Clinical trials: Primary Immune Deficiency Disorders
Hereditary Angioedema
Efficacy and Safety of GNR-038 vs Berinert® in Patients With Hereditary Angioedema
It is a placebo-controlled randomized trial to evaluate the efficacy and safety of GNR-038 in comparison with Berinert® in patients with hereditary angioedema
- Trial name
- NCT04898309
- Testing
- GNR-038, 50 МЕ/ kg, GNR-038, 100 МЕ/ kg, Berinert®, 20 МЕ/ kg
- Where
- 5 sites in Russia
- Sponsor
- AO GENERIUM
Chronic Granulomatous Disease
Analysis of Patients Treated for Chronic Granulomatous Disease Since January 1, 1995
This study is a longitudinal and cross-sectional evaluation of patients with Chronic Granulomatous Disease (CGD) who received or are receiving hematopoietic cell transplantation (HCT) for their disease under a variety of…
- Trial name
- NCT02116764
- Where
- United States
- Volunteers
- 137 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
C1 Esterase Inhibitor Deficiency, Angio Edema
Biomarker for Hereditary AngioEdema Disease
International, multicenter, observational, longitudinal monitoring study to identify, validate and/or monitor Mass Spectrometry (MS)-based biomarker/s for Hereditary Angioedeme (HAE) disease and to test the clinical robustness…
- Conditions
- C1 Esterase Inhibitor Deficiency, Angio Edema, C4 Deficiency, HAE
- Trial name
- BioHAE (NCT03029728)
- Where
- 7 sites in Armenia, Georgia and 5 more
- Volunteers
- 42 people
- Sponsor
- CENTOGENE GmbH Rostock
Severe Combined Immune Deficiency (SCID)
Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus
Background: Severe combined immune deficiency (SCID) is a group of conditions where the immune system does not work properly. The only cure for most SCIDs is a stem cell transplant (getting cells from a donor).
- Trial name
- NCT04370795
- Testing
- Sirolimus, Busulfan, Horse -Anti-thymocyte
- Where
- United States
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
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
Accelerated Phase Chronic Myelogenous Leukemia, Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome
Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure Disorders
This clinical trial is studying how well giving fludarabine phosphate and melphalan together with total-body irradiation followed by donor stem cell transplant works in treating patients with hematologic cancer or bone marrow…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission
- Trial name
- NCT00856388
- Testing
- fludarabine phosphate, melphalan, total-body irradiation
- Where
- United States
- Volunteers
- 62 people
- Sponsor
- Roswell Park Cancer Institute
Common Variable Immunodeficiency (CVID), Primary Immune Regulatory Disorder
Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD)
This is a research protocol that will examine Hematopoietic Stem Cell Transplantation (HSCT) using a reduced conditioning regimen (RIC) with total body Irradiation (TBI) in those diagnosed with Common Variable Immunodeficiency…
- Conditions
- Common Variable Immunodeficiency (CVID), Primary Immune Regulatory Disorder, Immune Dysregulation, DiGeorge Syndrome
- Trial name
- CVID/PIRD (NCT07284641)
- Testing
- Hematopoietic stem cell transplant (HSCT)
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Paul Szabolcs
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
Sickle Cell Disease, Thalassemia
Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells
This is a clinical trial of bone marrow transplantation for patients with the diagnosis of a genetic disease of blood cells that do not have an HLA-matched sibling donor.
- Conditions
- Sickle Cell Disease, Thalassemia, Anemia, Granuloma
- Trial name
- NCT00730314
- Testing
- Hematopoietic stem cell transplantation
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Children's Hospital Los Angeles
HIV Infections
Evaluating the Pharmacokinetics, Feasibility, Acceptability, and Safety of Oral Pre-Exposure Prophylaxis for HIV Prevention During Pregnancy and Postpartum
The purpose of this study was to evaluate the pharmacokinetics, feasibility, acceptability, and safety of a fixed-dose combination of emtricitabine and tenofovir disoproxil fumarate (FTC/TDF) as oral daily pre-exposure…
- Trial name
- NCT03386578
- Testing
- Emtricitabine/Tenofovir Disoproxil Fumarate (FTC/TDF), Behavioral HIV risk reduction package, Enhanced adherence support
- Where
- 7 sites in Malawi, South Africa and 2 more
- Volunteers
- 780 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Hereditary Angioedema, HAE
A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema
This is a Phase 1/2, single-arm, open-label, dose-escalation and dose-expansion study of BMN 331 for the treatment of hereditary angioedema (HAE) due to C1 Esterase Inhibitor (C1-INH) protein deficiency.
- Trial name
- HAErmony-1 (NCT05121376)
- Testing
- Dose 1 of BMN 331, Dose 2 of BMN 331, Dose 3 of BMN 331
- Where
- 16 sites in United States, Australia and 1 more
- Volunteers
- 44 people
- Sponsor
- BioMarin Pharmaceutical
Autophagy, Galectins
Study of Autophagy and the Effects of GALIG Gene Products in HIV-1 Infected Patients Who Are Under Antiretroviral Therapy Since Primary-infection, Chronic Phase, or Never Treated.
Little is known about autophagy during HIV infection. Recently, two different teams reported important dysfunctions of autophagy in HIV-infected patients despite sustained suppressive antiretroviral therapy.
- Conditions
- Autophagy, Galectins, HIV Infections, Highly Active Antiretroviral Therapy
- Trial name
- ATGALIG-HIV (NCT04160455)
- Testing
- expression of a panel
- Where
- France
- Volunteers
- 180 people
- Sponsor
- Centre Hospitalier Régional d'Orléans
Hereditary Angioedema
A Study of Icatibant in Patients With Acute Attacks of Hereditary Angioedema (FAST-3)
This study is being conducted to evaluate the efficacy and safety of icatibant compared to placebo in patients experiencing acute attacks of hereditary angioedema (HAE).
- Trial name
- NCT00912093
- Testing
- Icatibant
- Where
- 64 sites in United States, Australia and 7 more
- Volunteers
- 98 people
- Sponsor
- Shire
HIV Infections, Sexually Transmitted Diseases
Parents Matter!: Interventions to Promote Effective Parent-Child Communication
Parents Matter is a community-based project whose goal it is to develop and test an intervention designed to promote effective parent-child communication about sexuality in order to promote sexual health among adolescents.
- Conditions
- HIV Infections, Sexually Transmitted Diseases, Pregnancy
- Trial name
- NCT00137943
- Testing
- Full Parent communication intervention, Brief Parent Communication Intervention, Control Parent Intervention
- Where
- 3 sites in United States
- Volunteers
- 2,210 people, healthy volunteers welcome
- Sponsor
- Centers for Disease Control and Prevention
Primary Immune Deficiency
Retrospective Chart Review of Subcutaneous IgG Use in Infants
This study is a retrospective chart review of the use of subcutaneous IgG (SCIG) in infants less than two years old.
- Trial name
- NCT01166074
- Testing
- Subcutaneous IgG (SCIG)
- Where
- 5 sites in United States
- Volunteers
- 27 people
- Sponsor
- CSL Behring
Primary Immune Deficiency, COVID
COVID-19 in PID Survey
With the emergence of SARS-CoV-2 and the COVID-19 pandemic, there is an urgent need to understand the impact of infection on immunodeficient individuals.
- Trial name
- COPID19 (NCT04459689)
- Where
- France
- Volunteers
- 200 people
- Sponsor
- Imagine Institute
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
Adenovirus, Primary Immune Deficiency Disorder
Adenovirus-specific Cytotoxic T-lymphocytes for Refractory Adenovirus Infection
Related donor Adenovirus (ADV) specific cytotoxic T cells (CTLs) manufactured with the Miltenyi CliniMACS Prodigy Cytokine Capture System will be administered intravenously in in children, adolescents and young adults with…
- Trial name
- NCT03266627
- Testing
- cytotoxic t-lymphocytes
- Where
- 10 sites in United States
- Volunteers
- 20 people
- Sponsor
- New York Medical College
Hereditary Angioedema
C1 Inhibitor Registry in the Treatment of Hereditary Angioedema (HAE) Attacks
This is a non-interventional treatment Registry of Hereditary Angioedema (HAE) patients treated with C1 inhibitor, either plasma-derived (pdC1INH) or the recombinant human form (rhC1INH / Ruconest), to observe adverse events and…
- Trial name
- NCT01397864
- Testing
- rhC1INH or pdC1INH
- Where
- 17 sites in Bulgaria, Croatia and 11 more
- Volunteers
- 181 people
- Sponsor
- Pharming Technologies B.V.
Hereditary Angioedema (HAE)
Phase 1 Study to Assess the Safety, Tolerability, and Pharmacokinetics of Recombinant Human C1 Esterase Inhibitor in Healthy Adult Subjects
This trial is looking to gain information about the safety and tolerability of an investigational treatment (SHP623) in healthy adult volunteers.
- Trial name
- NCT02663687
- Testing
- Recombinant human C1 esterase inhibitor, SHP623
- Where
- United States
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Shire