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

October 19, 2022 updated by: Swedish Orphan Biovitrum
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).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Terminated

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

This clinical study is designed to investigate IFNγ activity in two cohorts of patients.

  • First group will include patients post HSCT at risk of graft failure (GF) based on their underlying diseases and on the transplant procedure.
  • Second group will contain patients with conditions where HSC proliferation is impaired (e.g. aplastic anemia) and with matched controls (healthy volunteers (HV) samples collected outside this clinical protocol).

IFNɣ activity will be assessed by measuring IFNγ and CXCL9 in serum.

For HSCT cohort, the following sampling time points are required: on day -7, pre HSCT on day 0, 1, 3, 5, 9, 13, 17, 21, 28, 31, 38 and one additional sample at the time when primary or secondary GF is suspected if not on the planned schedule. In addition, the following time points are recommended: day 7, 11, 15, 19, 24, 35, 42. It is also suggested to collect a sample when Graft vs Host Disease (GVHD) is diagnosed during any visit that the patients will attend as part of his/her standard treatment during the first 100 days post-transplant. The patient will be followed up until around day 100 post-transplant. This follow up will consist of capturing HSCT outcome information from patient hospital records around day 100.

For IHSCP cohort pre-transplant, it is recommended that, one sample per patient at the time of diagnosis (if possible not more than 1 week from the date of diagnosis) is collected. Age/sex matched control samples should be collected from healthy volunteers or patients with malignant disease outside of this protocol after appropriate consent.

Different sets of data will be collected for the HSCT and IHSCP cohorts respectively as described below:

Data collected for both cohorts

  • Age and sex
  • Inflammatory markers
  • IFNɣ
  • CXCL9
  • Other potential relevant exploratory biomarkers
  • Diagnosis
  • Date of disease diagnosis
  • Relevant medical history
  • Date and time of sample collection

Data collected for HSCT cohort only

  • Laboratory parameters assessed at the site laboratory on the date of sample collection and between collection dates when available:
  • Absolute neutrophile count (ANC) and Platelets will be measured as per the schedule of assessment, if possible when routine monitoring of patient health is conducted
  • Ferritin and Chimerism data will be collected when available (if measured as per site routine practice)
  • Concomitant medications at the time of sample collection and between collection dates
  • Presence of infection at the time of sample collection with the date of onset
  • Presence of donor specific antibodies (DSA)
  • Transplant information
  • Date of start of conditioning
  • Type of conditioning (Reduced Intensity Conditioning (RIC) / Myeloablative Conditioning (MAC) / Non-myeloablative Conditioning (NMAC) and medications
  • Transplant details (donor type, degree of match, transplant manipulation, stem cell source)
  • Date of transplant
  • Date of primary / secondary GF or of confirmed engraftment
  • GVHD with the date of onset
  • Post-transplant treatment and date (Donor Lymphocyte Infusion (DLI), Stem Cell (SC) boost, growth factor, GVHD prophylaxis, second HSCT procedure)

Data collected for IHSCP cohort only

  • Disease severity
  • In addition, the following data will be recorded for pediatric patients up to 18 years old, if available:
  • PNH clones
  • History of hepatitis
  • Karyotype

Study duration:

The study will be conducted, until the required number of patients is recruited.

  • HSCT cohort: At patient level, the study will last about 100 days from pre-transplant blood collection to last follow up data collection around day 100 post HSCT, matching the standard HSCT patient care
  • IHSCP cohort: At patient level the study will last 1 day.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
101

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Belgium
      • Brussels, Belgium, 1200
        • Cliniques Universitaires Saint-Luc
    • West-Vlaanderen
      • Roeselare, West-Vlaanderen, Belgium, 8800
        • Algemeen Ziekenhuis Delta - Campus Rumbeke
  • France
    • Basse-Normandie
      • Caen cedex 9, Basse-Normandie, France, 14033
        • Hôpital Côte de Nacre
    • Bretagne
      • Rennes cedex 9, Bretagne, France, 35033
        • Hopital Pontchaillou
    • Franche-Comte
      • Besançon Cedex, Franche-Comte, France, 25030
        • Centre Hospitalier Régional et Universitaire de Besançon - Hôpital Jean-Minjoz
    • Ile De France
      • Paris, Ile De France, France, 75010
        • Hôpital Saint-Louis
    • Ile-de-France
      • Paris, Ile-de-France, France, 75019
        • Hopital Universitaire Robert-Debre
    • Lorraine
      • Vandœuvre-lès-Nancy, Lorraine, France, 54511
        • Hopitaux de Brabois
    • Maine Et Loire
      • Angers Cedex 9, Maine Et Loire, France, 49933
        • Centre Hosptitalier Universitaire d'Angers
    • Nouvelle Aquitaine
      • Pessac, Nouvelle Aquitaine, France, 33604
        • Hôpital Haut-Lévêque
    • Provence Alpes Cote d'Azur
      • Montpellier, Provence Alpes Cote d'Azur, France, 34090
        • Hopital Arnaud de Villeneuve
      • Montpellier Cedex 5, Provence Alpes Cote d'Azur, France, 34295
        • Hopital Saint-Eloi
    • Rhone-Alpes
      • La Tronche, Rhone-Alpes, France, 38700
        • Centre Hospitalier Universitaire Grenoble Alpes
    • Rhône
      • Clermont-Ferrand, Rhône, France, 63003
        • Centre Hospitalier Universitaire Estaing
  • Germany
    • Baden-Württemberg
      • Tübingen, Baden-Württemberg, Germany
        • Universitatsklinikum Tubingen
  • Italy
      • Avellino, Italy, 83100
        • Azienda Ospedaliera San Giuseppe Moscati
      • Genova, Italy, 16147
        • Instituto Giannina Gaslini
      • Milano, Italy, 20132
        • Ospedale San Raffaele
      • Pavia, Italy, 27100
        • Fondazione IRCCS Policlinico San Matteo
      • Torino, Italy, 10126
        • Ospedale Regina Margherita
    • Lombardia
      • Roma, Lombardia, Italy, 00165
        • Ospedale Pediatrico Bambino Gesù - Roma - Gianicolo
  • Netherlands
      • Utrecht, Netherlands, 3584 CS
        • Prinses Maxima Centrum Kinderoncologie
  • United Kingdom
      • London, United Kingdom, SW3 6JJ
        • The Royal Marsden Hospital - London
      • London, United Kingdom, W12 0HS
        • Imperial College Healthcare NHS Trust NHS Trust
    • Wales
      • Cardiff, Wales, United Kingdom, CF14 4XW
        • Cardiff and Vale University Health Board

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

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)).

Description

Inclusion Criteria:

  • 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)

Exclusion Criteria:

  • HLH patients
  • Body weight < 10kg

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
HSCT - Hematopoetic Stem Cell Proliferation
Patients who received hematopoietic stem cell transplant
Procedure: blood collection
Blood samples will be collected as per protocol defined schedule. There is no investigation drug in this study.
IHSCP - Impaired HSC proliferation
Patients with impaired hematopoietic stem cell proliferation
Procedure: blood collection
Blood samples will be collected as per protocol defined schedule. There is no investigation drug in this study.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
HSCT cohort: IFNγ signature pre-and post-transplant
Time Frame: Day (-7) to day 100
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
Time Frame: Day (-7) to day 100
IFNγ and CXCL-9 in serum samples
Day (-7) to day 100
HSCT cohort: Relationship between IFNγ and the occurrence of GVHD
Time Frame: Day (-7) to day 100
IFNγ and CXCL-9 in serum samples
Day (-7) to day 100
IHSCP cohort: IFNγ signature pre-transplant
Time Frame: Day 1
IFNγ, CXCL-9 and exploratory biomarkers in serum samples
Day 1

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Swedish Orphan Biovitrum

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
PRA Health Sciences
BioMérieux
Cytel Inc.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Regis Peffault de Latour, MD, Hôpital Saint Louis Paris

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
November 16, 2020

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
August 31, 2022

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
August 31, 2022

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
July 28, 2020

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 28, 2020

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
July 31, 2020

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
October 21, 2022

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 19, 2022

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
October 1, 2022

More Information

Terms related to this study

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • NI-0501-13

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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