---
title: Safety and Efficacy Evaluation of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia
nct_id: NCT04390971
overall_status: COMPLETED
phase: NA
sponsor: Institute of Hematology & Blood Diseases Hospital, China
study_type: INTERVENTIONAL
primary_condition: Transfusion Dependent Beta-Thalassaemia
countries: China
canonical_url: "https://parkinsonspathways.com/agent/trials/NCT04390971.md"
clinicaltrials_gov: "https://clinicaltrials.gov/study/NCT04390971"
ct_last_update_post_date: 2026-02-11
last_seen_at: "2026-05-12T07:21:46.985Z"
source: ClinicalTrials.gov (mirrored, no enrichment)
---
# Safety and Efficacy Evaluation of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia

**Official Title:** A Multi-center, Open Label Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassemia

**NCT ID:** [NCT04390971](https://clinicaltrials.gov/study/NCT04390971)

## Key Facts

- **Status:** COMPLETED
- **Phase:** NA
- **Study Type:** INTERVENTIONAL
- **Target Enrollment:** 3
- **Lead Sponsor:** Institute of Hematology & Blood Diseases Hospital, China
- **Collaborators:** EdiGene Inc., The Affiliated Hospital Of Guizhou Medical University, Zunyi Medical College
- **Conditions:** Transfusion Dependent Beta-Thalassaemia
- **Start Date:** 2023-02-10
- **Completion Date:** 2023-03-05
- **CT.gov Last Update:** 2026-02-11

## Brief Summary

This is an open label, multi-center study to evaluate the safety and Efficacy of ET-01 Transplantation in subjects with Transfusion Dependent β-Thalassaemia.

## Detailed Description

After the proper subject is recruited, subject will go through steps generally as stem cell mobilization, apheresis, conditioning and ET-01 infusion to complete therapy. Two years follow-up will be carried out post-transplantation and related data will be collected. Subjects who have been treated by ET-01 will be asked to participate in a long-term follow up study. Monitoring the long-term efficacy and safety up to 15 years after ET-01 transplant.

## Eligibility

- **Minimum age:** 6 Years
- **Maximum age:** 35 Years
- **Sex:** ALL
- **Healthy Volunteers:** No

```
Inclusion Criteria:

* Subject and/or subject's legal personal representative fully understand and voluntarily sign informed consent forms;
* 6\~35 years old, all gender;
* Diagnosis of transfusion dependent β-thalassemia (β-TDT) as defined by protocol;
* Eligible for autologous stem cell transplant;
* Eligible for autologous stem cell transplant;
* Organs in good function.
* Other protocol defined inclusion criteria may apply.

Exclusion Criteria:

* Subjects with associated α-thalassemia;
* Active bacterial, virus, fungal or parasitic infection, including HIV infection, HbsAg and HBV DNA positive, HCV DNA positive, or Treponema Pallidum infection;
* HLA identical sibling or unrelated donors are available;
* Prior allo-HSCT or gene therapy.
* Other protocol defined exclusion criteria may apply.
```

## Arms

- **ET-01** (EXPERIMENTAL) — BCL11A Enhancer modified Autologous Hematopoietic Stem Cells.

## Interventions

- **ET-01** (BIOLOGICAL) — Recruited participants will receive ET-01 IV infusion after conditioning.

## Primary Outcomes

- **Frequency and severity of AEs & SAEs identified according to NCI CTCAE 5.0.** _(time frame: From ET-01 infusion to 104 weeks post-transplant)_
- **All-cause mortality** _(time frame: From signing of informed consent to 104 weeks post-transplant)_
- **Incidence of transplant-related mortality** _(time frame: Within 100 days post-transplant)_
- **Proportion of subjects with engraftment** _(time frame: Up to 42 days post-transplant)_

## Secondary Outcomes

- **Change of total hemoglobin from baseline** _(time frame: Within 104 weeks post-transplant)_
- **Change of HbF from baseline** _(time frame: Within 104 weeks post-transplant)_
- **Change of proportion of HbF/Hb** _(time frame: Within 104 weeks post-transplant)_
- **Change of frequency of packed RBC transfusions** _(time frame: From 6 months before recruitment to 104 weeks post-transplant)_
- **Change of volume of packed RBC transfusions** _(time frame: From 6 months before recruitment to 104 weeks post-transplant)_

## Locations (3)

- The Affiliated Hospital of Guizhou Medical University, Guiyang, Guizhou, China
- Affiliated Hospital of Zunyi Medical University, Zunyi, Guizhou, China
- Institute of Hematology & Blood Diseases Hospital, Tianjin, Tianjin Municipality, China

## Recent Field Changes (last 30 days)

- `status.completionDate` — added _(2026-05-12)_
- `status.lastUpdatePostDate` — added _(2026-05-12)_
- `design.phases` — added _(2026-05-12)_
- `design.enrollmentCount` — added _(2026-05-12)_
- `status.overallStatus` — added _(2026-05-12)_
- `status.primaryCompletionDate` — added _(2026-05-12)_
- `eligibility.criteria` — added _(2026-05-12)_
- `eligibility.minAge` — added _(2026-05-12)_
- `eligibility.maxAge` — added _(2026-05-12)_
- `eligibility.sex` — added _(2026-05-12)_
- `outcomes.primary` — added _(2026-05-12)_
- `outcomes.secondary` — added _(2026-05-12)_
- `armsInterventions.arms` — added _(2026-05-12)_
- `armsInterventions.interventions` — added _(2026-05-12)_
- `sponsor.lead` — added _(2026-05-12)_
- `sponsor.collaborators` — added _(2026-05-12)_
- `results.hasResults` — added _(2026-05-12)_
- `locations.the affiliated hospital of guizhou medical university|guiyang|guizhou|china` — added _(2026-05-12)_
- `locations.affiliated hospital of zunyi medical university|zunyi|guizhou|china` — added _(2026-05-12)_
- `locations.institute of hematology & blood diseases hospital|tianjin|tianjin municipality|china` — added _(2026-05-12)_

---

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*Source data (authoritative): https://clinicaltrials.gov/study/NCT04390971*  
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