NCT03222453

Brief Summary

In order to study the transplantation effect of hematopoetic stem cells from beta-thalassemia induced pluripotent stem cells. We applied clinical grade source of autologous hematopoietic stem cell for the treatment of beta-thalassemia patients, detecting the homing of hematopoietic stem cell transplantation, the differentiation of hematopoietic stem cells in vivo and the hemoglobin beta-chain (HBB) protein expression in the body of recovery, etc., as well as to make a research on the efficacy and safety of hematopoietic stem cells from beta-thalassemia induced pluripotent stem cells.

Trial Health

35
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
2

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started Jan 2015

Typical duration for not_applicable

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 1, 2015

Completed
1.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 14, 2016

Completed
1 year until next milestone

First Submitted

Initial submission to the registry

June 23, 2017

Completed
26 days until next milestone

First Posted

Study publicly available on registry

July 19, 2017

Completed
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

December 31, 2017

Completed
Last Updated

July 19, 2017

Status Verified

July 1, 2017

Enrollment Period

1.5 years

First QC Date

June 23, 2017

Last Update Submit

July 17, 2017

Conditions

Beta-Thalassemia

Keywords

thalassemiastem cell

Outcome Measures

Primary Outcomes (3)

  • The check of granulocyte transplantation-granulocyte plant living standards

    Granulocyte plant living standards for three days in a row after transplantation granulocyte should be greater than 0.5 x 109 / L,

    3 days

  • The check of granulocyte transplantation-platelet plant living standards

    Platelet plant living standard for seven consecutive days after transplantation the platelet should be greater than 20 x 109 / L and infusion.

    seven consecutive days

  • Effect of cell transplantation

    The granulocyte plant living standards and platelet plant living standards will be combined to measure the effect of cell transplantation.

    seven days

Study Arms (2)

Intervention Patient

EXPERIMENTAL

Hematopoetic stem cells differentiated from beta-thalassemia induced pluripotent stem cells.

Biological: Hematopoetic stem cells

non-intervention Patient

NO INTERVENTION

No treatment

Interventions

Hematopoetic stem cellsBIOLOGICAL

Patient will inject with hematopoetic stem cells differentiated from beta-thalassemia induced pluripotent stem cells

Intervention Patient

Eligibility Criteria

Age1 Year - 18 Years
Sexmale(Gender-based eligibility)
Gender Eligibility DetailsClinical diagnosis.
Healthy VolunteersYes
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • The genetic diagnosis confirmed homozygous for beta to poverty or double heterozygote, clinical severe anemia;
  • Aged 1 \~ 18 years old, no obvious iron overload cause organ damage;
  • Has a suitable donor HLA high resolution;
  • The informed consent

You may not qualify if:

  • There has been a significant increase in the level of tumor markers (AFP/CEA/CA199 / CA125) in the past five years.
  • Serious primary diseases such as cardiovascular, liver and hematopoietic systems; Those who have major organs with serious function; An adrenal disease or other disease that causes the organ failure of the organ;
  • An autoimmune disease, a family history of genetic disease, and an abnormal thyroid function;
  • Peripheral blood chromosome checking for nuclear aliens;
  • HIV, hepatitis b or hepatitis c;
  • A person with a history of severe drug allergies or an allergic person;
  • Those who do not expect to live for more than one year;
  • The researchers suggest that the patient may have a potential or have a disorder (such as an uncontrolled infection, right heart failure, pulmonary hypertension) that is interfering with this study.
  • In the first six months, alcohol and other substance abuse were not allowed;
  • Subjects who participated in other clinical trials or participated in other clinical trials within 3 months.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

beta-ThalassemiaThalassemia

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NON RANDOMIZED
Masking
SINGLE
Who Masked
PARTICIPANT
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Principal Investigator

Study Record Dates

First Submitted

June 23, 2017

First Posted

July 19, 2017

Study Start

January 1, 2015

Primary Completion

June 14, 2016

Study Completion

December 31, 2017

Last Updated

July 19, 2017

Record last verified: 2017-07