Clinical trials: Untreated
Congenital Bleeding Disorder, Haemophilia A
Safety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Previously Untreated Patients With Haemophilia A
This trial is conducted globally. The aim of the trial is to investigate the safety and efficacy of turoctocog alfa pegol (N8-GP) in previously untreated patients (PUPs) with haemophilia A.
- Trial name
- pathfinder™6 (NCT02137850)
- Testing
- turoctocog alfa pegol
- Where
- 96 sites in United States, Algeria and 21 more
- Volunteers
- 124 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A
Efficacy and Safety Study of a Recombinant Protein-Free Manufactured Factor VIII (rAHF-PFM) in Previously Untreated Hemophilia A Patients
The purpose of this study is to evaluate whether Antihemophilic factor, recombinant, manufactured protein-free (rAHF-PFM) is effective and safe in the treatment of hemophilia A patients who have not been treated with factor VIII…
- Trial name
- NCT00157157
- Testing
- Recombinant Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM)
- Where
- 35 sites in United States, Austria and 8 more
- Volunteers
- 66 people
- Sponsor
- Baxalta now part of Shire
Haemophilia A
Efficacy, Safety & Utilisation of Nuwiq, Octanate and Wilate in Previously Untreated & Minimally Treated Haemophilia A Patients
International, post-authorisation non-interventional study to evaluate real-life effectiveness, safety and utilisation patterns of Octapharma's FVIII concentrates Nuwiq, Octanate, and Wilate in previously untreated and minimally…
- Trial name
- Protect-NOW (NCT03695978)
- Where
- 59 sites in United States, Argentina and 16 more
- Volunteers
- 200 people
- Sponsor
- Octapharma
Congenital Bleeding Disorder, Haemophilia A
Safety and Efficacy of Turoctocog Alfa in Prevention and Treatment of Bleeds in Previously Untreated Children With Haemophilia A
This trial is conducted in Asia, Europe and North America. The purpose of the trial is to evaluate the safety and efficacy of turoctocog alfa in prevention and treatment of bleeds in previously untreated children with haemophilia…
- Trial name
- guardian™4 (NCT01493778)
- Testing
- turoctocog alfa
- Where
- 69 sites in United States, Algeria and 16 more
- Volunteers
- 60 people
- Sponsor
- Novo Nordisk A/S
Epilepsy
A Study to Evaluate the Efficacy and Safety of Perampanel Monotherapy in Untreated Participants With Focal Onset Seizures With or Without Focal to Bilateral Tonic-clonic Seizures
The primary purpose of this study is to evaluate the efficacy of perampanel monotherapy measured by the seizure-free rate during the Maintenance Period (24 weeks) of the Treatment Phase in untreated participants with focal onset…
- Trial name
- NCT05533814
- Testing
- Perampanel
- Where
- 10 sites in South Korea
- Volunteers
- 125 people
- Sponsor
- Eisai Korea Inc.
Epilepsy
A Multicenter Comparative Trial of Zonisamide and Topiramate as Initial Monotherapy in Untreated Epilepsies
To assess the efficacy and safety of Zonisamide and Topiramate as Initial Monotherapy in Untreated Epilepsies
- Trial name
- NCT00154076
- Testing
- Zonisamide, Topiramate
- Where
- South Korea
- Volunteers
- 140 people
- Sponsor
- Eisai Korea Inc.
Aplastic Anemia
A Phase II Dose-escalation Study Characterizing the PK of Eltrombopag in Pediatric Patients With Previously Untreated or Relapsed Severe Aplastic Anemia or Recurrent Aplastic Anemia
This is a phase II, open label, multi-center, intra-patient dose escalation study to characterize the pharmacokinetics (PK) after oral administration of eltrombopag in combination with immunosuppressive therapy in pediatric…
- Trial name
- NCT03025698
- Testing
- Eltrombopag, hATG, CsA
- Where
- 20 sites in United States, Hong Kong and 4 more
- Volunteers
- 51 people
- Sponsor
- Novartis Pharmaceuticals
FLT3-mutated Acute Myeloid Leukemia
A Global Study of Midostaurin in Combination With Chemotherapy to Evaluate Safety, Efficacy and Pharmacokinetics in Newly Diagnosed Pediatric Patients With FLT3 Mutated AML
This study will evaluate the safety, efficacy and pharmacokinetics of midostaurin in combination with standard chemotherapy in pediatrics patients with newly diagnosed FLT3-mutated Acute Myeloid Leukemia.
- Trial name
- NCT03591510
- Testing
- Midostaurin, Fludarabine, Cytarabine
- Where
- 15 sites in Czechia, Germany and 8 more
- Volunteers
- 22 people
- Sponsor
- Novartis Pharmaceuticals
Duchenne Muscular Dystrophy (DMD)
Study of Eteplirsen in DMD Patients
The main objective of this study is to provide evidence of efficacy of eteplirsen (AVI-4658) in Duchenne muscular dystrophy (DMD) patients that are amenable to skipping exon 51.
- Trial name
- PROMOVI (NCT02255552)
- Testing
- eteplirsen
- Where
- 37 sites in United States
- Volunteers
- 109 people
- Sponsor
- Sarepta Therapeutics, Inc.
Hodgkin Lymphoma
A Survey of Brentuximab Vedotin in Pediatric Participants With Hodgkin Lymphoma
This study is a survey in Japan of Brentuximab Vedotin used to treat children or teenagers with Hodgkin lymphoma (HL). The study sponsor will not be involved in how the participants are treated but will provide instructions on…
- Trial name
- NCT05481437
- Testing
- Brentuximab Vedotin
- Where
- Japan
- Volunteers
- 8 people
- Sponsor
- Takeda
Leukemia, Lymphocytic, Chronic, B-Cell
A Study to Confirm the Efficacy and Safety of Fludarabine Phosphate Administered in Untreated Chronic Lymphocytic Leukemia Patients With Anemia and/or Thrombocytopenia
The purpose of this study is to confirm the efficacy and safety of fludarabine phosphate administered with dose increasing in 6 cycles (1 cycle: 5 treatment days every 28 days) in untreated chronic lymphocytic leukemia (CLL)…
- Trial name
- NCT00220311
- Testing
- Fludarabine Phosphate (Fludara)
- Volunteers
- 10 people
- Sponsor
- Genzyme, a Sanofi Company
Melanoma
Phase II Safety Study of Vemurafenib Followed by Ipilimumab in Subjects With V600 BRAF Mutated Advanced Melanoma
The purpose of this study is to assess the safety profile of vemurafenib, 960 mg, administered for 6 weeks, followed by ipilimumab monotherapy in patients with BRAF V600 mutated advanced/metastatic melanoma.
- Trial name
- NCT01673854
- Testing
- Ipilimumab, Vemurafenib
- Where
- 14 sites in United States
- Volunteers
- 70 people
- Sponsor
- Bristol-Myers Squibb
Congenital Bleeding Disorder, Haemophilia B
Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B
This trial is conducted globally. The aim of the trial is to investigate the safety and efficacy of nonacog beta pegol (N9-GP) in previously untreated patients with Haemophilia B.
- Trial name
- paradigm™6 (NCT02141074)
- Testing
- nonacog beta pegol
- Where
- 93 sites in United States, Algeria and 22 more
- Volunteers
- 54 people
- Sponsor
- Novo Nordisk A/S
Acute Lymphoid Leukemia
Efficacy and Safety of Recombinant Asparaginase in Infants With Previously Untreated Acute Lymphoid Leukemia (ALL)
This non-controlled multicentre phase II study is designed to assess the safety and to describe (in relation to children of higher age) the pharmacodynamics of recombinant ASNase (rASNase) for first-line treatment of infants (< 1…
- Trial name
- NCT00983138
- Testing
- recombinant asparaginase
- Where
- 17 sites in Germany, Netherlands
- Volunteers
- 12 people
- Sponsor
- medac GmbH
Melanoma
A Study of Relatlimab Plus Nivolumab Versus Nivolumab Alone in Participants With Advanced Melanoma
The purpose of this study is to determine whether relatlimab in combination with nivolumab is more effective than nivolumab monotherapy in treating unresectable melanoma or melanoma that has spread.
- Trial name
- RELATIVITY-047 (NCT03470922)
- Testing
- Relatlimab, Nivolumab
- Where
- 127 sites in United States, Argentina and 23 more
- Volunteers
- 714 people
- Sponsor
- Bristol-Myers Squibb
LCA5
Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) and Non-interventional Arm With Untreated Patients
The goals of this clinical trial are assess the natural course of LCA5-IRD over 6 months and to evaluate the safety and preliminary efficacy of subretinal gene therapy with OPGx-001 in patients with inherited retinal degeneration…
- Trial name
- NCT05616793
- Testing
- AAV8.hLCA5
- Where
- 2 sites in United States
- Volunteers
- 22 people
- Sponsor
- Opus Genetics, Inc
Pediatric Immuno-Tolerant Chronic Hepatitis B
A Study of Pegylated Interferon Alfa-2A in Combination With Lamivudine or Entecavir Compared With Untreated Control Group in Children With Hepatitis B Envelope Antigen (HBeAg)-Positive Chronic Hepatitis B (CHB) in the Immune-Tolerant Phase
This randomized, controlled, parallel group, open-label multicenter study will evaluate the efficacy and safety of a combination of pegylated interferon alfa-2A (Pegasys) plus lamivudine or entecavir compared with an untreated…
- Trial name
- NCT02263079
- Testing
- Entecavir, Lamivudine, Pegylated Interferon Alfa-2A
- Where
- 25 sites in United States, Australia and 10 more
- Volunteers
- 62 people
- Sponsor
- Hoffmann-La Roche
Dwarfism
A Study of PEG-somatropin in the Treatment of Children With Idiopathic Short Stature
This study aims to explore the optimal dose of pegylated recombinant human growth hormone (PEG-rhGH) injection to treat children with idiopathic short stature (ISS), evaluate its safety and efficacy, and provide scientific and…
- Trial name
- NCT03221088
- Testing
- Jintrolong® low dose group, Jintrolong® high dose group
- Where
- 7 sites in China
- Volunteers
- 360 people
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
Brain and Central Nervous System Tumors, Lymphoma
DX-8951f in Treating Children With Advanced Solid Tumors or Lymphomas
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. PURPOSE: Phase I trial to study the effectiveness of DX-8951f in treating children who have advanced solid…
- Conditions
- Brain and Central Nervous System Tumors, Lymphoma, Unspecified Childhood Solid Tumor, Protocol Specific
- Trial name
- NCT00004212
- Testing
- filgrastim, exatecan mesylate
- Where
- 4 sites in United States
- Sponsor
- Daiichi Sankyo
Common Variable Immunodeficiency, Agammaglobulinemia
Efficacy and Safety of Vivaglobin® in Previously Untreated Patients With Primary Immunodeficiency
The objective of this study is to assess the efficacy and safety of Vivaglobin in previously untreated patients (PUPs) with primary immunodeficiency (PID) over a 25-week observation period.
- Trial name
- NCT00520494
- Testing
- Vivaglobin
- Where
- 6 sites in Canada, Germany and 2 more
- Volunteers
- 18 people
- Sponsor
- CSL Behring