Clinical trials: Primary Immunodeficiency
Hereditary Angioedema
Open-Label C1 Esterase Inhibitor (C1INH-nf) for the Prevention of Acute Hereditary Angioedema (HAE) Attacks
The study objective was to evaluate the safety and efficacy of prophylactic use of C1INH-nf for the prevention of acute HAE attacks.
- Trial name
- CHANGE 3 (NCT00462709)
- Testing
- C1 esterase inhibitor [human] (C1INH-nf)
- Where
- 48 sites in United States
- Volunteers
- 146 people
- Sponsor
- Shire
Primary Immunodeficiency Disease
Study to Investigate Efficacy, Safety and Pharmacokinetics of BT595 in Subjects With PID
This Phase III clinical study is to test efficacy, safety and pharmacokinetics of BT595 in treating patients with Primary Immunodeficiency (PID)
- Trial name
- NCT02810444
- Testing
- IgG Next Generation (BT595)
- Where
- 19 sites in United States, Germany and 3 more
- Volunteers
- 81 people
- Sponsor
- Biotest
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
Accelerated Phase Chronic Myelogenous Leukemia, Acute Undifferentiated Leukemia
Fludarabine and Total-Body Irradiation Followed By Donor Stem Cell Transplant and Cyclosporine and Mycophenolate Mofetil in Treating HIV-Positive Patients With or Without Cancer
This clinical trial studies the side effects and best dose of giving fludarabine and total-body irradiation (TBI) together followed by a donor stem cell transplant and cyclosporine and mycophenolate mofetil in treating human…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Acute Undifferentiated Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission
- Trial name
- NCT00112593
- Testing
- fludarabine phosphate, total-body irradiation, peripheral blood stem cell transplantation
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Fred Hutchinson Cancer Center
Hereditary Angioedema (HAE)
Open-label, Single-arm Study to Assess the Pharmacokinetics, Safety, and Tolerability of a Single Subcutaneous Dose of Icatibant in Healthy Japanese Volunteers
This is a single-dose study to evaluate the pharmacokinetics, safety, and tolerability of icatibant administered to adult Japanese subjects.
- Trial name
- NCT02045264
- Testing
- Icatibant (30 mg)
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Shire
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.
Hereditary Angioedema (HAE)
Efficacy and Safety Study of DX-88 to Treat Acute Attacks of Hereditary Angioedema (HAE)
The purpose of this study is to determine if a subcutaneous dose of DX-88 (ecallantide; an investigational product) is safe and relieves symptoms of HAE in patients suffering from moderate to severe acute attacks of HAE.
- Trial name
- NCT00262080
- Testing
- ecallantide, Phosphate Buffer Saline (PBS),
- Where
- United States
- Volunteers
- 91 people
- Sponsor
- Shire
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies
This phase II trial studies how well giving fludarabine phosphate, melphalan, and low-dose total-body irradiation (TBI) followed by donor peripheral blood stem cell transplant (PBSCT) works in treating patients with hematologic…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
- Trial name
- NCT01529827
- Testing
- fludarabine phosphate, melphalan, total-body irradiation
- Where
- United States
- Volunteers
- 94 people
- Sponsor
- Roswell Park Cancer Institute
Hereditary Angioedema (HAE)
Efficacy and Safety Study of DX-2930 to Prevent Acute Angioedema Attacks in Patients With Type I and Type II HAE
This is a phase 3, multicenter, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of DX-2930 in preventing acute angioedema attacks in patients with Type I and Type II HAE.
- Trial name
- NCT02586805
- Testing
- DX-2930 - 300mg/2wk, DX-2930 - 300mg/4wk, DX-2930 - 150mg/4wk
- Where
- 41 sites in United States, Canada and 5 more
- Volunteers
- 125 people
- Sponsor
- Shire
Thalassemia, Sickle Cell Disease
Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation
Allogeneic blood and marrow transplantation remains the only viable cure for children who suffer from many serious non-malignant hematological diseases. Transplantation, however, carries a high risk of fatal complications.
- Conditions
- Thalassemia, Sickle Cell Disease, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome
- Trial name
- NCT01319851
- Testing
- Alefacept
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Emory University
Hereditary Angioedema
A Phase II, Cross-over Clinical Trial Evaluating the Efficacy and Safety of KVD900 (Sebetralstat) in the On-demand Treatment of Angioedema Attacks in Adult Subjects With Hereditary Angioedema Type I or II
This study is a randomized, double-blind, placebo-controlled, phase II, cross-over clinical trial evaluating the efficacy and safety of KVD900, in the treatment of hereditary angioedema attacks in adult subjects.
- Trial name
- NCT04208412
- Testing
- KVD900
- Where
- 25 sites in United States, Austria and 8 more
- Volunteers
- 84 people
- Sponsor
- KalVista Pharmaceuticals, Ltd.
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)
Primary Immunodeficiency (PID)
Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy (EU Extension Study)
This study is a continuation of the study ZLB06_001CR with the objective of assessing efficacy, tolerability, safety of IgPro, as well as long-term health-related quality of life in patients with PID.
- Trial name
- NCT00751621
- Testing
- IgPro20
- Where
- 13 sites in France, Germany and 6 more
- Volunteers
- 40 people
- Sponsor
- CSL Behring
Hereditary Angioedema (HAE)
A Study of Lanadelumab (SHP643) in Chinese Participants With Hereditary Angioedema (HAE)
The main aim of this study is to evaluate the safety of lanadelumab in Chinese participants with HAE. Participants will be treated with lanadelumab for 26 weeks.
- Trial name
- NCT05460325
- Testing
- Lanadelumab
- Where
- 4 sites in China
- Volunteers
- 20 people
- Sponsor
- Takeda
ADA-SCID, Adenosine Deaminase Deficiency
EZN-2279 in Patients With ADA-SCID
The purpose of this study is to evaluate the safety, efficacy, and pharmacokinetics of EZN-2279 in patients with ADA-deficient combined immunodeficiency currently being treated with Adagen.
- Conditions
- ADA-SCID, Adenosine Deaminase Deficiency, Severe Combined Immunodeficiency
- Trial name
- NCT01420627
- Testing
- EZN-2279, Adagen
- Where
- 6 sites in United States
- Volunteers
- 7 people
- Sponsor
- Leadiant Biosciences, Inc.
Primary Immunodeficiency Diseases (PID)
Efficacy, Safety, Tolerability, Immunogenicity and Pharmacokinetic Evaluation of HYQVIA in Pediatric PIDD Subjects
The purpose of the study is to acquire additional data on efficacy, safety, tolerability, immunogenicity, pharmacokinetic (PK) and other parameters of HYQVIA in pediatric (age ≥ 2 to <16 years) participants with primary…
- Trial name
- NCT03277313
- Testing
- HYQVIA
- Where
- 19 sites in United States
- Volunteers
- 44 people
- Sponsor
- Baxalta now part of Shire
Primary Immune Deficiency Disorder
Study to Evaluate the Efficacy, Tolerability and Safety of Octanorm in Patients With Primary Immunodeficiency Diseases
Clinical phase 3 study to evaluate the efficacy, tolerability and safety of subcutaneous human immunoglobulin (octanorm) in patients with primary immunodeficiency diseases.
- Trial name
- NCT03988426
- Testing
- Octanorm
- Where
- 5 sites in Russia
- Volunteers
- 25 people
- Sponsor
- Octapharma
Primary Care Appointment Keeping
Intervention to Retain HIV-positive Patients in Medical Care
PROJECT OVERVIEW This research is a collaboration between the Centers for Disease Control and Prevention (CDC), the Health Resources and Services Administration (HRSA), and six university-affiliated HIV clinics in the United…
- Trial name
- Phase II (NCT01537367)
- Testing
- Enhanced contact plus behavioral skills, Enhanced contact only, Standard of Care
- Where
- 6 sites in United States
- Volunteers
- 1,838 people
- Sponsor
- Centers for Disease Control and Prevention
HIV Infections
Love Infection: A Dramatized Story Intervention (Telenovela/Soap Opera) for HIV Prevention
Purpose: To test the feasibility, acceptability of the IA intervention (four telenovela episodes), assessment of the mechanisms of action (self-efficacy, narrative engagement, and emotional elicitation) and conduct a randomized…
- Trial name
- IA (NCT05358366)
- Testing
- Infeccion de Amor (Love Infection)
- Where
- United States
- Volunteers
- 58 people, healthy volunteers welcome
- Sponsor
- University of North Carolina, Chapel Hill
Hereditary Angioedema, Hereditary Angioedema Type I
Dose-ranging Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema
This study evaluates the efficacy of orally administered deucrictibant for the acute treatment of attacks in patients with hereditary angioedema (HAE).
- Conditions
- Hereditary Angioedema, Hereditary Angioedema Type I, Hereditary Angioedema Type II, Hereditary Angioedema Types I and II
- Trial name
- RAPIDe-1 (NCT04618211)
- Testing
- Deucrictibant
- Where
- 36 sites in United States, Bulgaria and 11 more
- Volunteers
- 74 people
- Sponsor
- Pharvaris Netherlands B.V.