Clinical trials: Neuromuscular Diseases in Infants
Sjogren-Larsson Syndrome
ADX-629 Therapy for Sjogren-Larsson Syndrome
This research study will determine whether orally administered ADX-629 is safe and has biochemical efficacy in participants with Sjögren-Larsson syndrome (SLS), a rare inherited disorder of fatty aldehyde metabolism The disease…
- Trial name
- NCT05443685
- Testing
- ADX-629
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- University of Nebraska
Poliomyelitis, Poliomyelitis Vaccines
Immunogenicity and Safety Study of GlaxoSmithKline Biologicals' Poliorix™ Vaccine
The purpose of the study is to evaluate the immunogenicity and safety of Poliorix™ when administered to healthy Chinese infants at 2, 3 and 4 months of age.
- Trial name
- NCT01021293
- Testing
- Poliorix™, Oral Poliovirus vaccine
- Where
- China
- Volunteers
- 1,101 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Spinal Muscular Atrophy
An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA)
An open-label, multi-part, first-in-human study of oral branaplam in infants with Type 1 spinal muscular atrophy. The purpose of this study was to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD)…
- Trial name
- NCT02268552
- Testing
- branaplam
- Where
- 14 sites in Belgium, Bulgaria and 5 more
- Volunteers
- 40 people
- Sponsor
- Novartis Pharmaceuticals
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
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
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
Evaluation of Integrating MIYCN Interventions in Existing CGPP of PCI
The study has two arms; both experimental and control areas will receive standard interventions given under polio eradication program of PCI.
- Trial name
- NCT03368885
- Testing
- Maternal dietary diversity, Diet diversity in complementary feeding, Exclusive Breast Feeding
- Where
- India
- Volunteers
- 2,590 people, healthy volunteers welcome
- Sponsor
- FHI 360
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
Diphtheria, Tetanus
Immunogenicity and Safety of Sanofi Pasteur Pentaxim Combined Vaccine in Infants in Thailand
The present clinical study will assess the immunogenicity and reactogenicity of Sanofi Pasteur's DTacP-IPV// PRP~T combined vaccine (Pentavac™ or Pentaxim™) as a three-dose primary vaccination at 2, 4, and 6 months of age…
- Conditions
- Diphtheria, Tetanus, Poliomyelitis, Haemophilus Infections
- Trial name
- NCT00255021
- Testing
- Diphtheria, Tetanus, Polio, Acellular Pertussis and Hib Vac
- Where
- Thailand
- Volunteers
- 186 people, healthy volunteers welcome
- Sponsor
- Sanofi
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
Diphtheria, Tetanus
Immunogenicity and Safety of Sanofi Pasteur's Combined Vaccine Given as a Three-Dose Primary Series at 2, 3,4 Months of Age and Followed by a Booster Dose Given at 16 to 17 Months of Age in Vietnamese Infants Who Previously Received a Dose of Hepatitis B Vaccine at Birth or Within 1 Week After Birth
The purpose of this study is to describe the immunogenicity and safety of Sanofi Pasteur's DTaP-IPV-Hep B-PRP-T fully liquid combined hexavalent vaccine (Hexaxim®) administered at 2, 3, and 4 months of age and at 16 to 17 months…
- Conditions
- Diphtheria, Tetanus, Pertussis, Poliomyelitis
- Trial name
- NCT02428491
- Testing
- Hexaxim®
- Where
- Vietnam
- Volunteers
- 354 people, healthy volunteers welcome
- Sponsor
- Sanofi Pasteur, a Sanofi Company
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 Atrophy, Spinal
A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
This study will evaluate the pharmacokinetics (PK) and safety of risdiplam in participants with spinal muscular atrophy (SMA) under 20 days of age at first dose.
- Trial name
- PUPFISH (NCT05808764)
- Testing
- Risdiplam
- Where
- 13 sites in United States, Belgium and 6 more
- Volunteers
- 11 people
- Sponsor
- Hoffmann-La Roche
Diphtheria, Tetanus
Lot Consistency Study of DTaP-IPV-HB-PRP~T Vaccine Administered at 2-4-6 Months of Age in Healthy Infants
The purpose of this trial is to clinically confirm that the manufacturing process of the final bulk products of the investigational DTaP-IPV-HB-PRP~T vaccine is consistent.
- Conditions
- Diphtheria, Tetanus, Pertussis, Hepatitis B
- Trial name
- NCT00404651
- Testing
- DTaP-IPV-HB-PRP~T vaccine, DTaP-HBV-IPV vaccine
- Where
- 6 sites in Mexico
- Volunteers
- 1,189 people, healthy volunteers welcome
- Sponsor
- Sanofi Pasteur, a Sanofi Company
Tetanus, Diphtheria
Immunogenicity and Safety Study of GSK Biologicals' GSK2202083A Vaccine in Healthy Infants at 2, 3 and 4 Months of Age
This study will evaluate the safety and immunogenicity of GSK Biologicals' GSK2202083A vaccine co-administered with GSK Biologicals' 10-valent pneumococcal conjugate (GSK1024850A) vaccine given as a three-dose primary vaccination…
- Conditions
- Tetanus, Diphtheria, Haemophilus Influenzae Type b, Poliomyelitis
- Trial name
- NCT00970307
- Testing
- GSK2202083A vaccine, 10-valent pneumococcal vaccine (GSK 1024850A), Infanrix hexa™
- Where
- 9 sites in Poland
- Volunteers
- 421 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Spinal Muscular Atrophy (SMA)
Spinal Muscular Atrophy (SMA) Biomarkers Study in the Immediate Postnatal Period of Development
Spinal muscular atrophy (SMA) is the leading genetic cause of death of infants. Strong preclinical evidence suggests that effective therapy must be delivered as early as possible to prevent progression of the disease.
- Trial name
- NCT01736553
- Where
- 15 sites in United States
- Volunteers
- 53 people, healthy volunteers welcome
- Sponsor
- Ohio State University
Poliomyelitis
A Clinical Trial to Evaluate the Immunogenicity and Safety of sIPV in a "2+1"Sequential Schedule With bOPV in Infants
The purpose of this phrase III clinical trial is to evaluate the immunogenicity and safety of Sabin Inactivated Poliovirus Vaccine (Vero cell) in a '2+1'sequential schedule with bivalent oral poliovirus vaccine in 2-month-old…
- Trial name
- NCT03822767
- Testing
- sIPV-sIPV-bOPV vaccination schedule, wIPV-wIPV-bOPV vaccination schedule
- Where
- China
- Volunteers
- 240 people, healthy volunteers welcome
- Sponsor
- Sinovac Biotech Co., Ltd
Pertussis, Diphtheria
Comparison of Safety and Immunogenicity of Pediacel™ and Infanrix™ IPV+Hib (Penta) Given in a 3 Dose Schedule in Infants
Pediacel™ is currently licensed in the UK for use as a 3 dose regimen for the active immunisation of infants against Diphtheria, Tetanus, Pertussis, Poliomyelitis and invasive infections caused by Haemophilus influenzae type b.
- Conditions
- Pertussis, Diphtheria, Tetanus, Poliomyelitis
- Trial name
- NCT00287092
- Testing
- DT5aP-IPV-Hib 5-component Pertussis vaccine, Infanrix® -IPV+Hib
- Where
- 14 sites in Finland, Sweden
- Volunteers
- 807 people, healthy volunteers welcome
- Sponsor
- Sanofi Pasteur, a Sanofi Company
Poliomyelitis
Studies of the Safety and Immunogenicity of a Sabin Inactivated Poliovirus Vaccine
The purpose of this study is to evaluate the safety of sIPVs of different dosages in adults, children and infants in a phase I open-label study, and then assess its immunogenicity and safety in healthy infants between 60 and 90…
- Trial name
- NCT02985320
- Testing
- Single-dose regimen of high dosage investigational sIPV, Single-dose regimen of medium dosage investigational sIPV, Three-dose regimen of high dosage investigational sIPV
- Where
- China
- Volunteers
- 708 people, healthy volunteers welcome
- Sponsor
- Sinovac Biotech Co., Ltd
Poliomyelitis, Tetanus
Immunogenicity and Safety Study in Infants of GlaxoSmithKline Biologicals' Infanrix Hexa™ (DTPa-HBV-IPV/Hib) Vaccine
This study evaluates the immunogenicity and safety of Infanrix hexa™ (DTPa-HBV-IPV/Hib) when administered as a primary vaccination course to Indian infants according to a 6-10-14 weeks or a 2-4-6 months schedule.
- Conditions
- Poliomyelitis, Tetanus, Acellular Pertussis, Haemophilus Influenzae Type b
- Trial name
- NCT01353703
- Testing
- Infanrix hexa™
- Where
- 4 sites in India
- Volunteers
- 224 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline