Clinical trials: Primary Immune Deficiency Disorders
Hereditary Angioedema (HAE)
Study of C1 Inhibitor (Human) for the Prevention of Angioedema Attacks and Treatment of Breakthrough Attacks in Japanese Subjects With Hereditary Angioedema (HAE)
The purpose of this study is to determine if an investigational treatment is safe and well tolerated when administered by intravenous (IV) infusion in Japanese subjects with HAE.
- Trial name
- NCT02865720
- Testing
- CINRYZE 500 U, CINRYZE 1000 U
- Where
- 10 sites in Japan
- Volunteers
- 8 people
- Sponsor
- Shire
HIV Infections, Substance Use
Reducing HIV: Safer Sex Skill Building in Pregnant Drug Abusing Women
This study will examine safer sex skills building (SSB), a targeted behavioral HIV prevention and risk reduction group intervention in two samples of pregnant drug abusing women.
- Trial name
- NCT00619320
- Testing
- Safer Sex Skills Building (SSB)
- Where
- 2 sites in United States
- Volunteers
- 380 people, healthy volunteers welcome
- Sponsor
- Virginia Commonwealth University
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
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
Primary Immunodeficiency
Quality of Life Among Children With Inborn Error of Immunity
In this study, we aim to assess the QOL of pediatric IEI patients compared with healthy controls and to determine different factors affecting QOL in these patients.
- Trial name
- NCT07262047
- Testing
- Quality of Life Assessment
- Volunteers
- 50 people
- Sponsor
- Sohag University
Opioid-Related Disorders, HIV Infections
Buprenorphine and Integrated HIV Care Evaluation
The purpose of this study is to assess the feasibility, cost and effectiveness of interventions designed to integrate buprenorphine treatment for opioid dependence into HIV primary care in ten HIV care centers in the U.S.
- Conditions
- Opioid-Related Disorders, HIV Infections, AIDS
- Trial name
- NCT00124358
- Testing
- Buprenorphine, Integrated HIV care and office-based opioid dependence treatment
- Where
- 10 sites in United States
- Volunteers
- 1,350 people
- Sponsor
- The New York Academy of Medicine
Adenosine Deaminase Deficiency, Severe Combined Immunodeficiencies (SCID)
Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency
This is a historically controlled, non-randomized Phase I/II clinical trial to assess the safety and efficacy of autologous transplantation of CD34+ hematopoietic stem/progenitor cells (HSPCs), obtained from infants affected by…
- Trial name
- NCT01380990
- Testing
- Infusion of autologous EFS-ADA LV CD34+ cells, Haematopoietic Stem Cell Transplantation (HSCT), Busulfan
- Where
- United Kingdom
- Volunteers
- 36 people
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
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
AIDS-Related-Primary Central Nervous System Lymphoma
Chemotherapy and HAART to Treat AIDS-related Primary Brain Lymphoma
This study will investigate the use of chemotherapy plus highly active antiretroviral therapy (HAART) in patients with Acquired Immunodeficiency Syndrome (AIDS)-related primary brain lymphoma.
- Trial name
- NCT00267865
- Testing
- Methotrexate, Rituximab, Leucovorin
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- National Cancer Institute (NCI)
Hereditary Angioedema
Efficacy, Safety and Tolerability of Icatibant for the Treatment of HAE
The investigators propose a study to evaluate the safety, local tolerability, convenience, and efficacy of self-administered Icatibant for the treatment of acute attacks of hereditary angioedema.
- Trial name
- IHA (NCT01457430)
- Testing
- Icatibant
- Where
- 5 sites in United States
- Volunteers
- 19 people
- Sponsor
- Massachusetts General Hospital
Trauma, Inflammation
Effect of C1-esterase Inhibitor on Systemic Inflammation in Trauma Patients With a Femur or Pelvic Fracture
Trauma and major operation are associated with an excessive inflammation reaction due to tissue injury. This overwhelming immune response is considered to be a major risk factor in the pathogenesis of late inflammatory…
- Conditions
- Trauma, Inflammation, Sepsis, Multiple Organ Dysfunction Syndrome
- Trial name
- CAESAR (NCT01275976)
- Testing
- C1-esterase inhibitor, Saline 0.9%
- Where
- Netherlands
- Volunteers
- 11 people
- Sponsor
- UMC Utrecht
Osteoarthritis, Rheumatoid Arthritis
Outcomes Study of the Trabecular Metal (TM) Reverse Shoulder System
The objectives of this study are to obtain survival and outcome data on the Trabecular Metal Reverse Shoulder System when used in primary or revision reverse total shoulder arthroplasty.
- Conditions
- Osteoarthritis, Rheumatoid Arthritis, Post-traumatic Arthritis, Ununited Humeral Head Fracture
- Trial name
- NCT01480440
- Testing
- Trabecular Metal Reverse Shoulder System
- Where
- 5 sites in United States, United Kingdom
- Volunteers
- 200 people
- Sponsor
- Zimmer Biomet
Hereditary Angioedema, HAE
STOP-HAE: A Phase 3 Study of ADX-324 in HAE
This study will evaluate the efficacy and safety of ADX-324 in participants with Type 1 or Type 2 hereditary angioedema. The study will also evaluate safety, pharmacokinetics (PK), pharmacodynamics (PD), and health-related…
- Conditions
- Hereditary Angioedema, HAE, Hereditary Angioedema - Type 1, Hereditary Angioedema - Type 2
- Trial name
- STOP-HAE (NCT06960213)
- Testing
- ADX-324
- Where
- 70 sites in United States, Argentina and 22 more
- Volunteers
- 90 people
- Sponsor
- ADARx Pharmaceuticals, Inc.
Substance Abuse
Integrated Care (iCare) at Mount Sinai to Improve Substance Use Diagnosis and Treatment Practices in HIV Clinic Settings
Substance abuse and dependence is highly prevalent among people living with HIV and significantly exacerbates morbidity and mortality and accelerates HIV disease progression.
- Trial name
- NCT03092596
- Testing
- Patient-administered screening tool, Patient health navigator-administered screening tool, Patient health navigator-assisted linkage to treatment
- Where
- United States
- Volunteers
- 3,358 people, healthy volunteers welcome
- Sponsor
- Icahn School of Medicine at Mount Sinai
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
Nervous System Disease, Immune System Disease
Detection and Characterization of Neurologic Manifestations of Inborn and Acquired Errors of Immunity
Background: Immune system and nervous system have significant interaction so that People with immunity diseases can have complications that affect the nervous system and people with some neurological disease may have defects in…
- Trial name
- NCT06169150
- Where
- United States
- Volunteers
- 350 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Primary Immunodeficiency Diseases
Immunoglobulin Efficacy and Immune Profiling in Antibody Immunodeficiency
This study aims to systematically evaluate the dynamic alterations in peripheral immune subsets among patients with antibody Immunodeficiency undergoing Immunoglobulin therapy
- Trial name
- IEIPAI (NCT07516002)
- Volunteers
- 15 people
- Sponsor
- Peking University People's Hospital
Epstein-Barr Virus Infections, Primary Immune Deficiency Disorder
EBV-specific Cytotoxic T-lymphocytes (CTLs) for Refractory EBV Infection
Related donor Epstein-Barr Virus (EBV) specific cytotoxic T cells (CTLs) manufactured with the Miltenyi CliniMACS Prodigy Cytokine Capture System will be administered in children, adolescents and young adults with refractory EBV…
- Trial name
- NCT03266653
- Testing
- cytotoxic t-lymphocytes
- Where
- 8 sites in United States
- Volunteers
- 20 people
- Sponsor
- New York Medical College
HIV Infections, Histoplasmosis
A Study of Itraconazole in the Treatment and Prevention of Histoplasmosis, a Fungal Infection, in Patients With AIDS
To evaluate the feasibility of itraconazole as (1) primary therapy in histoplasmosis and (2) maintenance therapy after completion of primary therapy. To evaluate the effect of therapy of CNS histoplasmosis.
- Trial name
- NCT00000975
- Testing
- Itraconazole
- Where
- 12 sites in United States
- Volunteers
- 30 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Saccular and Fusiform Aneurysms, Pseudoaneurysm
Targeting Real World Usage In Stroke Treatment
TRUST Registry is an observational, prospective, long-term, post-market surveillance registry of subjects treated with WallabyPhenox flow modulation devices, stent systems, bifurcation aneurysm implants including their HPC…
- Conditions
- Saccular and Fusiform Aneurysms, Pseudoaneurysm, Vascular Dissection, Perforation
- Trial name
- TRUST (NCT07474935)
- Testing
- Aneurysm Treatment with a Neurovascular Flow Diverter, Dissection Treatment with a Neurovascular Flow Diverter, Perforation Treatment with a Neurovascular Flow Diverter
- Where
- 13 sites in France, Germany and 3 more
- Volunteers
- 5,000 people
- Sponsor
- Phenox GmbH