Clinical trials: Primary Immunodeficiency Disease(s)
Hereditary Angioedema
PK Subtrial in Adolescent Patients With HAE Type I or II Participating in the KVD900-302 Trial
This is a multicenter pharmacokinetic (PK) subtrial to investigate the PK profile of KVD900 (sebetralstat) in adolescent patients 12 to 17 years of age with Hereditary Angioedema (HAE) type I or II.
- Trial name
- NCT05511922
- Testing
- KVD900 600 mg, Drug: KVD900 300 mg
- Where
- 61 sites in United States, Australia and 15 more
- Volunteers
- 11 people
- Sponsor
- KalVista Pharmaceuticals, Ltd.
Primary Immunodeficiency Diseases (PID)
Inbon Errors of Immunity Attending Assiut University Children&Amp;#39;s Hospital: a Single Center Study
Aim of Study The aim of this study to describe the clinical profile of children diagnosed as IEIs who were admitted to in Assiut university children\'s Hospital.
- Trial name
- NCT06649643
- Testing
- Inborn errors of immunity, A. Routine .1CBC with blood film. .2Liver Function Test. .3Kidney Function Test. 4. Inflamatory marker ESR, CRP B.Immunology 1-Immunoglobulins IgG, IgM, IgA, IgE 2-Flow cytometric assessment: B
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Assiut University
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
Hereditary Angioedema
An Open-label Extension Trial to Evaluate the Long-term Safety of KVD900 (Sebetralstat) for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema (HAE)
This is an open-label, multicenter extension trial to evaluate the long-term safety of KVD900 in patients who are 12 years or older with HAE type I or II.
- Trial name
- NCT05505916
- Testing
- KVD900 600 mg, KVD900 300 mg
- Where
- 71 sites in United States, Australia and 21 more
- Volunteers
- 145 people
- Sponsor
- KalVista Pharmaceuticals, Ltd.
Hereditary Angioedema (HAE)
EDEMA2: Evaluation of DX-88's Effect in Mitigating Angioedema
EDEMA2 is an open-label, Phase 2 dose-ranging study designed to assess the safety and efficacy of repeated dosing of DX-88 (recombinant plasma kallikrein inhibitor) in Patients with Hereditary Angioedema.
- Trial name
- NCT01826916
- Testing
- DX-88 (ecallantide)
- Volunteers
- 77 people
- Sponsor
- Shire
Waning Donor Chimerism, Waning Immune Function
CD34+ Stem Cell Infusion to Augment Graft Function
The purpose of this study is to determine if infusing additional special donor cells will help to improve graft or immune function in previously transplanted children with immune deficiencies and bone marrow failures.
- Conditions
- Waning Donor Chimerism, Waning Immune Function, Primary Immunodeficiency Disease(s), Bone Marrow Failure
- Trial name
- NCT01856582
- Testing
- CD34+
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health