Trials Sponsors Conditions Drugs Pricing

Conditions

β-thalassemia Major

Outcome Measures

Primary Outcomes (2)

Proportion of subjects with engraftment;
up to 42 days post transplant
Incidence and severity of adverse events as a measure of safety and tolerability. Adverse events assessed according to NCI-CTCAE v5.0 criteria
up to 60 days post transplant

Secondary Outcomes (2)

Proportion of subjects achieving transfusion independence;
up to 24 months post transplant
Proportion of subjects with a > = 50% reduced annualized volume of packed RBC transfusions.
up to 24 months post transplant

Study Arms (1)

β-globin restored autologous HSC

EXPERIMENTAL

each subject will accept one dose of β-globin restored autologous hematopoietic stem cells

Biological: β-globin restored autologous HSC

Interventions

β-globin restored autologous HSCBiological

gene edited autologous hematopoietic stem cells with β-globin restoration

β-globin restored autologous HSC

Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.

Eligibility Criteria

Age5 Years - 15 Years

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17)

You may qualify if:

years old. Clinically diagnosed as β-thalassemia major with IVS-654 gene mutation phenotype;
Subjects or at least one legal guardian/agent understand and voluntarily sign informed consent.

Sponsors & Collaborators

Bioray Laboratories
Industrylead
The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
Othercollaborator

Study Sites (1)

Shanghai Bioraylaboratory Inc

Shanghai, Shanghai Municipality, 200241, China

Related Publications (1)

Brusson M, Miccio A. Genome editing approaches to beta-hemoglobinopathies. Prog Mol Biol Transl Sci. 2021;182:153-183. doi: 10.1016/bs.pmbts.2021.01.025. Epub 2021 Mar 1.
PMID: 34175041DERIVED

Study Officials

Xinhua Zhang, Prof
The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
PRINCIPAL INVESTIGATOR

Enrollment

2 participants (actual enrollment reported by the registry).

Study Design

Study Type: interventional
Phase: phase 1 2
Allocation: NA
Masking: NONE
Purpose: TREATMENT
Intervention Model: SINGLE GROUP
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

First Submitted

December 18, 2019

First Posted

December 19, 2019

Study Start

November 1, 2021

Actual (reported)

Study Stopped

β-globin Restored Autologous HSC in β-thalassemia Major Patients

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Outcome Measures

Primary Outcomes (2)

Secondary Outcomes (2)

Study Arms (1)

β-globin restored autologous HSC

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

Related Publications (1)

Study Officials

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Outcome Measures

Primary Outcomes (2)

Secondary Outcomes (2)

Study Arms (1)

β-globin restored autologous HSC

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

Related Publications (1)

Study Officials

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations