Clinical trials: Malformations
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
Familial Dysautonomia
Carbidopa for the Treatment of Nausea and Vomiting in Familial Dysautonomia
This is a pilot clinical trial of carbidopa to treat disabling attacks of nausea and vomiting in patients with familial dysautonomia (FD, also known as Riley Day syndrome or hereditary sensory and autonomic neuropathy type III).
- Trial name
- NCT01212484
- Testing
- Carbidopa
- Volunteers
- 12 people
- Sponsor
- NYU Langone Health
Osteoporosis, Cerebral Palsy
Prevention of Post Operative Bone Loss in Children
Hypothesis: one-dose pamidronate will prevent post-operative bone loss in children at risk for low bone density Plan: children with chronic disease such as CP, spina bifida, etc.
- Conditions
- Osteoporosis, Cerebral Palsy, Spina Bifida, Osteopenia
- Trial name
- NCT00655681
- Testing
- pamidronate, saline
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- University of New Mexico
Neural Tube Defects, Contraception
Efficacy and Safety Study for an Oral Contraceptive Containing Folate
The purpose of this study is to determine whether the study drug is safe and effective
- Conditions
- Neural Tube Defects, Contraception, Oral Contraceptives (OC)
- Trial name
- NCT00468481
- Testing
- Drospirenone/Ethinylestradiol/Methyltetrahydrofolate, Drospirenone/Ethinylestradiol (Yaz)
- Where
- 9 sites in United States
- Volunteers
- 385 people, healthy volunteers welcome
- Sponsor
- Bayer
Cerebral Cavernous Malformation
Atorvastatin Treatment of Cavernous Angiomas With Symptomatic Hemorrhage Exploratory Proof of Concept (AT CASH EPOC) Trial
This phase I/II randomized, placebo-controlled, double-blinded, single-site clinical trial is designed to investigate the effect of a prolonged course of atorvastatin versus placebo on CCM lesional iron deposition assessed by…
- Trial name
- AT CASH EPOC (NCT02603328)
- Testing
- Atorvastatin
- Where
- United States
- Volunteers
- 80 people
- Sponsor
- University of Chicago
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.
Advanced Stage Solid Tumors
ATRi Transition Rollover Study
The main purpose of the study was to monitor the safety of participant receiving long-term treatment of Berzosertib as monotherapy.
- Trial name
- NCT03309150
- Testing
- Berzosertib
- Where
- United Kingdom
- Volunteers
- 1 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Lymphangioleiomyomatosis, Tuberous Sclerosis Complex
Safety of Simvastatin in LAM and TSC
The purpose of this research study is to see if simvastatin can be taken safely in patients with either LAM or TSC, who are already being treated with everolimus or sirolimus.
- Trial name
- SOS (NCT02061397)
- Testing
- Simvastatin, Sirolimus Oral Product, Everolimus Oral Product
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- University of Pennsylvania
Chronic Kidney Disease
A Study of Vonapanitase (PRT-201) Administered Immediately After Radiocephalic Arteriovenous Fistula(AVF) Creation in Patients With Chronic Kidney Disease (CKD) (PATENCY-2)
This research study is designed to assess the safety and effectiveness of an experimental drug called vonapanitase (PRT-201) in patients both receiving or expecting to receive hemodialysis who have chronic kidney disease and who…
- Trial name
- NCT02414841
- Testing
- Vonapanitase
- Where
- 41 sites in United States, Canada
- Volunteers
- 696 people
- Sponsor
- Proteon Therapeutics
Lower Urinary Tract Symptoms
Impact of Probiotics on Urinary Symptoms in Spinal Cord Injury SCI and SB
As a collaborative effort between MedStar National Rehabilitation Hospital (NRH)/MedStar Health Research Institute (MHRI), Children's National Medical Center (CNMC) Department of Urology, Children's Research Institute (CRI)…
- Trial name
- NCT02748317
- Testing
- Lactobacillus rhamnosus GG
- Where
- 2 sites in United States
- Volunteers
- 96 people
- Sponsor
- Medstar Health Research Institute
Bardet Biedl Syndrome (BBS), Alström Syndrome (AS)
Setmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Participants With Moderate to Severe Obesity
This pivotal, phase 3 study is designed to confirm the efficacy and safety of setmelanotide, a potent melanocortin receptor type 4 (MC4R) agonist, for the treatment of obesity and hyperphagia in participants with Bardet Biedl…
- Trial name
- NCT03746522
- Testing
- Setmelanotide
- Where
- 12 sites in United States, Canada and 4 more
- Volunteers
- 52 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Vein of Galen Malformations
Fetal Treatment of Galenic Malformations
This is a prospective, single-arm non-randomized interventional study of fetuses to assess the safety and efficacy of fetal embolization of Vein of Galen Malformation (VOGM).
- Trial name
- NCT04434729
- Testing
- Fetal embolization of vein of Galen malformation using Target XL and XXL Detachable Coils
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Darren Orbach
Spina Bifida, Myelomeningocele
Fetoscopic NEOX Cord 1K® Spina Bifida Repair
To fetoscopically use cryopreserved human umbilical cord allografts, named NEOX Cord 1K®, as a spinal cord cover of spina bifida defects.
- Conditions
- Spina Bifida, Myelomeningocele, Myeloschisis
- Trial name
- NCT04243889
- Testing
- NEOX Cord 1K
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- The University of Texas Health Science Center, Houston
Hereditary Hemorrhagic Telangiectasia
Topical Anti-angiogenic Therapy for Telangiectasia in HHT: Proof of Concept
Hereditary hemorrhagic telangiectasia (HHT) is a hereditary vascular condition characterized by the development of abnormal connections between arteries and veins throughout the body, called vascular malformations.
- Trial name
- NCT01752049
- Testing
- Topical timolol maleate
- Where
- Canada
- Volunteers
- 5 people
- Sponsor
- Unity Health Toronto
Arterio-Venous Fistula, ESRD
VasQ External Support for Arteriovenous Fistula
This is a prospective clinical study of the VasQ external support for arteriovenous fistulas. The device is designed to improve fistula outcomes by optimizing the geometrical configuration of the fistula, influencing…
- Conditions
- Arterio-Venous Fistula, ESRD, Steal Syndrome, Aneurysm
- Trial name
- NCT03242343
- Testing
- VasQ
- Where
- 15 sites in United States
- Volunteers
- 144 people
- Sponsor
- Laminate Medical Technologies
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
Autoimmune Hemolytic Anemia
A Study of INCB050465 in Participants With Autoimmune Hemolytic Anemia
The purpose of this study is to evaluate the safety and efficacy of parsaclisib administered orally to participants with autoimmune hemolytic anemia (AIHA) who have decreased hemoglobin and evidence of ongoing hemolysis that…
- Trial name
- NCT03538041
- Testing
- Parsaclisib
- Where
- 12 sites in United States, Austria and 2 more
- Volunteers
- 25 people
- Sponsor
- Incyte Corporation
Angelman Syndrome, Nervous System Diseases
Dietary Supplements for the Treatment of Angelman Syndrome
Angelman syndrome (AS) is a complex genetic disorder that affects the nervous system. The purpose of this study is to determine the effectiveness of certain dietary supplements in treating the symptoms of AS.
- Trial name
- NCT00348933
- Testing
- Betaine, Creatine, Metafolin
- Where
- 4 sites in United States
- Volunteers
- 90 people
- Sponsor
- University of California, San Diego
Intracranial Arteriovenous Malformations (AVM)
Protocol for Staged Stereotactic Radiosurgery for Large Arteriovenous Malformations
Stereotactic radiosurgery is a well established treatment option for arteriovenous malformations (AVMs). The potential complications related to radiosurgery are well documented and are predominately related to radiation effects…
- Trial name
- NCT02576535
- Testing
- Fractionated stereotactic radiosurgery, Leskell gamma unit
- Volunteers
- 18 people
- Sponsor
- NYU Langone Health
Dysautonomia, Familial, Baroreflex Failure Syndrome
Carbidopa for the Treatment of Excessive Blood Pressure Variability
The overall study objectives are to determine whether carbidopa (Lodosyn®) is safe and well tolerated and to assess whether it can inhibit catecholamine-induced paroxysmal hypertension and normalize or reduce the exaggerated…
- Trial name
- CarbiFD (NCT02553265)
- Testing
- Carbidopa Low-Dose, Carbidopa High-Dose
- Where
- 2 sites in United States
- Volunteers
- 22 people
- Sponsor
- NYU Langone Health