Hematologic Disease and DisordersNCT07857746
Outcome of HSCT in Children at Ramathibodi Hospital
An observational study of Hematologic Disease and Disorders and Malignancies, run by Mahidol University at 1 site in Thailand.
Trial summary
Firstly, to study the treatment outcomes of hematopoietic stem cell transplantation (HSCT) in patients with hematologic diseases, cancers, and other conditions treated at the Department of Pediatrics, Faculty of Medicine Ramathibodi Hospital. Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Hematologic Disease and Disorders, Malignancies, Genetic Diseases
- How many people
- 200 participants
- Where
- 1 siteThailand
- Study dates
- 8 May 2020to 7 May 2035
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Patients in the Department of Pediatrics, Faculty of Medicine Ramathibodi Hospital, Mahidol University
Who may be able to join
- Patients with hematologic, malignant, or other diseases who underwent HSCT
Who cannot join
- Incomplete or loss of medical records
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Thailand1 site, 1 recruiting
- Recruiting
Ramathibodi Hospital
Ratchathewi, Bangkok, 10400
Usanarat Anurathapan, MD, 6622011803, usanarat.anu@mahidol.ac.th
Usanarat Anurathapan, MD
About this study
Official title: Outcome of Hematopoietic Stem Cell Transplantation in Children With Hematologic, Malignant and Other Diseases at Ramathibodi Hospital
Firstly, to study the treatment outcomes of hematopoietic stem cell transplantation (HSCT) in patients with hematologic diseases, cancers, and other conditions treated at the Department of Pediatrics, Faculty of Medicine Ramathibodi Hospital. Secondly, to evaluate factors that may influence treatment outcomes in patients with hematologic diseases, cancers, and other conditions undergoing hematopoietic stem cell transplantation. Lastly, to study complications occurring after hematopoietic stem cell transplantation in patients with hematologic diseases, cancers, and other conditions, in order to identify opportunities to further improve treatment outcomes.
Read the detailed description
Hematopoietic stem cell transplantation (HSCT) is considered a standard treatment for patients with hematologic diseases, cancers, and other conditions such as Gaucher disease. Most stem cell transplantations involve using cells from a donor (allogeneic HSCT) with matching human leukocyte antigen (HLA) types, such as full siblings or volunteer donors.
Currently, the Department of Pediatrics, Faculty of Medicine Ramathibodi Hospital, is capable of performing stem cell transplantations in patients who lack fully matched donors, using donors such as the patient's father or mother. Patient preparation before transplantation is a critical factor affecting treatment outcomes. Proper preparation increases the likelihood of successful treatment, particularly as this patient population typically presents with severe conditions. Additionally, selecting an appropriate conditioning regimen (pre-transplant chemotherapy) and providing optimal care during and after transplantation are equally vital to the treatment outcomes.
Collecting data to study the various factors influencing HSCT outcomes in pediatric patients, as well as associated complications, will yield essential information for treatment planning and pre-transplant patient preparation. This will help advance the treatment of hematologic diseases, cancers, and other conditions in children through stem cell transplantation to achieve optimal therapeutic outcomes.
- Keywords
- outcomes, HSCT, Single institute
- Medical subject headings
- Hematologic Diseases, Disease, Neoplasms, Genetic Diseases, Inborn
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall survival
Measured: 5 years
Disease free survival
Measured: 5 years
Secondary outcomes (2)
GVHD free rate
Measured: 5 years
Infection rate
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Oct 2026
- Study start8 May 2020
- Primary completion7 May 2030 (estimated)
- Study completion7 May 2035 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematologic Disease and DisordersStudies of the same condition, open and under way.
- More trials run by Mahidol UniversityThe sponsor's other registered studies.
- 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
- Case only
- Time perspective
- Retrospective
- Enrolment
- 200 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Mahidol University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Usanarat Anurathapan, Mahidol University
- Investigators
- Usanarat Anurathapan, MD, Principal investigator, Department of Pediatrics, Faculty of Medicine Ramathibodi Hospital, Mahidol University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07857746
- Sponsor's protocol ID
- MURA2020/791
- Status last verified
- Sep 2026
- First submitted
- 29 Sep 2026
- Last updated
- 5 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Due to personal information \& PDPA law
Study at a glance
From the registry record, last updated by the sponsor on 5 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Outcome of Hematopoietic Stem Cell Transplantation in Children With Hematologic, Malignant and Other Diseases at Ramathibodi Hospital
- ClinicalTrials.gov ID
- NCT07857746
- Acronym / sponsor ID
- MURA2020/791
- Keywords
- outcomes, HSCT, Single institute
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- Mahidol UniversityUniversity, hospital or other
- Study start
- 8 May 2020 (actual)
- Primary completion
- 7 May 2030 (estimated)
- Study completion
- 7 May 2035 (estimated)
- First posted
- 5 Oct 2026 (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.
Firstly, to study the treatment outcomes of hematopoietic stem cell transplantation (HSCT) in patients with hematologic diseases, cancers, and other conditions treated at the Department of Pediatrics, Faculty of Medicine Ramathibodi Hospital. Secondly, to evaluate factors that may influence treatment outcomes in patients with hematologic diseases, cancers, and other conditions undergoing hematopoietic stem cell transplantation. Lastly, to study complications occurring after hematopoietic stem cell transplantation in patients with hematologic diseases, cancers, and other conditions, in order to identify opportunities to further improve treatment outcomes.
Detailed description
Hematopoietic stem cell transplantation (HSCT) is considered a standard treatment for patients with hematologic diseases, cancers, and other conditions such as Gaucher disease. Most stem cell transplantations involve using cells from a donor (allogeneic HSCT) with matching human leukocyte antigen (HLA) types, such as full siblings or volunteer donors.
Currently, the Department of Pediatrics, Faculty of Medicine Ramathibodi Hospital, is capable of performing stem cell transplantations in patients who lack fully matched donors, using donors such as the patient's father or mother. Patient preparation before transplantation is a critical factor affecting treatment outcomes. Proper preparation increases the likelihood of successful treatment, particularly as this patient population typically presents with severe conditions. Additionally, selecting an appropriate conditioning regimen (pre-transplant chemotherapy) and providing optimal care during and after transplantation are equally vital to the treatment outcomes.
Collecting data to study the various factors influencing HSCT outcomes in pediatric patients, as well as associated complications, will yield essential information for treatment planning and pre-transplant patient preparation. This will help advance the treatment of hematologic diseases, cancers, and other conditions in children through stem cell transplantation to achieve optimal therapeutic outcomes.
Study design
- Observational model
- Case only
- Time perspective
- Retrospective
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Patients in the Department of Pediatrics, Faculty of Medicine Ramathibodi Hospital, Mahidol University
Key inclusion criteria (1 listed)
- Patients with hematologic, malignant, or other diseases who underwent HSCT
Key exclusion criteria (1 listed)
- Incomplete or loss of medical records
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Overall survival
Time frame: 5 years
Disease free survival
Time frame: 5 years
2 secondary outcomes
GVHD free rate
Time frame: 5 years
Infection rate
Time frame: 5 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Oct 2026.
- Individual participant dataSharing planned: No. Due to personal information \& PDPA law
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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.
Usanarat Anurathapan, MD principal investigator
Department of Pediatrics, Faculty of Medicine Ramathibodi Hospital, Mahidol University
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 Hematologic Disease and Disorders and Malignancies, run by Mahidol University at 1 site in Thailand.
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 1 inclusion and 1 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: looking for participants now, as of the sponsor's last update on 5 Oct 2026. 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 Thailand. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Usanarat Anurathapan, MD (6622011803, usanarat.anu@mahidol.ac.th), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall survival (time frame: 5 years). The study lists 2 primary outcomes in all. 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 5 Oct 2026.