Clinical trials: Primary Immune Deficiency Disorder
HIV Infections, Yellow Fever
Immunity to Yellow Fever in HIV-infected Patients 10 Years After a Primary Anti-yellow Fever Vaccination
ANRS 0146s NovaaTen study aims to determine the vaccine responses in the participants of the ANRS EP46 Novaa trial 10 years after a primary anti-yellow fever vaccination
- Trial name
- NCT05450770
- Testing
- Yellow fever vaccination (STAMARIL)
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- ANRS, Emerging Infectious Diseases
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
Hereditary Angioedema
Assessment of the State of Health, Quality of Life and Expectations of Patients With Hereditary Angioedema
8 years after the establishment of the therapeutic education program and 14 years after the creation of the National Reference Center for Angioedemas (CREAK), it is necessary to make an inventory in 2021 on the disease…
- Trial name
- BESQAOH (NCT04888650)
- Testing
- questionnaire
- Where
- France
- Volunteers
- 314 people
- Sponsor
- University Hospital, Grenoble
Pneumonia, Pneumocystis Carinii, HIV Infections
A Study of Three Drugs Plus Zidovudine in the Prevention of Infections in HIV-Infected Patients
To evaluate and compare 3 anti-pneumocystis regimens plus zidovudine (AZT) in persons with HIV infection and T4 cell count less than 200 cells/mm3.
- Trial name
- NCT00000991
- Testing
- Pentamidine isethionate, Sulfamethoxazole-Trimethoprim, Dapsone
- Where
- 21 sites in United States, Tanzania
- Volunteers
- 600 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Severe Combined Immunodeficiency Syndrome
Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency
This study will evaluate a new method for delivering gene transfer therapy to patients with severe combined immunodeficiency disease (SCID) due to a defective adenosine deaminase (ADA) gene.
- Trial name
- NCT00018018
- Testing
- CD34+ cells transduced with ADA retrovir
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
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 Ciliary Dyskinesia, Primary Immune Deficiency
Defining the Genetic Etiology of Suppurative Lung Disease in Children and Adults
The investigators will utilize a systematic approach for the diagnostic evaluation of patients to identify characteristics which may distinguish between Primary Immunodeficiency (PID) disorders versus Primary Ciliary Dyskinesia…
- Conditions
- Primary Ciliary Dyskinesia, Primary Immune Deficiency, Kartagener Syndrome
- Trial name
- NCT04702243
- Testing
- Genetic Testing for PCD or PID, Unaffected Family Member Genetic Testing
- Where
- 8 sites in United States, Canada
- Volunteers
- 436 people
- Sponsor
- University of North Carolina, Chapel Hill
Lymphoma, Non-Hodgkin, HIV Infections
Phase II Trial of Sequential Chemotherapy and Radiotherapy for AIDS-Related Primary Central Nervous System Lymphoma
To estimate the response rate, overall and disease-free survival, toxicities, factors associated with outcome, and effect on quality of life in patients with AIDS-related primary CNS lymphoma treated with CHOD (cyclophosphamide…
- Trial name
- NCT00000801
- Testing
- Filgrastim, Vincristine sulfate, Doxorubicin hydrochloride
- Where
- 7 sites in United States
- Volunteers
- 33 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Wiskott-Aldrich Syndrome
Romiplostim Treatment for Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome.
The rationale for this retrospective study is to evaluate the efficacy and safety of thrombopoietin-receptor agonist (TPO-RA) romiplostim for reducing thrombocytopenia and bleeding tendency in pediatric participants with…
- Trial name
- NCT04350164
- Testing
- Romiplostim
- Where
- Russia
- Volunteers
- 67 people
- Sponsor
- Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Primary Immunodeficiency, DOCK8
Establishing Fibroblast-Derived Cell Lines From Skin Biopsies of Patients With Immunodeficiency or Immunodysregulation Disorders
Background: National Institutes of Health (NIH) researchers have been studying immune cells (white blood cells) to better understand how the human body s defense system works and adjusts or regulates itself, and how changes in…
- Conditions
- Primary Immunodeficiency, DOCK8, Virus Susceptibility
- Trial name
- NCT00895271
- Where
- United States
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Severe Combined Immunodeficiency, Neonatal Screening
Neonatal Screening of Severe Combined Immunodeficiencies
The goal of the proposed research is to observe the prevalence and establish the validity of a newborn screening method for severe combined immunodeficiency (SCID).
- Trial name
- NCT02590328
- Where
- China
- Volunteers
- 180,000 people
- Sponsor
- Children's Hospital of Fudan University
JAK1 GOF, STAT1 GOF
Upadacitinib in Treatment of JAK/STAT Pathway Disorders With Activating Mutations
This study focuses on a genetic condition that affects the Janus Kinase-Signal Transducer and Activator of Transcription (JAK-STAT) immune signaling pathway.
- Conditions
- JAK1 GOF, STAT1 GOF, STAT3 GOF, STAT5B GOF
- Trial name
- SAFE-JAKi (NCT07670156)
- Testing
- Upadacitinib
- Where
- 3 sites in United States
- Volunteers
- 30 people
- Sponsor
- Lisa Satter
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
HIV/AIDS and Infections, Drug Abuse
A Community-University Approach to Preventing HIV
Examine the feasibility and acceptability of delivering the S4E intervention to 20 African-American youths between the ages 16-21 in Flint, Michigan.
- Conditions
- HIV/AIDS and Infections, Drug Abuse, Adolescent Behavior, Drug Usage
- Trial name
- NCT03938077
- Testing
- Storytelling 4 Empowerment
- Where
- 2 sites in United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- University of Michigan
Hereditary Angioedema
Safety and Efficacy of Oral BCX4161 as a Prophylactic Treatment for HAE
The purpose of this study is to determine whether BCX4161 given as a daily oral prophylactic treatment is safe and effective in reducing the number of acute attacks in patients with hereditary angioedema.
- Trial name
- OPuS-1 (NCT01984788)
- Testing
- BCX4161
- Where
- 6 sites in Germany, United Kingdom
- Volunteers
- 24 people
- Sponsor
- BioCryst Pharmaceuticals
Primary Immunodeficiency Diseases (PID)
A Study About the Long-Term Safety of TAK-881 in People With Primary Immunodeficiency Diseases
The main aim of this study is to learn if TAK-881 is safe and well tolerated during long-term use in adults and children with PIDD who are eligible and completed study TAK-881-3001 (NCT05755035).
- Trial name
- NCT06076642
- Testing
- TAK-881, Koru 24 G HIgH Flo Subcutaneous Safety Needle Set (investigational in the US)
- Where
- 25 sites in United States, Czechia and 5 more
- Volunteers
- 53 people
- Sponsor
- Takeda
Primary Immune Deficiency Disorder, Hematopoietic Stem Cell Transplantation
TCR Alpha/Beta Depletion for HSCT From Haploidentical and Unrelated Donors in the Treatment of PID
Treatment Study to assess of safety and efficiency of T cells receptor (TCR) alfa beta depleted graft for hematopoietic stem cell transplantation (HSCT) from haploidentical and unrelated donors in patients with primary…
- Trial name
- NCT02327351
- Testing
- Biological: TCR alfa beta T cell depletion
- Where
- Russia
- Volunteers
- 98 people
- Sponsor
- Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Primary Immunodeficiency Disease
Study to Investigate Efficacy, Safety and Pharmacokinetics of BT595 in Subjects With PID
This Phase III clinical study is to test efficacy, safety and pharmacokinetics of BT595 in treating patients with Primary Immunodeficiency (PID)
- Trial name
- NCT02810444
- Testing
- IgG Next Generation (BT595)
- Where
- 19 sites in United States, Germany and 3 more
- Volunteers
- 81 people
- Sponsor
- Biotest
Hereditary Angioedema, HAE
Oral Berotralstat Expanded Access Program
This expanded access program will provide access to berotralstat for eligible participants with hereditary angioedema in the U.S.
- Conditions
- Hereditary Angioedema, HAE, Prophylaxis
- Trial name
- NCT04428632
- Testing
- Berotralstat
- Sponsor
- BioCryst Pharmaceuticals
Immunoproliferative Disorder
Study of Immune Responses and Safety of Recombinant Human CD40 Ligand in Patients With X-Linked Hyper-IgM Syndrome
The primary goal of this Phase I/II study is to assess the immune response and safety of recombinant human CD40 ligand (rhuCD40L) in patients with X-linked hyper IgM syndrome (XHIM).
- Trial name
- NCT00001145
- Testing
- Bacteriophage, rhuCD40L, KLH
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)