Clinical trials: Primary Immune Deficiency Disorders
Ataxia Telangiectasia
Biomarkers for Babies and Young Children With Ataxia Telangiectasia
The goal of this observational study is to identify progressive changes of quantitative brain and lung imaging, serum and movement-related biomarkers reflecting disease progression in pre-symptomatic infants and very young…
- Trial name
- BOBCAT (NCT07709728)
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- University of Nottingham
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study
This is a long-term follow up study evaluating the safety of BPX-501 T cells (rivogenlecleucel) and infused in pediatric patients previously enrolled on the BP-004 study.
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes
- Trial name
- NCT03733249
- Testing
- Rimiducid, rivogenlecleucel
- Where
- Italy
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Hereditary Angioedema (HAE)
Donidalorsen Treatment in Children With Hereditary Angioedema
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of donidalorsen in pediatric participants with hereditary angioedema (HAE) Type I (HAE-1) or Type II (HAE-2).
- Trial name
- NCT07298447
- Testing
- Donidalorsen
- Where
- 18 sites in United States, Australia and 3 more
- Volunteers
- 20 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
DiGeorge Syndrome, Complete Typical DiGeorge Anomaly
Thymus Transplantation in DiGeorge Syndrome #668
The study purpose is to determine whether cultured thymus tissue implantation (CTTI) is effective in treating typical complete DiGeorge syndrome.
- Trial name
- NCT00576407
- Testing
- Cultured Thymus Tissue for Implantation (CTTI)
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH
Primary Antibody Deficiency
A Multi-centre Open Study to Assess the Safety and Efficacy of Subgam®
The primary objective was to determine the efficacy of Human Normal Immunoglobulin (Subgam®) given subcutaneously by weekly infusion to patients with primary antibody deficiency.
- Trial name
- NCT02247141
- Testing
- Subgam® (Human Normal Immunoglobulin Solution)
- Where
- 14 sites in United Kingdom
- Sponsor
- Bio Products Laboratory
Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML)
CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant
The purpose of this study is to learn more about the effects of (classification determinant) CD34+ stem cell selection on graft versus host disease (GVHD) in children, adolescents, and young adults.
- Conditions
- Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome (MDS), Juvenile Myelomonocytic Leukemia (JMML)
- Trial name
- NCT02061800
- Testing
- CliniMACS CD34+ Reagent System, Thiotepa, Cyclophosphamide
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Diane George
HIV, Acquired Immunodeficiency Syndrome
Primary Care-Based Interventions to Reduce Alcohol Use Among HIV Patients
This randomized clinical trial uses a health plan's electronic medical record (EMR) alcohol screen; and examines innovative behavioral interventions, and their cost effectiveness, for hazardous drinking within a large HIV primary…
- Conditions
- HIV, Acquired Immunodeficiency Syndrome, Alcoholism, Alcoholic Intoxication
- Trial name
- NCT01671501
- Testing
- Motivational Interviewing, Email Feedback, Usual Care
- Where
- United States
- Volunteers
- 614 people
- Sponsor
- University of California, San Francisco
Acute Ischemic Stroke
Research Into the Effect of a Clot-dissolving Agent and Its Inhibitor
Single-chain urokinase-type plasminogen activator (pro-urokinase) is a highly effective thrombolytic drug. At pharmacologic concentrations however, pro-urokinase is converted to urokinase - a non specific thrombolytic, limiting…
- Trial name
- NCT01694381
- Testing
- M5, C1-inhibitor
- Where
- Netherlands
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- TSI, LLC
Hereditary Angioedema (HAE)
Long-Term, Open-label Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE
This is a Phase 3, multicenter, long-term, open-label study to evaluate the safety and efficacy of once-daily orally administered deucrictibant extended-release tablet for prophylaxis to prevent angioedema attacks in participants…
- Trial name
- CHAPTER-4 (NCT06679881)
- Testing
- Deucrictibant
- Where
- 67 sites in United States, Argentina and 21 more
- Volunteers
- 170 people
- Sponsor
- Pharvaris Netherlands B.V.
Bone Marrow Failure Syndrome, Severe Aplastic Anemia
CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation
This study's goal is to determine the frequency and severity of acute graft versus host disease, to evaluate incidence of primary and secondary graft rejection, to assess event free survival and overall survival, to determine the…
- Conditions
- Bone Marrow Failure Syndrome, Severe Aplastic Anemia, Severe Congenital Neutropenia, Amegakaryocytic Thrombocytopenia
- Trial name
- NCT01966367
- Testing
- CD34 Stem Cell Selection Therapy
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- Diane George
Primary Immune Deficiency
Antibody Production in Immune Disorders
This study will evaluate immune function in people with a known or suspected immune disorder. It will determine participants immune response to vaccines by measuring blood antibody levels after vaccination.
- Trial name
- NCT00023504
- Testing
- Pneumovax, Rabavert, Prevnar
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Primary Immunodeficiency Diseases (PIDD)
Expanded Access to TAK-881 for People With Primary Immunodeficiency Diseases in the USA and Czech Republic
The expanded access program allows people to gain access to an unlicensed treatment on compassionate grounds. This study will provide access to TAK-881 for participants with PIDD who have completed study TAK-881-3002…
- Trial name
- NCT06955793
- Testing
- TAK-881, SC Investigational Needle Sets
- Sponsor
- Takeda
Hereditary Angioedema (HAE)
Study of BCX7353 as a Treatment for Attacks of Hereditary Angioedema
This 3-part study will evaluate the efficacy and safety of an oral kallikrein inhibitor, BCX7353, in the treatment angioedema attacks in subjects with Type I or II hereditary angioedema (HAE).
- Trial name
- NCT03240133
- Testing
- BCX7353
- Where
- 24 sites in Austria, Denmark and 10 more
- Volunteers
- 58 people
- Sponsor
- BioCryst Pharmaceuticals
DOK 8, STAT1
Genetic Analysis of Immune Disorders
The purposes of this study are to 1) identify the genes responsible for certain immune disorders, 2) learn about the medical problems they cause, and 3) learn how to predict who is likely to develop these disorders and what the…
- Conditions
- DOK 8, STAT1, GATA2, Immunodeficiency
- Trial name
- NCT00001467
- Where
- United States
- Volunteers
- 5,000 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
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
Lymphoma, Non-Hodgkin, HIV Infections
The Use of Chemotherapy Plus Radiotherapy Plus Azidothymidine in Patients With AIDS-Related Lymph Node Cancer
To determine the safety and toxicity of high-dose systemic methotrexate (MTX) and dexamethasone (DEX) combined with zidovudine (AZT) and brain irradiation in patients with AIDS-related primary central nervous system (CNS)…
- Trial name
- NCT00000723
- Testing
- Methotrexate, Leucovorin calcium, Zidovudine
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Hereditary Angioedema
Hereditary Angioedema Kininogen Assay
A multicenter epidemiological observational study aiming to explore the cleaved high-molecular weight kininogen (cHMWK) including identification and characterization of other metabolite/biomarkers in HAE type 1/2 patients
- Trial name
- HAEKA (NCT04091113)
- Where
- 7 sites in Germany
- Volunteers
- 59 people
- Sponsor
- CENTOGENE GmbH Rostock
Septic Shock
Pro-inflammatory Role of Blood Platelets in Critically Ill Patients With Septic Shock.
Blood platelets play a major role in the inflammatory response. A dysregulation of platelets activation may be one of the contributors to tissue damage in critically ill patients with septic shock.
- Trial name
- PLAQSIS (NCT04080453)
- Testing
- Septic shock, Systemic Inflammatory Response Syndrome
- Where
- France
- Volunteers
- 202 people
- Sponsor
- University Hospital, Bordeaux
Epstein-Barr Virus (EBV) Infections, Lymphoproliferative Disorders
Expanded Access Protocol for Tabelecleucel for Patients With Epstein-Barr Virus-Associated Viremia or Malignancies
The primary objective of this protocol is to provide expanded access to tabelecleucel to participants with Epstein-Barr virus-associated diseases and malignancies for whom there are no other appropriate therapeutic options, and…
- Conditions
- Epstein-Barr Virus (EBV) Infections, Lymphoproliferative Disorders, EBV+ Associated Lymphoma, EBV+ Associated Post-transplant Lymphoproliferative Disease (EBV+ PTLD)
- Trial name
- NCT02822495
- Testing
- tabelecleucel
- Sponsor
- Atara Biotherapeutics
Haematological Malignancy, Acute Leukemia
Feasibility Study to Assess an Adapted Physical Activity Program in Children, Adolescents and Young Adults Requiring Hematopoietic Stem Cell Transplantation
Allogeneic haematopoietic stem cell transplantation (aHSCT) is the only curative treatment for many paediatric and young adult haematological pathologies (acute leukaemia, myelodysplastic syndromes haemoglobinopathies, bone…
- Conditions
- Haematological Malignancy, Acute Leukemia, Myelodysplastic Syndromes, Hemoglobinopathy in Children
- Trial name
- EVAADE (NCT05298930)
- Testing
- Physical activity program
- Where
- France
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- Centre Leon Berard