Clinical trials: Immune Deficiency Disease
Pyruvate Kinase Deficiency, Pyruvate Kinase Deficiency Anemia
Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms
This cross-sectional prevalence assessment study involves a single blood draw in specific patient populations to assess for enzymatic and genomic evidence for acquired pyruvate kinase deficiency.
- Conditions
- Pyruvate Kinase Deficiency, Pyruvate Kinase Deficiency Anemia, Hereditary Hemolytic Anemia, Myelodysplastic Syndromes
- Trial name
- NCT04902833
- Testing
- Blood Draw
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Massachusetts General Hospital
Common Variable Immunodeficiency (CVID), Primary Immune Regulatory Disorder
Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD)
This is a research protocol that will examine Hematopoietic Stem Cell Transplantation (HSCT) using a reduced conditioning regimen (RIC) with total body Irradiation (TBI) in those diagnosed with Common Variable Immunodeficiency…
- Conditions
- Common Variable Immunodeficiency (CVID), Primary Immune Regulatory Disorder, Immune Dysregulation, DiGeorge Syndrome
- Trial name
- CVID/PIRD (NCT07284641)
- Testing
- Hematopoietic stem cell transplant (HSCT)
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Paul Szabolcs
Immunologic Deficiency Syndromes, Primary Immune Deficiency (PID)
Genetic Study of Immunodeficiency: Search for New Genetic Causes for Primary Immunodeficiencies
Individuals with suspected primary immunodeficiency will be studied and the results compared with healthy controls. Primary immunodeficiency may manifest as recurrent, severe or unusual infections as well as signs and symptoms of…
- Trial name
- GSI (NCT02735824)
- Testing
- Skin Biopsy, Mouth Swab or Saliva Collection, Blood Sampling
- Where
- Switzerland
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- University Children's Hospital, Zurich
Primary Immune Deficiency Disorder, Secondary Immune Deficiency
Observational Study of Subcutaneous Immunoglobulin (Cutaquig) in Patients With Primary and Secondary Immunodeficiency.
This study will assess the relative safety, tolerability, and participant satisfaction in participants using the rapid manual push method with Cutaquig®.
- Trial name
- NCT04354129
- Testing
- Cutaquig®
- Where
- Canada
- Volunteers
- 36 people
- Sponsor
- University of Alberta
Human Immunodeficiency Virus, Alcohol-Related Disorders
Project Khanya: Peer-Delivered Intervention to Improve HIV Medication Adherence and Substance Use in South Africa
The purpose of this study is to evaluate a stepped care behavioral intervention for HIV medication adherence and substance use ("Khanya") integrated into an HIV primary care setting in South Africa.
- Conditions
- Human Immunodeficiency Virus, Alcohol-Related Disorders, Drug Use, Substance Use
- Trial name
- NCT05933226
- Testing
- Khanya
- Where
- South Africa
- Volunteers
- 160 people
- Sponsor
- University of Maryland, College Park
Primary Immune Deficiency Disorder
Identification of New Inborn Errors of Immunity
Analysis of DNA samples of patients with molecularly undetermined PID by whole exome/genome sequencing. Transcriptome analysis of patients with molecularly undetermined PID.
- Trial name
- NCT03414528
- Testing
- Diagnostic Test
- Where
- Switzerland
- Volunteers
- 9 people, healthy volunteers welcome
- Sponsor
- University of Zurich
Primary Immune Deficiency Disorder
Pathophysiology of Inborn Immunodeficiencies
The pathophysiology of primary immunodeficiencies (PID), which encompass a broad range of different diseases with susceptibility to infection and/or a deregulated inflammatory response, is poorly understood.
- Trial name
- NCT03422614
- Testing
- Diagnostic Test
- Where
- Switzerland
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- University of Zurich
Viral Infection, Primary Immune Deficiency Disorder
Treatment of Refractory BK Infections With Related Donor BK Specific Cytotoxic T-cells (CTLs)
BK cytotoxic T cells (CTLs) manufactured with the Miltenyi CliniMACS Prodigy Cytokine Capture System will be safe and effective in decreasing specific viral load in children, adolescents and young adults (CAYA) with refractory BK…
- Trial name
- NCT04197596
- Testing
- BK CTL
- Where
- 7 sites in United States
- Volunteers
- 40 people
- Sponsor
- New York Medical College
Primary Immune Deficiencies, Benign Lymphoproliferative Disorder
Longitudinal Prognosis of Lymphoproliferative Manifestations in Primary Immunodeficiencies
Primary immunodeficiencies (PIDs) are a heterogeneous group of inborn errors of immunity characterized not only by increased susceptibility to infections but also by immune dysregulation.
- Trial name
- PID-LP (NCT07526714)
- Volunteers
- 60 people
- Sponsor
- Central Hospital, Nancy, France
Primary Immune Deficiency
Primitive Immunodeficiency and Pregnancy
The management of patients with primary immune deficiency is increasingly codified, however contraception and pregnancy have not yet extensively studied or codified, and the medical monitoring and the prevention of infectious…
- Trial name
- PREPI (NCT04581460)
- Testing
- Questionnaire
- Where
- France
- Volunteers
- 99 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Immunological Disease, Autoimmune Diseases
Biologics And Clinical Immunology Cohort at Sinai
The study team plans to establish a bioregistry of patients receiving biologic therapy as part of their standard treatment at the Mount Sinai Therapeutic Infusion Center and affiliated practices.
- Conditions
- Immunological Disease, Autoimmune Diseases, Primary Immune Deficiency
- Trial name
- BACICS (NCT04784364)
- Where
- United States
- Volunteers
- 800 people
- Sponsor
- Icahn School of Medicine at Mount Sinai
Obesity, Immune Deficiency
Immunoglobulin G Therapy Dose Optimization
The overall goal of this proposal is to investigate effects of obesity on pharmacokinetics of immunoglobulin G (IgG) and to develop strategies for optimization of dosing of IgG in obese patients.
- Trial name
- NCT04818177
- Testing
- Institutional standard intravenous immune globulin treatment
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Rutgers, The State University of New Jersey
Immune Deficiency, Immune Dysregulation Disorder
Allergy and Immunology Natural History Study
This protocol is a natural history study designed to evaluate subjects (and some family members) with suspected or identified genetic diseases of allergic inflammation or Immune Dysregulation.
- Conditions
- Immune Deficiency, Immune Dysregulation Disorder, Allergic Inflammation
- Trial name
- NCT05584488
- Where
- United States
- Volunteers
- 10,000 people, healthy volunteers welcome
- Sponsor
- Columbia University
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
Primary Immune-Deficiency (PID) Common Variable Immune Deficiency (CVID)
Severe PID With Lymphoproliferation and Neutropenia
The purpose of this study is to analyse the phenotype in a sub-population of adults with severe primary immunodeficiency with lymphoproliferation and neutropenia and to decipher the possible pathways involved, especially under…
- Trial name
- DICEP (NCT03427593)
- Testing
- FACS analyses, Target Sequencing by NGS ( Next-generation sequencing), Whole Exome Sequencing
- Where
- France
- Volunteers
- 27 people, healthy volunteers welcome
- Sponsor
- University Hospital, Strasbourg, France
Severe Combined Immunodeficiency (SCID), Immunodeficiency With Predominant T-cell Defect, Unspecified
Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases
The purpose of this study is to determine whether bilateral orthotopic lung transplantation (BOLT) followed by cadaveric partially-matched hematopoietic stem cell transplantation (HSCT) is safe and effective for patients aged…
- Conditions
- Severe Combined Immunodeficiency (SCID), Immunodeficiency With Predominant T-cell Defect, Unspecified, Severe Chronic Neutropenia, Chronic Granulomatous Disease (CGD)
- Trial name
- BOLT+BMT (NCT01852370)
- Testing
- CD3/CD19 negative allogeneic hematopoietic stem cells
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Paul Szabolcs
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
Severe Combined Immune Deficiency (SCID)
Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus
Background: Severe combined immune deficiency (SCID) is a group of conditions where the immune system does not work properly. The only cure for most SCIDs is a stem cell transplant (getting cells from a donor).
- Trial name
- NCT04370795
- Testing
- Sirolimus, Busulfan, Horse -Anti-thymocyte
- Where
- United States
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Primary Immune Deficiency Disorder, Immune Deficiency Disease
Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures
Phase II prospective trial to assess the rates of donor engraftment using reduced intensity conditioning (RIC) hematopoietic stem cell transplant (HSCT) and post-transplant cyclophosphamide (PTCy) for patients with primary immune…
- Conditions
- Primary Immune Deficiency Disorder, Immune Deficiency Disease, Bone Marrow Failure, Short Telomere Length
- Trial name
- NCT04232085
- Testing
- Alemtuzumab, Fludarabine, Melphalan
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Hematologic Malignancies, Inborn Errors of Metabolism Disorders
Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood Transplants
The objective of this study is to make T-cell depleted stem cells from a family member who is a half match (haplo-identical) available on an expanded access basis to patients receiving one or two unrelated cord blood transplants…
- Conditions
- Hematologic Malignancies, Inborn Errors of Metabolism Disorders, Immune Deficiencies
- Trial name
- NCT01881334
- Testing
- CliniMACS CD34 Reagent System
- Where
- United States
- Sponsor
- Joanne Kurtzberg, MD