for the desired histopathologic response, ie, reduction in mast cell burden. Serum tryptase levels are reported as the median of the percent reduction, with full range, from baseline up to 2 years.
Secondary	Total Symptom Score (TSS)	30 days	The totality of systemic mastocytosis was assessed by the total symptom score as measured by a Myeloproliferative Neoplasm Symptom Assessment Form modified for mast cell disorders \[MPN-SAF (MCD)\], and reported as the change in median score with standard deviation at baseline and 30 days. The MPN-SAF is a single, 27-question questionnaire that scores the following general measures on a scale of 0 (best) to 10 (worst): fatigue levels, effects of fatigue, satiety, pain, activity, concentration, dizziness, sleep, mood, anxiety, sexual function, itching, flushing, fever, weight loss, respiratory functions, diarrhea, lesions, and allergic reactions (some of these general terms may describe more than 1 assessment). The score on the MPN-SAF is the sum total of all 27 scores, and the range of scores is from a minimum of 0 (best; symptoms for all assessment absent) to a maximum of 270 (worst; score of 10 on all assessments).
Secondary	Change in Quality of Life (QoL)	30 days	The quality of life (QoL) component of the Myeloproliferative Neoplasm Symptom Assessment Form (MPNSAF) modified for mast cell symptoms, a scale of life quality ranking from 0 (best) to 10 (worst), was assessed at baseline and after 1 cycle of ibrutinib treatment (30 days), and reported as the median change in score with standard deviation.
Secondary	Duration of Response (DoR)	2 years	Duration of response (DoR) was assessed through 2 years of treatment, and reported as the median with standard deviation, with response duration censored at last response assessment in the event of death or progression not documented.
Secondary	Time-to-Response (TTR)	2 years	Time-to-response (TTR) was assessed through 2 years of treatment, and reported as the median with standard deviation, censored at last response assessment in the event of death or progression not documented.
Secondary	Progression-free Survival (PFS)	2 years	Participants were assessed for progression-free survival (PFS) from the start of treatment through 2 years of treatment. The outcome is reported as the number of participants who were alive without disease progression after 2 years of treatment.

1 to 10 of 11Source ClinicalTrials.gov

See all 11 registered outcomes

Outcomes11

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Overall Response Rate (ORR)	Up to 6 months		Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Number of Participants With Adverse Events	30 days	All study participants are included in this analysis.	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Ibrutinib Pharmacokinetics (PK)	28 days	Because this study terminated with low total accrual, the funding sponsor elected not to analyze the samples for ibrutinib levels. There are no pharmacokinetics values on which to conduct the outcome analysis.
SECONDARY	Change of Mast Cell Burden	2 years	The result was only calculated for those participants for whom a post-treatment mast cell level could be determined.	Percent reduction of mast cells	MEDIAN
SECONDARY	Serum Tryptase Levels	2 years	Results were determined for all participants.	Percent reduction serum tryptase level	MEDIAN
SECONDARY	Total Symptom Score (TSS)	30 days	Results were analyzed for all participants.	score on a scale	MEDIAN
SECONDARY	Change in Quality of Life (QoL)	30 days	Results were analyzed for all participants.	score on a scale	MEDIAN
SECONDARY	Duration of Response (DoR)	2 years	No participants achieved clinical response per protocol (minimum of any clinical improvement ≥ 12 weeks). On that basis, the overall duration of that response can not be determined.
SECONDARY	Time-to-Response (TTR)	2 years	No participants achieved clinical response per protocol (minimum of any clinical improvement ≥ 12 weeks). On that basis, the time to achieve per-protocol clinical response can not be determined.
SECONDARY	Progression-free Survival (PFS)	2 years	Results were analyzed for all participants. Values were censored at the last assessment if the participant was lost-to-follow-up or otherwise did not have a 2-year assessment.	Participants	COUNT_OF_PARTICIPANTS

1 to 10 of 11Source ClinicalTrials.gov

See all 11 outcomes

Results by group49

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Units
Overall Response Rate (ORR)	Ibrutinib 420 mg/Day		0	Participants
Overall Response Rate (ORR)	Ibrutinib 560 mg/Day		0	Participants
Number of Participants With Adverse Events	Ibrutinib 420 mg/Day	Any Adverse event	3	Participants
Number of Participants With Adverse Events	Ibrutinib 560 mg/Day	Any Adverse event	1	Participants
Number of Participants With Adverse Events	Ibrutinib 420 mg/Day	Any Grade 1 Adverse Event (mild)	3	Participants
Number of Participants With Adverse Events	Ibrutinib 560 mg/Day	Any Grade 1 Adverse Event (mild)	1	Participants
Number of Participants With Adverse Events	Ibrutinib 420 mg/Day	Any Grade 2 Adverse Event (moderate)	3	Participants
Number of Participants With Adverse Events	Ibrutinib 560 mg/Day	Any Grade 2 Adverse Event (moderate)	1	Participants
Number of Participants With Adverse Events	Ibrutinib 420 mg/Day	Any Grade 3 Adverse Event (severe)	2	Participants
Number of Participants With Adverse Events	Ibrutinib 560 mg/Day	Any Grade 3 Adverse Event (severe)	1	Participants

1 to 10 of 49Source ClinicalTrials.gov

See all 49 results by group

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Ibrutinib 420 mg/Day	3
Overall Study	STARTED	Ibrutinib 560 mg/Day	1
Overall Study	COMPLETED	Ibrutinib 420 mg/Day	3
Overall Study	COMPLETED	Ibrutinib 560 mg/Day	1
Overall Study	NOT COMPLETED	Ibrutinib 420 mg/Day	0
Overall Study	NOT COMPLETED	Ibrutinib 560 mg/Day	0

Source ClinicalTrials.gov

Adverse events58

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Anemia	Blood and lymphatic system disorders	Ibrutinib 420 mg/Day	1	3	1
other	Anemia	Blood and lymphatic system disorders	Ibrutinib 560 mg/Day	1	1	1
other	Diarrhea	Gastrointestinal disorders	Ibrutinib 420 mg/Day	1	3	1
other	Diarrhea	Gastrointestinal disorders	Ibrutinib 560 mg/Day	1	1	1
other	Mouth sores	Gastrointestinal disorders	Ibrutinib 420 mg/Day	1	3	1
other	Mouth sores	Gastrointestinal disorders	Ibrutinib 560 mg/Day	0	1	0
other	Tongue sensitivity	Gastrointestinal disorders	Ibrutinib 420 mg/Day	0	3	0
other	Tongue sensitivity	Gastrointestinal disorders	Ibrutinib 560 mg/Day	1	1	1
other	Vomiting	Gastrointestinal disorders	Ibrutinib 420 mg/Day	1	3	1
other	Vomiting	Gastrointestinal disorders	Ibrutinib 560 mg/Day	0	1	0

1 to 10 of 58Source ClinicalTrials.gov

See all 58 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Ibrutinib 420 mg/Day	1	3
Total, all-cause mortality	Ibrutinib 560 mg/Day	0	1
Total, other adverse events	Ibrutinib 420 mg/Day	3	3
Total, other adverse events	Ibrutinib 560 mg/Day	1	1
Total, serious adverse events	Ibrutinib 420 mg/Day	1	3
Total, serious adverse events	Ibrutinib 560 mg/Day	0	1

Source ClinicalTrials.gov

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

Grants1

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

Project	Title	Principal investigators	Organization	Funder	Fiscal years
P30CA124435	Stanford Cancer Institute	ARTANDI, STEVEN E	STANFORD UNIVERSITY	NATIONAL CANCER INSTITUTE	2007 to 2025

Source NIH RePORTER

Trial comparisons4

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Ibrutinib 420 mg/Day vs Ibrutinib 560 mg/Day: Progression-free Survival (PFS)	Trial comparisons
Ibrutinib 420 mg/Day vs Ibrutinib 560 mg/Day: Overall Response Rate (ORR)	Trial comparisons
Ibrutinib 420 mg/Day vs Ibrutinib 560 mg/Day: deaths from any cause	Trial comparisons
Ibrutinib 420 mg/Day vs Ibrutinib 560 mg/Day: serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

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