Clinical trials: Chronic Granulomatous Disease
IFN-Gamma Therapy, CGD Gene Mutation
Biochemical Response to Interferon-Gamma in Subjects With Specific Gene Mutation in Chronic Granulomatous Disease
Background: Chronic granulomatous disease (CGD) is an immunodeficiency disease in which white blood cells are unable to kill certain bacteria and fungi.
- Conditions
- IFN-Gamma Therapy, CGD Gene Mutation, CGD Response to IFNg, CGD - Chronic Granulomatous Disease
- Trial name
- NCT01147042
- Testing
- IFN-gamma
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Chronic Granulomatous Disease
IL-1 Receptor Inhibitor for Granulomatous Complications in Patients With Chronic Granulomatous Disease
"Kineret" (INN: Anakinra) neutralizes the biological activity of interleukin-1α (IL-1α) and interleukin-1β (IL-1β) by the concurrent inhibition of binding to interleukin-1 receptor I (IL-1RI).
- Trial name
- NCT04136028
- Testing
- Kineret
- Where
- Russia
- Volunteers
- 13 people
- Sponsor
- Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Vitamin D3, Chronic-granulomatous Disease
Vitamin D3 For CGD Patients With BCGosis/Itis
When children with chronic granulomatous disease (CGD) got BCG infection the treatment would be a tough task. The goal of the proposed research is to observe weather vitamin D supplementation can help the CGD children get through…
- Conditions
- Vitamin D3, Chronic-granulomatous Disease, BCG
- Trial name
- NCT03984890
- Testing
- Vitamin D3, Traditional treatment of CGD and TB
- Where
- 2 sites in China
- Volunteers
- 50 people
- Sponsor
- Children's Hospital of Fudan University
Granulomatous Disease, Chronic, Job's Syndrome
Posaconazole to Treat Invasive Fungal Infections
This study will evaluate the safety and effectiveness of posaconazole for treating invasive fungal infections. New therapies for these infections are needed for patients who do not respond, to or cannot tolerate, standard…
- Conditions
- Granulomatous Disease, Chronic, Job's Syndrome, Infection
- Trial name
- NCT00033982
- Testing
- Posaconazole
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Chronic Granulomatous Disease Transplant
High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous Disease
Chronic granulomatous disease (CGD) affects white blood cell function. Currently, the only curative treatment is bone marrow transplant to replace the abnormal stem cells with new ones (donor cells) capable of making a normal…
- Trial name
- NCT02629120
- Testing
- Alemtuzumab, Busulfan, Sirolimus
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Chronic Granulomatous Diseases (CGD) and Liver Lesions
Radiofrequency Ablation for Liver Abscesses From Chronic Granulomatous Disease
Background: Abscesses are a pocket of infection in an organ or tissue. Patients with a disease called chronic granulomatous disease (CGD) often develop these abscesses.
- Trial name
- NCT01851460
- Testing
- Cool-tip RF Ablation System
- Where
- United States
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
X-Linked Chronic Granulomatous Disease
Gene Therapy for X-linked Chronic Granulomatous Disease (X-CGD)
X-linked chronic granulomatous disease (X-CGD) is a rare genetic disorder, which affects boys. It is caused by an error in a gene that makes part of the immune system.
- Trial name
- CGD (NCT01855685)
- Testing
- X vivo gene therapy
- Where
- 2 sites in United Kingdom
- Volunteers
- 3 people
- Sponsor
- Genethon
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.
- Conditions
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies
- Trial name
- NCT02231710
- Testing
- BPX-501 and Rimiducid
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Bellicum Pharmaceuticals
Thalassemia, Sickle Cell Disease
Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation
Allogeneic blood and marrow transplantation remains the only viable cure for children who suffer from many serious non-malignant hematological diseases. Transplantation, however, carries a high risk of fatal complications.
- Conditions
- Thalassemia, Sickle Cell Disease, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome
- Trial name
- NCT01319851
- Testing
- Alefacept
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Emory University
HIV, Cancer
Study of Voicing My CHOiCES as a Tool for Advanced Care Planning in Young Adults With Cancer
Background: There are very few documents to help young adults living with advanced cancer discuss their concerns and end-of-life preferences.
- Conditions
- HIV, Cancer, Chronic Granulomatous Disease, Dock 8 Deficiency
- Trial name
- NCT02108028
- Where
- 9 sites in United States
- Volunteers
- 272 people
- Sponsor
- National Cancer Institute (NCI)
Chronic Granulomatous Disease (CGD)
Open-label Gene Therapy Study in p47-CGD
Chronic granulomatous disease (CGD) caused by p47phox deficiency (p47-CGD) is a life-threatening genetic disorder causing nicotinamide adenine dinucleotide phosphate (NADPH) oxidase deficiency in phagocytes.
- Trial name
- NCT07826767
- Testing
- SGX-001
- Where
- 3 sites in Germany, Spain and 1 more
- Volunteers
- 5 people
- Sponsor
- Somagenetix AG
Chronic Granulomatous Disease
Treatment of Chronic Granulomatous Disease With Allogeneic Stem Cell Transplantation Versus Standard of Care
This study will compare the health and well being of children treated with a modified stem cell transplantation procedure for chronic granulomatous disease (CGD) with that of children receiving standard of care treatment.
- Trial name
- NCT00023192
- Testing
- T-Cell Depleted & CD34+Select/w/StemCell Enriched Product
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Chronic Granulomatous Disease
Effect of IFN-γ on Innate Immune Cells
The investigators hypothesize that neutrophils and monocytes developed under the influence of Interferon- gamma-1b (IFN-γ-1b, Actimmune*) in vivo will display enhanced function across a broad range of activities related in large…
- Trial name
- NCT02609932
- Testing
- Administration of drug (Interferon-gamma 1-b) subcutaneously
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- University of Colorado, Denver
X-linked Chronic Granulomatous Disease
Gene Therapy for X-CGD
X-linked chronic granulomatous disease (X-CGD) is a rare inherited immune defect, which is caused by the inability of phagocytic cells to produce reactive oxygen species due to a defect in the gp91phox subunit of the NADPH…
- Trial name
- NCT01906541
- Testing
- ex-vivo gene-therapy
- Where
- Germany
- Volunteers
- 5 people
- Sponsor
- Hubert Serve, Prof., MD
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission
Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer
This phase II trial studies how well fludarabine phosphate, cyclophosphamide, total body irradiation, and donor stem cell transplant work in treating patients with blood cancer.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
- Trial name
- NCT03333486
- Testing
- Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Roswell Park Cancer Institute
Chronic Granulomatous Disease (CGD)
Prevalence of Antibodies and Cytokines in Participants With Chronic Granulomatous Disease
This research study is designed to assess the prevalence of specific antibodies and inflammatory cytokines in adult and pediatric participants with CGD.
- Trial name
- NCT06605378
- Where
- 52 sites in United States, United Kingdom
- Volunteers
- 60 people
- Sponsor
- Ensoma
Stem Cell Transplantation, Bone Marrow Transplantation
Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells
Many genetic diseases of lymphohematopoietic cells (such as sickle cell anemia, thalassemia, Diamond-Blackfan anemia, Combined Immune Deficiency (CID), Wiskott-Aldrich syndrome, chronic granulomatous disease, X-linked…
- Conditions
- Stem Cell Transplantation, Bone Marrow Transplantation, Peripheral Blood Stem Cell Transplantation, Allogeneic Transplantation
- Trial name
- NCT02512679
- Testing
- Cyclophosphamide Dose Level 1, Cyclophosphamide Dose Level 2, Cyclophosphamide Dose Level 3
- Volunteers
- 20 people
- Sponsor
- Children's Hospital Los Angeles
Chronic Granulomatous Disease
Analysis of Patients Treated for Chronic Granulomatous Disease Since January 1, 1995
This study is a longitudinal and cross-sectional evaluation of patients with Chronic Granulomatous Disease (CGD) who received or are receiving hematopoietic cell transplantation (HCT) for their disease under a variety of…
- Trial name
- NCT02116764
- Where
- United States
- Volunteers
- 137 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Mycoses
Itraconazole for the Prevention of Fungal Infections in Chronic Granulomatous Disease
This protocol describes a prospective, randomized study examining the safety and efficacy of Itraconazole for preventing fungal infections in patients with Chronic Granulomatous Disease (CGD).
- Trial name
- NCT00001280
- Testing
- itraconazole
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)