Clinical trials: Primary Immune Deficiency Disorders
Severe Combined Immunodeficiency (SCID), Immunodeficiency With Predominant T-cell Defect, Unspecified
Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases
The purpose of this study is to determine whether bilateral orthotopic lung transplantation (BOLT) followed by cadaveric partially-matched hematopoietic stem cell transplantation (HSCT) is safe and effective for patients aged…
- Conditions
- Severe Combined Immunodeficiency (SCID), Immunodeficiency With Predominant T-cell Defect, Unspecified, Severe Chronic Neutropenia, Chronic Granulomatous Disease (CGD)
- Trial name
- BOLT+BMT (NCT01852370)
- Testing
- CD3/CD19 negative allogeneic hematopoietic stem cells
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Paul Szabolcs
The Immune Dysregulation Polyendocrinopathy Enteropathy X-linked Syndrome is a Primary Immunodeficiency Caused by Pathogenic Variants in Forkhead Box Protein 3
Study Evaluating a Gene Therapy for IPEX Syndrome Through the Expression of FOXP3 on Deficient T Cells to Produce Tregs-like.
The purpose of this study is to evaluate the safety and efficacy of FOXP3-T4 (an autologous gene therapy) alone or in combination with low-dose IL-2 for the treatment of IPEX syndrome.
- Trial name
- THERIPEX (NCT07697118)
- Testing
- FOXP3-T4 drug product, ILT-101
- Where
- France
- Volunteers
- 5 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Wiskott-Aldrich Syndrome (WAS)
Gene Therapy for Wiskott-Aldrich Syndrome
This is phase I/II protocol to evaluate the safety and efficacy of WAS gene transfer into hematopoietic stem/progenitor cells for the treatment of Wiskott Aldrich Syndrome.
- Trial name
- TIGET-WAS (NCT01515462)
- Testing
- TLT003
- Where
- Italy
- Volunteers
- 8 people
- Sponsor
- Fondazione Telethon
Primary Ciliary Dyskinesia, Kartagener Syndrome
Characterizing the Upper Airway Manifestations in Primary Ciliary Dyskinesia and Primary Immunodeficiencies
Though common, morbidities related to upper airway disease in primary ciliary dyskinesia (PCD) and primary immunodeficiencies (PID) have not been fully characterized.
- Conditions
- Primary Ciliary Dyskinesia, Kartagener Syndrome, Primary Immune Deficiency
- Trial name
- NCT04919018
- Where
- 4 sites in United States, Canada
- Volunteers
- 159 people
- Sponsor
- University of North Carolina, Chapel Hill
Ataxia-Telangiectasia (A-T), Ataxia-Telangiectasia
Sirolimus Treatment in Pediatric Patients With Ataxia-Telangiectasia
Ataxia-telangiectasia (A-T) is a progressive disease which is, to date, incurable, affecting motor skills, and a high cancer and recurrent infections risk.
- Trial name
- NCT07820579
- Testing
- Rapamune (sirolimus/rapamycin)
- Where
- Mexico
- Volunteers
- 6 people
- Sponsor
- Sara Elva Espinosa Padilla, MD
Pneumonia, Pneumocystis Carinii, HIV Infections
Dapsone 100 Mg Versus 50 as Primary Prophylaxis for Pneumocystis Carinii Pneumonia (PCP) in Patients With AIDS-Related Complex (ARC)
To determine which of 2 doses of dapsone is effective prophylaxis for Pneumocystis carinii pneumonia (PCP) in patients with oral thrush or hairy leukoplakia and less than 400 CD4 lymphocytes per mm3.
- Trial name
- NCT00002043
- Testing
- Dapsone
- Where
- United States
- Sponsor
- Jacobus Pharmaceutical
Primary Immunodeficiency
Pharmacokinetics, Safety, and Tolerability of Subcutaneous GAMUNEX-C in Pediatric Subjects With Primary Immunodeficiency
The purpose of this open-label study is to evaluate the pharmacokinetics, safety, and tolerability of subcutaneously (SC; under the skin) administered GAMUNEX-C compared to intravenously (IV; through the vein) administered…
- Trial name
- KIDS (NCT01465958)
- Testing
- GAMUNEX-C
- Where
- 4 sites in United States
- Volunteers
- 12 people
- Sponsor
- Grifols Therapeutics LLC
Coronary Artery Calcification, HIV Infections
French CAC-HIV Cohort Study
Clinical study: Methods: observational transversal two-arm cohort study including adults living with HIV (PLHIV) and HIV negative subjects (HIV-) at intermediate cardiovascular risk.
- Trial name
- CAC-HIV (NCT05563116)
- Testing
- Computed tomography coronary artery calcium (CAC) score (part of routine workup)
- Volunteers
- 689 people
- Sponsor
- Franck Boccara
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
Chronic Lymphocytic Leukemia, Hypogammaglobulinemia
Double-blind, Randomized, Placebo-controlled, Prospective Phase III Study Evaluating Efficacy and Safety of Panzyga in Primary Infection Prophylaxis in Patients With Chronic Lymphocytic Leukemia ("PRO-SID" Study)
Study Evaluating Efficacy and Safety of Panzyga in Primary Infection Prophylaxis in Patients with Chronic Lymphocytic Leukemia
- Trial name
- NCT04502030
- Testing
- Panzyga
- Where
- 84 sites in United States, Czechia and 10 more
- Volunteers
- 247 people
- Sponsor
- Octapharma
Primary Immunodeficiency
IgG Level in Primary Immunodeficiency Switching From Standard SCIG to Every Other Week HyQvia
Most immunodeficiencies are related to severe immunoglobulin deficiencies which require lifelong replacement therapy with immunoglobulin G (IgG) to reduce the incidence and severity of infections.
- Trial name
- NCT02881437
- Testing
- IgHy10
- Where
- 7 sites in France
- Volunteers
- 22 people
- Sponsor
- University Hospital, Lille
Common Variable Immunodeficiency, Granulomatous Disease
Lymphocyte Immunophenotyping in Common Variable Immunodeficiency
The purpose of this study is to discover if differences in the surface markers of B-cells (antibody producing cells of the immune system) in Common Variable Immune Deficiency (CVID) are related to CVID or its…
- Conditions
- Common Variable Immunodeficiency, Granulomatous Disease, Bronchiectasis, Immunoglobulin Treatment
- Trial name
- NCT01196702
- Where
- United Kingdom
- Volunteers
- 210 people, healthy volunteers welcome
- Sponsor
- Barts & The London NHS Trust
Hereditary Angioedema (HAE)
A Study to Learn About the C1-Inhibitor Function as Diagnosis for Hereditary Angioedema
Hereditary angioedema (HAE) is a rare condition. It causes sudden swelling under the skin and inside the body, like in the belly, throat, or genitals.
- Trial name
- AHAE (NCT07293364)
- Testing
- Technochrom C1-INH Kit
- Where
- Algeria
- Volunteers
- 514 people
- Sponsor
- Takeda
Primary Immune Deficiency Disorders, Common Variable Immunodeficiency
A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary Immunodeficiency
The main objective is to determine the efficacy of Gammaplex by measuring the number of serious acute bacterial infections during treatment with Gammaplex over a 12 month period.
- Conditions
- Primary Immune Deficiency Disorders, Common Variable Immunodeficiency, X-linked Agammaglobulinemia, Hyper-IgM Syndrome
- Trial name
- NCT01289847
- Testing
- Gammaplex
- Where
- 10 sites in United States, Chile and 1 more
- Volunteers
- 25 people
- Sponsor
- Bio Products Laboratory
Epstein-Barr Virus (EBV)-Associated Diseases, EBV+ Lymphoproliferative Disease With Primary Immunodeficiency (EBV+ PID LPD)
A Study to Evaluate Tabelecleucel in Participants With Epstein Barr Virus (EBV) Associated Diseases
The purpose of this study is to assess the efficacy and safety of tabelecleucel in participants with EBV-associated diseases.
- Conditions
- Epstein-Barr Virus (EBV)-Associated Diseases, EBV+ Lymphoproliferative Disease With Primary Immunodeficiency (EBV+ PID LPD), EBV+ Lymphoproliferative Disease With Acquired (Non-congenital) Immunodeficiency (EBV+ AID LPD), EBV+ Posttransplant Lymphoproliferative Disease in Central Nervous System (EBV+ CNS PTLD)
- Trial name
- NCT04554914
- Testing
- Tabelecleucel
- Where
- 40 sites in United States, Austria and 5 more
- Volunteers
- 190 people
- Sponsor
- Pierre Fabre Medicament
Hereditary Angioedema (HAE)
A Study of Lanadelumab in Children With Hereditary Angioedema (HAE) in Multiple Countries
HAE is a rare condition. It causes sudden swelling under the skin and inside the body, like in the belly, throat, or genitals. This swelling happens because of a temporary leak in blood vessels but does not cause itching or…
- Trial name
- NCT07251933
- Testing
- No intervention
- Where
- 24 sites in Argentina, France and 4 more
- Volunteers
- 37 people
- Sponsor
- Takeda
Primary Immune Deficiency Disorder, Immune Deficiency Disease
Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures
Phase II prospective trial to assess the rates of donor engraftment using reduced intensity conditioning (RIC) hematopoietic stem cell transplant (HSCT) and post-transplant cyclophosphamide (PTCy) for patients with primary immune…
- Conditions
- Primary Immune Deficiency Disorder, Immune Deficiency Disease, Bone Marrow Failure, Short Telomere Length
- Trial name
- NCT04232085
- Testing
- Alemtuzumab, Fludarabine, Melphalan
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Substance Use, HIV/AIDS
Feasibility of SBIRT-PN
Substance misuse is a common problem among HIV+ individuals. Research suggests that a Screening, Brief Intervention, and Treatment (SBIRT) model can be effective in reducing substance misuse in the general older adult population…
- Conditions
- Substance Use, HIV/AIDS, Older Adults, Behavioral Intervention
- Trial name
- NCT03057119
- Testing
- Psycho-educational content (5 Minutes), Readiness Assessment (10 minutes), Goal-Setting (5 minutes)
- Where
- 3 sites in United States
- Volunteers
- 86 people
- Sponsor
- Florida State University
Cytomegalovirus Infections, Adenovirus Infection
Antiviral Cellular Therapy for Enhancing T-cell Reconstitution Before or After Hematopoietic Stem Cell Transplantation
The purpose of this study is to evaluate whether virus-specific T cell lines (VSTs) are safe and can effectively control three viruses (EBV, CMV, and adenovirus) in patients who have had a stem cell transplant and also in…
- Conditions
- Cytomegalovirus Infections, Adenovirus Infection, EBV Infection
- Trial name
- ACES (NCT03475212)
- Testing
- Virus Specific T-cell (VST) infusion
- Where
- 30 sites in United States
- Volunteers
- 52 people
- Sponsor
- Pediatric Transplantation & Cellular Therapy Consortium
Primary Immune Deficiency Disorder
Study to Evaluate the Efficacy, Tolerability and Safety of Octanorm in Patients With Primary Immunodeficiency Diseases
Clinical phase 3 study to evaluate the efficacy, tolerability and safety of subcutaneous human immunoglobulin (octanorm) in patients with primary immunodeficiency diseases.
- Trial name
- NCT03988426
- Testing
- Octanorm
- Where
- 5 sites in Russia
- Volunteers
- 25 people
- Sponsor
- Octapharma