Clinical trials: Open Label
Cystic Fibrosis
Safety of Tobramycin Inhalation Powder (TIP) vs Tobramycin Solution for Inhalation in Patients With Cystic Fibrosis
This study compares the safety of the tobramycin solution for inhalation with the tobramycin dry powder formulation, used with a simple inhaler
- Trial name
- EAGER (NCT00388505)
- Testing
- Tobramycin Inhalation Powder, Tobramycin Solution for Inhalation
- Where
- 72 sites in United States, Australia and 12 more
- Volunteers
- 517 people
- Sponsor
- Novartis Pharmaceuticals
Epilepsy, Generalized Tonic-clonic Seizures
A Study of Levetiracetam in Japanese Pediatric Patients With Generalized Tonic-clonic Seizures
To evaluate the efficacy and safety of Levetiracetam dry syrup at doses up to 60 mg/kg/day or 3000 mg/day used as adjunctive therapy in Japanese pediatric patients aged ≥4 to <16 years with uncontrolled Generalized Tonic-Clonic…
- Trial name
- NCT01292837
- Testing
- Levetiracetam
- Where
- 26 sites in Japan
- Volunteers
- 13 people
- Sponsor
- UCB Japan Co. Ltd.
Hypertension, Pulmonary
Safety of Terbogrel in Patients With Primary Pulmonary Hypertension
Study to assess the safety of terbogrel during open-label, long-term treatment in medically stable or improving patients with primary pulmonary hypertension who have completed the 12-week, double-blind randomized trial of…
- Trial name
- NCT02223494
- Testing
- Terbogrel
- Volunteers
- 33 people
- Sponsor
- Boehringer Ingelheim
EBV Infection After Allogenic HSCT
Clinical Study of EBV-TCR-T Cells for EBV Infection After Allogenic HSCT
This is a multi-center, single arm, open-label, phase I study to determine the safety and effectiveness of EBV-TCR-T cell immunotherapy in treating EBV virus infection after allogenic HSCT.
- Trial name
- NCT06119256
- Testing
- EBV-TCR-T cells
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Chinese PLA General Hospital
Partial Onset Seizures, Secondarily Generalized Seizures
Study to Evaluate the Efficacy and Safety of Perampanel as Monotherapy or First Adjunctive Therapy in Subjects With Partial Onset Seizures With or Without Secondarily Generalized Seizures or With Primary Generalized Tonic-Clonic Seizures
This study will assess the retention rate of perampanel when given as monotherapy or first adjunctive therapy in participants with partial-onset seizures or primary generalized tonic clonic seizures.
- Conditions
- Partial Onset Seizures, Secondarily Generalized Seizures, Primary Generalized Tonic-Clonic Seizures
- Trial name
- NCT03288129
- Testing
- Perampanel
- Where
- 24 sites in United States
- Volunteers
- 54 people
- Sponsor
- Eisai Inc.
Angelman Syndrome
An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman Syndrome
A study of the safety, tolerability and pharmacokinetics of NNZ-2591 and measures of efficacy in children and adolescents with Angelman syndrome
- Trial name
- AS-001 (NCT05011851)
- Testing
- NNZ-2591
- Where
- 3 sites in Australia
- Volunteers
- 17 people
- Sponsor
- Neuren Pharmaceuticals Limited
Moderate to Severe Acute Postoperative Pain
Use of Tapentadol Oral Solution for Pain After Surgery in Children From Newborn to Less Than 2 Years Old
This is a multicenter, open-label (all people involved know the identity of the intervention), single dose trial to evaluate the pharmacokinetic (PK) profile (how drugs are absorbed in the body, how are they distributed within…
- Trial name
- NCT02221674
- Testing
- Tapentadol
- Where
- 7 sites in United States, Poland and 1 more
- Volunteers
- 40 people
- Sponsor
- Grünenthal GmbH
Juvenile Idiopathic Arthritis
A Study of RoActemra/Actemra (Tocilizumab) in Patients With Active Systemic Juvenile Idiopathic Arthritis (JIA)
This study will evaluate the efficacy and safety of RoActemra/Actemra (tocilizumab) in patients with active systemic juvenile idiopathic arthritis (sJIA) who have an inadequate clinical response to NSAIDs and corticosteroids.
- Trial name
- NCT00642460
- Testing
- tocilizumab [RoActemra/Actemra], Non-steroidal anti-inflammatory drugs (NSAIDs), methotrexate
- Where
- 54 sites in United States, Argentina and 17 more
- Volunteers
- 112 people
- Sponsor
- Hoffmann-La Roche
Type1 Diabetes Mellitus
Comparison of Real-time CGMS With Intermittently-scanned CGMS in Adolescents and Adults With Type 1 Diabetes Mellitus
Type 1 Diabetes Mellitus (T1DM) is characterized by absolute insulin deficiency. Despite multiple daily insulin injections, glycemic targets are usually not achieved in T1DM patients.Use of continuos glucose monitoring system…
- Trial name
- NCT05928260
- Testing
- Medtronic Guardian Connect Sensor 3, Abott Freestyle Libre Sensor, Self blood Glucose monitoring by a glucometer
- Where
- India
- Volunteers
- 80 people
- Sponsor
- Post Graduate Institute of Medical Education and Research, Chandigarh
Plaque Psoriasis
A Study to Assess the Efficacy and Safety of Apremilast in Japanese Pediatric Participants With Moderate to Severe Plaque Psoriasis
The primary objective of this study is to evaluate the efficacy of apremilast in children and adolescents (ages 6 through 17 years) with moderate to severe plaque psoriasis.
- Trial name
- NCT05565560
- Testing
- Apremilast
- Where
- 29 sites in Japan
- Volunteers
- 21 people
- Sponsor
- Amgen
Haemophilia B
A Research Study Investigating Nonacog Beta Pegol (N9-GP) for Treatment and Prevention of Bleedings in Chinese People With Haemophilia B
The study investigates how well the medicine called nonacog beta pegol (N9-GP) works in Chinese people with haemophilia B. Participants will be treated with N9-GP.
- Trial name
- Paradigm9 (NCT05365217)
- Testing
- Nonacog beta pegol
- Where
- 15 sites in China
- Volunteers
- 30 people
- Sponsor
- Novo Nordisk A/S
Psoriasis
Safety, Tolerability, PD & PK of Calcipotriene 0.005% Foam, Under Maximal Use in 12-16 Year Olds With Plaque Psoriasis
Calcipotriene is a vitamin D3 analog that has been used as topical therapy in adult subjects with plaque-type psoriasis since 1993.
- Trial name
- NCT01563068
- Testing
- Calcipotriene 0.005% Foam
- Where
- 9 sites in United States
- Volunteers
- 18 people
- Sponsor
- Mayne Pharma International Pty Ltd
Mitochondrial Diseases
Clinical Study of Hydroxytyrosol (HT) in Mitochondrial Diseases
Mitochondrial diseases (MDs) are the commonest group of inborn errors resulting from primary dysfunction of mitochondrial respiratory chain (MRC).
- Trial name
- NCT04543968
- Testing
- hydroxytyrosol (HT) intake, hydroxytyrosol (HT) intake and withdraw
- Where
- Hong Kong
- Volunteers
- 10 people
- Sponsor
- Hong Kong Children's Hospital
Kawasaki Disease
Efficacy of Immunoglobulin Plus Infliximab for the Early Regression of Coronary Artery Lesion in Kawasaki Disease
This study evaluates the efficacy of the addition of infliximab to conventional initial treatment (intravenous immunoglobulin [IVIG] plus aspirin) in early regression of coronary artery lesion in patients with Kawasaki disease…
- Trial name
- NCT04535518
- Testing
- IVIG, Aspirin, Infliximab
- Where
- 5 sites in China
- Sponsor
- Children's Hospital of Fudan University
Spinal Muscular Atrophy (SMA)
SPX-01 Gene Therapy in Pediatric Patients With Spinal Muscular Atrophy Type 1 (RISE)
The goal of this clinical trial is to learn about the safety, and tolerability of giving an experimental gene therapy called SPX-01 to an infant with spinal muscular atrophy (SMA) type 1.
- Trial name
- NCT07903376
- Testing
- SPX-01
- Where
- Iran
- Volunteers
- 1 people
- Sponsor
- Exir Gene Pharmed
Pulmonary Fibrosis
Long-term Extension Study to Evaluate Safety and Tolerability of Admilparant in Participants With Pulmonary Fibrosis
The purpose of this study is to evaluate the long-term safety and tolerability of Admilparant in participants who completed participation in parent studies IM027-068 (for idiopathic pulmonary fibrosis (IPF)) and IM027-1015 (for…
- Trial name
- NCT07441408
- Testing
- BMS-986278
- Where
- 275 sites in United States, Argentina and 25 more
- Volunteers
- 2,380 people
- Sponsor
- Bristol-Myers Squibb
Chronic Graft-versus-host-disease
A Study of Axatilimab at 3 Different Doses in Participants With Chronic Graft Versus Host Disease (cGVHD)
This is a Phase 2 study to evaluate the efficacy, safety, and tolerability of axatilimab at 3 different dose levels in participants with recurrent or refractory active chronic graft versus host disease (cGVHD) who have received…
- Trial name
- AGAVE-201 (NCT04710576)
- Testing
- Axatilimab
- Where
- 121 sites in United States, Australia and 14 more
- Volunteers
- 296 people
- Sponsor
- Syndax Pharmaceuticals
Syphilis, Congenital
Congenital Syphilis Treatment Trial (CONSISTENT) in Neonates
The Investigator hypothesize that the treatment efficacy will be similar in both study arms. Secondary outcomes and endpoints will characterize safety by comparing adverse events (AEs), tolerability, and adherence to therapy in…
- Trial name
- NCT07457385
- Testing
- Benzathine Penicillin, Amoxicillin
- Where
- 11 sites in United States
- Volunteers
- 374 people
- Sponsor
- University of Alabama at Birmingham
Infantile GM2 Gangliosidosis (Disorder)
First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis
GM2 gangliosidoses are a group of autosomal recessive neurodegenerative diseases characterized by a deficiency of the Hex A enzyme to catabolize GM2, thereby causing GM2 accumulation within cellular lysosomes.Hex A is composed of…
- Trial name
- NCT04798235
- Testing
- TSHA-101
- Where
- Canada
- Volunteers
- 3 people
- Sponsor
- Dr. Anupam Sehgal
Atopic Dermatitis
A Study of CM310 in Children Patients With Moderate-to-severe Atopic Dermatis
This is a multi-center, single arm, open-label study to evaluate safety and primary efficacy in children patients with moderate-to severe atopic dermatis.
- Trial name
- NCT05579925
- Testing
- CM310
- Where
- China
- Volunteers
- 24 people
- Sponsor
- Keymed Biosciences Co.Ltd
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