Clinical trials: Primary Immune Deficiency Disorders
Immunologic Deficiency Syndromes, Primary Immune Deficiency (PID)
Genetic Study of Immunodeficiency: Search for New Genetic Causes for Primary Immunodeficiencies
Individuals with suspected primary immunodeficiency will be studied and the results compared with healthy controls. Primary immunodeficiency may manifest as recurrent, severe or unusual infections as well as signs and symptoms of…
- Trial name
- GSI (NCT02735824)
- Testing
- Skin Biopsy, Mouth Swab or Saliva Collection, Blood Sampling
- Where
- Switzerland
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- University Children's Hospital, Zurich
Hereditary Angioedema (HAE)
Firazyr® Patient Registry (Icatibant Outcome Survey - IOS)
The Icatibant Outcome Survey (IOS) is a prospective, observational disease registry designed to document the routine clinical outcomes over time in participants with angioedema treated with Firazyr® (icatibant) and/or Cinryze®…
- Trial name
- NCT01034969
- Where
- 74 sites in Australia, Austria and 12 more
- Volunteers
- 1,761 people
- Sponsor
- Shire
HIV Infections
New Era Study: Treatment With Multi Drug Class (MDC) HAART in HIV Infected Patients
This is a multi-center, open-label, non-randomized proof-of-concept trial. Two cooperating HIV-specialized centres represented by Dr. med. Hans Jaeger and Prof. Dr.
- Trial name
- NewEra (NCT00908544)
- Testing
- PHI-patients, CHI-patients
- Where
- 8 sites in Germany
- Volunteers
- 47 people
- Sponsor
- MUC Research GmbH
Primary Immune Deficiency Disorder, Common Variable Immunodeficiency
The Impact of Exercise on Stress, Fatigue, and Quality of Life in Individuals With Primary Immunodeficiency Disease
This aim of this research project is to determine if low to moderate level exercise can have an impact on stress, fatigue, and quality of life for individuals diagnosed with a primary immunodeficiency disease.
- Conditions
- Primary Immune Deficiency Disorder, Common Variable Immunodeficiency, Specific Antibody Deficiency, Hypergammaglobulinemia
- Trial name
- NCT03211689
- Testing
- Exercise Program
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Stockton University
Bone Marrow Failure, Hemoglobinopathies
Naive T Cell Deplete Grafts for GVHD Prevention in Non-Malignant Diseases
This phase II trial investigates how well a naive T cell depleted graft work for the reduction of graft versus host disease in patients with non-malignant diseases requiring hematopoietic cell transplantation.
- Conditions
- Bone Marrow Failure, Hemoglobinopathies, Primary Immune Deficiency (PID), Autoimmune Cytopenia
- Trial name
- NCT07660783
- Testing
- ALLOGENEIC CD34+ ENRICHED AND CD45RA- DEPLETED PBSCs
- Where
- 2 sites in United States
- Volunteers
- 40 people
- Sponsor
- Fred Hutchinson Cancer Center
Viral Infection, Primary Immune Deficiency Disorder
Treatment of Refractory BK Infections With Related Donor BK Specific Cytotoxic T-cells (CTLs)
BK cytotoxic T cells (CTLs) manufactured with the Miltenyi CliniMACS Prodigy Cytokine Capture System will be safe and effective in decreasing specific viral load in children, adolescents and young adults (CAYA) with refractory BK…
- Trial name
- NCT04197596
- Testing
- BK CTL
- Where
- 7 sites in United States
- Volunteers
- 40 people
- Sponsor
- New York Medical College
Primary Immunodeficiency
Investigation of the Effects of Pulmonary Rehabilitation in Children With Primary Immunodeficiency
Primary immunodeficiencies (PID) are a heterogeneous group of diseases that occur as a result of disorders that affect the development, differentiation and/or function of various cells and building blocks in the immune system.
- Trial name
- NCT06092528
- Testing
- Inspiratory muscle training, Upper extremity aerobic exercise training, Lower extremity aerobic exercise training
- Where
- Turkey (Türkiye)
- Volunteers
- 40 people
- Sponsor
- Gazi University
Primary Immunodeficiency Diseases (PID)
A Database Survey to Evaluate the Safety of Immune Globulin Subcutaneous (Human), 20% Solution in Participants With Primary Immunodeficiency
This study is a retrospective database study in Japan to evaluate the safety of Immune Globulin Subcutaneous (Human), 20% Solution in participants with primary immunodeficiency disease (PID).
- Trial name
- NCT06565078
- Testing
- Immune Globulin Subcutaneous (Human), 20% Solution
- Where
- Japan
- Volunteers
- 100 people
- Sponsor
- Takeda
Hereditary Angioedema
C1-INH Compared to Placebo at the Time of Prodromal Symptoms for Hereditary Angioedema (HAE) Exacerbation
The study hypothesis is that treatment of Hereditary Angioedema at the time of prodromal symptoms will decrease morbidity associated with the disease
- Trial name
- NCT01151735
- Testing
- C-1-esterase
- Sponsor
- Penn State University
Agammaglobulinemia, IgG Deficiency
Efficacy and Safety of Intravenous Immunoglobulin IgPro10 in Patients With Primary Immunodeficiencies (PID)
The purpose of this study is to demonstrate the effect of IgPro10 on the prevention of serious bacterial infections in patients with primary immunodeficiency.
- Conditions
- Agammaglobulinemia, IgG Deficiency, Common Variable Immunodeficiency
- Trial name
- NCT00168025
- Testing
- Immunoglobulins Intravenous (Human)
- Volunteers
- 89 people
- Sponsor
- CSL Behring
Autophagy, Galectins
Study of Autophagy and the Effects of GALIG Gene Products in HIV-1 Infected Patients Who Are Under Antiretroviral Therapy Since Primary-infection, Chronic Phase, or Never Treated.
Little is known about autophagy during HIV infection. Recently, two different teams reported important dysfunctions of autophagy in HIV-infected patients despite sustained suppressive antiretroviral therapy.
- Conditions
- Autophagy, Galectins, HIV Infections, Highly Active Antiretroviral Therapy
- Trial name
- ATGALIG-HIV (NCT04160455)
- Testing
- expression of a panel
- Where
- France
- Volunteers
- 180 people
- Sponsor
- Centre Hospitalier Régional d'Orléans
Genetic Disorders, Hereditary Angioedema
A Study of the Safety and Immunogenicity of Repeated rhC1INH Administration
Hereditary angioedema ("HAE") is a disease characterized by recurrent tissue swelling affecting various body locations. Recent literature shows that patients with frequent attacks may benefit from long-term prophylaxis.
- Trial name
- OPERA (NCT00851409)
- Testing
- Recombinant Human C1 Inhibitor
- Where
- 2 sites in Netherlands, Romania
- Volunteers
- 25 people
- Sponsor
- Pharming Technologies B.V.
Primary Immunodeficiency, Agammaglobulinemia
Efficacy and Safety Study of Kedrion IVIG 10% to Treat Subjects With Primary Immunodeficiency (PID)
The purpose of this study is to determine whether Kedrion IVIG 10% (an immunoglobulin solution) is effective in treating Primary Immunodeficiency (PID).
- Conditions
- Primary Immunodeficiency, Agammaglobulinemia, Hypogammaglobulinemia, Antibody Deficiency
- Trial name
- NCT01581593
- Testing
- Kedrion IVIG 10%
- Where
- 15 sites in United States, Canada
- Volunteers
- 45 people
- Sponsor
- Kedrion S.p.A.
Hereditary Angioedema
A Single Dose Safety, Tolerability, Pharmacokinetic and Food Effect Study of KVD900 (Sebetralstat) in Healthy Volunteers
A safety, tolerability, pharmacokinetic and food effect study of KVD900 in healthy volunteers.
- Trial name
- NCT04349800
- Testing
- KVD900
- Where
- United Kingdom
- Volunteers
- 84 people, healthy volunteers welcome
- Sponsor
- KalVista Pharmaceuticals, Ltd.
Hereditary Angioedema (HAE)
Open-label, Single-arm Study to Assess the Pharmacokinetics, Safety, and Tolerability of a Single Subcutaneous Dose of Icatibant in Healthy Japanese Volunteers
This is a single-dose study to evaluate the pharmacokinetics, safety, and tolerability of icatibant administered to adult Japanese subjects.
- Trial name
- NCT02045264
- Testing
- Icatibant (30 mg)
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Shire
Hematologic Malignancies
A Single-arm Safety Study of Transplantation Using Umbilical Cord Blood and Human Placental-derived Stem Cells From Partially Matched Related Donors in Persons With Certain Malignant Blood Diseases and Non-malignant Disorders
To investigate the safety of partially matched related human placental-derived stem cells (HPDSC) administered in conjunction with umbilical cord blood (UCB) stem cells from the same donor in subjects with various malignant or…
- Trial name
- HPDSC (NCT00596999)
- Testing
- UCB and HPDSC
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Celgene Corporation
Bronchiectasis, Cystic Fibrosis
Natural History of Bronchiectasis
Bronchiectasis is a chronic inflammatory and muco-obstructive condition that results in abnormal dilation of the airways, decline in lung function, frequent exacerbations, and chronic respiratory tract infections.
- Conditions
- Bronchiectasis, Cystic Fibrosis, Autoimmune Disease, Common Variable Immunodeficiency
- Trial name
- NCT00943514
- Where
- United States
- Volunteers
- 900 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Hereditary Angioedema (HAE)
A Study With Lanadelumab in Persons With Hereditary Angioedema (HAE) in Poland
The main aim of this study is to learn about how many persons with HAE type I or type II are attack-free when treated with lanadelumab in real life, how many attacks occur and how many of these attacks need rescue treatment and…
- Trial name
- CHOPIN (NCT05147181)
- Where
- 14 sites in Poland
- Volunteers
- 48 people
- Sponsor
- Takeda
Immune Deficiency, Antibody
Construction of a Health-related Quality of Life (HR-QOL) Questionnaire for Patients With Primary Antibody Deficiency Disease
This research is being done to construct a survey questionnaire, a quality of life tool, to determine the health, well-being, ability to perform daily activities, and physical, social and emotional functioning in participants…
- Trial name
- HR-QOLPIDD (NCT02542228)
- Testing
- questionnaire survey
- Volunteers
- 76 people
- Sponsor
- University of South Florida
Ataxia Telangiectasia
Open-Label Extension of EryDex Study IEDAT-04-2022
This is an international, multi-center, prospective, open-label, non-comparative study aiming to provide access to treatment with EryDex to ataxia telangiectasia (A-T) patients who completed the IEDAT-04-2022 trial which studied…
- Trial name
- OLE_NEAT (NCT06664853)
- Testing
- Dexamethasone sodium phosphate
- Where
- 21 sites in United States, Denmark and 7 more
- Volunteers
- 101 people
- Sponsor
- Quince Therapeutics S.p.A.