Clinical trials: Neuromuscular Diseases in Infants
Cystic Fibrosis, Motor Neuron Disease
MN4000 for Treatment of CF and MND Patients in the Home Setting
The study was a non-randomized open label pilot study. It was an observational design conducted at one (1) site in the US. All enrolled subjects received treatment with the MN4000.
- Conditions
- Cystic Fibrosis, Motor Neuron Disease, Airway Clearance Impairment
- Trial name
- NCT03797677
- Testing
- MN 4000
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Hill-Rom
Poliomyelitis
Safety and Reactogenicity of GSK Biologicals' Inactivated Poliomyelitis Vaccine (IPV) (Poliorix) in Infants
The study will evaluate the safety and reactogenicity of Poliorix given as primary vaccination course to Chinese children at 2, 3 and 4 months of age.
- Trial name
- NCT00937404
- Testing
- Poliorix
- Where
- China
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Muscular Atrophy, Spinal
A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
A global study of oral risdiplam in pre-symptomatic participants with spinal muscular atrophy (SMA).
- Trial name
- Rainbowfish (NCT03779334)
- Testing
- Risdiplam
- Where
- 7 sites in United States, Australia and 5 more
- Volunteers
- 26 people
- Sponsor
- Hoffmann-La Roche
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
Poliomyelitis
A Study to Evaluate the Safety and Immunogenicity of VLP-Polio in Infants and Toddlers
This is a phase I/II randomized, double-blind, and positive-controlled study. Participants in the phase I toddler group will receive 1 dose of either high dose VLP-Polio (Dose H) or control vaccine in a ratio of 3:1.
- Trial name
- NCT06577298
- Testing
- Recombinant Trivalent Poliomyelitis Vaccine (Sf-RVN Cell) (VLP-Polio) low adjuvant dose, VLP-Polio medium dose, VLP-Polio high dose
- Where
- Indonesia
- Volunteers
- 480 people, healthy volunteers welcome
- Sponsor
- CanSino Biologics Inc.
Poliomyelitis
Safety and Immunogenicity of Adjuvanted Reduced Dose Inactivated Polio Vaccine in 2, 4, 6 Months of Age
The trial is a phase III, non-inferiority, observer-blind, randomised, active controlled, multicentre clinical trial with 2 parallel groups: IPV-Al SSI (investigational vaccine) and IPV SSI (comparator vaccine).
- Trial name
- VIPV-07 (NCT03025750)
- Testing
- IPV-Al SSI, IPV SSI
- Where
- Panama
- Volunteers
- 800 people, healthy volunteers welcome
- Sponsor
- Statens Serum Institut
Spinal Muscular Atrophy
A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)
The primary objective is to examine the clinical efficacy of multiple doses of nusinersen (ISIS 396443) administered intrathecally to participants with Infantile-Onset Spinal Muscular Atrophy (SMA).
- Trial name
- NCT01839656
- Testing
- nusinersen
- Where
- 4 sites in United States, Canada
- Volunteers
- 21 people
- Sponsor
- Biogen
Poliomyelitis
An Immunogenicity and Safety Study of Sabin Inactivated Poliovirus Vaccine (Vero Cell) in 2-month-old Infants
The purpose of this phase III study is to evaluate the immunogenicity and safety of Sabin Inactivated Poliovirus Vaccine (Vero cell) in 2-month-old infants.
- Trial name
- NCT03526978
- Testing
- Investigational sIPV, Control IPV
- Where
- China
- Volunteers
- 1,200 people, healthy volunteers welcome
- Sponsor
- Sinovac Biotech Co., Ltd
Bacterial Infections, Virus Diseases
Safety, Tolerability, and Immunogenicity of V419 in Healthy Infants When Given at 2, 4, and 11 to 12 Months (V419-008)
This study will determine whether participants who receive V419 (PR5I) at 2, 4, and 11 to 12 months of age have an acceptable response to the vaccine.
- Trial name
- NCT01480258
- Testing
- PR5I, Rotavirus vaccine, Prevenar 13™
- Volunteers
- 1,315 people, healthy volunteers welcome
- Sponsor
- MCM Vaccines B.V.
Polio
Safety Study of a Single Dose of Monovalent High-dose Inactivated Poliovirus Type 2 Vaccine (m-IPV2 HD) in Infants Early in Life
Phase II, observer-blind, randomized study on the safety, reactogenicity, immunogenicity and impact on intestinal shedding of a single dose of monovalent high-dose inactivated poliovirus type 2 vaccine (m-IPV2 HD) or a single…
- Trial name
- IPV005 (NCT02111135)
- Testing
- b-OPV, m-OPV2, m-IPV HD
- Where
- Panama
- Volunteers
- 240 people, healthy volunteers welcome
- Sponsor
- Vaxtrials S.A.
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Sleep Apnea Syndromes in Children, Obesity in Children
Overview: Use of Respiratory Support At Home in Children
the primary outcome is to describe the perceived success of the use of the respiratory support from the parent's point of view
- Conditions
- Sleep Apnea Syndromes in Children, Obesity in Children, Bronchopulmonary Dysplasia (BPD), Neuromuscular Disease
- Trial name
- ELIPSE (NCT06692660)
- Where
- 2 sites in France
- Volunteers
- 30 people
- Sponsor
- University Hospital, Grenoble
Immunity to Oral Rotavirus Vaccine, Immunity to Oral Polio Vaccine
Zinc and/or Probiotic Supplementation of Rotavirus and Oral Polio Virus Vaccines
Background: Strategies are needed to improve oral rotavirus vaccine (RV), which provides suboptimal protection in developing countries.
- Conditions
- Immunity to Oral Rotavirus Vaccine, Immunity to Oral Polio Vaccine, Shedding of Oral Rotavirus Vaccine
- Trial name
- NCT01616693
- Testing
- Probiotic, Zinc, Rotavirus vaccine
- Where
- India
- Volunteers
- 620 people, healthy volunteers welcome
- Sponsor
- PATH
Neonatal Brachial Plexus Palsy
Forced Lefthandedness in Neonatal Brachial Plexus Palsy (NBPP) Children
The aim of the project is to assess the neurophysiological and functional consequences of forced lefthandedness in a population with right neonatal brachial plexus palsy (NBPP).
- Trial name
- NBPP (NCT04376372)
- Testing
- Functional testing, Magnetic resonance imaging, Electromyography
- Where
- Belgium
- Volunteers
- 82 people, healthy volunteers welcome
- Sponsor
- University Hospital, Ghent
Diphtheria, Tetanus
DTaP-IPV-Hep B-PRP~T Combined Vaccine Versus DTaP-IPV//PRP~T Combined Vaccine + Hep B Vaccine in Hep B Primed Infants
The aim of this study is to evaluate the immunogenicity and safety of a novel DTaP-IPV-Hep B-PRP~T fully liquid combined hexavalent vaccine (study vaccine) administered at 2, 4, and 6 months of age compared to Sanofi Pasteur's…
- Conditions
- Diphtheria, Tetanus, Pertussis, Haemophilus Influenzae Type b Infection
- Trial name
- NCT02094833
- Testing
- DTaP-IPV-Hep B-PRP~T combined vaccine, DTaP-IPV//PRP~T and Hepatitis B vaccine
- Where
- 16 sites in South Korea
- Volunteers
- 310 people, healthy volunteers welcome
- Sponsor
- Sanofi Pasteur, a Sanofi Company
Hepatitis B, Polio
Immunogenicity of DTaP-IPV-Hep B-PRP~T Combined Vaccine Compared With PENTAXIM™ and ENGERIX B® at 2-3-4 Months Schedule
To demonstrate that the immune response to hepatitis B antigen of the DTaP-IPV-Hep B-PRP~T is non-inferior to that of the association of PENTAXIM™ and ENGERIX B® one month after a three dose (2-3-4 month) primary series.
- Conditions
- Hepatitis B, Polio, Diphtheria, Pertussis
- Trial name
- NCT00315055
- Testing
- DTaP-IPV-HB-PRP~T vaccine, DTaP-IPV//PRP~T combined, Hepatitis B vaccine
- Where
- Turkey (Türkiye)
- Volunteers
- 310 people, healthy volunteers welcome
- Sponsor
- Sanofi Pasteur, a Sanofi Company
Poliomyelitis
Immunogenicity and Safety of Fractional Doses of IPV Intradermally vs Full Doses Intramuscularly
The present study intends to investigate the use of fractional doses of sanofi pasteur's IMOVAX Polio injected intradermally. The primary objective will be to demonstrate the non-inferiority of fractional doses of IMOVAX Polio…
- Trial name
- NCT00604058
- Testing
- Inactivated Poliomyelitis vaccine (IMOVAX)
- Where
- Philippines
- Volunteers
- 236 people, healthy volunteers welcome
- Sponsor
- Sanofi Pasteur, a Sanofi Company
Polio, Poliomyelitis
Sequential Inactivated Poliomyelitis Vaccine Followed by Oral Poliomyelitis Vaccine Versus Oral Poliomyelitis Vaccine
The purpose of this study is to generate data to support the registration extension of IMOVAX Polio to be used in a sequential vaccination.
- Trial name
- NCT01475539
- Testing
- Injectable inactivated types 1, 2 and 3 poliovirus + Live oral Polio, Live oral poliovirus type 1, 2 and 3
- Where
- China
- Volunteers
- 456 people, healthy volunteers welcome
- Sponsor
- Sanofi Pasteur, a Sanofi Company
Cystic Fibrosis, Bone Loss
CFTR Modulator Effects on Bone and Muscle in Adults With Cystic Fibrosis
Study is looking at the effects of cystic fibrosis treatment on bone muscle.
- Conditions
- Cystic Fibrosis, Bone Loss, Muscle Loss
- Trial name
- NCT04206436
- Testing
- Cftr Modulators
- Where
- United States
- Volunteers
- 63 people
- Sponsor
- Indiana University
Diphtheria, Tetanus
Study of DTaP-IPV-Hep B-PRP~T Combined Vaccine in Indian Infants Previously Given a Dose of Hepatitis B Vaccine at Birth
The purpose of this study is to describe the immunogenicity and safety of a novel DTaP- IPV- Hep B-PRT~T fully liquid combined hexavalent vaccine (Hexaxim™) administered at 6, 10 and 14 weeks of age in infants born to mothers…
- Conditions
- Diphtheria, Tetanus, Whooping Cough, Hepatitis B
- Trial name
- NCT01948193
- Testing
- Hexaxim™: DTaP-IPV-Hep B-PRP~T combined vaccine
- Where
- 2 sites in India
- Volunteers
- 177 people, healthy volunteers welcome
- Sponsor
- Sanofi Pasteur, a Sanofi Company