Recent Updates
Recently added Catalysts

Ravulizumab OBDS

Phase 3

Paroxysmal Nocturnal Hemoglobinuria | Small molecule | Hematology |AstraZeneca PLC|Last Updated: Sep 19, 2024

Success Probability

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

RandomizedACTIVE_CONTROLLED
Total Trials1
Total Enrollment139

FDA Designations

No designations recorded

Clinical trial landscape

Ravulizumab OBDS · 1 trial · 1 indication

Phase 3 1
NCT03748823Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With EculizumabParoxysmal Nocturnal Hemoglobinuria
COMPLETED139 Analytics
PHASE3COMPLETED
Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
Paroxysmal Nocturnal HemoglobinuriaUnlock trial analytics

Study Endpoints

Primary Endpoints

Ctrough Serum Concentration of Ravulizumab
Predose at Day 71

Secondary Endpoints

Ctrough Serum Concentration of Ravulizumab at Day 351
Predose at Day 351
Free Serum Complement Component 5 (C5) Concentrations at Day 71
Predose at Day 71
Free Serum Complement Component 5 (C5) Concentrations at Day 351
Predose at Day 351
Unlock Study Endpoints

Study Design & Arms

AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Ravulizumab SC Treatment GroupEXPERIMENTALIn the Randomized Treatment Period, participants will receive an IV loading dose of ravulizumab on Day 1 followed by SC maintenance doses of ravulizumab administered via the ravulizumab OBDS on Day 15 and every week (qw) thereafter for a total of 10 weeks of study treatment. In the Extension Period, ravulizumab SC will be administered via the ravulizumab OBDS from Day 71 qw through Day 1274.
Ravulizumab IV Treatment GroupACTIVE_COMPARATORIn the Randomized Treatment Period, participants will receive an IV loading dose of ravulizumab on Day 1 followed by IV maintenance doses of ravulizumab on Day 15. In the Extension Period, ravulizumab SC will be administered via the ravulizumab OBDS from Day 71 qw through Day 1274.

Interventions

NameTypeDescription
Ravulizumab OBDSCOMBINATION_PRODUCTThe ravulizumab OBDS is a biological-device combination product consisting of a prefilled cartridge containing ravulizumab SC and an on-body injector.
RavulizumabBIOLOGICALAdministered by IV infusion. Ravulizumab IV doses will be based on participant body weight.
Unlock Study Design Details

Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites51

Inclusion Criteria: * Male or female ≥18 years of age * Treated with eculizumab for PNH for at least 3 months prior to Day 1 * LDH level ≤1.5 × upper limit of normal (ULN) at screening * PNH diagnosis confirmed by documented high-sensitivity flow cytometry. * Documented meningococcal vaccination no...

Countries:United StatesAustraliaAustriaBelgiumBrazilCanadaFinlandFranceItalyNetherlandsRussiaSpainSwedenTurkey (Türkiye)
Unlock Eligibility Criteria

Frequently asked questions about Ravulizumab OBDS

What is Ravulizumab OBDS used for?

Ravulizumab OBDS is used for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. It is an investigational small molecule being studied in adults with PNH who are currently treated with eculizumab. The drug is in Phase 3 clinical development and is not yet approved.

Who makes Ravulizumab OBDS?

Ravulizumab OBDS is developed by AstraZeneca PLC, a biopharmaceutical company traded on the stock exchange under the ticker AZN. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with paroxysmal nocturnal hemoglobinuria.

What phase is Ravulizumab OBDS in?

Ravulizumab OBDS is in Phase 3 clinical development. It is an investigational drug, meaning it has not been approved by regulatory authorities. A Phase 3 trial has been completed, evaluating the drug in adults with paroxysmal nocturnal hemoglobinuria.

What clinical trials is Ravulizumab OBDS in?

Ravulizumab OBDS has one completed Phase 3 trial, identified as NCT03748823. This trial compared subcutaneous administration of ravulizumab to intravenous administration in adults with paroxysmal nocturnal hemoglobinuria who were currently treated with eculizumab. The trial enrolled 139 participants across multiple countries.

Is Ravulizumab OBDS the same as eculizumab?

No, Ravulizumab OBDS is not the same as eculizumab. The clinical trial NCT03748823 specifically studied ravulizumab in patients with paroxysmal nocturnal hemoglobinuria who were already being treated with eculizumab, indicating they are distinct drugs. Ravulizumab is being developed as a potential alternative treatment.