Clinical trials: Vascular Anomalies
Tuberous Sclerosis, Angiofibroma
Phase III Trial of Topical Formulation of Sirolimus to Skin Lesions in Patients With Tuberous Sclerosis Complex (TSC)
The purpose of this trial is to evaluate the efficacy and safety of NPC-12G gel (topical formulation of sirolimus) versus placebo gel to facial angiofibroma and other skin lesions in patients with tuberous sclerosis complex (TSC)
- Conditions
- Tuberous Sclerosis, Angiofibroma, Hypomelanotic Macule, Plaque
- Trial name
- NCT02635789
- Testing
- NPC-12G gel
- Where
- Japan
- Volunteers
- 62 people
- Sponsor
- Nobelpharma
Fabry Disease
A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
Study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of migalastat hydrochloride (HCl) (migalastat) in participants with Fabry disease.
- Trial name
- NCT00214500
- Testing
- migalastat HCl
- Where
- 5 sites in United States
- Volunteers
- 9 people
- Sponsor
- Amicus Therapeutics
Fabry Disease, Alpha Galactosidase A Deficiency
Study of the Effects of Fabrazyme Treatment on Lactation and Infants
The study was planned for up to 2 years (24 months). Planned full participation for both mother and infant was 24 months, planned full participation of mother and development of infant was 24 months, while planned full…
- Trial name
- NCT00230607
- Testing
- agalsidase beta
- Where
- 5 sites in United States, Austria and 1 more
- Volunteers
- 7 people
- Sponsor
- Genzyme, a Sanofi Company
Cancer
BCX-1777 in Treating Patients With Refractory Cancer
RATIONALE: BCX-1777 may stop the growth of cancer cells by blocking the enzymes necessary for their growth. PURPOSE: Phase I trial to study the effectiveness of BCX-1777 in treating patients who have refractory cancer.
- Trial name
- NCT00073944
- Testing
- forodesine hydrochloride
- Where
- United States
- Sponsor
- BioCryst Pharmaceuticals
Arteriovenous Fistula
ONYX Evaluation in the Endovascular Treatment of Intracranial Dural Arteriovenous Fistulae
The objective of this study is to evaluate the safety and performance post last embolization with Onyx
- Trial name
- dAVF (NCT02180945)
- Where
- France
- Volunteers
- 110 people
- Sponsor
- Medtronic Neurovascular Clinical Affairs
All Diagnosed Health Conditions, ADD/ADHD
At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions
We are the missing link in clinical trials, connecting patients and researchers seamlessly and conveniently using a mobile health platform to advance medical research.
- Conditions
- All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis
- Trial name
- NCT01931644
- Where
- United States
- Volunteers
- 17,667 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
Fabry Disease
A Study in Patients With Fabry Disease Who Are on Chronic Hemodialysis Therapy for Treatment of End-stage Renal Insufficiency.
People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme.
- Trial name
- NCT00312767
- Testing
- Fabrazyme (agalsidase beta)
- Where
- United States
- Sponsor
- Genzyme, a Sanofi Company
Diabetic Retinopathy
Computer-based Screening for Diabetic Retinopathy
The study is an Observational, Phase 0 designed to establish that the risk for diabetic retinopathy assigned by the RiskAnalyzer improves the reading accuracy and consistency of any reader and it decreases the inter-reader…
- Trial name
- NCT01614327
- Where
- 2 sites in United States
- Volunteers
- 19,199 people
- Sponsor
- VisionQuest Biomedical LLC
Fabry Disease
A Long Term Safety and Efficacy Study of Fabrazyme Replacement Therapy in Japanese Patients With Fabry Disease.
The purpose of this survey is to identify any concerns regarding the following efficacy and safety-related issues in clinical practice with the new drugs "Fabrazyme for intravenous infusion 5mg" and "Fabrazyme for intravenous…
- Trial name
- NCT00233870
- Testing
- Agalsidase beta (recombinant form)
- Where
- Japan
- Volunteers
- 405 people
- Sponsor
- Genzyme, a Sanofi Company
Hereditary Angioedema
Safety of Ruconest in 2-13 Year Old Hereditary Angioedema (HAE) Patients
This open-label study is being conducted to confirm the safety, pharmacokinetic profile and efficacy of Ruconest at a dose of 50 U/kg when used for the treatment of acute angioedema attacks in patients, from 2 up to and including…
- Trial name
- NCT01359969
- Testing
- rhC1INH
- Where
- 16 sites in United States, Czechia and 8 more
- Volunteers
- 57 people
- Sponsor
- Pharming Technologies B.V.
Retinal Disease, Age-Related Macular Degeneration
Stem Cell Ophthalmology Treatment Study II
This study will evaluate the use of autologous bone marrow derived stem cells (BMSC) for the treatment of retinal and optic nerve damage or disease.
- Conditions
- Retinal Disease, Age-Related Macular Degeneration, Retinitis Pigmentosa, Stargardt Disease
- Trial name
- SCOTS2 (NCT03011541)
- Testing
- Arm 1
- Where
- 4 sites in United States, Austria and 1 more
- Volunteers
- 500 people
- Sponsor
- MD Stem Cells
Cryopyrin-associated Periodic Syndromes (CAPS)
Study to Evaluate the Safety, Tolerability, Efficacy and Pharmacokinetics of JTE-162 in Subjects With Cryopyrin-Associated Periodic Syndrome (CAPS)
This study will evaluate the efficacy, safety, tolerability and pharmacokinetics of JTE-162 administered once daily for 2 weeks in subjects with cryopyrin-associated periodic syndrome (CAPS)
- Trial name
- NCT07247266
- Testing
- JTE-162
- Where
- Canada
- Volunteers
- 5 people
- Sponsor
- Shionogi Inc.
Hereditary Angioedema (HAE)
Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE
This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of once-daily orally administered deucrictibant extended-release tablet compared to placebo for prophylaxis to…
- Trial name
- CHAPTER-3 (NCT06669754)
- Testing
- Deucrictibant
- Where
- 56 sites in United States, Argentina and 22 more
- Volunteers
- 81 people
- Sponsor
- Pharvaris Netherlands B.V.
Solid Tumor, Renal Cell Carcinoma
A Study of BPI-452080 in Subjects With Solid Tumors
This open-label Phase 1 study will evaluate the efficacy and safety of BPI-452080 in patients with Solid Tumors
- Conditions
- Solid Tumor, Renal Cell Carcinoma, Von Hippel-Lindau Disease
- Trial name
- NCT05843305
- Testing
- BPI-452080
- Where
- 5 sites in China
- Volunteers
- 87 people
- Sponsor
- Betta Pharmaceuticals Co., Ltd.
Chronic Obstructive Pulmonary, Idiopathic Pulmonary Fibrosis
EZ-2053 in the Prophylaxis of Acute Pulmonary Allograft Rejection
The purpose of this study is to assess the efficacy and safety of the study drug, known as "ATG Fresenius S," which is sometimes called "EZ-2053," to prevent a lung transplant patient's body from rejecting a transplanted lung or…
- Conditions
- Chronic Obstructive Pulmonary, Idiopathic Pulmonary Fibrosis, Cystic Fibrosis, Bronchiectasis
- Trial name
- NCT00105183
- Testing
- EZ-2053, EZ-2053 5mg/kg
- Where
- 19 sites in United States, Australia and 2 more
- Volunteers
- 223 people
- Sponsor
- Neovii Biotech
Hereditary Angioedema
Pharmacokinetics of C1 Esterase Inhibitor in Hereditary Angioedema Subjects
The study objective was to describe the pharmacokinetics (PK) of one or two doses of C1 esterase inhibitor (C1INH-nf) in hereditary angioedema (HAE) subjects who were not experiencing an HAE attack.
- Trial name
- NCT00432510
- Testing
- C1 esterase inhibitor [human] (C1INH-nf)
- Where
- 6 sites in United States
- Volunteers
- 27 people
- Sponsor
- Shire
Fabry Disease
A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
Study to evaluate the safety, tolerability, and pharmacodynamics of migalastat hydrochloride (HCl) (migalastat) in participants with Fabry disease.
- Trial name
- NCT00283933
- Testing
- migalastat HCl
- Where
- 2 sites in France, United Kingdom
- Volunteers
- 5 people
- Sponsor
- Amicus Therapeutics
AV Fistula
SAVE Trial - Use of the Selution Sirolimus Eluting Balloon for Dysfunctional AV accEss Treatment Indications
Prospective multi-center single-blinded randomized controlled trial (RCT) investigating the safety and feasibility of the SELUTION SLR™ (Sustained Limus Release) 018 drug eluting balloon (DEB) for the treatment of failed AV…
- Trial name
- SAVE (NCT04327609)
- Testing
- SELUTION SLR™ DEB, Control Arm
- Where
- 3 sites in Greece, Singapore
- Volunteers
- 84 people
- Sponsor
- M.A. Med Alliance S.A.
Fabry Disease, Lysosomal Storage Diseases
A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190
Fabry disease is a rare, X-linked inborn error of glycosphingolipid metabolism caused by an abnormal gene encoding the α-galactosidase A (αGLA) enzyme.
- Trial name
- NCT04455230
- Testing
- FLT190
- Where
- 2 sites in Germany, United Kingdom
- Volunteers
- 3 people
- Sponsor
- Spur Therapeutics
Familial Hypoalphalipoproteinemia
CER-001 Therapy as a Novel Approach to Treat Genetic Orphan Diseases
The purpose of this study is to assess the impact of 29 intravenous infusions of CER-001 vs. placebo, given at weekly (9 infusions) and biweekly (20 infusions) intervals on carotid vessel wall area as measured by 3TMRI, when…
- Trial name
- TANGO (NCT02697136)
- Testing
- CER-001
- Where
- 19 sites in United States, Belgium and 5 more
- Volunteers
- 30 people
- Sponsor
- Cerenis Therapeutics, SA