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β-thalassemiaEarly Phase 1NCT05864170

the Safety and Efficacy Evaluation of HGI-001 Injection in Patients With Transfusion-Dependent β-Thalassemia

A Early Phase 1 study testing β-globin restored autologous hematopoietic stem cells in people with β-thalassemia, run by Shenzhen Hemogen at 1 site in China.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

This is an open label study to evaluate the safety and efficacy of β-globin Restored Autologous Hematopoietic Stem Cells in ß-Thalassemia Major Patients Read more about this study

Who is it for?
Ages 18 to 35 years
Conditions the study is for
β-thalassemia
What is being tested?
β-globin restored autologous hematopoietic stem cells
Could I receive a placebo?
No, everyone receives an active treatment
How many people
3 participants
Where
1 siteChina
Study dates
27 May 2022to 30 Dec 2025
Phase
Early 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 35 years
  • All sexes
  • No healthy volunteers
  • Adult

Who may be able to join

  • Aged 18-35 years (inclusive), ICF can be provided by the patient and/or legal guardian;
  • Definitively diagnosed with severe TDT without genotype restriction, and a valid test report can be provided;
  • Average transfusion volume > 100 mL/kg/year or transfusion frequency > 8 times/year within 2 years prior to enrollment, or has been definitively diagnosed with TDT;
  • At least 3 months of full volume transfusion (verification of blood transfusion records can be provided) prior to screening, and Hb is maintained at ≥ 9.0 g/dL;
  • Ferritin load < 3000 μg/L, cardiac and liver iron indicates moderate or lesser iron overload; records of iron chelation treatments within 3 months before screening (including prescription or receipt) can be provided;
  • Acceptable organ functions (including heart, liver, kidney, lung and coagulation functions), stable disease condition, and suitable for busulfan pre-treatment and hematopoietic stem cell (HSC) transplantation as judged by the investigator;
  • Meets follow-up requirements, adheres to treatment arrangements, and is able to return to the hospital regularly to undergo various examinations within 2 years after reinfusion of HGI-001 injection.

Who cannot join

  • Patients with fully HLA-matched donors;
  • Received allogeneic transplantation, which needs to be weighed and evaluated by an expert committee; received other gene therapies;
  • Have previously undergone splenectomy;
  • Uncorrected bleeding disorder;
  • Uncontrolled epilepsy and mental illness;
  • Received hydroxyurea, ruxolitinib, decitabine, or cytarabine within 3 months prior to enrollment;
Show all 19Show fewer
  • Psychoactive substance abuse, drug or alcohol abuse within 6 months prior to enrollment;
  • Patients with pulmonary hypertension who have not been given effective intervention;
  • Persistent toxicity (≥ CTCAE grade 2) induced by previous treatment;
  • Positive for anti-RBC antibodies in antibody screening;
  • Positive for hepatitis B surface antigen (HBsAg) and HBV DNA copy number > upper limit of normal (ULN) (HBV DNA test not required for patients negative for HBsAg), positive for hepatitis C virus (HCV) antibody, positive human immunodeficiency virus (HIV), or positive for Treponema pallidum antibody (TP-Ab) (subjects who are positive for the antibody due to vaccination can be enrolled). In certain clinical environments/regions, subjects who are positive for other tests can also be excluded from the trial, such as, human lymphocytic virus-1 (HTLV-1) or -2 (HTLV-2), tuberculosis, and toxoplasmosis.
  • Has or has had malignant tumors or myeloproliferative disease or immunodeficiency disease;
  • Immediate family member with or suspected of having a familial cancer (including but not limited to hereditary breast and ovarian cancers, nonpolyposis colorectal cancer, and adenomatous polyposis);
  • Severe bacterial, viral, fungal or parasitic infection;
  • Other illnesses which render the subject unsuitable for participation (e.g., severe liver, kidney or heart disease); Definition of severe liver and kidney disease: a. Aspartate aminotransferase (AST), alanine aminotransferase (ALT), or total bilirubin > 3 × ULN; b. Liver magnetic resonance imaging (MRI) indicates significant cirrhosis; c. Liver biopsy indicates cirrhosis, severe fibrosis or active hepatitis (liver biopsy is only performed when liver MRI indicates active hepatitis and significant fibrosis without evidence for cirrhosis); d. Creatinine clearance < 30% of normal;
  • WBC < 3 × 109/L and/or PLT < 100 × 109/L;
  • Has diabetes, abnormal thyroid functions or other endocrine disorder;
  • Participated in other interventional clinical studies within 4 weeks before the trial;
  • Poor adherence or other conditions that renders the subject unsuitable for participation as judged by the investigator.

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Haigang Sun contact

13823168465

sunhaigang@genomics.cn

China1 site, 1 recruiting

  • Shenzhen University General Hospital

    Shenzhen, Guangdong

    Haigang Sun, 13823168465, sunhaigang@genomics.cn

    Recruiting

Other trials in China

About this study

Official title: the Safety and Efficacy Evaluation of HGI-001 Injection in Patients With Transfusion-Dependent β-Thalassemia(Child)

This is an open label study to evaluate the safety and efficacy of β-globin Restored Autologous Hematopoietic Stem Cells in ß-Thalassemia Major Patients

Read the detailed description

We will recruit ß-thalassaemia major patients and collect their autologous hematopoietic stem cells, which will be modified with the LentiHBBT87Q system to restore β-globin expression. After conditioning, the autologous hematopoietic stem cells with restored β-globin will be reinfused to the patients and followed up for two years to collect data.

Conditions
β-thalassemia
Being tested
β-globin restored autologous hematopoietic stem cells

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • β-globin restored autologous hematopoietic stem cellsBiological

    β-globin-restored autologous hematopoietic stem cells modified with LentiHBBT87Q

Study groups in NCT05864170
Study groupRoleWhat they receive
ExperimentalExperimentalThree transfusion-dependent β-thalassaemia subjects aged 18-35 years will be reinfused with β-globin restored autologous hematopoietic stem cells modified with LentiHBBT87Q
Receives: Biological: β-globin restored autologous hematopoietic stem cells

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Overall response rate

    Percent of patients with average VCN > 0.1 in peripheral blood mononuclear cells (PBMCs) and average expression of exogenous adult hemoglobin HbAT87Q > 2.0 g/dL

    Measured: 24 months

  2. Incidence and severity of AEs

    The number and the percentage of adverse events related to transplantation will be summarized according to NCI CTCAE 5.0

    Measured: 0-24 months

  3. Incidence of SAEs

    The number of SAE related to transplantation will be summarized according to NCI CTCAE 5.0

    Measured: 0-24 months

  4. Transplantation-related fatal and disabling events within day 100 after transplantation

    Transplantation-related fatal and disabling events

    Measured: Day 100

  5. Overall survival rate during the clinical trial

    Number of patients alive through the whole trial will be record

    Measured: 0-24 months

  6. HGI-001 injection-related replicating lentivirus test

    The percentage of RCL should be negative in the 24 months after transplant

    Measured: 0-24 months

  7. Change from baseline in Clonal variations containing specific viral integration sites

    Evaluation of the percentage of participants without abnormal clonal proliferation and polyclonal engraftment at 6, 12, 18 and 24 months after transplant. More than 1000 VIS retrieved from peripheral blood should be checked.

    Measured: 0-24 months

  8. Number of patients with abnormal hematology and bone marrow cytology within 24 months after reinfusion, and percent of patients with abnormal RBC proliferation

    Number of patients with abnormal hematology and bone marrow cytology

    Measured: 0-24 months

Secondary outcomes (11)

  1. Treatment response rate

    Percent of patients with average VCN > 0.1 in PBMCs and average expression of exogenous adult hemoglobin HbAT87Q > 2.0 g/dL after reinfusion of HGI-001 injection

    Measured: 12 Months

  2. Percent of subjects with successful HSC engraftment

    Criteria for successful engraftment: Absolute neutrophil count > 0.5 × 10 9 /L for 3 consecutive days; platelet count is maintained at > 20 × 10 9 /L for 7 consecutive days without platelet transfusion

    Measured: 1 month

  3. Change in transfusion volume or frequency

    Change in average annual transfusion volume or frequency from baseline or change in percentage

    Measured: 0-24 Months

  4. Transfusion improvement rate

    Percent of subjects with ≥ 30% decrease in the average annual (0-12 months, 12-24 months) transfusion volume or frequency from baseline after reinfusion of HGI-001 injection

    Measured: 0-24 Months

  5. Transfusion independence (TI) rate

    Percent of subjects who do not require transfusion for at least 12 consecutive months after reinfusion of HGI-001 injection and have a weighted average Hb of ≥ 9.0 g/dL

    Measured: 0-24 Months

  6. Transfusion-free survival

    the time when a subject meets the TI criteria and maintains transfusion-free survival

    Measured: 0-24 Months

  7. Changes in VCN and exogenous adult HbAT87Q expression

    Vector copy number

    Measured: 0-24 Months

  8. Changes in cardiac iron load after reinfusion of HGI-001 injection

    T2 MRI

    Measured: 0-24 Months

  9. Changes in liver iron load after reinfusion of HGI-001 injection

    T2 MRI

    Measured: 0-24 Months

  10. Changes in serum ferritin after reinfusion of HGI-001 injection

    serum ferritin

    Measured: 0-24 Months

  11. Changes use of iron chelation medications after reinfusion of HGI-001 injection

    iron chelation medications

    Measured: 0-24 Months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted18 May 2023
  2. Study start27 May 2022
  3. Primary completion30 Dec 2025 (estimated)
  4. Study completion30 Dec 2025 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Early Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
3 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05864170
Sponsor's protocol ID
HGI-001-CTP
Status last verified
Nov 2024
First submitted
28 Feb 2023
Last updated
29 Nov 2024
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Early Phase 1 study testing β-globin restored autologous hematopoietic stem cells in people with β-thalassemia, run by Shenzhen Hemogen 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 7 inclusion and 19 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 29 Nov 2024. 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?

Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.

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 Haigang Sun (13823168465, sunhaigang@genomics.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Overall response rate (time frame: 24 months). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05864170.

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 29 Nov 2024.

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