Clinical trials: Malformations
Telangiectasia, Hereditary Hemorrhagic, Epistaxis
North American Study of Epistaxis in Hereditary Hemorrhagic Telangiectasia (HHT)
The purpose of the NOSE Study is to carefully examine the efficacy and safety of 3 nasal sprays (bevacizumab, estriol, and tranexamic acid), compared to placebo, for the treatment of HHT related nosebleeds.
- Trial name
- NOSE (NCT01408030)
- Testing
- Sterile saline, Bevacizumab, Estriol
- Where
- 6 sites in United States
- Volunteers
- 123 people
- Sponsor
- James Gossage
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
Port Wine Stains
Combined Photodynamic and Pulsed Dye Laser Treatment of Port Wine Stains
This research study was designed to determine the effect on port wine stains (PWS) of liposomal benzoporphyrin derivative monoacid (BPD-MA) termed verteporfin by intravenous (IV) infusion for photodynamic therapy (PDT) or…
- Trial name
- PDT/PDL (NCT00556946)
- Testing
- Combined Photodynamic & Pulsed Dye Laser Treatment
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- University of California, Irvine
Small-cell Lung Cancer
Berzosertib + Topotecan in Relapsed Platinum-Resistant Small-Cell Lung Cancer (DDRiver SCLC 250)
The main purpose of this study is to assess efficacy, safety, tolerability and pharmacokinetics (PK) of Berzosertib in combination with Topotecan in participants with relapsed, platinum-resistant small-cell lung cancer (SCLC).
- Trial name
- NCT04768296
- Testing
- Berzosertib, Topotecan
- Where
- 51 sites in United States, Belgium and 5 more
- Volunteers
- 76 people
- Sponsor
- EMD Serono Research & Development Institute, Inc.
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
Breast Cancer
Study Assessing the Efficacy and Safety of Alpelisib Plus Fulvestrant or Letrozole, Based on Prior Endocrine Therapy, in Patients With PIK3CA Mutant, HR+, HER2- Advanced Breast Cancer Who Have Progressed on or After Prior Treatments
This was a Phase II, multicenter, open-label, three-cohort, non-comparative study of alpelisib plus endocrine therapy (either fulvestrant or letrozole) in subjects (pre- and post-menopausal women and men) with HR-positive…
- Trial name
- BYLieve (NCT03056755)
- Testing
- Alpelisib, Fulvestrant, Letrozole
- Where
- 94 sites in United States, Argentina and 17 more
- Volunteers
- 383 people
- Sponsor
- Novartis Pharmaceuticals
Renal Angiomyolipoma
Safety and Efficacy of Everolimus (Afinitor®) in Chinese Adult Patients With Angiomyolipoma Associated With Tuberous Sclerosis Complex.
The purpose of this study was to assess the safety and efficacy of everolimus (Afinitor®) in Chinese patients with renal angiomyolipoma (AML) associated with tuberous sclerosis complex (TSC).
- Trial name
- NCT03525834
- Testing
- Everolimus
- Where
- 5 sites in China
- Volunteers
- 40 people
- Sponsor
- Novartis Pharmaceuticals
May-Thurner Syndrome, Deep Vein Thrombosis
Venous Stent for the Iliofemoral Vein Investigational Clinical Trial Using the DUO Venous Stent System
This is a prospective, multi-center, single-arm, non-blinded clinical trial designed to investigate the safety and efficacy of the Vesper DUO Venous Stent System as compared to a pre-defined performance goal (PG) established from…
- Conditions
- May-Thurner Syndrome, Deep Vein Thrombosis, Chronic Venous Insufficiency
- Trial name
- VIVID (NCT04580160)
- Testing
- Duo Venous Stent System
- Where
- 30 sites in United States, Poland
- Volunteers
- 162 people
- Sponsor
- Vesper Medical, Inc.
Pregnancy, Neonates
Pregnancy and Neonatal Follow-up of Ongoing Pregnancies Established in Clinical Trial 38833 (P05783)
The objective of this trial is to evaluate whether corifollitropin alfa (MK-8962, Org 36286) treatment for the induction of multifollicular growth in women undergoing Controlled Ovarian Stimulation (COS) prior to in vitro…
- Trial name
- Care (NCT00702520)
- Testing
- Corifollitropin alpha (MK-8962, Org 36286) 100 ug, Corifollitropin alpha (MK-8962, Org 36286) 150 ug, Triptorelin
- Volunteers
- 15 people
- Sponsor
- Organon and Co
Seizure in Participants With Tuberous Sclerosis Complex, Seizure in Participants With Dravet Syndrome
Assessment of Adjunctive Cannabidiol Oral Solution (GWP42003-P) in Children With Tuberous Sclerosis Complex (TSC), Dravet Syndrome (DS), or Lennox-Gastaut Syndrome (LGS) Who Experience Inadequately-controlled Seizures
This study will be conducted to evaluate the safety, pharmacokinetics (PK), and efficacy of adjunctive GWP42003-P in participants < 2 years of age with tuberous sclerosis complex (TSC), Lennox-Gastaut syndrome (LGS), or Dravet…
- Conditions
- Seizure in Participants With Tuberous Sclerosis Complex, Seizure in Participants With Dravet Syndrome, Seizure in Participants With Lennox-Gastaut Syndrome
- Trial name
- NCT04485104
- Testing
- GWP42003-P
- Where
- 11 sites in United States, Italy and 1 more
- Volunteers
- 3 people
- Sponsor
- Jazz Pharmaceuticals
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
Hypervascular Tumor and Arteriovenous Malformation
A Multicenter, Open-label Study for E7040 in Japanese Subjects With Hypervascular Tumor and Subjects With Arteriovenous Malformation
To evaluate the efficacy and safety of transcatheter arterial embolization with E7040 in Japanese subjects with hypervascular tumor or arteriovenous malformation
- Trial name
- NCT01677624
- Testing
- E7040
- Where
- 10 sites in Japan
- Volunteers
- 28 people
- Sponsor
- Eisai Co., Ltd.
Epilepsy; Seizure, Neuromuscular Diseases
North Carolina Genomic Evaluation by Next-generation Exome Sequencing, 2
The "North Carolina Clinical Genomic Evaluation by Next-gen Exome Sequencing, 2 (NCGENES 2)" study is part of a larger consortium project investigating the clinical utility, or net benefit of an intervention on patient and family…
- Conditions
- Epilepsy; Seizure, Neuromuscular Diseases, Brain Malformation, Intellectual Disability
- Trial name
- NCGENES2 (NCT03548779)
- Testing
- Pre-visit prep, usual care + exome seq
- Where
- 3 sites in United States
- Volunteers
- 548 people
- Sponsor
- University of North Carolina, Chapel Hill
Spinal Cord Injuries
Effect of Botulinum Toxin A on Detrusor Overactivity and Renal Function in Chronic Spinal Cord Injured Patients
To investigate the clinical effect of detrusor botulinum toxin A (BoNT-A) injection on neurogenic detrusor overactivity (NDO) and renal function and to compare the difference of expressions of sensory receptors and nerve growth…
- Trial name
- NCT01682603
- Testing
- Botulinum toxin A
- Where
- Taiwan
- Volunteers
- 34 people
- Sponsor
- Buddhist Tzu Chi General Hospital
Tuberous Sclerosis Complex
Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex
Study design is a Phase IIb prospective multi-center, randomized, placebo-controlled, double-blind clinical trial. The goal will be to enroll 80 infants with Tuberous Sclerosis Complex who are less than 6 months of age prior to…
- Trial name
- NCT02849457
- Testing
- Early Vigabatrin
- Where
- 13 sites in United States
- Volunteers
- 84 people
- Sponsor
- Martina Bebin
End Stage Renal Disease, AV Fistula
Safety and Efficacy Study of the Venous Window Needle Guide to Access Arteriovenous (AV)Fistulae
The SAVE Study will evaluate the safety and efficacy of the Venous Window Needle Guide in achieving access of a deep, un-cannulatable arteriovenous fistula to complete hemodialysis as prescribed.
- Conditions
- End Stage Renal Disease, AV Fistula, Kidney Failure
- Trial name
- SAVE (NCT01471041)
- Testing
- Venous Window Needle Guide
- Where
- 2 sites in United States
- Volunteers
- 54 people
- Sponsor
- Vital Access Corp.
Autism Spectrum Disorders
Genetics, Brain Structure and Thinking Skills in Autism
The purpose of this research is to better understand the genetic, biochemical, cognitive and behavioral symptom abnormalities that contribute to autism spectrum disorders.
- Trial name
- NCT01451983
- Where
- United States
- Volunteers
- 138 people, healthy volunteers welcome
- Sponsor
- The Cleveland Clinic
Charcot-Marie-Tooth Disease Type 1A
Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (PLEO-CMT)
The purpose of this study is to determine whether PXT3003 is effective and safe in the treatment of Charcot-Marie-Tooth disease - Type 1 A (CMT1A).
- Trial name
- PLEO-CMT (NCT02579759)
- Testing
- PXT3003 dose 1, PXT3003 dose 2
- Where
- 30 sites in United States, Belgium and 6 more
- Volunteers
- 323 people
- Sponsor
- Pharnext S.C.A.
Spina Bifida, Latex Allergy
Yulex Glove Prospective Study in Spina Bifida
This study is to evaluate the safety of Yulex gloves when used by individuals who have documented allergy to natural rubber latex from Hevea brasiliensis.
- Trial name
- NCT01133288
- Testing
- Yulex gloves
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Obscure Gastrointestinal Bleeding, Angiodysplasia
Long-term Effects of Thalidomide for Recurrent Gastrointestinal Bleeding Due to Vascular Malformation
Background: Repeated episodes of bleeding from gastrointestinal vascular malformations refractory to endoscopic or surgical therapy often pose a major therapeutic challenge.
- Conditions
- Obscure Gastrointestinal Bleeding, Angiodysplasia, Gastric Antral Vascular Ectasia, Thalidomide
- Trial name
- NCT00964496
- Testing
- Thalidomide, Iron
- Volunteers
- 55 people
- Sponsor
- Shanghai Jiao Tong University School of Medicine