NCT06093529

Brief Summary

Immune thrombocytopenia (ITP) is a common autoimmune disease characterized by low platelet count and increased risk of bleeding. It affects approximately 50 to 100 cases per million people per year, with children accounting for half of the cases.

Trial Health

35
At Risk

Trial Health Score

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

Trial has exceeded expected completion date
Enrollment
42

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started Jan 2024

Status
unknown

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

First Submitted

Initial submission to the registry

October 17, 2023

Completed
6 days until next milestone

First Posted

Study publicly available on registry

October 23, 2023

Completed
2 months until next milestone

Study Start

First participant enrolled

January 1, 2024

Completed
1 year until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 31, 2024

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2025

Completed
Last Updated

November 14, 2023

Status Verified

November 1, 2023

Enrollment Period

1 year

First QC Date

October 17, 2023

Last Update Submit

November 10, 2023

Conditions

ITP - Immune Thrombocytopenia

Outcome Measures

Primary Outcomes (1)

  • Correlation analysis

    Analysis of the correlation between specific markers expression ( CD3,CD4,CD8,Interferon gamma and natural killer cells) and treatment response in pediatric patients with immune thrombocytopenia who are receiving second line therapy.

    One year

Secondary Outcomes (1)

  • Assessment of treatment response

    One year

Study Arms (2)

Respondents to second-line therapy

Immune markers in ITPpatients Respondant to second line therapy

Diagnostic Test: complete blood count, CD3+ , CD4+ , CD8+, CD16+, CD56+, IFN-γ.

Non-Respondents to second-line therapy

Immune markers in ITPpatients nonrespondant to second line therapy

Diagnostic Test: complete blood count, CD3+ , CD4+ , CD8+, CD16+, CD56+, IFN-γ.

Interventions

complete blood count, CD3+ , CD4+ , CD8+, CD16+, CD56+, IFN-γ.DIAGNOSTIC_TEST

1. Full history 2. Thorough clinical examinations 3. Laboratory investigations will include: 1. complete blood count with focus on platelet count, platelet distribution width and mean platelet volume. Platelet count will be confirmed by direct blood film and blood smear. 2. Measurements of CD3+, CD4+, CD8+ and natural killer cells (CD16+, CD56+) will be conducted using flow cytometry. 3. Serum IFN-γ levels will be determined using an ELISA kit. 4. Response to the treatment will be assessed according to The International Working Group criteria which defines Response as platelet count ≥ 30 x 10⁹/L and \>2-fold increase in platelet count from baseline and absence of bleeding, measured on 2 occasions greater than 7 days apart. No response is characterized by a platelet count \<30 x 10⁹/L or a less than 2-fold increase in platelet count from baseline, or the presence of bleeding.

Non-Respondents to second-line therapyRespondents to second-line therapy

Eligibility Criteria

AgeUp to 17 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)
Sampling MethodProbability Sample
Study Population

Pediatric patients with ITP who are receiving second-line therapy who are attending the outpatient pediatric hematology clinic at Assiut University Children Hospital or the clinic at Central Insurance Agency of Assiut.

You may qualify if:

  • Pediatric patients diagnosed with immune thrombocytopenia who are undergoing second line therapy with either eltrombopag or romiplostim.

You may not qualify if:

  • Include acute immune thrombocytopenic children receiving first line therapy and those with secondary immune thrombocytopenia.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Related Publications (14)

  • Kayal L, Jayachandran S, Singh K. Idiopathic thrombocytopenic purpura. Contemp Clin Dent. 2014 Jul;5(3):410-4. doi: 10.4103/0976-237X.137976.

    PMID: 25191085BACKGROUND

  • Zufferey A, Kapur R, Semple JW. Pathogenesis and Therapeutic Mechanisms in Immune Thrombocytopenia (ITP). J Clin Med. 2017 Feb 9;6(2):16. doi: 10.3390/jcm6020016.

    PMID: 28208757BACKGROUND

  • Cancro MP. The BLyS/BAFF family of ligands and receptors: key targets in the therapy and understanding of autoimmunity. Ann Rheum Dis. 2006 Nov;65 Suppl 3(Suppl 3):iii34-6. doi: 10.1136/ard.2006.058412.

    PMID: 17038469BACKGROUND

  • Kuwana M, Kaburaki J, Kitasato H, Kato M, Kawai S, Kawakami Y, Ikeda Y. Immunodominant epitopes on glycoprotein IIb-IIIa recognized by autoreactive T cells in patients with immune thrombocytopenic purpura. Blood. 2001 Jul 1;98(1):130-9. doi: 10.1182/blood.v98.1.130.

    PMID: 11418472BACKGROUND

  • Malik A, Sayed AA, Han P, Tan MMH, Watt E, Constantinescu-Bercu A, Cocker ATH, Khoder A, Saputil RC, Thorley E, Teklemichael A, Ding Y, Hart ACJ, Zhang H, Mitchell WA, Imami N, Crawley JTB, Salles-Crawley II, Bussel JB, Zehnder JL, Adams S, Zhang BM, Cooper N. The role of CD8+ T-cell clones in immune thrombocytopenia. Blood. 2023 May 18;141(20):2417-2429. doi: 10.1182/blood.2022018380.

    PMID: 36749920BACKGROUND

  • Pollard KM, Cauvi DM, Toomey CB, Morris KV, Kono DH. Interferon-gamma and systemic autoimmunity. Discov Med. 2013 Sep;16(87):123-31.

    PMID: 23998448BACKGROUND

  • HARRINGTON WJ, MINNICH V, HOLLINGSWORTH JW, MOORE CV. Demonstration of a thrombocytopenic factor in the blood of patients with thrombocytopenic purpura. J Lab Clin Med. 1951 Jul;38(1):1-10. No abstract available.

    PMID: 14850832BACKGROUND

  • Shulman NR, Marder VJ, Weinrach RS. Similarities between known antiplatelet antibodies and the factor responsible for thrombocytopenia in idiopathic purpura. Physiologic, serologic and isotopic studies. Ann N Y Acad Sci. 1965 Jun 30;124(2):499-542. doi: 10.1111/j.1749-6632.1965.tb18984.x. No abstract available.

    PMID: 5214832BACKGROUND

  • van Leeuwen EF, van der Ven JT, Engelfriet CP, von dem Borne AE. Specificity of autoantibodies in autoimmune thrombocytopenia. Blood. 1982 Jan;59(1):23-6.

    PMID: 7032627BACKGROUND

  • Ghanima W, Khelif A, Waage A, Michel M, Tjonnfjord GE, Romdhan NB, Kahrs J, Darne B, Holme PA; RITP study group. Rituximab as second-line treatment for adult immune thrombocytopenia (the RITP trial): a multicentre, randomised, double-blind, placebo-controlled trial. Lancet. 2015 Apr 25;385(9978):1653-61. doi: 10.1016/S0140-6736(14)61495-1. Epub 2015 Feb 5.

    PMID: 25662413BACKGROUND

  • Mahevas M, Azzaoui I, Crickx E, Canoui-Poitrine F, Gobert D, Languille L, Limal N, Guillaud C, Croisille L, Jeljeli M, Batteux F, Baloul S, Fain O, Pirenne F, Weill JC, Reynaud CA, Godeau B, Michel M. Efficacy, safety and immunological profile of combining rituximab with belimumab for adults with persistent or chronic immune thrombocytopenia: results from a prospective phase 2b trial. Haematologica. 2021 Sep 1;106(9):2449-2457. doi: 10.3324/haematol.2020.259481.

    PMID: 32817288BACKGROUND

  • Newland AC, Sanchez-Gonzalez B, Rejto L, Egyed M, Romanyuk N, Godar M, Verschueren K, Gandini D, Ulrichts P, Beauchamp J, Dreier T, Ward ES, Michel M, Liebman HA, de Haard H, Leupin N, Kuter DJ. Phase 2 study of efgartigimod, a novel FcRn antagonist, in adult patients with primary immune thrombocytopenia. Am J Hematol. 2020 Feb;95(2):178-187. doi: 10.1002/ajh.25680. Epub 2019 Dec 10.

    PMID: 31821591BACKGROUND

  • Faul F, Erdfelder E, Lang AG, Buchner A. G*Power 3: a flexible statistical power analysis program for the social, behavioral, and biomedical sciences. Behav Res Methods. 2007 May;39(2):175-91. doi: 10.3758/bf03193146.

    PMID: 17695343BACKGROUND

  • Rodeghiero F, Stasi R, Gernsheimer T, Michel M, Provan D, Arnold DM, Bussel JB, Cines DB, Chong BH, Cooper N, Godeau B, Lechner K, Mazzucconi MG, McMillan R, Sanz MA, Imbach P, Blanchette V, Kuhne T, Ruggeri M, George JN. Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group. Blood. 2009 Mar 12;113(11):2386-93. doi: 10.1182/blood-2008-07-162503. Epub 2008 Nov 12.

    PMID: 19005182BACKGROUND

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

Blood Cell CountCD4 Lymphocyte Count

Condition Hierarchy (Ancestors)

Purpura, ThrombocytopenicPurpuraBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesThrombotic MicroangiopathiesThrombocytopeniaBlood Platelet DisordersCytopeniaHemorrhagic DisordersAutoimmune DiseasesImmune System DiseasesHemorrhagePathologic ProcessesPathological Conditions, Signs and SymptomsSkin ManifestationsSigns and Symptoms

Intervention Hierarchy (Ancestors)

Cell CountCytological TechniquesClinical Laboratory TechniquesDiagnostic Techniques and ProceduresDiagnosisHematologic TestsInvestigative TechniquesCell Physiological PhenomenaBlood Physiological PhenomenaCirculatory and Respiratory Physiological PhenomenaLymphocyte CountLeukocyte Count

Study Officials

  • Khaled I El-sayeh, M.D.

    Assiut University

    STUDY DIRECTOR

  • Mostafa M Embaby, M.D.

    Assiut University

    STUDY DIRECTOR

  • Azhar A Mohammed, M.D.

    Assiut University

    STUDY DIRECTOR

Central Study Contacts

Gehad M Abdelsalam, M.Sc.

CONTACT

+201091294596drgehadabdelsalam@aun.edu.eg

Study Design

Study Type
observational
Observational Model
CASE ONLY
Time Perspective
CROSS SECTIONAL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Principle Investigator

Study Record Dates

First Submitted

October 17, 2023

First Posted

October 23, 2023

Study Start

January 1, 2024

Primary Completion

December 31, 2024

Study Completion

February 1, 2025

Last Updated

November 14, 2023

Record last verified: 2023-11