Clinical trials: Primary Immune Deficiency Disorders
Primary Immunodeficiency
Efficacy, Pharmacokinetics, Safety, and Tolerability of IGSC 20% in Subjects With Primary Immunodeficiency
Approximately 60 subjects will be enrolled in order to have approximately 20 adult subjects and 20 pediatric subjects treated with subcutaneously administered Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography…
- Trial name
- NCT02806986
- Testing
- IGSC 20%
- Where
- 23 sites in Australia, Czechia and 7 more
- Volunteers
- 61 people
- Sponsor
- Grifols Therapeutics LLC
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
Primary Immunodeficiency Diseases (PID)
Gammagard Liquid and rHuPH20 in PID
The purpose of the study is to develop a subcutaneous treatment option for participants with Primary Immunodeficiency Diseases (PID) that allows an administration of Immune Globulin Intravenous (Human), 10% at the same frequency…
- Trial name
- NCT00814320
- Testing
- Recombinant human hyaluronidase (rHuPH20)+ immune globulin intravenous (IGIV)
- Where
- 14 sites in United States, Canada
- Volunteers
- 89 people
- Sponsor
- Baxalta now part of Shire
Primary Immunodeficiency Diseases (PID)
Tolerability and Safety of Immune Globulin Subcutaneous Solution (IGSC) and rHuPH20 in PID
The original purpose of the study is to assess the long-term safety, tolerability, and practicability of the subcutaneous (SC) treatment with Immune Globulin Subcutaneous Solution (IGSC), 10% facilitated with recombinant human…
- Trial name
- NCT01175213
- Testing
- SC treatment with IGSC, 10% with rHuPH20 followed by SC/IGSC, 10% only (safety), SC treatment with IGSC, 10% with rHuPH20 followed by IV/IGSC, 10% only (safety), IV treatment with IGSC, 10%
- Where
- 11 sites in United States
- Volunteers
- 66 people
- Sponsor
- Baxalta now part of Shire
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
Hereditary Angioedema (HAE)
Efficacy and Safety of BCX7353 to Prevent Angioedema Attacks in Subjects With Hereditary Angioedema
This 3-part study will evaluate the safety and efficacy of an oral treatment, BCX7353, in preventing angioedema attacks in subjects with hereditary angioedema (HAE).
- Trial name
- APeX-1 (NCT02870972)
- Testing
- BCX7353
- Where
- 24 sites in Australia, Austria and 9 more
- Volunteers
- 75 people
- Sponsor
- BioCryst Pharmaceuticals
Hereditary Angioedema
A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous CINRYZE Administration
The objectives of the study are to: 1. Evaluate the safety and tolerability of CINRYZE administered by subcutaneous injection in subjects with hereditary angioedema 2.
- Trial name
- NCT01095497
- Testing
- CINRYZE
- Where
- 7 sites in United States
- Volunteers
- 26 people
- Sponsor
- Shire
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially…
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndrome
- Trial name
- NCT02065869
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 4 sites in Italy, United Kingdom
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Immunologic Deficiency Syndrome
A Trial of the Pharmacokinetics, Safety, and Tolerability of Subcutaneous Gamunex® in Primary Immunodeficiency
This study will compare the blood level of Gamunex in patients. Patients will take it as an injection under the skin or in a vein. The study will compare how safe and tolerable the two methods are in the patients.
- Trial name
- NCT00389324
- Testing
- Immune Globulin Intravenous (Human)
- Where
- 8 sites in United States, Canada
- Volunteers
- 35 people
- Sponsor
- Grifols Therapeutics LLC
Hereditary Angioedema (HAE)
Long-term Safety and Efficacy Study of DX-2930 (SHP643) to Prevent Acute Angioedema Attacks in Patients With Type I and Type II HAE
This study is an open-label, long term safety and efficacy study to evaluate DX-2930 in preventing acute angioedema attacks in participants with Type I and Type II HAE.
- Trial name
- NCT02741596
- Testing
- DX-2930
- Where
- 43 sites in United States, Canada and 5 more
- Volunteers
- 212 people
- Sponsor
- Shire
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
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
Human Immunodeficiency Virus, Acquired Immunodeficiency Syndrome
Self-Test Strategies and Linkage Incentives to Improve ART and PrEP Uptake in Men
This is a randomized controlled trial to test a combination behavioral and biomedical interventions to improve the HIV prevention and care cascades in a population of mobile men in a high priority setting (fishermen in Kenya).
- Conditions
- Human Immunodeficiency Virus, Acquired Immunodeficiency Syndrome, HIV Infections, Sexually Transmitted Diseases, Viral
- Trial name
- NCT04772469
- Testing
- Provision of multiple self-tests, Small monetary incentives, Use of SMS/text reminders to motivate adherence to ART or PrEP
- Where
- Kenya
- Volunteers
- 934 people, healthy volunteers welcome
- Sponsor
- University of California, San Francisco
Primary Immune Deficiency
Study of Subcutaneous Immunoglobulin in Patients With PID Requiring IgG Replacement Therapy
The objective of this study is to assess the efficacy, tolerability, safety and pharmacokinetics of IgPro20 in patients with primary humoral immunodeficiency (PID).
- Trial name
- NCT00419341
- Testing
- Human Normal Immunoglobulin for Subcutaneous Administration
- Where
- 13 sites in United States
- Volunteers
- 49 people
- Sponsor
- CSL Behring
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.
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
Hereditary Angioedema (HAE)
Safety and Efficacy Study of Repeated Doses of DX-88 (Ecallantide) to Treat Attacks of Hereditary Angioedema (HAE)
The purpose of this study is to evaluate the efficacy and safety of repeated doses of ecallantide in the treatment of acute attacks of hereditary angioedema and to allow HAE patients continued access to ecallantide.
- Trial name
- NCT00456508
- Testing
- ecallantide
- Where
- 41 sites in United States, Canada and 1 more
- Volunteers
- 147 people
- Sponsor
- Shire
Immunodeficiency Syndrome, Non-Cancer Diagnosis
Alemtuzumab, Fludarabine Phosphate, and Total-Body Irradiation Followed by a Donor Stem Cell Transplant in Treating Patients With Immunodeficiency or Other Nonmalignant Inherited Disorders
This phase II trial studies fludarabine phosphate and total-body irradiation with or without alemtuzumab followed by donor stem cell transplant to see how well it works in treating patients with immunodeficiency or other…
- Trial name
- NCT00553098
- Testing
- Alemtuzumab, Allogeneic Bone Marrow Transplantation, Allogeneic Hematopoietic Stem Cell Transplantation
- Where
- 6 sites in United States
- Volunteers
- 29 people
- Sponsor
- Fred Hutchinson Cancer Center
Hereditary Angioedema
Efficacy, Safety and Immunogenicity Study of Recombinant Human C1 Inhibitor for the Treatment of Acute HAE Attacks
This study is being conducted to confirm the efficacy, safety, and immunogenicity of recombinant human C1 inhibitor (rhC1INH) at a dose of 50 U/kg when used for the treatment of acute angioedema attacks in Hereditary Angioedema…
- Trial name
- NCT01188564
- Testing
- rhC1INH
- Where
- 27 sites in United States, Bulgaria and 9 more
- Volunteers
- 75 people
- Sponsor
- Pharming Technologies B.V.
Severe Combined Immunodeficiency
SCID Bu/Flu/ATG Study With T Cell Depletion
This is a pilot clinical trial of hematopoietic stem cell transplantation for patients with a diagnosis of Severe Combined Immune Deficiency (SCID) who do not have an HLA-matched sibling donor.
- Trial name
- NCT02127892
- Testing
- unrelated BM with T cell depletion, unrelated cord blood, haplo BM with T cell depletion
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Neena Kapoor, M.D.