Clinical trials: SC Disease
Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia
Pharmacokinetics of Oral Hydroxyurea Solution
An open label, safety and pharmacokinetic study of oral hydroxyurea solution administered to children from 6 months to 17.99 years (i.e.
- Conditions
- Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia, Sickle Cell-beta-thalassemia, Sickle Cell Hemoglobin C
- Trial name
- HUPK (NCT03763656)
- Testing
- Oral Hydroxyurea (100 mg/mL) Solution
- Where
- 6 sites in Jamaica, United Kingdom
- Volunteers
- 33 people
- Sponsor
- Nova Laboratories Limited
Dehydration
Efficacy and Time and Resources for Hylenex-facilitated SC Rehydration Versus ORT for Dehydrated Children in the Emergency Department (ED)
The purpose is to evaluate the safety and efficacy as well as the time and resources needed in the ED setting to achieve rehydration in young pediatric subjects with mild to moderate dehydration using hylenex-facilitated SC…
- Trial name
- INFUSE-TM (NCT01120431)
- Testing
- Oral rehydration fluid, Isotonic hydration fluid and recombinant human hyaluronidase
- Sponsor
- Baxter Healthcare Corporation
Pelizaeus-Merzbacher Disease
Study of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) Subjects
The purpose of this study is to determine the safety and preliminary effectiveness of human central nervous system stem cells (HuCNS-SC®) transplantation in patients with Connatal Pelizaeus-Merzbacher Disease (PMD).
- Trial name
- NCT01005004
- Testing
- HuCNS-SC cells
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- StemCells, Inc.
Degenerative Disc Disease in Cervical Spine, Stenosis Cervical Spine
PMCF Study Fortilink-SC
This study is setup to be able to gather information on the safety and performance of the Fortilink-SC Ti cage of Paradigm Spine GmbH, up to one year after the surgery.
- Trial name
- NCT07834749
- Testing
- Cervical interbody fusion surgery (standalone)
- Where
- 2 sites in Spain
- Volunteers
- 50 people
- Sponsor
- BAAT Medical Products B.V.
Pelizaeus-Merzbacher Disease, PMD
Long-Term Follow-Up Study of Human Stem Cells Transplanted in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD)
The purpose of this study is to determine the long term safety and preliminary effect of HuCNS-SC cells transplanted in subjects with Connatal Pelizaeus-Merzbacher Disease (PMD).
- Trial name
- NCT01391637
- Testing
- HuCNS-SC transplant in the lead-in phase
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- StemCells, Inc.
Adenoma, Colo-rectal Cancer
A Dual Tandem Study - SC vs. CAD-EYE vs. CAD-EYE With G-EYE
Single-center, four-arm dual-tandem, randomized, open-label study involving the following devices: 1. The Standard Colonoscope is a high-definition colonoscope employing advanced optical filtering and enhancement techniques …
- Trial name
- NCT05318495
- Testing
- Standard Colonoscopy, CAD-EYE, G-EYE
- Where
- Germany
- Volunteers
- 372 people, healthy volunteers welcome
- Sponsor
- Dr. Horst Schmidt Klinik GmbH
Rhinitis, Allergic, Rhinoconjunctivitis
Efficacy and Safety Evaluation of the Treatment of Allergy Against Cupressaceae and Grasses.
A Randomized, Double-Blinded, Placebo-Controlled, Prospective, Multicenter Clinical Trial to Evaluate the Efficacy and Safety of Subcutaneous Immunotherapy in Patients With Rhinitis/Rhinoconjunctivitis With or Without Mild to…
- Conditions
- Rhinitis, Allergic, Rhinoconjunctivitis, Asthma, Allergic
- Trial name
- NCT04898283
- Testing
- 10,000 MG01 +10,000 T521, 30,000 MG01 +10,000 T521
- Where
- 27 sites in Spain
- Volunteers
- 180 people
- Sponsor
- Inmunotek S.L.
Post Menopausal Osteoporosis
Phase 2 Study of BA058 (Abaloparatide) Transdermal Delivery in Postmenopausal Women With Osteoporosis
To determine the clinical safety and efficacy of abaloparatide transdermal in otherwise healthy postmenopausal women with osteoporosis as assessed by changes in bone mineral density (BMD) and serum markers of bone metabolism when…
- Trial name
- NCT01674621
- Testing
- Abaloparatide Transdermal (50 mcg), Abaloparatide Transdermal (100 mcg), Abaloparatide Transdermal (150 mcg)
- Where
- 9 sites in United States, Denmark and 2 more
- Volunteers
- 250 people
- Sponsor
- Radius Health, Inc.
Sickle Cell Disease
A Sickle CEll Disease ComplicatioN Trial
The objective of this study is to assess the efficacy of SC411 in reducing the number of sickle cell crisis (SCC) events in sickle cell disease (SCD) subjects receiving SC411 compared to those subjects receiving placebo.
- Trial name
- ASCENT (NCT02604368)
- Testing
- SC411
- Volunteers
- 210 people
- Sponsor
- Micelle BioPharma Inc
Generalized Lipodystrophy
Immunogenicity of Metreleptin in Patients With Generalized Lipodystrophy
MYALEPT™ (metreleptin) has been approved as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy (MYALEPT Prescribing…
- Trial name
- NCT04026178
- Testing
- Metreleptin
- Where
- 8 sites in United States
- Volunteers
- 11 people
- Sponsor
- Aegerion Pharmaceuticals, Inc.
Muscular Dystrophies
A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess Effect, Safety, Tolerability and PK of Multiple SC Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for IV Dosing as an Alternative Route of Administration
The purpose of the extension phase of this study is to determine whether Drisapersen is effective in the treatment of boys with Duchenne muscular dystrophy resulting from a mutation thought to be corrected by exon 51 skipping.
- Trial name
- NCT01910649
- Testing
- Drisapersen
- Volunteers
- 12 people
- Sponsor
- BioMarin Pharmaceutical
Hemophilia A, Hemophilia B
Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B
Treatment with PF-06741086 is anticipated to demonstrate a clinically relevant advantage and/or a major contribution to patient care in comparison to current methods of treatment for hemophilia A or B because it works differently…
- Trial name
- NCT03938792
- Testing
- PF-06741086
- Where
- 62 sites in United States, Bulgaria and 18 more
- Volunteers
- 188 people
- Sponsor
- Pfizer
Generalized Lipodystrophy
Efficacy and Safety of Mibavademab in Adult and Pediatric Patients With Generalized Lipodystrophy
This study is researching a new drug called mibavademab (called "study drug"). The study involves participants with a condition called Generalized Lipodystrophy (GLD).
- Trial name
- LAGO (NCT07220785)
- Testing
- Mibavademab
- Where
- 9 sites in United States, Brazil and 1 more
- Volunteers
- 28 people
- Sponsor
- Regeneron Pharmaceuticals
Juvenile Idiopathic Arthritis
A Repeated Dose-finding Study of Sarilumab in Children and Adolescents With Systemic Juvenile Idiopathic Arthritis (SKYPS)
Primary Objective: To describe the pharmacokinetic (PK) profile of sarilumab in patients aged 1-17 years with Systemic Juvenile Idiopathic Arthritis (sJIA) in order to identify the dose and regimen for adequate treatment of this…
- Trial name
- SKYPS (NCT02991469)
- Testing
- Sarilumab SAR153191 (REGN88)
- Where
- 40 sites in Argentina, Canada and 12 more
- Volunteers
- 51 people
- Sponsor
- Sanofi
Diabetes Mellitus, Type 1
Pre-Approval Access to Golimumab Subcutaneous for the Treatment of Stage 3 Type-1 Diabetes Mellitus in Children and Young Adults
This is a Single Patient Investigational New Drug (IND) to golimumab subcutaneous (SC) for the treatment of recently diagnosed Stage 3 type-1 diabetes mellitus (T1D) in children and young adults.
- Trial name
- NCT03945903
- Testing
- Golimumab
- Where
- United States
- Sponsor
- Janssen Research & Development, LLC
Sickle Cell Disease (SCD)
An Indian Multi-centric Phase IV Study to Assess the Safety of Crizanlizumab in Sickle Cell Disease Patients
Sickle cell disease (SCD) is a genetic blood disorder. Crizanlizumab is indicated to reduce the frequency of vaso-occlusive crises (VOCs) in patients with SCD aged 16 years and older.
- Trial name
- NCT04662931
- Testing
- crizanlizumab
- Where
- 7 sites in India
- Volunteers
- 140 people
- Sponsor
- Novartis Pharmaceuticals
Opioid-use Disorder
Evaluation of Long-term Buprenorphine Plasma Exposure
The study objective is to characterise the long-term plasma exposure to buprenorphine after stopping SUBLOCADE treatment. There are no interventions planned.
- Trial name
- NCT03752528
- Testing
- Blood sample collection, Urine sample collection
- Where
- 11 sites in United States
- Volunteers
- 53 people
- Sponsor
- Indivior Inc.
Hemophilia A, Hemophilia B
A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
The purpose of this clinical trial is to learn about the safety and effects of the study medicine (called marstacimab) for the potential treatment of hemophilia in pediatric patients.
- Trial name
- BASIS KIDS (NCT05611801)
- Testing
- marstacimab
- Where
- 63 sites in United States, Argentina and 21 more
- Volunteers
- 100 people
- Sponsor
- Pfizer
Asthma
Efficacy and Safety of Omalizumab in Patients With Severe Persistent Asthma
The purpose of this study is to determine the effect of omalizumab, compared to placebo, on clinically significant asthma exacerbation rates in adolescents and adults with asthma.
- Trial name
- NCT00046748
- Testing
- Omalizumab
- Volunteers
- 484 people
- Sponsor
- Novartis Pharmaceuticals
Von Willebrand Disease (VWD)
Study to Evaluate Subcutaneous (SC) VGA039 in Patients With Von Willebrand Disease (VWD)
This is a phase 3 study that will evaluate subcutaneous (SC) VGA039 in patients with von Willebrand Disease (VWD)
- Trial name
- VIVID-6 (NCT07115004)
- Testing
- VGA039
- Where
- 32 sites in United States, Australia and 8 more
- Volunteers
- 60 people
- Sponsor
- Vega Therapeutics, Inc