Clinical trials: Leukocyte Adhesion Deficiency 1 (LAD)
Breast Cancer, Radiation-Induced Vascular Disease
4D CT Scan Versus 3D CT Scan Concerning Cardiac Dosimetry Assesment for Left Sided Breast Cancers Radiotherapy
To establish if the cardiac radiation dose assesment is well aproximated with routine 3D CT scan compared to 4D CT experimental scan with respiratory gating (breath motion monitoring).
- Conditions
- Breast Cancer, Radiation-Induced Vascular Disease, Left Anterior Descending Coronary Artery Stenosis, Radiotherapy Side Effect
- Trial name
- RD3D4 (NCT05726604)
- Testing
- Respiratory gating
- Where
- France
- Volunteers
- 15 people
- Sponsor
- Central Hospital Saint Quentin
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
Leukocyte Adhesion Deficiency
Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I)
This Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I) is a continuation of a Phase 1/2 clinical study to evaluate the safety and efficacy of the infusion of autologous hematopoietic stem…
- Trial name
- NCT06282432
- Where
- 3 sites in United States, Spain and 1 more
- Volunteers
- 9 people
- Sponsor
- Rocket Pharmaceuticals Inc.
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
LAD1
Ustekinumab (Anti-IL-12/23p40 Monoclonal Antibody) in Patients With Leukocyte Adhesion Deficiency Type 1 (LAD1) Who Have Inflammatory Pathology
Background: The disease leukocyte adhesion deficiency type 1 (LAD1) affects white blood cells. Those are immune system cells. In people with LAD1, white blood cells do not properly communicate with the rest of the body.
- Trial name
- NCT03366142
- Testing
- Ustekinumab
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Leukocyte Adhesion Deficiency
A Study to Evaluate Efficacy and Safety of AVTX-803 in Patients With Leukocyte Adhesion Deficiency Type II
The primary objective of this study is to evaluate the efficacy and safety of AVTX-803 compared to withdrawal in patients with Leukocyte Adhesion Deficiency, Type II (LAD II).
- Trial name
- NCT05462587
- Testing
- AVTX-803 (L-Fucose)
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- AUG Therapeutics
LAD (Left Anterior Descending) Coronary Artery Stenosis
Reconstruction of Diffusely Diseased Left Anterior Descending with Left Internal Mammary Artery On-lay Patch or Saphenous Vein Patch Without Endarterectomy by Opening the Whole Wall of the Diseased Segment(s) Has Less Risk and Fewer Complications Compared to Endarterectomy Technique
The objective of this study is to compare different surgical strategies for management of severely diseased LAD artery during CABG i.e. LIMA on-lay patch vs.
- Trial name
- NCT06835114
- Testing
- CABG, LIMA on lay patch, Safenous vein patch
- Volunteers
- 90 people
- Sponsor
- Assiut University
Chronic Granulomatous Disease (CGD), X-Linked Severe Combined Immune Deficiency (XSCID)
Evaluation of Patients With Immune Function Abnormalities
This study will evaluate patients with abnormal immune function that results in recurrent or unusual infections or chronic inflammation.
- Conditions
- Chronic Granulomatous Disease (CGD), X-Linked Severe Combined Immune Deficiency (XSCID), Leukocyte Adhesion Deficiency 1 (LAD), Graft Versus Host Disease (cGvHD)
- Trial name
- NCT00128973
- Where
- United States
- Volunteers
- 3,500 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Leukocyte Adhesion Deficiency Syndrome
Gene Therapy for Patients With Leukocyte Adherence Deficiency (Follow-Up of Phase 1 Trial)
This study will provide long-term monitoring of two patients who received gene therapy for leukocyte adherence deficiency (LAD) under the Food and Drug Administration investigational new drug study BB-IND-7949.
- Trial name
- NCT00023010
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- National Cancer Institute (NCI)
Sickle Cell Disease, Thalassemia
Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells
This is a clinical trial of bone marrow transplantation for patients with the diagnosis of a genetic disease of blood cells that do not have an HLA-matched sibling donor.
- Conditions
- Sickle Cell Disease, Thalassemia, Anemia, Granuloma
- Trial name
- NCT00730314
- Testing
- Hematopoietic stem cell transplantation
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Children's Hospital Los Angeles
X-Linked Agammaglobulinemia, X-Linked Hyper IgM Syndrome
Study of Genetic and Molecular Defects in Primary Immunodeficiency Disorders
OBJECTIVES: I. Identify the molecular defects responsible for primary immunodeficiency disorders. II. Explore the mutations within each syndrome to better understand the genetics of these disorders. III.
- Conditions
- X-Linked Agammaglobulinemia, X-Linked Hyper IgM Syndrome, Wiskott-Aldrich Syndrome, Leukocyte Adhesion Deficiency Syndrome
- Trial name
- NCT00004341
- Where
- United States
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Leukocyte Adhesion Defect - Type I
A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I
The primary purpose of the Phase I portion of the study is to assess the therapeutic safety and preliminary efficacy of a hematopoietic cell-based gene therapy consisting of autologous CD34+ enriched cells transduced with the…
- Trial name
- NCT03812263
- Testing
- RP-L201
- Where
- 3 sites in United States, Spain and 1 more
- Volunteers
- 9 people
- Sponsor
- Rocket Pharmaceuticals Inc.
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
Leukocyte Adhesion Defect - Type I
A Gene Therapy Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I
The primary purpose of the Phase I portion of the study is to determine the safety profile and preliminary evidence of efficacy associated with infusion of autologous gene-corrected hematopoietic stem cells.
- Trial name
- NCT03825783
- Testing
- RP-L201
- Where
- Spain
- Sponsor
- Rocket Pharmaceuticals Inc.
Immunologic Deficiency Syndromes, Chediak-Higashi Syndrome
Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies
OBJECTIVES: I. Provide curative immunoreconstituting allogeneic bone marrow transplantation for patients with primary immunodeficiencies. II.
- Conditions
- Immunologic Deficiency Syndromes, Chediak-Higashi Syndrome, Common Variable Immunodeficiency, Graft Versus Host Disease
- Trial name
- NCT00006054
- Testing
- anti-thymocyte globulin, busulfan, cyclophosphamide
- Where
- United States
- Sponsor
- Fairview University Medical Center
Leukocyte Adhesion Deficiency Syndrome
Interferon Gamma to Treat Leukocyte Adhesion Deficiency Type I
This study will evaluate the safety and effectiveness of the drug, interferon gamma, in treating leukocyte adhesion deficiency type I (LAD I).
- Trial name
- NCT00001905
- Testing
- Interferon gamma
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant…
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias
- Trial name
- HSCT+RIC (NCT01962415)
- Testing
- Hydroxyurea, Alemtuzumab, Fludarabine
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Paul Szabolcs
Coronary Arterial Disease (CAD), Coronary Artery Bypass
Comparison of Bidirectional Palpation Test and Transit Time Flow Measurement for LIMA-LAD Graft Patency
The most important factor determining mortality and morbidity after coronary artery bypass graft (CABG) surgery is graft patency.
- Conditions
- Coronary Arterial Disease (CAD), Coronary Artery Bypass, LIMA, Graft Patency
- Trial name
- NCT06934993
- Where
- Turkey (Türkiye)
- Volunteers
- 116 people
- Sponsor
- Abdulgani Orhun YENİGÜN
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
Leukocyte-Adhesion Deficiency Syndrome
Stem Cell Transplantation to Treat Leukocyte Adhesion Deficiency
This study will investigate the safety and effectiveness of a modified stem cell transplant procedure for treating leukocyte adhesion deficiency (LAD).
- Trial name
- NCT00031005
- Testing
- modified stem cell transplant
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)