Cognitive Status, Fatigue and Inflammation in Patients With Immune Thrombocytopenia (ITP)

Recruiting

• Conditions: Chronic Immune Thrombocytopenia; Primary Immune Thrombocytopenia; Adult Immune Thrombocytopenia

• Registration Number: NCT06665308
• Lead Sponsor: Sanofi

Brief Summary

This is a multi-center, hospital based, cross-sectional study based on data from patient medical records, including laboratory results. The study will include adult patients with chronic (\> 1 year duration) primary Immune Thrombocytopenia (ITP) only. Patients will be identified based on a laboratory confirmed diagnosis of ITP in the medical records, where there is also a physician-confirmed diagnosis of primary ITP. If patients consent to take part in the study, a routine clinical visit will also serve as the study visit.

All study participants will have a routine blood test as part of their standard clinical care at the study visit, and this same procedure will be used to collect supplementary blood samples to assess a variety of biomarkers.

Data will be collected using a combination of chart review, clinical outcome assessment administration, and laboratory results through blood tests.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-10-10
• Study First Submitted QC: 2024-10-29
• Study First Posted: 2024-10-30
• Study Start: 2025-01-13
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-01
• Last Update Posted: 2025-04-02
• Primary Completion: 2025-05-23
• Study Completion: 2025-05-23

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 110

Inclusion Criteria

• Patient diagnosed with chronic (ITP with > 1 year duration) primary ITP living in the USA or the UK:
• On ITP pharmacologic treatment, regardless of last platelet count (below or above 100 x 10^9 counts/L);
• Patients in remission as per their most recent blood test (platelet count >100 x 10^9 counts/L) and without ITP-specific treatment for less than 1 year
• Adult patient who are 18 years or older at index date
• Patient who has received at least one initial first line therapy (corticosteroids [CS]/ intravenous immunoglobulin [IVIg]/ANTI d) with initial response (platelet count ≥ 50 x 10^9 counts/L) as of index date

Exclusion Criteria

• Secondary ITP
• Patients in remission as per their most recent blood test (platelet count >100 x 10^9 counts/L) and without ITP-specific treatment for 1 year or more
• Patients with recent or active infection recorded 14 days or less before index date; patients with uncontrolled chronic infections or who were recently diagnosed with a chronic infection (≤14 days of index date).
• Patients with vaccination in 28 days before index date
• Patients treated with rilzabrutinib on or before index date

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Prevalence of impaired cognitive status among adult Immune Thrombocytopenia patients	Day 1 (Index date)	Impaired cognitive status is defined as a CBB (CogState Brief Battery) z-score of -1 or less on at least one of 2 composite domains (attention/psychomotor composite and memory/learning composite domains).; The CBB is a computerized outcome measure of cognitive function in human subjects aged 6 to 99 years and has been previously used in ITP. The CBB test for psychomotor function, attention, visual learning and working memory.

Secondary Outcome Measures

Name	Time	Method
Age, sex, ethnicity, educational attainment	Day 1 (Index date)
Smoking, alcohol, marijuana, cocaine use	Day 1 (Index date)
ITP Bleeding Scale (IBLS)	Day 1 (Index date)
FACIT (Functional Assessment of Chronic Illness Therapy) - Fatigue score	Day 1 (Index date)	The FACIT-Fatigue is a 13-item tool that measures self-reported fatigue and its impact on daily life. Each question is rated by the participant on a Likert-like scale of 0 ('not at all') to 4 ('very much'). A higher FACIT-Fatigue score represents greater quality of life (QoL).
Comorbidities at index categorical	Day 1 (Index date)
Medical history	Day 1 (Index date)	Family history of dementia, family history of cognitive impairment, ITP medication and treatment history, age of ITP diagnosis, duration of ITP, history of clinically relevant or severe bleeding complications, history of thromboembolism complications, history of brain injury or neurological sequalae, history of COVID-19, brain imaging results, platelet counts results
Current medications	Day 1 (Index date)	ITP-specific treatments, antiplatelet,anticoagulant, psychiatric and pain medications
Laboratory characteristics	Day 1 (Index date)	Complete Blood Count
ITP-PAQ	Day 1 (Index date)	A validated, disease-specifictool to measure health-related quality of life HRQoL in adult ITP patients
Assessment of key inflammatory biomarkers	Day 1 (Index date)	Transferrin, Ferritin, Iron, Prothrombin fragment, Fibrinogen, D dimer, Interleukin (IL)-1 beta, IL-6, IL-10, IL-18, TNF alpha, Gamma Interferon, high-sensitivity C-reactive protein (hsCRP), MCP1, NETosis markers (cell-free deoxyribonucleic acid (DNA), neutrophil elastase (NE), myeloperoxidase (MPO)), antinuclear antibodies (ANA), Lupus anticoagulant, Anticardiolipin antibodies (ACA), thrombopoietin level, soluble P-selectin, L-selectin, ICAM-1; whole blood RNA sequencing (transcriptome), C1q, sC5b-9

Trial Locations

Locations (4)

Massachusetts General Hospital- Site Number : 8400001; 🇺🇸 Boston, Massachusetts, United States

Investigational Site Number: 8260001; 🇬🇧 London, City Of London, United Kingdom

USC Norris Comprehensive Cancer Center- Site Number : 8400002; 🇺🇸 Los Angeles, California, United States

University of Washington-Fred Hutchinson Cancer Center_Site Number: 8400003; 🇺🇸 Seattle, Washington, United States