Clinical trials: Malformations
Tuberous Sclerosis
Adjunctive Ganaxolone Treatment (Part A) in TSC Followed by Long-term Treatment (Part B)
To assess preliminary safety and efficacy of ganaxolone as adjunctive therapy for the treatment of primary seizure types in patients with genetically- or clinically-confirmed TSC-related epilepsy through the end of the 12 week…
- Trial name
- TSC (NCT04285346)
- Testing
- Ganaxolone
- Where
- 7 sites in United States
- Volunteers
- 23 people
- Sponsor
- Marinus Pharmaceuticals
Chronic Inflammatory Demyelinating Polyneuropathy, Peripheral Neuropathy
SERENDEM : MD1003 in Patients Suffering From Demyelinating Neuropathies, an Open Label Pilot Study
The single-center, open-label Phase II study has the objective of assess the effect of MD1003 on motor and sensory conduction in patients suffering from demyelinating polyneuropathies in 15 subjects.
- Conditions
- Chronic Inflammatory Demyelinating Polyneuropathy, Peripheral Neuropathy, Charcot-Marie-Tooth Disease, Charcot-Marie-Tooth Disease Type 1A
- Trial name
- NCT02967679
- Testing
- MD1003
- Where
- France
- Volunteers
- 15 people
- Sponsor
- MedDay Pharmaceuticals SA
Improve Endothelial Function and Decrease Vascular Stenosis
Phosphodiesterase Type 5 Inhibition to Improve Endothelial Function and Vascular Remodeling in Chronic Kidney Disease and End Stage Renal Disease Patients Requiring New Arteriovenous Fistula
Patients with stage IV and V chronic kidney disease and end stage renal disease requiring hemodialysis at University of Alabama at Birmingham (UAB) Dialysis Clinics will be recruited from the UAB Vascular Access Clinic, which has…
- Trial name
- NCT02414204
- Testing
- Sildenafil
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- University of Alabama at Birmingham
Advanced Solid Tumours
A Study Investigating DNA-damage Response Agents in Molecularly Altered Advanced Cancer
The study is investigating efficacy, safety and tolerability of DNA-damage Response Agents (or Combinations), in participants with advanced/metastatic solid malignancies whose tumours contain molecular alterations
- Trial name
- NCT04564027
- Testing
- Ceralasertib
- Where
- 18 sites in United States, France and 1 more
- Volunteers
- 54 people
- Sponsor
- AstraZeneca
Cerebral Cavernous Malformation
The Symptomatic Cerebral Cavernous Malformation Trial of REC-994
This is a two-part, multi-center, randomized, double-blind, placebo-controlled study to investigate the safety, efficacy and pharmacokinetics of REC-994 (200 mg and 400 mg) compared to placebo in participants with symptomatic…
- Trial name
- SYCAMORE (NCT05085561)
- Testing
- REC-994
- Where
- 15 sites in United States
- Volunteers
- 62 people
- Sponsor
- Recursion Pharmaceuticals Inc.
Angiofibromas, Tuberous Sclerosis
Topical Rapamycin to Erase Angiofibromas in TSC
The study is a multi-center prospective, randomized, double-blind, placebo-controlled evaluation of the safety and efficacy of a topically applied formulation of rapamycin to cutaneous angiofibromas in subjects with Tuberous…
- Trial name
- Treatment (NCT01526356)
- Testing
- Rapamycin
- Where
- 10 sites in United States, Australia
- Volunteers
- 179 people
- Sponsor
- The University of Texas Health Science Center, Houston
Malformations
Varenicline Pregnancy Cohort Study
A prospective population-based cohort study to examine whether varenicline use during pregnancy is associated with an increased risk of major congenital malformations in infants above that associated with smoking during pregnancy.
- Trial name
- NCT01290445
- Testing
- varenicline, cigarette smoke from maternal smoking, Non-Exposure
- Volunteers
- 885,185 people
- Sponsor
- Pfizer
Neurogenic Detrusor Overactivity
A Two-week Open-label Pharmacodynamic and Pharmacokinetic Study of Multiple Doses of a Darifenacin Liquid Oral Suspension in Children (2 - 15 Years) With Neurogenic Detrusor Overactivity
This study will evaluate the pharmacodynamics and pharmacokinetics of darifenacin liquid oral suspension in children, ages 2-15 years, with neurogenic detrusor overactivity.
- Trial name
- NCT00712322
- Testing
- Darifenacin
- Where
- 12 sites in United States, Colombia
- Volunteers
- 35 people
- Sponsor
- Warner Chilcott
Arteriovenous Dural Fistula
PHIL in the Treatment of Intracranial dAVF.
This study is a prospective, multicenter, single-arm study. Patients with Dural Arteriovenous Fistulas (dAVF) have a few choice for safe treatment.
- Trial name
- PHIL dAVF (NCT03467542)
- Testing
- PHIL® Liquid Embolic System
- Where
- United States
- Volunteers
- 64 people
- Sponsor
- Microvention-Terumo, Inc.
Arteriovenous Malformations
Apollo™ Onyx™ Delivery Microcatheter Post Market Safety Study
The purpose of this study is to evaluate the safety of the Apollo™ Onyx™ Delivery Microcatheter used for delivery of the Onyx™ Liquid Embolic System during brain arteriovenous malformation (AVM) embolization procedures.
- Trial name
- NCT02378883
- Testing
- Apollo™ Onyx™ Delivery Micro Catheter
- Where
- 2 sites in United States
- Volunteers
- 112 people
- Sponsor
- Medtronic Neurovascular Clinical Affairs
Telangiectasia, Hereditary Hemorrhagic
Pomalidomide for the Treatment of Bleeding in HHT
This is a Phase II placebo-controlled double-blind study of pomalidomide in patients with hereditary hemorrhagic telangiectasia (HHT) with moderate to severe epistaxis who have anemia and/or require parenteral iron infusions or…
- Trial name
- PATH-HHT (NCT03910244)
- Testing
- Pomalidomide Oral Product
- Where
- 14 sites in United States
- Volunteers
- 145 people
- Sponsor
- The Cleveland Clinic
Arteriovenous Fistula
Use of Topical Tranexamic Acid and Bacitracin in Dialysis Patients
This study will investigate how the use of Tranexamic acid and Bacitracin applied to the bleeding site after the hemodialysis fistula needle is removed will affect Time to Clot and Infection Rate
- Trial name
- NCT02106962
- Testing
- Topical Tranexamic Acid 5% with bacitracin, Topical Tranexamic Acid 25% with bacitracin
- Where
- 3 sites in United States
- Volunteers
- 17 people
- Sponsor
- California Institute of Renal Research
Radiation Necrosis
Low-dose Intra-arterial Bevacizumab for Edema and Radiation Necrosis Therapeutic Intervention (LIBERTI)
To assess the overall safety and efficacy of intra-arterial (IA) bevacizumab for the treatment of radiation necrosis. A single 2.5 mg/kg dose of bevacizumab will be given intra-arterially after osmotic blood-brain-barrier…
- Trial name
- LIBERTI (NCT02819479)
- Testing
- 25% Mannitol, Low-dose Intra-arterial Bevacizumab
- Where
- 2 sites in United States
- Volunteers
- 10 people
- Sponsor
- Norton Healthcare
Spina Bifida, Myelomeningocele
Minimally Invasive Fetoscopic Regenerative Repair of Spina Bifida - A Pilot Study
Researchers are studying a new minimally invasive technique (fetoscopic repair) for repair of spina bifida (MMC) during the second trimester of pregnancy.
- Conditions
- Spina Bifida, Myelomeningocele, Neural Tube Defects
- Trial name
- NCT03936322
- Testing
- Minimally invasive fetoscopic repair of MMC
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Rodrigo Ruano
Arthritis, Osteoarthritis
Tanezumab In Osteoarthritis Of The Knee (2)
The purpose of this study is to test the efficacy and safety of 2 doses of tanezumab compared with naproxen and placebo in patients with osteoarthritis.
- Trial name
- NCT00830063
- Testing
- tanezumab 10 mg, tanezumab 5 mg, naproxen
- Where
- 94 sites in United States
- Volunteers
- 832 people
- Sponsor
- Pfizer
MPN (Myeloproliferative Neoplasms)
A Study of INCB050465 in Combination With Ruxolitinib in Subjects With Myelofibrosis
The purpose of this study is to evaluate the safety, tolerability, and efficacy of the combination of parsaclisib and ruxolitinib in subjects with myelofibrosis.
- Trial name
- NCT02718300
- Testing
- Parsaclisib, Ruxolitinib
- Where
- 39 sites in United States
- Volunteers
- 74 people
- Sponsor
- Incyte Corporation
Venous Stenosis, Venous Occlusion
The Merit WRAPSODY AV Access Efficacy Study (WAVE)
The purpose of the study is to demonstrate the safety and efficacy of the Merit WRAPSODY Endovascular Stent Graft for treatment of stenosis or occlusion within the dialysis access outflow circuit
- Trial name
- WAVE (NCT04540302)
- Testing
- Merit WRAPSODY Endovascular Stent Graft, PTA
- Where
- 43 sites in United States, Brazil and 2 more
- Volunteers
- 357 people
- Sponsor
- Merit Medical Systems, Inc.
Epilepsy, Dravet Syndrome
A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of TAK-935 (OV935) as an Adjunctive Therapy in Pediatric Participants With Developmental and/or Epileptic Encephalopathies
The purpose of this study is to investigate the effect on the frequency of all seizures (convulsive and drop) in participants treated with TAK-935 compared to placebo.
- Conditions
- Epilepsy, Dravet Syndrome, Lennox-Gastaut Syndrome
- Trial name
- ELEKTRA (NCT03650452)
- Testing
- TAK-935
- Where
- 44 sites in United States, Australia and 6 more
- Volunteers
- 141 people
- Sponsor
- Takeda
Solid Tumors
Safety, Tolerability and Pharmacokinetics of Cobimetinib in Combination With Pictilisib in Patients With Locally Advanced or Metastatic Solid Tumors
This is an open-label, multicenter, Phase Ib dose-escalation study designed to assess the safety, tolerability and pharmacokinetics of oral dosing of cobimetinib and pictilisib administered in combination in patients with locally…
- Trial name
- NCT00996892
- Testing
- Cobimetinib, Pictilisib
- Where
- 6 sites in United States
- Volunteers
- 178 people
- Sponsor
- Genentech, Inc.
Hereditary Haemorrhagic Telangiectasia
Hereditary Haemorrhagic Telangiectasia Flight Safety Study
Hereditary Haemorrhagic Telangiectasia (HHT) is a condition in which sufferers have abnormal blood vessels which makes them more likely to bleed than other people, particularly in the lungs, which results in low blood oxygen…
- Trial name
- Flying and HHT (NCT01590121)
- Testing
- Aeroplane flight in the past- no active intervention for study, Questionnaire
- Where
- United Kingdom
- Volunteers
- 156 people
- Sponsor
- Imperial College London