Clinical trials: Primary Immune Deficiency Disorders
Cystic Fibrosis, Pseudohypoaldosteronism
Genetic Disorders of Mucociliary Clearance in Nontuberculous Mycobacterial Lung Disease
Healthy volunteers and patients with diseases that involve problems clearing mucus from the lungs will be examined and tested to better understand the reasons for recurring lung infections in these patients and to try to develop…
- Conditions
- Cystic Fibrosis, Pseudohypoaldosteronism, Nontuberculous Mycobacterial Infection, Chronic Granulomatous Disease
- Trial name
- NCT00368446
- Where
- United States
- Volunteers
- 87 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Ataxia-Telangiectasia (A-T)
Cell-Based Approaches For Modeling and Treating Ataxia-Telangiectasia
This research is being done to better understand the causes of the disease Ataxia-Telangiectasia and, in the longer-term, develop new therapies for the disease using stem cells.
- Trial name
- NCT02246491
- Testing
- A-T iPS cell line, Carrier patients iPS cell line
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Johns Hopkins University
HIV Infections
Cyclosporine A in Combination With Abacavir Sulfate, Lamivudine, and Zidovudine and Lopinavir/Ritonavir in HIV
Cyclosporine A (CsA) is a common long-term treatment used to inhibit the immune response in transplant patients who receive donor organs. CsA may also help people with HIV.
- Trial name
- NCT00084149
- Testing
- Abacavir sulfate, Lamivudine, and Zidovudine, Lopinavir/Ritonavir
- Where
- 4 sites in United States
- Volunteers
- 54 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Severe Combined Immunodeficiency
Influences on Female Adolescents' Decisions Regarding Testing for Carrier Status of XSCID
The purpose of this study is to learn what factors influence adolescent girls' decisions regarding testing for carrier status of X-Linked Severe Combined Immunodeficiency (XSCID).
- Trial name
- NCT00006335
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
HIV-1-infection, Cancer
Coronavirus (COVID-19) Outcomes Registries in Immunocompromised Individuals Australia (CORIA)
CORIA is an observational cohort study of immunosuppressed populations who test positive for COVID-19. This includes people living with HIV, cancer, acquired immunodeficiency associated with other immunosuppressive therapy…
- Conditions
- HIV-1-infection, Cancer, Primary Immune Deficiency Disorder, Immunosuppression Disorders
- Trial name
- CORIA (NCT04354818)
- Where
- 11 sites in Australia
- Volunteers
- 27 people
- Sponsor
- Kirby Institute
Chronic Granulomatous Disease, Inflammatory Bowel Disease
Natural History of Intestinal Inflammation in People With Primary Immune Dysregulations
Background: PIDD stands for primary immune dysregulation. It is a general term that includes many different inherited immune system disorders. The immune system is the part of the body that helps fight disease and infection.
- Trial name
- NCT03278912
- Where
- United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
HIV/AIDS
Using Open Contest and Neuro-influence Experiment to Develop and Evaluate PrEP Promotion Messages for High Risk Men
The aim of this study is to examine the utility of neuroimaging technique to evaluate pre-exposure prophylaxis (PrEP) promotion messages for men who have sex with men (MSM) at risk of HIV in Baltimore.
- Trial name
- NCT04166851
- Testing
- PrEP campaign messages developed via open contest, PrEP campaign messages developed via social marketing approach
- Where
- United States
- Volunteers
- 23 people, healthy volunteers welcome
- Sponsor
- Johns Hopkins Bloomberg School of Public Health
Severe Combined Immunodeficiency
Hematopoietic Stem Cell Transplantation (HSCT) for Children With SCID Utilizing Alemtuzumab, Plerixafor & Filgrastim
The goal of this study is to develop a novel approach to hematopoietic stem cell transplantation for children with Severe Combined Immunodeficiency Disease (SCID) that eliminates the use of toxic chemotherapy conditioning and…
- Trial name
- NCT01182675
- Testing
- Transplant Conditioning with Mobilization Only, Transplant Conditioning with Mobilization and Alemtuzumab
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- University of California, San Francisco
Primary Immunodeficiency
Diagnostic Immunization With Rabies Vaccine in Patients With PID
The purpose of this study is to evaluate diagnostic immunization protocol of rabies vaccine for diagnosis the patients with primary immunodeficiency disorders and study humoral and cellular immune response to rabies vaccine in…
- Trial name
- NCT02490956
- Testing
- Verorab® (PVRV; Purified Vero Cell Vaccine)
- Where
- Thailand
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Chulalongkorn University
Candidiasis, Chronic Mucocutaneous
CAMB/MAT2203 in Patients With Mucocutaneous Candidiasis
This is an open-label, dose-titration trial to study the efficacy, safety, and pharmacokinetics of oral cochleate amphotericin B (CAMB) in the treatment of mucocutaneous candidiasis infections in patients who are refractory or…
- Trial name
- CAMB (NCT02629419)
- Testing
- Amphotericin B
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Matinas BioPharma Nanotechnologies, Inc.
Hiv, Hpv
Real-world Effectiveness of HPV Vaccine in Women Living With HIV and Its Impact on Cervical Cancer Screening Accuracies
This study will examine both Human papillomavirus (HPV) vaccine effectiveness and Primary high-risk HPV PHS screening triage strategies in women living with HIV (WLHIV) by partnering with the Pediatric HIV/AIDs Cohort Study…
- Trial name
- LiVes-LLC (NCT06144229)
- Testing
- Self-Collection HPV testing kit from Abbott
- Where
- 9 sites in United States
- Volunteers
- 650 people
- Sponsor
- University of California, Los Angeles
Epstein-Barr Virus Infections, Primary Immune Deficiency Disorder
EBV-specific Cytotoxic T-lymphocytes (CTLs) for Refractory EBV Infection
Related donor Epstein-Barr Virus (EBV) specific cytotoxic T cells (CTLs) manufactured with the Miltenyi CliniMACS Prodigy Cytokine Capture System will be administered in children, adolescents and young adults with refractory EBV…
- Trial name
- NCT03266653
- Testing
- cytotoxic t-lymphocytes
- Where
- 8 sites in United States
- Volunteers
- 20 people
- Sponsor
- New York Medical College
Severe Combined Immunodeficiency Syndrome
Gene Therapy for ADA-SCID
This study investigated the safety and efficacy of different gene therapy approaches for Severe Combined Immunodeficiency (SCID) caused by the deficiency of adenosine deaminase (ADA) enzyme.
- Trial name
- NCT00599781
- Testing
- gene transduced PBL and/or gene transduced HSC
- Volunteers
- 8 people
- Sponsor
- IRCCS San Raffaele
Hereditary Angioedema (HAE)
Subcutaneous CINRYZE With Recombinant Human Hyaluronidase for Prevention of Angioedema Attacks
The primary objectives of the study are to evaluate the safety, tolerability, and efficacy of two doses of CINRYZE with recombinant human hyaluronidase (rHuPH20) administered by subcutaneous (SC) injection to prevent angioedema…
- Trial name
- NCT01756157
- Testing
- CINRYZE with rHuPH20
- Where
- 23 sites in United States, Germany and 2 more
- Volunteers
- 47 people
- Sponsor
- Shire
Primary Immune Deficiency Disease
Safety and Efficacy Study of Flebogamma 5% DIF IGIV in Pediatric Subjects
This is a multi-center, open-label study to assess the efficacy and safety of Flebogamma 5% DIF in the pediatric population.
- Trial name
- NCT00634569
- Testing
- Flebogamma 5% DIF
- Where
- 7 sites in United States
- Volunteers
- 24 people
- Sponsor
- Instituto Grifols, S.A.
Primary Immune Deficiency Disorder
Pharmacokinetics, Efficacy, and Safety Study of RI-002 (IGIV) in Subjects With Primary Immunodeficiency Diseases (PIDD)
This is a Phase III, multicenter, open-label study of RI-002 administered as an intravenous infusion of RI-002 (IGIV) every 21 or 28 days in approximately 60 subjects with Primary Immunodeficiency Diseases (PIDD).
- Trial name
- NCT01814800
- Testing
- RI-002
- Where
- 9 sites in United States
- Volunteers
- 59 people
- Sponsor
- ADMA Biologics, Inc.
Primary Immunodeficiency Disease
High Infusion Rate Study of Immunoglobulin Intravenous (Human) 10% (NewGam)
This was a prospective, open-label, non-controlled, non-randomized multicenter Phase III study of 2 multiple-dose intravenous NewGam regimens (every 3 weeks or every 4 weeks, continuing the patient's infusion interval in the main…
- Trial name
- NGAM-05 (NCT01313507)
- Testing
- NewGam
- Where
- 6 sites in United States
- Volunteers
- 21 people
- Sponsor
- Octapharma
Hereditary Angioedema, HAE
Efficacy and Safety Study of BCX7353 as an Oral Treatment for the Prevention of Attacks in HAE
This is a phase 3, multicenter, 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.
- Trial name
- APeX-2 (NCT03485911)
- Testing
- BCX7353 capsules
- Where
- 47 sites in United States, Austria and 9 more
- Volunteers
- 121 people
- Sponsor
- BioCryst Pharmaceuticals
Ataxia Telangiectasia in Children, Ataxia Telangiectasia
Home-based Complex Intervention for Children With Ataxia Telangiectasia
Ataxia telangiectasia is a rare, genetic and progressive condition with no known cure. Therapies present a mainstream management option and have the potential to offer optimisation of fitness and general health.
- Trial name
- NCT05692622
- Testing
- Whole-body exercise and respiratory exercise
- Where
- United Kingdom
- Volunteers
- 40 people
- Sponsor
- University of Plymouth
SCID, ADA-SCID
Patients Treated for SCID (1968-Present)
Individuals with a past diagnosis of severe combined immune deficiency (including many cases of "leaky SCID", Omenn syndrome, and reticular dysgenesis) who have undergone blood and marrow transplant, gene therapy, or enzyme…
- Conditions
- SCID, ADA-SCID, XSCID, Leaky SCID
- Trial name
- NCT01346150
- Where
- 36 sites in United States, Canada
- Volunteers
- 1,007 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)