Clinical trials: Arm
Dravet Syndrome
A Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Fenfluramine (Hydrochloride) in Infants 1 Year to Less Than 2 Years of Age With Dravet Syndrome
The primary purpose of this study is evaluate the safety and tolerability of fenfluramine hydrochloride (HCl) 0.2 to 0.8 mg/kg/day in infants 1 year to less than 2 years of age with Dravet syndrome.
- Trial name
- ORCHID (NCT06118255)
- Testing
- fenfluramine
- Where
- 12 sites in United States, Belgium and 3 more
- Volunteers
- 25 people
- Sponsor
- UCB BIOSCIENCES, Inc.
T-ALL
CD7 CAR-T in the Treatment of CD7 Positive Refractory Relapsed Acute Leukemia
Patients with acute leukemia derived from T lymphocytes have the characteristics of high expression of CD7 antigen, such as acute T lymphocyte leukemia (T-ALL).CAR-T therapy is to genetically modify the patient's T lymphocytes to…
- Trial name
- NCT04785833
- Testing
- T cell injection targeting CD7 chimeric antigen receptor
- Where
- China
- Volunteers
- 20 people
- Sponsor
- PersonGen BioTherapeutics (Suzhou) Co., Ltd.
Ankle Fractures, Knee Fractures
Performance and Safety Follow-up of Bioabsorbable Headless Inion CompressOnTM Screws in the Fixation of Bone Fractures in Ankle, Knee and Elbow of Pediatric and Adolescent Patients
The study in question is a post market clinical follow up (PMCF) study to follow up the safety and performance of bioabsorbable headless compression screw Inion CompressOn in fracture fixation of the ankle, knee and elbow of…
- Conditions
- Ankle Fractures, Knee Fractures, Elbow Fractures
- Trial name
- NCT07473947
- Testing
- Fracture fixation operation
- Where
- Finland
- Volunteers
- 80 people
- Sponsor
- Inion Oy
Vernal Keratoconjunctivitis
Double-Masked Trial of NOVA22007 (1mg/mL Ciclosporin/Cyclosporine) Versus Vehicle in Pediatric Patients With Active Severe Vernal Keratoconjunctivitis
The objective of this study is to compare the efficacy of two different dosing regimen of NOVA22007 (1mg/ml ciclosporin/cyclosporine) eye drops, emulsion versus placebo (vehicle of the formulation) administered four times a day…
- Trial name
- NCT01751126
- Testing
- NOVA22007 ''Ciclosporin''
- Where
- 46 sites in United States, Croatia and 9 more
- Volunteers
- 169 people
- Sponsor
- Santen SAS
Severe Eosinophilic Asthma
Real World Evidence of Benralizumab in Eosinophilic Severe AsThma in Russia (BEST)
Benralizumab is a humanised, afucosylated, monoclonal antibody against the interleukin (IL)-5 receptor (IL-5R) α subunit that induces direct, rapid, and near-complete depletion of eosinophils in blood, airway tissue, and bone…
- Trial name
- NCT05271526
- Where
- Russia
- Volunteers
- 3 people
- Sponsor
- AstraZeneca
Friedreich Ataxia
A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
The primary objective of the study is to evaluate the efficacy (using the modified Friedreich Ataxia Rating Scale [mFARS]) and safety of vatiquinone in participants with Friedreich ataxia (FA).
- Trial name
- MOVE-FA (NCT04577352)
- Testing
- Vatiquinone
- Where
- 14 sites in United States, Australia and 7 more
- Volunteers
- 146 people
- Sponsor
- PTC Therapeutics
Type 2 Diabetes Mellitus
Multi-center, Single Arm, Observational Study to Evaluate the Safety of Dapagliflozin in Type 2 Diabetes Mellitus Patients in China
DONATE study is a multi-center, prospective cohort, single arm, observational study to be performed in 3000 Chinese type 2 diabetes mellitus (T2DM) patients recruited from 100 tier 2 or 3 hospitals, China.It is a drug intense…
- Trial name
- DONATE (NCT03156985)
- Where
- 83 sites in China
- Volunteers
- 3,000 people
- Sponsor
- AstraZeneca
Infant Nutritional Physiological Phenomena, Infant Formula
Effect of Partially Hydrolyzed, Whey-based Infant Formulas on Growth and Tolerability in Healthy Term Infants
This study will assess the effect of partially hydrolyzed, whey-based infant formulas on growth and gastrointestinal tolerance in healthy term infants.
- Conditions
- Infant Nutritional Physiological Phenomena, Infant Formula, Infant Health, Infant Development
- Trial name
- NCT05868408
- Testing
- Control Formula, Experimental Formula 1, Experimental Formula 2
- Where
- 5 sites in Philippines, Saudi Arabia
- Volunteers
- 234 people, healthy volunteers welcome
- Sponsor
- Société des Produits Nestlé (SPN)
Upper Extremity Impairments, Cerebral Palsy (CP)
Obi Medical Robot: Evaluating Effectiveness Related to Usability
This mixed-methods usability study evaluates the effectiveness of the Obi Gen 3 robotic feeding device in meeting user needs among providers, caregivers, and patients.
- Conditions
- Upper Extremity Impairments, Cerebral Palsy (CP), ALS - Amyotrophic Lateral Sclerosis, Rett Syndrome
- Trial name
- NCT07151950
- Testing
- One-week trial of Obi Medical device, which provides robotic assistance with feeding to persons with upper extremity impairments who are unable to self-feed in the traditional manner using utensils
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Desin LLC
Neuromyelitis Optica, Neuromyelitis Optica Spectrum Disorder
A Study of the Safety and Activity of Eculizumab in Pediatric Participants With Relapsing Neuromyelitis Optica Spectrum Disorder
The objective of this study is to evaluate the safety and efficacy of eculizumab in pediatric participants (aged 2 to < 18 years) with relapsing neuromyelitis optica spectrum disorder (NMOSD).
- Trial name
- NCT04155424
- Testing
- Eculizumab
- Where
- 35 sites in United States, Canada and 5 more
- Volunteers
- 5 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Warts Hand
Tirbanibulin for Pediatric Warts
Warts are common, benign skin lesions caused by the human papillomavirus (HPV). Treatment is challenging, particularly in the pediatric population, where standard modalities such as cryotherapy and intralesional immunotherapy are…
- Trial name
- NCT06791525
- Testing
- Tirbanibulin ointment 1%
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- The Skin Center Dermatology Group
Liver Transplantation, Infection
Efficacy and Safety of Basiliximab, Cyclosporine/Cyclosporine Microemulsion, and Steroids in Pediatric de Novo Liver Transplant Recipients Avoiding Intraoperative Steroids
Systemic infection is still a major concern in young children with liver transplantation. The approach of this study is to reduce the risk of systemic infections by avoiding intraoperative steroids (another class of…
- Trial name
- NCT00149890
- Testing
- Basiliximab, Cyclosporine/cyclosporine microemulsion, Steroid
- Where
- Germany
- Volunteers
- 77 people
- Sponsor
- Novartis
Hemophilia A
Evaluation of SCT800 in Prophylaxis Therapy on Previous Treated Patients With Severe Hemophilia A
This study is a multi-center, open-label, single-arm trial to evaluate the efficacy,safety and pharmacokinetics of SCT800 in regular prophylaxis and perioperative treatment in patients (≥12 years old) with severe hemophilia A who…
- Trial name
- NCT03815318
- Testing
- Recombinant Human Coagulation FVIII
- Where
- China
- Volunteers
- 73 people
- Sponsor
- Sinocelltech Ltd.
Multiple Myeloma
Clinical Study of T Cell Infusion Targeting BCMA Chimeric Antigen Receptor
Chimeric antigen receptor T cells (car-t) is one of the most effective therapies for malignant tumors (especially hematological tumors).
- Trial name
- NCT04650724
- Testing
- T cell infusion agent targeting BCMA chimeric antigen receptor
- Where
- China
- Volunteers
- 3 people
- Sponsor
- PersonGen BioTherapeutics (Suzhou) Co., Ltd.
Cow Milk Allergy
Growth of Infants With Cow's Milk Allergy Fed an Amino Acid-based Formula Containing Two Human Milk Oligosaccharides
The main aim of this study is to provide longitudinal growth data in infants with moderate-to-severe cow's milk allergy fed an amino acid-based formula with two added human milk oligosaccharides.
- Trial name
- PLATYPUS (NCT03661736)
- Testing
- Food for Specific Medical Purpose
- Where
- Australia
- Volunteers
- 33 people, healthy volunteers welcome
- Sponsor
- Société des Produits Nestlé (SPN)
Homozygous Familial Hypercholesterolemia
Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia
This study will evaluate the efficacy and safety of zodasiran subcutaneous (sc) injection in participants 12 to <18 years of age with genetically or clinically diagnosed homozygous familial hypercholesterolemia (HoFH) and…
- Trial name
- SPRUCE (NCT07473843)
- Testing
- Zodasiran
- Where
- 3 sites in United States, Denmark and 1 more
- Volunteers
- 10 people
- Sponsor
- Arrowhead Pharmaceuticals
Precursor Cell Lymphoblastic Leukemia-Lymphoma, Lymphoma, Non-Hodgkin
A Study to Evaluate the Safety and Efficacy of JCAR017 in Pediatric Subjects With Relapsed/Refractory (r/r) B-cell Acute Lymphoblastic Leukemia (B-ALL) and B-cell Non-Hodgkin Lymphoma (B-NHL)
This is a Phase 1/2, open-label, single arm, multicohort study to evaluate the safety and efficacy of JCAR017 in pediatric subjects aged ≤ 25 years with CD19+ r/r B-ALL and B-NHL.
- Trial name
- NCT03743246
- Testing
- JCAR017, Lymphodepleting, Fludarabine
- Where
- 19 sites in United States, France and 4 more
- Volunteers
- 21 people
- Sponsor
- Celgene
Tuberous Sclerosis Complex-associated Refractory Seizures
A Placebo-controlled Study of Efficacy & Safety of 2 Trough-ranges of Everolimus as Adjunctive Therapy in Patients With Tuberous Sclerosis Complex (TSC) & Refractory Partial-onset Seizures
This study evaluated the efficacy and safety of two trough-ranges of everolimus given as adjunctive therapy in patients with tuberous sclerosis complex (TSC) who had refractory partial-onset seizures.
- Trial name
- EXIST-3 (NCT01713946)
- Testing
- RAD001, Antiepileptic drug (1 to 3 only), open label RAD001 (only used for post-extension phase)
- Where
- 104 sites in United States, Argentina and 23 more
- Volunteers
- 366 people
- Sponsor
- Novartis Pharmaceuticals
B Cell Lymphoma
Clinical Study of U01（ssCART-19） in Patients With B-Cell Lymphoma
This is a single-arm, open-label clinical study evaluating the efficacy and safety of U01 (ssCART-19) in patients with relapsed or refractory B-cell lymphoma.
- Trial name
- NCT07093073
- Testing
- ssCART-19
- Where
- China
- Volunteers
- 30 people
- Sponsor
- Shanghai Unicar-Therapy Bio-medicine Technology Co.,Ltd
Asthma
Study of Mepolizumab Autoinjector in Asthmatics
This study is aimed to assess the correct real-world use of an autoinjector for the repeat self-administration of mepolizumab SC, so to improve subject / physician convenience and to enable repeat dose self injection themselves…
- Trial name
- NCT03099096
- Testing
- Mepolizumab
- Where
- 38 sites in United States, Australia and 5 more
- Volunteers
- 159 people
- Sponsor
- GlaxoSmithKline