ALPHA Study: Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH) | Aplastic Anemia & MDS International Foundation Return to top.

ClinicalTrials.gov Identifier:

NCT04469465

Contact Info

clinicaltrials@alexion.com

855-752-2356

Dates

Start: January 2021
End: October 2023

Official Title

ALPHA Study: Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)

Purpose

The purpose of this study is to evaluate the efficacy of danicopan as add-on therapy to a complement component 5 (C5) inhibitor (eculizumab or ravulizumab) in participants with PNH who have clinically evident EVH.

Participants will be assigned to different groups receiving danicopan on top of their C5 inhibitor (eculizumab or ramulizumab) or placebo (a sugar pill) in a ratio of 2:1 (which means that for every participant receiving placebo, two participants will receive danicopan ). At Week 12, participants who received placebo in the first 12 weeks will be switched to receive danicopan in addition to their C5 inhibitor for an additional 12 weeks (Treatment Period 2) and participants randomized to danicopan will continue on danicopan for an additional 12 weeks, while remaining on their ongoing C5 inhibitor therapy.

At the end of the 2 treatment periods (Week 24), participants may enter a 1-year Long-Term Extension (LTE) Period and continue to receive danicopan in addition to their C5 inhibitor therapy.

ALPHA Study: Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)

Clinical Trial: NCT04469465

Instructions

If you are interested in learning more about your possible participation in this clinical trial, please complete the form. Your information will be forwarded directly to the sponsoring company.

Status: 
Recruiting
Associated Drug(s): 
Phase: 
Phase 3
Gender: 
Female
Male
Age Group: 
18 years and older
Accepts Healthy Volunteers: 
No
Inclusion Criteria: 
  • Diagnosis of PNH
  • Clinically Evident EVH defined by:
  • Receiving an approved C5 inhibitor (eculizumab or ravulizumab) for at least 6 months prior to study Day 1
  • Platelet count ≥30,000/microliters (µL)
  • Absolute neutrophil counts ≥750/μL
  • Documentation of/or willingness to receive vaccinations for N. meningiditis and prophylactic antibiotics as required
Exclusion Criteria: 
  • History of a major organ transplant or hematopoietic stem cell transplantation (HSCT)
  • Participants with known aplastic anemia or other bone marrow failure that requires HSCT or other therapies including anti-thymocyte globulin and/or immunosuppressants
  • Known or suspected complement deficiency
  • Laboratory abnormalities at screening, including:
    • Alanine aminotransferase >2 x ULN
    • Direct bilirubin >2 x ULN (unless due to EVH or documented Gilbert's Syndrome)
  • Known underlying bleeding disorders (eg, coagulation factor deficiencies, idiopathic thrombocytopenic purpura, Von Willebrand disease, etc.) or any conditions leading to anemia that are not primarily due to PNH.
  • Current evidence of biliary cholestasis
  • Estimated glomerular filtration rate <30 milliliters/minute/1.73 meter squared and/or are on dialysis
  • Evidence of human immunodeficiency virus, hepatitis B, or active hepatitis C infection at screening

Find Out More:

Disclaimer:

AAMDSIF does not recommend, endorse, or make any representation about the efficacy, appropriateness or suitability of any clinical trial listed on this website. Pharmaceutical company sponsored content is highlighted only to give additional information about the trial. All trials are listed on https://clinicaltrials.gov/. Always seek the advice of your physician or other qualified health care provider with any questions you may have regarding a clinical trial, and never disregard professional medical advice or delay in seeking it because of something you have read on this website.

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