Clinical trials: Open Label
Osteopetrosis
ACTIMMUNE in Intermediate Osteopetrosis
This study evaluates the effects of ACTIMMUNE (IFN-γ1b) in children and adults with intermediate osteoporosis. All participants will receive treatment with ACTIMMUNE for 12 months.
- Trial name
- NCT02666768
- Testing
- Interferon gamma-1b
- Where
- 2 sites in United States
- Volunteers
- 5 people
- Sponsor
- Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Pneumonia
Amoxicillin Versus Benzyl Penicillin for Treatment of Children Hospitalised With Severe Pneumonia
This study seeks to determine whether clinical outcome following initial treatment of severe pneumonia with oral amoxicillin is as effective as the current standard benzyl penicillin.
- Trial name
- NCT01399723
- Testing
- Amoxicillin, Benzyl penicillin
- Where
- 6 sites in Kenya
- Volunteers
- 561 people
- Sponsor
- KEMRI-Wellcome Trust Collaborative Research Program
Cystic Fibrosis
Study of Ataluren (PTC124) in Cystic Fibrosis
The primary objective of this study is to determine the long-term safety and tolerability of ataluren in participants with nonsense mutation cystic fibrosis (nmCF) who completed participation in the double-blind study…
- Trial name
- NCT02107859
- Testing
- Ataluren
- Where
- 17 sites in United States, Belgium and 5 more
- Volunteers
- 61 people
- Sponsor
- PTC Therapeutics
Moderate to Severe Chronic Plaque-type Psoriasis
Study to Assess the Long-term Safety, Tolerability, Efficacy of Secukinumab in Pediatric Patients of Age 6 to <18 Years, With Moderate to Severe Plaque Psoriasis
This was an open-label, parallel-group, two-arm, multicenter study in pediatric subjects aged 6 years to less than 18 years, at randomization, with moderate to severe chronic plaque psoriasis.
- Trial name
- NCT03668613
- Testing
- secukinumab low dose, secukinumab high dose
- Where
- 23 sites in United States, Belgium and 7 more
- Volunteers
- 84 people
- Sponsor
- Novartis Pharmaceuticals
Hepatitis B
Durability of Antiviral Activity in Chronic HBV Patients Who Showed Complete Response in L-FMAU-301,302 or 303 Trial
The purpose of this study is to evaluate the durability of antiviral activity in chronic hepatitis B patients who showed complete response in L-FMAU-301,L-FMAU-302 or L-FMAU-303 trial.
- Trial name
- NCT00362674
- Where
- 27 sites in South Korea
- Sponsor
- Bukwang Pharmaceutical
Glycogen Storage Disease Type II Pompe Disease
Avalglucosidase Alfa Extension Study
Primary Objective: Long-term safety and pharmacokinetics (PK) of avalglucosidase alfa Secondary Objective: Long-term effect of avalglucosidase alfa on pharmacodynamic variables
- Trial name
- NEO-EXT (NCT02032524)
- Testing
- Avalglucosidase Alfa
- Where
- 17 sites in United States, Belgium and 5 more
- Volunteers
- 19 people
- Sponsor
- Genzyme, a Sanofi Company
Mucopolysaccharidosis I, Hurler's Syndrome
A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients
The purpose of this study is to determine whether the development of antibodies to laronidase in patients with MPS I receiving Aldurazyme® impairs the clearance of GAG substrate.
- Conditions
- Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie's Syndrome
- Trial name
- NCT00144768
- Testing
- laronidase
- Where
- 3 sites in United States
- Volunteers
- 25 people
- Sponsor
- Genzyme, a Sanofi Company
Autism Spectrum Disorder
Open Label Phase I hCT-MSC in Toddlers With Autism Spectrum Disorder
This is a single site, prospective study of one intravenous infusion of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) in toddlers with autism spectrum disorder (ASD).
- Trial name
- TACT (NCT04294290)
- Testing
- hCT-MSC infusion
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Duke University
Cystic Fibrosis
A Study to Evaluate ELX/TEZ/IVA on Cough and Physical Activity in Participants With Cystic Fibrosis (CF)
This study will evaluate the effects of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) on cough and physical activity using wearable technology in CF participants.
- Trial name
- NCT04969224
- Testing
- ELX/TEZ/IVA, IVA
- Where
- 19 sites in Australia, Belgium and 2 more
- Volunteers
- 82 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Continuous Renal Replacement Therapy, Hyperlactatemia
RCA for CRRT in Hyperlactatemia Patient With Increased Bleeding Risk
The purpose of this single center, randomized, control, open-labeled study is to evaluate the effect and safety of RCA versus no anticoagulation for CRRT in hyperlactatemia patients with increased bleeding risk.
- Conditions
- Continuous Renal Replacement Therapy, Hyperlactatemia, Bleeding
- Trial name
- NCT04315623
- Testing
- Regional citrate anticoagulation CRRT, No-anticoagulation CRRT
- Where
- China
- Volunteers
- 80 people
- Sponsor
- Air Force Military Medical University, China
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
Uncomplicated Plasmodium Knowlesi Malaria
P. Knowlesi Trial of Artesunate-mefloquine Versus Chloroquine
Preliminary studies have supported the background efficacy of local standard anti-malarial medications in the treatment of uncomplicated knowlesi malaria, however this has not been tested systematically and there are no current…
- Trial name
- ACT KNOW (NCT01708876)
- Testing
- Artesunate-mefloquine, Chloroquine, Primaquine
- Where
- 3 sites in Malaysia
- Volunteers
- 250 people
- Sponsor
- Menzies School of Health Research
Hemophilia A
A Study of PEGylated Recombinant Factor VIII (BAX855) in Previously Untreated Young Children With Severe Hemophilia A
This study is for young children with severe hemophilia A who have previously not been treated with BAX855 or other FVIII concentrates. The main aim of the study is to check for side effects from treatment with BAX855.
- Trial name
- NCT02615691
- Testing
- PEGylated Recombinant Factor VIII, ITI
- Where
- 91 sites in United States, Austria and 21 more
- Volunteers
- 120 people
- Sponsor
- Baxalta now part of Shire
Genetic Disease, Nonsense Mutation
A Phase 2 Study of ELX-02 in Patients With Nephropathic Cystinosis
This is a Phase 2 open label study to evaluate the safety, tolerability, PK, and PD of multiple dose levels of SC administered ELX-02 in patients with cystinosis with nonsense mutation in at least one allele.
- Conditions
- Genetic Disease, Nonsense Mutation, Cystinosis
- Trial name
- NCT04069260
- Testing
- ELX-02
- Where
- Canada
- Volunteers
- 3 people
- Sponsor
- Eloxx Pharmaceuticals, Inc.
Infection, Human Immunodeficiency Virus I, HIV Infection
Observational Study Of A Protease Inhibitor (PI) Containing Regimen In Subjects With PI Mutations Selected During Prior Treatment With GW433908 (Fosamprenavir)
This study will assess the efficacy of subsequent protease inhibitor (PI)-containing therapy in subjects who have acquired HIV-1 protease mutations whilst receiving a GW433908 (fosamprenavir)-containing regimen.
- Trial name
- NCT00242840
- Testing
- No intervention; Observational study
- Where
- 7 sites in United States, Italy
- Volunteers
- 20 people
- Sponsor
- ViiV Healthcare
B Precursor Acute Lymphoblastic Leukemia, Relapsed/Refractory B Precursor Acute Lymphoblastic Leukemia
Study of Blinatumomab Administration in Chinese Pediatric Participants With Relapsed/Refractory B Precursor Acute Lymphoblastic Leukemia (R/R B-ALL)
The primary objective of this study is to evaluate the efficacy of blinatumomab.
- Trial name
- NCT06054113
- Testing
- Blinatumomab
- Where
- 7 sites in China
- Volunteers
- 18 people
- Sponsor
- Amgen
OTC Deficiency, Ornithine Transcarbamylase Deficiency
A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD
Evaluate the safety and pharmacodynamics of multiple doses of ARCT-810 in adolescent and adult participants with OTC deficiency.
- Conditions
- OTC Deficiency, Ornithine Transcarbamylase Deficiency, OTCD
- Trial name
- NCT06488313
- Testing
- ARCT-810
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Arcturus Therapeutics, Inc.
Epilepsy
An Open-Label, Safety and Tolerability Study of Chronic Intermittent Use of Diazepam Nasal Spray.
This study will assess long-term safety and tolerability of repeat dose diazepam nasal spray in adolescents and adults with cluster seizures, with a focus on potential local effects (changes in nasal mucosa, olfaction, and taste).
- Trial name
- NCT02316847
- Testing
- diazepam nasal spray
- Where
- 33 sites in United States
- Volunteers
- 126 people
- Sponsor
- Acorda Therapeutics
Asthma
Post-marketing Surveillance (PMS) Study to Observe the Safety and Effectiveness of ARNUITY in Asthma Subjects in a Real World Setting
This is an open-label, multi-center, PMS study to observe the safety and effectiveness of ARNUITY administered in asthma subjects in a real world setting.
- Trial name
- NCT03595930
- Testing
- Arnuity
- Where
- South Korea
- Volunteers
- 668 people
- Sponsor
- GlaxoSmithKline
Pulmonary Hypertension, Sickle Cell Anemia
A Study of Patients Having Pulmonary Hypertension Associated With Sickle Cell Disease and Completing an ASSET Study
This study will assess the safety and efficacy of bosentan therapy (in a study known as ASSET) for patients who have high blood pressure in the lungs associated with sickle cell disease.
- Trial name
- ASSET-3 (NCT00360087)
- Testing
- Bosentan
- Volunteers
- 236 people
- Sponsor
- Actelion
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