Clinical trials: Paraneoplastic Syndromes
- Condition Paraneoplastic Syndromes
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Congenital Myasthenic Syndrome, Lambert-Eaton Myasthenic Syndrome
Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic Syndrome
Primary: The primary objective of this study under the original protocol was to provide neuromuscular specialists and neurologists access to amifampridine phosphate therapy for their patients with LEMS, CMS or downbeat nystagmus…
- Conditions
- Congenital Myasthenic Syndrome, Lambert-Eaton Myasthenic Syndrome, Nystagmus, Acquired
- Trial name
- EAP-001 (NCT02189720)
- Testing
- Amifampridine Phosphate
- Where
- United States
- Sponsor
- Catalyst Pharmaceuticals, Inc.
Generalized Myasthenia Gravis
Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis
The purpose of this trial is to investigate the PK, PD, safety, and activity of efgartigimod IV in children and adolescents aged from 2 to less than 18 years of age with gMG.
- Trial name
- ADAPT Jr (NCT04833894)
- Testing
- Efgartigimod IV
- Where
- 25 sites in United States, Austria and 10 more
- Volunteers
- 15 people
- Sponsor
- argenx
Cushing Syndrome
Extension Study to Evaluate the Safety of Long-Term Use of Relacorilant in Patients With Cushing Syndrome
This is an open-label extension study to evaluate the long-term safety of relacorilant in patients with endogenous Cushing syndrome who successfully completed participation in a Corcept-sponsored study of relacorilant and may…
- Trial name
- NCT03604198
- Testing
- relacorilant
- Where
- 48 sites in United States, Austria and 8 more
- Volunteers
- 125 people
- Sponsor
- Corcept Therapeutics
Ultomiris-exposed Pregnant/ Postpartum, Pregnancy
Study of Ultomiris® (Ravulizumab) Safety in Pregnancy
The primary objective of this study is to describe the frequency and characteristics of pregnancy outcomes and maternal complications among participants exposed to Ultomiris and to describe the frequency and characteristics of…
- Conditions
- Ultomiris-exposed Pregnant/ Postpartum, Pregnancy, Paroxysmal Nocturnal Hemoglobinuria (PNH), Atypical Hemolytic Uremic Syndrome (aHUS)
- Trial name
- NCT06312644
- Testing
- Ultomiris
- Where
- 7 sites in United States, Australia and 5 more
- Volunteers
- 75 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Generalized Myasthenia Gravis
A Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis
The purpose of this study is to assess the pharmacokinetics, pharmacodynamics, safety, tolerability, immunogenicity and activity of zilucoplan (ZLP) in pediatric study participants with generalized myasthenia gravis (gMG).
- Trial name
- ziMyG (NCT06055959)
- Testing
- Zilucoplan
- Where
- 9 sites in United States, Italy and 3 more
- Volunteers
- 8 people
- Sponsor
- UCB Biopharma SRL
Generalized Myasthenia Gravis
A Study of Rozanolixizumab in Pediatric Study Participants With Moderate to Severe Generalized Myasthenia Gravis
The purpose of the study is to assess the safety and tolerability of subcutaneous (sc) administration of rozanolixizumab in pediatric participants aged ≥2 to <18 years with generalized Myasthenia Gravis (gMG).
- Trial name
- roMyG (NCT06149559)
- Testing
- rozanolixizumab
- Where
- 9 sites in Italy, Japan and 2 more
- Volunteers
- 14 people
- Sponsor
- UCB Biopharma SRL
Neurogenic Detrusor Overactivity
Open-label, Long-term Safety, Efficacy, and Pharmacokinetics Study of Vibegron in Pediatric Subjects 2 Years to < 18 Years of Age With NDO and on CIC
The purpose of this study is to evaluate the safety, efficacy, and PK of Vibegron in pediatric participants with NDO who are regularly using CIC
- Trial name
- KANGUROO (NCT05491525)
- Testing
- Vibegron
- Where
- 38 sites in United States, Belgium and 14 more
- Volunteers
- 71 people
- Sponsor
- Urovant Sciences GmbH
Generalized Myasthenia Gravis
Safety and Efficacy Study of Ravulizumab in Adults With Generalized Myasthenia Gravis
The primary purpose of this study is to evaluate the safety and efficacy of ravulizumab for the treatment of participants with generalized myasthenia gravis (gMG).
- Trial name
- NCT03920293
- Testing
- Ravulizumab
- Where
- 106 sites in United States, Austria and 13 more
- Volunteers
- 175 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Myasthenia Gravis, CIDP - Chronic Inflammatory Demyelinating Polyneuropathy
A Worldwide Pregnancy Safety Study to Assess Maternal, Fetal, and Infant Outcomes Following Exposure to Efgartigimod During Pregnancy and/or Breastfeeding.
This is a multi-country, prospective safety study of pregnant women exposed to efgartigimod or efgartigimod PH20 SC any time within 25 days prior to conception or any time during pregnancy.
- Trial name
- NCT06299748
- Testing
- Efgartigimod
- Where
- 4 sites in United States, Germany and 2 more
- Volunteers
- 279 people
- Sponsor
- argenx
Myasthenia Gravis
An Open Study for Steroid Resistant, Non-Thymectomized MG Patients
The purpose of the study is to investigate the efficacy and safety for steroid resistant, non-thymectomized MG patients.
- Trial name
- NCT00309101
- Testing
- tacrolimus
- Where
- 9 sites in Japan
- Volunteers
- 11 people
- Sponsor
- Astellas Pharma Inc
Generalized Myasthenia Gravis, gMG
Evaluation of PK, PD, Efficacy, Safety, and Immunogenicity of IV Ravulizumab in Pediatric Participants With Generalized Myasthenia Gravis
The primary purpose of this study is to characterize the pharmacokinetics and pharmacodynamics of treatment with ravulizumab intravenous infusion in pediatric participants with gMG.
- Trial name
- NCT05644561
- Testing
- Ravulizumab
- Where
- 11 sites in United States, Italy and 3 more
- Volunteers
- 12 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Myasthenia Gravis, Myasthenia Gravis, Juvenile Form
A Phase 3 Open-Label Study of Eculizumab in Pediatric Participants With Refractory Generalized Myasthenia Gravis (gMG)
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of eculizumab in the treatment of pediatric refractory gMG based on change from Baseline in the Quantitative Myasthenia Gravis…
- Conditions
- Myasthenia Gravis, Myasthenia Gravis, Juvenile Form, Myasthenia Gravis, Generalized
- Trial name
- NCT03759366
- Testing
- Eculizumab
- Where
- 11 sites in United States, Japan
- Volunteers
- 12 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Generalized Myasthenia Gravis
A Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Tolerability of Inebilizumab in Children With Generalized Myasthenia Gravis (gMG)
The primary objectives of this study are to characterize the pharmacokinetics (PK) and pharmacodynamics (PD) of inebilizumab administered in pediatric participants with gMG, and to assess the safety and tolerability of…
- Trial name
- THYME (NCT06987539)
- Testing
- Inebilizumab
- Where
- 14 sites in United States, Argentina and 6 more
- Volunteers
- 15 people
- Sponsor
- Amgen
Generalized Myasthenia Gravis, gMG
PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis
The primary objective of this study is to assess the pharmacokinetics and pharmacodynamics of gefurulimab in pediatric participants with AChR+ gMG for the duration of the study.
- Trial name
- NCT06607627
- Testing
- Gefurulimab
- Where
- 13 sites in United States, Brazil and 3 more
- Volunteers
- 12 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Neuromyelitis Optica, Neuromyelitis Optica Spectrum Disorder
A Study of the Safety and Activity of Eculizumab in Pediatric Participants With Relapsing Neuromyelitis Optica Spectrum Disorder
The objective of this study is to evaluate the safety and efficacy of eculizumab in pediatric participants (aged 2 to < 18 years) with relapsing neuromyelitis optica spectrum disorder (NMOSD).
- Trial name
- NCT04155424
- Testing
- Eculizumab
- Where
- 35 sites in United States, Canada and 5 more
- Volunteers
- 5 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Generalized Myasthenia Gravis
An Open-label Extension Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis
The purpose of this study is to assess the long-term safety and tolerability of an additional 52 weeks of Zilucoplan treatment administered by subcutaneous injection once daily in pediatric study participants
- Trial name
- ziMyG+ (NCT06435312)
- Testing
- Zilucoplan
- Where
- 6 sites in Italy, Poland and 2 more
- Volunteers
- 8 people
- Sponsor
- UCB Biopharma SRL
Myasthenia Gravis
FK506 Phase 3 Study: a Study for Steroid Non-resistant Myasthenia Gravis (MG) Patients
The purpose of the study is to investigate the efficacy and safety for steroid non-resistant MG patients in a double blind, placebo controlled study.
- Trial name
- NCT00309088
- Testing
- tacrolimus
- Where
- 9 sites in Japan
- Volunteers
- 80 people
- Sponsor
- Astellas Pharma Inc
Generalized Myasthenia Gravis
A Study To Evaluate Efficacy, Safety, Pharmacokinetics, And Pharmacodynamics Of Satralizumab In Patients With Generalized Myasthenia Gravis
This study will evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of satralizumab compared with placebo in participants with generalized myasthenia gravis (gMG).
- Trial name
- NCT04963270
- Testing
- Satralizumab
- Where
- 76 sites in United States, Argentina and 15 more
- Volunteers
- 188 people
- Sponsor
- Hoffmann-La Roche
Generalized Myasthenia Gravis
A Study of Efgartigimod PH20 SC in Children Between 2 and Less Than 18 Years of Age With Generalized Myasthenia Gravis
The purpose of this study is to measure the pharmacokinetics (PK), pharmacodynamics (PD), safety, tolerability, and immunogenicity of efgartigimod PH20 SC in pediatric participants with gMG aged 2 to <18 years.
- Trial name
- ADAPT Jr SC (NCT06392386)
- Testing
- Efgartigimod PH20 SC
- Where
- 22 sites in United States, Australia and 12 more
- Volunteers
- 12 people
- Sponsor
- argenx
Gastric Ulcer (GU), Duodenal Ulcer (DU)
A Phase I/III Study of D961H 10 mg and 20 mg in Japanese Paediatric Patients With Gastrointestinal Acid Related Diseases
The objective of this study is to assess the safety, pharmacokinetics, pharmacodynamics and efficacy of repeated once daily oral administration of D961H 10 mg and D961H 20 mg in Japanese paediatric patients aged 1 to 14 years old…
- Conditions
- Gastric Ulcer (GU), Duodenal Ulcer (DU), Anastomotic Ulcer (AU), Non-erosive Reflux Esophagitis Disease (NERD)
- Trial name
- NCT02153398
- Testing
- D961H sachet 10 mg, D961H capsule 10mg, D961H capsule 20 mg
- Where
- 15 sites in Japan
- Volunteers
- 55 people
- Sponsor
- AstraZeneca