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Griscelli Syndrome clinical trials

View clinical trials related to Griscelli Syndrome.

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NCT ID: NCT05687474 Recruiting - Cystic Fibrosis Clinical Trials

Baby Detect : Genomic Newborn Screening

Start date: September 1, 2022
Phase:
Study type: Observational

Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions. With a simple blood test, rare genetic conditions can be easily detected, and the early start of transformative treatment will help avoid severe disabilities and increase the quality of life. Baby Detect Project is an innovative NBS program using a panel of target sequencing that aims to identify 126 treatable severe early onset genetic diseases at birth caused by 361 genes. The list of diseases has been established in close collaboration with the Paediatricians of the University Hospital in Liege. The investigators use dedicated dried blood spots collected between the first day and 28 days of life of babies, after a consent sign by parents.

NCT ID: NCT01652092 Recruiting - Clinical trials for Chronic Granulomatous Disease

Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

Start date: September 4, 2012
Phase: N/A
Study type: Interventional

This is a standard of care treatment guideline for allogeneic hematopoetic stem cell transplant (HSCT) in patients with primary immune deficiencies.

NCT ID: NCT00176865 Completed - Clinical trials for Hemophagocytic Lymphohistiocytosis

Stem Cell Transplant for Immunologic or Histiocytic Disorders

Start date: August 2002
Phase: Phase 2
Study type: Interventional

This study tests the clinical outcomes of a preparative regimen of fludarabine (FLU), anti-thymocyte globulin (ATG)/or Campath, and melphalan; followed by hematopoietic stem cell transplant, and a post transplant regimen of Cyclosporin A (CsA) in patients with immunologic or histiocytic disorders. The researchers hypothesize that this regimen will have a positive effect on post transplant engraftment and the incidence of graft-versus-host-disease (GVHD). Patients will be randomized biologically into one of 3 arms based upon donor availability: (a) human leukocyte antigen (HLA) genotypic matched sibling donor, (b) HLA phenotypic matched unrelated peripheral blood stem cell (PBSC) donor, (c) two HLA 0-2 antigen mismatched unrelated cord blood donors (double cord).

NCT ID: NCT00176826 Terminated - Clinical trials for Hematologic Diseases

T-Cell Depletion and Stem Cell Transplant for Immune Deficiencies and Histiocytic Disorders

Start date: September 2000
Phase: Phase 2/Phase 3
Study type: Interventional

The hypothesis is to determine if a preparative regimen of busulfan, cyclophosphamide, and antithymocyte globulin (ATG) plus allogeneic stem cell transplantation will be effective in the treatment of immune deficiencies and histiocytic disorders.