Clinical trials: Primary Immune Deficiency Disorders
Primary Immune Deficiency Disease
Safety and Efficacy Study of Flebogamma 5% DIF IGIV in Pediatric Subjects
This is a multi-center, open-label study to assess the efficacy and safety of Flebogamma 5% DIF in the pediatric population.
- Trial name
- NCT00634569
- Testing
- Flebogamma 5% DIF
- Where
- 7 sites in United States
- Volunteers
- 24 people
- Sponsor
- Instituto Grifols, S.A.
Abdominal Pain, Functional Abdominal Pain
Epidemiological Analysis for Hereditary Angioedema Disease
An international, multicenter, epidemiological, observational study investigating the prevalence of Hereditary Angioedema (HAE) disease among participants with recurrent episodes of abdominal pain of no obvious etiology.
- Trial name
- EHA (NCT03558009)
- Where
- 35 sites in Germany, Italy and 4 more
- Volunteers
- 2,318 people
- Sponsor
- CENTOGENE GmbH Rostock
Melanoma, Non-small Cell Lung Cancer
A Study of CDX-1140 (CD40) as Monotherapy or in Combination in Patients With Advanced Malignancies
This is a study to determine the maximum tolerated dose (MTD) for CDX-1140 (CD40 antibody), either alone or in combination with CDX-301 (FLT3L), pembrolizumab, or chemotherapy and to further evaluate its tolerability and efficacy…
- Conditions
- Melanoma, Non-small Cell Lung Cancer, Breast Cancer, Gastric Cancer
- Trial name
- NCT03329950
- Testing
- CDX-1140, CDX-301, pembrolizumab
- Where
- 11 sites in United States
- Volunteers
- 132 people
- Sponsor
- Celldex Therapeutics
Hereditary Angioedema (HAE)
A European Post-Authorisation Observational Study Of Patients With Hereditary Angioedema
This observational (non-interventional) study is being conducted to characterize the safety and use of CINRYZE in routine clinical practice when administered for (1) routine prevention of angioedema attacks, (2) pre-procedure…
- Trial name
- NCT01541423
- Where
- 20 sites in Belgium, France and 4 more
- Volunteers
- 83 people
- Sponsor
- Shire
Ataxia Telangiectasia
N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T)
This is a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of Ataxia-Telangiectasia (A-T).
- Trial name
- NCT03759678
- Testing
- IB1001
- Where
- 4 sites in United States, Germany and 2 more
- Volunteers
- 17 people
- Sponsor
- IntraBio Inc
Primary Immunodeficiency
Study of ProMetic BioTherapeutics Immune Globulin Intravenous (Human) 10%
Phase 3 multicenter, open-label study of safety, tolerability, efficacy, and pharmacokinetics (PK) of ProMetic's Immune Globulin Intravenous (Human) 10%, the investigational medicinal product [IMP]), in Adults and Children with…
- Trial name
- NCT02269163
- Testing
- Immune Globulin Intravenous, Prometic's Immune Globulin Intravenous 10%
- Where
- 13 sites in United States
- Volunteers
- 82 people
- Sponsor
- Prometic Biotherapeutics, Inc.
Hereditary Angioedema
Treatment of Angioedema Attacks in Pediatric (Ages 2-11) Post-Trial and Naive Patients With HAE With Sebetralstat
The sebetralstat Early Access Program (EAP) provides early access to the investigational medicinal product (IMP) sebetralstat to eligible and approved Hereditary Angioedema (HAE) pediatric (ages 2-11) post-trial and naïve…
- Trial name
- NCT07216378
- Testing
- Sebetralstat
- Where
- 6 sites in United States
- Sponsor
- KalVista Pharmaceuticals, Ltd.
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.
Wiskott-Aldrich Syndrome
Targeted Literature Review and Subject Interviews in Wiskott-Aldrich Syndrome (WAS)
WAS is a rare primary immune deficiency disease caused by genetic mutation and is more common in males than females. The purpose of this study is to understand experiences of WAS subjects and caregivers to identify important…
- Trial name
- NCT03399461
- Testing
- Patient Interview guide, Caregiver interview guide, Sociodemographic questionnaire
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- GlaxoSmithKline
Primary Immune Deficiency
Study to Evaluate the Pharmacokinetics, Efficacy, Tolerability, and Safety of Subcutaneous Human Immunoglobulin (Newnorm) in Patients With Primary Immunodeficiency Diseases
Prospective, open-label, single-arm, multicentre Phase 3 study to evaluate the pharmacokinetics, efficacy, tolerability, and safety of subcutaneous human immunoglobulin (Newnorm) in patients with primary immunodeficiency diseases
- Trial name
- NCT04640142
- Testing
- Newnorm
- Where
- 23 sites in United States, Germany and 5 more
- Volunteers
- 50 people
- Sponsor
- Octapharma
Hereditary Angioedema
A Single Dose Safety, Tolerability, Pharmacokinetic and Food Effect Study of KVD900 (Sebetralstat) in Healthy Volunteers
A safety, tolerability, pharmacokinetic and food effect study of KVD900 in healthy volunteers.
- Trial name
- NCT04349800
- Testing
- KVD900
- Where
- United Kingdom
- Volunteers
- 84 people, healthy volunteers welcome
- Sponsor
- KalVista Pharmaceuticals, Ltd.
Hereditary Angioedema
Treatment of Angioedema Attacks in Adolescent and Adult Patients 12 Years and Older With HAE Type I or II With Sebetralstat
The sebetralstat Early Access Program (EAP) provides early access to the investigational medicinal product (IMP) sebetralstat to eligible and approved Type I or II Hereditary Angioedema (HAE) adolescent and adult post-trial and…
- Trial name
- NCT06628713
- Testing
- Sebetralstat
- Where
- United States
- Sponsor
- KalVista Pharmaceuticals, Ltd.
Primary Immunodeficiency
Safety and Pharmacokinetics of IGSC 20% in Subjects With Primary Immunodeficiency
This study was designed to determine a dose of weekly subcutaneously administered Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography Purified (Grifols) (IGSC 20%) that produces steady-state AUC of total IgG that…
- Trial name
- NCT02604810
- Testing
- IGIV-C 10%, IGSC 20%
- Where
- 25 sites in United States, Canada
- Volunteers
- 53 people
- Sponsor
- Grifols Therapeutics LLC
Immunologic Deficiency Syndrome, Agammaglobulinemia
Rapid Infusion of Immune Globulin Intravenous (Human) In Primary Immunodeficiency Patients
The objective of this study is to determine if the safety and tolerability of Immune Globulin Intravenous (Human), 10% caprylate/chromatography (IGIV-C)purified is similar when infused at two different infusion rates.
- Conditions
- Immunologic Deficiency Syndrome, Agammaglobulinemia, Severe Combined Immunodeficiency, Wiskott-Aldrich Syndrome
- Trial name
- NCT00220766
- Testing
- Immune Globulin Intravenous [Human], 10% Caprylate/Chromatography Purified, Dextrose, 5% in Water
- Where
- 13 sites in United States, Canada
- Volunteers
- 100 people
- Sponsor
- Grifols Therapeutics LLC
Hereditary Angioedema, HAE
Efficacy and Safety Study of BCX7353 as an Oral Treatment for the Prevention of Attacks in HAE
This is a phase 3, multicenter, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of oral BCX7353 in preventing acute angioedema attacks in patients with Type I and Type II HAE.
- Trial name
- APeX-2 (NCT03485911)
- Testing
- BCX7353 capsules
- Where
- 47 sites in United States, Austria and 9 more
- Volunteers
- 121 people
- Sponsor
- BioCryst Pharmaceuticals
Hereditary Angioedema (HAE)
A Study of Lanadelumab in Persons With Hereditary Angioedema (HAE) Type I or II in North America
The main aim of this study is to compare the HAE attack rate before and after lanadelumab treatment was started in persons with Hereditary Angioedeme Type I or II. Data from participants will be collected for at least 24 months.
- Trial name
- EMPOWER (NCT03845400)
- Where
- 29 sites in United States, Canada and 1 more
- Volunteers
- 168 people
- Sponsor
- Shire
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.
Primary Immunodeficiency Diseases (PID)
A Database Survey to Evaluate the Safety of Immune Globulin Subcutaneous (Human), 20% Solution in Participants With Primary Immunodeficiency
This study is a retrospective database study in Japan to evaluate the safety of Immune Globulin Subcutaneous (Human), 20% Solution in participants with primary immunodeficiency disease (PID).
- Trial name
- NCT06565078
- Testing
- Immune Globulin Subcutaneous (Human), 20% Solution
- Where
- Japan
- Volunteers
- 100 people
- Sponsor
- Takeda
Hereditary Angioedema
A Single and Multiple Doses Safety, Tolerability, Pharmacokinetics and Food Effect Study of KVD824 in Healthy Volunteers
This is a 3 part phase 1, randomized, double-blind, placebo-controlled, study of the safety, tolerability, and pharmacokinetics of KVD824 following administration of single and multiple ascending oral doses; followed by a…
- Trial name
- NCT05178355
- Testing
- KVD824
- Where
- United Kingdom
- Volunteers
- 108 people, healthy volunteers welcome
- Sponsor
- KalVista Pharmaceuticals, Ltd.
Immunologic Deficiency Syndromes
Study of Immune Globulin Intravenous (Human) GC5107 in Subjects With Primary Humoral Immunodeficiency
The purpose of this study is to evaluate the safety, efficacy and Pharmacokinetics of Immune Globulin Intravenous (Human) GC5107 in subjects with Primary Humoral Immunodeficiency (PHID).
- Trial name
- NCT02783482
- Testing
- GC5107
- Where
- 20 sites in United States, Canada
- Volunteers
- 49 people
- Sponsor
- Green Cross Corporation