Clinical trials: Primary Immune Deficiency Disorders
Hereditary Angioedema (HAE)
A Study of Icatibant for Acute Attacks of Hereditary Angioedema in Japanese Participants
The objective of this study is to evaluate the efficacy, pharmacokinetics (PK), and safety of icatibant for the treatment of acute attacks in Japanese participants with type I or type II hereditary angioedema (HAE).
- Trial name
- NCT03888755
- Testing
- Icatibant
- Where
- 8 sites in Japan
- Volunteers
- 8 people
- Sponsor
- Shire
Leukemia, Leukemia, Lymphoblastic
Observational Study of People Living With HIV Treated With CD19-directed CAR T Cell
This protocol will develop an observational cohort of PLWH who have been or are being treated with CAR19 therapy outside of an AMC clinical trial.
- Conditions
- Leukemia, Leukemia, Lymphoblastic, Lymphoma, Large B-Cell, Diffuse, Follicular Lymphoma
- Trial name
- CS22-03 (NCT05784415)
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- AIDS Malignancy Consortium
HIV Primary Infection
Bictegravir/FTC/TAF for the Treatment of Primary HIV Infection
The purpose of this study is to assess the efficacy of Bictegravir/FTC/TAF in patients with less of 100 days post HIV infection
- Trial name
- BIC-PHI (NCT04483674)
- Testing
- 50mg bictegravir/200mg emtricitabine/25mg tenofovir alafenamide
- Where
- Spain
- Volunteers
- 66 people
- Sponsor
- Anna Cruceta
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
Angio Edema Hereditary
Status of Dental Care Practices in Patients With Hereditary Angioedema
Hereditary angioedema (HAE) is a rare genetic disease, it can occur at any age and evolves in flare-ups. These are subcutaneous or submucosal edemas responsible for tightness and pain. All areas can be reached.
- Trial name
- PRADA (NCT05776784)
- Testing
- dental procedure
- Where
- France
- Volunteers
- 800 people
- Sponsor
- University Hospital, Grenoble
Hereditary Angioedema (HAE)
Post Study Access of CSL312 (Garadacimab) for Pediatric Participants With Hereditary Angioedema Who Have Completed the CSL312_3003 Study
This protocol for post study access allows pediatric participants (2-11 years old at the time of consent) with HAE who have completed study CSL312_3003 (NCT05819775) to continue treatment with CSL312 for routine prevention of HAE…
- Trial name
- NCT07159464
- Testing
- CSL312
- Sponsor
- CSL Behring
Wiskott-Aldrich Syndrome, Thrombocytopenia
Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients
The purpose of this project is to describe the pathophysiology of thrombocytopenia and bleeding in patients with Wiskott-Aldrich Syndrome (WAS) and determine the response to thrombopoietic agents in vitro and in vivo.
- Conditions
- Wiskott-Aldrich Syndrome, Thrombocytopenia, Bleeding
- Trial name
- WAS (NCT00909363)
- Testing
- Promacta, blood drawing in patients with WAS, blood drawing in healthy controls
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Weill Medical College of Cornell University
Severe Combined Immunodeficiency
Autologous Gene Therapy for Artemis-Deficient SCID
This study aims to determine if a new method can be used to treat Artemis-deficient Severe Combined Immunodeficiency (ART-SCID), a severe form of primary immunodeficiency caused by mutations in the DCLRE1C gene.
- Trial name
- NCT03538899
- Testing
- AProArt-CD34, CliniMACS® CD34 Reagent System cell sorter device, Busulfan
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- University of California, San Francisco
Primary Immunodeficiency Diseases (PID)
Study to Determine the Dose of Recombinant Human Hyaluronidase Needed to Infuse a Full Dose of IGIV Subcutaneously
The purpose of the study is to determine the feasibility of infusing a full 4-week dose of Immune Globulin Intravenous (Human), 10% in a single subcutaneous site and the amount of recombinant human hyaluronidase needed to infuse…
- Trial name
- NCT00782106
- Testing
- Recombinant human hyaluronidase + immune globulin intravenous
- Where
- 3 sites in United States
- Volunteers
- 11 people
- Sponsor
- Baxalta now part of Shire
Acne Vulgaris, Allergic Rhinitis
MedlinePlus Health Prescriptions: Developing a Pragmatic Approach for Clinic Use
The specific aim of this proposed pilot study is to compare two standardized processes (paper and electronic) to deliver a customized MedlinePlus health information prescription.
- Conditions
- Acne Vulgaris, Allergic Rhinitis, Anxiety, Asthma
- Trial name
- NCT01050465
- Testing
- health information prescription
- Volunteers
- 907 people, healthy volunteers welcome
- Sponsor
- University of Missouri-Columbia
Severe Combined Immunodeficiency
Genetic Basis of Immunodeficiency
This study will examine the role of hereditary factors in different forms of severe combined immunodeficiency (SCID). Patients with immunodeficiencies may be eligible for this study.
- Trial name
- NCT00055172
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Primary Immune Deficiency Diseases (PIDD)
Study of PlasmaCap IG in Adults and Children With PIDD
The purpose of this study is to investigate the efficacy, safety, tolerability, and pharmacokinetic profile of the investigational medicinal product (IMP) and to determine, on the basis of historical control data, how it compares…
- Trial name
- NCT03238079
- Testing
- 10% IGIV
- Where
- 12 sites in United States, Canada
- Volunteers
- 63 people
- Sponsor
- Evolve Biologics
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
Subcutaneous Treatment With Icatibant for Acute Attacks of Hereditary Angioedema (HAE)
Primary Outcome Measures: The primary endpoint was the time to onset of symptom relief of the first attack in the double blind phase.
- Trial name
- FAST2 (NCT00500656)
- Testing
- Icatibant, Tranexamic Acid
- Where
- Italy
- Volunteers
- 85 people
- Sponsor
- Shire
Bloom Syndrome
Biological Significance of the Bloom's Syndrome Protein
Since 1960, persons with the very rare disorder Bloom's syndrome (BS) have been followed clinically, documenting clinical matters as obtained from their doctors.
- Trial name
- NCT00021437
- Where
- United States
- Sponsor
- National Center for Research Resources (NCRR)
Common Variable Immunodeficiency, Agammaglobulinemia
Efficacy and Safety of Vivaglobin® in Previously Untreated Patients With Primary Immunodeficiency
The objective of this study is to assess the efficacy and safety of Vivaglobin in previously untreated patients (PUPs) with primary immunodeficiency (PID) over a 25-week observation period.
- Trial name
- NCT00520494
- Testing
- Vivaglobin
- Where
- 6 sites in Canada, Germany and 2 more
- Volunteers
- 18 people
- Sponsor
- CSL Behring
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)
Hereditary Angioedema
Safety, Tolerability, PK, PD of ADX-324 in Healthy Volunteers and Hereditary Angioedema Patients
The first-in-human Phase 1 study will evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ADX-324 in healthy volunteers (HV) and in patients with Hereditary Angioedema (HAE).
- Trial name
- NCT05691361
- Testing
- ADX-324
- Where
- Australia
- Volunteers
- 53 people, healthy volunteers welcome
- Sponsor
- ADARx Pharmaceuticals, Inc.
Ataxia Telangiectasia
The Validity of Forced Expiratory Maneuvers in Ataxia Telangiectasia Studied Longitudinally
Rationale: Forced spirometry maneuvers are not routinely performed in patients with Ataxia Telangiectasia (A-T), even though they suffer from respiratory illnesses.
- Trial name
- NCT00951886
- Where
- Israel
- Volunteers
- 28 people
- Sponsor
- Sheba Medical Center
Asthma
C1-inhibitor in Allergic ASThma Patients
The purpose of this proof-of-concept study is to determine the effect of Intestinal Microbiota Depletion or Intravenous Administration of C1-inhibitor on Inflammation and Coagulation after Bronchial Instillation of House Dust…
- Trial name
- CAST (NCT03051698)
- Testing
- C1-inhibitor, Saline, Antibiotics
- Where
- Netherlands
- Volunteers
- 37 people
- Sponsor
- T. van der Poll