Clinical trials: Primary Immunodeficiency
Severe Combined Immunodeficiency, X-Linked
Lentiviral Gene Therapy for X-linked Severe Combined Immunodeficiency
Severe combined immunodeficiency disorder (SCID) is a heterogeneous group of inherited disorders characterized by a profound reduction or absence of T lymphocyte function, resulting in lack of both cellular and humoral immunity.
- Trial name
- NCT03601286
- Testing
- Lentiviral vector transduced CD34+ cells
- Where
- United Kingdom
- Volunteers
- 5 people
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
Wiskott-Aldrich Syndrome
Gene Therapy for WAS
This is a phase I/II study to evaluate the safety and efficacy of Hematopoietic Stem Cell genetherapy for the Wiskott-Aldrich Syndrome.
- Trial name
- NCT01347346
- Testing
- Autologous CD34 positive cells transduced with a lentiviral vector containing human WAS gene
- Where
- France
- Volunteers
- 5 people
- Sponsor
- Genethon
Hereditary Angioedema
HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)
This Phase 3 study aims to evaluate the efficacy and safety of NTLA-2002 compared to placebo in participants with HAE.
- Trial name
- NCT06634420
- Testing
- NTLA-2002, Normal Saline IV Administration
- Where
- 29 sites in United States, Australia and 7 more
- Volunteers
- 80 people
- Sponsor
- Intellia Therapeutics
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
Primary Immune Deficiency
Study to Evaluate the Pharmacokinetics, Efficacy, Tolerability, and Safety of Subcutaneous Human Immunoglobulin (Newnorm) in Patients With Primary Immunodeficiency Diseases
Prospective, open-label, single-arm, multicentre Phase 3 study to evaluate the pharmacokinetics, efficacy, tolerability, and safety of subcutaneous human immunoglobulin (Newnorm) in patients with primary immunodeficiency diseases
- Trial name
- NCT04640142
- Testing
- Newnorm
- Where
- 23 sites in United States, Germany and 5 more
- Volunteers
- 50 people
- Sponsor
- Octapharma
Shwachman-Diamond Syndrome, SDS
Shwachman-Diamond Syndrome Global Patient Survey and Partnering Platform
The Shwachman-Diamond Syndrome Global Patient Survey and Collaboration Program (SDS-GPS) is an opportunity for patients and their families - from anywhere in the world - to share their experience living with SDS via a safe…
- Conditions
- Shwachman-Diamond Syndrome, SDS, IBMF, Congenital Neutropenia
- Trial name
- SDS-GPS (NCT06999954)
- Where
- United States
- Volunteers
- 8,000 people
- Sponsor
- Shwachman-Diamond Syndrome Alliance Inc
Viral Infection, Hematopoietic Stem Cell Transplantation (HSCT)
Adoptive T Lymphocyte Administration for Chronic Norovirus Treatment in Immunocompromised Hosts
This is a Phase I dose-escalation study to evaluate the safety of norovirus -specific T-cell (NST) therapy for chronic norovirus infection in participants following hematopoietic stem cell transplantation (HSCT) or who are…
- Conditions
- Viral Infection, Hematopoietic Stem Cell Transplantation (HSCT), Primary Immunodeficiency Disorders (PID)
- Trial name
- ATLANTIC (NCT04691622)
- Testing
- Norovirus -specific T-cell (NST) therapy
- Where
- 3 sites in United States
- Volunteers
- 48 people
- Sponsor
- Children's National Research Institute
Primary Immunodeficiency (PID)
Subcutaneous Ig NextGen 16% in PID Patients
This study aims to assess the safety, tolerability, efficacy and pharmacokinetics of Ig NextGen 16% in people with antibody deficiency currently being treated with IntragamP.
- Trial name
- NCT00391131
- Testing
- IgNextGen 16%
- Where
- 12 sites in Australia, New Zealand
- Volunteers
- 35 people
- Sponsor
- CSL Limited
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)
Agammaglobulinemia, IgG Deficiency
Efficacy and Safety of Intravenous Immunoglobulin IVIG-F10 in Patients With Primary Immunodeficiencies (PID)
The purpose of this study is to demonstrate the effect of IVIG-F10 on the prevention of serious bacterial infections in patients with primary immunodeficiency.
- Conditions
- Agammaglobulinemia, IgG Deficiency, Common Variable Immunodeficiency
- Trial name
- NCT00168012
- Testing
- Immunoglobulins Intravenous (Human)
- Volunteers
- 42 people
- Sponsor
- CSL Behring
Leukocyte Adhesion Deficiency Syndrome
Interferon Gamma to Treat Leukocyte Adhesion Deficiency Type I
This study will evaluate the safety and effectiveness of the drug, interferon gamma, in treating leukocyte adhesion deficiency type I (LAD I).
- Trial name
- NCT00001905
- Testing
- Interferon gamma
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Activated PI3K-delta Syndrome, Immunodeficiency Primary
Rapamycin Treatment for Activated Phosphoinositide 3-Kinase δ Syndrome
The purpose of this proposed research is to evaluate the efficacy and safety of the rapamycin therapy in patients with activated phosphoinositide 3-kinase δ syndrome (APDS).
- Trial name
- NCT03383380
- Testing
- Rapamycin
- Where
- 2 sites in China
- Volunteers
- 30 people
- Sponsor
- Children's Hospital of Fudan University
Pneumonia, Pneumocystis Carinii, HIV Infections
A Study of Three Drugs Plus Zidovudine in the Prevention of Infections in HIV-Infected Patients
To evaluate and compare 3 anti-pneumocystis regimens plus zidovudine (AZT) in persons with HIV infection and T4 cell count less than 200 cells/mm3.
- Trial name
- NCT00000991
- Testing
- Pentamidine isethionate, Sulfamethoxazole-Trimethoprim, Dapsone
- Where
- 21 sites in United States, Tanzania
- Volunteers
- 600 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.
- Conditions
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies
- Trial name
- NCT02231710
- Testing
- BPX-501 and Rimiducid
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Bellicum Pharmaceuticals
Primary Immunodeficiency Diseases
Evaluation of an IgG Deficiency Rapid Screening Test: A Performance Study With Primary Immunodeficiency (PID) Patients in Tunisia
To evaluate the usability and utility of the device, % agreement between the PID-RDT and the referent assay (serum/plasma), and % agreement between capillary blood and venous blood samples using the PID-RDT within confirmed PID…
- Trial name
- NCT05621876
- Testing
- IgG deficiency rapid screening test
- Where
- Tunisia
- Volunteers
- 50 people
- Sponsor
- PATH
Hereditary Angioedema (HAE)
Study of C1 Inhibitor (Human) for the Prevention of Angioedema Attacks and Treatment of Breakthrough Attacks in Japanese Subjects With Hereditary Angioedema (HAE)
The purpose of this study is to determine if an investigational treatment is safe and well tolerated when administered by intravenous (IV) infusion in Japanese subjects with HAE.
- Trial name
- NCT02865720
- Testing
- CINRYZE 500 U, CINRYZE 1000 U
- Where
- 10 sites in Japan
- Volunteers
- 8 people
- Sponsor
- Shire
Hereditary Angioedema
A Study to Evaluate the Safety and Effect of Escalating Doses of CINRYZE
The objectives of the study were: 1. To assess the safety and tolerability of escalating doses of CINRYZE. 2. To assess the effect of an escalating dose algorithm for CINRYZE on hereditary angioedema (HAE) attack rates. 3.
- Trial name
- NCT00914966
- Testing
- C1 inhibitor (human) [C1 INH]
- Where
- 11 sites in United States
- Volunteers
- 20 people
- Sponsor
- Shire
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
Hereditary Angioedema (HAE)
Safety and Efficacy Study of CINRYZE for Prevention of Angioedema Attacks in Children Ages 6-11 With Hereditary Angioedema
Primary Objective - To assess the relative efficacy of two dose levels of CINRYZE (500 Units and 1000 Units) administered by intravenous (IV) injection every 3 or 4 days to prevent angioedema attacks in children 6 to 11 years of…
- Trial name
- NCT02052141
- Testing
- CINRYZE 500, CINRYZE 1000
- Where
- 8 sites in United States, Germany and 3 more
- Volunteers
- 12 people
- Sponsor
- Shire
Primary Immunodeficiency Diseases (PID)
Non-Interventional Post-Marketing Safety Study on the Long-Term Safety of HYQVIA (Global)
The purpose of the proposed study is to acquire additional data (including the assessment of anti-rHuPH20 antibodies) on the long-term safety of HYQVIA and to assess the prescribed treatment regimens and treatment administration…
- Trial name
- NCT02593188
- Testing
- HYQVIA
- Where
- 30 sites in United States
- Volunteers
- 264 people
- Sponsor
- Baxalta now part of Shire