Clinical trials: Primary Immunodeficiency Diseases
Primary Immune Deficiency Disorder
Study to Evaluate the Efficacy, Tolerability and Safety of Octanorm in Patients With Primary Immunodeficiency Diseases
Clinical phase 3 study to evaluate the efficacy, tolerability and safety of subcutaneous human immunoglobulin (octanorm) in patients with primary immunodeficiency diseases.
- Trial name
- NCT03988426
- Testing
- Octanorm
- Where
- 5 sites in Russia
- Volunteers
- 25 people
- Sponsor
- Octapharma
Primary Immunodeficiency (PID)
Gammanorm Quality of Life Study in Immunodeficient Patients Using Rapid Push or Pumps
The purpose of this study is to compare satisfaction (LQI questionnaire, factor I: treatment interference) in PID patients receiving subcutaneous injections of Gammanorm® 165 mg/mL according to the delivery device.
- Trial name
- NCT02180763
- Testing
- Gammanorm
- Where
- 8 sites in France
- Volunteers
- 31 people
- Sponsor
- Octapharma
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
Chronic Lymphocytic Leukemia, Hypogammaglobulinemia
Double-blind, Randomized, Placebo-controlled, Prospective Phase III Study Evaluating Efficacy and Safety of Panzyga in Primary Infection Prophylaxis in Patients With Chronic Lymphocytic Leukemia ("PRO-SID" Study)
Study Evaluating Efficacy and Safety of Panzyga in Primary Infection Prophylaxis in Patients with Chronic Lymphocytic Leukemia
- Trial name
- NCT04502030
- Testing
- Panzyga
- Where
- 84 sites in United States, Czechia and 10 more
- Volunteers
- 247 people
- Sponsor
- Octapharma
Ataxia Telangiectasia
Evaluate the Neurological Effects of EryDex on Subjects With A-T
This is an international, multi-center, randomized, prospective, double-blind, placebo-controlled, Phase 3 study, designed to assess the effect of EryDex (dexamethasone sodium phosphate [DSP] in autologous erythrocytes)…
- Trial name
- NEAT (NCT06193200)
- Testing
- Dexamethasone sodium phosphate
- Where
- 19 sites in United States, Denmark and 7 more
- Volunteers
- 105 people
- Sponsor
- Quince Therapeutics S.p.A.
Hereditary Angioedema, Hereditary Angioedema Type I
Study of Oral Deucrictibant Soft Capsule for On-Demand Treatment of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema
This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled, 2-period, 2-treatment cross-over study to evaluate the efficacy and safety of orally administered deucrictibant compared to placebo for the on-demand…
- Conditions
- Hereditary Angioedema, Hereditary Angioedema Type I, Hereditary Angioedema Type II, Hereditary Angioedema Types I and II
- Trial name
- RAPIDe-3 (NCT06343779)
- Where
- 76 sites in United States, Argentina and 27 more
- Volunteers
- 134 people
- Sponsor
- Pharvaris Netherlands B.V.
Agammaglobulinemia, IgG Deficiency
Safety and Efficacy of Intravenous Immunoglobulin IgPro10 in Patients With Primary Immunodeficiencies (PID)
The objectives of this trial are the assessment of safety and efficacy of IgPro10 in patients with PID, and the assessment of tolerability of high infusion rates.
- Conditions
- Agammaglobulinemia, IgG Deficiency, Common Variable Immunodeficiency
- Trial name
- NCT00322556
- Testing
- Immunoglobulins Intravenous (Human)
- Where
- 10 sites in United States
- Volunteers
- 55 people
- Sponsor
- CSL Behring
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
Hereditary Angioedema
An Open-label Drug-Drug Interaction Study to Evaluate the Effect of BCX7353 on Cytochrome P450 Enzyme Activity Using Probe Substrates
This is an open-label, single sequence study to evaluate the effect of BCX7353 on hepatic and intestinal cytochrome P450 enzymes using probe substrate drugs in healthy subjects.
- Trial name
- NCT02819102
- Testing
- BCX7353 and probes
- Where
- United Kingdom
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- BioCryst Pharmaceuticals
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
Primary Immunodeficiency Diseases (PID), Immune Thrombocytopenic Purpura (ITP)
Safety and Efficacy Study of a 10% Intravenous Immune Globulin Solution in Subjects With Primary Immunodeficiency Disorders
The purpose of this study is to assess the safety and efficacy of Immune Globulin Intravenous (Human), 10% (IGIV 10%) in subjects with primary immunodeficiency disorders.
- Conditions
- Primary Immunodeficiency Diseases (PID), Immune Thrombocytopenic Purpura (ITP), Kawasaki Syndrome
- Trial name
- NCT00157079
- Testing
- Immune Globulin Intravenous (Human), 10%
- Where
- 11 sites in United States
- Volunteers
- 61 people
- Sponsor
- Baxalta now part of Shire
Hereditary Angioedema (HAE)
A Study in Adults With Hereditary Angioedema (HAE) Who Currently Receive Icatibant at Home
The main aim of this study is to describe the treatment patterns, characteristics and outcomes of people with HAE who are currently receiving icatibant in the homecare setting in the United Kingdom (UK).
- Trial name
- NCT05489640
- Testing
- No Intervention
- Where
- United Kingdom
- Volunteers
- 85 people
- Sponsor
- Takeda
Primary Immunodeficiency Diseases (PID)
A Study About the Long-Term Safety of TAK-881 in People With Primary Immunodeficiency Diseases
The main aim of this study is to learn if TAK-881 is safe and well tolerated during long-term use in adults and children with PIDD who are eligible and completed study TAK-881-3001 (NCT05755035).
- Trial name
- NCT06076642
- Testing
- TAK-881, Koru 24 G HIgH Flo Subcutaneous Safety Needle Set (investigational in the US)
- Where
- 25 sites in United States, Czechia and 5 more
- Volunteers
- 53 people
- Sponsor
- Takeda
Primary Immunodeficiency
ASIS for GAMMAGARD in Primary Immunodeficiency
ASIS Corporation (ASIS) has developed the only automatic injection system for delivery of injectable products to it's optimum/right spot, just outside of the fascia, which exists subdermally (between the skin and muscle).
- Trial name
- ASISinPI (NCT02123615)
- Testing
- Gadolinium For abdomen, Gadolinium For lower back, Efficacy of Gammagard subcutaneously at Week 12
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- ASIS Corporation
Hereditary Angioedema (HAE)
A Double-Blind, Multiple Ascending Dose Study to Assess Safety, Tolerability and Pharmacokinetics of DX-2930 in Hereditary Angioedema Participants
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) profile of multiple subcutaneous administrations of DX-2930 across a range of doses in HAE participants.
- Trial name
- NCT02093923
- Testing
- DX-2930
- Where
- 14 sites in United States, Italy and 1 more
- Volunteers
- 38 people
- Sponsor
- Shire
Hereditary Angioedema (HAE)
Efficacy and Safety Study of DX-2930 to Prevent Acute Angioedema Attacks in Patients With Type I and Type II HAE
This is a phase 3, multicenter, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of DX-2930 in preventing acute angioedema attacks in patients with Type I and Type II HAE.
- Trial name
- NCT02586805
- Testing
- DX-2930 - 300mg/2wk, DX-2930 - 300mg/4wk, DX-2930 - 150mg/4wk
- Where
- 41 sites in United States, Canada and 5 more
- Volunteers
- 125 people
- Sponsor
- Shire
Hereditary Angioedema (HAE)
A Survey of Icatibant in Pediatric Participants With Hereditary Angioedema
This study is a survey in Japan of Icatibant subcutaneous injection 30 mg syringe used to treat children or teenagers with acute attacks of hereditary angioedema (HAE).
- Trial name
- NCT05509569
- Testing
- Icatibant
- Where
- Japan
- Volunteers
- 32 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
A Relative Bioavailability Study of Two Formulations of BCX7353
This is an open-label, randomized study to investigate the relative bioavailability of two formulations of BCX7353 and to determine if there is a food effect
- Trial name
- NCT03202784
- Testing
- BCX7353
- Where
- United Kingdom
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- BioCryst Pharmaceuticals
Hereditary Angioedema
A Phase II, Cross-over Clinical Trial Evaluating the Efficacy and Safety of KVD900 (Sebetralstat) in the On-demand Treatment of Angioedema Attacks in Adult Subjects With Hereditary Angioedema Type I or II
This study is a randomized, double-blind, placebo-controlled, phase II, cross-over clinical trial evaluating the efficacy and safety of KVD900, in the treatment of hereditary angioedema attacks in adult subjects.
- Trial name
- NCT04208412
- Testing
- KVD900
- Where
- 25 sites in United States, Austria and 8 more
- Volunteers
- 84 people
- Sponsor
- KalVista Pharmaceuticals, Ltd.