Clinical trials: System Safety
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 Liver Failure, Fulminant Hepatic Failure
TReatment for ImmUne Mediated PathopHysiology
TReatment for ImmUne Mediated PathopHysiology (TRIUMPH) is a multi-center, three arm, randomized, controlled trial of immunosuppressive therapy for children with acute liver failure.
- Conditions
- Acute Liver Failure, Fulminant Hepatic Failure, Hepatic Encephalopathy, Acute Liver Injury
- Trial name
- TRIUMPH (NCT04862221)
- Testing
- High-dose methylprednisolone, Equine anti-thymocyte globulin, Prednisolone
- Where
- 17 sites in United States
- Volunteers
- 44 people
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
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.
HIV Infections
The Safety of Zidovudine Plus Interferon-Alpha in HIV-Infected Children
PRIMARY: To determine the maximum tolerated dose of interferon-alfa (IFN-A) alone and in combination with zidovudine (AZT); to assess the safety and tolerance of IFN-A alone and in combination with AZT.
- Trial name
- NCT00000967
- Testing
- Interferon alfa-2a, Zidovudine
- Where
- 9 sites in United States, Puerto Rico
- Volunteers
- 52 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Type 1 Diabetes Mellitus, Hypercalciuria
Safety and Efficacy of Chlorthalidone in Type 1 Diabetes
This open-label study will determine if chlorthalidone is safe and effective for the use of reducing urinary calcium excretion over 4 weeks in subjects with type 1 diabetes
- Trial name
- NCT03325114
- Testing
- Chlorthalidone
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- University of Rochester
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.
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
Atopic Dermatitis
Combination Therapy for Atopic Dermatitis
Atopic dermatitis is a chronic relapsing disease with acute flares. The standard therapy is to treat acute flares using topical medications.
- Trial name
- NCT00119158
- Testing
- Combination of pimecrolimus and fluticasone, pimecrolimus
- Where
- 3 sites in United States
- Volunteers
- 90 people
- Sponsor
- Children's Hospital of Philadelphia
Acute Spinal Cord Injury, Spinal Cord Injuries
Safety, Feasibility, and Efficacy of TSCS on Stabilizing Blood Pressure for Acute Inpatients With SCI
Current forms of pharmacologic and non-pharmacologic treatments for hypotension and orthostatic hypotension (OH) remain inadequate during acute inpatient rehabilitation (AIR) following a traumatic spinal cord injury (SCI).
- Conditions
- Acute Spinal Cord Injury, Spinal Cord Injuries, Neuromodulation, Traumatic Spinal Cord Injury
- Trial name
- NCT06000592
- Testing
- Digitimer
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Jill M. Wecht, Ed.D.
Type II Gaucher Disease
An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease
This exploratory trial is to prove the tolerability and safety of VGN-R08b to treat infants with type II Gaucher disease.
- Trial name
- NCT06272149
- Testing
- VGN-R08b
- Where
- China
- Volunteers
- 6 people
- Sponsor
- Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
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
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.
Atopic Dermatitis
CorEvitas International Adolescent Atopic Dermatitis (AD) Drug Safety and Effectiveness Registry
Prospective observational registry for an adolescent cohort with AD under the care of a dermatology provider. Approximately 1500 subjects and 75 clinical sites in North America and select European countries will be recruited to…
- Trial name
- NCT06238765
- Where
- United States
- Volunteers
- 1,500 people, healthy volunteers welcome
- Sponsor
- CorEvitas
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
GM2 Gangliosidosis, Niemann-Pick Type C Disease
A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease
This open-label study aims to gather long-term safety, tolerability, PK, biomarker, and clinical efficacy data relating to daily administration of Nizubaglustat in participants previously enrolled in the Phase 2 RAINBOW study…
- Trial name
- PRISMA (NCT07399704)
- Testing
- AZ-3102
- Where
- 3 sites in Brazil
- Volunteers
- 21 people
- Sponsor
- Azafaros B.V.
Juvenile Primary Fibromyalgia Syndrome (JPFS), Fibromyalgia
Safety and Efficacy of Fluoxetine in Juvenile Fibromyalgia
The purpose of this research is to conduct an open, pilot trial to assess the efficacy and safety of fluoxetine in the treatment of Juvenile Primary Fibromyalgia Syndrome (JPFS).
- Trial name
- NCT00115804
- Testing
- Fluoxetine
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Cincinnati
Leukemia, B-cell
CD19/CD22-targeted Chimeric Antigen Receptor Engineered T Cell (CART) in B-Cell Acute Lymphoblastic Leukemia.
This is a single center, open-label ,phase 1/2 study to evaluate the safety and efficacy of targeted CD19/CD22 chimeric antigen receptor engineered T cell immunotherapy (CART) in the treatment of CD19/CD22 positive Relapsed or…
- Trial name
- NCT03614858
- Testing
- CART-19/22
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Shanghai Unicar-Therapy Bio-medicine Technology Co.,Ltd
Diabetes Mellitus, Type 1
Safety And Efficacy Of Exubera Compared With Subcutaneous Human Insulin Therapy In Children And Adolescents
To assess the safety and efficacy of Exubera on pulmonary function and glycemic control in subjects ages 6-17 over a 1 year period.
- Trial name
- NCT00479258
- Testing
- Inhaled insulin (Exubera), Subcutaneous Insulin (subject's prescribed)
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Pfizer
Rhinitis, Allergic, Perennial and Seasonal
Regulatory AVAMYS Nasal Spray PMS
Non-interventional, open-label, single group, multicentric post-marketing surveillance to monitor the safety and effectiveness of AVAMYS nasal spray administered in Korean patients according to the prescribing information.
- Trial name
- NCT01001130
- Testing
- fluticasone furoate group
- Where
- South Korea
- Volunteers
- 3,244 people
- Sponsor
- GlaxoSmithKline
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