Clinical trials: Systemic Therapy
- Condition Systemic Therapy
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
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.
Pompe Disease
Alglucosidase Alfa Pompe Safety Sub-Registry
To collect uniform and meaningful data on patients with Pompe disease who experience anaphylaxis, severe allergic reactions, and/or signals of severe cutaneous and/or systemic immune complex-mediated reactions following treatment…
- Trial name
- NCT01710813
- Testing
- alglucosidase alfa
- Where
- 18 sites in United States, Belgium and 4 more
- Volunteers
- 110 people
- Sponsor
- Genzyme, a Sanofi Company
Systemic Lupus Erythematosus
A Study to Evaluate the Safety and Tolerability of Dapirolizumab Pegol in Study Participants With Systemic Lupus Erythematosus
The purpose of this study is to evaluate long-term safety and tolerability of dapirolizumab pegol treatment.
- Trial name
- NCT04976322
- Testing
- Dapirolizumab pegol
- Where
- 123 sites in United States, Argentina and 22 more
- Volunteers
- 760 people
- Sponsor
- UCB Biopharma SRL
Lupus Nephritis
A Study to Evaluate the Safety and Efficacy of Tacrolimus for Lupus Nephritis Under Actual Use Situations
To evaluate the safety and efficacy of tacrolimus for lupus nephritis under actual-use.
- Trial name
- TRUST (NCT01410747)
- Testing
- Tacrolimus
- Where
- 8 sites in Japan
- Volunteers
- 1,484 people
- Sponsor
- Astellas Pharma Inc
Adolescent Lupus Nephritis, Pediatric Lupus Nephritis
Long-Term Voclosporin Treatment in Adolescent and Pediatric Subjects With Lupus Nephritis
The purpose of this study is to assess the long-term safety and tolerability of voclosporin for up to an additional 12 months following completion of treatment in the AUR-VCS-2020-03 study (VOCAL) in adolescent and pediatric…
- Trial name
- VOCAL-EXT (NCT05962788)
- Testing
- voclosporin
- Where
- 6 sites in United States, Colombia and 3 more
- Volunteers
- 4 people
- Sponsor
- Aurinia Pharmaceuticals Inc.
Raynaud's Disease, Raynaud's Disease Secondary to Scleroderma
A Study of a Topical Form of Nitroglycerin and Placebo in the Treatment and Prevention of Raynaud's Phenomenon
The purpose of this research study is to test the safety, tolerability, and effectiveness of Topical AmphiMatrix with Nitroglycerin (MQX0503) to relieve Raynaud's symptoms and increase blood flow to the fingers.
- Conditions
- Raynaud's Disease, Raynaud's Disease Secondary to Scleroderma, Raynaud's Disease Secondary to Other Autoimmune Disease
- Trial name
- NCT00577304
- Testing
- Nitroglycerin, Topical AmphiMatrix
- Where
- 17 sites in United States
- Volunteers
- 200 people
- Sponsor
- MediQuest Therapeutics
Still's Disease, Adult-Onset, Still's Disease, Juvenile-Onset
A Study to Evaluate Efficacy and Safety of Anakinra in the Treatment of Still's Disease (SJIA and AOSD)
The aim of this study is to demonstrate the efficacy and to evaluate the safety, pharmacokinetics (PK) and immunogenicity of anakinra in patients with newly diagnosed Still's disease, including SJIA (Systemic juvenile idiopathic…
- Trial name
- anaSTILLs (NCT03265132)
- Testing
- anakinra
- Where
- 39 sites in United States, Canada
- Volunteers
- 13 people
- Sponsor
- Swedish Orphan Biovitrum
Non-Segmental Vitiligo
A Study to Learn How Effective, Safe, and Tolerable Upadacitinib is for Young Participants With Non-segmental Vitiligo
Vitiligo is a common, long-lasting disease that happens when the immune system attacks melanocytes (skin cells that produce the skin-coloring pigment melanin) in the skin leading to loss of color.
- Trial name
- Ph3 Peds Viti (NCT07896434)
- Testing
- Upadacitinib
- Volunteers
- 154 people
- Sponsor
- AbbVie
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
Limb-girdle Muscular Dystrophy
A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4
This is a multicenter, global study of the effects of a single systemic dose of SRP-9003 on beta-sarcoglycan (β-SG) gene expression in participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4).
- Trial name
- EMERGENE (NCT06246513)
- Testing
- SRP-9003, Glucocorticoid
- Where
- 10 sites in United States, Belgium and 4 more
- Volunteers
- 17 people
- Sponsor
- Sarepta Therapeutics, Inc.
Candidates for Systemic Therapy for Psoriasis, Psoriasis
A Multicentre, Open Label Phase IIIb/IV Study of Subcutaneously Administered Raptiva in the Treatment of Adult Subjects With Moderate to Severe Plaque Psoriasis
An open-label, multi-center study to establish psoriasis control of moderate to severe plaque psoriasis with Raptiva therapy administered subcutaneously for 24 weeks.
- Trial name
- NCT00287118
- Testing
- Efalizumab
- Where
- Switzerland
- Volunteers
- 189 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Acute Kidney Injury, Renal Failure
Analysis of Continuous Kidney Replacement Therapy (CKRT) Treatments With multiFlux Hemofilters Using Regional Citrate Anticoagulation (RCA) or Systemic Heparin Anticoagulation
The goal of this observational study is to assess the performance of the multiFlux hemofilterwith regional or systemic anticoagulation during CKRT treatments.
- Trial name
- RCA-Flux (NCT07679568)
- Testing
- multiFlux 160 hemofilter with regional heparin anticoagulation, multiFlux 160 hemofilter with RCA with regional citrate anticoagulation, multiFlux 130 hemofilter with regional heparin anticoagulation
- Where
- Germany
- Volunteers
- 62 people
- Sponsor
- Fresenius Medical Care Deutschland GmbH
Chronic Graft-versus-host-disease
A Ph1b/2 Study of Axatilimab Monotherapy in Chinese Participants With Recurrent or Refractory cGVHD
This study will be conducted to evaluate the safety, efficacy, and pharmacokinetics of axatilimab monotherapy in Chinese participants with recurrent or refractory active chronic graft-versus-host disease after systemic therapy.
- Trial name
- NCT06843408
- Testing
- INCA034176
- Where
- 9 sites in China
- Volunteers
- 32 people
- Sponsor
- Incyte Corporation
Septic Shock, Acute Respiratory Distress Syndrome
CytOSorb TreatMent Of Critically Ill PatientS Registry
Registry intended to provide a data repository and reporting infrastructure for the surveillance of CytoSorb device use in real-world critical care settings, and to serve as an objective, comprehensive, and scientifically-based…
- Conditions
- Septic Shock, Acute Respiratory Distress Syndrome, Trauma, Rhabdomyolysis
- Trial name
- COSMOS (NCT05146336)
- Testing
- CytoSorb
- Where
- 28 sites in Austria, Germany and 4 more
- Volunteers
- 3,000 people
- Sponsor
- CytoSorbents, Inc
Psoriasis
Ustekinumab Safety and Surveillance Program Using the Ingenix NHI Database
The patients included in this observational study will be drawn from a research database containing claims and enrollment data for members of a large, geographically diverse US health plan.
- Trial name
- NCT01081730
- Testing
- anti-TNF biologics, general population, non-anti-TNF biologics
- Volunteers
- 2,040 people
- Sponsor
- Janssen Biotech, Inc.
Systemic Lupus Erythematosus
A Study of Telitacicept for the Treatment of Moderately to Severely Active Systemic Lupus Erythematosus (REMESLE-2)
The purpose of this study is to evaluate the efficacy and safety of telitacicept in the treatment of moderately to severely active SLE.
- Trial name
- NCT06456567
- Testing
- Telitacicept
- Where
- 4 sites in United States
- Sponsor
- Vor Biopharma
Mycoses
Antifugal Effect of Recombinant Human Granulocyte-macrophage Stimulating Factor (rhGM-CSF) in Patients Post Allo-HSCT
We conducted a prospective, multicenter, open-label randomized trial to compare the antifungal effect of Recombinant Human Granulocyte-macrophage Stimulating Factor (rhGM-CSF), Recombinant Human Granulocyte Stimulating Factor…
- Trial name
- NCT01232504
- Testing
- rhGM-CSF group, rhG-CSF+rhGM-CSF group, rhG-CSF group
- Where
- 5 sites in China
- Volunteers
- 206 people
- Sponsor
- Xiamen Amoytop Biotech Co., Ltd.
Systemic Sclerosis Cutaneous Lupus E Myopathies Systemic Lupus Erythematosus
Rollover Study for Participants Who Have Completed a Previous Clinical Study With Anifrolumab (Saphnelo) and Clinically Benefited From Continued Treatment
The purpose of this study is to provide continued access to anifrolumab to eligible participants who are continuing to derive clinical benefit from treatment at the end of a parent clinical study, as assessed by the Investigator…
- Trial name
- ROSY-S (NCT07691970)
- Testing
- Anifrolumab
- Where
- 153 sites in United States, Argentina and 33 more
- Volunteers
- 1,072 people
- Sponsor
- AstraZeneca
Low-grade Glioma, Rapidly Accelerated Fibrosarcoma (RAF) Altered Glioma
DAY101 vs. Standard of Care Chemotherapy in Pediatric Participants With Low-Grade Glioma Requiring First-Line Systemic Therapy (LOGGIC/FIREFLY-2)
This is a 2-arm, randomized, open-label, multicenter, global, Phase 3 trial to evaluate the efficacy, safety, and tolerability of tovorafenib monotherapy versus standard of care (SoC) chemotherapy in participants with pediatric…
- Conditions
- Low-grade Glioma, Rapidly Accelerated Fibrosarcoma (RAF) Altered Glioma, Pediatric Low-grade Glioma
- Trial name
- NCT05566795
- Testing
- Tovorafenib, Chemotherapeutic Agent
- Where
- 138 sites in United States, Australia and 28 more
- Volunteers
- 418 people
- Sponsor
- Day One Biopharmaceuticals, Inc.
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