Multi-center Clinical Study of Cord Blood Stem Cell Transplantation for SCID

Related Clinical Trial
cliniMACs HUD for T Cell Depletion Cord Blood Stem Cell Transplantation Study (COBLT) Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products Lentiviral Gene Therapy for Adenosine Deaminase (ADA) Deficiency Genetic Basis of Immunodeficiency IMM 0212: Busulfan With Fludarabine and Antithymocyte Globulin as Preparative Therapy for Hematopoietic Stem Cell Transplant for the Treatment of Severe Congenital T-Cell Immunodeficiency Sirolimus Prophylaxis for aGVHD in TME SCID Study Through Imaging of Visceral Lymphoid Organs in Patients With SCID Who Have Recieved Bone Marrow Allograft Influences on Female Adolescents’ Decisions Regarding Testing for Carrier Status of XSCID Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow Transplant EZN-2279 in Patients With ADA-SCID AMG191 Conditioning/CD34+CD90 Stem Cell Transplant Study for SCID Patients Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID-X1) Newborn Screening for Severe Combined Immunodeficiency (SCID) in a High-Risk Population Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning Allogeneic SCT Of Pts With SCID And Other Primary Immunodeficiency Disorders Stem Cell Gene Therapy to Treat X-Linked Severe Combined Immunodeficiency (XSCID) Patients Treated for SCID (1968-Present) Clinical Characteristics and Genetic Profiles of Severe Combined Immunodeficiency in China Gene Therapy for ADA-SCID MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID Transplantation of Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome Hematopoietic Stem Cell Transplantation (HSCT) for Children With SCID Utilizing Alemtuzumab, Plerixafor & Filgrastim Natural History Study of SCID Disorders Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study Safety and Early Efficacy Study of TBX-1400 in Patients With Severe Combined Immunodeficiency SCID Bu/Flu/ATG Study With T Cell Depletion Generalized Neonatal Screening of Severe Combined Immunodeficiencies Neonatal Screening of Severe Combined Immunodeficiencies Gene Therapy for X-linked Severe Combined Immunodeficiency An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID Haplocompatible Transplant Using TCRα/β Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID) Registry Study of Revcovi Treatment in Patients With ADA-SCID Multi-center Clinical Study of Cord Blood Stem Cell Transplantation for SCID

Brief Title

Multi-center Clinical Study of Cord Blood Stem Cell Transplantation for SCID

Official Title

Multi-center Clinical Study of Cord Blood Stem Cell Transplantation for Severe Combined Immunodeficiency Disease

Brief Summary

      Severe combined immunodeficiency (SCID) is a rare disease caused by a group of genetic
      disorders that leads to early death from recurrent infections in affected children.The only
      curative therapy for SCID is allogeneic hematopoietic stem cell transplantation.Unrelated
      umbilical cord blood(UCB) is increasingly used as an alternative to bone marrow.
    

Detailed Description

      Severe combined immunodeficiency (SCID) is a rare disease caused by a group of genetic
      disorders that leads to early death from recurrent infections in affected children.The only
      curative therapy for SCID is allogeneic hematopoietic stem cell transplantation.Unrelated
      umbilical cord blood(UCB) is increasingly used as an alternative to bone marrow.The
      development of NBS in Europe and America has enabled more SCID patients to be diagnosed
      before symptoms appear, and to enter the normative assessment and intervention procedures as
      soon as possible. In China SCID has not been included in the NBS program,and there are few
      units that can diagnose SCID.Patients often delay diagnosis and miss the best period of
      transplantation. Department of Hematology, Children's Hospital of Fudan University, has used
      UCBT for the treatment of SCID patients with reduced intensity conditioning(RIC)since 2014.6
      of 8 cases were treated with disease-free survival. The encouraging efficacy of these
      patients suggests that RIC of UCBT may be an effective treatment for SCID patients to
      urgently hematopoietic stem cell transplantation. The aim of this study is to investigate the
      efficacy of UCBT in the treatment of SCID, including engraftment rate, disease-free survival
      rate,overall survival rate and immune reconstitution, and to evaluate transplant-related
      mortality and complications. All the selected cases are diagnosed as severe combined
      immunodeficiency disease by immunological function and gene detection. These patients have no
      matched sibling donors. Their organs function should be normal. The guardian of the patient
      has the desire and requirement for UCBT and signs the informed consent before treatment. Cord
      blood stem cell selection:HLA high-resolution detection of patients before transplantation,
      searching through cord blood stem cell bank, selecting cord blood stem cells that meet the
      following criteria: HLA-A, B, C, DRB1 high-resolution (genotype) > 6/8matching,total number
      of nuclear cells >5x10^7/kg.Conditioning:busulfan+ cyclophosphamide.GVHD prevention:
      tacrolimus (FK506) or cyclosporine A. Infection prevention: Micafungin/caspofungin before
      engraftment, voriconazole after engraftment to prevent fungi. Ganciclovir is used from the
      beginning of conditioning to the infusion of cord blood stem cells, and acyclovir is used to
      prevent virus infection. SMZ prevents Pneumocystis carinii infection after engraftment until
      half a year after the withdrawal of immunosuppressive agents.

      This is a multicenter prospective observational study. Procedure/Surgery: Cord Blood Stem
      Cell Transplantation Unrelated cord blood stem cell selection: HLA high-resolution detection
      is performed before transplantation. High-resolution (genotype) matching of HLA-A, B, C and
      DRB1 was selected. The total number of nuclear cells is more than 5*107/kg.

      Reduced intensity conditioning : Busulphan 3.2mg/kg per day in divided doses for 3 days(total
      dose 9.6 mg/kg) and cyclophosphamide 50 mg/kg for 2 days (total dose 100 mg/kg).

      GVHD prevention: tacrolimus (FK506) 0.1 mg/kg/day, started 4 days before transplantation, is
      taken orally twice, and the blood concentration is monitored to maintain the concentration at
      5-10 ng/ml. If the patient does not have a GVHD transplant +100 days after the slow
      reduction, until the transplant 6 months after the withdrawal.

      Infection prevention: Micafungin/caspofungin before engraftment, voriconazole after
      engraftment to prevent fungi. Ganciclovir is used from the beginning of pretreatment to the
      beginning of reinfusion, and acyclovir is used to prevent virus infection until
      immunosuppressive agents are discontinued after reinfusion. SMZ prevents Pneumocystis carinii
      infection after engraftment.
    


Study Type

Observational


Primary Outcome

Overall survival rate

Secondary Outcome

 Disease free survival rate

Condition

Severe Combined Immunodeficiency Disease

Intervention

cord blood stem cell transplantation

Study Arms / Comparison Groups

 UCBT-SCID-Case
Description:  SCID patients who underwent cord blood stem cell transplantation.The only curative therapy for SCID is allogeneic hematopoietic stem cell transplantation.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Procedure

Estimated Enrollment

50

Start Date

December 1, 2019

Completion Date

October 31, 2023

Primary Completion Date

October 31, 2022

Eligibility Criteria

        Inclusion Criteria:

          1. All patients were diagnosed as severe combined immunodeficiency disease by
             immunological function and genetic diagnosis center.

          2. Patients have no HLA-matched related donor.

          3. Each organ functions normally and conforms the following inspection criteria:

        Liver function ALT, AST ≤ 10 times the upper limit of normal value, TBIL ≤ 5 times the
        upper limit of normal value.

        Renal function BUN, Cr ≤ 1.25 times the upper limit of normal value. ECG, cardiac
        examination normal

        Exclusion Criteria:

          1. Patients have any contraindications to hematopoietic stem cell transplantation.

          2. Patients have other serious diseases, such as serious damage to vital organ function:
             respiratory failure, cardiac insufficiency, decompensated liver dysfunction, renal
             insufficiency, uncontrollable infection, etc.

          3. The patient is undergoing other drug clinical research.

          4. At the same time suffering from other serious acute or chronic physical or mental
             illness, or laboratory abnormalities, may affect patient safety and compliance,
             affecting informed consent, research participation, follow-up or interpretation of
             results.
      

Gender

All

Ages

1 Month - 18 Years

Accepts Healthy Volunteers

No

Contacts

jiang hui, master, , 

Location Countries

China

Location Countries

China

Administrative Informations


NCT ID

NCT04172181

Organization ID

CPBMT-SCID-2019


Responsible Party

Principal Investigator

Study Sponsor

Children's Hospital of Fudan University

Collaborators

 Shanghai Children's Hospital

Study Sponsor

jiang hui, master, Study Director, Shanghai Children's Hospital


Verification Date

January 2020