Clinical trials: Primary Immune Deficiency Disorders
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
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.
Pain
Evaluation of the Tolerability of Switching Subjects on Chronic ATC Opioid Therapy to Buprenorphine HCl Buccal Film
The primary aim of this study is to determine if chronic pain subjects on around-the-clock opioids who are receiving 100 to 220 mg oral morphine sulfate equivalent (MSE) can be safely transitioned on to buprenorphine…
- Trial name
- NCT01871285
- Testing
- Buprenorphine, Oxycodone, Morphine sulfate
- Where
- 3 sites in United States
- Volunteers
- 39 people
- Sponsor
- BioDelivery Sciences International
Hereditary Angioedema (HAE)
A Study of Lanadelumab in Teenagers and Adults With Hereditary Angioedema (HAE)
The main aim of this study is to compare the HAE attacks (both total and those requiring on-demand treatment) in the 12 months before and up to 24 months after initiation of treatment with lanadelumab.
- Trial name
- NCT05469789
- Testing
- No Intervention
- Where
- United Kingdom
- Volunteers
- 50 people
- Sponsor
- Takeda
Primary Immunodeficiency
A Study to Evaluate IGSC 20% Biweekly Dosing in Treatment-Experienced Participants and Loading/Maintenance Dosing in Treatment-Naïve Participants With Primary Immunodeficiency
The purpose of the study is to determine whether biweekly (every 2 weeks) administration of Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography Purified (IGSC 20%) produces a steady-state area under the…
- Trial name
- NCT04566692
- Testing
- IGSC 20%
- Where
- 12 sites in United States
- Volunteers
- 33 people
- Sponsor
- Grifols Therapeutics LLC
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
Severe Combined Immunodeficiency
Safety and Early Efficacy Study of TBX-1400 in Patients With Severe Combined Immunodeficiency
This is a study of stem cell transplantation with TBX-1400 in pediatric subjects with severe combined immunodeficiency (SCID). The donor cells are exposed to a protein that has been shown in the laboratory to improve the ability…
- Trial name
- NCT02860559
- Testing
- TBX-1400
- Where
- 2 sites in Israel
- Volunteers
- 8 people
- Sponsor
- Taiga Biotechnologies, Inc.
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
Immunologic Deficiency Syndromes
A Clinical Study of Intravenous Immunoglobulin
The purpose of this study is to measure the pharmacokinetics, efficacy and safety of Immune Globulin Intravenous (Human) [IGIV], 5% Solution Omr-IgG-am™ in patients with primary immunodeficiency diseases.
- Trial name
- IGIV (NCT00468273)
- Testing
- Immune Globulin Intravenous (Human) Omr-IgG-am IGIV
- Where
- 10 sites in United States, Canada
- Volunteers
- 57 people
- Sponsor
- FFF Enterprises
Hereditary Angioedema (HAE)
Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE
This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of once-daily orally administered deucrictibant extended-release tablet compared to placebo for prophylaxis to…
- Trial name
- CHAPTER-3 (NCT06669754)
- Testing
- Deucrictibant
- Where
- 56 sites in United States, Argentina and 22 more
- Volunteers
- 81 people
- Sponsor
- Pharvaris Netherlands B.V.
Hereditary Angioedema
Hereditary Angioedema Kininogen Assay
A multicenter epidemiological observational study aiming to explore the cleaved high-molecular weight kininogen (cHMWK) including identification and characterization of other metabolite/biomarkers in HAE type 1/2 patients
- Trial name
- HAEKA (NCT04091113)
- Where
- 7 sites in Germany
- Volunteers
- 59 people
- Sponsor
- CENTOGENE GmbH Rostock
WHIM Syndrome
A Retrospective and Prospective Natural History Study of Patients With WHIM Syndrome
This natural history study is a prospective and retrospective, observational study of WHIM patients. WHIM syndrome is a rare, genetic, primary immunodeficiency disorder (a disorder in which the body's immune system does not…
- Trial name
- NCT03087370
- Testing
- No intervention
- Sponsor
- X4 Pharmaceuticals
Hereditary Angioedema
CSL312 (Garadacimab) in the Prevention of Hereditary Angioedema Attacks
This is a multicenter, double-blind, randomized, placebo-controlled, parallel-arm study to investigate the efficacy and safety of subcutaneous administration of CSL312 (garadacimab) in the prophylactic treatment of hereditary…
- Trial name
- NCT04656418
- Testing
- CSL312
- Where
- 28 sites in United States, Canada and 5 more
- Volunteers
- 64 people
- Sponsor
- CSL Behring
Hereditary Angioedema (HAE), Angioedema
A Study to Review the Treatment and Outcomes of Teenagers and Adults With Non-histaminergic Angioedema With Normal C1 Inhibitor in Canada
The main aim of this study is to describe the characteristics of participants, check for number of hereditary angioedema (HAE) cases, their treatment and outcomes.
- Trial name
- PROSPECT (NCT05578417)
- Testing
- No Intervention
- Where
- 6 sites in Canada
- Volunteers
- 60 people
- Sponsor
- Takeda
Primary Immunodeficiency Disease
An Extension Study of TAK-664 for Japanese People With Primary Immunodeficiency Disease
This study is an extension study for participants with primary immunodeficiency disorders who were previously treated with IGSC, 20% in the TAK-664-3001 study.
- Trial name
- NCT04842643
- Testing
- IGSC 20% infusion
- Where
- 8 sites in Japan
- Volunteers
- 12 people
- Sponsor
- Takeda
Primary Immunodeficiency Diseases (PID)
Comparison of Intravenous and Subcutaneous Administration of IGIV, 10% in Primary Immunodeficiency (PID) Subjects
The purpose of this study is to evaluate the tolerability of IGIV, 10% given subcutaneously and the pharmacokinetics of immunoglobulin G (IgG) following subcutaneous (SC) treatment with IGIV, 10% in subjects with primary…
- Trial name
- NCT00546871
- Testing
- Immune Globulin Intravenous (Human), 10%
- Where
- 9 sites in United States
- Volunteers
- 49 people
- Sponsor
- Baxalta now part of Shire
Hereditary Angioedema, Hereditary Angioedema Type I
Dose-ranging Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema
This study evaluates the efficacy of orally administered deucrictibant for the acute treatment of attacks in patients with hereditary angioedema (HAE).
- Conditions
- Hereditary Angioedema, Hereditary Angioedema Type I, Hereditary Angioedema Type II, Hereditary Angioedema Types I and II
- Trial name
- RAPIDe-1 (NCT04618211)
- Testing
- Deucrictibant
- Where
- 36 sites in United States, Bulgaria and 11 more
- Volunteers
- 74 people
- Sponsor
- Pharvaris Netherlands B.V.
Agammaglobulinemia, IgG Deficiency
Efficacy and Safety of Intravenous Immunoglobulin IVIG-F10 in Patients With Primary Immunodeficiencies (PID)
The purpose of this study is to demonstrate the effect of IVIG-F10 on the prevention of serious bacterial infections in patients with primary immunodeficiency.
- Conditions
- Agammaglobulinemia, IgG Deficiency, Common Variable Immunodeficiency
- Trial name
- NCT00168012
- Testing
- Immunoglobulins Intravenous (Human)
- Volunteers
- 42 people
- Sponsor
- CSL Behring
Hereditary Angioedema (HAE)
A Study of Lanadelumab in Children With Hereditary Angioedema (HAE) in Multiple Countries
HAE is a rare condition. It causes sudden swelling under the skin and inside the body, like in the belly, throat, or genitals. This swelling happens because of a temporary leak in blood vessels but does not cause itching or…
- Trial name
- NCT07251933
- Testing
- No intervention
- Where
- 24 sites in Argentina, France and 4 more
- Volunteers
- 37 people
- Sponsor
- Takeda
Hereditary Angioedema (HAE)
Study of BCX7353 as a Treatment for Attacks of Hereditary Angioedema
This 3-part study will evaluate the efficacy and safety of an oral kallikrein inhibitor, BCX7353, in the treatment angioedema attacks in subjects with Type I or II hereditary angioedema (HAE).
- Trial name
- NCT03240133
- Testing
- BCX7353
- Where
- 24 sites in Austria, Denmark and 10 more
- Volunteers
- 58 people
- Sponsor
- BioCryst Pharmaceuticals