Clinical trials: Primary Immune Deficiency Disorders
Primary Immunodeficiency
Quality of Life Among Children With Inborn Error of Immunity
In this study, we aim to assess the QOL of pediatric IEI patients compared with healthy controls and to determine different factors affecting QOL in these patients.
- Trial name
- NCT07262047
- Testing
- Quality of Life Assessment
- Volunteers
- 50 people
- Sponsor
- Sohag University
Hereditary Angioedema
Phase 1 Crossover Study in Healthy Subjects to Evaluate the PK Profile of KVD824 Following Single and Multiple Doses of Modified Release (MR) Formulations
This is a 3 part, phase 1 crossover study in healthy subjects to evaluate the pharmacokinetic profile of KVD824 following single and multiple doses of novel KVD824 modified-release formulations compared with a reference KVD824…
- Trial name
- NCT05118958
- Testing
- KVD824 Prototype 1 modified-release tablet, KVD824 Prototype 2 modified-release tablet, KVD824 Immediate-Release Capsule
- Where
- United Kingdom
- Volunteers
- 37 people, healthy volunteers welcome
- Sponsor
- KalVista Pharmaceuticals, Ltd.
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, Neonatal Screening
Neonatal Screening of Severe Combined Immunodeficiencies
The goal of the proposed research is to observe the prevalence and establish the validity of a newborn screening method for severe combined immunodeficiency (SCID).
- Trial name
- NCT02590328
- Where
- China
- Volunteers
- 180,000 people
- Sponsor
- Children's Hospital of Fudan University
Angioedema
Subcutaneous Treatment With Icatibant for Acute Attacks of Hereditary Angioedema
The purpose of this study is to assess the efficacy and safety of Icatibant, a bradykinin antagonist in the treatment of acute cutaneous and/or abdominal attacks in patients with hereditary angioedema (HAE).
- Trial name
- NCT00097695
- Testing
- Icatibant
- Where
- United States
- Volunteers
- 84 people
- Sponsor
- Shire
Severe Combined Immune Deficiency (SCID)
Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus
Background: Severe combined immune deficiency (SCID) is a group of conditions where the immune system does not work properly. The only cure for most SCIDs is a stem cell transplant (getting cells from a donor).
- Trial name
- NCT04370795
- Testing
- Sirolimus, Busulfan, Horse -Anti-thymocyte
- Where
- United States
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Cancer
Caspofungin Acetate in Treating Aspergillosis in Patients With Hematologic Cancer or in Patients Who Have Undergone a Stem Cell Transplant
RATIONALE: Antifungals, such as caspofungin acetate, may be effective in treating fungal infections caused by chemotherapy or stem cell transplant.
- Trial name
- NCT00110045
- Testing
- caspofungin acetate
- Where
- 13 sites in Belgium, France and 5 more
- Volunteers
- 171 people
- Sponsor
- European Organisation for Research and Treatment of Cancer - EORTC
Common Variable Immunodeficiency, Agammaglobulinemia
Specific IgG Antibody in Patients With Primary Antibody Deficiencies Treated With Subcutaneous Immunoglobulin
Objective: Measure serum IgG antibody to Streptococcus pneumoniae serotypes 1, 3, 5, 6B, 9V e 14, Haemophilus influenzae type b and tetanus toxoid in patients with primary antibody deficiencies who were treated with subcutaneous…
- Trial name
- NCT00661401
- Testing
- gammaglobulin
- Where
- Brazil
- Volunteers
- 5 people
- Sponsor
- Federal University of São Paulo
Primary Immunodeficiency Diseases (PID)
Post-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric PIDD Subjects
The purpose of the study is to acquire additional data on safety, tolerability and immunogenicity of HyQvia in pediatric (age two to <18 years) patients with Primary Immunodeficiency Diseases (PIDD)
- Trial name
- NCT03116347
- Testing
- HYQVIA, KIOVIG, Cuvitru
- Where
- 20 sites in Czechia, Denmark and 6 more
- Volunteers
- 42 people
- Sponsor
- Baxalta now part of Shire
HIV Infections
Evaluating the Pharmacokinetics, Feasibility, Acceptability, and Safety of Oral Pre-Exposure Prophylaxis for HIV Prevention During Pregnancy and Postpartum
The purpose of this study was to evaluate the pharmacokinetics, feasibility, acceptability, and safety of a fixed-dose combination of emtricitabine and tenofovir disoproxil fumarate (FTC/TDF) as oral daily pre-exposure…
- Trial name
- NCT03386578
- Testing
- Emtricitabine/Tenofovir Disoproxil Fumarate (FTC/TDF), Behavioral HIV risk reduction package, Enhanced adherence support
- Where
- 7 sites in Malawi, South Africa and 2 more
- Volunteers
- 780 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Primary Immunodeficiency
The Benefit of 5% IVIG for Patients With Primary Immunodeficiency Disorders Who Experience Adverse Events on 10% IVIG Preparations
Patients with primary immunodeficiency disorders (PID) on intravenous immunoglobulin (IVIG) treatment may experience adverse events (AEs).
- Trial name
- NCT03339778
- Volunteers
- 15 people
- Sponsor
- IMMUNOe Research Centers
Severe Infection, Severe Immune Deficiency
Systematic Screening for Primary Immunodeficiencies in Patients Admitted for Severe Infection in Pediatric Intensive Care Unit
Severe infections in pediatric intensive care unit are not uncommon. Historically, the diagnosis of hereditary (primary) immune deficiency required a combination of recurrent clinical signs and biological stigmas.
- Trial name
- SPYSI (NCT04990908)
- Where
- France
- Volunteers
- 100 people
- Sponsor
- University Hospital, Montpellier
Hereditary Angioedema
A Relative Bioavailability Study of Two Formulations of BCX7353
This is an open-label, randomized study to investigate the relative bioavailability of two formulations of BCX7353 and to determine if there is a food effect
- Trial name
- NCT03202784
- Testing
- BCX7353
- Where
- United Kingdom
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- BioCryst Pharmaceuticals
Primary Immunodeficiency
A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Immune Globulin (Human) 10% (Gamunex-C) PEG Process (IVIG-PEG) Compared to Gamunex-C in Participants With Primary Humoral Immunodeficiency
The purpose of this study is to demonstrate bioequivalence of IVIG-PEG with Gamunex-C (IVIG-C) at steady-state as determined by comparing total Immunoglobulin G (IgG) area under the concentration-time curve during the defined…
- Trial name
- NCT04561115
- Testing
- Gamunex-C, IVIG-PEG
- Where
- 10 sites in United States
- Volunteers
- 33 people
- Sponsor
- Grifols Therapeutics LLC
Primary Immune Deficiency Disorder
Primary Immunodeficiency in Kuwait
Background/Rationale: Epidemiological data about Primary Immunodeficiency Disorders (PIDD) in Kuwait is needed to better understand peculiarities and to compare them with other regions and ethnicity.
- Trial name
- NCT03618147
- Where
- Kuwait
- Volunteers
- 314 people
- Sponsor
- Kuwait Society for Allergy and Clinical Immunology
Hiv
Confidential Care and Adolescent HIV Testing
The purpose of this study is to assess the feasibility and acceptability of structural intervention components to increase adolescent HIV testing uptake by improving the implementation of confidential care as standard practice in…
- Trial name
- NCT06279949
- Testing
- Provider training, Clinic protocols
- Volunteers
- 4 people
- Sponsor
- Yale University
Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)
Natural History of Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)
This is a prospective longitudinal natural history study with a retrospective cross-sectional arm aimed at determining the natural history of sphingosine phosphate lyase insufficiency syndrome (SPLIS), a recently recognized…
- Trial name
- SPLIS-HIS (NCT06669949)
- Testing
- no intervention
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- University of California, San Francisco
Infections, Pneumonia
Natural History, Management, and Genetics of the Hyperimmunoglobulin E Recurrent Infection Syndrome (HIES)
The Hyper IgE Syndromes (HIES) are primary immunodeficiencies resulting in eczema and recurrent skin and lung infections. Autosomal dominant Hyper IgE syndrome (AD-HIIES; Job's syndrome) is caused by STAT3 mutations, and is a…
- Conditions
- Infections, Pneumonia, Immune System Diseases, STAT3 Transcription Factor
- Trial name
- NCT00006150
- Where
- United States
- Volunteers
- 600 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Hereditary Angioedema (HAE), Suicidal Ideation
Suicide Ideation in Hereditary Angioedema
This study focuses on patients with Hereditary Angioedema (HAE) to better understand how mental health affects overall well-being.
- Conditions
- Hereditary Angioedema (HAE), Suicidal Ideation, Cardiovascular Risk
- Trial name
- HAE-SAFE (NCT06811467)
- Volunteers
- 100 people
- Sponsor
- Ivan Cherrez Ojeda
Severe Combined Immunodeficiency
Transplantation of Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome
Treatment for severe combined immunodeficiency (SCID) is a medical emergency. A stem cell transplant (immature blood cells that can make other blood cells) from a (MSD) matched sibling donor (brother or sister who is a "match"…
- Trial name
- NCT00152100
- Testing
- Stem cell transplant, Filgrastim, Alemtuzumab, Miltenyi CliniMACS
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- St. Jude Children's Research Hospital