Clinical trials: M-protein
Severe Hemophilia A
Safety and Pharmacokinetics of Subcutaneous Injection of OCTA101 in Adult Patients With Severe Hemophilia A
This Phase 1/2 study will be a dose escalation study in adults in 5 cohorts (named cohorts 1, 2, 3, 5 and 6), with the main purpose to assess the safety of subcutaneous injection of OCTA101 (a human-cl rhFVIII and recombinant…
- Trial name
- NCT04046848
- Testing
- OCTA101
- Where
- Bulgaria
- Volunteers
- 36 people
- Sponsor
- Octapharma
Elranatamab, Myeloma
A Study of Elranatamab (PF-06863135) in Chinese Participants With Refractory Multiple Myeloma.
The purpose of this study is to understand the study medicine (called Elranatamab, or PF-06863135) as potential treatment for refractory multiple myeloma.
- Conditions
- Elranatamab, Myeloma, Multiple Myeloma, Relapsed Multiple Myeloma
- Trial name
- NCT05228470
- Testing
- Elranatamab
- Where
- 15 sites in China
- Volunteers
- 39 people
- Sponsor
- Pfizer
Chronic Kidney Diseases, Chronic Kidney Insufficiency
A Study to Evaluate TOUR006 in Patients With Chronic Kidney Disease and Elevated Hs-CRP
This study will evaluate the safety, tolerability, pharmacokinetics, and CRP-lowering effect of quarterly and monthly subcutaneous administration of TOUR006 (also known as pacibekitug) in participants with chronic kidney disease…
- Conditions
- Chronic Kidney Diseases, Chronic Kidney Insufficiency, Chronic Renal Diseases, Chronic Renal Insufficiency
- Trial name
- TRANQUILITY (NCT06362759)
- Testing
- TOUR006 - 50 MG, TOUR006 - 25 MG, TOUR006 - 15 MG
- Where
- 38 sites in United States
- Volunteers
- 143 people
- Sponsor
- Tourmaline Bio, Inc., a Novartis Company
Carcinoma, Carcinoma, Renal Cell
Savolitinib vs. Sunitinib in MET-driven PRCC.
This study is designed for patients diagnosed with MET-driven, unresectable and locally advanced or metastatic Papillary Renal Cell Carcinoma.
- Conditions
- Carcinoma, Carcinoma, Renal Cell, Kidney Neoplasms, Urologic Neoplasms
- Trial name
- NCT03091192
- Testing
- Savolitinib, Sunitinib
- Where
- 59 sites in United States, Brazil and 5 more
- Volunteers
- 60 people
- Sponsor
- AstraZeneca
Hemophilia A
Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A
This Phase III clinical study will evaluate the safety and effectiveness of valoctocogene roxaparvovec in combination with prophylactic corticosteroids in patients with severe hemophilia A.
- Trial name
- GENEr8-3 (NCT04323098)
- Testing
- valoctocogene roxaparvovec
- Where
- 12 sites in United States, Australia and 2 more
- Volunteers
- 22 people
- Sponsor
- BioMarin Pharmaceutical
CD55-deficient Protein-losing Enteropathy, CHAPLE
Open-Label Efficacy and Safety Study of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy (CHAPLE Disease)
The primary objective of the study is to determine the effect of pozelimab on active CD55-deficient protein-losing enteropathy (PLE; CHAPLE).
- Trial name
- NCT04209634
- Testing
- Pozelimab
- Where
- 3 sites in United States, Thailand and 1 more
- Volunteers
- 10 people
- Sponsor
- Regeneron Pharmaceuticals
Hemophilia A, Gene Therapy
Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5
This study is being conducted by BioMarin Pharmaceutical Inc. as an open label, single dose study to determine the safety of valoctocogene roxaparvovec (an Adenovirus-Associated Virus (AAV) based gene therapy vector) in severe…
- Conditions
- Hemophilia A, Gene Therapy, Clotting Disorders, Blood Disorder
- Trial name
- GENEr8-AAV5+ (NCT03520712)
- Testing
- Valoctocogene Roxaparvovec
- Where
- 9 sites in South Africa, South Korea and 2 more
- Volunteers
- 3 people
- Sponsor
- BioMarin Pharmaceutical
Congenital Protein C Deficiency
A Study of TAK-662 for Japanese Patients With Congenital Protein C Deficiency
Pharmacokinetic Part: This study is for Japanese participants with congenital protein C deficiency. The main aim of this study is to check how much TAK-662 stays in their blood over time.
- Trial name
- NCT04984889
- Testing
- TAK-662
- Where
- 4 sites in Japan
- Volunteers
- 5 people
- Sponsor
- Takeda
Severe Haemophilia A
Gene Therapy Study in Severe Haemophilia A Patients (270-201)
This study is being conducted by BioMarin Pharmaceutical Inc. as an open label, dose escalation study in order to determine the safety and efficacy of valoctocogene roxaparvovec (an Adenovirus-Associated Virus based gene therapy…
- Trial name
- NCT02576795
- Testing
- valoctocogene roxaparvovec
- Where
- 5 sites in United Kingdom
- Volunteers
- 15 people
- Sponsor
- BioMarin Pharmaceutical
Hemophilia A
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)
This Phase III clinical study will assess the impact of BMN 270 (compared to FVIII prophylaxis) on the number of bleeding episodes irrespective of exogenous FVIII replacement treatment in the efficacy evaluation period (EEP)…
- Trial name
- BMN 270-301 (NCT03370913)
- Testing
- valoctocogene roxaparvovec
- Where
- 49 sites in United States, Australia and 11 more
- Volunteers
- 144 people
- Sponsor
- BioMarin Pharmaceutical
Multiple Myeloma
ACY-1215 (Ricolinostat) in Combination With Pomalidomide and Low-dose Dex in Relapsed-and-Refractory Multiple Myeloma
Phase 1b: To evaluate the side effects and determine the best dose of ACY-1215 in combination with Pomalidomide and low-dose dexamethasone in patients with relapsed-and-refractory multiple myeloma.
- Trial name
- NCT01997840
- Testing
- ACY-1215 (Ricolinostat) in combination with pomalidomide and dexamethasone
- Where
- 2 sites in United States
- Volunteers
- 103 people
- Sponsor
- Celgene
Hemophilia A, Hemophilia B
An 18-month Low-interventional Study to Assess Joint Health in Haemophilia A and B Patients on Prophylaxis With Efmoroctocog Alfa or Eftrenonacog Alfa
The goal of this low-interventional study is to describe the overall joint health in patients with haemophilia A or haemophilia B prophylactically treated with rFVIIIFc or rFIXFc.
- Trial name
- JOIN-us (NCT05856266)
- Testing
- Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US), Haemophilia Joint Health Score (HJHS)
- Where
- 56 sites in Bulgaria, Croatia and 9 more
- Volunteers
- 10 people
- Sponsor
- Swedish Orphan Biovitrum
Hemophilia A
A Gene Transfer Study for Hemophilia A
This clinical research study is being conducted by Spark Therapeutics, Inc. to determine the safety and efficacy of the factor VIII gene transfer treatment with SPK-8011 in individuals with hemophilia A.
- Trial name
- NCT03003533
- Testing
- SPK-8011
- Where
- 16 sites in United States, Australia and 3 more
- Volunteers
- 25 people
- Sponsor
- Spark Therapeutics, Inc.
Multiple Myeloma
A Study of KW-2478 in Combination With Bortezomib in Subjects With Relapsed and/or Refractory Multiple Myeloma
The purpose of this study is to assess the safety and benefits of the investigational study drug, KW-2478, when given with bortezomib (Velcade®), a drug approved for the treatment of Multiple Myeloma (MM).
- Trial name
- NCT01063907
- Testing
- KW-2478, Bortezomib
- Where
- 26 sites in United States, Philippines and 1 more
- Volunteers
- 95 people
- Sponsor
- Kyowa Kirin Co., Ltd.
Adeno-Associated Virus (AAV), Blood Coagulation Disorder
Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors
SPK-8016 is in development for the treatment of patients with inhibitors to FVIII. This Phase 1/2, open-label, non-randomized, dose-finding study to evaluate the safety, efficacy, and tolerability of SPK-8016 in adult males with…
- Conditions
- Adeno-Associated Virus (AAV), Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders
- Trial name
- NCT03734588
- Testing
- SPK-8016
- Where
- 11 sites in United States
- Volunteers
- 4 people
- Sponsor
- Spark Therapeutics, Inc.
Hemophilia A
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
This Phase III clinical study will assess the efficacy of BMN 270 defined as FVIII activity, during weeks 49-52 following intravenous infusion of BMN 270 and assess the impact of BMN 270 on usage of exogenous FVIII replacement…
- Trial name
- GENEr8-2 (NCT03392974)
- Testing
- Valoctocogene Roxaparvovec
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- BioMarin Pharmaceutical
Carnitine Palmitoyltransferase (CPT I or CPT II) Deficiency, Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency
Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies
The primary objective of this study is to evaluate the long-term safety and efficacy of UX007 in participants with LC-FAOD. The secondary objectives of this study are to evaluate the effect of UX007 on energy metabolism in…
- Conditions
- Carnitine Palmitoyltransferase (CPT I or CPT II) Deficiency, Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency, Long-chain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency, Trifunctional Protein (TFP) Deficiency
- Trial name
- NCT02214160
- Testing
- UX007
- Where
- 11 sites in United States, United Kingdom
- Volunteers
- 94 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Cancer, Hematologic Malignancies
Denosumab Compared to Zoledronic Acid in the Treatment of Bone Disease in Patients With Multiple Myeloma
The purpose of this study is to determine if denosumab is non-inferior to zoledronic acid in the treatment of bone disease from multiple myeloma.
- Conditions
- Cancer, Hematologic Malignancies, Multiple Myeloma, Oncology
- Trial name
- NCT01345019
- Testing
- Denosumab, Zoledronic acid, Denosumab (for the open-label treatment phase)
- Where
- 309 sites in United States, Australia and 27 more
- Volunteers
- 1,718 people
- Sponsor
- Amgen
Hemophilia A, Hemophilia B
A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors
The purpose of this study was to determine the frequency of bleeding episodes in participants receiving fitusiran as prophylactic treatment of hemophilia compared to participants who were assigned to continue with their regular…
- Trial name
- ATLAS-INH (NCT03417102)
- Testing
- fitusiran, Bypassing agents
- Where
- 58 sites in United States, Australia and 15 more
- Volunteers
- 60 people
- Sponsor
- Genzyme, a Sanofi Company
Hemophilia A, Hemophilia B
A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors
Primary Objective: -To evaluate the efficacy of fitusiran compared to on-demand treatment with factor concentrates, as determined by the frequency of bleeding episodes.
- Trial name
- NCT03417245
- Testing
- fitusiran, factor concentrates
- Where
- 64 sites in United States, Australia and 17 more
- Volunteers
- 120 people
- Sponsor
- Genzyme, a Sanofi Company