Hematopoietic Stem Cell TransplantationNCT04580576
Gender Differences in Pediatric Hematopoietic Stem Cell Transplantation (HSCT)
An observational study of Hematopoietic Stem Cell Transplantation, run by IRCCS Burlo Garofolo.
Trial summary
Gender medicine considers the way in which gender, male or female, affects the development and impact of diseases and the response to therapies. It can be said that it is a new transversal dimension of medicine, which evaluates the gender differences in the physiology, pathophysiology and clinic of many diseases and… Read more about this study
- Who is it for?
- Ages 4 months to 17 years
- Conditions the study is for
- Hematopoietic Stem Cell Transplantation
- What is being tested?
- Hematopoietic stem cell transplantation
- Type of treatment
- Surgery or procedure
- How many people
- 200 participants
- Where
- No sites listed yet
- Study dates
- 1 Jan 2000to 1 Oct 2019
- 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 4 months to 17 years
- All sexes
- No healthy volunteers
- Child
Study population: Population of pediatric patients undergoing hematopoietic stem cell transplantation
Who may be able to join
- Patients aged between 4 months and 17 years
- Diagnosis of oncohaematological disease subjected to hematopoietic stem cell transplantation
- Allogeneic or autologous bone marrow transplantation from January 2000 to October 2018
- Consent acquired for the processing of data for research purposes
Who cannot join
None listed.
About this study
Official title: Pilot Study on Gender Differences in Hematopoietic Cell Transplantation Outcomes in the Pediatric Population
Gender medicine considers the way in which gender, male or female, affects the development and impact of diseases and the response to therapies. It can be said that it is a new transversal dimension of medicine, which evaluates the gender differences in the physiology, pathophysiology and clinic of many diseases and thus sets itself the goal of reaching optimal therapeutic decisions both in men and women based on proven scientific evidence.
Although knowledge of gender medicine has increased significantly in recent years, a gender approach has not been much developed in pediatrics. In the field of bone marrow transplants, hematopoietic stem cell transplantation is known to be the most effective consolidation therapy in some high-risk hematology malignancies such as acute lymphoblastic leukemia and acute myeloid leukemia, and represents one of the potential treatment for patients suffering from solid tumors and genetic hematological, metabolic diseases and primary immunodeficiencies. Huge progress has been made in high resolution donor typing, choice of conditioning regimens, manipulation of hematopoietic stem cells (HSC) and prevention of serious infections in recent years, which have significantly improved the survival rate of patients undergoing to this procedure.
Read more
International literature regarding the response and outcomes from hematopoietic cell transplantation in a gender perspective is completely absent, for these reasons this pilot study was born from the need to understand from a broader perspective and in order to better understand how the gender may or not influence the outcome of transplantation in pediatric patients.
This retrospective analysis of the data will concern all patients who underwent allogeneic or autologous bone marrow transplant. The data will be collected from clinical records and from Regional electronic databases. All data will be collected anonymously and an identification code will be assigned to each case.
- Conditions
- Hematopoietic Stem Cell Transplantation
- Being tested
- Hematopoietic stem cell transplantation
- Keywords
- gender, pediatrics, hematopoietic cell transplantation
- Medical subject headings
- Coitus
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Hematopoietic stem cell transplantationProcedure or surgery
Allogeneic or autologous bone marrow transplant
| Study group | Role | What they receive |
|---|---|---|
| Female group | Pediatric female patients undergoing hematopoietic stem cell transplantation Receives: Procedure: Hematopoietic stem cell transplantation | |
| Male group | Pediatric male patients undergoing hematopoietic stem cell transplantation Receives: Procedure: Hematopoietic stem cell transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Gender-related difference in overall 12-month toxicity
Differences in toxicity (hepatic, renal, pulmonary, gastrointestinal) in males and females recipients
Measured: 12 months after transplant
Secondary outcomes (9)
Gender difference in overall survival (OS)
Overall survival comparison from males and females recipients
Measured: 12 months after transplant
Gender difference in post-transplant primary disease recurrence
Incidence of post-transplant leukemic relapse in males and females recipients
Measured: 12 months after transplant
Gender difference in the frequency of transplant-related toxicity at 12 months
Frequency of post-transplant liver, kidney, pulmonary, gastrointestinal, endocrine, cardiac toxicity
Measured: 12 months after transplant
Gender difference in infectious complications
Number of episodes of sepsis / fungal infections / viral reactivations after HSCT
Measured: 12 months after transplant
Gender difference in the frequency of adverse events due to pre-transplant conditioning regimen
Number of chemo- radiotherapy-related adverse events. Toxicity was graded according to National Cancer Institute (NCI) common toxicity criteria
Measured: 12 months after transplant
Gender difference in severity of adverse events due to pre-transplant conditioning regimen
Severity of chemo- radiotherapy-related adverse events. Toxicity wil be graded according to National Cancer Institute (NCI) common toxicity criteria
Measured: 12 months after transplant
Gender difference in timing of hematological engraftment
Engraftment defined as the engraftment of polymorphonuclear neutrophils (PMN) on the first day of 3 consecutive days with PMN number greater than 500 / ml3 and engraftment of platelets defined as number of platelets> 20,000 / ml3 in the absence of platelet transfusion in the previous 5 days.
Measured: 12 months after transplant
Gender difference in frequency of primary graft failure
Engraftment defined as the engraftment of polymorphonuclear neutrophils (PMN) on the first day of 3 consecutive days with PMN number greater than 500 / ml3 and engraftment of platelets defined as number of platelets> 20,000 / ml3 in the absence of platelet transfusion in the previous 5 days.
Primary graft failure is defined as no evidence of engraftment or hematological recovery of donor cells, within the first month after transplant, without evidence of disease relapse.
Measured: 12 months after transplant
Gender difference in frequency of secondary graft failure
Engraftment defined as the engraftment of polymorphonuclear neutrophils (PMN) on the first day of 3 consecutive days with PMN number greater than 500 / ml3 and engraftment of platelets defined as number of platelets> 20,000 / ml3 in the absence of platelet transfusion in the previous 5 days.
Secondary graft failure refers to the loss of a previously functioning graft, resulting in cytopenia involving at least two blood cell lineages.
Measured: 12 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 posted8 Oct 2020
- Study start1 Jan 2000
- Primary completion1 Oct 2018
- Study completion1 Oct 2019
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
- 200 participants (actual)
Sponsor and investigators
- Lead sponsor
- IRCCS Burlo Garofolo
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Alessandra Maestro, PharmD, PhD, Study director, Institute for Maternal and Child Health IRCCS Burlo GarofoloNatalia Maximova, MD, Principal investigator, Institute for Maternal and Child Health IRCCS Burlo Garofolo
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04580576
- Sponsor's protocol ID
- RC 25/2019
- Status last verified
- Oct 2020
- First submitted
- 2 Oct 2020
- Last updated
- 8 Oct 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Oct 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study on Gender Differences in Hematopoietic Cell Transplantation Outcomes in the Pediatric Population
- ClinicalTrials.gov ID
- NCT04580576
- Acronym / sponsor ID
- RC 25/2019
- Conditions
- Hematopoietic Stem Cell Transplantation
- Keywords
- gender, pediatrics, hematopoietic cell transplantation
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 200 participants (actual)
- Lead sponsor
- IRCCS Burlo GarofoloUniversity, hospital or other
- Study start
- 1 Jan 2000 (actual)
- Primary completion
- 1 Oct 2018 (actual)
- Study completion
- 1 Oct 2019 (actual)
- First posted
- 8 Oct 2020 (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.
Gender medicine considers the way in which gender, male or female, affects the development and impact of diseases and the response to therapies. It can be said that it is a new transversal dimension of medicine, which evaluates the gender differences in the physiology, pathophysiology and clinic of many diseases and thus sets itself the goal of reaching optimal therapeutic decisions both in men and women based on proven scientific evidence.
Although knowledge of gender medicine has increased significantly in recent years, a gender approach has not been much developed in pediatrics. In the field of bone marrow transplants, hematopoietic stem cell transplantation is known to be the most effective consolidation therapy in some high-risk hematology malignancies such as acute lymphoblastic leukemia and acute myeloid leukemia, and represents one of the potential treatment for patients suffering from solid tumors and genetic hematological, metabolic diseases and primary immunodeficiencies. Huge progress has been made in high resolution donor typing, choice of conditioning regimens, manipulation of hematopoietic stem cells (HSC) and prevention of serious infections in recent years, which have significantly improved the survival rate of patients undergoing to this procedure.
International literature regarding the response and outcomes from hematopoietic cell transplantation in a gender perspective is completely absent, for these reasons this pilot study was born from the need to understand from a broader perspective and in order to better understand how the gender may or not influence the outcome of transplantation in pediatric patients.
This retrospective analysis of the data will concern all patients who underwent allogeneic or autologous bone marrow transplant. The data will be collected from clinical records and from Regional electronic databases. All data will be collected anonymously and an identification code will be assigned to each case.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Female group | – | Procedure: Hematopoietic stem cell transplantationPediatric female patients undergoing hematopoietic stem cell transplantation |
| Male group | – | Procedure: Hematopoietic stem cell transplantationPediatric male patients undergoing hematopoietic stem cell transplantation |
Who may qualify
- Ages
- 4 months to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Population of pediatric patients undergoing hematopoietic stem cell transplantation
Key inclusion criteria (4 listed)
- Patients aged between 4 months and 17 years
- Diagnosis of oncohaematological disease subjected to hematopoietic stem cell transplantation
- Allogeneic or autologous bone marrow transplantation from January 2000 to October 2018
- Consent acquired for the processing of data for research purposes
Key exclusion criteria (0 listed)
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 |
|---|---|---|
| Hematopoietic stem cell transplantationAllogeneic or autologous bone marrow transplant | Procedure or surgery | Female group, Male group |
Primary outcome
Gender-related difference in overall 12-month toxicity
Time frame: 12 months after transplant
Differences in toxicity (hepatic, renal, pulmonary, gastrointestinal) in males and females recipients
9 secondary outcomes
Gender difference in overall survival (OS)
Time frame: 12 months after transplant
Gender difference in post-transplant primary disease recurrence
Time frame: 12 months after transplant
Gender difference in the frequency of transplant-related toxicity at 12 months
Time frame: 12 months after transplant
Gender difference in infectious complications
Time frame: 12 months after transplant
Gender difference in the frequency of adverse events due to pre-transplant conditioning regimen
Time frame: 12 months after transplant
Gender difference in severity of adverse events due to pre-transplant conditioning regimen
Time frame: 12 months after transplant
Gender difference in timing of hematological engraftment
Time frame: 12 months after transplant
Gender difference in frequency of primary graft failure
Time frame: 12 months after transplant
Gender difference in frequency of secondary graft failure
Time frame: 12 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 8 Oct 2020.
- Individual participant dataSharing planned: No
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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.
Alessandra Maestro, PharmD, PhD study director
Institute for Maternal and Child Health IRCCS Burlo Garofolo
Natalia Maximova, MD principal investigator
Institute for Maternal and Child Health IRCCS Burlo Garofolo
Questions about this trial
What is this clinical trial studying?
An observational study of Hematopoietic Stem Cell Transplantation, run by IRCCS Burlo Garofolo.
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 4 inclusion and 0 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 8 Oct 2020. Status can change between updates, so confirm with the study contact before making plans.
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?
Gender-related difference in overall 12-month toxicity (time frame: 12 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 8 Oct 2020.