Haemoglobin Barts HydropsNCT05797272
In-utero Hematopoietic Stem Cell Transplantation for the Treatment of Fetuses With Bart's Hydrops Fetalis Syndrome
A study testing in-utero HSCT in people with Haemoglobin Barts Hydrops, run by Chinese University of Hong Kong at 1 site in Hong Kong.
Trial summary
This is a prospective observational pilot study on pregnant women who are diagnosed to have Bart's hydrops fetalis syndrome (BHFS) affected fetuses and opt for continuation of pregnancy will be invited to consider undergoing in-utero hematopoietic stem cell transplantation under a research protocol, aiming to… Read more about this study
- Who is it for?
- Ages 18 years and older, women only
- Conditions the study is for
- Haemoglobin Barts Hydrops
- What is being tested?
- in-utero HSCT
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteHong Kong
- Study dates
- 1 Oct 2021to 30 Sep 2027
- Phase
- Interventional
- 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 18 years and older
- Women only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Pregnancy before 26 weeks and 0 day of gestational age with a diagnosis of BHFS confirmed by chorionic villus sampling, amniocentesis or cordocentesis;
- The parents elected to pursue IUT and are willing to undergo subsequent IUT for the remainder of gestation.
Who cannot join
- Fetal subjects:
- Fetuses having a second major anatomic anomaly (not related to the underlying thalassemia) that contributes a significant morbidity or mortality risk;
- Fetuses having a genetic or chromosomal abnormalities other than BHFS that contributes a significant morbidity or mortality risk;
- Echocardiogram or ultrasound findings that indicate a high risk of fetal demise after fetal intervention;
- Fetuses diagnosed with in-utero death prior to the actual intervention.
- Maternal subjects:
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- Maternal age < 18 years, mentally handicapped or severely ill;
- Maternal participants having one or more morbidities that would preclude bone marrow or peripheral blood stem cells harvest and fetal intervention including, but not limited to, bleeding disorder, maternal cardiac disease, maternal mirror syndrome, symptomatic maternal anemia, or if they develop preterm premature rupture of membranes or active preterm labor;
- Unable to understand English or Chinese to give consent.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Hong Kong1 site, 1 recruiting
- Recruiting
The Chinese University of Hong Kong
Shatin
Tak Yeung LEUNG, MD, 852-35052806, tyleung@cuhk.edu.hk
About this study
Official title: Feasibility of In-utero Hematopoietic Stem Cell Transplantation as a Conjunctive Treatment During In-utero Blood Transfusion for Hemoglobin Bart's Hydrops Fetalis Syndrome in Hong Kong
This is a prospective observational pilot study on pregnant women who are diagnosed to have Bart's hydrops fetalis syndrome (BHFS) affected fetuses and opt for continuation of pregnancy will be invited to consider undergoing in-utero hematopoietic stem cell transplantation under a research protocol, aiming to determine whether in-utero hematopoietic stem cell transplantation (HSCT) for fetuses with confirmed BHFS at the time of in-utero transfusion (IUT) of red blood cells could be feasible in Hong Kong. The participants will undergo bone marrow or peripheral blood stem cells harvest and an IUT combined with maternal stem cells.
Read the detailed description
Alpha thalassemia major or hemoglobin BHFS is thought to be incompatible of life. A prevalence study conducted in Hong Kong showed 4.6% of the screened population are heterozygous carriers of deletional type alpha thalassemia, who would have 25% risk of conceiving fetuses with BHFS if both couple are carrier. Fetuses with BHFS often develop severe anaemia in utero, feature with cardiomegaly, high peak systolic velocity in middle cerebral artery (MCA PSV), thickened placenta, and eventually hydrops, which can lead to intrauterine fetal demise or early neonatal death; while the mother may also be at risk of maternal mirror syndrome which will cause significant morbidity and even mortality. As a result, when the foetuses are diagnosed with BHFS, most couple will terminate the pregnancy.
With the advance of technology, if the couples are both known to be alpha thalassemia carrier (alpha-alpha couples), they may consider pre-implantation genetic diagnosis. However, even though preimplantation genetic testing for monogenic disease (PGT-M) is a mature technique over the decades, it is still technically challenging, requires assisted reproductive technology even if the couple are fertile and relative costly. As a result, most of the alpha-alpha couples still conceive naturally. Prenatal ultrasonic surveillance is usually offered for the couples and prenatal diagnosis is required for genetic analysis to confirm BHFS when there is ultrasound feature of fetal anaemia.
Thus, the investigators propose this pilot study to determine whether in-utero hematopoietic stem cell transplantation for fetuses with confirmed alpha thalassemia major at the time of IUT of red blood cells could be feasible in Hong Kong.
- Conditions
- Haemoglobin Barts Hydrops
- Being tested
- in-utero HSCT
- Keywords
- in-utero transplantation, hematopoietic stem cell, Bart's syndrome, hydrops fetalis
- Medical subject headings
- Hydrops Fetalis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
in-utero HSCTProcedure or surgery
Pregnant women who are diagnosed to have BHFS affected fetuses and opt for continuation of pregnancy will undergo in-utero HSCT.
| Study group | Role | What they receive |
|---|---|---|
| Fetuses with BHFS | Experimental | Fetuses of pregnant women confirmed with BHFS Receives: Procedure: in-utero HSCT |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of maternal complications
Maternal participant tolerance of bone marrow or peripheral blood stem cells harvest with prior granulocyte colony-stimulating factor (G-CSF) administration:
- Maternal participant tolerance defined as not requiring interventions for preterm labor, bleeding, infection or prolonged hospitalization. Any side effects or adverse outcome related to the procedure or related medications observed will be well documented.
Measured: during pregnancy (up to week 40)
Rate of survival of fetus
Safety of in utero HSCT when performed at the same time as in utero blood transfusion for the fetal participant:
- Safety for fetal participant defined by survival 24 hours after procedure, fetal survival till birth, neonatal survival through discharge of hospitalization and no evidence of graft versus host disease.
Measured: during pregnancy (up to week 40)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Apr 2023
- Study start1 Oct 2021
- Primary completion30 Sep 2027 (estimated)
- Study completion30 Sep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Haemoglobin Barts HydropsStudies of the same condition, open and under way.
- More trials run by Chinese University of Hong KongThe 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
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chinese University of Hong Kong
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Tak Yeung LEUNG, Chinese University of Hong Kong
- Investigators
- Tak Yeung LEUNG, MD, Principal investigator, Chinese University of Hong Kong
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05797272
- Sponsor's protocol ID
- 2021.551-T
- Status last verified
- Apr 2023
- First submitted
- 7 Mar 2023
- Last updated
- 12 Apr 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Feasibility of In-utero Hematopoietic Stem Cell Transplantation as a Conjunctive Treatment During In-utero Blood Transfusion for Hemoglobin Bart's Hydrops Fetalis Syndrome in Hong Kong
- ClinicalTrials.gov ID
- NCT05797272
- Acronym / sponsor ID
- 2021.551-T
- Conditions
- Haemoglobin Barts Hydrops
- Keywords
- in-utero transplantation, hematopoietic stem cell, Bart's syndrome, hydrops fetalis
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Chinese University of Hong KongUniversity, hospital or other
- Study start
- 1 Oct 2021 (actual)
- Primary completion
- 30 Sep 2027 (estimated)
- Study completion
- 30 Sep 2027 (estimated)
- First posted
- 4 Apr 2023 (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.
This is a prospective observational pilot study on pregnant women who are diagnosed to have Bart's hydrops fetalis syndrome (BHFS) affected fetuses and opt for continuation of pregnancy will be invited to consider undergoing in-utero hematopoietic stem cell transplantation under a research protocol, aiming to determine whether in-utero hematopoietic stem cell transplantation (HSCT) for fetuses with confirmed BHFS at the time of in-utero transfusion (IUT) of red blood cells could be feasible in Hong Kong. The participants will undergo bone marrow or peripheral blood stem cells harvest and an IUT combined with maternal stem cells.
Detailed description
Alpha thalassemia major or hemoglobin BHFS is thought to be incompatible of life. A prevalence study conducted in Hong Kong showed 4.6% of the screened population are heterozygous carriers of deletional type alpha thalassemia, who would have 25% risk of conceiving fetuses with BHFS if both couple are carrier. Fetuses with BHFS often develop severe anaemia in utero, feature with cardiomegaly, high peak systolic velocity in middle cerebral artery (MCA PSV), thickened placenta, and eventually hydrops, which can lead to intrauterine fetal demise or early neonatal death; while the mother may also be at risk of maternal mirror syndrome which will cause significant morbidity and even mortality. As a result, when the foetuses are diagnosed with BHFS, most couple will terminate the pregnancy.
With the advance of technology, if the couples are both known to be alpha thalassemia carrier (alpha-alpha couples), they may consider pre-implantation genetic diagnosis. However, even though preimplantation genetic testing for monogenic disease (PGT-M) is a mature technique over the decades, it is still technically challenging, requires assisted reproductive technology even if the couple are fertile and relative costly. As a result, most of the alpha-alpha couples still conceive naturally. Prenatal ultrasonic surveillance is usually offered for the couples and prenatal diagnosis is required for genetic analysis to confirm BHFS when there is ultrasound feature of fetal anaemia.
Thus, the investigators propose this pilot study to determine whether in-utero hematopoietic stem cell transplantation for fetuses with confirmed alpha thalassemia major at the time of IUT of red blood cells could be feasible in Hong Kong.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Fetuses with BHFS | Experimental | Procedure: in-utero HSCTFetuses of pregnant women confirmed with BHFS |
Who may qualify
- Ages
- 18 years and older
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Pregnancy before 26 weeks and 0 day of gestational age with a diagnosis of BHFS confirmed by chorionic villus sampling, amniocentesis or cordocentesis;
- The parents elected to pursue IUT and are willing to undergo subsequent IUT for the remainder of gestation.
Key exclusion criteria (9 listed)
- Fetal subjects:
- Fetuses having a second major anatomic anomaly (not related to the underlying thalassemia) that contributes a significant morbidity or mortality risk;
- Fetuses having a genetic or chromosomal abnormalities other than BHFS that contributes a significant morbidity or mortality risk;
- Echocardiogram or ultrasound findings that indicate a high risk of fetal demise after fetal intervention;
- Fetuses diagnosed with in-utero death prior to the actual intervention.
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 |
|---|---|---|
| in-utero HSCTPregnant women who are diagnosed to have BHFS affected fetuses and opt for continuation of pregnancy will undergo in-utero HSCT. | Procedure or surgery | Fetuses with BHFS |
Primary outcome
Rate of maternal complications
Time frame: during pregnancy (up to week 40)
Maternal participant tolerance of bone marrow or peripheral blood stem cells harvest with prior granulocyte colony-stimulating factor (G-CSF) administration: - Maternal participant tolerance defined as not requiring interventions for preterm labor, bleeding, infection or prolonged hospitalization. Any side effects or adverse outcome related to the procedure or related medications observed will be well documented.
Rate of survival of fetus
Time frame: during pregnancy (up to week 40)
Safety of in utero HSCT when performed at the same time as in utero blood transfusion for the fetal participant: - Safety for fetal participant defined by survival 24 hours after procedure, fetal survival till birth, neonatal survival through discharge of hospitalization and no evidence of graft versus host disease.
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 12 Apr 2023.
Related trials for Haemoglobin Barts Hydrops
RecruitingObservational study
International Registry of Patients With Alpha Thalassemia
Alpha-Thalassemia, Alpha Thalassemia Major
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.
Tak Yeung LEUNG, MD principal investigator
Chinese University of Hong Kong
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing in-utero HSCT in people with Haemoglobin Barts Hydrops, run by Chinese University of Hong Kong at 1 site in Hong Kong.
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 2 inclusion and 9 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 12 Apr 2023. 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 Hong Kong. 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 Tak Yeung LEUNG, MD (852-35052806, tyleung@cuhk.edu.hk), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Rate of maternal complications (time frame: during pregnancy (up to week 40)). 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 12 Apr 2023.