Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID-X1)

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Brief Title

Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID-X1)

Official Title

Gene Therapy for SCID-X1 Using a Self-inactivating (SIN) Gammaretroviral Vector

Brief Summary

      X-linked severe combined immunodeficiency (SCID-X1) is an inherited disorder that results in
      failure of development of the immune system in boys. This trial aims to treat SCID-X1
      patients using gene therapy to replace the defective gene.
    



Study Type

Interventional


Primary Outcome

Immunological reconstitution

Secondary Outcome

 Incidence of adverse reactions

Condition

X-linked Severe Combined Immunodeficiency

Intervention

Single infusion of autologous CD34+ cells transduced with the self-inactivating (SIN) gammaretroviral vector pSRS11.EFS.IL2RG.pre

Study Arms / Comparison Groups

 Single infusion of autologous CD34+ cells
Description:  

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

Genetic

Estimated Enrollment

1

Start Date

April 2011

Completion Date

December 2018

Primary Completion Date

December 2018

Eligibility Criteria

        Inclusion Criteria:

          1. No HLA identical (A,B,C,DR,DQ) family donor and no HLA identical unrelated donor
             available within 3 months of diagnosis or patients whose underlying clinical problems
             and prognosis would be significantly compromised by chemotherapy conditioning
             (including persisting pneumonitis, protracted diarrhoea requiring parental nutrition,
             ongoing visceral viral infection (herpes viruses, HSV,VZV,CMV, EBV or adenovirus),
             systemic BCG infection, virus-induced lymphoproliferation.

          2. Diagnosis of classical SCID-X1 based on immunophenotype (absent, or reduced numbers of
             non-functional T lymphocytes) and confirmed by DNA sequencing

          3. Parental/guardian voluntary consent

          4. Boys between the ages of 0 and 16
      

Gender

Male

Ages

N/A - 16 Years

Accepts Healthy Volunteers

No

Contacts

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Location Countries

United Kingdom

Location Countries

United Kingdom

Administrative Informations


NCT ID

NCT01175239

Organization ID

06MI10


Responsible Party

Sponsor

Study Sponsor

Great Ormond Street Hospital for Children NHS Foundation Trust


Study Sponsor

, , 


Verification Date

October 2016