Clinical trials: Primary Immune Deficiency Disorders
Hereditary Angioedema
HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)
This Phase 3 study aims to evaluate the efficacy and safety of NTLA-2002 compared to placebo in participants with HAE.
- Trial name
- NCT06634420
- Testing
- NTLA-2002, Normal Saline IV Administration
- Where
- 29 sites in United States, Australia and 7 more
- Volunteers
- 80 people
- Sponsor
- Intellia Therapeutics
Hereditary Angioedema (HAE)
Efficacy and Safety Study of DX-88 to Treat Acute Attacks of Hereditary Angioedema (HAE)
The purpose of this study is to determine if a subcutaneous dose of DX-88 (ecallantide; an investigational product) is safe and relieves symptoms of HAE in patients suffering from moderate to severe acute attacks of HAE.
- Trial name
- NCT00262080
- Testing
- ecallantide, Phosphate Buffer Saline (PBS),
- Where
- United States
- Volunteers
- 91 people
- Sponsor
- Shire
Primary Immunodeficiency Disease
Clinical Assessment of Pharmacokinetics, Efficacy, and Safety of 10% IVIg in Pediatric PID Patients (KIDCARES10)
The purpose of this study is to assess efficacy, safety and pharmacokinetics of Kedrion Immunoglobulin 10% (KIg10) in pediatric patients with Primary Immunodeficiency Disease (PID).
- Trial name
- KIDCARES10 (NCT04944979)
- Testing
- Kedrion IVIG 10%
- Where
- 21 sites in United States, Hungary and 4 more
- Volunteers
- 30 people
- Sponsor
- Kedrion S.p.A.
Ataxia-Telangiectasia, Ataxia-Telangiectasia (A-T)
A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T)
A pivotal, randomized, double-blind, placebo-controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of Ataxia-Telangiectasia (A-T).
- Trial name
- NCT06673056
- Testing
- N-Acetyl-L-Leucine
- Where
- 11 sites in United States, Germany and 4 more
- Volunteers
- 60 people
- Sponsor
- IntraBio Inc
CVI - Common Variable Immunodeficiency
A Study to Evaluate the Benefit of RUCONEST® in Subjects Who Experience ADRs Related to IVIG Infusions
Patients receiving intravenous immunoglobulin (IVIG) therapy for primary immunodeficiency and neurologic conditions may experience adverse drug reactions (ADRs). The mechanism of the ADR is unknown.
- Trial name
- NCT03576469
- Testing
- C1-esterase inhibitor [recombinant] (C1-INH-R)
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- IMMUNOe Research Centers
Hereditary Angioedema
A Study Observing US Patients With HAE Type I or II Who Take Icatibant to Treat HAE Attacks
Patients with HAE Type I or II who enroll in the study are asked to complete a patient diary when they experience an HAE attack.
- Trial name
- ICTOS (NCT07009262)
- Testing
- Inapplicable
- Where
- United States
- Volunteers
- 88 people
- Sponsor
- KalVista Pharmaceuticals, Ltd.
Hereditary Angioedema (HAE)
Post Study Access of CSL312 (Garadacimab) for Pediatric Participants With Hereditary Angioedema Who Have Completed the CSL312_3003 Study
This protocol for post study access allows pediatric participants (2-11 years old at the time of consent) with HAE who have completed study CSL312_3003 (NCT05819775) to continue treatment with CSL312 for routine prevention of HAE…
- Trial name
- NCT07159464
- Testing
- CSL312
- Sponsor
- CSL Behring
Intracranial Aneurysm
Diversion-p64 Post Market Clinical Follow-Up Study to Assess Safety and Effectiveness of the p64 Flow Modulation Device
The purpose of this study is to assess safety and effectiveness of the p64 Flow Modulation Device.
- Trial name
- NCT02600364
- Where
- 26 sites in Argentina, Belgium and 8 more
- Volunteers
- 450 people
- Sponsor
- Phenox GmbH
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
Hereditary Angioedema
Safety and Effectiveness of Sebetralstat (KVD900) for Short-Term Prophylaxis Before Procedures in People With Hereditary Angioedema (KONTROL)
This is a Phase 4, prospective, open-label trial to evaluate the safety and effectiveness of sebetralstat when used for STP for a qualifying procedures in patients 12 years of age or older with hereditary angioedema (HAE).
- Trial name
- NCT07654829
- Testing
- KVD900 600 mg
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- KalVista Pharmaceuticals, Ltd.
Hereditary Angioedema (HAE)
Donidalorsen Treatment in Children With Hereditary Angioedema
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of donidalorsen in pediatric participants with hereditary angioedema (HAE) Type I (HAE-1) or Type II (HAE-2).
- Trial name
- NCT07298447
- Testing
- Donidalorsen
- Where
- 18 sites in United States, Australia and 3 more
- Volunteers
- 20 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Immune Thrombocytopenia
The Efficacy and Safety of Huaiqihuang Granule in Children With Chronic Primary Immune Thrombocytopenia
This is a multisite, double-blind, randomized, placebo-controlled, and parallel study designed to evaluate the clinical efficacy and safety of Huaiqihuang granule for treatment to children with chronic primary immune…
- Trial name
- ITP (NCT03201848)
- Testing
- Huaiqihuang Granule
- Where
- 14 sites in China
- Volunteers
- 216 people
- Sponsor
- Qidong Gaitianli Medicines Co., Ltd
Primary Immunodeficiency Diseases (PID)
Study to Determine the Dose of Recombinant Human Hyaluronidase Needed to Infuse a Full Dose of IGIV Subcutaneously
The purpose of the study is to determine the feasibility of infusing a full 4-week dose of Immune Globulin Intravenous (Human), 10% in a single subcutaneous site and the amount of recombinant human hyaluronidase needed to infuse…
- Trial name
- NCT00782106
- Testing
- Recombinant human hyaluronidase + immune globulin intravenous
- Where
- 3 sites in United States
- Volunteers
- 11 people
- Sponsor
- Baxalta now part of Shire
Hereditary Angioedema (HAE)
A Study About the Diagnosis and Management of Hereditary Angioedema (HAE) in Egypt
Hereditary angioedema (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.
- Trial name
- EXPLORHAE (NCT07218393)
- Testing
- No intervention
- Where
- 7 sites in Egypt
- Volunteers
- 100 people
- Sponsor
- Takeda
Hematologic Malignancies
A Single-arm Safety Study of Transplantation Using Umbilical Cord Blood and Human Placental-derived Stem Cells From Partially Matched Related Donors in Persons With Certain Malignant Blood Diseases and Non-malignant Disorders
To investigate the safety of partially matched related human placental-derived stem cells (HPDSC) administered in conjunction with umbilical cord blood (UCB) stem cells from the same donor in subjects with various malignant or…
- Trial name
- HPDSC (NCT00596999)
- Testing
- UCB and HPDSC
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Celgene Corporation
Primary Immune Deficiency (PID)
Ig NextGen 10% in Patients With Primary Immune Deficiency (PID)
This study aims to assess the safety, tolerability and pharmacokinetics of Ig NextGen 10% in patients with primary immune deficiency currently being treated with Intragam P.
- Trial name
- NCT00405184
- Testing
- IntragamP, Ig NextGen 10%
- Where
- 4 sites in Australia
- Volunteers
- 19 people
- Sponsor
- CSL Limited
Hereditary Angioedema (HAE)
A Study of Lanadelumab in Teenagers and Adults With Hereditary Angioedema (HAE) in the Kingdom of Saudi Arabia
Hereditary angioedema (HAE) is a rare condition. It causes sudden swelling under the skin and inside the body, like in the belly, throat, or genitals.
- Trial name
- NCT07263685
- Testing
- No intervention
- Where
- Saudi Arabia
- Volunteers
- 50 people
- Sponsor
- Takeda
Primary Immune Deficiency Disorder
Study to Monitor Subcutaneous Human Immunoglobulin Administered at Modified Dosing Regimens in Patients With Primary Immunodeficiency Diseases
CLINICAL PHASE 3 STUDY TO MONITOR THE SAFETY, TOLERABILITY, AND EFFICACY OF SUBCUTANEOUS HUMAN IMMUNOGLOBULIN (CUTAQUIG®) ADMINISTERED AT MODIFIED DOSING REGIMENS IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES
- Trial name
- NCT03939533
- Testing
- CUTAQUIG
- Where
- 18 sites in United States
- Volunteers
- 64 people
- Sponsor
- Octapharma
Hereditary Angioedema (HAE)
Safety and Efficacy Study of Repeated Doses of DX-88 (Ecallantide) to Treat Attacks of Hereditary Angioedema (HAE)
The purpose of this study is to evaluate the efficacy and safety of repeated doses of ecallantide in the treatment of acute attacks of hereditary angioedema and to allow HAE patients continued access to ecallantide.
- Trial name
- NCT00456508
- Testing
- ecallantide
- Where
- 41 sites in United States, Canada and 1 more
- Volunteers
- 147 people
- Sponsor
- Shire
Angioedema
Subcutaneous Treatment With Icatibant for Acute Attacks of Hereditary Angioedema
The purpose of this study is to assess the efficacy and safety of Icatibant, a bradykinin antagonist in the treatment of acute cutaneous and/or abdominal attacks in patients with hereditary angioedema (HAE).
- Trial name
- NCT00097695
- Testing
- Icatibant
- Where
- United States
- Volunteers
- 84 people
- Sponsor
- Shire