Clinical trials: Vascular Anomaly
- Condition Vascular Anomaly
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Fabry Disease, Chronic Kidney Disease, Stage IV (Severe)
Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme
This study was designed to determine appropriate treatment with Fabrazyme at a biweekly dose of either 1 mg/kg or 3 mg/kg in a population of patients with severe renal disease burden.
- Trial name
- NCT00837824
- Testing
- Fabrazyme (agalsidase beta)
- Where
- 9 sites in United States
- Volunteers
- 20 people
- Sponsor
- Genzyme, a Sanofi Company
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
Fabry Disease
Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease
The primary objective of this study was to compare the effect of migalastat (123 milligrams [mg] of migalastat [equivalent to 150 mg of migalastat hydrochloride]) (migalastat) versus placebo on kidney globotriaosylceramide (GL-3).
- Trial name
- NCT00925301
- Testing
- migalastat hydrochloride
- Where
- 28 sites in United States, Argentina and 11 more
- Volunteers
- 67 people
- Sponsor
- Amicus Therapeutics
Hereditary Angioedema (HAE)
Study of C1 Inhibitor (Human) for the Prevention of Angioedema Attacks and Treatment of Breakthrough Attacks in Japanese Subjects With Hereditary Angioedema (HAE)
The purpose of this study is to determine if an investigational treatment is safe and well tolerated when administered by intravenous (IV) infusion in Japanese subjects with HAE.
- Trial name
- NCT02865720
- Testing
- CINRYZE 500 U, CINRYZE 1000 U
- Where
- 10 sites in Japan
- Volunteers
- 8 people
- Sponsor
- Shire
Hereditary Angioedema
A Study to Evaluate the Safety and Effect of Escalating Doses of CINRYZE
The objectives of the study were: 1. To assess the safety and tolerability of escalating doses of CINRYZE. 2. To assess the effect of an escalating dose algorithm for CINRYZE on hereditary angioedema (HAE) attack rates. 3.
- Trial name
- NCT00914966
- Testing
- C1 inhibitor (human) [C1 INH]
- Where
- 11 sites in United States
- Volunteers
- 20 people
- Sponsor
- Shire
Hereditary Angioedema (HAE)
Safety and Efficacy Study of CINRYZE for Prevention of Angioedema Attacks in Children Ages 6-11 With Hereditary Angioedema
Primary Objective - To assess the relative efficacy of two dose levels of CINRYZE (500 Units and 1000 Units) administered by intravenous (IV) injection every 3 or 4 days to prevent angioedema attacks in children 6 to 11 years of…
- Trial name
- NCT02052141
- Testing
- CINRYZE 500, CINRYZE 1000
- Where
- 8 sites in United States, Germany and 3 more
- Volunteers
- 12 people
- Sponsor
- Shire
Pulmonary Hypertension
Phase 2 Study of Ambrisentan for Liver Function Test Rescue in Pulmonary Arterial Hypertension
This Phase 2 study was to determine the incidence of increased serum aminotransferase concentrations (alanine aminotransferase [ALT] and/or aspartate aminotransferase [AST]), as well as the overall safety and tolerability of…
- Trial name
- NCT00423592
- Testing
- ambrisentan
- Volunteers
- 36 people
- Sponsor
- Gilead Sciences
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.
Hereditary Angioedema
C1 Esterase Inhibitor (C1INH-nf) for the Treatment of Acute Hereditary Angioedema (HAE) Attacks
The study objective was to determine the safety and efficacy of C1INH-nf for the treatment of acute HAE attacks.
- Trial name
- NCT00289211
- Testing
- C1 esterase inhibitor [human] (C1INH-nf)
- Where
- 37 sites in United States
- Volunteers
- 83 people
- Sponsor
- Shire
Behçet Disease
Apremilast Pediatric Study in Children With Active Oral Ulcers Associated With Behçet's Disease
The aim of this study is to estimate the efficacy of apremilast compared to placebo in the treatment of oral ulcers in pediatric participants from 2 to < 18 years of age with oral ulcers associated with Behçet's disease (BD)…
- Trial name
- BEAN (NCT04528082)
- Testing
- Apremilast
- Where
- 27 sites in France, Greece and 6 more
- Volunteers
- 60 people
- Sponsor
- Amgen
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
Fabry Disease
Treatment Protocol of Replagal for Patients With Fabry Disease
The study will evaluate the safety and efficacy of Replagal® (agalsidase alfa) at a dose of 0.2 mg/kg infused intravenously (IV) over 40 minutes, every other week.
- Trial name
- NCT01031173
- Testing
- agalsidase alfa
- Where
- 25 sites in United States
- Sponsor
- Shire
Asphyxia Neonatorum
Biomarkers And Neurological Outcome in Neonates 2
Follow-up of participants of BANON1 study at age of at least 2 years. BANON1 (ClinicalTrials.gov ID: NCT03357250): Validation of biomarkers based on 24 months neuro-developmental outcome data in a human population for their…
- Trial name
- BANON2 (NCT04714775)
- Where
- 13 sites in Germany, Turkey (Türkiye)
- Volunteers
- 500 people
- Sponsor
- InfanDx AG
Fabry Disease
To Assess the Glycosphingolipid Clearance and Clinical Effects of Switching to Agalsidase Beta (Fabrazyme) Versus Continuing on Agalsidase Alfa (Replagal) in Male Patients With Classic Fabry Disease
Primary Objective: To assess reduction of plasma lyso-GL3 level after switch to agalsidase beta from agalsidase alfa Secondary Objectives: To assess reduction of kidney podocyte GL3 content after switch to agalsidase beta from…
- Trial name
- BCLEAR1 (NCT04143958)
- Testing
- agalsidase beta (GZ419828), agalsidase alfa
- Sponsor
- Sanofi
Hereditary Angioedema
OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)
The purpose of this study was to evaluate the safety and efficacy of donidalorsen in participants with HAE and effect of donidalorsen on the quality and pattern of HAE attacks and their impact on quality of life (QoL).
- Trial name
- NCT05139810
- Testing
- Donidalorsen
- Where
- 51 sites in United States, Belgium and 12 more
- Volunteers
- 91 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Hypertension
Congenital Malformations and Maternal Use of Anti-hypertensive Medication in the United Kingdom
Assess Fetal Mortality and Malformations in Women treated with antihypertensive medication during preganancy. Mother - Child pairs will be analysed in a cohort selected from the UK in the CPRD database.
- Trial name
- NCT02459990
- Where
- United Kingdom
- Volunteers
- 22,000 people
- Sponsor
- AstraZeneca
Ataxia Telangiectasia, Genetic Syndrome
Extension Treatment Using EryDex System in Patients With AT Who Participated in the ATTeST-IEDAT-02-2015 Study
Primary Objective To monitor and evaluate the long-term safety and tolerability of EDS-EP in AT patients. Secondary Objective To evaluate the long-term effect of EDS-EP on health-related Quality of Life (QoL; EQ-5D-5L scale).
- Trial name
- OLE-IEDAT (NCT03563053)
- Testing
- EryDex System
- Where
- 17 sites in United States, Australia and 9 more
- Volunteers
- 104 people
- Sponsor
- Quince Therapeutics S.p.A.
Antithrombin III Deficiency
Safety, Pharmacokinetics and Efficacy of an ATIII Concentrate
To assess the safety, pharmacokinetics and efficacy of a plasma-derived AT-III concentrate in the treatment of subjects with congenital AT-III deficiency.
- Trial name
- NCT00319228
- Testing
- Plasma-derived AT-III concentrate
- Volunteers
- 3 people
- Sponsor
- Instituto Grifols, S.A.
Ataxia Telangiectasia Louis-Bar, Ataxia Telangiectasia in Children
MBM-01 (Tempol) for the Treatment of Ataxia Telangiectasia
Ataxia Telangiectasia (A-T) is an autosomal recessively inherited neurodegenerative disorder that also has dramatic effects on the immune and endocrine systems.
- Conditions
- Ataxia Telangiectasia Louis-Bar, Ataxia Telangiectasia in Children, Ataxia Telangiectasia
- Trial name
- NCT04887311
- Testing
- MBM-01
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Matrix Biomed, Inc.
Hairy Cell Leukemia
US Post-Marketing Safety Study of Moxetumomab Pasudotox-tdfk (LUMOXITI)
This study is being conducted to satisfy a post-marketing requirement (PMR) to provide evidence characterizing 1) the safety of moxetumomab pasudotox-tdfk in patients who are 65 years of age and older and/or 2) the safety of…
- Trial name
- PROXY (NCT04125290)
- Where
- 2 sites in United States
- Volunteers
- 2 people
- Sponsor
- AstraZeneca