Clinical trials: Infantile
Dravet Syndrome
Natural History Study of Infants and Children With Developmental and Epileptic Encephalopathies
This is a multicenter, prospective, 2-year observational study in infants and children with developmental and epileptic encephalopathies (DEEs). The DEE currently being investigated is SCN1A-positive Dravet Syndrome.
- Trial name
- ENVISION (NCT04537832)
- Testing
- No Intervention
- Where
- 16 sites in United States, Australia and 2 more
- Volunteers
- 58 people
- Sponsor
- Encoded Therapeutics
Pompe Disease Infantile-Onset
Evaluation of the Safety and Efficacy of Infantile-onset Pompe Disease Gene Therapy Drug
This study is being conducted to evaluate the safety and effectiveness of GC301 adeno-associated virus vector expressing codon-optimized human acid alpha-glucosidase (GAA) as potential gene therapy for Pompe disease.
- Trial name
- NCT05793307
- Testing
- GC301
- Where
- 5 sites in China
- Volunteers
- 16 people
- Sponsor
- GeneCradle Inc
Solid Tumors Harboring NTRK Fusion
A Study to Test the Safety and Efficacy of the Drug Larotrectinib for the Treatment of Tumors With NTRK-fusion in Children
The study is being done to test the safety of a cancer drug called larotrectinib in children. The cancer must have a change in a particular gene (NTRK1, NTRK2 or NTRK3).
- Trial name
- SCOUT (NCT02637687)
- Testing
- Larotrectinib (Vitrakvi, BAY2757556)
- Where
- 45 sites in United States, Australia and 19 more
- Volunteers
- 154 people
- Sponsor
- Bayer
Colic
The Effect of Probiotics on Symptoms of Infantile Colic
The aim of this study is to evaluate the effects of a probiotic formulation on the daily crying patterns of infants. It is hypothesized that participants given the probiotic formulation will show a significant reduction in daily…
- Trial name
- PROCOLIN (NCT05198700)
- Testing
- Probiotic Formulation
- Where
- 5 sites in Spain
- Sponsor
- Lallemand Health Solutions
GM1 Gangliosidosis, GM1 Gangliosidosis, Type I
Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis
PBGM01 is a gene therapy for GM1 gangliosidosis intended to deliver a functional copy of the GLB1 gene to the brain and peripheral tissues.
- Conditions
- GM1 Gangliosidosis, GM1 Gangliosidosis, Type I, GM1 Gangliosidosis, Type 2, Beta-Galactosidase-1 (GLB1) Deficiency
- Trial name
- Imagine-1 (NCT04713475)
- Testing
- PBGM01
- Where
- 7 sites in United States, Brazil and 2 more
- Volunteers
- 26 people
- Sponsor
- Gemma Biotherapeutics
Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 Deficiency, ATP-Binding Cassette Subfamily C Member 6 Deficiency
PROPEL - A Prospective Observational Patient Registry to Evaluate ENPP1 and ABCC6 Deficiency
The purpose of this prospective registry is to characterize the natural history of ectonucleotide pyrophosphatase/phosphodiesterase1(ENPP1) Deficiency and the infantile-onset form of adenosine triphosphate (ATP) binding cassette…
- Trial name
- NCT06302439
- Testing
- No Intervention for this observational study
- Where
- 14 sites in United States, Canada and 7 more
- Volunteers
- 54 people
- Sponsor
- Inozyme Pharma
Hygiene
Dermacyd Infantile - Acceptability.
Primary Objective: To prove the safety of the formulation in normal conditions of use.
- Trial name
- NCT00881270
- Testing
- LACTIC ACID(ND)
- Where
- Brazil
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- Sanofi
Infantile Colic
Preliminary Efficacy and Safety Study of Oral Nepadutant in Infant With Colic Not Responding to Conventional Treatment
This phase IIa study is designed as a multi-centre, single country, 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
- nocry-a (NCT01309009)
- Testing
- Nepadutant oral solution
- Sponsor
- Menarini Group
Infantile Globoid Cell Leukodystrophy
The Natural History of Infantile Globoid Cell Leukodystrophy
The purpose of this natural history study is to understand more about the progression of infantile Krabbe disease, a very rare genetic disease. There is very little published longitudinal data with only anecdotal cases.
- Trial name
- NCT00983879
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Zymenex A/S
Infantile Colic
Evaluation of Safety and Efficacy of New Infant Formula in Infantile Colic
The study is conducted to examine the safety and efficacy of a new infant formula and its effects on the gastrointestinal tolerance in infants suffering from colic.
- Trial name
- Coco (NCT01721850)
- Testing
- control formula, intervention formula 1, intervention formula 2
- Where
- 18 sites in Germany, Italy
- Volunteers
- 169 people, healthy volunteers welcome
- Sponsor
- HiPP GmbH & Co. Vertrieb KG
Diarrhea, Chronic Diarrhea
Safety, Tolerability and Preliminary Efficacy of Lenodiar Pediatric in Diarrhea
Evaluation of the efficacy of a treatment with Actitan-F, a natural molecular complex of tannins (from Agrimony and Tormentil) and flavonoids (Chamomile) in a pediatric population of children affected by acture/prolonged/chronic…
- Conditions
- Diarrhea, Chronic Diarrhea, Acute Diarrhea, Diarrhea, Infantile
- Trial name
- NCT03598010
- Testing
- Actitan-F (7dd), Actitan-F (28dd)
- Volunteers
- 240 people
- Sponsor
- Aboca Spa Societa' Agricola
Pompe Disease Infantile-Onset
Expanded Access for ATB200/AT2221 for the Treatment of IOPD
This is an expanded access program (EAP) for eligible participants designed to provide access to ATB200/AT2221.
- Trial name
- NCT04327973
- Testing
- ATB200, AT2221
- Where
- 4 sites in United States, Italy and 1 more
- Sponsor
- Amicus Therapeutics
Infantile Spasm
Tricaprilin Infantile Spasms Pilot Study
The purpose of this study is to assess the safety, tolerability, and efficacy of tricaprilin in subjects with infantile spasms. This is a single-arm, open-label, pilot study in up to 10 subjects with infantile spasms.
- Trial name
- NCT04727970
- Testing
- Tricaprilin
- Where
- 4 sites in Australia, Singapore
- Volunteers
- 8 people
- Sponsor
- Cerecin
Hypophosphatasia (HPP)
A Retrospective Study of the Natural History of Patients With Severe Perinatal and Infantile Hypophosphatasia (HPP)
This study aims to characterize the natural history of patients with severe perinatal or infantile onset HPP.
- Trial name
- NCT01419028
- Where
- 12 sites in United States, Australia and 5 more
- Volunteers
- 48 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Niemann-Pick Type C Disease
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease
- Trial name
- NCT07082725
- Testing
- Nizubaglustat
- Where
- 34 sites in United States, Argentina and 13 more
- Volunteers
- 72 people
- Sponsor
- Azafaros B.V.
Psoriasis
Exploratory Study to Investigate Efficacy of LAS41004 in a Psoriasis Plaque Test
The aim of the exploratory study is to compare the dose-related efficacy of LAS41004 in a Psoriasis Plaque Test (PPT).
- Trial name
- NCT01360944
- Testing
- LAS41004, reference
- Where
- Germany
- Volunteers
- 22 people
- Sponsor
- Almirall, S.A.
Infantile Malignant Osteopetrosis
A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis
The primary objective of this Phase 1 study is to evaluate the therapeutic safety and feasibility of the investigational product (IP), RP-L401.
- Trial name
- NCT04525352
- Testing
- RP-L401
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Metachromatic Leukodystrophy (MLD)
Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
Objectives: The overall objective is to evaluate the safety, efficacy and pharmacokinetics (PK) of rhASA treatment in patients with late infantile MLD.
- Trial name
- NCT00418561
- Testing
- rhASA - Dose Level 1, rhASA - Dose Level 2, rhASA - Dose Level 3
- Where
- Denmark
- Volunteers
- 13 people
- Sponsor
- Shire
ATP-Binding Cassette Subfamily C Member 6 Deficiency, Pseudoxanthoma Elasticum
Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ABCC6 Deficiency Causing PXE
The purpose of this study is to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics PD) of multiple ascending doses of INZ-701, an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) recombinant…
- Conditions
- ATP-Binding Cassette Subfamily C Member 6 Deficiency, Pseudoxanthoma Elasticum, Generalized Arterial Calcification of Infancy
- Trial name
- NCT05030831
- Testing
- INZ-701
- Where
- 2 sites in United States, United Kingdom
- Volunteers
- 10 people
- Sponsor
- Inozyme Pharma
Pompe Disease, Glycogen Storage Disease Type II
An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease
The purpose of this study was to evaluate the efficacy, clinical benefits and safety of a prophylactic immunomodulatory regimen given prior to first treatment with alglucosidase alfa (Myozyme®) in patients with infantile-onset…
- Trial name
- NCT00701129
- Testing
- Alglucosidase Alfa, Methotrexate, Rituximab
- Where
- 2 sites in United States
- Volunteers
- 4 people
- Sponsor
- Genzyme, a Sanofi Company