Clinical trials: Primary Immunodeficiency Diseases
Risk Behavior, HIV/AIDS
Testing an Integrated Bio-Behavioral Primary HIV Prevention Intervention Among High-Risk People Who Use Drugs
This study will evaluate the efficacy and cost-effectiveness of CHRP-BB - an integrated bio-behavioral approach that incorporates the use of PrEP - with an evidence-based behavioral approach aimed at enhancing PrEP adherence and…
- Conditions
- Risk Behavior, HIV/AIDS, Medication Adherence, Substance Abuse
- Trial name
- CHRP-BB (NCT03282890)
- Testing
- CHRP-BB
- Where
- United States
- Volunteers
- 237 people
- Sponsor
- University of Connecticut
Primary Ciliary Dyskinesia, Kartagener Syndrome
Characterizing the Upper Airway Manifestations in Primary Ciliary Dyskinesia and Primary Immunodeficiencies
Though common, morbidities related to upper airway disease in primary ciliary dyskinesia (PCD) and primary immunodeficiencies (PID) have not been fully characterized.
- Conditions
- Primary Ciliary Dyskinesia, Kartagener Syndrome, Primary Immune Deficiency
- Trial name
- NCT04919018
- Where
- 4 sites in United States, Canada
- Volunteers
- 159 people
- Sponsor
- University of North Carolina, Chapel Hill
Hereditary Angioedema, HAE
Open-label, Long-term Safety Study of Avoralstat in Subjects With Hereditary Angioedema
This is an open-label study designed to evaluate the long-term safety of prophylactic avoralstat (500 mg three times daily) when given to approximately 150 patients with hereditary angioedema (HAE) for a duration of up to 72…
- Trial name
- OPuS-4 (NCT02670720)
- Testing
- avoralstat
- Where
- 3 sites in Belgium, France
- Volunteers
- 6 people
- Sponsor
- BioCryst Pharmaceuticals
Hereditary Angioedemas
A Study Investigating the Effectiveness and Safety of Garadacimab for Treating Patients With Hereditary Angioedema (HAE)
This is a multinational, multicenter, prospective, observational cohort study of patients with HAE in the real-world setting. The study will include patients newly initiating garadacimab in routine clinical practice.
- Trial name
- GREAT (NCT07001280)
- Testing
- No intervention
- Where
- 22 sites in United States, Austria and 3 more
- Volunteers
- 200 people
- Sponsor
- CSL Behring
Hereditary Angioedema
Pharmacokinetics of C1 Esterase Inhibitor in Hereditary Angioedema Subjects
The study objective was to describe the pharmacokinetics (PK) of one or two doses of C1 esterase inhibitor (C1INH-nf) in hereditary angioedema (HAE) subjects who were not experiencing an HAE attack.
- Trial name
- NCT00432510
- Testing
- C1 esterase inhibitor [human] (C1INH-nf)
- Where
- 6 sites in United States
- Volunteers
- 27 people
- Sponsor
- Shire
Ataxia-Telangiectasia (A-T)
Expanded Access Program (EAP) of Levacetylleucine for Ataxia-Telangiectasia (A-T)
This expanded access program provides investigational levacetylleucine to patients with Ataxia-Telangiectasia who are not eligible for clinical trials and have no satisfactory alternative therapies.
- Trial name
- NCT07380165
- Testing
- Levacetylleucine
- Sponsor
- IntraBio Inc
Alcohol Dependence
Reducing Alcohol Dependence Among HIV-Positive Individuals
The purpose of this study is to compare the effects of interventions for drinking-reduction and antiretroviral therapy (ART) adherence among HIV-positive primary care patients.
- Trial name
- NCT02501057
- Testing
- Clinician's Guide, Enhanced Motivational Interviewing, Enhanced Clinician's Guide
- Volunteers
- 133 people
- Sponsor
- Research Foundation for Mental Hygiene, Inc.
Chronic Pain, Depression Disorders
Analysis of the Therapeutic and Clinical Profile of Cannabinoid Treatments: A Real-World Study
What is the purpose of this study? The use of medicinal cannabis has grown significantly in Brazil. However, because many patients use these products through "compassionate use" (when traditional treatments haven't worked), there…
- Conditions
- Chronic Pain, Depression Disorders, Anxiety Disorders, Quality of Life
- Trial name
- CANAREAL (NCT07362407)
- Testing
- Participants are followed while using cannabis-based products (such as CBD or THC oils, extracts, or isolates) as prescribed by their own physicians for therapeutic purposes.
- Where
- Brazil
- Volunteers
- 384 people
- Sponsor
- Santa Catarina Federal University
Hereditary Angioedema (HAE)
A Single Increasing Dose Study to Assess Safety and Tolerability of DX-2930 in Healthy Subjects
The purpose of this study is to assess the safety and tolerability of the study drug (DX-2930) administered subcutaneously in healthy subjects. Subjects will be randomized to receive DX-2930 or placebo.
- Trial name
- NCT01923207
- Testing
- DX-2930
- Where
- United States
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Shire
Acute Bacterial Infections, Acute Viral Infections
300 Antibody Diagnostic Test Kit
For the development of a Point of Care IVD test kit for acute phase disease detection against a variety of bacterial and viral infections.
- Trial name
- Antibody 300 (NCT01646411)
- Where
- United States
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- The Plasma Source
HIV/AIDS and Infections, Drug Abuse
Preventing HIV/STI in Urban Adolescents Via an mHealth Primary Care Intervention
The study evaluates the preliminary efficacy of an innovative mobile-health (mHealth) intervention (hereon referred to as S4E) to improve human immunodeficiency virus and sexually transmitted infection testing and reduce HIV/STI…
- Conditions
- HIV/AIDS and Infections, Drug Abuse, Adolescent Behavior, Drug Usage
- Trial name
- NCT03368456
- Testing
- S4E, Usual Care
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- University of Michigan
PIDs Linked to PI3K
Long-term Safety and Efficacy of Leniolisib in PIDs With Immune Dysregulation
This is an open-label extension (OLE) study to extend treatment to patients with primary immunodeficiency (PID) disorders linked to phosphoinositide 3-kinase delta signaling who participated in a prior study of leniolisib, LE…
- Trial name
- NCT06990529
- Testing
- Leniolisib
- Where
- 5 sites in United States, Spain and 1 more
- Volunteers
- 12 people
- Sponsor
- Pharming Technologies B.V.
X-Linked Agammaglobulinemia, X-Linked Hyper IgM Syndrome
Study of Genetic and Molecular Defects in Primary Immunodeficiency Disorders
OBJECTIVES: I. Identify the molecular defects responsible for primary immunodeficiency disorders. II. Explore the mutations within each syndrome to better understand the genetics of these disorders. III.
- Conditions
- X-Linked Agammaglobulinemia, X-Linked Hyper IgM Syndrome, Wiskott-Aldrich Syndrome, Leukocyte Adhesion Deficiency Syndrome
- Trial name
- NCT00004341
- Where
- United States
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
HIV Infections, AIDS
The CORE Buprenorphine Project - An HIV Primary Care Program Demonstration
The goal of this study is to develop and evaluate an innovative model of care to better serve patients who are both HIV-infected and opioid-dependent.
- Conditions
- HIV Infections, AIDS, Opiate Dependence, Post-Traumatic Stress Disorders
- Trial name
- BUP (NCT00227357)
- Testing
- On-site - buprenorphine/naloxone (Suboxone), Off-site - methadone or no agonist
- Where
- United States
- Volunteers
- 101 people
- Sponsor
- Ruth M. Rothstein CORE Center
Hereditary Angioedema
Safety Study in Subjects ≥ 12 Years of Age With Hereditary Angioedema Switching to Garadacimab
This study is designed to evaluate the safety after switching to garadacimab from another prophylactic hereditary angioedema (HAE) treatment (marketed kallikrein [KK] inhibitor or plasma-derived C1-esterase inhibitor…
- Trial name
- NCT06806657
- Testing
- Garadacimab
- Where
- 11 sites in United States, Canada and 1 more
- Volunteers
- 18 people
- Sponsor
- CSL Behring
Ataxia Telangiectasia (AT)
Conjugate Pneumococcal Vaccine in Ataxia Telangiectasia (AT)
Ataxia Telangiectasia (AT) is an autosomal recessive inherited condition caused by mutations in the ATM gene1. Patients suffer from neuro-degenerative problems, usually commencing in the second year of life, and affecting…
- Trial name
- NCT00656409
- Testing
- Conjugated pneumococcal vaccine (Prevenar)
- Where
- United Kingdom
- Volunteers
- 30 people
- Sponsor
- Institute of Child Health
Hereditary Angioedema
PK Subtrial in Adolescent Patients With HAE Type I or II Participating in the KVD900-302 Trial
This is a multicenter pharmacokinetic (PK) subtrial to investigate the PK profile of KVD900 (sebetralstat) in adolescent patients 12 to 17 years of age with Hereditary Angioedema (HAE) type I or II.
- Trial name
- NCT05511922
- Testing
- KVD900 600 mg, Drug: KVD900 300 mg
- Where
- 61 sites in United States, Australia and 15 more
- Volunteers
- 11 people
- Sponsor
- KalVista Pharmaceuticals, Ltd.
Hereditary Angioedema (HAE)
A Study in Teenagers and Adults With Hereditary Angioedema (HAE) Type I or Type II Who Use Lanadelumab as Long-Term Prophylaxis
The main aims of this study are to learn how many people with HAE Type I or Type II are attack-free when treated with lanadelumab in real life.
- Trial name
- INTEGRATED (NCT04861090)
- Where
- 20 sites in Austria, France and 2 more
- Volunteers
- 207 people
- Sponsor
- Takeda
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
Primary Immunodeficiency Diseases (PID)
A Study of Immune Globulin Subcutaneous (Human), 20% Solution (IGSC, 20%) in Japanese Participants With Primary Immunodeficiency Diseases (PID)
In this study, Japanese participants with primary immunodeficiency diseases were treated with Immune Globulin Subcutaneous (Human), 20% solution, (IGSC, 20%).
- Trial name
- NCT04346108
- Testing
- Immune Globulin Intravenous (IGIV), Immune Globulin Subcutaneous, 20% Solution (IGSC, 20%)
- Where
- 8 sites in Japan
- Volunteers
- 17 people
- Sponsor
- Baxalta now part of Shire