Clinical trials: Congenital Spinal Deformities
Friedreich Ataxia
Single Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia
To evaluate the safety and tolerability of single ascending doses of CTI-1601 in participants with Friedreich's ataxia
- Trial name
- NCT04176991
- Testing
- CTI-1601
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Larimar Therapeutics, Inc.
Spinocerebellar Ataxia
Wearable Gait Sensors
This research study is testing body-worn sensors to measure movement during simple tests of coordination, in order to evaluate the progression and severity of Spinocerebellar ataxia.
- Trial name
- NCT06628934
- Where
- United States
- Volunteers
- 600 people, healthy volunteers welcome
- Sponsor
- University of Chicago
Kyphoscoliosis
Indication and Treatment of Adult Kyphoscoliosis
Degeneration, iatrogenic-/idiopathic causes and fractures can lead to kypho- scoliotic deformities potentially resulting in pain and loss of function.
- Trial name
- INTRAKS (NCT04536909)
- Testing
- Non-surgical, Surgical
- Where
- 5 sites in Japan, Norway and 1 more
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- University of Bergen
Spinal Muscular Atrophy, SMA
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
This double-blind, Phase 2, multiple-dose study will be conducted to evaluate the PK/PD, efficacy, safety, and tolerability of apitegromab in subjects <2 years old with 5q autosomal recessive SMA who have delayed motor milestones…
- Conditions
- Spinal Muscular Atrophy, SMA, Spinal Muscular Atrophy Type 2, Spinal Muscular Atrophy Type 3
- Trial name
- OPAL (NCT07047144)
- Testing
- Apitegromab, Nusinersen, Risdiplam
- Where
- 23 sites in United States, Belgium and 5 more
- Volunteers
- 52 people
- Sponsor
- Scholar Rock, Inc.
Spinocerebellar Degeneration
An Extension Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD)
The purpose of this study is to evaluate the long-term safety, efficacy, and pharmacokinetics of KPS-0373 in SCD patients.
- Trial name
- NCT01970111
- Testing
- KPS-0373, High dose, KPS-0373, Low dose
- Where
- Japan
- Sponsor
- Kissei Pharmaceutical Co., Ltd.
Prostate Cancer, Prostatic Neoplasms
Safety and Efficacy Studies of Panobinostat and Bicalutamide in Patients With Recurrent Prostate Cancer After Castration
This trial is designed to investigate the safety, dosing schedule, and efficacy of the combination treatment of Panobinostat (a histone deacetylase inhibitor) and hormone therapy for recurrent prostate cancer.
- Trial name
- NCT00878436
- Testing
- Panobinostat, Bicalutamide
- Where
- 4 sites in United States
- Volunteers
- 52 people
- Sponsor
- NYU Langone Health
Spina Bifida
Effects of Progressive Resistance Exercise Training on Endurance and Functional Mobility in Children With Spina Bifida
In this research the effects of progressive resistance exercise training on endurance and functional mobility in children with spina bifida are assessed.
- Trial name
- NCT05719636
- Testing
- Conventional treatment, Progressive resistance exercise
- Where
- Pakistan
- Volunteers
- 20 people
- Sponsor
- Riphah International University
Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia 3
Clinical Trial Readiness for SCA1 and SCA3
The investigators plan to fill the gap between the current state of clinical trial readiness and the optimal one for SCA1 and SCA3, which are fatal rare diseases with no treatments.
- Trial name
- READISCA (NCT03487367)
- Where
- United States
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- The Methodist Hospital Research Institute
Friedreich Ataxia
Efficacy of Stabilometric Platform to Improve Standing Balance in Patients With Friedreich's Ataxia
The primary objective is to evaluate the potential effectiveness of an individualized intensive rehabilitation intervention using the "Prokin 252" stabilometric platform in the treatment of adolescent and adult patients with…
- Trial name
- NCT06692296
- Testing
- Balance training over stabilometric platform, Balance training by means of conventional rehabilitation
- Where
- Italy
- Volunteers
- 24 people
- Sponsor
- IRCCS Eugenio Medea
Hereditary Ataxia
Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Hereditary Ataxia
The Hereditary Ataxias are a group of genetic disorders characterized by slowly progressive incoordination of gait and often associated with poor coordination of hands, speech, and eye movements.
- Trial name
- NCT01360164
- Testing
- human umbilical cord mesenchymal stem cells
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Shenzhen Beike Bio-Technology Co., Ltd.
Friedreich Ataxia, Cardiomyopathies
Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia
The purpose of this study is to test the safety and preliminary efficacy of AAVrh.10hFXN to treat the cardiomyopathy associated with Friedreich's ataxia (FA).
- Conditions
- Friedreich Ataxia, Cardiomyopathies, Cardiac Hypertrophy, Myocardial Fibrosis
- Trial name
- NCT05302271
- Testing
- AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN, Prednisone
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Weill Medical College of Cornell University
Friedreich Ataxia
Evaluation of the Effect of Artesunate in Friedreich Ataxia (FA)
This dose-escalation study is aimed at investigating a novel application for artesunate in the treatment of Friedreich ataxia. It will evaluate this novel application of oral artesunate using a surrogate biological marker as…
- Trial name
- ARTEMIS (NCT04921930)
- Testing
- Artesunate Oral Product
- Where
- France
- Volunteers
- 20 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Neuromuscular Diseases, Congenital Diaphragmatic Hernia
Respiratory Muscles and Work of Breathing in Children
Respiratory muscle testing allows a quantitative assessment of inspiratory and expiratory muscles in children of any age with primary or secondary respiratory muscle impairment, in order to better understand the pathophysiology…
- Conditions
- Neuromuscular Diseases, Congenital Diaphragmatic Hernia, Lung Diseases, Cardiac Diseases
- Trial name
- WOB&MR_Ped (NCT05051254)
- Testing
- Esogastric pressure measurement
- Where
- France
- Volunteers
- 550 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
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
Alzheimer's Disease, Mild Cognitive Impairment
Retinal Imaging in Neurodegenerative Disease
This study aims to develop and evaluate biomarkers using non-invasive optical coherence tomography (OCT) and OCT angiography (OCTA) as well as ultra-widefield (UWF) fundus photography to assess the structure and function of the…
- Conditions
- Alzheimer's Disease, Mild Cognitive Impairment, Parkinson's Disease, Multiple Sclerosis
- Trial name
- NCT03233646
- Testing
- Retinal and Choroidal Imaging
- Where
- United States
- Volunteers
- 2,000 people, healthy volunteers welcome
- Sponsor
- Duke University
Neurogenic Bladder, Bladder Dysfunction
Urinary Artificial Sphincter in Children
Retrospective monocentric study of the outcomes of patients with neurogenic bladder, who had a urinary artificial sphincter before the age of 18 years old.
- Conditions
- Neurogenic Bladder, Bladder Dysfunction, Spina Bifida, Child, Only
- Trial name
- SUA (NCT05858840)
- Testing
- Urinary Artificial Sphincter (UAS) in Children
- Where
- France
- Volunteers
- 17 people
- Sponsor
- University of Lorraine
Rheumatoid Arthritis (RA), Atopic Dermatitis (AD)
A Study of Oral Upadacitinib (RINVOQ) Tablets to Assess Adverse Events and Change in Disease Symptoms in Korean Participants With Moderate to Severe Active Rheumatoid Arthritis, Atopic Dermatitis, Ankylosing Spondylitis or Psoriatic Arthritis
Rheumatoid Arthritis (RA) is an inflammatory disease of the joints characterized by the swelling of multiple joints and tenderness caused by progressive inflammatory synovitis, which leads to serious and debilitating diseases.
- Conditions
- Rheumatoid Arthritis (RA), Atopic Dermatitis (AD), Ankylosing Spondylitis (AS), Psoriatic Arthritis (PsA)
- Trial name
- NCT04541810
- Where
- 116 sites in South Korea
- Volunteers
- 2,369 people
- Sponsor
- AbbVie
Pregnancy, Fetal and Neonatal Health
Outcome of Fetal Spina Bifida
Neural tube defects are one of the most prevalent congenital abnormalities, surpassed only by cardiac malformations. Spina bifida accounts for the majority of the neural tube defects and is comprised of a wide spectrum of…
- Trial name
- NCT01100697
- Where
- Germany
- Volunteers
- 103 people
- Sponsor
- University of Luebeck
Friedreich Ataxia, Cardiomyopathy
Clinical Course Of Disease In Participants With FA-CM
Characteristics and clinical course of disease In participants with cardiomyopathy associated with Friedreich Ataxia (CLARITY-FA)
- Trial name
- NCT06865482
- Where
- 19 sites in United States, Brazil and 6 more
- Volunteers
- 65 people
- Sponsor
- Lexeo Therapeutics
High Tumor Mutation Burden, High TMB (Tumor Mutation Burden)
Study of MK-3475 (Pembrolizumab) in Patients With Microsatellite Unstable (MSI) Tumors (Cohort D)
This study will be looking at whether MK-3475 (pembrolizumab) is effective (anti-tumor activity) and safe in patients with MSI (Microsatellite Unstable) negative cancer with a mutator phenotype.
- Conditions
- High Tumor Mutation Burden, High TMB (Tumor Mutation Burden), MSS (Microsatellite Stable)
- Trial name
- NCT04098068
- Testing
- MK-3475
- Where
- 3 sites in United States
- Volunteers
- 12 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins