Clinical trials: Malformations
Arteriovenous Malformations
Treating Arteriovenous Malformation With Stereotactic Radiosurgery Using CT Angiography for Treatment Planning
Evaluate whether a treatment plan based on CT angiography can accurately and precisely identify the target nidus as compared to standard cerebral arteriography fused to MRI.
- Trial name
- NCT03559556
- Testing
- CT angiography
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- University of Texas Southwestern Medical Center
Tuberous Sclerosis, Subependymal Giant Cell Astrocytoma
Everolimus (RAD001) Therapy of Giant Cell Astrocytoma in Patients With Tuberous Sclerosis Complex
The purpose of the study is to evaluate the safety and potential side effects of everolimus (an experimental drug) on a person with Tuberous Sclerosis Complex who also has been diagnosed with a brain tumor (astrocytoma) The…
- Trial name
- NCT00411619
- Testing
- Everolimus
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Hereditary Sensory and Autonomic Neuropathy Type I
L-Serine Supplementation in Hereditary Sensory Neuropathy Type 1
In hereditary sensory and autonomic neuropathy type 1 (HSAN1) the investigators recently discovered the accumulation of two neurotoxic sphingolipids.
- Trial name
- NCT01733407
- Testing
- L-serine
- Where
- 2 sites in United States
- Volunteers
- 18 people
- Sponsor
- Massachusetts General Hospital
Hereditary Hemorrhagic Telangiectasia, Nosebleeds
Sirolimus for Nosebleeds in HHT
This pilot study is to determine the safety and efficacy of oral sirolimus (blood trough level 6-10ng/ml) in patients with HHT that are experiencing moderate or severe epistaxis.
- Conditions
- Hereditary Hemorrhagic Telangiectasia, Nosebleeds, Epistaxis
- Trial name
- NCT05269849
- Testing
- Sirolimus
- Where
- Canada
- Volunteers
- 10 people
- Sponsor
- Unity Health Toronto
Pregnancy, Neonates
Pregnancy and Neonatal Follow-up of Ongoing Pregnancies Established in Clinical Trial P05714 (Care Program)(P05715)
The objective of this trial is to evaluate whether corifollitropin alfa (Org 36286) treatment for the induction of multifollicular growth in women undergoing controlled ovarian stimulation (COS) prior to in vitro fertilization…
- Trial name
- Care (NCT00702234)
- Testing
- Corifollitropin alfa, GnRH antagonist, (rec)hCG
- Volunteers
- 268 people
- Sponsor
- Organon and Co
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
Uterine Fibroid, Arteriovenous Malformations
An Observational Study of Radial Access Embolization Procedures Using HydroPearl Microspheres
This registry study is designed to collect data on the procedural success and complication rates in real-world patients undergoing HydroPearl embolization procedures via radial access.
- Conditions
- Uterine Fibroid, Arteriovenous Malformations, Hypervascular Tumors, Benign Prostatic Hyperplasia
- Trial name
- RAVI Registry (NCT04272216)
- Testing
- Treatment with HydroPearl via radial access
- Where
- 6 sites in United States
- Volunteers
- 99 people
- Sponsor
- Terumo Medical Corporation
Meningomyelocele, Spinal Dysraphism
Management of Myelomeningocele Study (MOMS)
Spina bifida (myelomeningocele) is a complex birth defect in which a portion of the spinal cord is not fully developed. The overlying bones and skin are incompletely formed and the underdeveloped area of the spinal cord is…
- Trial name
- MOMS (NCT00060606)
- Testing
- Prenatal Myelomeningocele Repair Surgery, Postnatal Myelomeningocele Repair Surgery
- Where
- 3 sites in United States
- Volunteers
- 183 people, healthy volunteers welcome
- Sponsor
- The George Washington University Biostatistics Center
Cerebral Palsy, Spina Bifida
The Effectiveness of Participation-focused Interventions on Body Functions of Youth With Physical Disabilities
Youth with physical disabilities face greater restrictions to participation in community-based activities than their typically developing peers, which can lead to poor health outcomes.
- Conditions
- Cerebral Palsy, Spina Bifida, Muscular Dystrophies, Musculoskeletal Disorder
- Trial name
- NCT03851107
- Testing
- Engagement in 6-week community-based activity program
- Where
- Canada
- Volunteers
- 8 people
- Sponsor
- McGill University
Hereditary Hemorrhagic Telangiectasia (HHT)
Topical Bevacizumab for the Management of Recurrent Epistaxis in Patients With Hereditary Hemorrhagic Telangiectasia (HHT)
Terence M. Davidson, MD is conducting a research study to find out more about the topical application of Avastin (proper chemical name is bevacizumab) in the treatment of epistaxis (nose bleeding) in patients with Hereditary…
- Trial name
- NCT01397695
- Testing
- Bevacizumab
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of California, San Diego
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
Pregnancy, Neonates
Pregnancy and Neonatal Follow-up of Ongoing Pregnancies Established in Clinical Trial P05690 (Care Program) (P05710)
The objective of this follow-up study is to evaluate whether corifollitropin alfa (Org 36286) treatment for the induction of multifollicular growth in women undergoing controlled ovarian stimulation (COS) prior to in vitro…
- Trial name
- Care (NCT00702624)
- Testing
- Corifollitropin alfa, recFSH (follitropin beta), gonadatropin releasing hormone (GnRH) antagonist ganirelix
- Volunteers
- 113 people
- Sponsor
- Organon and Co
Microcystic Lymphatic Malformation
Study Evaluating the Safety and Efficacy of PTX-022 (QTORIN Sirolimus) in the Treatment of Microcystic Lymphatic Malformations
This study evaluates the safety and efficacy of PTX-022 (sirolimus) Topical Gel 3.9% w/w in the treatment of Microcystic Lymphatic Malformations. The participant will receive 3 months of PTX-022 treatment by the end of the study.
- Trial name
- NCT05050149
- Testing
- PTX-022
- Where
- 7 sites in United States
- Volunteers
- 12 people
- Sponsor
- Palvella Therapeutics, Inc.
Kidney Diseases, Arteriovenous Fistula
Prospective Feasibility Study Evaluating EchoMark LP Placement and EchoSure Measurements for Subjects Requiring Arteriovenous Fistulae
Multi-center trial to assess the feasibility and safety of the EchoMark LP and the EchoMark diagnostic ultrasound system for assessing AV fistula blood flow, diameter, and depth.
- Trial name
- NCT04896476
- Testing
- EchoMark / EchoSure
- Where
- 4 sites in United States
- Volunteers
- 20 people
- Sponsor
- Sonavex, Inc.
Tuberous Sclerosis Complex, Epilepsy
Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC Infants
This phase I/II clinical trial is an open-label clinical trial design to verify safety and dosing for TAVT-18 (sirolimus) powder for oral solution in TSC infants (N=5).
- Trial name
- STOP2 (NCT04595513)
- Testing
- TAVT-18 (sirolimus)
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Sleep Disorders
Efficacy and Safety of Circadin® in the Treatment of Sleep Disturbances in Children With Neurodevelopment Disabilities
The purpose of this study is to establish the efficacy and safety of Circadin in children with neurodevelopmental disorders and to determine the dose, this randomized, placebo-controlled study is planned to evaluate the efficacy…
- Trial name
- NCT01906866
- Testing
- Circadin 2/5/10 mg
- Where
- 28 sites in United States, Finland and 3 more
- Volunteers
- 125 people
- Sponsor
- Neurim Pharmaceuticals Ltd.
Lymphoma
A Study of INCB050465 in Relapsed or Refractory Follicular Lymphoma
The purpose of this study is to assess the objective response rate of parsaclisib treatment in participants with relapsed or refractory follicular lymphoma.
- Trial name
- CITADEL-203 (NCT03126019)
- Testing
- Parsaclisib
- Where
- 83 sites in United States, Australia and 11 more
- Volunteers
- 126 people
- Sponsor
- Incyte Corporation
Epilepsy, Tuberous Sclerosis Complex
Everolimus (RAD001) Therapy for Epilepsy in Patients With Tuberous Sclerosis Complex (TSC)
The goal of this study is to learn if the study drug RAD001 can reduced the number of epileptic seizures, and can be taken safety by people who have epilepsy associated with Tuberous Sclerosis Complex.
- Trial name
- NCT01070316
- Testing
- Everolimus
- Where
- 2 sites in United States
- Volunteers
- 20 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Epistaxis, Hereditary Hemorrhagic Telangiectasia
Office-sclerotherapy for Epistaxis Due to Hereditary Hemorrhagic Telangiectasia
The purpose of this study is to test a novel and tolerable office-based treatment method, sclerotherapy with sodium tetradecyl sulfate, for recurrent epistaxis (nosebleeds) related to Hereditary Hemorrhagic Telangiectasia (HHT)…
- Trial name
- NCT01408732
- Testing
- Sclerotherapy, Standard Treatment
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- University of Minnesota
Congenital Cystic Adenomatoid Malformation
Prenatal Steroids for Treatment of Congenital Cystic Adenomatoid Malformations (CCAM)
Congenital cystic adenomatoid malformations (CCAMs) are theorized to be growing immature lung tissue. Administration of maternal steroids in the mid-trimester may stop the growth or decrease the size of the CCAM, thus increasing…
- Trial name
- CCAM Steroids (NCT00670956)
- Testing
- Betamethasone
- Where
- 3 sites in United States
- Volunteers
- 1 people, healthy volunteers welcome
- Sponsor
- University of California, San Francisco