Clinical trials: Open Label
Neurofibromatosis Type 1
Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN
This study is designed to define a dosing regimen and assess the pharmacokinetics(PK) and safety of the granule formulation; the study will also include descriptive analyses of exploratory efficacy endpoints.
- Trial name
- SPRINKLE (NCT05309668)
- Testing
- Selumetinib granule formulation, Selumetinib capsule formulation
- Where
- 16 sites in United States, Germany and 5 more
- Volunteers
- 36 people
- Sponsor
- AstraZeneca
Acute Myeloid Leukemia in Children
CHIP-AML22/Master: An Open Label Complex Clinical Trial in Newly Diagnosed Pediatric de Novo AML Patients
The CHIP-AML22 Master protocol has the overall aim of increasing the cure rate in newly diagnosed pediatric de novo AML patients, while avoiding unnecessary toxicity.
- Trial name
- NCT05994690
- Testing
- Standard Intervention Rc, Investigational Intervention Rc, Standard Intervention Ri
- Where
- Netherlands
- Volunteers
- 905 people
- Sponsor
- Princess Maxima Center for Pediatric Oncology
X-Linked Retinitis Pigmentosa
Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR)
Phase 1 of the study is a dose escalation of the subretinal administration of AAV5-hRKp.RPGR vector to assess the safety of this vector in participants with XLRP caused by mutations in RPGR.
- Trial name
- NCT03252847
- Testing
- AAV5-RPGR
- Where
- 5 sites in United States, United Kingdom
- Volunteers
- 49 people
- Sponsor
- MeiraGTx UK II Ltd
Relapsing-remitting Multiple Sclerosis
A Study to Evaluate Pharmacokinetics, Safety, Tolerability, Immunogenicity and Pharmacodynamic Effects of Subcutaneous Ocrelizumab Administration in Children and Adolescents With Relapsing-remitting Multiple Sclerosis (RRMS)
The main purpose of this study is to evaluate the pharmacokinetics (PK) of ocrelizumab administered subcutaneously (SC) in children and adolescents aged 10 to <18 years with RRMS.
- Trial name
- Operetta III (NCT07503340)
- Testing
- Ocrelizumab co-formulated with rHuPH20
- Volunteers
- 25 people
- Sponsor
- Hoffmann-La Roche
X-Linked Hypophosphatemia
Study of the Safety, Pharmacodynamics (PD) and Efficacy of KRN23 in Children From 1 to 4 Years Old With X-linked Hypophosphatemia (XLH)
The primary objectives of the study are to: Establish the safety profile of KRN23 for the treatment of XLH in children between 1 and 4 years old Determine the PD effects of KRN23 treatment on serum phosphorus and other PD markers…
- Trial name
- NCT02750618
- Testing
- Burosumab
- Where
- 3 sites in United States
- Volunteers
- 13 people
- Sponsor
- Kyowa Kirin, Inc.
Cystic Fibrosis
Trial of Aeroquin Versus Tobramycin Inhalation Solution (TIS) in Cystic Fibrosis (CF) Patients
Patients with cystic fibrosis (CF) suffer from chronic infections of the lower respiratory tract that can be caused by one or multiple bacteria, including Pseudomonas aeruginosa, which has been particularly problematic to…
- Trial name
- TIS (NCT01270347)
- Testing
- MP-376 (Levofloxacin Solution for Inhalation), TIS (Tobramycin Inhalation Solution)
- Where
- 130 sites in United States, France and 4 more
- Volunteers
- 282 people
- Sponsor
- Amgen
Nervous System Diseases
Study of Midazolam Hydrochloride Oromucosal Solution (MHOS/SHP615) in Pediatric Patients With Status Epilepticus (Convulsive) in the Community Setting
The purpose of this study is to determine if the investigational treatment, MHOS/SHP615, is safe and effective in children with status epilepticus (SE) (convulsive) in the community setting.
- Trial name
- NCT03336450
- Testing
- SHP615, MHOS/SHP615
- Where
- 23 sites in Japan
- Volunteers
- 3 people
- Sponsor
- Shire
Surgical Procedures, Operative
The T REX Pilot Study: a Study to Investigate the Use of an Alternative Anaesthetic in Infants.
Animal studies suggest general anaesthetics harm the developing brain. It is unclear if these findings are relevant to humans but the issue has become a major concern.
- Trial name
- NCT02353182
- Testing
- Dexmedetomidine, Remifentanil, Bupivacaine
- Where
- 6 sites in United States, Australia and 1 more
- Volunteers
- 60 people
- Sponsor
- Murdoch Childrens Research Institute
Herpes Simplex Virus, Neonatal Sepsis
A Study to Describe the Pharmacokinetics of Acyclovir in Premature Infants (PTN_Acyclo)
Acyclovir is a drug used to treat herpes simplex virus (HSV) infections in babies. Appropriate dosing of acyclovir is known for adults and children but acyclovir has not been adequately studied in full-term or premature neonates.
- Trial name
- NCT00942084
- Testing
- Acyclovir
- Where
- 3 sites in United States
- Volunteers
- 32 people
- Sponsor
- Phillip Brian Smith
Short Bowel Syndrome
Long-term Safety and Efficacy Study of Teduglutide in Pediatric Participants With Short Bowel Syndrome (SBS)
This study will follow participants who completed the TED-C14-006 study. The purpose of this study is to evaluate the long-term safety and efficacy of teduglutide in pediatric participants with Short Bowel Syndrome (SBS).
- Trial name
- NCT02954458
- Testing
- TED, SOC, Syringe
- Where
- 22 sites in United States, Belgium and 4 more
- Volunteers
- 61 people
- Sponsor
- Shire
Contrast Enhancement in Magnetic Resonance Imaging, Children
A Study to Learn How Gadoquatrane Moves Into, Through, and Out of the Body and How Safe it is in Children (From Birth to <18 Years), Who Will Undergo a Contrast Enhanced MRI (Quanti Pediatric)
Researchers are looking for a better way to help children under the age of 18 with any known or suspected problems scheduled for a "contrast-enhanced" Magnetic Resonance Imaging (MRI).
- Trial name
- NCT05915026
- Testing
- Gadoquatrane (BAY1747846)
- Where
- 34 sites in United States, Argentina and 8 more
- Volunteers
- 105 people
- Sponsor
- Bayer
Mycobacterium Avium-Intracellulare Infection, HIV Infections
An Open-Label Study of the Use of Azithromycin in Patients With Symptomatic Disseminated Mycobacterium Avium-Intracellulare Complex (MAC) Infection Failing Current Therapy
To evaluate the efficacy and safety of azithromycin given chronically for the treatment of Mycobacterium avium (MAC) bacteremia in patients failing or intolerant of current available MAC therapy.
- Trial name
- NCT00002089
- Testing
- Azithromycin
- Where
- United States
- Sponsor
- Pfizer
Giant Axonal Neuropathy (GAN)
Intraneural Administration of scAAV9/JeT-GAN Into the Vagus Nerve for Patients With Giant Axonal Neuropathy (GAN)
Giant axonal neuropathy (GAN) is a rare pediatric disorder caused by autosomal recessive mutations in the GAN gene. GAN is a multisystem, neurodegenerative disorder affecting the peripheral nervous system (PNS), central nervous…
- Trial name
- NCT07543991
- Testing
- scAAV9/JeT-GAN
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- University of Texas Southwestern Medical Center
Heterozygous or Homozygous Familial Hypercholesterolemia
Long-term Safety and Tolerability of Inclisiran in Participants With HeFH or HoFH Who Have Completed the Pediatric ORION-16, ORION-13, ORION-20, or ORION-19 Studies
The purpose of this open-label, single arm, multicenter extension study is to evaluate the long-term safety and tolerability of inclisiran in participants with HeFH or HoFH who have completed the ORION-16 (CKJX839C12301)…
- Trial name
- V-PEDS-OLE (NCT05682378)
- Testing
- Inclisiran
- Where
- 52 sites in United States, Argentina and 24 more
- Volunteers
- 195 people
- Sponsor
- Novartis Pharmaceuticals
Growth Failure, Congenital Heart Disease
A Study to Evaluate the Effects of a New Formula on the Growth, Safety and Tolerance of Infants With Growth Failure
This study is a prospective, open-label, multi-site, growth, safety and tolerance study to evaluate a NF (New Formula). A minimum of 45 evaluable infants with confirmed growth failure will be enrolled.
- Trial name
- NCT03563391
- Testing
- New Infant Formula
- Where
- 5 sites in United States
- Volunteers
- 30 people
- Sponsor
- Nutricia North America
Prader-Willi Syndrome
A Study of Oral ARD-101 in Patients With Prader-Willi Syndrome
A Phase 2, Single-Arm, Open-Label Study to Evaluate the Safety and Efficacy of ARD-101 in Patients with Prader-Willi Syndrome
- Trial name
- NCT05153434
- Testing
- ARD-101
- Where
- 2 sites in United States
- Volunteers
- 19 people
- Sponsor
- Aardvark Therapeutics, Inc.
Pediatric Ulcerative Colitis
Proof of Concept Trial of Vamorolone in Pediatric Ulcerative Colitis
This is a Phase I/II, multi-center, open-label proof-of-concept study of vamorolone. Twenty participants with a flare of mild or moderately active Ulcerative Colitis (defined as a Pediatric Ulcerative Colitis Activity Index…
- Trial name
- NCT04348890
- Testing
- Vamorolone 4% suspension for oral dosing
- Sponsor
- ReveraGen BioPharma, Inc.
Dravet Syndrome, Lennox Gastaut Syndrome
A Study to Assess the Safety and Tolerability of ZX008 in Children and Young Adults With Dravet Syndrome or Lennox Gastaut Syndrome Currently Taking Cannabidiol
This is an open label study to evaluate the safety of ZX008 (fenfluramine) in patients with Dravet syndrome (DS) or Lennox Gastaut syndrome (LGS) who are being administered cannabidiol (CBD).
- Trial name
- NCT03467113
- Testing
- ZX008 0.2 to 0.8 mg/kg/day, Cannabidiol
- Where
- 2 sites in United States
- Volunteers
- 9 people
- Sponsor
- Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
Neutropenia, Severe Chronic
ANF-Rho in the Treatment of Chronic Neutropenia
A 6 month safety, tolerability, efficacy and pharmacokinetic study with ANF-Rho to treat patients with chronic neutropenia.
- Trial name
- NCT02533362
- Testing
- ANF-Rho
- Sponsor
- Prolong Pharmaceuticals
Short Bowel Syndrome
Long-term Study of Teduglutide in Pediatric Subjects With Short Bowel Syndrome Who Completed the TED-C13-003 Study
The purpose of this study is to evaluate the safety and efficacy of teduglutide treatment of children with short bowel syndrome (SBS) who completed the TED-C13-003 study over a long-term period.
- Trial name
- NCT02949362
- Testing
- Teduglutide, SOC
- Where
- 11 sites in United States, United Kingdom
- Volunteers
- 29 people
- Sponsor
- Shire
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