Clinical trials: Infantile
Infantile Spasms
A Randomized, Controlled Trial of Ganaxolone in Patients With Infantile Spasms
The study consists of cohorts where participants are randomized, in a 2:1 ratio, to 1 of 2 sequences, A and B. In each cohort, Sequence A, comprised of participants, who will receive ascending doses of ganaxolone and ascending…
- Trial name
- NCT00441896
- Testing
- Ganaxolone
- Where
- 15 sites in United States
- Volunteers
- 57 people
- Sponsor
- Marinus Pharmaceuticals
Late Infantile Metachromatic Leukodystrophy
Long-term Metazym Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
This is a single center, open-label study of patients with late infantile MLD. All patients were previous treated 26 weeks in the phase I trial (EudraCT number: 2006-005341-11, NCT00418561).
- Trial name
- NCT00633139
- Testing
- Recombinant human Arylsulfatase A (rhASA)
- Where
- Denmark
- Volunteers
- 13 people
- Sponsor
- Shire
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
Hepatitis B
Hepatitis B Booster Study in Adolescence
This study looks at protection in 10-11 and 15-16 year olds, immunized as infants with Hepatitis B (HB) vaccine to see if they still have residual protection against Hepatitis B disease.
- Trial name
- NCT02169674
- Testing
- EngerixB, GlaxoSmithKline Vaccines
- Where
- Canada
- Volunteers
- 359 people, healthy volunteers welcome
- Sponsor
- University of British Columbia
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.
Cradle Cap, Infantile Seborrheic Dermatitis
Promiseb Topical Cream for Cradle Cap
The purpose of this study is to evaluate the efficacy and safety of Promiseb Topical Cream in cradle cap.
- Trial name
- NCT01214434
- Testing
- Promiseb Topical Cream, Bland emollient
- Where
- 4 sites in United States
- Volunteers
- 42 people
- Sponsor
- Promius Pharma, LLC
Hypophosphatasia (HPP)
Safety and Efficacy Study of Asfotase Alfa in Severely Affected Infants With Hypophosphatasia (HPP)
This clinical trial studies the safety and efficacy of asfotase alfa in infants and young children with infantile onset HPP.
- Trial name
- NCT00744042
- Testing
- asfotase alfa
- Where
- 10 sites in United States, Canada and 2 more
- Volunteers
- 11 people
- Sponsor
- Alexion 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
Nakajo-Nishimura Syndrome, Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature Syndrome
A Study of Baricitinib (LY3009104) in Adult and Pediatric Japanese Participants With NNS/CANDLE, SAVI, and AGS
The main purpose of this study is to evaluate the efficacy and safety of baricitinib in adult and pediatric Japanese participants with Nakajo-Nishimura Syndrome/chronic atypical neutrophilic dermatosis with lipodystrophy and…
- Conditions
- Nakajo-Nishimura Syndrome, Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature Syndrome, STING-Associated Vasculopathy With Onset in Infancy, Aicardi Goutieres Syndrome
- Trial name
- NCT04517253
- Testing
- Baricitinib
- Where
- 5 sites in Japan
- Volunteers
- 10 people
- Sponsor
- Eli Lilly and Company
Hemangioma of Infancy
Propranolol Versus Prednisolone for Treatment of Symptomatic Hemangiomas
Hemangiomas are relatively common lesions in infants. Most go away spontaneously after one year of life and do not need treatment.
- Trial name
- NCT00967226
- Testing
- propranolol, Prednisolone
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- Nancy Bauman
Jansky-Bielschowsky Disease, Batten Disease
An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease
The Phase 1/2 study (190-201) evaluated the efficacy and safety of a 300 mg dose of BMN 190 administered every other week (qow) to patients with CLN2.
- Conditions
- Jansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, CLN2 Disease
- Trial name
- NCT02485899
- Testing
- BMN 190, Intracerebroventricular (ICV) access device
- Where
- 4 sites in United States, Germany and 2 more
- Volunteers
- 23 people
- Sponsor
- BioMarin Pharmaceutical
Sarcoma
Surgery and/or Chemotherapy in Treating Children With Infantile, Congenital, or Childhood Fibrosarcoma
RATIONALE: Drugs used in chemotherapy work in different ways to stop tumor cells from dividing so they stop growing or die. Giving combination chemotherapy before surgery may shrink the tumor so that it can be removed.
- Trial name
- NCT00072280
- Testing
- dactinomycin, cyclophosphamide, etoposide
- Where
- 74 sites in United States, Australia and 2 more
- Volunteers
- 7 people
- Sponsor
- Children's Oncology Group
13-valent Pneumococcal Vaccine, Immunization
Study Evaluating Antibody Response Of 13-Valent Pneumococcal Conjugate Vaccine (13vPnC) 24 Months After Toddler Dose.
This is an open-label study (a study in which the doctors and participants know which drug or vaccine is being administered) in children who previously received a 4-dose series of a pneumococcal conjugate vaccine (PnC) during…
- Conditions
- 13-valent Pneumococcal Vaccine, Immunization, Safety, Antibody Response
- Trial name
- NCT01026038
- Testing
- 13-valent pneumococcal conjugate vaccine
- Where
- 25 sites in France
- Volunteers
- 263 people, healthy volunteers welcome
- Sponsor
- Pfizer
Meningococcal Immunisation, Healthy Volunteers
Study of Immunogenicity and Safety of MenQuadfi® as a Booster Vaccine in Toddlers 12 to 23 Months, Regardless of the Quadrivalent Meningococcal Conjugate Vaccine Used for Priming in Infancy
This study evaluated the immunogenicity and safety of a single dose of MenQuadfi® administered as a booster vaccine in toddlers 12 - 23 months of age in Argentina who had been primed with at least 1 dose of the quadrivalent…
- Trial name
- NCT05929651
- Testing
- MenACYW conjugate vaccine
- Where
- 2 sites in Argentina
- Volunteers
- 71 people, healthy volunteers welcome
- Sponsor
- Sanofi Pasteur, a Sanofi Company
Infantile Neronal Ceroid Lipofuscinosis
Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis
This study will examine the effectiveness of a drug called Cystagon in treating infantile neuronal ceroid lipofuscinosis (INCL), a progressive neurological disease affecting children.
- Trial name
- NCT00028262
- Testing
- Cystagon
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Development
Effect of Infant Formula on Energy Balance
The overarching goal of the research we propose here is to conduct a randomized clinical trial to specify the physiologic and behavioral mechanisms by which infant-formula composition affects all aspects of energy balance and…
- Trial name
- GRO (NCT01700205)
- Testing
- Type of Formula
- Where
- United States
- Volunteers
- 113 people, healthy volunteers welcome
- Sponsor
- Monell Chemical Senses Center
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
Hemangioma
Incidence of Hepatic Hemangiomatosis in Patients With Cutaneous Hemangiomas
This study will attempt to determine how common liver hemangiomas are in children with infantile hemangiomas by comparing liver ultrasound results in patients with 1-4 cutaneous hemangiomas, 5 or more cutaneous hemangiomas, or at…
- Trial name
- NCT00374335
- Testing
- abdominal ultrasound, Dermatologic Examination
- Where
- United States
- Volunteers
- 261 people
- Sponsor
- Children's Mercy Hospital Kansas City
Infantile Spasm
A Study to Assess Cannabidiol Oral Solution With Vigabatrin as Initial Therapy in Participants With Infantile Spasms
The primary purpose of this study was to evaluate the efficacy, safety, and tolerability of Cannabidiol Oral Solution (CBD) as adjunctive therapy with vigabatrin as initial therapy, compared to vigabatrin alone in the treatment…
- Trial name
- NCT03421496
- Testing
- Cannabidiol Oral Solution, Vigabatrin
- Where
- 5 sites in United States
- Volunteers
- 2 people
- Sponsor
- Radius Pharmaceuticals, Inc.
Infantile Neuroaxonal Dystrophy
Desipramine in Infantile Neuroaxonal Dystrophy (INAD).
This is a research study to find out if clinically prescribed desipramine is effective at improving the symptoms and slowing the progression of Infantile Neuroaxonal Dystrophy (INAD) in affected children.
- Trial name
- NCT03726996
- Testing
- Desipramine
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Duke University