SNPMiner Trials by Shray Alag


SNPMiner Trials: Clinical Trial Report


Report for Clinical Trial NCT03794297

Developed by Shray Alag, 2019.
SNP Clinical Trial Gene

A Phase II Therapeutic Trial of the Use of Dabrafenib and Trametinib in Patients With BRAF V600 Mutation Positive Lesions in Erdheim Chester Disease

This phase II trial studies the side effects and how well dabrafenib and trametinib work in treating patients with Erdheim Chester disease that have BRAF V600 gene mutations. Dabrafenib and trametinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

NCT03794297 BRAF NP_004324.2:p.V600X Erdheim-Chester Disease
MeSH: Erdheim-Chester Disease

4 Interventions

Name: Dabrafenib Mesylate

Description: Given PO

Type: Drug

Treatment (dabrafenib mesylate, trametinib dimethyl sulfoxide)

Name: Quality-of-Life Assessment

Description: Ancillary studies

Type: Other

Treatment (dabrafenib mesylate, trametinib dimethyl sulfoxide)

Name: Questionnaire Administration

Description: Ancillary studies

Type: Other

Treatment (dabrafenib mesylate, trametinib dimethyl sulfoxide)

Name: Trametinib Dimethyl Sulfoxide

Description: Given PO

Type: Drug

Treatment (dabrafenib mesylate, trametinib dimethyl sulfoxide)


Primary Outcomes

Description: Measured by Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 criteria. Response to therapy will be evident on follow up imaging studies revealing a decrease in lesion size of 30% or more, which is defined as a partial response as per RECIST criteria, when compared to baseline studies.

Measure: Clinical response rate

Time: Up to 48 weeks after completion of study treatment

Description: Assessed using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.

Measure: Incidence of toxicities

Time: Up to 48 weeks after completion of study treatment

Secondary Outcomes

Description: Will be reported using Kaplan-Meier curves and appropriate 95% confidence intervals.

Measure: Time to response

Time: Up to 48 weeks after completion of study treatment

Description: Will be reported using Kaplan-Meier curves and appropriate 95% confidence intervals.

Measure: Duration of response

Time: From the time measurement criteria are met for complete or partial response (whichever is first recorded) until the first date that recurrent or progressive disease is objectively documented, assessed up to 48 weeks after completion of study treatment

Description: Will be reported using Kaplan-Meier curves and appropriate 95% confidence intervals.

Measure: Progression-free survival

Time: From start of treatment to time of progression or death, whichever occurs first, assessed up to 48 weeks after completion of study treatment

Description: Will be reported using Kaplan-Meier curves and appropriate 95% confidence intervals.

Measure: Overall survival

Time: Up to 48 weeks after completion of study treatment

Measure: Degree of histiocytic infiltration using fludeoxyglucose (FDG) positron emission tomography (PET)/computed tomography (CT) and bone scans

Time: Up to 48 weeks after completion of study treatment

Measure: Levels of C-reactive protein

Time: Up to 48 weeks after completion of study treatment

Measure: Levels of estrogen receptor (ESR)

Time: Up to 48 weeks after completion of study treatment

Measure: Levels of cytokines

Time: Up to 48 weeks after completion of study treatment

Description: Patients will be evaluated using the multi-dimensional fatigue inventory.

Measure: Change in fatigue

Time: Baseline up to 48 weeks after completion of study treatment

Description: Patients will be evaluated using the 6 minute walk test, single leg stance, and functional reach and grip strength using a dynamometer.

Measure: Change in level of functioning

Time: Baseline up to 48 weeks after completion of study treatment

Description: Patients will be evaluated using the human activity profile and activity card sort.

Measure: Change in ability to perform routine activities

Time: Baseline up to 48 weeks after completion of study treatment

Description: Patients will be evaluated using the comparative pain scale.

Measure: Change in pain

Time: Baseline up to 48 weeks after completion of study treatment

Description: Patients will be evaluated using the the group peg board test.

Measure: Change in fine motor dexterity

Time: Baseline up to 48 weeks after completion of study treatment

Description: Improvement of patients' overall quality of life will be evidence of response, and assessments will be made at baseline and throughout the trial, as well as at the conclusion of the trial, to evaluate for any improvement in quality of life. Patients will be evaluated using the National Institute of Neurological Disorders and Stroke-Neuro-Quality of Life scale.

Measure: Change in overall quality of life

Time: Baseline up to 48 weeks after completion of study treatment

Description: Resistance to therapy will be evaluated through imaging studies and patient follow up for at least one year, but this is not expected with the combination therapy.

Measure: Resistance to therapy

Time: Up to 1 year

Purpose: Treatment

Single Group Assignment


There is one SNP

SNPs


1 V600E

These potential risks may also apply to other agents used in this study - History of retinal vein occlusion (RVO) - Interstitial lung disease or pneumonitis not secondary to ECD - Central serous retinopathy (CSR) including presence of predisposing factors to RVO or CSR (e.g., uncontrolled glaucoma or ocular hypertension, uncontrolled diabetes mellitus, or a history of hyperviscosity or hypercoagulability syndromes); or visible pathology (e.g., evidence of optic disc cupping, evidence of new visual field defects on automated perimetry, or intraocular pressure > 21 mmHg as measured by tonography) as assessed by ophthalmic examination - Inability to travel to the treating center - Patients with confirmed diagnosis of ECD that are asymptomatic and with no visceral involvement are not eligible for this trial (patients with no target lesions as per RECIST 1.1 criteria) BRAF NP_004324.2:p.V600X Erdheim-Chester Disease Erdheim-Chester Disease PRIMARY OBJECTIVES: I. To study the efficacy and safety of dabrafenib mesylate (dabrafenib) and trametinib dimethyl sulfoxide (trametinib) as combination therapy in patients with BRAF V600E positive Erdheim Chester disease. --- V600E ---

To determine the clinical response rate to dabrafenib and trametinib combination therapy in patients with BRAF V600E positive Erdheim Chester disease. --- V600E ---

V. To establish duration of treatment-endpoints in patients with BRAF V600E positive Erdheim-Chester disease (ECD) lesions. --- V600E ---



HPO Nodes