Clinical trials: Primary Immune Deficiency Disorders
Stimulant Use (Diagnosis), Hiv
Contingency Management in HIV Care for Both Stimulant Use & ART Adherence
Methamphetamine use and associated sequelae have been rising, and represent a major barrier to successful control of the HIV epidemic.
- Trial name
- CoMBo (NCT06564792)
- Testing
- Contingency management
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- University of California, San Francisco
Hereditary Angioedema (HAE), Angioedema
Oral Deucrictibant for Prophylactic and Acute Treatment in Hereditary Angioedema Patients
To assess the efficacy of prophylactic treatment with deucrictibant extended release (XR) tablet versus placebo in preventing angioedema attacks, and to also assess the efficacy of deucrictibant soft capsules as on-demand…
- Conditions
- Hereditary Angioedema (HAE), Angioedema, Bradykinin-mediated Angioedema, C1 Inhibitor Deficiency
- Trial name
- BK-AE-nC1INH (NCT07046806)
- Testing
- Deucrictibant XR tablet, Deucrictibant 20 mg capsule
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Institute for Asthma and Allergy
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)
Angioedemas, Hereditary
A Study of Lanadelumab in Teenagers and Adults With Hereditary Angioedema (HAE) in Argentina
The main aim of this study is to learn about the safety profile of lanadelumab in teenagers and adults with hereditary angioedema (HAE) in Argentina as part of routine routine practice. This study is about collecting data only.
- Trial name
- NCT04955964
- Where
- Argentina
- Volunteers
- 48 people
- Sponsor
- Takeda
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
Cancer, Breast Cancer
Liquid Biopsy Evaluation and Repository Development at Princess Margaret
The objective of this protocol is to develop an institution-wide liquid biopsy protocol that will establish a common process for collecting blood and corresponding archived tumor specimens for future research studies at the…
- Conditions
- Cancer, Breast Cancer, Lung Cancer, Colon Cancer
- Trial name
- LIBERATE (NCT03702309)
- Where
- Canada
- Volunteers
- 2,500 people, healthy volunteers welcome
- Sponsor
- University Health Network, Toronto
Human Immunodeficiency Virus Transmission
PC4PrEP: Integrating PrEP Into Primary Care
Oral pre-exposure prophylaxis (PrEP) has been proven effective in reducing HIV infection in high-risk men who have sex with men, heterosexually active women and men, and injecting drug users.
- Trial name
- PC4PrEP (NCT03617874)
- Testing
- PC4PrEP- Intervention Clinics, Standard of Care Clinics
- Where
- United States
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- Albert Einstein College of Medicine
Wiskott-Aldrich Syndrome
Romiplostim Treatment for Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome.
The rationale for this retrospective study is to evaluate the efficacy and safety of thrombopoietin-receptor agonist (TPO-RA) romiplostim for reducing thrombocytopenia and bleeding tendency in pediatric participants with…
- Trial name
- NCT04350164
- Testing
- Romiplostim
- Where
- Russia
- Volunteers
- 67 people
- Sponsor
- Federal Research Institute of Pediatric Hematology, Oncology and Immunology
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.
Advanced Solid Tumor, Unresectable Solid Tumor
A Beta-only IL-2 ImmunoTherapY Study
This is a Phase 1/2, multi-center, open-label, dose-escalation and expansion study to evaluate safety and tolerability, PK, pharmacodynamic, and early signal of anti-tumor activity of MDNA11 alone or in combination with a…
- Conditions
- Advanced Solid Tumor, Unresectable Solid Tumor, Clear Cell Renal Cell Carcinoma, Triple Negative Breast Cancer
- Trial name
- ABILITY-1 (NCT05086692)
- Testing
- MDNA11, Pembrolizumab (KEYTRUDA®)
- Where
- 25 sites in United States, Australia and 6 more
- Volunteers
- 160 people
- Sponsor
- Medicenna Therapeutics, Inc.
Adenine Phosphoribosyltransferase Deficiency, AH Amyloidosis
National Registry of Rare Kidney Diseases
The goal of this National Registry is to is to collect information from patients with rare kidney diseases, so that it that can be used for research.
- Conditions
- Adenine Phosphoribosyltransferase Deficiency, AH Amyloidosis, AHL Amyloidosis, AL Amyloidosis
- Trial name
- RaDaR (NCT06065852)
- Where
- United Kingdom
- Volunteers
- 35,000 people
- Sponsor
- UK Kidney Association
HIV Infections
A Study to Monitor Patients With Primary or Early HIV Infection
The purpose of this study is to monitor patients who recently have been infected with HIV in order to learn how their immune systems respond to HIV infection and to study how the virus acts in their bodies.
- Trial name
- NCT00000911
- Where
- United States
- Volunteers
- 1,500 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Hereditary Angioedema (HAE)
A Study of Takhzyro in Teenagers and Adults With Hereditary Angioedema (HAE) in South Korea
Takhzyro is an approved treatment for hereditary angioedema (HAE) in South Korea. HAE is a rare condition which causes sudden swelling under the skin and inside the body, like in the belly, throat, or genitals.
- Trial name
- NCT07445087
- Testing
- No intervention
- Where
- 3 sites in South Korea
- Volunteers
- 35 people
- Sponsor
- Takeda
Ataxia Telangiectasia, ATM Gene Mutation
Effects of Vitamin B3 in Patients With Ataxia Telangiectasia
This clinical trial investigates the effects of nicotinamide riboside (vitamin B3) on the disease course of patients with ataxia telangiectasia.
- Trial name
- NCT03962114
- Testing
- Vitamin B3
- Where
- Netherlands
- Volunteers
- 24 people
- Sponsor
- Radboud University Medical Center
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.
HIV Infections
Preliminary Study of Early, Primary HIV Infection in a High Risk Cohort
The study of early, acute HIV infection is critical to understanding subtype-specific pathophysiologic differences, since up to 50% of acute HIV infections may be incapacitating.
- Trial name
- SEARCH004 (NCT00989144)
- Where
- Thailand
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Thai Red Cross AIDS Research Centre
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.
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
X-linked Severe Combined Immunodeficiency (XSCID)
Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)
This Phase I/II clinical trial will study the safety and potential benefits of gene therapy for up to 35 people aged 2 to 50 with X-linked severe combined immunodeficiency (XSCID), a rare genetic condition that makes it difficult…
- Trial name
- NCT01306019
- Testing
- Campath, Palifermin, Busulfan
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)