Clinical trials: Congenital Spinal Deformities
Kennedy's Disease, Spinal and Bulbar Muscular Atrophy
Dutasteride to Treat Spinal and Bulbar Muscular Atrophy (SBMA)
This study will determine if the drug dutasteride can improve weakness, mobility, functioning, nerve function, and quality of life in patients with spinal and bulbar muscular atrophy (SBMA).
- Trial name
- NCT00303446
- Testing
- Dutasteride
- Where
- United States
- Volunteers
- 57 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Prostate Cancer
Study to Compare Capsule and Liquid Formulations of Enzalutamide After Single Dose Administration Under Fasting Conditions in Prostate Cancer
Background: Enzalutamide is given to treat metastatic prostate cancer. But it takes 4 pills to deliver the effective dose. This can be difficult for people already taking multiple drugs.
- Trial name
- NCT03478904
- Testing
- Enzalutamide Liquid, Enzalutamide Capsule
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- National Cancer Institute (NCI)
Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor
Dasatinib, Ifosfamide, Carboplatin, and Etoposide in Treating Young Patients With Metastatic or Recurrent Malignant Solid Tumors
RATIONALE: Dasatinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Drugs in chemotherapy, such as ifosfamide, carboplatin, and etoposide, work in different ways to stop the growth of…
- Conditions
- Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor, Kidney Cancer
- Trial name
- NCT00788125
- Testing
- carboplatin, dasatinib, etoposide phosphate
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- City of Hope Medical Center
Unresectable or Metastatic Microsatellite Stable (MSS) Solid Tumor, Microsatellite Stable (MSS) Colon Cancer
Study of PI3Kinase Inhibition (Copanlisib) and Anti-PD-1 Antibody Nivolumab in Relapsed/Refractory Solid Tumors With Expansions in Mismatch-repair Proficient (MSS) Colorectal Cancer
A phase I/II study of PI3Kinase inhibition (copanlisib) and anti-PD-1 antibody nivolumab in relapsed/refractory solid tumors with expansions in mismatch-repair proficient (MSS) colorectal cancer.
- Trial name
- NCT03711058
- Testing
- Copanlisib, Nivolumab
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Friedreich's Ataxia
Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia Study
The purpose of this phase 3 multi-center, open-label extension study is to evaluate the long-term safety of ACTIMMUNE® (interferon-γ 1b) in participants with Friedreich's Ataxia.
- Trial name
- STEADFAST (NCT02593773)
- Testing
- Interferon γ-1b
- Where
- 4 sites in United States
- Volunteers
- 86 people
- Sponsor
- Amgen
Sickle Cell Disease, Sickle Cell Anemia
Sickle Cell Disease (SCD) Bone Pain Study
A prospective study to determine how low bone mineral density and/or vertebral compression fractures associate with pain in adults with sickle cell disease
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia, Low Bone Density, Osteoporosis
- Trial name
- NCT05283148
- Testing
- Dual-energy X-ray absorptiometry, Vertebral fracture analysis, Adult Sickle Cell Quality of Life Measurement System pain impact questionnaire
- Where
- United States
- Volunteers
- 53 people
- Sponsor
- University of California, Davis
Friedreich's Ataxia
Safety and Pharmacology Study of VP 20629 in Adults With Friedreich's Ataxia
The objectives of the study are: To evaluate the safety and tolerability of single and multiple oral doses of VP 20629 in subjects with Friedreich's ataxia (FA).
- Trial name
- NCT01898884
- Testing
- VP 20629
- Where
- 5 sites in United States
- Volunteers
- 46 people
- Sponsor
- Shire
Spinal Muscular Atrophy Type I
Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I
The purpose of this study is to identify the maximum tolerated dosage of sodium phenylbutyrate in children with spinal muscular atrophy type I; and to determine if the drug has an effect on SMN mRNA and protein levels.
- Trial name
- NPTUNE 02 (NCT00439218)
- Testing
- sodium phenylbutyrate
- Where
- 5 sites in United States
- Volunteers
- 5 people
- Sponsor
- Westat
Neoplasm, Arteriovenous Malformations
A Phase II Study of Spinal Radiosurgery
Phase I of the study (motion and quality assurance [QA] study) is being used to determine intrafraction target motion and define quality assurance procedures for single fraction spinal radiosurgery.
- Trial name
- RAD0408 (NCT00573872)
- Testing
- Radiosurgery
- Where
- United States
- Volunteers
- 43 people
- Sponsor
- University of Alabama at Birmingham
Spinal and Bulbar Muscular Atrophy
Safety, Tolerability, and Efficacy of BVS857 in Patients With Spinal and Bulbar Muscular Atrophy
The purpose of this study was to determine if BVS857 is safe, tolerable and increases thigh muscle thickness in patients with spinal bulbar and muscular atrophy (SBMA).
- Trial name
- NCT02024932
- Testing
- BVS857
- Where
- 6 sites in United States, Denmark and 2 more
- Volunteers
- 37 people
- Sponsor
- Novartis Pharmaceuticals
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), Children's National Medical Center (CNMC) Department of Urology, Children's Research Institute (CRI) Center for Genetic Medicine Research…
- Trial name
- NCT02748356
- Testing
- Lactobacillus
- Where
- 2 sites in United States
- Volunteers
- 7 people
- Sponsor
- Medstar Health Research Institute
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
Friedreich Ataxia
An Objective Double-blind Evaluation of Bupropion and Citalopram in an Individual With Friedreich Ataxia
The purpose of this study is to document the clinical effects of bupropion and citalopram in a single subject with Friedreich Ataxia.
- Trial name
- NCT01716221
- Testing
- bupropion & Citalopram
- Volunteers
- 1 people
- Sponsor
- University of Colorado, Denver
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
Neurofibromatosis 2, Vestibular Schwannoma
Lapatinib Study for Children and Adults With Neurofibromatosis Type 2 (NF2) and NF2-Related Tumors
The purpose of this study is to determine if Lapatinib has any effect on tumors found in patients with Neurofibromatosis Type 2 (NF2). NF2 is a condition that mainly affects the skin and nervous system.
- Trial name
- NCT00973739
- Testing
- Lapatinib
- Where
- United States
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- NYU Langone Health
Degenerative Joint Disease, Congenital Deformity
Augment® Injectable Bone Graft Compared to Autologous Bone Graft as a Bone Regeneration Device in Hindfoot Fusions
STUDY OBJECTIVES: To demonstrate equivalent clinical and radiologic outcomes as "gold standard" (Autologous Bone Graft) in a representative clinical model (hindfoot fusions) STUDY HYPOTHESIS: Augment® Injectable is an equivalent…
- Conditions
- Degenerative Joint Disease, Congenital Deformity, Arthritis, Osteoarthritis
- Trial name
- NCT01305356
- Testing
- Augment® Injectable Bone Graft, Autologous bone graft
- Where
- 25 sites in United States, Canada
- Volunteers
- 299 people
- Sponsor
- BioMimetic Therapeutics
Trigeminal Nerve Injuries, Post-herpetic Neuralgia
Transcranial Magnetic Stimulation for Facial Pain
Complex oro-facial pain is a poorly diagnosed and poorly treated cause of significant suffering and disability. This study will explore the ability of fMRI to identify patients with complex oro-facial pain who respond to…
- Conditions
- Trigeminal Nerve Injuries, Post-herpetic Neuralgia, Facial Pain, Nervus Intermedius Neuralgia
- Trial name
- NCT02633306
- Testing
- transcranial magnetic stimulation
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- University of Minnesota
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
Spinal Muscular Atrophy Type I
Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies
This is a Phase 3, open-label, single-arm, single-dose, trial of onasemnogene abeparvovec-xioi (gene replacement therapy) in participants with spinal muscular atrophy (SMA) Type 1 and who are genetically defined by a biallelic…
- Trial name
- NCT03837184
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 4 sites in Japan, South Korea and 1 more
- Volunteers
- 2 people
- Sponsor
- Novartis Gene Therapies
Spinal Muscular Atrophy Type 3
Whole Body Vibration Therapy in Children With Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) are one of the common physical disabilities in childhood. For SMA, progressive muscle weakness and early fatigue hamper the mobility of the sufferers.
- Trial name
- NCT03056144
- Testing
- whole body vibration therapy
- Where
- Hong Kong
- Volunteers
- 1 people
- Sponsor
- The Hong Kong Polytechnic University