Clinical trials: Safety Study
Duchenne Muscular Dystrophy
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study.
- Trial name
- EMBARK (NCT05096221)
- Testing
- delandistrogene moxeparvovec
- Where
- 42 sites in United States, Belgium and 7 more
- Volunteers
- 126 people
- Sponsor
- Sarepta Therapeutics, Inc.
Acute Blunt Soft Tissue Injuries/Contusions
Efficacy and Safety of Diclofenac Sodium Topical Gel 1% Compared to Placebo in Subjects With Acute Blunt Trauma Injuries
This study will assess the analgesic efficacy of DSG 1% compared to placebo in the reduction of the pain associated with acute blunt trauma injuries.
- Trial name
- NCT02290821
- Where
- 10 sites in United States
- Volunteers
- 215 people
- Sponsor
- Novartis
Metastatic Prostate Cancer
64Cu-TLX592 Phase I Safety, PK, Biodistribution and Dosimetry Study (CUPID Study)
This is a Phase 1 trial of TLX592, a humanised, engineered monoclonal antibody HuX592r conjugated with a DOTA chelator and radiolabelled with 64Cu (64Cu-TLX592).
- Trial name
- CUPID (NCT04726033)
- Testing
- 64Cu-DOTA-TLX592
- Where
- 2 sites in Australia
- Volunteers
- 14 people
- Sponsor
- Telix Pharmaceuticals (Innovations) Pty Limited
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.
Hypervolemic and Euvolemic Hyponatremia
Samsca Post Marketing Surveillance Study
This Post-Marketing Surveillance will be conducted in accordance with the local regulation of New Drug Re-examination. The surveillance will be conducted for 6 years of the re-examination period (01Sep2011~31Aug2017).
- Trial name
- Samsca PMS (NCT02722863)
- Where
- 4 sites in South Korea
- Volunteers
- 908 people
- Sponsor
- Korea Otsuka Pharmaceutical Co., Ltd.
Hypoplasminogenemia, Congenital Plasminogen Deficiency
A Study of Prometic Plasminogen IV Infusion in Subjects With Hypoplasminogenemia
This is a Phase 2/3 pivotal study to evaluate pharmacokinetics (PK), efficacy, and safety of Prometic Plasminogen (Human) Intravenous Lyophilized Solution, the investigational medicinal product (IMP), in pediatric and adult…
- Trial name
- NCT02690714
- Testing
- Plasminogen (Human) intravenous
- Where
- 2 sites in United States, Norway
- Volunteers
- 15 people
- Sponsor
- Prometic Biotherapeutics, Inc.
Pompe Disease Late-Onset, Glycogen Storage Disease Type II GSD II
Extension Study of Long-term Safety and Efficacy of Myozyme for a Single Patient With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored ERT Studies.
This extension study was to monitor the long-term safety and efficacy of rhGAA treatment in a single patient with infantile-onset Pompe disease who were previously treated with rhGAA in a Genzyme study.
- Trial name
- NCT00765414
- Testing
- Myozyme
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Genzyme, a Sanofi Company
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
Cystic Fibrosis
A Phase 3 Study of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
This study will evaluate the efficacy of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function mutation (F/MF…
- Trial name
- NCT03447249
- Testing
- VX-659/TEZ/IVA, IVA
- Where
- 101 sites in United States, Australia and 9 more
- Volunteers
- 385 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Acute Pain
Pharmacokinetics and Safety of MNK-155 in Postsurgical Adolescent Subjects With Moderate to Severe Acute Pain
An open-label study of the pharmacokinetics (PK) and safety of MNK-155 in postsurgical adolescent subjects aged 12 to 17 years with moderate to severe acute pain.
- Trial name
- NCT02767349
- Testing
- MNK-155
- Where
- United States
- Sponsor
- Mallinckrodt
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
Patellofemoral Pain Syndrome
A Confirmatory Trial of Multidisciplinary Digital Therapeutics for Patellofemoral Pain Syndrome
This randomized controlled confirmatory study will be evaluating an mobile application, MORA Cure (ETH-01K), owned by EverEx, Inc., to examine safety and effectiveness in individuals with patellofemoral pain syndrome.
- Trial name
- NCT06260865
- Testing
- MORA Cure, Treatment as Usual
- Where
- 10 sites in South Korea
- Volunteers
- 216 people
- Sponsor
- EverEx Inc.
Congenital Bleeding Disorder, Haemophilia A
A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients With Severe Haemophilia A
This trial is conducted globally. The aim of the trial is to investigate safety, efficacy and pharmacokinetics (the exposure of the trial drug in the body) of NNC 0129-0000-1003 (N8-GP) in children with severe haemophilia A who…
- Trial name
- pathfinder™5 (NCT01731600)
- Testing
- turoctocog alfa pegol
- Where
- 51 sites in United States, Brazil and 14 more
- Volunteers
- 68 people
- Sponsor
- Novo Nordisk A/S
Mycobacterium Avium-intracellulare Infection, HIV Infections
The Safety and Effectiveness of Clarithromycin Plus Zidovudine or Dideoxyinosine in the Treatment of Mycobacterium Avium Complex (MAC) Infections in Children With AIDS
To evaluate three doses of clarithromycin in children with AIDS and Mycobacterium avium complex (MAC) infection who are receiving concurrent antiretroviral therapy.
- Trial name
- NCT00000971
- Testing
- Clarithromycin, Zidovudine, Didanosine
- Where
- 2 sites in United States
- Volunteers
- 24 people
- Sponsor
- Abbott
Fibromyalgia
Pregabalin In Adolescent Patients With Fibromyalgia
The main purpose of this study is to evaluate the long-term safety of pregabalin in adolescent patients who participated in the previous fibromyalgia study (A0081180) and who wish to recieve open-label pregabalin
- Trial name
- NCT01020526
- Testing
- pregabalin
- Where
- 26 sites in United States, Czechia and 1 more
- Volunteers
- 63 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Schizophrenia
Trial to Evaluate the Short-term Safety & Efficacy of Brexpiprazole Monotherapy in the Treatment of Adolescents With Schizophrenia
To determine the safety \& efficacy of brexpiprazole monotherapy in the treatment of adolescents with schizophrenia.
- Trial name
- NCT03198078
- Testing
- Brexpiprazole (OPC-34712), Aripiprazole
- Where
- United States
- Volunteers
- 316 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Cystic Fibrosis
A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
This study will evaluate the efficacy of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function mutation (F/MF…
- Trial name
- NCT03525444
- Testing
- VX-445/TEZ/IVA, IVA
- Where
- 115 sites in United States, Australia and 11 more
- Volunteers
- 405 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Autism Spectrum Disorder
Study of Cariprazine Oral Capsules or Solution to Assess Adverse Events and Change in Irritability Due to Autism Spectrum Disorder (ASD) in Participants Aged 5-17 Years With ASD
Autism spectrum disorder (ASD) consists of deficits in social, communication, and cognitive development, repetitive and stereotypic behaviors.
- Trial name
- NCT05439616
- Testing
- Cariprazine
- Where
- 36 sites in United States, Puerto Rico
- Volunteers
- 161 people
- Sponsor
- AbbVie
B-ALL
Assessment of Senl_B19 CAR-T Cells in Relapsed/Refractory CD19+ B-ALL
To evaluate the efficacy and safety of S1904 in patients with relapsed or refractory CD19+B-ALL.
- Trial name
- NCT07244406
- Testing
- S1904 CD19 CAR-T
- Where
- China
- Volunteers
- 59 people
- Sponsor
- Hebei Senlang Biotechnology Inc., Ltd.
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