Clinical trials: Primary Immune Deficiency
Primary Immunodeficiency (PID), Secondary Immunodeficiency (SID)
Assessment of Immunoglobulins (IgG) in a Long-term Non-interventional Study
This non-interventional, epidemiological study assesses long-term outcomes in subjects receiving immunoglobulins (IgG) for any treatment purpose, irrespective of the regimen prescribed by the treating physician, under routine…
- Conditions
- Primary Immunodeficiency (PID), Secondary Immunodeficiency (SID), Neurological Autoimmune Disease
- Trial name
- SIGNS (NCT01287689)
- Testing
- Immunoglobulin G (IgG)
- Where
- 7 sites in Germany
- Volunteers
- 685 people
- Sponsor
- Technische Universität Dresden
Hereditary Angioedema (HAE)
A Study of Lanadelumab in Persons With Hereditary Angioedema (HAE) Type I or II
The main aim of this study is to compare the number of HAE attacks occuring in persons using lanadelumab with the number of HAE attacks before lanadelumab treatment was started.
- Trial name
- ENABLE (NCT04130191)
- Where
- 19 sites in Austria, Germany and 5 more
- Volunteers
- 140 people
- Sponsor
- Shire
Primary Immune Deficiency Disorder
Intensive Care Unit and Secondary and Primary Immune Deficiency
Primary immune deficiencies (PID) are characterized by a failure of the immune system that is not explained by any infectious, neoplastic, or iatrogenic cause.
- Trial name
- ICUSPID (NCT02888535)
- Testing
- Internal Medicine consultation
- Where
- France
- Volunteers
- 16 people
- Sponsor
- University Hospital, Caen
Hereditary Angioedema (HAE)
A Study in Adults With Hereditary Angioedema (HAE) Who Currently Receive Icatibant at Home
The main aim of this study is to describe the treatment patterns, characteristics and outcomes of people with HAE who are currently receiving icatibant in the homecare setting in the United Kingdom (UK).
- Trial name
- NCT05489640
- Testing
- No Intervention
- Where
- United Kingdom
- Volunteers
- 85 people
- Sponsor
- Takeda
Ataxia Telangiectasia, Infections
Susceptibility to Infections in Ataxia Telangiectasia
Death in Ataxia telangiectasia (A-T) is usually due to cancer or chronic lung failure around 20 years of age. Despite low lymphocyte counts (CD3, CD4, CD8 and CD19), IgA and IgG subclass deficiency opportunistic and acute severe…
- Conditions
- Ataxia Telangiectasia, Infections, Immunodeficiency, Lung Disease
- Trial name
- NCT02345135
- Testing
- Blood collection, Lung function measurement, Symptom diary
- Volunteers
- 41 people, healthy volunteers welcome
- Sponsor
- Johann Wolfgang Goethe University Hospital
SCAD, Addison Disease
Spontaneous Coronary Artery Dissection (SCAD) and Autoimmunity
This case control study aims to determine whether spontaneous coronary artery dissection (SCAD) is associated with autoimmune diseases and to update the incidence of SCAD in a population-based cohort.
- Conditions
- SCAD, Addison Disease, Ankylosing Spondylitis, Antiphospholipid Antibody Syndrome
- Trial name
- NCT03941184
- Where
- United States
- Volunteers
- 114 people
- Sponsor
- Mayo Clinic
Primary Immune Deficiency
An Open Label, Multicenter Study to Evaluate the Pharmacokinetics, Efficacy and Safety of ASCENIV™ (IGIV) in Pediatric Subjects With Primary Immunodeficiency Diseases (PIDD)
This is a Phase IV, multicenter, open-label study of Asceniv™ administered as an intravenous infusion of Asceniv™ (IGIV) 300-800 mg/kg every 21 or 28 days in approximately 12 pediatric subjects with Primary Immunodeficiency…
- Trial name
- NCT05070455
- Testing
- Asceniv™
- Where
- 3 sites in United States
- Volunteers
- 12 people
- Sponsor
- ADMA Biologics, Inc.
Gene Therapy
Gene Therapy for X Linked Severe Combined Immunodeficiency
A safety and efficacy clinical study of a lentiviral vector to transfer IL2RG complementary DNA to bone marrow stem cells in ten children with genetic diagnosed X-SCID（severe combined immune deficiency ）.The ten children will be…
- Trial name
- NCT04286815
- Testing
- Lentiviral Vector Gene Therapy
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Children's Hospital of Chongqing Medical University
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
Hereditary Angioedema
Treatment of Angioedema Attacks in Pediatric (Ages 2-11) Post-Trial and Naive Patients With HAE With Sebetralstat
The sebetralstat Early Access Program (EAP) provides early access to the investigational medicinal product (IMP) sebetralstat to eligible and approved Hereditary Angioedema (HAE) pediatric (ages 2-11) post-trial and naïve…
- Trial name
- NCT07216378
- Testing
- Sebetralstat
- Where
- 6 sites in United States
- Sponsor
- KalVista Pharmaceuticals, Ltd.
Primary Immune Deficiency (PID)
Ig NextGen 10% in Patients With Primary Immune Deficiency (PID)
This study aims to assess the safety, tolerability and pharmacokinetics of Ig NextGen 10% in patients with primary immune deficiency currently being treated with Intragam P.
- Trial name
- NCT00405184
- Testing
- IntragamP, Ig NextGen 10%
- Where
- 4 sites in Australia
- Volunteers
- 19 people
- Sponsor
- CSL Limited
Secondary or Primary Immunodeficiency
Tolerance and Patients' Satisfaction With IGHy
Primary immunodeficiencies (PID) represent more than 150 diseases affecting the immune system. More than 50% of PIDs are due to a lack or an insufficiency in antibody production.
- Trial name
- NCT03668288
- Testing
- Data record and questionnaires passation
- Where
- France
- Volunteers
- 20 people
- Sponsor
- CHU de Reims
Primary Immunodeficiency
An Open-label, Multicenter Study to Assess the Pharmacokinetics (PK), Safety, and Tolerability of Subcutaneous IgPro20 in Immunoglobulin (IG) Treatment-naïve Participants With Primary Immunodeficiency (PID)
This is an open-label, multicenter, phase 4 study in IG treatment-naïve participants with PID, conducted in the United States (US), to assess the PK, safety, and tolerability of IgPro20.
- Trial name
- NCT07076446
- Testing
- IgPro
- Where
- 8 sites in United States
- Volunteers
- 8 people
- Sponsor
- CSL Behring
Hereditary Angioedema (HAE)
Study to Evaluate Ecallantide in Paediatric Patients With Acute Attacks of Hereditary Angioedema
The primary objective of this study is: assess the safety and tolerability of ecallantide in paediatric patients for acute attacks of HAE The secondary objectives are: evaluate the pharmacokinetic profile of ecallantide in…
- Trial name
- NCT01253382
- Testing
- ecallantide
- Sponsor
- Shire
Autosomal-dominant Hyper-IgE Syndrome
Safety, Tolerability, and Immunogenicity of One Dose of NDV 3A Vaccine in People With STAT3-Mutated Hyper-IgE Syndrome
Background: AD-HIES is a disease that weakens the immune system. It puts people at risk for infections, particularly Staph and Candida infections.
- Trial name
- NCT02996448
- Testing
- NDV-3A
- Where
- United States
- Volunteers
- 3 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Hereditary Angioedema
Donidalorsen Expanded Access Program for Patients With Hereditary Angioedema
The purpose of the Expanded Access Program is to provide pre-approval access of donidalorsen to eligible patients with Hereditary Angioedema (HAE) who complete the ISIS 721744-CS3 clinical trial.
- Trial name
- NCT06415448
- Testing
- Donidalorsen
- Sponsor
- Ionis Pharmaceuticals, Inc.
Hiv
Engaging Men Through HIV Self-Testing With Differentiated Care to Improve ART Initiation and Viral Suppression Among Men in Malawi
Men in sub-Saharan Africa who test HIV-positive continue to have poor ART initiation and retention outcomes. The proposed project will test a home-based ART intervention for men who are living with HIV compared to facility-based…
- Trial name
- NCT04858243
- Testing
- Home-Based ART, Facility-Based ART
- Where
- Malawi
- Volunteers
- 930 people, healthy volunteers welcome
- Sponsor
- University of California, Los Angeles
Bradykinin-mediated Angioedema
Characterization of Rhythmicity Profiles of Bradykinin-mediated Angioedema Attacks Using a Tracking Smartphone Application.
Bradykinin-mediated angioedema is a rare and disabling disease, characterized by the occurrence of attacks marked by localized swelling of skin, but also of the airways, which can be life-threatening.
- Trial name
- ANGIOAPP (NCT05304091)
- Testing
- Questionnaire on a smartphone application
- Sponsor
- University Hospital, Lille
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
Hereditary Angioedema, HAE
Study to Evaluate the Efficacy and Safety of BCX7353 as an Oral Treatment for the Prevention of HAE Attacks in Japan
This is a phase 3, multi-center, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of oral BCX7353 in preventing acute angioedema attacks in patients with Type I and Type II HAE who live in…
- Trial name
- APeX-J (NCT03873116)
- Testing
- BCX7353 capsules
- Where
- 10 sites in Japan
- Volunteers
- 19 people
- Sponsor
- BioCryst Pharmaceuticals