Clinical trials: Primary Immune Deficiency Disorder
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 Immunodeficiency
Pharmacokinetics, Safety, and Tolerability of Subcutaneous GAMUNEX-C in Pediatric Subjects With Primary Immunodeficiency
The purpose of this open-label study is to evaluate the pharmacokinetics, safety, and tolerability of subcutaneously (SC; under the skin) administered GAMUNEX-C compared to intravenously (IV; through the vein) administered…
- Trial name
- KIDS (NCT01465958)
- Testing
- GAMUNEX-C
- Where
- 4 sites in United States
- Volunteers
- 12 people
- Sponsor
- Grifols Therapeutics LLC
Hereditary Angioedema
C1 Esterase Inhibitor in Hereditary Angioedema (HAE)(Extension Study)
Hereditary angioedema (HAE) is a rare disorder characterized by congenital lack of functional C1 esterase inhibitor. If not treated adequately, the acute attacks of HAE can be life-threatening and may even result in fatalities…
- Trial name
- NCT00292981
- Testing
- C1 Esterase Inhibitor
- Where
- 14 sites in United States, Canada
- Volunteers
- 57 people
- Sponsor
- CSL Behring
Hereditary Angioedema
An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema
The purpose of this study was to evaluate the safety and efficacy of extended dosing of donidalorsen administered subcutaneously (SC), with alternative dosing and/or dose frequency with donidalorsen in participants with…
- Trial name
- NCT04307381
- Testing
- Donidalorsen
- Where
- 7 sites in United States, Netherlands
- Volunteers
- 20 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Hereditary Angioedema
A Study of Icatibant (TAK-667) in Japanese Children and Teenagers With Acute Attacks of Hereditary Angioedema
The main aim of the study is to check for side effects from icatibant in children and teenagers with hereditary angioedema (HAE).
- Trial name
- NCT04654351
- Testing
- TAK-667
- Where
- 3 sites in Japan
- Volunteers
- 2 people
- Sponsor
- Takeda
Opioid Use Disorder, HIV Infections
Combination Primary Care and Prevention Services for Women Who Inject Drugs and Exchange Sex in Seattle, Washington
The investigators will implement a non-randomized observational clinical trial that will include a pop-up clinic for women who inject drugs (WWID) near venues for exchange sex and drug use in north Seattle.
- Conditions
- Opioid Use Disorder, HIV Infections, Chlamydia, Gonorrhea
- Trial name
- NCT06187064
- Testing
- Mobile Van Clinic
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- University of Washington
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.
Growth Failure, X-linked Severe Combined Immunodeficiency (XSCID)
Treatment for Growth Failure in Patients With X-Linked Severe Combined Immunodeficiency: Phase 2 Study of Insulin-Like Growth Factor-1
This study will evaluate the safety and effectiveness of insulin-like growth factor-1 (IGF-1) to treat patients with X-linked severe combined immunodeficiency (XSCID).
- Conditions
- Growth Failure, X-linked Severe Combined Immunodeficiency (XSCID), Growth Hormone Resistence
- Trial name
- NCT00490100
- Testing
- Increlex
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Severe Combined Immunodeficiency
Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector
Researchers are working on ways to treat SCID patients who don't have a matched brother or sister. One of the goals is to avoid the problems that happen with stem cell transplant from parents and unrelated people, such as repeat…
- Trial name
- NCT01129544
- Testing
- Gene transfer
- Where
- 3 sites in United States
- Volunteers
- 8 people
- Sponsor
- David Williams
Meningococcal Vaccine
Trial to Describe the Safety, Tolerability, and Immunogenicity of Trumenba When Administered to Immunocompromised Participants ≥10 Years of Age
The aim of this study is to evaluate the safety, tolerability, and immunogenicity of 2 doses of Trumenba® (on a 0- and 6-month schedule) in immunocompromised participants by functionally assessing antibody production in asplenic…
- Trial name
- NCT04893811
- Testing
- Trumenba
- Where
- 14 sites in Czechia, Poland and 1 more
- Volunteers
- 53 people, healthy volunteers welcome
- Sponsor
- Pfizer
Hereditary Angioedema
A Study to Evaluate the Safety and Pharmacology of Subcutaneous Administration of CINRYZE With Recombinant Human Hyaluronidase
The objectives of the study are to: 1. Evaluate the safety and tolerability of subcutaneously administered CINRYZE with recombinant human hyaluronidase (rHuPH20) in subjects with hereditary angioedema (HAE) who previously…
- Trial name
- NCT01426763
- Testing
- CINRYZE with rHuPH20
- Where
- 4 sites in United States
- Volunteers
- 12 people
- Sponsor
- Shire
HIV, AIDS
Effectiveness of a Smoking Cessation Algorithm Integrated Into HIV Primary Care
To determine the efficacy of an algorithm designed to recommend smoking cessation-related pharmacotherapy options to the primary care providers of smokers living with HIV/AIDS.
- Conditions
- HIV, AIDS, Smoking Cessation
- Trial name
- NCT03670316
- Testing
- Algorithm Treatment, Quitline only
- Where
- United States
- Volunteers
- 371 people
- Sponsor
- University of Alabama at Birmingham
Hereditary Angioedema
C1 Esterase Inhibitor (C1INH-nf) for the Prevention of Acute Hereditary Angioedema (HAE) Attacks
The study objective was to determine the safety and efficacy of C1INH-nf for the prevention of acute HAE attacks.
- Trial name
- NCT01005888
- Testing
- C1 esterase inhibitor [human] (C1INH-nf)
- Where
- 16 sites in United States
- Volunteers
- 26 people
- Sponsor
- Shire
Non-Neoplastic Hematologic and Lymphocytic Disorder
Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders
This phase II clinical trial studies how well treosulfan and fludarabine phosphate with or without low dose radiation before donor stem cell transplantation works in treating patients with nonmalignant (noncancerous) diseases.
- Trial name
- NCT00919503
- Testing
- Allogeneic Bone Marrow Transplantation, Anti-Thymocyte Globulin, Cyclosporine
- Where
- 6 sites in United States
- Volunteers
- 98 people
- Sponsor
- Fred Hutchinson Cancer Center
HIV Infection
The Effect of Raltegravir on HIV Decay During Primary and Chronic Infection
The purpose of this study is to measure the decay characteristics of HIV in the blood of patients after taking a combination of anti-HIV drugs, which includes a new class of anti-HIV drug, an integrase inhibitor.
- Trial name
- PINT (NCT00641641)
- Testing
- Tenofovir + emtricitabine + raltegravir.
- Where
- 4 sites in Australia
- Volunteers
- 16 people
- Sponsor
- Kirby Institute
Bone Marrow Failure Syndrome, Thalassemia
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum…
- Conditions
- Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia
- Trial name
- NCT03513328
- Testing
- Thiotepa--single daily dose, Thiotepa--escalated dose
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Florida
Hereditary Angioedema
Human C1 Esterase Inhibitor (C1-INH) in Subjects With Acute Abdominal or Facial Hereditary Angioedema (HAE) Attacks
HAE is a rare disorder characterized by functional C1 esterase inhibitor deficiency. If not treated adequately, the acute attacks of HAE can be life-threatening and may even result in fatalities, especially in case of swelling of…
- Trial name
- NCT00168103
- Testing
- C1 Esterase Inhibitor
- Where
- 36 sites in United States, Argentina and 13 more
- Volunteers
- 126 people
- Sponsor
- CSL Behring
Hemophagocytic Lymphohistiocytosis, X-Linked Lymphoproliferative Disorders
Stem Cell Transplant for Immunologic or Histiocytic Disorders
This study tests the clinical outcomes of a preparative regimen of fludarabine (FLU), anti-thymocyte globulin (ATG)/or Campath, and melphalan; followed by hematopoietic stem cell transplant, and a post transplant regimen of…
- Conditions
- Hemophagocytic Lymphohistiocytosis, X-Linked Lymphoproliferative Disorders, Chediak-Higashi Syndrome, Griscelli Syndrome
- Trial name
- NCT00176865
- Testing
- Stem Cell Transplant, Fludarabine, Melphalan
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
HIV Infections
Anti-HIV Medications for People Recently Infected With HIV
It is not known if anti-HIV treatment for recently infected patients improves long-term patient prognosis. The purpose of this study is to determine if a one year course of anti-HIV medications slows progression of HIV disease in…
- Trial name
- NCT00106171
- Testing
- Highly active antiretroviral therapy (HAART)
- Where
- 5 sites in United States, Canada
- Volunteers
- 113 people
- Sponsor
- Johns Hopkins Bloomberg School of Public Health
Wiskott-Aldrich Syndrome, Thrombocytopenia
Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients
The purpose of this project is to describe the pathophysiology of thrombocytopenia and bleeding in patients with Wiskott-Aldrich Syndrome (WAS) and determine the response to thrombopoietic agents in vitro and in vivo.
- Conditions
- Wiskott-Aldrich Syndrome, Thrombocytopenia, Bleeding
- Trial name
- WAS (NCT00909363)
- Testing
- Promacta, blood drawing in patients with WAS, blood drawing in healthy controls
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Weill Medical College of Cornell University