Clinical trials: Musculoskeletal Function
Irritable Bowel Syndrome (IBS), Functional Abdominal Pain
Peer Mentorship: An Intervention To Promote Effective Pain Self-Management In Adolescents
This protocol matches child subjects with peer mentors of similar age who have learned to function successfully with a chronic pain disorder.
- Conditions
- Irritable Bowel Syndrome (IBS), Functional Abdominal Pain, Fibromyalgia, Complex Regional Pain Syndrome (CRPS)
- Trial name
- NCT01118988
- Testing
- Mentorship
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- University of California, Los Angeles
Duchenne Muscular Dystrophy (DMD)
Twice Weekly Steroids and Exercise as Therapy for DMD
The study team will determine the potential of low dose twice weekly prednisone and whether exercise training can synergize to delay disease progression and improve muscle strength/physical function in boys with Duchenne muscular…
- Trial name
- NCT04322357
- Testing
- Prednisone, Exercise Training on standard steroid regimen, Exercise training on twice-weekly steroids
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- University of Florida
Muscular Dystrophy, Duchenne, Ambulatory Care
Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD)
The aim of this Phase III study was to assess the efficacy of idebenone on pulmonary function, motor function, muscle strength and quality of life in patients with DMD.
- Trial name
- DELOS (NCT01027884)
- Testing
- Idebenone
- Where
- 23 sites in United States, Austria and 8 more
- Volunteers
- 65 people
- Sponsor
- Santhera Pharmaceuticals
Duchenne Muscular Dystrophy
Use of Dynamic Arm Supports to Promote Activities of Daily Living in Individuals With DMD
This study is a longitudinal, randomized control trial evaluating the use of two commercially available dynamic arm support devices (1) Armon Ayura-Kinova and 2) JAECO WREX) to promote participation in activities of daily living…
- Trial name
- NCT03531788
- Testing
- Armon Ayura (Kinova), JAECO Wrex
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Roxanna Marie Bendixen
Growth Hormone Deficiency, Panhypopituitarism
Growth Hormone and Endothelial Function in Children
Objective: This study is designed to determine whether growth hormone treatment in children 8 to 18 years of age alters function of the lining of the arteries.
- Conditions
- Growth Hormone Deficiency, Panhypopituitarism, Short Stature
- Trial name
- NCT00373386
- Testing
- growth hormone
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Ohio State University
Spinal Muscular Atrophy
Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)
To evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene weighing ≥…
- Trial name
- SMART (NCT04851873)
- Testing
- OAV101
- Where
- 13 sites in United States, Australia and 7 more
- Volunteers
- 24 people
- Sponsor
- Novartis Pharmaceuticals
Osteoarthritis of the Knee
Structured Treatment of Osteoarthritis of the Knee With or Without Total Knee Replacement
The purpose of this study is to test whether surgical insertion of total knee replacement provides further improvement in quality of life, pain and function in addition to an algorithm for systematic non-surgical treatment…
- Trial name
- MEDIC (NCT01410409)
- Testing
- Neuromuscular training (NEMEX-TJR), Paracetamol, Burana
- Where
- 3 sites in Denmark
- Volunteers
- 100 people
- Sponsor
- Northern Orthopaedic Division, Denmark
Gastroschisis
Evaluation of Glycerin Suppositories to Improve Bowel Function in Gastroschisis
Gastroschisis is a congenital defect of the abdominal wall that leads to evisceration of various amounts of the abdominal organs.
- Trial name
- NCT03355326
- Testing
- Glycerin Suppository
- Where
- United States
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Indiana University
Haemophilia
Multidisciplinary Evaluation of Patients With Hemophilia
Multidisciplinary assessment of the physical, functional and psychosocial alterations in patients with hemophilia around the country.
- Trial name
- HOLISTIC (NCT02198430)
- Where
- Spain
- Volunteers
- 104 people
- Sponsor
- Universidad de Murcia
Muscular Dystrophies, Muscular Dystrophy, Duchenne
A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
HOPE-2 is a double-blind clinical trial evaluating the safety and efficacy of a cell therapy called CAP-1002 in study participants with Duchenne Muscular Dystrophy (DMD).
- Conditions
- Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- HOPE-2 (NCT03406780)
- Testing
- CAP-1002
- Where
- 9 sites in United States
- Volunteers
- 20 people
- Sponsor
- Capricor Inc.
Type 2 Spinal Muscular Atrophy
Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)
This was a Phase III multi-center, single dose (1.2 x 10^14 vector genomes), randomized, sham controlled, double-blind study that investigates the efficacy, safety and tolerability of OAV101B in treatment naive, sitting and never…
- Trial name
- STEER (NCT05089656)
- Testing
- OAV101
- Where
- 42 sites in United States, Brazil and 11 more
- Volunteers
- 126 people
- Sponsor
- Novartis Pharmaceuticals
Knee Osteoarthritis
Implementing Group Physical Therapy (PT) for Veterans With Knee Osteoarthritis (Group PT): Function QUERI 2.0
Implementing Group Physical Therapy (PT) for Veterans with Knee Osteoarthritis (OA): Function QUERI 2.0 (Group PT) aims to implement, evaluate and sustain Group PT in 16 VA sites using a type III effectiveness-implementation…
- Trial name
- Group PT (NCT05282927)
- Testing
- Implementation Strategy: Foundational REP, Implementation Strategy: Enhanced REP (enREP)
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- VA Office of Research and Development
Exercise-related Amenorrhea
Fat Mediated Modulation of Reproductive and Endocrine Function in Young Athletes
One aim of this study is to determine changes in body composition and hormones that differentiate athletes who stop getting their periods versus those who continue to get their periods and non-athletes.
- Trial name
- NCT00946192
- Testing
- Transdermal 17Beta-estradiol, progesterone, Ethinyl Estradiol + Desogestrel
- Where
- United States
- Volunteers
- 121 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
Hypophosphatasia, Bone Disease
Non-interventional Substudy of ALX-HPP-502 to Assess Natural History of Patients With Juvenile-onset HPP Who Served as Historical Controls in ENB-006-09
The purpose of this study is to characterize the natural history of HPP in patients with Juvenile-onset HPP who served as historical controls in ENB-006-09.
- Trial name
- NCT02235493
- Testing
- Retrospective Case Only
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Muscular Atrophy, Spinal
Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA)
This was a phase IV Open-label, single-arm, single-dose, multicenter study, to evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with SMA with bi-allelic mutations in…
- Trial name
- OFELIA (NCT05073133)
- Testing
- OAV101
- Where
- 5 sites in Argentina, Brazil
- Volunteers
- 16 people
- Sponsor
- Novartis Pharmaceuticals
Vitamin D Deficiency
Treatment Study of Vitamin D Deficiency in Adolescents
To compare the efficacy of two high dose vitamin D3 regimens (5,000 IU daily vs. 50,000 IU weekly) used clinically for the treatment of vitamin D deficiency versus a low dose of vitamin D3 used for supplementation (1,000 IU…
- Trial name
- NCT01784029
- Testing
- Vitamin D3
- Where
- United States
- Volunteers
- 183 people, healthy volunteers welcome
- Sponsor
- Montefiore Medical Center
Immune Deficiency Disorders, Severe Combined Immunodeficiency
Immune Disorder HSCT Protocol
This study hypothesizes that a reduced intensity immunosuppressive preparative regimen will establish engraftment of donor hematopoietic cells with acceptable early and delayed toxicity in patients with immune function disorders.
- Conditions
- Immune Deficiency Disorders, Severe Combined Immunodeficiency, Chronic Granulomatous Disease, X-linked Agammaglobulinemia
- Trial name
- NCT01821781
- Testing
- Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Washington University School of Medicine
Duchenne Muscular Dystrophy
Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy
The purpose of this study is to determine whether IGF-1 therapy improves or preserves muscle function in Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT01207908
- Testing
- IGF-1
- Where
- United States
- Volunteers
- 44 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Cleft Palate, Tube Disorders Eustachian
Exercises for Improving Soft Palate and Eustachian Tube Function in Children With Ear Tubes With or Without Cleft Palate
Elevation of the soft palate (the soft part of the roof of the mouth) during swallowing helps the Eustachian tube to open and keep the ear healthy.
- Conditions
- Cleft Palate, Tube Disorders Eustachian, Velopharyngeal Incompetence Due to Cleft Palate, Velopharyngeal Insufficiency
- Trial name
- NCT03868891
- Testing
- EMST150
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Noel Jabbour
Adolescent Idiopathic Scoliosis
MESA Rail™ AIS Study - Preservation of Curve Correction
To evaluate the restoration and maintenance of thoracic kyphosis and coronal curve correction demonstrated through the surgical implantation of the K2M MESA Rail™ Deformity System as compared to literature reported outcomes for…
- Trial name
- NCT02062606
- Testing
- MESA Rail™ Deformity System
- Volunteers
- 188 people
- Sponsor
- K2M, Inc.