Clinical trials: Primary Immune Deficiency Disorders
Hereditary Angioedema (HAE)
A Study of Icatibant for Acute Attacks of Hereditary Angioedema in Japanese Participants
The objective of this study is to evaluate the efficacy, pharmacokinetics (PK), and safety of icatibant for the treatment of acute attacks in Japanese participants with type I or type II hereditary angioedema (HAE).
- Trial name
- NCT03888755
- Testing
- Icatibant
- Where
- 8 sites in Japan
- Volunteers
- 8 people
- Sponsor
- Shire
Primary Immunodeficiency Disease
Clinical and Laboratory Online Patient- and Research Database for Primary Immunodeficiencies in Switzerland
A Swiss national, multi-centre, online patient and research database will be created, using the existing ESID database server system.
- Trial name
- NCT01150240
- Where
- 6 sites in Switzerland
- Volunteers
- 800 people
- Sponsor
- University of Zurich
Hereditary Angioedema (HAE)
A Study of Lanadelumab (Takhzyro) and Icatibant (Firazyr®) in Persons With HAE in China
The Chinese health authority has approved lanadelumab to prevent Hereditary Angioedema (HAE) attacks in persons of 12 years and older. It has also approved icatibant to treat acute HAE attacks in persons 2 years and older.
- Trial name
- NCT06346899
- Testing
- No intervention
- Where
- 13 sites in China
- Volunteers
- 115 people
- Sponsor
- Takeda
Primary Immune Deficiency Disorder
Study of Octanorm Subcutaneous IG in Patients With PID
This is an open-label phase III study with a 12-week wash-in/wash-out period followed by a 12-month efficacy period. The main goals of the study are to assess the efficacy of octanorm in preventing serious bacterial infections…
- Trial name
- NCT01888484
- Testing
- octanorm 16.5%
- Where
- 24 sites in United States, Canada and 5 more
- Volunteers
- 75 people
- Sponsor
- Octapharma
Hereditary Angioedema
Patient Registry to Evaluate the Real-world Safety of Ruconest®
This is a prospective, real-world, observational patient registry for patients with HAE who are receiving treatment with Ruconest for HAE.
- Trial name
- NCT03697187
- Testing
- rhC1INH
- Where
- United States
- Volunteers
- 152 people
- Sponsor
- Pharming Technologies B.V.
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
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)
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
JAK1 GOF, STAT1 GOF
Upadacitinib in Treatment of JAK/STAT Pathway Disorders With Activating Mutations
This study focuses on a genetic condition that affects the Janus Kinase-Signal Transducer and Activator of Transcription (JAK-STAT) immune signaling pathway.
- Conditions
- JAK1 GOF, STAT1 GOF, STAT3 GOF, STAT5B GOF
- Trial name
- SAFE-JAKi (NCT07670156)
- Testing
- Upadacitinib
- Where
- 3 sites in United States
- Volunteers
- 30 people
- Sponsor
- Lisa Satter
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
Wiskott-Aldrich Syndrome
A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome
This is an open-label, single arm study to evaluate the cryopreserved formulation of OTL-103 Gene Therapy. OTL-103 consists of autologous CD34+ hematopoietic stem cells in which the gene encoding for the Wiskott-Aldrich Syndrome…
- Trial name
- NCT03837483
- Testing
- OTL-103
- Where
- 2 sites in United States, Italy
- Volunteers
- 10 people
- Sponsor
- Fondazione Telethon
Common Variable Immunodeficiency, Agammaglobulinemia
Specific IgG Antibody in Patients With Primary Antibody Deficiencies Treated With Subcutaneous Immunoglobulin
Objective: Measure serum IgG antibody to Streptococcus pneumoniae serotypes 1, 3, 5, 6B, 9V e 14, Haemophilus influenzae type b and tetanus toxoid in patients with primary antibody deficiencies who were treated with subcutaneous…
- Trial name
- NCT00661401
- Testing
- gammaglobulin
- Where
- Brazil
- Volunteers
- 5 people
- Sponsor
- Federal University of São Paulo
Human Immunodeficiency Virus (HIV), Sexually Transmitted Disease (STD)
Steering Together in a New Direction: Reducing the Risk of HIV/STD Among African American Men
African American men have by far the highest rates of HIV in the US, but there are few randomized controlled trials (RCTs) of interventions to dissuade heterosexually active African American men from engaging sexual risk…
- Trial name
- STAND (NCT02572401)
- Testing
- STAND HIV Risk Reduction Intervention, Text Messaging Intervention
- Where
- United States
- Volunteers
- 391 people, healthy volunteers welcome
- Sponsor
- University of Pennsylvania
Ataxia Telangiectasia
Ataxia-telangiectasia: Treating Mitochondrial Dysfunction With a Novel Form of Anaplerosis
Study design: Parallel group, placebo-controlled, dose-escalation each 2 months for 12 months. Dose based on percent (%) of calculated caloric intake.
- Trial name
- A-TC7 (NCT04513002)
- Testing
- Triheptanoin
- Where
- Australia
- Volunteers
- 30 people
- Sponsor
- The University of Queensland
Obesity, Immune Deficiency
Immunoglobulin G Therapy Dose Optimization
The overall goal of this proposal is to investigate effects of obesity on pharmacokinetics of immunoglobulin G (IgG) and to develop strategies for optimization of dosing of IgG in obese patients.
- Trial name
- NCT04818177
- Testing
- Institutional standard intravenous immune globulin treatment
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Rutgers, The State University of New Jersey
Hereditary Angioedema (HAE)
A Study With Lanadelumab in Persons With Hereditary Angioedema (HAE) in Poland
The main aim of this study is to learn about how many persons with HAE type I or type II are attack-free when treated with lanadelumab in real life, how many attacks occur and how many of these attacks need rescue treatment and…
- Trial name
- CHOPIN (NCT05147181)
- Where
- 14 sites in Poland
- Volunteers
- 48 people
- Sponsor
- Takeda
Primary Immunodeficiency Disease, Secondary Immunodeficiency
Safety, Tolerability, Patient Satisfaction and Cost of 16.5% Subcutaneous Immunoglobulin (Cutaquig®) Treatment
Patients with primary or secondary immunodeficiency disease who have developed adverse reactions to products available on the market such as Cuvitru® (Shire), Hizentra® (CSL Behring) or 10% Gammunex® (Grifols), may benefit from…
- Trial name
- NCT03677557
- Testing
- 16,5% Cutaquig
- Where
- Canada
- Volunteers
- 30 people
- Sponsor
- Ottawa Hospital Research Institute
Severe Combined Immunodeficiency
Stem Cell Gene Therapy to Treat X-Linked Severe Combined Immunodeficiency (XSCID)
This is a clinical trial of gene therapy for X-linked severe combined immunodeficiency (XSCID), a genetic disease caused by defects in a protein called the common gamma chain, which is normally on the surface of immune cells…
- Trial name
- NCT00028236
- Testing
- Gene-Transduced Autologous CD34+ Stem Cells
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Invasive Pneumococcal Disease, Recurrent Isolated, 1, Invasive Pneumococcal Disease, Recurrent Isolated, 2
What is the Incidence of an Immune Disorder in Children With Invasive Pneumococcal Disease (IPD)?
This is a multicentre prospective audit to determine the incidence of immunodeficiency in children with IPD. Aims and/or research question of the project 1.
- Conditions
- Invasive Pneumococcal Disease, Recurrent Isolated, 1, Invasive Pneumococcal Disease, Recurrent Isolated, 2, Invasive Pneumococcal Disease, Protection Against, Primary Immunodeficiency
- Trial name
- NCT03815357
- Testing
- Referral to Immunology
- Where
- 6 sites in Australia, New Zealand
- Volunteers
- 380 people, healthy volunteers welcome
- Sponsor
- Murdoch Childrens Research Institute
Hereditary Angioedema, HAE
12-Week Safety and Efficacy Study of BCX4161 as an Oral Prophylaxis Against HAE Attacks
This study will evaluate the safety and efficacy of an oral treatment, BCX4161, in preventing acute attacks in participants with hereditary angioedema (HAE).
- Trial name
- OPuS-2 (NCT02303626)
- Testing
- BCX4161
- Where
- 40 sites in United States, Belgium and 6 more
- Volunteers
- 110 people
- Sponsor
- BioCryst Pharmaceuticals