Clinical trials: Primary Immune Deficiency Disorders
Hereditary Angioedema
Safety Study in Subjects ≥ 12 Years of Age With Hereditary Angioedema Switching to Garadacimab
This study is designed to evaluate the safety after switching to garadacimab from another prophylactic hereditary angioedema (HAE) treatment (marketed kallikrein [KK] inhibitor or plasma-derived C1-esterase inhibitor…
- Trial name
- NCT06806657
- Testing
- Garadacimab
- Where
- 11 sites in United States, Canada and 1 more
- Volunteers
- 18 people
- Sponsor
- CSL Behring
Ataxia Telangiectasia
Ataxia-telangiectasia: Treating Mitochondrial Dysfunction With Nicotinamide Riboside
Study design: Single arm open-label clinical trial in ataxia-telangiectasia to test the effects of nicotinamide riboside on ataxia scales, immune function, and neurofilament light chain.
- Trial name
- ATNAD (NCT06324877)
- Testing
- Nicotinamide riboside
- Where
- Australia
- Volunteers
- 10 people
- Sponsor
- The University of Queensland
Hereditary Angioedema
A Long-term Study of STAR-0215 in Participants With Hereditary Angioedema
The goal of this trial is to enable the collection of information about long-term safety and clinical activity of STAR-0215 in participants with hereditary angioedema (HAE).
- Trial name
- NCT06007677
- Testing
- STAR-0215
- Where
- 22 sites in United States, Bulgaria and 5 more
- Volunteers
- 56 people
- Sponsor
- Astria Therapeutics, Inc.
HIV Infection
Discontinuation of Primary and Secondary Prophylaxis for Opportunistic Infections in HIV-infected Patients
The purpose of this study is to compare the incidence of opportunistic infections between HIV-infected patients who continue and discontinue primary or secondary prophylaxis for opportunistic infections in whom receiving…
- Trial name
- NCT01392430
- Testing
- Discontinuation of prophylactic drugs i.e. co-trimoxazole, dapsone, fluconazole, itraconazole, azithromycin
- Where
- Thailand
- Volunteers
- 74 people
- Sponsor
- Chiang Mai University
Primary Immunodeficiency
Send-In Sample Collection to Achieve Genetic and Immunologic Characterization of Primary Immunodeficiencies
Background: The immune system helps the body fight infections. Primary immunodeficiency disorders (PIDs) are diseases that make it easier for people to get sick. Many PIDs are inherited.
- Trial name
- NCT03610802
- Where
- 3 sites in United States, Italy and 1 more
- Volunteers
- 3,000 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Primary Immunodeficiency Disorders
The Mechanistic Biology of Primary Immunodeficiency Disorders
Background: Primary immunodeficiency disorders, or PIDs, are diseases that weaken the immune system. This makes it easier for a person to get sick. Some PIDs are mild and may not be diagnosed until later in life.
- Trial name
- NCT03394053
- Where
- 2 sites in United States
- Volunteers
- 1,190 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Coronavirus Infections
Conestat Alfa in the Prevention of Severe SARS-CoV-2 Infection in Hospitalized Patients With COVID-19
The aim of this study is to analyze if administration of conestat alfa for 72 hours in addition to standard of care (SOC) in patients hospitalized with non-critical SARS-CoV-2 pneumonia (WHO Ordinal Scale Score 3 or 4) reduces…
- Trial name
- NCT04414631
- Testing
- Conestat alfa
- Where
- 5 sites in Brazil, Mexico and 1 more
- Volunteers
- 80 people
- Sponsor
- University Hospital, Basel, Switzerland
Lymphoma, Non-Hodgkin, HIV Infections
Phase II Trial of Sequential Chemotherapy and Radiotherapy for AIDS-Related Primary Central Nervous System Lymphoma
To estimate the response rate, overall and disease-free survival, toxicities, factors associated with outcome, and effect on quality of life in patients with AIDS-related primary CNS lymphoma treated with CHOD (cyclophosphamide…
- Trial name
- NCT00000801
- Testing
- Filgrastim, Vincristine sulfate, Doxorubicin hydrochloride
- Where
- 7 sites in United States
- Volunteers
- 33 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Primary Immunodeficiency
Safety and Tolerability of Higher Infusion Parameters of IgPro20 (Hizentra) in Subjects With Primary Immunodeficiency (PID)
This multicenter, open-label, parallel-arm, non-randomized study is designed to evaluate safety and tolerability of higher infusion parameters of IgPro20 in subjects with primary immunodeficiency (PID).
- Trial name
- NCT03033745
- Testing
- IgPro20
- Where
- 12 sites in United States, Canada
- Volunteers
- 49 people
- Sponsor
- CSL Behring
Wiskott- Aldrich Syndrome, ADA Deficient SCID
Molecular and Clinical Studies of Primary Immunodeficiency Diseases
This study will try to identify mutations in the genes responsible for primary immunodeficiency disorders (inherited diseases of the immune system) and evaluate the course of these diseases in patients over time to learn more…
- Trial name
- NCT00006319
- Where
- United States
- Volunteers
- 266 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
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
Severe Combined Immunodeficiency Disease, Severe Primary Immunodeficiency Disorder
Allogeneic SCT Of Pts With SCID And Other Primary Immunodeficiency Disorders
This study is to discover whether children with severe combined immunodeficiency disease (SCID) or other primary immunodeficiency disorder (PID) for which no satisfactory treatment other than stem cell transplantation (SCT)…
- Conditions
- Severe Combined Immunodeficiency Disease, Severe Primary Immunodeficiency Disorder, Undefined T Cell Deficiency Disorder, Wiskott-Aldrick Syndrome
- Trial name
- MASCI (NCT00579137)
- Testing
- Campath -1H, Fludarabine, Anti-CD45
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Baylor College of Medicine
Lymphoproliferative Disorders, Autoimmune Lymphoproliferative
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Inborn Errors of Immunity
Background: During a transplant, blood stem cells from one person are given to someone else. The cells grow into the different cells that make up the immune system. This can cure people with certain immunodeficiencies.
- Conditions
- Lymphoproliferative Disorders, Autoimmune Lymphoproliferative, Immune System Diseases, Common Variable Immunodeficiency
- Trial name
- NCT04339777
- Testing
- Busulfan test dose, Fludarabine, Busulfan
- Where
- United States
- Volunteers
- 66 people
- Sponsor
- National Cancer Institute (NCI)
Chronic Liver Disease
An Observational Study Evaluating Patients With Chronic Liver Diseases Associated With Hepatic Steatosis
Hepatic steatosis may cause inflammation and fibrosis within the liver potentially leading to end-stage liver disease cirrhosis, liver failure and death.
- Trial name
- FALL (NCT05335603)
- Where
- Denmark
- Volunteers
- 335 people, healthy volunteers welcome
- Sponsor
- University of Copenhagen
Primary Immune Deficiency Disorder
Octagam 5% Versus Comparator Post Marketing Trial
Study Design: This is a 2 arm non-interventional trial that will compare the occurrence of adverse drug reactions between Octagam 5% and other marketed IVIG infusion treatments.
- Trial name
- NCT01859754
- Testing
- Octagam 5%, Other marketed IVIG product
- Where
- 27 sites in United States
- Volunteers
- 623 people
- Sponsor
- Octapharma
Hereditary Angioedema (HAE)
Efficacy and Safety of Lanadelumab (SHP643) in Japanese Participants With Hereditary Angioedema (HAE)
The purpose of this phase 3, open-label, multi-center study is to evaluate the safety and efficacy of lanadelumab in Japanese participants with HAE Type I or II.
- Trial name
- NCT04180163
- Testing
- Lanadelumab
- Where
- 12 sites in Japan
- Volunteers
- 12 people
- Sponsor
- Shire
Severe Combined Immunodeficiency, T Cell Lymphocytopenia
Newborn Screening for Severe Combined Immunodeficiency (SCID) in a High-Risk Population
The goal of the proposed research is to establish the validity of a newborn screening method for severe combined immunodeficiency (SCID).
- Trial name
- NCT00845416
- Where
- 2 sites in United States
- Volunteers
- 1,800 people
- Sponsor
- University of California, San Francisco
Hereditary Angioedema, Angioneurotic Edema
Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
Hereditary angioedema ("HAE") is a genetic disorder characterized by sudden recurrent attacks of local swelling (angioedema). These attacks are often painful and disabling, and, in some cases, life-threatening.
- Trial name
- NCT00225147
- Testing
- Recombinant Human C1 Inhibitor
- Where
- Netherlands
- Volunteers
- 77 people
- Sponsor
- Pharming Technologies B.V.
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)
Primary Immune Deficiency
Study of Immune Globulin Intravenous (Human) GC5107 in Pediatric Subjects With Primary Humoral Immunodeficiency
The purpose of this study is to evaluate the pharmacokinetics and safety of Immune Globulin Intravenous (Human) GC5107 in pediatric subjects with Primary Humoral Immunodeficiency (PHID).
- Trial name
- NCT04565015
- Testing
- GC5107
- Where
- 8 sites in United States, Bosnia and Herzegovina and 1 more
- Volunteers
- 24 people
- Sponsor
- GC Biopharma Corp