Clinical trials: Infantile
Jansky-Bielschowsky Disease, Batten Disease
A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease
This Phase 2 open-label, multicenter study will evaluate the safety, tolerability, and efficacy of BMN 190 intracerebroventricular (ICV) administration every other week (qow) for a period of 144 weeks, in patients with CLN2.
- Conditions
- Jansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, CLN2 Disease
- Trial name
- NCT02678689
- Testing
- BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1), Intracerebroventricular access device
- Where
- 4 sites in United States, Germany and 2 more
- Volunteers
- 14 people
- Sponsor
- BioMarin Pharmaceutical
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
Infantile Spasms
Phase 3 Trial of Cannabidiol (CBD; GWP42003-P) for Infantile Spasms: Open-label Extension Phase (GWPCARE7)
This trial consists of 3 parts: a pilot safety phase, a pivotal randomized controlled phase, and an open-label extension phase. The open-label extension phase only will be described in this record.
- Trial name
- NCT02954887
- Testing
- GWP42003-P
- Where
- 7 sites in United States, Poland
- Volunteers
- 9 people
- Sponsor
- Jazz Pharmaceuticals
Hemangioma
Clinical Trial of Vincristine vs. Prednisolone for Treatment of Complicated Hemangiomas
The goal of this study is to determine the safety and efficacy of Prednisolone and Vincristine for treatment of large, complicated infantile hemangiomas.
- Trial name
- NCT00555464
- Testing
- Vincristine, Prednisone
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Medical College of Wisconsin
Pompe Disease (Infantile-Onset), Glycogen Storage Disease Type II (GSD II)
A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease
A study to demonstrate comparable safety, efficacy, and pharmacokinetics (PK) of alglucosidase alfa manufactured at the 160 litre (L) and 4000 L scales in participants who had been diagnosed with infantile-onset Pompe disease.
- Conditions
- Pompe Disease (Infantile-Onset), Glycogen Storage Disease Type II (GSD II), Glycogenosis 2, Acid Maltase Deficiency
- Trial name
- NCT01597596
- Testing
- alglucosidase alfa
- Where
- 17 sites in United States, Germany and 1 more
- Volunteers
- 4 people
- Sponsor
- Genzyme, a Sanofi Company
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
Hemangioma
Efficacy and Safety of Propranolol Versus Atenolol on the Proliferative Phase of Infantile Hemangioma
The purpose of this study is to compare the efficacy of orally administered propranolol versus atenolol in the treatment of potentially disfiguring or functionally threatening IHs.
- Trial name
- NCT02342275
- Testing
- Propranolol, Atenolol
- Where
- China
- Volunteers
- 377 people, healthy volunteers welcome
- Sponsor
- West China Hospital
Jansky-Bielschowsky Disease, Batten Disease
A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease
The purpose of this study is to determine whether BMN 190 is safe and effective in the treatment of patients with Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) disease.
- Conditions
- Jansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, CLN2 Disease
- Trial name
- NCT01907087
- Testing
- BMN 190
- Where
- 5 sites in United States, Germany and 2 more
- Volunteers
- 24 people
- Sponsor
- BioMarin Pharmaceutical
Metachromatic Leukodystrophy (MLD)
A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy
The main aim of the study is to determine if SHP611 given by injection into the spinal fluid that surrounds the brain and spinal cord (intrathecal; IT) prolongs the time for children with Metachromatic Leukodystrophy (MLD) to…
- Trial name
- EMBOLDEN (NCT03771898)
- Testing
- SHP611
- Where
- 30 sites in United States, Argentina and 12 more
- Volunteers
- 36 people
- Sponsor
- Shire
Infantile Hemangioma
Efficacy, Safety and Pharmacokinetics of Topical Timolol in Infants With Infantile Hemangioma (IH)
The purpose of this study is to assess the safety and efficacy of Timolol 0.25% and 0.5% doses.
- Trial name
- TIM01 (NCT02913612)
- Testing
- 0.25% Timolol Maleate Gel Forming Solution, 0.5% Timolol Maleate Gel Forming Solution
- Where
- 12 sites in United States
- Volunteers
- 105 people
- Sponsor
- Kanecia Obie Zimmerman
Hemangioma, Infant
Topical Timolol 0.5% Solution for Proliferating Infantile Hemangiomas
The purpose of this study is to learn about a new potential use for topical timolol 0.5% aqueous solution that may help treat small uncomplicated infantile hemangiomas.
- Trial name
- NCT01147601
- Testing
- topical 0.5% Timolol
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Oregon Health and Science University
Pompe Disease, Glycogen Storage Disease Type II (GSD-II)
Growth and Development Study of Alglucosidase Alfa
Pompe disease (also known as glycogen storage disease Type II) is a rare autosomal recessive metabolic muscle disease caused by the deficiency of acid α glucosidase (GAA), an enzyme that degrades lysosomal glycogen.
- Conditions
- Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease
- Trial name
- NCT00486889
- Testing
- alglucosidase alfa
- Where
- 3 sites in United States
- Volunteers
- 12 people
- Sponsor
- Genzyme, a Sanofi Company
Diarrhea
PROMISE EBF: Safety and Efficacy of Exclusive Breastfeeding Promotion in the Era of HIV in Sub-Saharan Africa
The objective of the project is to develop and test an intervention to promote exclusive breastfeeding (EBF), to assess its impact on infant health in African contexts where a high prevalence of HIV is a barrier, and to…
- Trial name
- NCT00397150
- Testing
- Peer-support for exclusive breastfeeding
- Where
- 4 sites in Burkina Faso, South Africa and 2 more
- Volunteers
- 2,579 people, healthy volunteers welcome
- Sponsor
- Centre For International Health
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