Clinical trials: Open Label
Leber Congenital Amaurosis, Inherited Retinal Diseases Caused by RPE65 Mutations
Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)
The purpose of the study is to determine whether HG004 as gene therapy is safe and effective for the treatment of Leber Congenital Amaurosis caused by mutations in RPE65 gene.
- Trial name
- NCT05906953
- Testing
- HG004
- Where
- 3 sites in United States, China
- Volunteers
- 20 people
- Sponsor
- HuidaGene Therapeutics Co., Ltd.
Malaria
Efficacy, Safety and Pharmacokinetic of ArtequinTM P. Falciparum Malaria
Treatment of Plasmodium falciparum malaria in Africa is increasingly difficult. Resistance to cheap efficient antimalarial drugs poses an increasing threat.
- Trial name
- NCT00243737
- Testing
- Artesunate-Mefloquine
- Where
- 2 sites in Gabon
- Volunteers
- 70 people
- Sponsor
- Albert Schweitzer Hospital
Progeria, Hutchinson-Gilford Syndrome
Phase II Trial of Lonafarnib (a Farnesyltransferase Inhibitor) for Progeria
This is an open label dose adjusted phase II trial of the oral farnesyltransferase inhibitor (FTI) lonafarnib (SCH66336) for patients with HGPS and progeroid laminopathies.
- Trial name
- NCT00425607
- Testing
- Lonafarnib
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- Monica E. Kleinman
Type 1 Diabetes Mellitus
Efficacy and Safety of Insulin Glargine Versus. Neutral Protamine Hagedorn (NPH) Insulin in Children With Type 1 Diabetes Above 6 Years Old.
Primary Objective: 6 To assess the efficacy of insulin glargine given once daily (QD) on glycosylated hemoglobin (HbA1c) levels over a period of 24 weeks in children with type 1 diabetes mellitus (T1DM) aged at least 6 years to…
- Trial name
- Lantus-P-CN (NCT01223131)
- Testing
- Insulin glargine (HOE901), NPH insulin
- Where
- 10 sites in China
- Volunteers
- 162 people
- Sponsor
- Sanofi
Alopecia Areata
A Study of ATI-50002 Topical Solution for the Treatment of Alopecia Areata
Primary: To assess safety, tolerability and efficacy of 2 dose strengths of ATI-50002 Topical Solution, compared to vehicle in subjects with alopecia areata (AA) Secondary: To evaluate key clinical outcome assessments
- Trial name
- NCT03354637
- Testing
- ATI-50002 high dose, ATI-50002 low dose
- Where
- 23 sites in United States
- Volunteers
- 129 people
- Sponsor
- Aclaris Therapeutics, Inc.
Angioedema
A Study of Long-Term Safety and Efficacy of Lanadelumab for Prevention of Acute Attacks of Non-histaminergic Angioedema With Normal C1-Inhibitor
The purpose of this study is to evaluate the long-term safety and efficacy of repeated subcutaneous (SC) administration of lanadelumab in adolescents and adults with non-histaminergic angioedema with normal C1-inhibitor who…
- Trial name
- NCT04444895
- Testing
- Lanadelumab
- Where
- 35 sites in United States, Canada and 8 more
- Volunteers
- 73 people
- Sponsor
- Shire
Plasmodium Falciparum
Comparison of Arterolane-piperaquine Versus Arterolane-piperaquine+Mefloquine Versus Artemether-lumefantrine in Kenyan Children
This open-label randomised controlled clinical trial will compare the safety, tolerability, therapeutic efficacy and pharmacokinetics and pharmacodynamics of arterolane-piperaquine, arterolane-piperaquine plus mefloquine versus…
- Trial name
- TACTKenya (NCT03452475)
- Testing
- Arterolane-piperaquine, Arterolane-piperaquine+mefloquine, Artemether-lumefantrine
- Where
- Kenya
- Volunteers
- 219 people
- Sponsor
- University of Oxford
Bipolar Disorder
Safety and Tolerability of Ziprasidone in Children and Adolescents With Bipolar I Disorder (Manic or Mixed)
The purpose of this study is to assess the safety and tolerability of ziprasidone during long-term open-label administration in children and adolescents (ages 10-17) with bipolar I disorder (manic or mixed)
- Trial name
- NCT00265330
- Testing
- Ziprasidone oral capsules
- Where
- 41 sites in United States
- Volunteers
- 169 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Human Immunodeficiency Virus
An Open Label Pharmacokinetic, Safety And Efficacy Study Of Maraviroc In Combination With Background Therapy For The Treatment Of HIV-1 Infected, CCR5 -Tropic Children
The primary purpose of this study is to determine the pharmacokinetic properties (what the body does to maraviroc) and to determine a suitable dosing schedule of maraviroc in HIV-1 infected children and adolescents.
- Trial name
- NCT00791700
- Testing
- Maraviroc
- Where
- 41 sites in United States, Brazil and 7 more
- Volunteers
- 103 people
- Sponsor
- ViiV Healthcare
Alopecia Areata
Long-Term PF-06651600 for the Treatment of Alopecia Areata
This is a global Phase 3 study to evaluate the safety and effectiveness of an investigational study drug (called PF-06651600) in adults and adolescents (12 years and older) who have alopecia areata.
- Trial name
- ALLEGRO-LT (NCT04006457)
- Testing
- PF-06651600, Tetanus and diphtheria toxoids and acellular pertussis (Tdap) vaccine, Meningococcal (groups A, C, W-135 and Y [ACWY]) oligosaccharide diphtheria CRM197 conjugate vaccine
- Where
- 144 sites in United States, Argentina and 15 more
- Volunteers
- 1,057 people
- Sponsor
- Pfizer
Pharmacokinetics
Phase 1 Pharmacokinetics of Intravenous Nexium in Children
The purpose of this research study is to evaluate how much of repeated once daily intravenous (IV, meaning through a vein) doses of esomeprazole gets into the bloodstream of hospitalized children aged 0-17 years old that require…
- Trial name
- NCT00474019
- Testing
- esomeprazole
- Where
- 15 sites in United States, Australia and 3 more
- Volunteers
- 42 people
- Sponsor
- AstraZeneca
Venous Leg Ulcer
Efficacy and Safety of URGOBD001 Compression System
Evaluation of the efficacy (wound epithelialization and time to closure) and safety (emergence and nature of adverse event) of the new URGO BD001 compression system versus a reference compression in the local treatment of venous…
- Trial name
- FUTURE (NCT05158764)
- Testing
- Venous compression system of the lower limbs stage C6 / C6r of the CEAP classification
- Where
- France
- Volunteers
- 178 people
- Sponsor
- Laboratoires URGO
Prevention of Meningococcal Infection
Persistence of Immune Response After Vaccination With MCC
Persistence of Immune response after vaccination with MCC
- Trial name
- NCT00310687
- Testing
- BLOOD DRAW
- Where
- United Kingdom
- Volunteers
- 1,244 people, healthy volunteers welcome
- Sponsor
- Novartis
Sleep Apnea, Obstructive
Safety Study of Quickclot for Bleeding Control After Adenotonsillectomy
Hemostasis will be achieved during Pediatric Tonsillectomy/Adenoidectomy with the use of QuickClot.
- Trial name
- NCT01863563
- Testing
- QuickClot
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Eastern Virginia Medical School
Leber Congenital Amaurosis
Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT)
The purpose of the study is to determine whether HG004 as gene therapy is safe and effective for the treatment of Leber Congenital Amaurosis caused by mutationsin RPE65 gene.
- Trial name
- NCT06088992
- Testing
- HG004
- Where
- China
- Volunteers
- 9 people
- Sponsor
- Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Osteoporosis
Teriparatide (Forsteo) Treatment in Postmenopausal Women: Mechanism of Action
Previously the approach to treatment of osteoporosis has been to use medications which prevent excessive resorption of bone. More recently medications that build up new bone, i.e.
- Trial name
- Forsteo (NCT01293292)
- Testing
- Teriparatide
- Where
- United Kingdom
- Volunteers
- 19 people
- Sponsor
- Sheffield Teaching Hospitals NHS Foundation Trust
Type 1 Diabetes Mellitus, Type 2 Diabetes Mellitus
Treatment Use of Generex Oral-lyn™ in Patients With Diabetes
The objective of this Treatment IND protocol is to provide Generex Oral-lyn™ to patients with serious or life-threatening Type 1 or Type 2 diabetes mellitus, with no satisfactory alternative therapy available for treatment of…
- Trial name
- NCT00948493
- Testing
- Buccal insulin spray
- Sponsor
- Generex Biotechnology Corp.
Childhood Cancer Survivors
Pilot Study of Perindopril in Childhood Cancer Survivors
The purpose of this study is to evaluate the feasibility of conducting a medical intervention trial in childhood cancer survivors with early echocardiographic evidence of cardiac remodeling.
- Trial name
- NCT01948232
- Testing
- Perindopril
- Where
- Canada
- Sponsor
- The Hospital for Sick Children
Duchenne Muscular Dystrophy
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of KER-065 administered to adult and pediatric ambulatory and nonambulatory male participants with…
- Trial name
- NCT07704099
- Testing
- KER-065
- Where
- 2 sites in New Zealand
- Volunteers
- 36 people
- Sponsor
- Keros Therapeutics, Inc.
BH4 Deficiency, Hyperphenylalaninemia
A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia
This study has been designed to demonstrate the safety, pharmacokinetics (PK) and preliminary efficacy of PTC923 (CNSA-001) in reducing blood phenylalanine concentrations in participants with hyperphenylalaninemia due to primary…
- Trial name
- NCT03519711
- Testing
- PTC923
- Where
- 4 sites in United States
- Volunteers
- 8 people
- Sponsor
- PTC Therapeutics
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