Clinical trials: Malformations
Hemorrhagic Hereditary Telangiectasia, HHT
Subsequent Bevacizumab Treatment in Patients With HHT. Follow up BABH
Bevacizumab is widely prescribed for the treatment of severe bleeding related to epistaxis or gastrointestinal bleeding in HHT. We studied the efficacy of bevacizumab on severe bleeding in HHT patients in a randomized study…
- Trial name
- BEST (NCT06039124)
- Testing
- bevacizumab treatment
- Where
- 3 sites in France
- Volunteers
- 23 people
- Sponsor
- Hospices Civils de Lyon
Spina Bifida Aperta, Acquired Traumatic Spinal Cord Injury
Self-esteem and Neuro-urological Follow-up in Patients With Spina Bifida or Spinal Cord Injury
With improved life expectancy over the last fifty years, spina bifida has become a disease of the adult. One of the major stakes for these patients is the preservation of a regular follow-up of uro-nephrologicals risk factors and…
- Trial name
- ESTIME (NCT01606618)
- Where
- 16 sites in France
- Volunteers
- 111 people
- Sponsor
- Rennes University Hospital
Charcot Marie Tooth Disease, Inherited Peripheral Neuropathy
Disability Severity Scale (DSI) and Hereditary Motor and Sensory Neuropathy Overall Disability Scale (HMSN-R-ODS)
The purpose of this research study is to create and validate two patient reported outcome (PRO) questionnaires. PRO questionnaires ask questions that help to measure disability in patients with inherited neuropathies.
- Trial name
- DSI and HMSN (NCT02194010)
- Where
- 3 sites in United States, United Kingdom
- Volunteers
- 563 people
- Sponsor
- University of Iowa
Cerebral Palsy, Spinal Muscular Atrophy
Pediatric Powered Wheelchair Standing Devices: An Exploratory Study
For children who use a power wheelchair, a powered wheelchair standing device (PWSD) may be considered for daily use. A PWSD allows a child to electronically move between sitting and standing and can be driven in either position.
- Conditions
- Cerebral Palsy, Spinal Muscular Atrophy, Spinal Cord Injuries, Spina Bifida
- Trial name
- NCT05117827
- Testing
- Power Wheelchair Standing Device
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Grand Valley State University
Hereditary Hemorrhagic Telangiectasia (HHT), Rendu Osler Disease
IV Bevacizumab Treatment of Patients With Hereditary Hemorrhagic Telangiectasia (HHT) or Rendu-Osler (RO) Disease: A Retrospective Analysis of French Cohort Data From 2009 to 2024
Rendu-Osler disease (RO), also known as Hereditary Haemorrhagic Telangiectasia (HHT), is an autosomal dominant genetic disorder. Its pathophysiology is associated with an imbalance in angiogenic balance.
- Trial name
- CoBé (NCT07772050)
- Testing
- Real World data study.
- Where
- France
- Volunteers
- 244 people
- Sponsor
- Hospices Civils de Lyon
Spina Bifida, Neurogenic Bowel
Bowel Continence Across the Lifespan in People With Spina Bifida
The goal of this observational study is to learn how different enema programs affect bowel control in children and adults with spina bifida.
- Conditions
- Spina Bifida, Neurogenic Bowel, Bowel Incontinence
- Trial name
- BCALS (NCT07390318)
- Testing
- Retrograde Enema, Antegrade Enema
- Where
- 24 sites in United States
- Volunteers
- 943 people
- Sponsor
- David Chu
Lymphatic Abnormality
Injection Sclerotherapy With Bleomycin VS Doxycycline in Patients With Lymphatic Malformations
This is a randomized clinical trial comparing the safety and effectiveness of injection sclerotherapy using bleomycin versus doxycycline in children under 12 years with lymphatic malformations.
- Trial name
- NCT07320001
- Testing
- Bleomycin, doxycycline (tetracycline)
- Volunteers
- 92 people
- Sponsor
- Basharat Hussain
Tuberous Sclerosis Complex
Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in Infants
The purpose of the study is to evaluate the efficacy, tolerability, and safety of vigabatrin versus rapamycin as a preventive treatment in infants with Tuberous Sclerosis Complex (TSC).
- Trial name
- ViRap (NCT04987463)
- Testing
- Vigabatrin, Rapamycin
- Where
- 2 sites in Poland
- Volunteers
- 60 people
- Sponsor
- Katarzyna Kotulska
Rathke's Cleft Cyst
Evaluation of the Pravalence of Rathke's Cleft Cyst of Children Under 15 Years
The goal of the study was to evaluate the pravalence of Rathke's Cleft Cyst of children under 15 years of age and demonstrate that their prevalence is higher than for patients over 15 years of age, due to their embryonic origin.
- Trial name
- PREVA-KPR (NCT03975712)
- Testing
- Not applicable, no intervention
- Where
- France
- Volunteers
- 460 people
- Sponsor
- Centre Hospitalier Universitaire de Besancon
Breast Cancer
Biomarker Testing and Treatment Patterns Among Patients With PIK3CA Mutation in Advanced Breast Cancer
This was a retrospective observational study of patients with advanced breast cancer (BC). This non-interventional study was conducted using discrete structured data and medical record abstraction, if needed, from patients…
- Trial name
- NCT05982886
- Where
- United States
- Volunteers
- 1,093 people
- Sponsor
- Novartis Pharmaceuticals
Neural Tube Defects, Spina Bifida
Laparotomy Versus Percutaneous Endoscopic Correction of Myelomeningocele
The purpose of this study is to evaluate the feasibility of a fetoscopic surgical technique for antenatal correction of fetal myelomeningocele. Two surgical approaches will be utilized.
- Conditions
- Neural Tube Defects, Spina Bifida, Myelomeningocele, Chiari Malformation
- Trial name
- NCT03856034
- Testing
- Fetoscopy
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- USFetus
Lipedema, Lymphedema
Investigation of Lipedema, Lymphedema and Vascular Malformations by Multispectral Optoacoustic Tomography (MSOT)
This study aims to analyze the fatty tissue architecture of the subcutaneous tissue in patients from the plastic surgery department.
- Conditions
- Lipedema, Lymphedema, Vascular Malformations
- Trial name
- NCT06399367
- Testing
- MSOT
- Where
- Germany
- Volunteers
- 100 people
- Sponsor
- University Hospital Goettingen
Colorectal Cancer Metastatic
Influence of BRAF and PIK3K Status on the Efficacy of 5-Fluorouracil/Leucovorin/Oxaliplatin (FOLFIRI) Plus Bevacizumab or Cetuximab in Patients With RAS Wild-type Metastatic Colorectal Carcinoma and < 3 Circulating Tumor Cells (CTC)
The purpose of the study is to explore the influence of BRAF and PIK3K status on the efficacy of FOLFIRI plus Bevacizumab or Cetuximab, as first line therapy of patients with RAS wild-type metastatic colorectal carcinoma and < 3…
- Trial name
- VISNU-2 (NCT01640444)
- Testing
- FOLFIRI + bevacizumab, FOLFIRI + cetuximab
- Where
- Spain
- Volunteers
- 240 people
- Sponsor
- Spanish Cooperative Group for the Treatment of Digestive Tumours (TTD)
Vascular Diseases, Cerebrovascular Diseases
Evaluation of the Accuracy of the Vessel Navigator Tool for Catheterization of Supra-aortic Vessels - ARCHIBALD
This study compares the accuracy of fusion imaging (Fusion Roadmap) versus real-time X-ray imaging (Roadmap) during catheterization of supra-aortic trunks of in patients with aneurysms or arteriovenous malformations.
- Conditions
- Vascular Diseases, Cerebrovascular Diseases, Cardiovascular Diseases, Aortic Aneurysm
- Trial name
- ARCHIBALD (NCT03269734)
- Where
- France
- Volunteers
- 15 people
- Sponsor
- Fondation Ophtalmologique Adolphe de Rothschild
Hereditary Hemorrhagic Telangiectasia (HHT)
Phase 1b Study of TX002100 in Participants With Hematologic Support-Dependent Hereditary Hemorrhagic Telangiectasia (HHT)
The primary purpose of this study is to assess the safety and tolerability of TX002100 in participants with hereditary hemorrhagic telangiectasia (HHT).
- Trial name
- NCT07863609
- Testing
- TX002100
- Volunteers
- 20 people
- Sponsor
- Tectonic Operating Company, Inc.
Digestive System Abnormalities
Follow-up of Children With Gastrointestinal Malformations and Postnatal Surgery
The aim of this single center study is to measure the impact of standardized neonatal pediatric surgeries due to gastrointestinal malformations on the children's motor and cognitive development and psycho-emotional competence.
- Trial name
- FraMal (NCT01451307)
- Where
- Germany
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Goethe University
Malformations, Multiple Abnormalies
Phenotype and Etiology of Pallister-Hall Syndrome
We aim to delineate the range of severity, natural history, molecular etiology, and pathophysiology of Pallister-Hall syndrome (PHS), Greig cephalopolysyndactyly syndrome (GCPS), McKusick-Kaufman syndrome (MKS), Bardet-Biedl…
- Conditions
- Malformations, Multiple Abnormalies, Polydactyly
- Trial name
- NCT00001404
- Where
- 4 sites in United States, Turkey (Türkiye)
- Volunteers
- 1,170 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
CCSVI, Chronic Cerebrospinal Venous Insufficiency
Multi-center Registry for CCSVI Testing and Treatment
Patients suspected of CCSVI (chronic cerebrospinal venous insufficiency) may be tested and if appropriate treated with catheter venoplasty by an interventional radiologist in their local community who has been credentialled…
- Conditions
- CCSVI, Chronic Cerebrospinal Venous Insufficiency, Venous Malformations of CNS Draining Veins
- Trial name
- CCSVI (NCT01205633)
- Testing
- venous angioplasty
- Where
- 9 sites in United States
- Volunteers
- 1,000 people
- Sponsor
- Hubbard Foundation
Dementia, Mild Cognitive Impairment
Safety and Modulation of ABCC9 Pathways by Nicorandil for the Treatment of Hippocampal Sclerosis of Aging
Widespread recognition of the current and projected impact of the dementia epidemic has spurred research into novel drug discovery efforts.
- Trial name
- SMArT-HS (NCT04120766)
- Testing
- Nicorandil
- Where
- United States
- Volunteers
- 64 people
- Sponsor
- Gregory Jicha, MD, PhD
Chronic Kidney Diseases, End Stage Renal Disease
SAVE-FistulaS: the SelfWrap-Assisted ArterioVEnous Fistulas Study
This is a prospective, randomized, multi-center clinical trial for chronic kidney disease (CKD) patients referred for creation of a new arteriovenous fistula (AVF) in order to assess the safety and effectiveness of SelfWrap, a…
- Conditions
- Chronic Kidney Diseases, End Stage Renal Disease, Arteriovenous Fistula, Hemodialysis Access Failure
- Trial name
- SAVE-FistulaS (NCT06001827)
- Testing
- SelfWrap Bioabsorbable Perivascular Wrap, Untreated AVF Control
- Where
- 28 sites in United States
- Volunteers
- 600 people
- Sponsor
- VenoStent