Thalassemia MajorNCT02126046
Unrelated Umbilical Cord Blood Following HLA-haploidentical Hematopoietic Stem Cell Transplantation in Patients With β-thalassemia Major
A study testing unrelated CB following haplo-identical hematopoietic stem cells transplantation in people with Thalassemia Major, run by Nanfang Hospital, Southern Medical University at 1 site in China.
Trial summary
Allo-hematopoietic stem cell transplantation(HSCT) is the only way to cure β-thalassemia major at present. To expand donor pool，we developed a haplo-identical HSCT (Hi-HSCT) platform. Read more about this study
- Who is it for?
- Ages 12 months to 18 years
- Conditions the study is for
- Thalassemia Major
- What is being tested?
- unrelated CB following haplo-identical hematopoietic stem cells transplantation
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteChina
- Study dates
- Sep 2012to Mar 2016
- Phase
- Interventional
- 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 12 months to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- β-thalassemia major
- < 18 year old
- Unrelated umbilical cord blood following Haplo-identical HSCT
Who cannot join
- ≥ 18 year old
- HLA- matched related donors
- Unrelated donor transplants
- Unrelated umbilical cord blood transplants
- Severe iron overload in heart by T2*
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Department of paediatrics，Nangfang Hospital, Southern Medical University
Guangzhou, Guangdong, 510515
Chunfu Li, professor, +86 20-61641921, chunfugzcn@126.com
About this study
Allo-hematopoietic stem cell transplantation(HSCT) is the only way to cure β-thalassemia major at present. To expand donor pool，we developed a haplo-identical HSCT (Hi-HSCT) platform. But in prior Hi-HSCT using high dose post-transplant Cyclophosphamide in patients with leukemia, cytopenia post-transplant often developed, which was considered as a symptom of GVHD. Therefore, the investigators add unrelated umbilical cord blood (UCB) to the Hi-HSCT. It has reported that, as third-party cells, UCB will reduce GVHD.The purpose of this study is to determine whether unrelated UCB following Hi-HSCT can improve outcomes of Hi-HSCT in patients with β-thalassemia major.
- Conditions
- Thalassemia Major
- Being tested
- unrelated CB following haplo-identical hematopoietic stem cells transplantation
- Keywords
- Cord Blood Transplant, Haploidentical HSCT, Thalassemia major
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
unrelated CB following haplo-identical hematopoietic stem cells transplantationGenetic
| Study group | Role | What they receive |
|---|---|---|
| Hi-HSC-CBT | Other | Not described Receives: Genetic: unrelated CB following haplo-identical hematopoietic stem cells transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
overall survival（OS）
the measure is a composite
Measured: 12 months
TM-free survival（TFS）
the measure is a composite
Measured: 12 months
Transplant Related Martality (TRM)
the measure is a composite
Measured: 12 months
Primary or Secondary Graft Rejection (GR)
the measure is a composite
Measured: 12 months
Secondary outcomes (2)
The cumulative incidences of acute graft-versus-host disease（GVHD）
the measure is a composite
Measured: 12 months
The cumulative incidences of chronic graft vesus host disease (cGVHD)
the measure is a composite
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Apr 2014 (estimated)
- Study startSep 2012
- Primary completionDec 2015 (estimated)
- Study completionMar 2016 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Thalassemia MajorStudies of the same condition, open and under way.
- More trials run by Nanfang Hospital, Southern Medical 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Nanfang Hospital, Southern Medical University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Chunfu Li, Nanfang Hospital, Southern Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02126046
- Sponsor's protocol ID
- NFTM20130101
- Status last verified
- Apr 2015
- First submitted
- 18 Apr 2014
- Last updated
- 16 Apr 2015
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Apr 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Unrelated Umbilical Cord Blood Following HLA-haploidentical Hematopoietic Stem Cell Transplantation in Patients With β-thalassemia Major
- ClinicalTrials.gov ID
- NCT02126046
- Acronym / sponsor ID
- NFTM20130101
- Conditions
- Thalassemia Major
- Keywords
- Cord Blood Transplant, Haploidentical HSCT, Thalassemia major
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Nanfang Hospital, Southern Medical UniversityUniversity, hospital or other
- Study start
- Sep 2012
- Primary completion
- Dec 2015 (estimated)
- Study completion
- Mar 2016 (estimated)
- First posted
- 29 Apr 2014 (estimated)
- 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.
Allo-hematopoietic stem cell transplantation(HSCT) is the only way to cure β-thalassemia major at present. To expand donor pool，we developed a haplo-identical HSCT (Hi-HSCT) platform. But in prior Hi-HSCT using high dose post-transplant Cyclophosphamide in patients with leukemia, cytopenia post-transplant often developed, which was considered as a symptom of GVHD. Therefore, the investigators add unrelated umbilical cord blood (UCB) to the Hi-HSCT. It has reported that, as third-party cells, UCB will reduce GVHD.The purpose of this study is to determine whether unrelated UCB following Hi-HSCT can improve outcomes of Hi-HSCT in patients with β-thalassemia major.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Hi-HSC-CBT | Other | Genetic: unrelated CB following haplo-identical hematopoietic stem cells transplantation |
Who may qualify
- Ages
- 12 months to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- β-thalassemia major
- < 18 year old
- Unrelated umbilical cord blood following Haplo-identical HSCT
Key exclusion criteria (5 listed)
- ≥ 18 year old
- HLA- matched related donors
- Unrelated donor transplants
- Unrelated umbilical cord blood transplants
- Severe iron overload in heart by T2*
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 |
|---|---|---|
| unrelated CB following haplo-identical hematopoietic stem cells transplantation | Genetic | Hi-HSC-CBT |
Primary outcome
overall survival（OS）
Time frame: 12 months
the measure is a composite
TM-free survival（TFS）
Time frame: 12 months
the measure is a composite
Transplant Related Martality (TRM)
Time frame: 12 months
the measure is a composite
Primary or Secondary Graft Rejection (GR)
Time frame: 12 months
the measure is a composite
2 secondary outcomes
The cumulative incidences of acute graft-versus-host disease（GVHD）
Time frame: 12 months
The cumulative incidences of chronic graft vesus host disease (cGVHD)
Time frame: 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Apr 2015.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing unrelated CB following haplo-identical hematopoietic stem cells transplantation in people with Thalassemia Major, run by Nanfang Hospital, Southern Medical University at 1 site in China.
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 3 inclusion and 5 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: status not confirmed, as of the sponsor's last update on 16 Apr 2015. 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 China. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Chunfu Li, professor (+86 20 61641921, chunfugzcn@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
overall survival（OS） (time frame: 12 months). The study lists 4 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 16 Apr 2015.