Clinical trials: Neuromuscular Diseases in Infants, open to volunteers
Poliomyelitis
Study of Co-administered (Types 1 & 2) Novel Oral Poliomyelitis Vaccines Evaluation
The purpose of this study is to evaluate the safety and tolerability of co-administration of nOPV1 + nOPV2 in infants, relative to those receiving monovalent nOPV vaccines alone and whether two and/or three doses of…
- Trial name
- SCOPE (NCT06895486)
- Testing
- Novel Oral Polio Vaccine Type 1 (nOPV1), Novel Oral Polio Vaccine Type 2 (nOPV2)
- Where
- 3 sites in Panama
- Volunteers
- 675 people, healthy volunteers welcome
- Sponsor
- PATH
Spinal Muscular Atrophy Type I
Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1
GB221 is a gene therapy that delivers a working SMN1 gene to the motor neurons of people with spinal muscular atrophy (SMA) Type 1. This study will evaluate the safety, tolerability and efficacy of GB221 in two groups: 1.
- Trial name
- NCT07070999
- Testing
- GB221
- Where
- Brazil
- Volunteers
- 22 people
- Sponsor
- Gemma Biotherapeutics
Poliomyelitis
Study of a Novel Type 3 Oral Poliomyelitis Vaccine in Panama
The purpose of this clinical trial is to assess the safety and tolerability (primary objective), immunogenicity (primary and secondary objectives), fecal shedding of vaccine viruses (secondary objective) and the potential for…
- Trial name
- NCT05654467
- Testing
- Novel Live Attenuated Type 3 Oral Poliomyelitis Vaccine (nOPV3), Sabin Monovalent Oral Poliomyelitis Vaccine Type 3 (mOPV3)
- Where
- 3 sites in Panama
- Volunteers
- 1,532 people, healthy volunteers welcome
- Sponsor
- PATH
Spastic Cerebral Palsy, Acquired Brain Injury
Evaluation of Macroscopic Muscle Growth in Infants and Young Children With Spastic Cerebral Palsy
A mixed longitudinal design study will be carried out to explore the onset and time course of morphological muscle changes on a macroscopic level in children with spastic cerebral palsy (SCP).
- Trial name
- 3D-MMAP (NCT05197764)
- Where
- Belgium
- Volunteers
- 229 people, healthy volunteers welcome
- Sponsor
- Universitaire Ziekenhuizen KU Leuven
Pertussis, Diphtheria
Study of BK1803 in Healthy Infants
This study is a phase 3, randomized, observer-blinded, active-controlled, multicenter study to evaluate the immunogenicity and safety of BK1803 in healthy infants aged 2 months to less than 7 months.
- Conditions
- Pertussis, Diphtheria, Tetanus, Poliomyelitis
- Trial name
- NCT07793513
- Testing
- BK1803, GOBIK Aqueous Suspension Syringes, Bimmugen® Injection
- Where
- 33 sites in Japan
- Volunteers
- 344 people, healthy volunteers welcome
- Sponsor
- Tanabe Pharma Corporation
Poliomyelitis
Phase IV Clinical Study of sIPV Administration in Adolescent and Adult Populations
The goal of this clinical study is to compare the immunogenicity and safety of one dose of sIPV in adolescents or adults aged 7-50 years with that of three doses of DTaP-IPV-Hib Pentavalent Vaccine in Infants Aged 3 Months
- Trial name
- NCT07354269
- Testing
- sIPV, DTaP-IPV-Hib
- Where
- 3 sites in China
- Volunteers
- 180 people, healthy volunteers welcome
- Sponsor
- Sinovac Biotech Co., Ltd
Premature Infant, Chronic Respiratory Disease
Genesis Electrical Impedance Tomography (EIT): A Preliminary Study
The purpose of this study is to evaluate the Genesis Electrical Impedance Tomography (EIT) imaging system for use in pediatric respiratory disease populations including neuromuscular and bronchopulmonary dysplasia, as well as in…
- Conditions
- Premature Infant, Chronic Respiratory Disease, Neuromuscular Diseases, Healthy
- Trial name
- NCT03797183
- Where
- United States
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- University of Colorado, Denver
Muscular Atrophy, Spinal
A Study to Learn About the Safety and Effects of Salanersen (BIIB115) When Given to Babies With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec
In this study, researchers will learn more about the safety and effects of BIIB115, also known as salanersen. Specifically, researchers will learn more about how salanersen works in babies who have already been treated with…
- Trial name
- STELLAR-2 (NCT07444450)
- Testing
- Salanersen
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- Biogen
Obstetrical Brachial Plexus Palsy
Effectiveness and Safety of Early Intramuscular Botulinum Toxin Injections to Prevent Shoulder Deformity in Babies With Obstetrical Brachial Plexus Palsy
In children population with obstetrical brachial plexus palsy (OBPP), shoulder musculoskeletal deformity is the main cause of morbidity, with a loss of range of shoulder motion, pain and a reduction in social participation.
- Trial name
- POPB-TOX (NCT03198702)
- Testing
- Botulinum toxin type A injection
- Where
- 7 sites in France
- Volunteers
- 62 people
- Sponsor
- University Hospital, Brest
Infantile Spinal Muscular Atrophy
Test of Aquatic Mobility of SMA Infants
Spinal muscular atrophy (SMA) is a genetic neurodegenerative disease impacting spinal cord motor neurons, leading to motor and respiratory issues and, ultimately, death.
- Trial name
- BAINSMA (NCT06152302)
- Testing
- Mobility study in dry conditions, Mobility study in water conditions
- Where
- France
- Volunteers
- 15 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Infant, Premature, Intubation, Intratracheal
Fentanyl and Rocuronium for Tracheal Intubation in Newborns
Tracheal intubation is a common and critical procedure in neonatal intensive care units (NICU). Despite its clinical importance, first-attempt success rates remain low (approximately 50%), and adverse events - including oxygen…
- Conditions
- Infant, Premature, Intubation, Intratracheal, Respiratory Distress Syndrome, Newborn, Apnea of Prematurity
- Trial name
- NEORIN (NCT07650552)
- Testing
- Rocuronium Bromide 10 MG/ML, Fentanyl 1 mcg/kg IV, single dose, administered before non-emergent tracheal intubation.
- Where
- Brazil
- Volunteers
- 102 people
- Sponsor
- UEA - Universidade Do Estado Do Amazonas
Muscular Atrophy, Spinal
A Study to Learn How Nusinersen (Spinraza) Affects Participants With Spinal Muscular Atrophy (SMA) Who Took it Before or During Pregnancy And About The Health of Their Babies
In this study, researchers will know more about the effects of nusinersen, also known as Spinraza®, in pregnant participants with spinal muscular atrophy, also known as SMA.
- Trial name
- NCT05789758
- Testing
- No Intervention
- Where
- 14 sites in United States, United Kingdom
- Volunteers
- 20 people
- Sponsor
- Biogen
Duchenne Muscular Dystrophy (DMD)
The Baby Duchenne Study: Characterizing Developmental and Clinical Outcomes in the First Three Years in Children With Duchenne Muscular Dystrophy
The aim of the BABY DUCHENNE study is to evaluate the natural history and characterize the early clinical outcomes in very young children (0-3 years) with Duchenne muscular dystrophy (DMD) identified by newborn screening programs.
- Trial name
- BABY DUCHENNE (NCT07092540)
- Where
- 4 sites in United States
- Volunteers
- 105 people
- Sponsor
- University of Rochester
Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Cell-free fetal DNA (cffDNA) is present in the maternal blood from the early first trimester of gestation and makes up 5%-20% of the total circulating cell-free DNA (cfDNA) in maternal plasma.
- Conditions
- Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease, Cystic Fibrosis, Fragile X Syndrome
- Trial name
- DANNIgene (NCT06147414)
- Testing
- Blood sample
- Where
- France
- Volunteers
- 550 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Neuromuscular Disorders, Motor Development
Wearable Sensors to Detect Atypical Muscle Activation in Young Infants
The purpose of this study is to see if wearable sensor technology can be used to evaluate muscle activity and/or identify atypical muscle tone in infants up to 48 weeks postmenstrual age (8 weeks corrected age).
- Conditions
- Neuromuscular Disorders, Motor Development, Muscle Tone
- Trial name
- NCT06970522
- Where
- 2 sites in United States
- Volunteers
- 40 people
- Sponsor
- Shirley Ryan AbilityLab
Cystic Fibrosis, Sarcopenia
Tele-Rehabilitation in Adults With Cystic Fibrosis
This study is a randomized controlled trial designed to evaluate the effectiveness of an 8-week tele-rehabilitation program in adults with cystic fibrosis.
- Conditions
- Cystic Fibrosis, Sarcopenia, Frailty
- Trial name
- CF-TR (NCT07500493)
- Testing
- Tele-Rehabilitation Exercise Program
- Volunteers
- 76 people
- Sponsor
- King Saud University
Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy)
A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping
A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1044 for the treatment of Duchenne Muscular Dystrophy (DMD) with Gene Mutations Amenable to Exon 44 Skipping
- Conditions
- Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Muscular Disorders, Atrophic, Muscular Disease
- Trial name
- SAFARI44 (NCT07587242)
- Testing
- AOC 1044
- Where
- 18 sites in Australia, Belgium and 6 more
- Volunteers
- 70 people
- Sponsor
- Avidity Biosciences, Inc.
Glenohumeral Subluxation, Neonatal Brachial Plexus Palsy
Investigation of the Relationship Between Shoulder Alpha Angle and Humeral Head Displacement Percentage Measured by Ultrasonography in Neonatal Brachial Plexus Palsy Infants With Clinical Parameters
This study will examine infants with a birth-related nerve injury called neonatal brachial plexus palsy, which can affect movement in the arm and sometimes cause the shoulder joint to shift out of its normal position…
- Conditions
- Glenohumeral Subluxation, Neonatal Brachial Plexus Palsy, Brachial Plexus Injuries
- Trial name
- NCT07768904
- Testing
- Shoulder Ultrasonography, Standardized Clinical Examination
- Where
- Turkey (Türkiye)
- Volunteers
- 19 people
- Sponsor
- Fatih Sultan Mehmet Training and Research Hospital
Muscular Atrophy, Spinal
A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA)
In this study, researchers will learn more about the effects and safety of BIIB115, also known as salanersen. Specifically, researchers will learn more about how salanersen works in babies who have been diagnosed with SMA through…
- Trial name
- STELLAR-1 (NCT07221669)
- Testing
- Salanersen
- Where
- 14 sites in United States, Australia and 5 more
- Volunteers
- 30 people
- Sponsor
- Biogen
Duchenne Muscular Dystrophy
Once Weekly Infant Corticosteroid Trial for DMD
The hypothesis tested here is that a lower dose of intermittent oral corticosteroids (5mg/kg/week) will be equally effective to the 10mg/kg/week dose.
- Trial name
- NCT05412394
- Testing
- Prednisolone
- Where
- 4 sites in United States
- Volunteers
- 26 people
- Sponsor
- Anne M. Connolly