Clinical trials: Primary Immune Deficiency Disorders, open to volunteers
Primary Immunodeficiencies (PID), Secondary Immunodeficiencies (SID)
Assessing Patient Preference for Infusion Systems
This crossover study evaluates patient experiences and preferences between mechanical and electrical infusion pumps for subcutaneous immunoglobulin (SCIg) therapy.
- Trial name
- NCT07189013
- Testing
- Mechanical infusion pump
- Where
- United Kingdom
- Volunteers
- 52 people
- Sponsor
- KORU Medical Systems, Inc.
Hereditary Angioedema (HAE)
A Long-Term Study of Navenibart in Participants With Hereditary Angioedema
This is a Phase 3 multicenter trial in 2 parts to evaluate the long-term safety and efficacy of navenibart in adult and adolescent participants with hereditary angioedema (HAE) who participated in STAR-0215-301 (NCT06842823…
- Trial name
- NCT07204938
- Testing
- navenibart
- Where
- 40 sites in United States, Brazil and 16 more
- Volunteers
- 145 people
- Sponsor
- Astria Therapeutics, Inc.
Primary Antibody Deficiency Disorders
Exercise Intervention and Immunodeficiency
The purpose of this study is to assess if a 12-week exercise program is safe, practical, and acceptable for adults with primary antibody deficiencies.
- Trial name
- NCT07698197
- Testing
- App-based exercise intervention
- Where
- 2 sites in United States
- Volunteers
- 20 people
- Sponsor
- Yale University
HIV, Kaposi's Sarcoma
Specimen Collections From Participants With HIV Infection, KSHV Infection, Viral-Related Pre-malignant Lesions and Cancer
BACKGROUND: A number of important scientific advances can be made through the study of blood, bone marrow, tumor, or other tissue samples from patients with HIV infection, infection with Kaposi s sarcoma associated herpesvirus…
- Conditions
- HIV, Kaposi's Sarcoma, Lymphomas, Multicentric Castleman's Disease
- Trial name
- NCT00006518
- Where
- United States
- Volunteers
- 1,029 people
- Sponsor
- National Cancer Institute (NCI)
Kidney Transplant
Improving Deceased-Donor Kidney Transplant Outcomes Via a Single Intragraft Injection of C1 Esterase Inhibitor (IMPROVE TRIAL)
The purpose of this study is to find out if Berinert can improve kidney function in the first year after transplant and to find out what effects, good or bad, Berinert will have in the kidney recipient.
- Trial name
- IMPROVE (NCT06919003)
- Testing
- Berinert
- Where
- 4 sites in United States
- Volunteers
- 180 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
HIV/AIDS and Infections
A Randomised Placebo Controlled Trial of ART Plus Dual Long-acting HIV-specific Broadly Neutralising Antibodies (bNAbs).
RIO is a placebo-controlled double-blinded two arm prospective phase II randomised controlled trial . This study will test the use of broadly neutralising antibodies (bNAbs) in participants with treated primary HIV infection…
- Trial name
- RIO (NCT04319367)
- Testing
- Investigational Medicinal Product
- Where
- 12 sites in Denmark, United Kingdom
- Volunteers
- 72 people
- Sponsor
- Imperial College London
Acquired Angioedema Due to C1-Inhibitor Deficiency (AAE-C1-INH)
Study of Oral Deucrictibant XR Tablet for Prophylaxis and Deucrictibant Soft Capsule for On-Demand Treatment of Angioedema Attacks in Adults With Acquired Angioedema Due to C1 Inhibitor Deficiency
This is a Phase 3, multicenter, 3-part study, with 2 randomized, double-blind, placebo-controlled parts and an open-label extension part, to evaluate the efficacy and safety of orally administered deucrictibant XR tablet for…
- Trial name
- CREAATE (NCT07266805)
- Testing
- Deucrictibant
- Where
- 32 sites in United States, Australia and 14 more
- Volunteers
- 48 people
- Sponsor
- Pharvaris Netherlands B.V.
Immunodeficiencies, Primary Immunodeficiencies (PID)
V-IMMUNE: A Novel Immunoglobulin Therapy for Immunodeficiency
This is a phase III, non-randomized clinical trial (VIP Study) designed to assess the safety and efficacy of V-IMMUNE®, a 5% human normal immunoglobulin preparation, in approximately 50 patients with primary immunodeficiency…
- Conditions
- Immunodeficiencies, Primary Immunodeficiencies (PID), Agammaglobulinemia
- Trial name
- VIP (NCT06954441)
- Testing
- Intravenous immunoglobulin (IVIG)
- Where
- Brazil
- Volunteers
- 50 people
- Sponsor
- On Pharma Importadora, Exportadora e Distribuidora de Medicamentos LTDA.
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
Cystic Fibrosis, Pulmonary Fibrosis
Role of Genetic Factors in the Development of Lung Disease
This study is designed to evaluate the genetics involved in the development of lung disease by surveying genes involved in the process of breathing and examining the genes in lung cells of patients with lung disease.
- Conditions
- Cystic Fibrosis, Pulmonary Fibrosis, Tuberous Sclerosis, Asthma
- Trial name
- NCT00001532
- Where
- 2 sites in United States
- Volunteers
- 3,500 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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
EBV Viremia, CMV Viremia
25BT056: Basket Study of Viral Cytotoxic T Lymphocytes
This study is evaluating the safety and effectiveness of virus-specific cytotoxic T lymphocytes (vCTLs) for treating difficult-to-control viral infections caused by cytomegalovirus (CMV), adenovirus (ADV), Epstein-Barr virus…
- Conditions
- EBV Viremia, CMV Viremia, BK Viremia, Adenovirus
- Trial name
- NCT07873970
- Testing
- Virus-Specific Cytotoxic T Lymphocytes (vCTLs)
- Volunteers
- 35 people
- Sponsor
- Children's Hospital of Philadelphia
Cytomegalovirus Infections, Primary Immune Deficiency Disorder
Virus Specific Cytotoxic T-Lymphocytes (CTLs) for Refractory Cytomegalovirus (CMV)
CMV cytotoxic T cells (CTLs) manufactured with the Miltenyi CliniMACS Prodigy Cytokine Capture System will be administered in children, adolescents and young adults (CAYA) with refractory cytomegalovirus (CMV) infection post…
- Trial name
- NCT03266640
- Testing
- viral specific cytotoxic t-lymphocytes
- Where
- 9 sites in United States
- Volunteers
- 20 people
- Sponsor
- New York Medical College
Chronic Granulomatous Disease, Infection
NADPH Oxidase Correction in mRNA-transfected Granulocyte-enriched Cells in Chronic Granulomatous Disease (CGD)
Background: CGD is caused by a gene mutation. For people with CGD, their cells cannot kill germs well, so they can get frequent or life-threatening infections.
- Trial name
- NCT05189925
- Testing
- gp91 Grans
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Severe Combined Immunodeficiencies (SCID)
Follow-up of the Cohort of Newborns Screened at Birth Using TREC Analysis
Since September 2025, neonatal screening for severe combined immunodeficiency (SCID) has been generalized in France. These genetic disorders, which are asymptomatic at birth, cause severe immunodeficiency, exposing infants to…
- Trial name
- DépisTrec (NCT07704281)
- Testing
- Data collection
- Where
- France
- Volunteers
- 450 people
- Sponsor
- Nantes University Hospital
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
Hereditary Angioedema
Safety and Effectiveness of Sebetralstat (KVD900) for Short-Term Prophylaxis Before Procedures in People With Hereditary Angioedema (KONTROL)
This is a Phase 4, prospective, open-label trial to evaluate the safety and effectiveness of sebetralstat when used for STP for a qualifying procedures in patients 12 years of age or older with hereditary angioedema (HAE).
- Trial name
- NCT07654829
- Testing
- KVD900 600 mg
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- KalVista Pharmaceuticals, Ltd.
SCID, X-Linked
SCID-X1 Gene Therapy Via Intravenous Lentiviral (Ivlv-X1) Injection
This is a Phase I/II gene therapy trial treating X-linked severe combined immunodeficiency (SCID-X1) using a self-inactivating lentiviral vector (ivlv-X1) to functionally correct the genetic defect.
- Trial name
- NCT03217617
- Testing
- Direct intravenous injection of ivlv-X1 lentiviral vector
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
Atopy, Primary Immunodeficiency
NIAID Centralized Sequencing Protocol
Background: Genetic testing called "sequencing" helps researchers look at DNA. Genes are made of DNA and are the instructions for our bodies to function. We all have thousands of genes.
- Conditions
- Atopy, Primary Immunodeficiency, Autoimmunity, Autoinflammation
- Trial name
- NCT03206099
- Where
- 2 sites in United States
- Volunteers
- 20,000 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
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