Clinical trials: Infantile
Late Infantile Metachromatic Leukodystrophy
Open-Label Extension Study of Recombinant Human Arylsulfatase A (HGT-1111) in Late Infantile MLD
This is a multi-center, open-label, extension study of patients with late infantile MLD who have previously completed clinical study HGT-MLD-048 (NCT00633139), defined as the completion of all Week 52 procedures.
- Trial name
- NCT00681811
- Testing
- HGT-1111
- Where
- Denmark
- Volunteers
- 11 people
- Sponsor
- Shire
STXBP1 Encephalopathy with Epilepsy
Natural History Study in Pediatric Patients with STXBP1 Encephalopathy with Epilepsy
This is a prospective, non-interventional, longitudinal study designed to characterize the natural history of STXBP1 related encephalopathy with epilepsy, in participants ≤ <5 years of age.
- Trial name
- NCT05462054
- Sponsor
- Capsida Biotherapeutics, Inc.
Diffuse Astrocytoma, Anaplastic Astrocytoma
Pediatric Long-Term Follow-up and Rollover Study
A roll-over study to assess long-term effect in pediatric patients treated with dabrafenib and/or trametinib.
- Conditions
- Diffuse Astrocytoma, Anaplastic Astrocytoma, Astrocytoma, Oligodendroglioma, Childhood
- Trial name
- NCT03975829
- Testing
- dabrafenib, trametinib
- Where
- 54 sites in United States, Argentina and 17 more
- Volunteers
- 163 people
- Sponsor
- Novartis Pharmaceuticals
Neisseria Meningitidis, Haemophilus Influenzae Type b
Study to Evaluate GSK Biologicals' GSK2197870A Vaccine Given as Primary Course in Infants
The purpose of this Phase II study is to evaluate the feasibility of GSK Biologicals' GSK2197870A vaccine co-administered with Wyeth-Lederle's Prevenar™ when given in healthy infants as a three-dose primary vaccination course at…
- Trial name
- NCT00871338
- Testing
- GSK2197870A, Prevenar™, Menitorix™
- Where
- 5 sites in United Kingdom
- Volunteers
- 284 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Hepatitis B
A Study to Evaluate Persistence of Hepatitis B Antibodies, Immunogenicity and Safety of Engerix™-B Kinder Challenge Dose, in Adolescents Vaccinated With Four Doses of Infanrix™ Hexa During Infancy
The purpose of this study is to assess the long-term persistence of immunity to hepatitis B in adolescents aged 14-15 years who were vaccinated with four doses of Infanrix™-Hexa in the first two years of life and to assess the…
- Trial name
- NCT02798952
- Testing
- Engerix-B Kinder
- Where
- 14 sites in Germany
- Volunteers
- 302 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets
The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency
The primary purpose of Study INZ701-104 (the ENERGY study) is to assess the safety and tolerability of INZ-701 in infants with ENPP1 Deficiency or with ABCC6 Deficiency.
- Conditions
- Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy, ATP-Binding Cassette Subfamily C Member 6 Deficiency
- Trial name
- ENERGY (NCT05734196)
- Testing
- INZ-701
- Where
- 5 sites in United States, Spain and 1 more
- Volunteers
- 9 people
- Sponsor
- Inozyme Pharma
Infantile Colic, Infantile Functional Gastrointestinal Disorders
A Phase I Study to Evaluate the Oral Absorption of Nepadutant in Infants
The purpose of the study is to evaluate the gastrointestinal absorption of nepadutant after single dose as oral solution (and the effect of age on its oral absorption) in infants.
- Trial name
- NCT00655083
- Testing
- Nepadutant
- Where
- 3 sites in United States
- Volunteers
- 21 people
- Sponsor
- Menarini Group
Seizure in Participants With Tuberous Sclerosis Complex, Seizure in Participants With Dravet Syndrome
Assessment of Adjunctive Cannabidiol Oral Solution (GWP42003-P) in Children With Tuberous Sclerosis Complex (TSC), Dravet Syndrome (DS), or Lennox-Gastaut Syndrome (LGS) Who Experience Inadequately-controlled Seizures
This study will be conducted to evaluate the safety, pharmacokinetics (PK), and efficacy of adjunctive GWP42003-P in participants < 2 years of age with tuberous sclerosis complex (TSC), Lennox-Gastaut syndrome (LGS), or Dravet…
- Conditions
- Seizure in Participants With Tuberous Sclerosis Complex, Seizure in Participants With Dravet Syndrome, Seizure in Participants With Lennox-Gastaut Syndrome
- Trial name
- NCT04485104
- Testing
- GWP42003-P
- Where
- 11 sites in United States, Italy and 1 more
- Volunteers
- 3 people
- Sponsor
- Jazz Pharmaceuticals
Cryopyrin Associated Periodic Syndrome
The Safety and Efficacy of Canakinumab in Patients Aged 4 Years or Older Diagnosed With Cryopyrin-associated Periodic Syndromes (CAPS) in Canada
This study will assess the safety, tolerability and efficacy of canakinumab treatment in Canadian patient diagnosed with cryopyrin-associated periodic syndrome over a period of 18 months.
- Trial name
- NCT01105507
- Testing
- canakinumab (company code: ACZ885D)
- Where
- 2 sites in Canada
- Volunteers
- 4 people
- Sponsor
- Novartis Pharmaceuticals
Locally Advanced or Metastatic Infantile Fibrosarcoma Harboring an NTRK Gene Fusion, Infantile Fibrosarcoma
A Study Called EPI VITRAKVI to Compare Treatment Results in Patients With Infantile Fibrosarcoma (IFS), a Type of Connective Soft Tissue Cancer, Who Received a Treatment Called Larotrectinib From a Study Called SCOUT With Patient Data From an External Database
This is an observational study in which data from the past of children and young people with a specific cancer, called NTRK gene fusion positive infantile fibrosarcoma (IFS) is studied.
- Trial name
- EPI VITRAKVI (NCT05236257)
- Testing
- Larotrectinib (Vitrakvi, BAY2757556), Standard of Care
- Where
- France
- Volunteers
- 93 people
- Sponsor
- Bayer
Hepatitis B
Persistence of Immunity Against Hepatitis B in 12-13 Year Old Adolescents After Infant Hepatitis B Vaccination
This phase IV open study will evaluate the persistence of humoral antibodies against hepatitis B as well as the immune response to a challenge dose of hepatitis B vaccine in adolescents aged 12-13 years, who received three…
- Trial name
- NCT00984139
- Testing
- Engerix™-B Kinder
- Where
- 24 sites in Germany
- Volunteers
- 306 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Infantile Neuroaxonal Dystrophy
A Study to Assess Efficacy and Safety of RT001 in Subjects With Infantile Neuroaxonal Dystrophy
The purpose of this study is to evaluate the efficacy and safety of RT001 in patients with Infantile Neuroaxonal Dystrophy (INAD).
- Trial name
- NCT03570931
- Testing
- RT001
- Where
- 2 sites in United States
- Volunteers
- 19 people
- Sponsor
- Biojiva LLC
Infantile Colic
Evaluating the Effect of Probiotic Limosilactobacillus Reuteri (L. Reuteri) on Crying and Fussing Time in Infants With Colic
This is a double-blind, randomized, placebo-controlled, parallel-group study in infants with colic with the primary objective to evaluate crying and fussing time.
- Trial name
- NCT07190859
- Testing
- L. reuteri
- Where
- 4 sites in Bulgaria
- Volunteers
- 50 people
- Sponsor
- BioGaia AB
Locally Advanced Solid Tumors, Metastatic Solid Tumors
A Study of Repotrectinib in Pediatric and Young Adult Subjects Harboring ALK, ROS1, OR NTRK1-3 Alterations
Phase 1 will evaluate the safety and tolerability at different dose levels of repotrectinib in pediatric and young adult subjects with advanced or metastatic malignancies harboring anaplastic lymphoma kinase (ALK), receptor…
- Conditions
- Locally Advanced Solid Tumors, Metastatic Solid Tumors, Lymphoma, Primary CNS Tumors
- Trial name
- NCT04094610
- Testing
- Oral repotrectinib (TPX-0005)
- Where
- 68 sites in United States, Australia and 9 more
- Volunteers
- 75 people
- Sponsor
- Turning Point Therapeutics, Inc.
Infantile Colic
Preliminary Efficacy and Safety Study of Oral Nepadutant in Infant Colic
This phase IIa study is designed as a multi-centre, multinational, randomised, double-blind, placebo controlled study in three parallel groups, with the aim to evaluate the efficacy and safety of Nepadutant given at two oral…
- Trial name
- no-cry (NCT01258153)
- Testing
- Nepadutant oral solution
- Where
- 9 sites in Germany, Poland and 2 more
- Volunteers
- 115 people
- Sponsor
- Menarini Group
Diarrhea, Infantile
Preventing Antibiotic-Associated DiarRhea Using Erceflora
To determine the effectiveness of the pre-biotic Bacillus clausii in preventing antibiotic associated diarrhea among hospitalized immunocompetent Filipino children.
- Trial name
- PADRE (NCT00447161)
- Testing
- Bacillus Clausii Multi ATB Resist
- Where
- Philippines
- Volunteers
- 323 people
- Sponsor
- Sanofi
Infantile Hemangioma
Study to Demonstrate the Efficacy and Safety of Propranolol Oral Solution in Infants With Proliferating Infantile Hemangiomas Requiring Systemic Therapy
There is an unsatisfied medical need for a first-line treatment of proliferating IHs with a good benefit/risk profile. Based on the recent findings of encouraging results obtained with propranolol in a series of infants with…
- Trial name
- NCT01056341
- Testing
- Propranolol
- Where
- 59 sites in United States, Australia and 14 more
- Volunteers
- 512 people
- Sponsor
- Pierre Fabre Dermatology
Generalized Arterial Calcification in Infancy, Autosomal Recessive Hypophosphatemic Rickets Type 2
Understanding the Spectrum of ENPP1 Deficiency and Acute ABCC6 Deficiency
To date, the investigators lack characterization of, the burden of, and the systemic progression of disease in ENPP1 deficiency and ABCC6 deficiency from a patient and/or parent perspective.
- Trial name
- NCT04372446
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Inozyme Pharma
Cradle Cap, Seborrheic Dermatitis
TRIDERM D.S. Crema Normalizzante for Neonatal Hyperseborrhea (Cradle Cap) and Associated Cutaneous Manifestations of Mild-to-Moderate Seborrheic Dermatitis in Children Aged 0 to 3 Years
This clinical study aims to evaluate the effects of using the cosmetic product TRIDERM D.S. Crema Normalizzante in children aged 0 to 3 years with neonatal hyperseborrhea ("cradle cap"), potentially associated with manifestations…
- Trial name
- NCT07860788
- Testing
- TRIDERM D.S. Crema Normalizzante
- Where
- Italy
- Volunteers
- 20 people
- Sponsor
- ICIM International S.r.l.
Glycogen Storage Disease Type II
Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa
This is a single group, treatment, Phase 3, open-label study to assess efficacy, safety, pharmacokinetic (PK), pharmacodynamics (PD) of avalglucosidase alfa in treatment-naïve male and female participants with IOPD.
- Trial name
- Baby-COMET (NCT04910776)
- Testing
- avalglucosidase alfa
- Where
- 16 sites in United States, Belgium and 7 more
- Volunteers
- 17 people
- Sponsor
- Sanofi