Clinical trials: Primary Immune Deficiency Disorders
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
Hereditary Angioedema (HAE)
Observational Safety Study for KALBITOR (Ecallantide) in the Treatment of Acute Attacks of Hereditary Angioedema
The objective of this study is to evaluate the formation of antibodies, the occurence of allergic reactions, and the risk of hypercoagulability and hypocoagulability in patients treated with KALBITOR (ecallantide).
- Trial name
- NCT01059526
- Testing
- ecallantide
- Where
- 40 sites in United States
- Volunteers
- 81 people
- Sponsor
- Shire
Primary Immunodeficiency
A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Immune Globulin (Human) 10% (Gamunex-C) PEG Process (IVIG-PEG) Compared to Gamunex-C in Participants With Primary Humoral Immunodeficiency
The purpose of this study is to demonstrate bioequivalence of IVIG-PEG with Gamunex-C (IVIG-C) at steady-state as determined by comparing total Immunoglobulin G (IgG) area under the concentration-time curve during the defined…
- Trial name
- NCT04561115
- Testing
- Gamunex-C, IVIG-PEG
- Where
- 10 sites in United States
- Volunteers
- 33 people
- Sponsor
- Grifols Therapeutics LLC
Primary Immunodeficiency
Pharmacokinetics, Safety, and Tolerability of Subcutaneous GAMUNEX-C in Pediatric Subjects With Primary Immunodeficiency
The purpose of this open-label study is to evaluate the pharmacokinetics, safety, and tolerability of subcutaneously (SC; under the skin) administered GAMUNEX-C compared to intravenously (IV; through the vein) administered…
- Trial name
- KIDS (NCT01465958)
- Testing
- GAMUNEX-C
- Where
- 4 sites in United States
- Volunteers
- 12 people
- Sponsor
- Grifols Therapeutics LLC
Hereditary Angioedema
Long-Term Follow-Up (LTFU) of Subjects Treated With NTLA 2002
This is a follow-up study of subjects who received NTLA-2002 in a previous clinical trial as an observational evaluation of the long-term effects of the investigational therapy.
- Trial name
- NCT06262399
- Where
- 7 sites in Australia, France and 4 more
- Volunteers
- 100 people
- Sponsor
- Intellia Therapeutics
Hereditary Angioedema (HAE)
A Phase 2 in Adult Subjects With Hereditary Angioedema
A Phase 2 in Adult Subjects with Hereditary Angioedema
- Trial name
- HAE (NCT06846398)
- Testing
- BW-20805
- Where
- 18 sites in United States, China and 4 more
- Volunteers
- 25 people
- Sponsor
- Shanghai Argo Biopharmaceutical Co., Ltd.
Hereditary Angioedema (HAE)
A Survey of Lanadelumab in Participants With Hereditary Angioedema
This study is a survey in Japan of Lanadelumab used to treat people with hereditary angioedema (HAE). The study sponsor will not be involved in how the participants are treated but will provide instructions on how the clinics…
- Trial name
- NCT05397431
- Testing
- Lanadelumab
- Where
- Japan
- Volunteers
- 155 people
- Sponsor
- Takeda
Hereditary Angioedema (HAE), Hereditary Angioedema - Type 1
Phase 3 Extension Study of ADX-324 in Participants With Hereditary Angioedema (HAE)
Study ADX-324-302 is an extension study for participants who complete the Phase 3 ADX-324-301 trial. The extension study will provide information about the safety and efficacy of additional dosing of ADX-324 in participants with…
- Conditions
- Hereditary Angioedema (HAE), Hereditary Angioedema - Type 1, Hereditary Angioedema - Type 2, HAE
- Trial name
- NCT07428499
- Testing
- ADX-324 Dose Level 1, ADX-324 Dose Level 2
- Where
- 48 sites in United States, Argentina and 17 more
- Volunteers
- 90 people
- Sponsor
- ADARx Pharmaceuticals, Inc.
Hereditary Angioedema
A Study of the Burden of Illness and Treatment Patterns in Teenagers and Adults With Hereditary Angioedema
This study is about teenagers and adults with hereditary angioedema (HAE) type I and type II. In this study, the burden of illness means the impact of HAE in terms of long-term health outcomes and the financial cost.
- Trial name
- BOISTERN (NCT04957641)
- Where
- 36 sites in Austria, Belgium and 20 more
- Volunteers
- 221 people
- Sponsor
- Takeda
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
C1 Esterase Inhibitor in Hereditary Angioedema (HAE)(Extension Study)
Hereditary angioedema (HAE) is a rare disorder characterized by congenital lack of functional C1 esterase inhibitor. If not treated adequately, the acute attacks of HAE can be life-threatening and may even result in fatalities…
- Trial name
- NCT00292981
- Testing
- C1 Esterase Inhibitor
- Where
- 14 sites in United States, Canada
- Volunteers
- 57 people
- Sponsor
- CSL Behring
Hereditary Angioedema
An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema
The purpose of this study was to evaluate the safety and efficacy of extended dosing of donidalorsen administered subcutaneously (SC), with alternative dosing and/or dose frequency with donidalorsen in participants with…
- Trial name
- NCT04307381
- Testing
- Donidalorsen
- Where
- 7 sites in United States, Netherlands
- Volunteers
- 20 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Hereditary Angioedema
A Study of Icatibant (TAK-667) in Japanese Children and Teenagers With Acute Attacks of Hereditary Angioedema
The main aim of the study is to check for side effects from icatibant in children and teenagers with hereditary angioedema (HAE).
- Trial name
- NCT04654351
- Testing
- TAK-667
- Where
- 3 sites in Japan
- Volunteers
- 2 people
- Sponsor
- Takeda
Primary Immunodeficiency Diseases (PID)
Efficacy, Safety and Pharmacokinetic of Virchow IVIG in PID Patients
The study duration for individual patients will be up to 55 weeks for the 4-weekly and 3- weekly schedule of the treatment which includes 3 weeks for screening, 48 weeks for Study Medication administration and 4 weeks for…
- Trial name
- VBPL-IVIGP3 (NCT07017036)
- Testing
- IVIG-VImmune, IVIG-Immuglo
- Where
- 2 sites in India
- Volunteers
- 44 people
- Sponsor
- Virchow Group
Bradykinin-mediated Angioedema, Hereditary Angioedema (HAE)
A Study to Evaluate the Safety and Efficacy of Icatibant in Patients With Bradykinin Induced Angioedema
This is a phase IV clinical study. The investigators collect patients with a diagnosis of bradykinin induced angioedema. For a specific study period and prospectively arrange, the eligible patients who has an acute attack of…
- Trial name
- NCT07290855
- Testing
- Icatibant Injection (Icanticure®)
- Where
- Taiwan
- Volunteers
- 5 people
- Sponsor
- Nang Kuang Pharmaceutical Co., Ltd.
Primary Immune Deficiency Disorder
Pharmacokinetics, Efficacy, and Safety Study of RI-002 (IGIV) in Subjects With Primary Immunodeficiency Diseases (PIDD)
This is a Phase III, multicenter, open-label study of RI-002 administered as an intravenous infusion of RI-002 (IGIV) every 21 or 28 days in approximately 60 subjects with Primary Immunodeficiency Diseases (PIDD).
- Trial name
- NCT01814800
- Testing
- RI-002
- Where
- 9 sites in United States
- Volunteers
- 59 people
- Sponsor
- ADMA Biologics, Inc.
Hereditary Angioedema
Study to Determine How BCX4161 is Metabolized and Eliminated by the Body
The purpose of the study is to assess the extent that radioactive dose of BCX4161 taken by mouth ends up in the urine, feces and expired air.
- Trial name
- NCT02218294
- Testing
- BCX4161
- Where
- United Kingdom
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- BioCryst Pharmaceuticals
Hereditary Angioedema
A Study of the Relative Bioavailability of a New Formulation of BCX4161 and the Effect of Food on BCX4161
The purpose of the study is to compare how the body takes up and then eliminates the study drug BCX4161 when it is taken orally as a hard gelatin capsule and as a soft gelatin capsule.
- Trial name
- NCT02125162
- Testing
- BCX4161
- Where
- United Kingdom
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- BioCryst Pharmaceuticals
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
Hereditary Angioedema
EAP for Deucrictibant Immediate-release
This is an expanded access program (EAP) designed to allow access to deucrictibant immediate-release (IR) capsule for eligible participants with hereditary angioedema (HAE) in the United States (US) and US territories who lack…
- Trial name
- NCT07759141
- Testing
- Deucrictibant IR
- Sponsor
- Pharvaris Netherlands B.V.