Clinical trials: Autoimmune Lymphoproliferative Syndrome, open to volunteers
- Condition Autoimmune Lymphoproliferative Syndrome
- Status Open to volunteers
- Age group Children (under 18)
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
Benign Lymphoproliferative Disorder
Study of Autoimmune Lymphoproliferative Syndrome (ALPS)
The purpose of the protocol is to allow for patients, and relatives of patients, who may have the newly described autoimmune lymphoproliferative syndrome, to be evaluated at the NIH Clinical Center.
- Trial name
- NCT00001350
- Where
- United States
- Volunteers
- 1,200 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Lymphoproliferative Disorders, Autoimmune Lymphoproliferative
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Inborn Errors of Immunity
Background: During a transplant, blood stem cells from one person are given to someone else. The cells grow into the different cells that make up the immune system. This can cure people with certain immunodeficiencies.
- Conditions
- Lymphoproliferative Disorders, Autoimmune Lymphoproliferative, Immune System Diseases, Common Variable Immunodeficiency
- Trial name
- NCT04339777
- Testing
- Busulfan test dose, Fludarabine, Busulfan
- Where
- United States
- Volunteers
- 66 people
- Sponsor
- National Cancer Institute (NCI)
Hereditary Autoinflammatory Diseases, Schnitzler Syndrome
AutoInflammatory Disease Alliance Registry (AIDA)
Autoinflammatory diseases (AID) are clinical entities characterized by recurrent inflammatory attacks in absence of infection, neoplasm or deregulation of the adaptive immune system.
- Conditions
- Hereditary Autoinflammatory Diseases, Schnitzler Syndrome, Behcet Syndrome, PFAPA Syndrome
- Trial name
- AIDA (NCT05200715)
- Testing
- No intervention is foreseen by the protocol.
- Where
- 112 sites in Algeria, Australia and 20 more
- Volunteers
- 3,500 people
- Sponsor
- University of Siena
Eosinophilic Granulomatosis With Polyangiitis (EGPA), Hypereosinophilia Syndrome (HES)
A Trial to Investigate Benralizumab in Children With Eosinophilic Diseases
The main purpose of study is to assess the safety, tolerability, pharmacokinetics (PK), and efficacy of benralizumab.
- Trial name
- CLIPS (NCT06512883)
- Testing
- Benralizumab
- Where
- 15 sites in United States, Brazil and 8 more
- Volunteers
- 14 people
- Sponsor
- AstraZeneca
Asthma, Eosinophilic Granulomatosis With Polyangiitis (EGPA)
Roll-over Study for Participants Who Have Completed a Previous Clinical Study With Benralizumab (Fasenra) and Benefit From Continued Treatment
The rationale of the roll-over study (ROSY) is to provide continuous access to study treatment for participants who have completed or exited a parent study and are deemed appropriate for continued benralizumab treatment, as…
- Conditions
- Asthma, Eosinophilic Granulomatosis With Polyangiitis (EGPA), Hypereosinophilic Syndrome (HES)
- Trial name
- ROSY-F (NCT07444567)
- Testing
- Benralizumab
- Where
- 47 sites in United States, Argentina and 12 more
- Volunteers
- 230 people
- Sponsor
- AstraZeneca
Cryoglobulinemic Vasculitis (CV), Drug-induced Vasculitis
Clinical Transcriptomics in Systemic Vasculitis (CUTIS)
Multi-center observational study to evaluate the histopathology and transcriptome of cutaneous lesions in patients with several different types of vasculitis.
- Conditions
- Cryoglobulinemic Vasculitis (CV), Drug-induced Vasculitis, Eosinophilic Granulomatosis With Polyangiitis (EGPA), IgA Vasculitis
- Trial name
- CUTIS (NCT03004326)
- Where
- 10 sites in United States, Canada
- Volunteers
- 50 people
- Sponsor
- Peter Merkel
Primary T-cell Immunodeficiency Disorders, Common Variable Immunodeficiency
Pilot Trial of Allogeneic Blood or Marrow Transplantation for Primary Immunodeficiencies
Background: Allogeneic blood or marrow transplant is when stem cells are taken from one person s blood or bone marrow and given to another person. Researchers think this may help people with immune system problems.
- Conditions
- Primary T-cell Immunodeficiency Disorders, Common Variable Immunodeficiency, Immune System Diseases, Autoimmune Lymphoproliferative
- Trial name
- NCT02579967
- Testing
- Immunosuppression Only Conditioning -Closed with amendment L, Reduced Intensity Conditioning, Myeloablative Conditioning-Closed with amendment L
- Where
- 2 sites in United States
- Volunteers
- 354 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Adenine Phosphoribosyltransferase Deficiency, AH Amyloidosis
National Registry of Rare Kidney Diseases
The goal of this National Registry is to is to collect information from patients with rare kidney diseases, so that it that can be used for research.
- Conditions
- Adenine Phosphoribosyltransferase Deficiency, AH Amyloidosis, AHL Amyloidosis, AL Amyloidosis
- Trial name
- RaDaR (NCT06065852)
- Where
- United Kingdom
- Volunteers
- 35,000 people
- Sponsor
- UK Kidney Association
Aortitis, Cutaneous Vasculitis
VCRC Tissue Repository
The purpose of this study is to collect existing tissue specimens from subjects enrolled in Vasculitis Clinical Research Consortium (VCRC) studies.
- Conditions
- Aortitis, Cutaneous Vasculitis, Eosinophilic Granulomatosis With Polyangiitis, Giant Cell Arteritis
- Trial name
- NCT02967068
- Where
- 8 sites in United States, Canada
- Volunteers
- 1,000 people
- Sponsor
- University of Pennsylvania
Eosinophilic Granulomatosis With Polyangiitis, Churg-Strauss Syndrome
Longitudinal Study for Eosinophilic Granulomatosis With Polyangiitis
Eosinophilic granulomatosis with polyangiitis (EGPA) is a rare immune system disorder that causes asthma, an excessive number of eosinophils (a type of white blood cell) in the blood, and the inflammation of blood vessels, or…
- Trial name
- NCT00315380
- Where
- 15 sites in United States, Canada and 3 more
- Volunteers
- 700 people
- Sponsor
- University of Pennsylvania
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
Multiple Myeloma, Acute Myeloid Leukemia
National Longitudinal Cohort of Hematological Diseases
Background Hematological diseases are disorders of the blood and hematopoietic organs. The current hematological cohorts are mostly based on single-center or multi-center cases, or cohorts with limited sample size in China.
- Conditions
- Multiple Myeloma, Acute Myeloid Leukemia, Hemophilia, Hemophilia A
- Trial name
- NICHE (NCT04645199)
- Where
- China
- Volunteers
- 2,300 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Rare Diseases, Amyloidosis
Institutional Registry of Rare Diseases
The goal of this observational study is to create a single macro registry system with data collection on common clinical features, grouping the different rare diseases (RD).
- Conditions
- Rare Diseases, Amyloidosis, Sarcoidosis, Phacomatosis
- Trial name
- NCT06573723
- Where
- Argentina
- Volunteers
- 380 people
- Sponsor
- Hospital Italiano de Buenos Aires
Kidney Transplant, Familial Hypercholesterolaemia
Cardiovascular Risk in Children With Chronic Conditions Study
Children living with chronic health conditions face a higher risk of developing cardiovascular diseases than their peers, largely due to the accelerated aging of the heart and blood vessels.
- Conditions
- Kidney Transplant, Familial Hypercholesterolaemia, Type 1 Diabetes Mellitus (T1DM), Type 2 Diabetes Mellitus (T2DM)
- Trial name
- CR3C (NCT07086989)
- Where
- 3 sites in Hungary
- Volunteers
- 300 people
- Sponsor
- Semmelweis University
Sjogren's Syndrome, Pediatric ALL
Belimumab After Rituximab in Resistant Primary Juvenile SS
The goal of this clinical trial is to explore the effectiveness of sequential use of rituximab and belimumab in the treatment of resistant primary juvenile Sjogren's syndrome.
- Trial name
- BRRPJS (NCT06410833)
- Testing
- Rituximab, Belimumab
- Where
- China
- Volunteers
- 15 people
- Sponsor
- Peking Union Medical College Hospital
Anterior Uveitis (AU), Birdshot Chorioretinitis
Quantification & Classification of Inflammatory Cells in Uveitis Using OCT
The goal of this study is to determine if it's possible to use a high resolution imaging device called optical coherence tomography (OCT) to develop an unbiased, standard method of counting and categorizing the various types of…
- Conditions
- Anterior Uveitis (AU), Birdshot Chorioretinitis, Behcet Disease, Herpes Simplex Virus
- Trial name
- NCT07065747
- Where
- United States
- Volunteers
- 125 people, healthy volunteers welcome
- Sponsor
- Oregon Health and Science University
Eosinophilic Granulomatosis With Polyangiitis (Churg-Strauss), Giant Cell Arteritis
One-Time DNA Study for Vasculitis
The purpose of this study is to identify genes that increase the risk of developing vasculitis, a group of severe diseases that feature inflammation of blood vessels.
- Conditions
- Eosinophilic Granulomatosis With Polyangiitis (Churg-Strauss), Giant Cell Arteritis, Granulomatosis With Polyangiitis (Wegener's), Microscopic Polyangiitis
- Trial name
- NCT01241305
- Where
- 14 sites in United States, Canada and 1 more
- Volunteers
- 1,000 people
- Sponsor
- University of Pennsylvania
Hemophagocytic Lymphohistiocytosis (HLH), Macrophage Activation Syndrome (MAS)
Long Term Safety Study in Patients With HLH/MAS in Still's Disease Exposed to Gamifant.
This study aims to evaluate safety information in patients with HLH/MAS in Still's disease exposed to Gamifant.
- Conditions
- Hemophagocytic Lymphohistiocytosis (HLH), Macrophage Activation Syndrome (MAS), Still Disease
- Trial name
- NCT07909005
- Testing
- Local laboratory testing, Local Lab Tests
- Where
- 18 sites in United States
- Volunteers
- 70 people
- Sponsor
- Swedish Orphan Biovitrum
Autoimmune Lymphoproliferative Syndrome
Study of the ITK Inhibitor Soquelitinib to Reduce Lymphoproliferation and Improve Cytopenias in Autoimmune Lymphoproliferative Syndrome (ALPS)-FAS Patients
Background: Autoimmune lymphoproliferative syndrome (ALPS) is a rare disorder of the immune system caused by a mutation in the FAS gene. In ALPS, the body stores too many germ-fighting cells called lymphocytes.
- Trial name
- NCT06730126
- Testing
- Soquelitinib
- Where
- 3 sites in United States
- Volunteers
- 15 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)