Clinical trials: Infantile
Spinal Muscular Atrophy
A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy
The primary objective of the study is to examine the clinical efficacy of nusinersen (ISIS 396443) administered intrathecally (IT) to participants with infantile-onset with infantile-onset spinal muscular atrophy (SMA).
- Trial name
- ENDEAR (NCT02193074)
- Testing
- nusinersen
- Where
- 31 sites in United States, Australia and 11 more
- Volunteers
- 122 people
- Sponsor
- Biogen
Epilepsy, Dravet Syndrome
A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of TAK-935 (OV935) as an Adjunctive Therapy in Pediatric Participants With Developmental and/or Epileptic Encephalopathies
The purpose of this study is to investigate the effect on the frequency of all seizures (convulsive and drop) in participants treated with TAK-935 compared to placebo.
- Conditions
- Epilepsy, Dravet Syndrome, Lennox-Gastaut Syndrome
- Trial name
- ELEKTRA (NCT03650452)
- Testing
- TAK-935
- Where
- 44 sites in United States, Australia and 6 more
- Volunteers
- 141 people
- Sponsor
- Takeda
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
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
Spasms, Infantile
Cannabidiol Oral Solution for Treatment of Refractory Infantile Spasms
Infantile Spasms (IS) is a diagnosis described as a fairly rare and terrible form of epilepsy that usually strikes children in the first year of life. There is a great need for safe and effective therapies in the treatment of IS.
- Trial name
- NCT02551731
- Testing
- Cannabidiol Oral Solution
- Where
- 4 sites in United States
- Volunteers
- 9 people
- Sponsor
- INSYS Therapeutics Inc
Pneumococcal Disease
A Study to Assess the Antibody Response of Healthy Chinese Children Who Have Been Vaccinated Previously With 4-doses of Prevenar (a Pneumococcal Vaccine) as Babies and Toddlers
In 2005, Wyeth conducted study 0887X-101518 (NCT00488826) in which Chinese infants received either Prevenar alone (Group 1), Prevenar given with a Diphtheria, Tetanus and acellular Pertussis (DTaP) vaccine (Group 2) or DTaP alone…
- Trial name
- NCT01298544
- Testing
- Blood draw
- Where
- 10 sites in China
- Volunteers
- 335 people, healthy volunteers welcome
- Sponsor
- Pfizer
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
Infantile Spasms
Trial of Cannabidiol (CBD; GWP42003-P) for Infantile Spasms (GWPCARE7)
This trial consists of 3 parts: a pilot safety phase, a pivotal randomized controlled phase, and an open-label extension phase. The pilot phase only will be described in this record.
- Trial name
- NCT02953548
- Testing
- GWP42003-P
- Where
- 8 sites in United States, Poland
- Volunteers
- 9 people
- Sponsor
- Jazz Pharmaceuticals
Obesity
Pattern - Reach Aim 2
The purpose of this research study is to learn about different approaches to promoting healthy growth among infants, and understanding parents' likes and dislikes of an approach to reducing risk factors for obesity later in life.
- Trial name
- NCT04216264
- Testing
- Standard response, Targeted response
- Where
- United States
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- Duke University
Glycogen Storage Disease Type II-Pompe's Disease
A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa
This is a multi-stage, phase 2, open-label, multicenter, multinational, ascending dose cohort, repeated intravenous (IV) infusion study of avalglucosidase alfa in pediatric patients with Infantile-Onset Pompe Disease (IOPD) who…
- Trial name
- Mini-COMET (NCT03019406)
- Testing
- Avalglucosidase alfa (GZ402666), Alglucosidase alfa (GZ419829)
- Where
- 12 sites in United States, France and 3 more
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- Genzyme, a Sanofi Company
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
Infantile Colic
Effects of Vivatlac Baby on Crying Behavior of Colicky Babies
Open trial with two parallel arms, assessing the effects of Simethicone and Vivatlac Baby in babies diagnosed for infantile colic.
- Trial name
- NCT04487834
- Testing
- Simethicone Solution, Vivatlac Baby
- Where
- 2 sites in Poland
- Volunteers
- 87 people
- Sponsor
- The President Stanisław Wojciechowski State University of Applied Sciences in Kalisz
Diffuse Astrocytoma, Anaplastic Astrocytoma
Study of Efficacy and Safety of Dabrafenib in Combination With Trametinib in Pediatric Patients With BRAF V600 Mutation Positive LGG or Relapsed or Refractory HGG Tumors
The purpose of this study was to investigate the activity of dabrafenib in combination with trametinib in children and adolescent patients with BRAF V600 mutation positive low grade glioma (LGG) or relapsed or refractory high…
- Conditions
- Diffuse Astrocytoma, Anaplastic Astrocytoma, Astrocytoma, Oligodendroglioma, Childhood
- Trial name
- NCT02684058
- Testing
- Dabrafenib, trametinib, Carboplatin
- Where
- 57 sites in United States, Argentina and 18 more
- Volunteers
- 151 people
- Sponsor
- Novartis Pharmaceuticals
Feeding Behavior, Feeding Disorder of Infancy or Early Childhood
iKanEat: A Randomized-controlled, Multi-center Trial of Megestrol for Chronic Oral Food Refusal in Children
This is a multi-center, randomized, placebo-controlled, double-blind clinical trial. The primary focus of the study is the evaluation of the effectiveness of treatment with megestrol as part of a 24 week behavioral feeding…
- Trial name
- iKanEat (NCT03815019)
- Testing
- Megestrol Acetate, iKanEat Behavioral Intervention
- Where
- 8 sites in United States
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- University of Kansas Medical Center
Medullary Thyroid Cancer, Infantile Myofibromatosis
A Study of Oral LOXO-292 (Selpercatinib) in Pediatric Participants With Advanced Solid or Primary Central Nervous System (CNS) Tumors
This is an open-label, multi-center Phase 1/2 study of oral LOXO-292 in pediatric participants with an activating rearranged during transfection (RET) alteration and an advanced solid or primary CNS tumor.
- Conditions
- Medullary Thyroid Cancer, Infantile Myofibromatosis, Infantile Fibrosarcoma, Papillary Thyroid Cancer
- Trial name
- LIBRETTO-121 (NCT03899792)
- Testing
- Selpercatinib
- Where
- 26 sites in United States, Australia and 9 more
- Volunteers
- 36 people
- Sponsor
- Eli Lilly and Company
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
Infantile Hemangioma
Assessing the Impact of Dosage Frequency of Propranolol on Sleep Patterns in Patients With Infantile Hemangiomas
The purpose of this study is to assess the baseline sleep pattern disruption for patients starting oral propranolol at the standard BID dosing regimen compared to the control (timolol) group and to determine if there is a…
- Trial name
- NCT05479123
- Testing
- Propranolol three times a day, Propranolol twice a day, Timolol
- Where
- United States
- Volunteers
- 52 people
- Sponsor
- The University of Texas Health Science Center, Houston
Central Nervous System Neoplasm, Infantile Fibrosarcoma
Larotrectinib in Treating Patients With Previously Untreated TRK Fusion Solid Tumors and TRK Fusion Relapsed Acute Leukemia
This phase II trial studies the side effects and how well larotrectinib works in treating patients with previously untreated TRK fusion solid tumors and TRK fusion acute leukemia that has come back.
- Conditions
- Central Nervous System Neoplasm, Infantile Fibrosarcoma, Recurrent Acute Leukemia, Refractory Acute Leukemia
- Trial name
- NCT03834961
- Testing
- Larotrectinib Sulfate
- Where
- 80 sites in United States, Canada
- Volunteers
- 33 people
- Sponsor
- Children's Oncology Group
Peroxisome Biogenesis Disorders
Betaine and Peroxisome Biogenesis Disorders
The PBD are a rare group of inherited disorders due to the failure to form functional cellular peroxisomes. Most patients have progressive hearing and visual loss, leading to deafness and blindness, as well as neurological…
- Trial name
- NCT01838941
- Testing
- Betaine
- Where
- Canada
- Volunteers
- 12 people
- Sponsor
- McGill University Health Centre/Research Institute of the McGill University Health Centre
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