Hematologic DiseasesPhase 1HIGHTNCT06809699
Human Leukocyte Antigen (HLA) Mismatched Related Allogeneic Hematopoietic Stem Cell Transplantation
A Phase 1 study testing Busulfan (Busulfex), Cyclophosphamide (CTX) and Fludarabine (Fludara) in people with Hematologic Diseases, run by He Huang at 1 site in China.
Trial summary
This study is a single center, prospective, single arm exploratory clinical trial that includes patients with hematological malignancies who are indicated for allogeneic hematopoietic stem cell transplantation (allo HSCT) but lack suitable donors. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Hematologic Diseases
- What is being tested?
- Busulfan (Busulfex), Cyclophosphamide (CTX) and Fludarabine (Fludara)
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 29 participants
- Where
- 1 siteChina
- Study dates
- 4 Aug 2022to 31 Oct 2027
- Phase
- Phase 1
- 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 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Adult patients (18-60 years old) with hematological malignancies and indications for hematopoietic stem cell transplantation with no available related human leukocyte antigen (HLA) identical sibling donors;
- No available donor with HLA high-resolution typing ≥ 9/10 or those who have difficulty finding donors due to urgent medical conditions;
- No suitable HLA matching haploidentical donor available;
- There is a suitable donor with mismatched HLA typing;
- The subjects or their legal representatives shall sign an informed consent form before the start of the clinical study.
Who cannot join
- Patients with severe liver and kidney function (alanine aminotransferase>2.5 times the upper limit of normal, blood creatinine>1.5 times the upper limit of normal) and cardiopulmonary dysfunction (New York Heart Association (NYHA) III/IV heart function, ejection fraction<50%, severe obstructive or restrictive ventilation dysfunction);
- Merge active infections;
- Eastern Cooperative Oncology Group (ECOG) score ≥ 2 points;
- Secondary tumors with merged activity;
- Severe central nervous system or mental illness leading to the inability to autonomously choose to enter or exit clinical trials;
- Combine other allo HSCT contraindications.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Yishan Ye, MD., PhD contact
China1 site, 1 recruiting
- Recruiting
The First Affiliated Hospital, Zhejiang University School of Medicine
Hangzhou, China, 310003
Yanmin Zhao, MD., PhD, +86 57187236706, yanminzhao@zju.edu.cn
He Huang
About this study
Official title: A Prospective Single-arm Trial on Human Leukocyte Antigen (HLA) Mismatched Related Allogeneic Hematopoietic Stem Cell Transplantation
This study is a single center, prospective, single arm exploratory clinical trial that includes patients with hematological malignancies who are indicated for allogeneic hematopoietic stem cell transplantation (allo HSCT) but lack suitable donors. This project plans to use human leukocyte antigen (HLA) mismatched donors. Ultimately, a HLA mismatched allo HSCT transplantation plan will be established to improve the disease prognosis of these patients and truly enter the era of "everyone has a donor".
Read the detailed description
This study is a single center, prospective, single arm exploratory clinical trial that includes patients with hematological malignancies who are indicated for allogeneic hematopoietic stem cell transplantation (allo HSCT) but lack suitable donors. This project plans to use human leukocyte antigen (HLA) mismatched donors. Ultimately, a HLA mismatched allo HSCT transplantation plan will be established to improve the disease prognosis of these patients and truly enter the era of "everyone has a donor".
- Conditions
- Hematologic Diseases
- Being tested
- Busulfan (Busulfex), Cyclophosphamide (CTX), Fludarabine (Fludara), Semustine (MeCCNU)
- Keywords
- Hematopoietic cell transplantation, HLA mismatch related, Hematologic diseases
- Medical subject headings
- Hematologic Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Busulfan (Busulfex)Drug
For myeloablative conditioning: Busulfan (Bu，3.2 mg/kg/d IV -8d ~-6d) For reduced intensity conditioning: (Bu，3.2 mg/kg/d IV -7d~-5d)
Cyclophosphamide (CTX)Drug
Cyclophosphamide is only used in myeloablative conditioning (Cy，1.8g/m2, -5d, -4d)
Fludarabine (Fludara)Drug
For myeloablative conditioning: Fludarabine (Flu，30 mg/m2/d IV -6d ~ -2d) For reduced intensity conditioning: Fludarabine（Flu，30mg/m2 /d IV -10d~-5d）
Semustine (MeCCNU)Drug
For both myeloablative and reduced intensity conditioning: Semustine (MECCNU，250 mg/m2 orally-3d).
| Study group | Role | What they receive |
|---|---|---|
| HLA-mismatch related | Experimental | The myeloablative conditioning is used when the patient is below 50 and with an HCT-CI<2. The reduced intensity conditioning is used when the patient is over 50 or with an HCT-CI≥2. Receives: Drug: Busulfan (Busulfex); Drug: Cyclophosphamide (CTX); Drug: Fludarabine (Fludara); Drug: Semustine (MeCCNU) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall survival
Overall survival after allogeneic cell transplantation refers to the length of time a patient remains alive from the date of transplantation, regardless of disease status or complications.
Measured: 1-year
Secondary outcomes (6)
Neutrophil engraftment rate
The neutrophil engraftment rate is assessed, and neutrophil engraftment is defined as achievement of an absolute neutrophil count (ANC) of ≥ 0.5 × 10⁹/L for three consecutive days
Measured: 28 days
Platelet engraftment rate
Platelet engraftment rate is assessed, and platelet engraftment is defined as achievement of a platelet count of ≥ 20 × 10⁹/L (or ≥ 50 × 10⁹/L in some definitions) without transfusion support for at least seven consecutive days.
Measured: 28 days
Relapse
Cumulative incidence of relapse
Measured: 1-year
Cumulative incidence of relapse
Progression-free survival after allogeneic cell transplantation refers to the length of time during which a patient remains alive without any signs of disease relapse or progression.
Measured: 1-year
Progression free survival
Progression-free survival after allogeneic cell transplantation refers to the length of time during which a patient remains alive without any signs of disease relapse or progression.
Measured: 1-year
GRFS
GRFS (Graft-versus-host disease-free, relapse-free survival) after allogeneic cell transplantation is the length of time a patient remains alive without experiencing significant graft-versus-host disease, disease relapse, or death.
Measured: 1-year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Feb 2025
- Study start4 Aug 2022
- Primary completion31 Oct 2026 (estimated)
- Study completion31 Oct 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematologic DiseasesStudies 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
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 29 participants (estimated)
Sponsor and investigators
- Lead sponsor
- He Huang
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, He Huang, Zhejiang University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06809699
- Sponsor's protocol ID
- MMR-001
- Acronym
- HIGHT
- Status last verified
- Oct 2024
- First submitted
- 4 Oct 2024
- Last updated
- 5 Feb 2025
- 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 5 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Single-arm Trial on Human Leukocyte Antigen (HLA) Mismatched Related Allogeneic Hematopoietic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT06809699
- Acronym / sponsor ID
- HIGHT / MMR-001
- Conditions
- Hematologic Diseases
- Keywords
- Hematopoietic cell transplantation, HLA mismatch related, Hematologic diseases
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 29 participants (estimated)
- Lead sponsor
- He HuangUniversity, hospital or other
- Study start
- 4 Aug 2022 (actual)
- Primary completion
- 31 Oct 2026 (estimated)
- Study completion
- 31 Oct 2027 (estimated)
- First posted
- 5 Feb 2025 (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 study is a single center, prospective, single arm exploratory clinical trial that includes patients with hematological malignancies who are indicated for allogeneic hematopoietic stem cell transplantation (allo HSCT) but lack suitable donors. This project plans to use human leukocyte antigen (HLA) mismatched donors. Ultimately, a HLA mismatched allo HSCT transplantation plan will be established to improve the disease prognosis of these patients and truly enter the era of "everyone has a donor".
Detailed description
This study is a single center, prospective, single arm exploratory clinical trial that includes patients with hematological malignancies who are indicated for allogeneic hematopoietic stem cell transplantation (allo HSCT) but lack suitable donors. This project plans to use human leukocyte antigen (HLA) mismatched donors. Ultimately, a HLA mismatched allo HSCT transplantation plan will be established to improve the disease prognosis of these patients and truly enter the era of "everyone has a donor".
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| HLA-mismatch related | Experimental | Drug: Busulfan (Busulfex), Drug: Cyclophosphamide (CTX), Drug: Fludarabine (Fludara), Drug: Semustine (MeCCNU)The myeloablative conditioning is used when the patient is below 50 and with an HCT-CI<2. The reduced intensity conditioning is used when the patient is over 50 or with an HCT-CI≥2. |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- Adult patients (18-60 years old) with hematological malignancies and indications for hematopoietic stem cell transplantation with no available related human leukocyte antigen (HLA) identical sibling donors;
- No available donor with HLA high-resolution typing ≥ 9/10 or those who have difficulty finding donors due to urgent medical conditions;
- No suitable HLA matching haploidentical donor available;
- There is a suitable donor with mismatched HLA typing;
- The subjects or their legal representatives shall sign an informed consent form before the start of the clinical study.
Key exclusion criteria (6 listed)
- Patients with severe liver and kidney function (alanine aminotransferase>2.5 times the upper limit of normal, blood creatinine>1.5 times the upper limit of normal) and cardiopulmonary dysfunction (New York Heart Association (NYHA) III/IV heart function, ejection fraction<50%, severe obstructive or restrictive ventilation dysfunction);
- Merge active infections;
- Eastern Cooperative Oncology Group (ECOG) score ≥ 2 points;
- Secondary tumors with merged activity;
- Severe central nervous system or mental illness leading to the inability to autonomously choose to enter or exit clinical trials;
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 |
|---|---|---|
| Busulfan (Busulfex)For myeloablative conditioning: Busulfan (Bu，3.2 mg/kg/d IV -8d ~-6d) For reduced intensity conditioning: (Bu，3.2 mg/kg/d IV -7d~-5d) | Drug | HLA-mismatch related |
| Cyclophosphamide (CTX)Cyclophosphamide is only used in myeloablative conditioning (Cy，1.8g/m2, -5d, -4d) | Drug | HLA-mismatch related |
| Fludarabine (Fludara)For myeloablative conditioning: Fludarabine (Flu，30 mg/m2/d IV -6d ~ -2d) For reduced intensity conditioning: Fludarabine（Flu，30mg/m2 /d IV -10d~-5d） | Drug | HLA-mismatch related |
| Semustine (MeCCNU)For both myeloablative and reduced intensity conditioning: Semustine (MECCNU，250 mg/m2 orally-3d). | Drug | HLA-mismatch related |
Primary outcome
Overall survival
Time frame: 1-year
Overall survival after allogeneic cell transplantation refers to the length of time a patient remains alive from the date of transplantation, regardless of disease status or complications.
6 secondary outcomes
Neutrophil engraftment rate
Time frame: 28 days
Platelet engraftment rate
Time frame: 28 days
Relapse
Time frame: 1-year
Cumulative incidence of relapse
Time frame: 1-year
Progression free survival
Time frame: 1-year
GRFS
Time frame: 1-year
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 Feb 2025.
- 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.
Yishan Ye, MD., PhD contact
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Busulfan (Busulfex), Cyclophosphamide (CTX) and Fludarabine (Fludara) in people with Hematologic Diseases, run by He Huang 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 5 inclusion and 6 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 Feb 2025. 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.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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 Yanmin Zhao, MD., PhD (+86 87236706, yanminzhao@zju.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall survival (time frame: 1-year). 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 Feb 2025; AzTrials last refreshed its copy on 7 Nov 2026.