Trial · NCT01711632

BRAF Inhibitor, Vemurafenib, in Patients With Relapsed or Refractory Hairy Cell Leukemia

NCT01711632, Phase 2, Completed, Memorial Sloan Kettering Cancer Center; results first posted 2025-10-20

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. BRAF Inhibitor, Vemurafenib, in Patients With Relapsed or Refractory Hairy Cell Leukemia. Trials, record NCT01711632. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01711632

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Trial

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

Title: BRAF Inhibitor, Vemurafenib, in Patients With Relapsed or Refractory Hairy Cell Leukemia
NCT number: NCT01711632
Status: Completed
Phases: Phase 2
Enrollment: 36 participants, actual
Lead sponsor: Memorial Sloan Kettering Cancer Center
Conditions: Hairy Cell Leukemia
Interventions: Vemurafenib
First posted: 2012-10-22
Last updated: 2025-10-20
Results first posted: 2025-10-20

Source ClinicalTrials.gov

Registered outcomes4

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

List	Outcome	Time frame	Description
Primary	Efficacy of Vemurafenib	3 months	as assessed by overall response rates after three months of treatment in patients with relapsed or refractory HCL. Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR
Secondary	Toxicity (Safety and Tolerability)	2 years	Toxicity will be graded and recorded using the NCI Common Toxicology Criteria version 4.0.
Secondary	To Assess the Pharmacodynamics Change From Baseline	2 years	Peripheral blood and/or bone marrow aspirate samples from pretreatment and post-treatment at specified time points will be assessed by Western Blot or by phospho-flow for the downstream targets of BRAF (MEK, pMEK, ERK, pERK) to assess the ontarget effect of the Vemurafenib. The droplet digital PCR assay was performed to quantify the concentration of molecules with mutated BRAF V600E DNA at baseline and again at 12 weeks of vermurafenib therapy.
Secondary	Evaluate Biomarkers of Resistance	2 years	Reactivation of MAPK pathways: Increased expression of the other RAF isoforms CRAF and ARAF), and MAPK (MAPK8 or COT) will be analyzed by Western Blot and/or real-time PCR39,40. Secondary BRAF mutations (all 18 BRAF exons) and RAS mutations40 will be analyzed by bidirectional Sanger sequencing and by Raindance multiplex PCR and Illumina next generation sequencing, respectively. Activation of RTKs (i.e. PDGFRβ and IGF-IR) will be assessed by Western Blot. Cell Biosciences NanoPro 1000 technology will be used to examine quantitative signaling on the entire MAPK, PI3K and JAK-STAT pathways41.

Source ClinicalTrials.gov

Outcomes4

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

Type	Outcome	Time frame	Units	Measured as
PRIMARY	Efficacy of Vemurafenib	3 months	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Toxicity (Safety and Tolerability)	2 years	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	To Assess the Pharmacodynamics Change From Baseline	2 years	participants	NUMBER
SECONDARY	Evaluate Biomarkers of Resistance	2 years	Participants	COUNT_OF_PARTICIPANTS

Source ClinicalTrials.gov

Results by group11

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

Outcome	Group	Category	Value	Units
Efficacy of Vemurafenib	Vemurafenib	Complete Response	10	Participants
Efficacy of Vemurafenib	Vemurafenib	Partial Response	14	Participants
Efficacy of Vemurafenib	Vemurafenib	No CR or PR	12	Participants
Toxicity (Safety and Tolerability)	Vemurafenib	Dose reduction to 720mg twice daily	2	Participants
Toxicity (Safety and Tolerability)	Vemurafenib	Dose reduction to 480mg twice daily	10	Participants
Toxicity (Safety and Tolerability)	Vemurafenib	Dose reduction to 240mg twice daily	1	Participants
Toxicity (Safety and Tolerability)	Vemurafenib	Did not need a dose reduction	23	Participants
To Assess the Pharmacodynamics Change From Baseline	Vemurafenib	Pts with a decrease of the concentration of molecules with mutated BRAF V600E DNA at 12 wks	24	participants
To Assess the Pharmacodynamics Change From Baseline	Vemurafenib	Pts without decrease of the concentration of molecules with mutated BRAF V600E DNA at 12 wks	12	participants
Evaluate Biomarkers of Resistance	Vemurafenib	Participants with KRAS mutations	1	Participants

1 to 10 of 11Source ClinicalTrials.gov

See all 11 results by group

Participant flow3

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

Period	Milestone	Group	Participants
Overall Study	STARTED	Vemurafenib	36
Overall Study	COMPLETED	Vemurafenib	14
Overall Study	NOT COMPLETED	Vemurafenib	22

Source ClinicalTrials.gov

Adverse events38

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
other	Hyperglycemia	Metabolism and nutrition disorders	Vemurafenib	21	36
other	Platelet count decreased	Blood and lymphatic system disorders	Vemurafenib	21	36
other	Anemia	Blood and lymphatic system disorders	Vemurafenib	20	36
other	Neutrophil count decreased	Investigations	Vemurafenib	19	36
other	White blood cell decreased	Investigations	Vemurafenib	19	36
other	Lymphocyte count decreased	Investigations	Vemurafenib	17	36
other	Blood bilirubin increased	Investigations	Vemurafenib	14	36
other	Alkaline phosphatase increased	Investigations	Vemurafenib	12	36
other	Alanine aminotransferase increased	Investigations	Vemurafenib	11	36
other	Hypoalbuminemia	Metabolism and nutrition disorders	Vemurafenib	10	36

1 to 10 of 38Source ClinicalTrials.gov

See all 38 adverse events

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Vemurafenib	7	36
Total, other adverse events	Vemurafenib	22	36
Total, serious adverse events	Vemurafenib	13	36

Source ClinicalTrials.gov

Papers2

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
26352686	DERIVED	PubMed
35930750	DERIVED	PubMed

Grants0

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.

None in this release.

Trial comparisons0

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

None in this release.

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.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT01711632

GET https://conductscience.org/api/trials/v1/NCT01711632/events?page=2

API reference For AI assistants