Hematopoietic Stem Cell TransplantationNCT04869254
Lymphocyte Count, ATG Dose and Incidence and Severity of GVHD in Pediatric Recipients of HSCT
An observational study of Hematopoietic Stem Cell Transplantation, run by IRCCS Burlo Garofolo at 1 site in Italy.
Trial summary
Despite increasing success rate in hematopoietic stem cell transplantation (HSCT) control of graft versus host disease (GVHD) remains a significative burden in mortality and morbidity. Read more about this study
- Who is it for?
- Ages 1 day to 17 years
- Conditions the study is for
- Hematopoietic Stem Cell Transplantation
- What is being tested?
- Lower lymphocyte/ATG ratio and Higher lymphocyte/ATG ratio
- How many people
- 102 participants
- Where
- 1 siteItaly
- Study dates
- 1 Jan 2000to 31 Dec 2020
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 day to 17 years
- All sexes
- No healthy volunteers
- Child
Study population: Pediatric recipients of hematopoietic stem cells transplantation
Who may be able to join
- Age of the patients between 0 and 17
- Diagnosis of hematological or oncological disease undergoing allogeneic bone marrow transplantation
- Patients undergoing myeloablative conditioning
- Patients that received ATG as GVHD prophylaxis
- Patients whose consent has already been acquired for the processing of data for research purposes
- Minimum follow-up of 12 months
Who cannot join
- Bacterial and / or fungal infection present at the time of bone marrow transplantation
- Use of Thymoglobulin in the 3 months prior to transplantation
- Allergy and / or intolerance to the active substances or excipients contained in Thymoglobulin
Trial locations and contacts
1 site in 1 country.
Italy1 site
Institute for Maternal and Child Health - IRCCS "Burlo Garofolo"
Trieste, 34137
About this study
Official title: Correlation Between the Pre-transplantation Lymphocyte Count, Administered Dose of Thymoglobulin and Graft Versus Host Disease (GVHD) in Pediatric Recipients of Hematopoietic Stem Cells Transplantation: a Retrospective Study
Despite increasing success rate in hematopoietic stem cell transplantation (HSCT) control of graft versus host disease (GVHD) remains a significative burden in mortality and morbidity. A lot of strategies could lower the incidence and gravity of the disease and immunosuppressive treatment as GVHD prophylaxis still represent the main method.
Although immunosuppressive treatment showed a good effect on GVHD mortality a lot of studies also highlight an increase of relapse and infection related mortality that jeopardize the effect on overall survival of HSCT recipient. Using anti thymocyte globulin (ATG) as GVHD prophylaxis shares the same double-edge effect as other immunosuppressive treatment although is still unclear how manage dose and timing of the infusion to minimize promoting effect on infections and maximize protective effect on GVHD. Biological effect of ATG lead to a dose- related delay in all class of T-cell reconstitution but our data are mostly from adult studies with high doses between 30 and 60 mg/kg due to the more important burden of GVHD in HSCT adult population. As for other treatment in HSCT conditioning we would like to study a personalized approach for ATG treatment: some studies focus on tuning of ATG dose for kilos but previous evidence showed that the same dose could made too little or too much immunosuppressive effect for different patients, even though same age and same stem cell source.
- Conditions
- Hematopoietic Stem Cell Transplantation
- Being tested
- Lower lymphocyte/ATG ratio, Higher lymphocyte/ATG ratio
- Keywords
- Gvhd prophylaxis, pediatric patients, hematopoietic cell transplantation
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Lower lymphocyte/ATG ratioOther
Also called Lymphocyte/ATG ratio <0.01
Higher lymphocyte/ATG ratioOther
Also called Lymphocyte/ATG ratio >0.01
| Study group | Role | What they receive |
|---|---|---|
| Hematopoietic stem cells transplantation | patients undergone allogeneic or autologous bone marrow transplant who received immunomodulatory therapy with Thymoglobulin Receives: Other: Lower lymphocyte/ATG ratio; Other: Higher lymphocyte/ATG ratio |
What is being measured
The primary outcome is the main question the study is designed to answer.
Development of GVHD according to lymphocyte/ATG ratio
Differences in the frequency of GVHD according to the two exposure's groups (lymphocyte/ATG ratio <0.01 vs >0.01). Adjustment for potentially associated variables (e.g., dose of drug actually received by patients, age of patients, type of conditioning, underlying disease, type of donor) will be carried out
Measured: 24 months after transplant
Secondary outcomes (6)
Transplant related mortality according to lymphocyte/ATG ratio
Comparison of transplant related mortality 24 months after HSCT in the two exposure's groups (lymphocyte/ATG ratio <0.01 vs >0.01)
Measured: 24 months after transplant
Incidence of Graft Failure according to lymphocyte/ATG ratio
Frequency of engraftment failure and hematological reconstitution time (defined as the first post-transplant day of at least three consecutive days with the presence of at least 500 neutrophils / mmc for myeloid engraftment and the first appearance of lymphoid cell subpopulations) in the two exposure's groups (lymphocyte/ATG ratio <0.01 vs >0.01)
Measured: 24 months after transplant
Number of episodes of sepsis during the post-transplant period according to lymphocyte/ATG ratio
Number of episodes of sepsis after HSCT and correlated mortality and morbidity in the two exposure's groups (lymphocyte/ATG ratio <0.01 vs >0.01)
Measured: 24 months after transplant
Number of episodes of fungal infections during the post transplant period according to lymphocyte/ATG ratio
Number of episodes of fungal infections after HSCT and correlated mortality and morbidity in the two exposure's groups (lymphocyte/ATG ratio <0.01 vs >0.01)
Measured: 24 months after transplant
Number of episodes of viral reactivations during the post transplant period according to lymphocyte/ATG ratio
Number of episodes of viral reactivations after HSCT and correlated mortality and morbidity in the two exposure's groups (lymphocyte/ATG ratio <0.01 vs >0.01)
Measured: 24 months after transplant
Entity of GVHD according to lymphocyte/ATG ratio
Severity and organ involvement of acute and chronic GVHD defined by the Glucksberg classification by degrees of severity in the two exposure's groups (lymphocyte/ATG ratio <0.01 vs >0.01)
Measured: 24 months after transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 May 2021
- Study start1 Jan 2000
- Primary completion31 Dec 2018
- Study completion31 Dec 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematopoietic Stem Cell TransplantationStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 102 participants (actual)
Sponsor and investigators
- Lead sponsor
- IRCCS Burlo Garofolo
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Natalia Maximova, MD, Study chair, IRCCS Burlo GarofoloAlessandra Maestro, PharmD PhD, Study director, IRCCS Burlo Garofolo
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04869254
- Sponsor's protocol ID
- RC 08/2019
- Status last verified
- Apr 2021
- First submitted
- 28 Apr 2021
- Last updated
- 3 May 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 3 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Correlation Between the Pre-transplantation Lymphocyte Count, Administered Dose of Thymoglobulin and Graft Versus Host Disease (GVHD) in Pediatric Recipients of Hematopoietic Stem Cells Transplantation: a Retrospective Study
- ClinicalTrials.gov ID
- NCT04869254
- Acronym / sponsor ID
- RC 08/2019
- Conditions
- Hematopoietic Stem Cell Transplantation
- Keywords
- Gvhd prophylaxis, pediatric patients, hematopoietic cell transplantation
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 102 participants (actual)
- Lead sponsor
- IRCCS Burlo GarofoloUniversity, hospital or other
- Study start
- 1 Jan 2000 (actual)
- Primary completion
- 31 Dec 2018 (actual)
- Study completion
- 31 Dec 2020 (actual)
- First posted
- 3 May 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Despite increasing success rate in hematopoietic stem cell transplantation (HSCT) control of graft versus host disease (GVHD) remains a significative burden in mortality and morbidity. A lot of strategies could lower the incidence and gravity of the disease and immunosuppressive treatment as GVHD prophylaxis still represent the main method.
Although immunosuppressive treatment showed a good effect on GVHD mortality a lot of studies also highlight an increase of relapse and infection related mortality that jeopardize the effect on overall survival of HSCT recipient. Using anti thymocyte globulin (ATG) as GVHD prophylaxis shares the same double-edge effect as other immunosuppressive treatment although is still unclear how manage dose and timing of the infusion to minimize promoting effect on infections and maximize protective effect on GVHD. Biological effect of ATG lead to a dose- related delay in all class of T-cell reconstitution but our data are mostly from adult studies with high doses between 30 and 60 mg/kg due to the more important burden of GVHD in HSCT adult population. As for other treatment in HSCT conditioning we would like to study a personalized approach for ATG treatment: some studies focus on tuning of ATG dose for kilos but previous evidence showed that the same dose could made too little or too much immunosuppressive effect for different patients, even though same age and same stem cell source.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Hematopoietic stem cells transplantation | – | Other: Lower lymphocyte/ATG ratio, Other: Higher lymphocyte/ATG ratiopatients undergone allogeneic or autologous bone marrow transplant who received immunomodulatory therapy with Thymoglobulin |
Who may qualify
- Ages
- 1 day to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Pediatric recipients of hematopoietic stem cells transplantation
Key inclusion criteria (6 listed)
- Age of the patients between 0 and 17
- Diagnosis of hematological or oncological disease undergoing allogeneic bone marrow transplantation
- Patients undergoing myeloablative conditioning
- Patients that received ATG as GVHD prophylaxis
- Patients whose consent has already been acquired for the processing of data for research purposes
Key exclusion criteria (3 listed)
- Bacterial and / or fungal infection present at the time of bone marrow transplantation
- Use of Thymoglobulin in the 3 months prior to transplantation
- Allergy and / or intolerance to the active substances or excipients contained in Thymoglobulin
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Lower lymphocyte/ATG ratioAlso: Lymphocyte/ATG ratio <0.01 | Other | Hematopoietic stem cells transplantation |
| Higher lymphocyte/ATG ratioAlso: Lymphocyte/ATG ratio >0.01 | Other | Hematopoietic stem cells transplantation |
Primary outcome
Development of GVHD according to lymphocyte/ATG ratio
Time frame: 24 months after transplant
Differences in the frequency of GVHD according to the two exposure's groups (lymphocyte/ATG ratio <0.01 vs >0.01). Adjustment for potentially associated variables (e.g., dose of drug actually received by patients, age of patients, type of conditioning, underlying disease, type of donor) will be carried out
6 secondary outcomes
Transplant related mortality according to lymphocyte/ATG ratio
Time frame: 24 months after transplant
Incidence of Graft Failure according to lymphocyte/ATG ratio
Time frame: 24 months after transplant
Number of episodes of sepsis during the post-transplant period according to lymphocyte/ATG ratio
Time frame: 24 months after transplant
Number of episodes of fungal infections during the post transplant period according to lymphocyte/ATG ratio
Time frame: 24 months after transplant
Number of episodes of viral reactivations during the post transplant period according to lymphocyte/ATG ratio
Time frame: 24 months after transplant
Entity of GVHD according to lymphocyte/ATG ratio
Time frame: 24 months after transplant
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 May 2021.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Natalia Maximova, MD study chair
IRCCS Burlo Garofolo
Alessandra Maestro, PharmD PhD study director
IRCCS Burlo Garofolo
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hematopoietic Stem Cell Transplantation, run by IRCCS Burlo Garofolo at 1 site in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 May 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Development of GVHD according to lymphocyte/ATG ratio (time frame: 24 months after transplant). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 May 2021.