NCT03127709

Brief Summary

The purpose of this study is to try to understand how histopcytosis can cause symptoms or problems in the brain. The tests being done in the study will look at memory and thinking as well as brain function via MRI scan.

Trial Health

55
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
13

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Apr 2017

Longer than P75 for all trials

Geographic Reach
1 country

4 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

April 7, 2017

Completed
4 days until next milestone

First Submitted

Initial submission to the registry

April 11, 2017

Completed
14 days until next milestone

First Posted

Study publicly available on registry

April 25, 2017

Completed
8.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2026

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2026

Completed
Last Updated

May 2, 2025

Status Verified

May 1, 2025

Enrollment Period

9 years

First QC Date

April 11, 2017

Last Update Submit

May 1, 2025

Conditions

Keywords

17-199histiocytosis

Outcome Measures

Primary Outcomes (3)

  • neurocognitive function

    Neurocognitive tests will yield raw scores that are converted to z-scores, normalized for participant's age, sex and educational level.The proportion of participants demonstrating cognitive impairment (as defined by 2 or more z-scores less than -1.5) will be summarized. Also, the proportion of participants with impairment (z \< -1.5) for each cognitive test will be summarized. In this way the cognitive assessment yields a dichotomous outcome of impaired versus not impaired for each participant but also allow for more granular assessment of impairment in specific cognitive domains.

    1 year

  • quality of life

    questionnaires will yield raw scores (total score for HADS and McGill QOL, as well as anxiety and depression subscores for the HADS). QOL scores will be analyzed as raw scores. Cognitive test scores will be transformed into z-sores (based on score distributions from established normative samples with a mean of 0 and standard deviation of 1) to define the presence/severity of cognitive dysfunction. Scores will be normalized for age, sex and education.

    1 year

  • comparing of grey matter volume

    In a whole-brain analysis, first, cortical thickness will be compared between participants and controls and regions of statistically significant grey matter loss will be identified. This is done computionally with a whole-brain, vertex-by-vertex approach, as stated above. Also, as stated above (Section 7), statistical significance thresholding is at p\<0.001, correcting for multiple comparisons using the False Discovery Rate (FDR) method and clustering thresholds, methods used in various high-throughput contexts, including MRI analysis.

    1 year

Study Arms (1)

Participants with a histiocytic disorder diagnosis

Participants will have a histiocytic disorder as determined by a corroborating constellation of histopathology, clinical, and/or radiologic findings.

Behavioral: Trail Making Test, Parts A & BBehavioral: Brief Test of AttentionBehavioral: Symbol SpanBehavioral: Controlled Oral Word Association TestBehavioral: The Hopkins Verbal Learning Test-RevisedBehavioral: Brief Visuospatial Memory Test-RevisedBehavioral: Hospital Anxiety and Depression ScaleBehavioral: McGill Quality of Life ScaleDiagnostic Test: MR Brain ImagingDiagnostic Test: Resting state functional MRIBehavioral: Functional Assessment of Cancer Therapy Cognitive Scale (FACT-Cog)

Interventions

Part A is a timed measure of visual scanning and graphomotor speed. Part B is a timed measure of cognitive flexibility.

Participants with a histiocytic disorder diagnosis

Assesses auditory working memory

Also known as: BTA
Participants with a histiocytic disorder diagnosis
Symbol SpanBEHAVIORAL

Assesses visual working memory

Also known as: WMS-IV
Participants with a histiocytic disorder diagnosis

The COWA timed test of phonemic verbal fluency. It requires the subject to generate as many words as possible, beginning with a given letter of the alphabet (i.e., F, A, and S).

Also known as: COWA
Participants with a histiocytic disorder diagnosis

HVLT-R is a test of verbal learning and memory. Scores obtained are the total number of words.

Also known as: HVLT-R
Participants with a histiocytic disorder diagnosis

The BVMT-R is a test of visuospatial learning and memory.

Also known as: BVMT-R
Participants with a histiocytic disorder diagnosis

The HADS is a brief assessment validated in many cancer populations.

Also known as: HADS
Participants with a histiocytic disorder diagnosis

This is a questionnaire that is a validated 17-item QOL instrument validated for ill patients and in the particular context of the ancer.

Also known as: McGill QOL
Participants with a histiocytic disorder diagnosis
MR Brain ImagingDIAGNOSTIC_TEST

Standard of care brain MR imaging includes high-resolution 3D T1-weighted images in the axial plane. The axial T1-weighted images will be isotropic with a typical matrix size of 256 x 256, 256 mm field-of-view (FOV) and 1 mm slice thickness covering the whole brain.

Participants with a histiocytic disorder diagnosis

Scanning will be performed on a 3 Tesla General Electric scanner (Optima 750W) with a GEM HNU 24-channel head coil. rsFMRI matching the FLAIR and T1 post-contrast images will be obtained. For resting state fMRI, T2\*-weighted images will be acquired with a single-shot gradient echo echo-planar imaging (EPI) sequence in the axial orientation (TR = 2500 ms, TE = 30 ms, FA = 80°, slice thickness = 4 mm, FOV= 240mm2, matrix=64×64) covering whole brain. For the resting state fMRI portion of the scan, patients will be instructed by the technologist or research staff to leave eyes open, focus on looking at a crosshair, and not think about anything during the scan.

Also known as: rsFMRI
Participants with a histiocytic disorder diagnosis

This is a validated 37-item QOL instrument for cancer patients with cognitive complaints. The measures consist of FACT-Cog total, perceived cognitive impairments, perceived cognitive abilities, and impact on quality of life.

Participants with a histiocytic disorder diagnosis

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Participants will have a diagnosis of a histiocytic disorder as determined, in the opinion of the study PI, by a corroborating constellation of histopathology, clinical, and/or radiologic findings.

You may qualify if:

  • Age greater than or equal to 18 years.
  • Fluency in English
  • Diagnosis of a histiocytic disorder as determined, in the opinion of the study PI, by a corroborating constellation of histopathology, clinical, and/or radiologic findings.
  • Will undergo Standard of Care MRI.

You may not qualify if:

  • Known intracranial involvement of histiocytosis (including dura, leptomeninges and brain parenchyma)
  • Prior stroke or intracranial hemorrhage
  • Other (non-histiocytic) intracranial neoplasm or neurological disorder deemed by the PI or Co-PI to confound neuroimaging studies (e.g., demyelinating disease)
  • Existing diagnosis of a psychiatric disorder or untreated mood disturbance
  • Existing diagnosis of a neurodegenerative disease, such as Alzheimer's disease
  • Chronic or daily excessive alcohol consumption as determined by the PI.
  • History of chronic use of corticosteroids, defined as continuous treatment for six months or longer at any time in the past
  • History of severe claustrophobia or other contraindications to patient SOC brain MRI
  • Prior intravenous cytarabine or cladribine
  • Other current or prior treatments (e.g., high-dose chemotherapy for a different cancer) deemed by the PI or Co-PI to confound imaging studies or cognitive performance

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

Memorial Sloan Kettering Basking Ridge

Basking Ridge, New Jersey, 07920, United States

Location

Memorial Sloan Kettering Monmouth

Middletown, New Jersey, 07748, United States

Location

Memorial Sloan Kettering Cancer Center @ Suffolk

Commack, New York, 11725, United States

Location

Memorial Sloan Kettering Westchester

Harrison, New York, 10604, United States

Location

Related Links

MeSH Terms

Conditions

Histiocytosis

Interventions

Trail Making TestWechsler ScalesNeuropsychological Tests4-amino-4'-hydroxylaminodiphenylsulfone

Condition Hierarchy (Ancestors)

Lymphatic DiseasesHemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Psychological TestsBehavioral Disciplines and ActivitiesIntelligence TestsAptitude Tests

Study Officials

  • Eli Diamond, MD

    Memorial Sloan Kettering Cancer Center

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 11, 2017

First Posted

April 25, 2017

Study Start

April 7, 2017

Primary Completion

April 1, 2026

Study Completion

April 1, 2026

Last Updated

May 2, 2025

Record last verified: 2025-05

Locations