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Wiskott-Aldrich Syndrome clinical trials

View clinical trials related to Wiskott-Aldrich 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: NCT04371939 Recruiting - Clinical trials for Wiskott-Aldrich Syndrome

Efficacy and Safety of Romiplostim Versus Eltrombopag in the Treatment of Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome

Start date: November 5, 2019
Phase: Phase 2
Study type: Interventional

This is a prospective, open-label, randomized, two-arm clinical trial conducted to evaluate the safety and efficacy of romiplostim in comparison with eltrombopag in the treatment of thrombocytopenia in patients with Wiskott-Aldrich syndrome

NCT ID: NCT03019809 Recruiting - Clinical trials for Hematopoietic Stem Cell Transplantation

A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS Patients

Start date: June 2016
Phase: Phase 2
Study type: Interventional

Treatment Study to assess of safety and efficiency of conditioning with Plerixafor and G-CSF as additional agents for prevention of graft failure after transplantation with TCR alpha/beta grafts depletion in patient with Wiskott-Aldrich syndrome.

NCT ID: NCT01821781 Recruiting - Clinical trials for Chronic Granulomatous Disease

Immune Disorder HSCT Protocol

Start date: March 2013
Phase: Phase 2
Study type: Interventional

This study hypothesizes that a reduced intensity immunosuppressive preparative regimen will establish engraftment of donor hematopoietic cells with acceptable early and delayed toxicity in patients with immune function disorders. A regimen that maximizes host immune suppression is expected to reduce graft rejection and optimize donor cell engraftment.

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.