Myelodysplastic Syndrome Clinical Trial

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

Summary

The main objective 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.

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Full Description

This is a multiple-region, randomized, double-blind, placebo controlled, multiple-dose, study in participants with PNH who have clinically evident EVH on a C5 inhibitor (eculizumab or ravulizumab).

Participants will be randomized to receive danicopan or placebo, in a 2:1 ratio for 12 weeks (Treatment Period 1) in addition to their C5 inhibitor (eculizumab or ravulizumab) therapy. At Week 12, participants randomized to receive placebo will be switched to 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 Long-Term Extension (LTE) Period and continue to receive danicopan in addition to their C5 inhibitor therapy. The Long-Term Extension period will consist of a first year of LTE(Year1) and a second year of optional LTE(Year2).All patients will complete 72 weeks of LTE(Year 1) assessments. After Week 72 (at the end of the first year of LTE), patients have the choice to complete participation in this study or continue to the optional second year (Year2) of LTE.

View Eligibility Criteria

Eligibility Criteria

Inclusion Criteria:

Diagnosis of PNH

Clinically Evident EVH defined by:

Anemia (Hgb ≤9.5 gram/deciliter) with absolute reticulocyte count ≥120 x 10^9/liter
Receiving an approved C5 inhibitor for at least 6 months prior to Day 1
Platelet count ≥30,000/microliters (µL)
Absolute neutrophil counts ≥500/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 (>3 x ULN in case of patients with documented liver iron overload defined by serum ferratin values

500 ng/ML)
Direct bilirubin >2 x ULN (unless due to EVH or documented Gilbert's Syndrome)
Current evidence of biliary cholestasis
Estimated glomerular filtration rate of <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

Study is for people with:

Myelodysplastic Syndrome

Phase:

Phase 3

Estimated Enrollment:

86

Study ID:

NCT04469465

Recruitment Status:

Completed

Sponsor:

Alexion Pharmaceuticals, Inc.

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There are 62 Locations for this study

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Research Site
Los Angeles California, 90089, United States
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Weston Florida, 33331, United States
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Chicago Illinois, 60612, United States
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Kalamazoo Michigan, 49007, United States
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New York New York, 10065, United States
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Dallas Texas, 75390, United States
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Milwaukee Wisconsin, 53212, United States
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Belem , 66053, Brazil
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Curitiba , 80810, Brazil
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Goiania , 74605, Brazil
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Porto Alegre , 90110, Brazil
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Rio De De Janeiro , 20211, Brazil
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Toronto Ontario, M5G 2, Canada
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Brno , 625 0, Czechia
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Lille , 59037, France
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Paris , 75010, France
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Pessac , 33604, France
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Pierre Benite Cedex , 69495, France
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Ulm , 89081, Germany
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Athens , 11527, Greece
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Thessaloniki , 57010, Greece
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Haifa , 31048, Israel
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Jerusalem , 91120, Israel
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Avellino , 83100, Italy
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Bassano del Grappa , 36061, Italy
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Firenze , 50134, Italy
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Milano , 20122, Italy
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Reggio Calabria , 89131, Italy
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Roma , 00161, Italy
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Bunkyo-Ku , 113 8, Japan
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Fukuoka , 812-8, Japan
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Kashiwa-shi , 277-8, Japan
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Kyoto-shi , 605-0, Japan
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Nagakute-shi , 480-1, Japan
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Ogaki-shi , 503-8, Japan
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Osaka-shi , 530-8, Japan
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Osakasayama , 589-8, Japan
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Shibuya-ku , 150-8, Japan
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Tanabe-shi , 646-8, Japan
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Toyoake-shi , 470-1, Japan
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Tsukuba-shi , 305-8, Japan
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Daejeon , 35015, Korea, Republic of
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Seoul , 03722, Korea, Republic of
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Seoul , 06351, Korea, Republic of
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Seoul , 06591, Korea, Republic of
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Suwon , 16247, Korea, Republic of
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Kota Kinabalu , 88586, Malaysia
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Kuching , 93586, Malaysia
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Miri , 98000, Malaysia
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Maastricht , 6229 , Netherlands
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Gdansk , 80-21, Poland
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Barcelona , 08036, Spain
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Barcelona , 08916, Spain
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Las Palmas de Gran Canaria , 35020, Spain
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Madrid , 28040, Spain
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Majadahonda , 28222, Spain
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Sevilla , 41013, Spain
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Taipei , 100, Taiwan
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Bangkok , 10330, Thailand
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Airdrie , ML6 0, United Kingdom
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Leeds , BD7 1, United Kingdom
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London , SE5 9, United Kingdom

How clear is this clinincal trial information?

Study is for people with:

Myelodysplastic Syndrome

Phase:

Phase 3

Estimated Enrollment:

86

Study ID:

NCT04469465

Recruitment Status:

Completed

Sponsor:


Alexion Pharmaceuticals, Inc.

How clear is this clinincal trial information?

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