Clinical trials: Chronic-granulomatous Disease
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
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
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
This is a data collection study that will examine the general diagnostic and treatment data associated with the reduced-intensity chemotherapy-based regimen paired with simple alemtuzumab dosing strata designed to prevented graft…
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias
- Trial name
- PRO-RIC (NCT04528355)
- Testing
- data collection
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Paul Szabolcs
Chronic Granulomatous Disease, Infection
NADPH Oxidase Correction in mRNA-transfected Granulocyte-enriched Cells in Chronic Granulomatous Disease (CGD)
Background: CGD is caused by a gene mutation. For people with CGD, their cells cannot kill germs well, so they can get frequent or life-threatening infections.
- Trial name
- NCT05189925
- Testing
- gp91 Grans
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Chronic Granulomatous Disease
Gene Therapy for Chronic Granulomatous Disease in Korea
The purpose of this study is to evaluate the safety and efficacy of administration of autologous hematopoietic stem cells transduced with MT-gp91 retroviral vector for patients with X-linked chronic granulomatous disease.
- Trial name
- NCT00778882
- Testing
- VM106
- Where
- South Korea
- Sponsor
- Helixmith Co., Ltd.
Hurler Syndrome, Fanconi Anemia
Bone Marrow Transplant With Abatacept for Non-Malignant Diseases
This is a single arm, phase I study to assess the tolerability of abatacept when combined with cyclosporine and mycophenolate mofetil as graft versus host disease prophylaxis in children undergoing unrelated hematopoietic stem…
- Conditions
- Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome
- Trial name
- NCT01917708
- Testing
- Abatacept
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Emory University
Crohn's Disease
An Open-Label, Proof of Consent Study of Vorinostat for the Treatment of Mdoerate-to-Severe Crohn s Disease and Maintenance Therapy With Ustekinumab
Background: Crohn s disease (CD) is an inflammatory bowel disease. It causes inflammation of the gut. Symptoms may include diarrhea, abdominal pain, fatigue, weight loss and malnutrition.
- Trial name
- NCT03167437
- Testing
- Vorinostat, Ustekinumab
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
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)
Hemophagocytic Lymphohistiocytosis, Chronic Active Epstein-Barr Virus Infection
Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204)
HLH, HLH-related disorders, Chronic Granulomatous (CGD), HIGM1, Immune dysregulation, polyendocrinopathy, enteropathy, and X-linked inheritance (IPEX) and severe LAD-I represent primary immune disorders that are typically fatal…
- Conditions
- Hemophagocytic Lymphohistiocytosis, Chronic Active Epstein-Barr Virus Infection, Chronic Granulomatous Disease, HIGM-1
- Trial name
- RICHI (NCT01998633)
- Testing
- Hematopoietic Stem Cell Transplant
- Where
- 22 sites in United States, Canada
- Volunteers
- 47 people
- Sponsor
- Medical College of Wisconsin
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
Granulomatous Disease, Chronic
Gene Therapy for Chronic Granulomatous Disease
The aim of the study is to evaluate the side effects and risks after infusion of retroviral gene corrected autologous CD34+ cells of the peripheral blood of chemotherapy conditioned (busulphan)patients with chronic granulomatous…
- Trial name
- CGD (NCT00564759)
- Testing
- retroviral SF71-gp91phox transduced CD34+ cells
- Where
- Germany
- Volunteers
- 2 people
- Sponsor
- Johann Wolfgang Goethe University Hospital
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
CGD
Immunological Profile and Clinical Characteristics of Children Diagnosed With Chronic Granulomatous Disease
Immunological profile and Clinical characteristics of children diagnosed with chronic granulomatous disease
- Trial name
- NCT05546775
- Testing
- DHR
- Volunteers
- 30 people
- Sponsor
- Assiut University
Chronic Granulomatous Disease
A Phase IV Study of Recombinant Human Gamma Interferon in Patients With Chronic Granulomatous Diseases of Childhood
In a recent double-blinded, placebo-controlled multi-center international study, subcutaneous injections of interferon-gamma administered three times weekly reduced the frequency of serious infections in patients with chronic…
- Trial name
- NCT00001317
- Testing
- interferon-gamma
- Where
- United States
- Volunteers
- 100 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)
p47, Autosomal Recessive
pCCLCHIM-p47 (Lentiviral Vector Transduced CD34 Plus Cells) in Patients With p47 Autosomal Recessive Chronic Granulomatous Disease (AR-CGD)
Background: Chronic granulomatous disease (CGD) is a genetic disorder. People with CGD are missing a gene that affects their white blood cells.
- Conditions
- p47, Autosomal Recessive, Chronic Granulomatous Disease
- Trial name
- NCT06253507
- Testing
- Cryopreserved Autologous CD34+ cells transduced with pCCLCHIM-p47
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Chronic Granulomatous Disease (CGD)
Part B- G1X-CGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease
Background: X-Linked Chronic Granulomatous Disease (X-CGD) is caused by a gene mutation that makes the immune system to not work properly.
- Trial name
- NCT07113743
- Testing
- Busulfan, Tocilizumab, Eltrombopag
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Atherosclerosis, Chronic Granulomatous Disease (CGD)
Non-Invasive Assessment of Atherosclerosis in Patients With CGD and Other Disorders of the Immune System
Background: Atherosclerosis, the arterial plaques or blockages that cause heart disease, develops in many people by the time they are in their mid-20s.
- Trial name
- NCT01063309
- Where
- United States
- Volunteers
- 156 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
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
Chediak Higashi Syndrome, Chronic Granulomatous Disease
Learning and Behavior Problems in Children With Chronic Granulomatous Disease and Related Disorders
This study will try to determine what causes learning, behavioral and emotional problems in children with chronic granulomatous disease (GCD) and other phagocyte disorders.
- Conditions
- Chediak Higashi Syndrome, Chronic Granulomatous Disease, Job's Syndrome, Leukocyte Disorder
- Trial name
- NCT00005933
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)