Clinical trials: SC Disease
Juvenile Idiopathic Arthritis
An Open-label, Ascending, Repeated Dose-finding Study of Sarilumab in Children and Adolescents With Polyarticular-course Juvenile Idiopathic Arthritis (pcJIA)
Primary Objective: To describe the pharmacokinetic (PK) profile of sarilumab in participants aged 2-17 years with Polyarticular-course Juvenile Idiopathic Arthritis (pcJIA) in order to identify the dose and regimen for adequate…
- Trial name
- SKYPP (NCT02776735)
- Testing
- Sarilumab
- Where
- 30 sites in United States, Argentina and 13 more
- Volunteers
- 102 people
- Sponsor
- Sanofi
Eosinophilic Asthma
Randomised Clinical Trial to Investigate Efficacy and Safety of Benralizumab 30 mg SC as an add-on Therapy in Uncontrolled Eosinophilic Asthma Patients Treated With Medium-dose ICS-LABA Compared to Conventional Escalation to High-dose ICS-LABA Treatment
This study evaluates the efficacy and safety of benralizumab as an add-on therapy in uncontrolled eosinophilic asthma participants treated with medium-dose ICS-LABA compared to the conventional treatment step of escalation of…
- Trial name
- BRISOTE (NCT06750289)
- Testing
- ICS-LABA, benralizumab
- Where
- 86 sites in United States, Bulgaria and 11 more
- Volunteers
- 130 people
- Sponsor
- AstraZeneca
Brain and Central Nervous System Tumors, Chordoma
Polyvinylpyrrolidone-Sodium Hyaluronate Gel in Reducing Pain From Oral Mucositis in Young Patients With Cancer
RATIONALE: Polyvinylpyrrolidone-sodium hyaluronate gel may lessen the pain of oral mucositis, or mouth sores, in patients undergoing treatment for cancer.
- Conditions
- Brain and Central Nervous System Tumors, Chordoma, Kidney Cancer, Leukemia
- Trial name
- NCT00349024
- Testing
- polyvinylpyrrolidone-sodium hyaluronate gel
- Where
- 21 sites in Ireland, United Kingdom
- Volunteers
- 50 people
- Sponsor
- Children's Cancer and Leukaemia Group
Sickle Cell Disease, Sickle B+ Thalassemia
Adherence to HU and HRQOL in Patients With Sickle Cell Disease: An Intervention Study Using HU-Go App
This project addresses three important research questions. First, adolescents and young adults (AYA) with sickle cell disease (SCD) and their parents/caregivers will be engaged to inform the (1) domains of health-related quality…
- Conditions
- Sickle Cell Disease, Sickle B+ Thalassemia, Sickle Beta Zero Thalassemia, Sickle Cell Hemoglobin C
- Trial name
- NCT04675645
- Testing
- HU-Go app
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Systemic Lupus Erythematosus
Study of Subcutaneous (SC) Belimumab in Pediatric Participants With Systemic Lupus Erythematosus (SLE)
The purpose of this study is to evaluate the pharmacokinetics (PK), safety, and pharmacodynamics (PD) of repeat doses of 200 milligrams per milliliter (mg/mL) belimumab administered via SC injection in pediatric participants 5 to…
- Trial name
- NCT04179032
- Testing
- Belimumab
- Where
- 11 sites in United States, Argentina and 5 more
- Volunteers
- 25 people
- Sponsor
- GlaxoSmithKline
Relapsed/Refractory B-Cell Precursor Acute Lymphoblastic Leukemia, Minimal Residual Disease + B-Cell Acute Lymphoblastic Leukemia
A Study of Subcutaneous Blinatumomab in Children With R/R and and MRD+ B-Cell Precursor Acute Lymphoblastic Leukemia
The main objective of this study is to evaluate the safety and efficacy of SC blinatumomab in children below 12 years of age.
- Trial name
- NCT07134088
- Testing
- Blinatumomab
- Where
- 5 sites in United States, Japan
- Volunteers
- 104 people
- Sponsor
- Amgen
Sickle Cell Disease
Sickle Cell Disease and the Genomic and Gene Therapy Needs of Stakeholders
The primary objectives of this prospective mixed-method interview study are to use semi-structured interviews in parents of sickle cell disease (SCD) patients to describe parental attitudes of research involving genomic…
- Trial name
- NCT04416178
- Where
- United States
- Volunteers
- 352 people, healthy volunteers welcome
- Sponsor
- St. Jude Children's Research Hospital
Neuronal Ceroid Lipofuscinosis
Safety and Efficacy Study of HuCNS-SC in Subjects With Neuronal Ceroid Lipofuscinosis
The purpose of this Phase Ib study is to determine if "Human Central Nervous System Stem Cells"(HuCNS-SC) is safe to be transplanted in subjects with infantile and late infantile neuronal ceroid lipofuscinosis.
- Trial name
- NCT01238315
- Testing
- HuCNS-SC
- Where
- United States
- Sponsor
- StemCells, Inc.
Hemoglobinopathies, Sickle Cell Disease
ERN-EuroBloodNet Registry on Patients With Rare Red Blood Cell Defects and COVID-19
Patients with red blood cell disorders (RBCDs), such as Sickle cell disease (SCD) and Thalassemia, are chronic, life-threatening conditions that can become multi-organ complications over time, and are likely at an increased risk…
- Conditions
- Hemoglobinopathies, Sickle Cell Disease, Sickle Cell Anemia, Sickle Beta Thalassemia
- Trial name
- NCT06831799
- Testing
- Non applicable, is a patient registry
- Where
- Spain
- Volunteers
- 684 people
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute
Active Polyarticular Juvenile Idiopathic Arthritis
Pharmacokinetics, Efficacy and Safety of Abatacept Administered Subcutaneously (SC) in Children and Adolescents With Active Polyarticular Juvenile Idiopathic Arthritis (pJIA) and Inadequate Response (IR) to Biologic or Non Biologic Disease Modifying Anti-rheumatic Drugs (DMARDs)
The purpose of this study is to estimate Abatacept steady-state trough concentration (Cmin) at Day 113 in children and adolescents with pJIA
- Trial name
- NCT01844518
- Testing
- Abatacept
- Where
- 57 sites in United States, Argentina and 10 more
- Volunteers
- 219 people
- Sponsor
- Bristol-Myers Squibb
Juvenile Idiopathic Arthritis
A Study of Subcutaneously (SC) Administered Tocilizumab (TCZ) in Participants With Polyarticular-Course Juvenile Idiopathic Arthritis (pJIA)
This open-label, multicenter study evaluated the pharmacokinetics, pharmacodynamics and safety of SC administered TCZ in participants with pJIA.
- Trial name
- JIGSAW 117 (NCT01904279)
- Testing
- Tocilizumab
- Where
- 35 sites in United States, Argentina and 10 more
- Volunteers
- 52 people
- Sponsor
- Hoffmann-La Roche
Hereditary Angioedema
A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous CINRYZE Administration
The objectives of the study are to: 1. Evaluate the safety and tolerability of CINRYZE administered by subcutaneous injection in subjects with hereditary angioedema 2.
- Trial name
- NCT01095497
- Testing
- CINRYZE
- Where
- 7 sites in United States
- Volunteers
- 26 people
- Sponsor
- Shire
Autoimmune Encephalitis (AE)
A Study to Learn About How Well the Medicine Efgartigimod Works to Treat Autoimmune Encephalitis In Children 12 Years or Older and Adults
The POLARIS study is designed to evaluate how well efgartigimod PH20 SC may work (called "efficacy") and how safe it is for people diagnosed with Autoimmune Encephalitis (AIE).
- Trial name
- Polaris (NCT07583641)
- Testing
- Efgartigimod PH20 (ARGX-113) SC
- Where
- 2 sites in United States
- Volunteers
- 170 people
- Sponsor
- argenx
Neuronal Ceroid Lipofuscinosis
Study of HuCNS-SC Cells in Patients With Infantile or Late Infantile Neuronal Ceroid Lipofuscinosis (NCL)
Patients with infantile or late infantile NCL have either a reduced amount of, or are missing, the palmitoyl protein thioesterase 1 (PPT1) enzyme or the tripeptidyl peptidase 1 (TPP-I) enzyme.
- Trial name
- NCT00337636
- Testing
- Surgery to implant human CNS stem cells (HuCNS-SC), Medication to suppress the immune system
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- StemCells, Inc.
Psoriasis
Study to Evaluate Adverse Events and Change in Disease Activity in Participants Between 6 and 17 Years With Moderate to Severe Plaque Psoriasis Treated With Subcutaneous (SC) Injection of Risankizumab Who Have Completed Participation in Study M19-977
Psoriasis is a chronic, systemic, inflammatory disease in which skin cells build up and develop thick, red and white scaly patches on the skin.
- Trial name
- OptIMMize-2 (NCT04862286)
- Testing
- Risankizumab
- Where
- 41 sites in United States, Canada and 5 more
- Volunteers
- 132 people
- Sponsor
- AbbVie
Anemia, Sickle Cell, Sickle Cell Anemia
Phase 2 Study of MP4CO to Treat Vaso-occlusive Sickle Crisis
Sickle Cell disease is caused by an inherited hemoglobin disorder. Healthy red blood cells are discoid and can deform and move through small blood vessels to carry oxygen to all parts of the body.
- Conditions
- Anemia, Sickle Cell, Sickle Cell Anemia, Sickle Cell Disease, Sickle Cell Disorders
- Trial name
- NCT01925001
- Testing
- MP4CO, Sodium chloride solution
- Where
- 14 sites in Bahrain, Belgium and 7 more
- Sponsor
- Sangart
Sickle Cell Anemia, Hemoglobinopathy
Bone Marrow Transplant From Related Donor for Patients With High Risk Hemoglobinopathies
The major goal of this study is to determine the risks and benefits of bone marrow transplants in patients with severe thalassemia or sickle cell disease. Participation in this project will be for two years.
- Conditions
- Sickle Cell Anemia, Hemoglobinopathy, Thalassemia
- Trial name
- NCT00040469
- Testing
- Campath -1H, Dilantin, Busulfan
- Where
- 2 sites in United States
- Volunteers
- 15 people
- Sponsor
- Baylor College of Medicine
Sickle Cell Disease, Sickle Cell Anemia in Children
PINPOINT: Gaming Technology for SCD Pain
Sickle cell disease (SCD) is a common genetic disorder characterized by episodes of pain, yet programs to assist SCD adolescents with better identification and communication about pain are lacking.
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia in Children, Sickle Cell Thalassemia, Sickle Cell SC Disease
- Trial name
- Pinpoint II (NCT04579926)
- Testing
- Pinpoint app
- Where
- 2 sites in United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Klein Buendel, Inc.
Measles, Mumps
Immunogenicity and Safety of Concomitant Administration of MMR™ rHA and VARIVAX® by Intramuscular Versus Subcutaneous Route (V205C-011)
Primary objective: To compare if, when given concomitantly with VARIVAX® by the same route at 12-18 months of age using separate injection sites, a single dose of M-M-RTMII administered by IM route is as immunogenic as a single…
- Conditions
- Measles, Mumps, Varicella
- Trial name
- NCT00432523
- Testing
- M-M-R™II manufactured with recombinant Human Albumin (rHA) and VARIVAX®
- Where
- 2 sites in France, Germany
- Volunteers
- 752 people, healthy volunteers welcome
- Sponsor
- Merck Sharp & Dohme LLC
Sickle Cell Disease, Hemoglobin SC
Bone Marrow Transplantation, Hemoglobinopathies, SCALLOP
Patients are being asked to participate in this study because they have severe sickle cell anemia (SCD) with or without the beta thalassemia trait.
- Trial name
- SCALLOP (NCT00578344)
- Testing
- Busulfan, Campath 1H, Cyclophosphamide and MESNA
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Tami D. John