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. Patients who do not have a routine visit scheduled during the study period but who would otherwise be eligible may be contacted by the site. In such cases, clinicians may use their discretion to schedule a visit specifically for study purposes.

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: 2025-11
• Last Update Submitted: 2025-11-05
• Last Update Posted: 2025-11-10
• Primary Completion: 2025-12-12
• Study Completion: 2025-12-12

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, , including on-demand treatment strategy, regardless of last platelet count (below or above 100 x 10^9 counts/L);
  • Patients with their most recent platelet count >50 x 109 counts/L) and without any ITP-specific treatment for 1 year or less.

• Adult patient who are 18 years or older at index date

• Patient who has received at least one initial first line ITP therapy (CS/ IVIg/ANTI d) with initial response as of index date and indicated by one of the following:

  • Platelet count ≥ 50 x 10^9 counts /L; OR
  • Platelet count ≥ 30 x 10^9 counts /L with at least a two-fold increase from baseline within 1 month since start of treatment, as per ASH 2019 guidelines; OR
  • A medical record of initial response as determined by clinical judgement, considering documented platelet count along with overall patient condition and relevant medical factors.

• Patients whose medical history related to the study objectives are available from the site covering 12 months prior to index date.

• Patient provided informed consent to participate in the study.

Exclusion Criteria

• Secondary ITP.
• Patients with their most recent platelet count >50 x 10^9 counts/L and without any ITP-specific treatment for more than 1 year.
• 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.
• Patients who participated in an interventional clinical trial for any investigational drug in the 180 days prior to 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
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
Age, sex, ethnicity, educational attainment	Day 1 (Index date)

Trial Locations

Locations (4)

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

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

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

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