A Clinical Study to Investigate Interferon Gamma (IFNɣ) Signature in Patients Post HSCT and in Patients With Impaired HSC Proliferation Pre-transplant

NCT04494061 | Terminated

• 1 other identifier: NI-0501-13
• Study Type: observational
• Target: 101
• Locations: 6 countries
• Sites: 25

Brief Summary

Clinical study designed to collect blood for research purposes in patients after hematopoietic stem cell transplantation (HSCT) or in patients with a medical condition where the blood cells production is impaired. The blood samples will be used to study the role of Interferon gamma (IFNɣ) in graft failure or impairment of hematopoietic stem cell proliferation. The IFNɣ signature will be assessed by measuring primarily IFNɣ and C-X-C Motif Chemokine Ligand 9 (CXCL9).

Conditions

Graft Failure

Outcome Measures

Primary Outcomes (4)

• HSCT cohort: IFNγ signature pre-and post-transplant

  IFNγ, CXCL-9 and exploratory biomarkers in serum samples

  Day (-7) to day 100

• HSCT cohort: Relationship between IFNγ and the risk of graft failure

  IFNγ and CXCL-9 in serum samples

  Day (-7) to day 100

• HSCT cohort: Relationship between IFNγ and the occurrence of GVHD

  IFNγ and CXCL-9 in serum samples

  Day (-7) to day 100

• IHSCP cohort: IFNγ signature pre-transplant

  IFNγ, CXCL-9 and exploratory biomarkers in serum samples

  Day 1

Study Arms (2)

HSCT - Hematopoetic Stem Cell Proliferation

Patients who received hematopoietic stem cell transplant

Procedure: blood collection

IHSCP - Impaired HSC proliferation

Patients with impaired hematopoietic stem cell proliferation

Procedure: blood collection

Interventions

blood collection PROCEDURE

Blood samples will be collected as per protocol defined schedule. There is no investigation drug in this study.

HSCT - Hematopoetic Stem Cell Proliferation; IHSCP - Impaired HSC proliferation

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

* Patients post HSCT at risk of graft failure based on their underlying diseases and on the transplant procedure. * Patients with conditions where HSC proliferation is impaired (e.g. aplastic anemia) and with respective controls (healthy volunteers (HV)).

You may qualify if:

• The patient must have consented to the use of their clinical data and biological samples for research investigations.
• In HSCT cohort:
• Patients with underlying:
• i. non-malignant hematological disease (e.g. autoimmune and metabolic disorders, aplastic anemia, Sickle cell anemia, Fanconi anemia, Diamond-blackfan anemia, thalassemia, osteopetrosis, Wiskott-Aldrich syndrome, severe combined immunodeficiency) or ii. malignant disease with higher risk of GF, i.e. Acute Myeloid Leukemia (AML) and Acute Lymphoblastic Leukemia (ALL) with primary induction failure, second partial remission or relapse; Chronic Myeloid Leukemia (CML) in blastic phase (circulating blast or blast above 5% in biopsy); Non Hodgkin and Hodgkin Lymphoma and multiple myeloma with primary induction failure, second partial remission or relapse, myelodysplastic syndromes (MDS) and myeloproliferative disorders (MPD) with splenomegaly, myelofibrosis with portal hypertension pre-transplant, MDS/MPD overlap syndromes
• and who received allogeneic HSCT and are at higher risk of graft failure based on at least one of the following criteria: i. Having received reduced intensity conditioning (RIC) or non myeloablative conditioning (NMA) combined with a non-malignant disease or having received graft from Bone Marrow (BM) ii. Ex vivo T cell depleted graft iii. Graft from mismatched unrelated donor or haploidentical donor iv. Graft from Umbilical Cord Blood (UCB)
• In the IHSCP cohort:
• Patients with IHSCP pre-transplant (e.g. aplastic anemia)

You may not qualify if:

• HLH patients
• Body weight \< 10kg

Sponsors & Collaborators

• Swedish Orphan Biovitrum: lead
• PRA Health Sciences: collaborator
• Cytel Inc.: collaborator
• BioMérieux: collaborator

Study Sites (25)

Algemeen Ziekenhuis Delta - Campus Rumbeke

Roeselare, West-Vlaanderen, 8800, Belgium

Location

Cliniques Universitaires Saint-Luc

Brussels, 1200, Belgium

Location

Centre Hospitalier Universitaire Grenoble Alpes

La Tronche, Auvergne-Rhône-Alpes, 38700, France

Location

Hôpital Côte De Nacre

Caen, Basse-Normandie, 14033, France

Location

Hôpital Pontchaillou

Rennes, Brittany Region, 35033, France

Location

Centre Hospitalier Régional et Universitaire de Besançon - Hôpital Jean-Minjoz

Besançon, Franche-Comte, 25030, France

Location

Hôpitaux de Brabois

Vandœuvre-lès-Nancy, Lorraine, 54511, France

Location

Centre Hosptitalier Universitaire d'Angers

Angers, Maine Et Loire, 49933, France

Location

Hôpital Haut-Lévêque

Pessac, Nouvelle-Aquitaine, 33604, France

Location

Hôpital Saint-Eloi

Montpellier Cedex 5, Provence-Alpes-Côte d'Azur Region, 34295, France

Location

Hôpital Arnaud de Villeneuve

Montpellier, Provence-Alpes-Côte d'Azur Region, 34090, France

Location

Centre Hospitalier Universitaire Estaing

Clermont-Ferrand, Rhône, 63003, France

Location

Hôpital Saint-Louis

Paris, Île-de-France Region, 75010, France

Location

Hôpital Universitaire Robert-Debré

Paris, Île-de-France Region, 75019, France

Location

Universitätsklinikum Tübingen

Tübingen, Baden-Wurttemberg, Germany

Location

Ospedale Pediatrico Bambino Gesù - Roma - Gianicolo

Roma, Lombardy, 00165, Italy

Location

Azienda Ospedaliera San Giuseppe Moscati

Avellino, 83100, Italy

Location

Instituto Giannina Gaslini

Genova, 16147, Italy

Location

Ospedale San Raffaele

Milan, 20132, Italy

Location

Fondazione IRCCS Policlinico San Matteo

Pavia, 27100, Italy

Location

Ospedale Regina Margherita

Torino, 10126, Italy

Location

Prinses Maxima Centrum Kinderoncologie

Utrecht, 3584 CS, Netherlands

Location

Cardiff and Vale University Health Board

Cardiff, Wales, CF14 4XW, United Kingdom

Location

The Royal Marsden Hospital - London

London, SW3 6JJ, United Kingdom

Location

Imperial College Healthcare NHS Trust NHS Trust

London, W12 0HS, United Kingdom

Location

MeSH Terms

Interventions

Blood Specimen Collection

Intervention Hierarchy (Ancestors)

Specimen Handling; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Punctures; Surgical Procedures, Operative; Investigative Techniques

Study Officials

• Regis Peffault de Latour, MD

  Hôpital Saint Louis Paris

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 28, 2020
• First Posted: July 31, 2020
• Study Start: November 16, 2020
• Primary Completion: August 31, 2022
• Study Completion: August 31, 2022
• Last Updated: October 21, 2022

Record last verified: 2022-10

Locations

NL (1); GB (3); FR (12); BE (2); DE (1); IT (6)