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

Primary Purpose

Paroxysmal Nocturnal Hemoglobinuria

Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Danicopan
Placebo
C5 Inhibitor
Sponsored by
Alexion
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Paroxysmal Nocturnal Hemoglobinuria focused on measuring Paroxysmal Nocturnal Hemoglobinuria (PNH), Extravascular Hemolysis (EVH), Factor D inhibitor, Complement, Danicopan, C5 inhibitor

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

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Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Danicopan + C5 Inhibitor

Placebo + C5 Inhibitor

Arm Description

Participants will receive danicopan, in addition to their C5 inhibitor therapy, for 24 weeks (12 weeks in Treatment Period 1, followed by 12 weeks in Treatment Period 2).

Participants will receive placebo, in addition to their C5 inhibitor therapy, for 12 weeks during Treatment Period 1. At Week 12, participants randomized to receive placebo will be switched to danicopan for an additional 12 weeks (Treatment Period 2).

Outcomes

Primary Outcome Measures

Change From Baseline in Hgb at Week 12
Baseline was defined as the lowest Hgb value observed between and including Screening and Day 1. The least square (LS) mean and standard error (SE) were produced using mixed-effect model for repeated measures (MMRM). Hgb values collected within 4 weeks after transfusion were not included in the MMRM.

Secondary Outcome Measures

Percentage of Participants With Hgb Increase of ≥2 g/dL (≥20 g/L) From Baseline in the Absence of Transfusion at Week 12
The criterion was defined as ≥20 g/L increase in Hgb from Baseline to Week 12 and remaining transfusion free during the 12-Week TP1. Participants who withdrew from the study early during the 12-Week TP1 or had missing Hgb value at Week 12 were considered as not achieving the criterion.
Percentage of Participants With Transfusion Avoidance Through Week 12
Participants achieved transfusion avoidance if they remained transfusion free and did not require a transfusion as per protocol-specified guidelines from Week 1 through Week 12. Participants who discontinued study treatment early before Week 12 were considered as not achieving transfusion avoidance.
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Score at Week 12
The FACIT-Fatigue was 13-item questionnaire scored on a 5-point Likert scale (0 = not at all, 4 = very much) that assesses self-reported fatigue and its impact on daily activities and function. Total scores range from 0 to 52 with higher score indicating less fatigue better health-related quality of life. LS mean and SE were produced using MMRM.
Change From Baseline in Absolute Reticulocyte Count at Week 12
LS mean and SE were produced using MMRM.
Change in the Number of Red Blood Cell (RBC) Units Transfused From 24 Weeks Prior to Initiation of Treatment to Post 24 Weeks of Treatment
Change in Number of Transfusion Instances From 24 Weeks Prior to Initiation of Treatment to Post 24 Weeks of Treatment
Percentage of Participants With Transfusion Avoidance Through Week 24
Change in the Number of RBC Units Transfused From 12 Weeks Prior to Initiation of Treatment to Post 12 Weeks of Treatment
LS mean and SE were produced using analysis of covariance (ANCOVA).
Change in Number of Transfusion Instances From 12 Weeks Prior to Initiation of Treatment to Post 12 Weeks of Treatment
LS mean and SE were produced using ANCOVA.
Change From Baseline FACIT Fatigue Scores at Week 24
Percentage of Participants With Hgb Stabilization During Last 12 Weeks of Treatment in Participants Receiving 24 Weeks of Danicopan
Percentage of Participants With Hgb Increase of ≥2 g/dL (≥ 20 g/L) From Baseline in the Absence of Transfusion at Week 24
Change From Baseline in Total and Direct Bilirubin at Week 12
Baseline was defined as the last nonmissing value prior to first dose of study intervention. LS mean and SE were produced using MMRM.
Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) RBC Clone Size at Week 12
The PNH clone size refers to the percentage of PNH-affected cells versus normal cells within the total cell population. Baseline was defined as the last nonmissing value prior to first dose of study intervention. LS mean and SE were produced using MMRM.
Change From Baseline in C3 Fragment Deposition (C3d PNH Type 3 Cells) on PNH RBCs at Week 12
Baseline was defined as the last nonmissing value prior to first dose of study intervention. LS mean and SE were produced using MMRM.
Change From Baseline in Lactate Dehydrogenase at Week 12
Baseline was defined as the average of all available assessments prior to the first dose of study intervention. LS mean and SE were produced using MMRM.
Percentage of Participants With Hgb Normalization at Week 12
Hgb normalization was defined as Hgb value above lower limit of normal (LLN) reference range. For male, the LLN was 125 grams (g)/liter (L), for female, the LLN was 110 g/L. Participants with transfusions within 4 weeks prior to Week 12 were considered as not meeting Hgb normalization regardless of actual value observed at Week 12.
Percentage of Participants With Hgb Normalization at Week 24

Full Information

First Posted
July 9, 2020
Last Updated
July 21, 2023
Sponsor
Alexion
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1. Study Identification

Unique Protocol Identification Number
NCT04469465
Brief Title
Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA)
Official Title
A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH)
Study Type
Interventional

2. Study Status

Record Verification Date
July 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
December 16, 2020 (Actual)
Primary Completion Date
June 28, 2022 (Actual)
Study Completion Date
December 31, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Alexion

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief 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.
Detailed 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Paroxysmal Nocturnal Hemoglobinuria
Keywords
Paroxysmal Nocturnal Hemoglobinuria (PNH), Extravascular Hemolysis (EVH), Factor D inhibitor, Complement, Danicopan, C5 inhibitor

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
86 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Danicopan + C5 Inhibitor
Arm Type
Experimental
Arm Description
Participants will receive danicopan, in addition to their C5 inhibitor therapy, for 24 weeks (12 weeks in Treatment Period 1, followed by 12 weeks in Treatment Period 2).
Arm Title
Placebo + C5 Inhibitor
Arm Type
Placebo Comparator
Arm Description
Participants will receive placebo, in addition to their C5 inhibitor therapy, for 12 weeks during Treatment Period 1. At Week 12, participants randomized to receive placebo will be switched to danicopan for an additional 12 weeks (Treatment Period 2).
Intervention Type
Drug
Intervention Name(s)
Danicopan
Other Intervention Name(s)
ALXN2040
Intervention Description
Oral tablet
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Oral tablet
Intervention Type
Drug
Intervention Name(s)
C5 Inhibitor
Intervention Description
Participants will continue to receive their ongoing C5 inhibitor (eculizumab or ravulizumab) therapy according to their usual dose and schedule.
Primary Outcome Measure Information:
Title
Change From Baseline in Hgb at Week 12
Description
Baseline was defined as the lowest Hgb value observed between and including Screening and Day 1. The least square (LS) mean and standard error (SE) were produced using mixed-effect model for repeated measures (MMRM). Hgb values collected within 4 weeks after transfusion were not included in the MMRM.
Time Frame
Baseline, Week 12
Secondary Outcome Measure Information:
Title
Percentage of Participants With Hgb Increase of ≥2 g/dL (≥20 g/L) From Baseline in the Absence of Transfusion at Week 12
Description
The criterion was defined as ≥20 g/L increase in Hgb from Baseline to Week 12 and remaining transfusion free during the 12-Week TP1. Participants who withdrew from the study early during the 12-Week TP1 or had missing Hgb value at Week 12 were considered as not achieving the criterion.
Time Frame
Week 12
Title
Percentage of Participants With Transfusion Avoidance Through Week 12
Description
Participants achieved transfusion avoidance if they remained transfusion free and did not require a transfusion as per protocol-specified guidelines from Week 1 through Week 12. Participants who discontinued study treatment early before Week 12 were considered as not achieving transfusion avoidance.
Time Frame
Week 12
Title
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Score at Week 12
Description
The FACIT-Fatigue was 13-item questionnaire scored on a 5-point Likert scale (0 = not at all, 4 = very much) that assesses self-reported fatigue and its impact on daily activities and function. Total scores range from 0 to 52 with higher score indicating less fatigue better health-related quality of life. LS mean and SE were produced using MMRM.
Time Frame
Baseline, Week 12
Title
Change From Baseline in Absolute Reticulocyte Count at Week 12
Description
LS mean and SE were produced using MMRM.
Time Frame
Baseline, Week 12
Title
Change in the Number of Red Blood Cell (RBC) Units Transfused From 24 Weeks Prior to Initiation of Treatment to Post 24 Weeks of Treatment
Time Frame
24 weeks prior to initiation of treatment to post 24 weeks of treatment
Title
Change in Number of Transfusion Instances From 24 Weeks Prior to Initiation of Treatment to Post 24 Weeks of Treatment
Time Frame
24 weeks prior to initiation of treatment to post 24 weeks of treatment
Title
Percentage of Participants With Transfusion Avoidance Through Week 24
Time Frame
Week 24
Title
Change in the Number of RBC Units Transfused From 12 Weeks Prior to Initiation of Treatment to Post 12 Weeks of Treatment
Description
LS mean and SE were produced using analysis of covariance (ANCOVA).
Time Frame
12 weeks prior to initiation of treatment to post 12 weeks of treatment
Title
Change in Number of Transfusion Instances From 12 Weeks Prior to Initiation of Treatment to Post 12 Weeks of Treatment
Description
LS mean and SE were produced using ANCOVA.
Time Frame
12 weeks prior to initiation of treatment to post 12 weeks of treatment
Title
Change From Baseline FACIT Fatigue Scores at Week 24
Time Frame
Baseline, Week 24
Title
Percentage of Participants With Hgb Stabilization During Last 12 Weeks of Treatment in Participants Receiving 24 Weeks of Danicopan
Time Frame
Week 12 to Week 24
Title
Percentage of Participants With Hgb Increase of ≥2 g/dL (≥ 20 g/L) From Baseline in the Absence of Transfusion at Week 24
Time Frame
Week 24
Title
Change From Baseline in Total and Direct Bilirubin at Week 12
Description
Baseline was defined as the last nonmissing value prior to first dose of study intervention. LS mean and SE were produced using MMRM.
Time Frame
Baseline, Week 12
Title
Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) RBC Clone Size at Week 12
Description
The PNH clone size refers to the percentage of PNH-affected cells versus normal cells within the total cell population. Baseline was defined as the last nonmissing value prior to first dose of study intervention. LS mean and SE were produced using MMRM.
Time Frame
Baseline, Week 12
Title
Change From Baseline in C3 Fragment Deposition (C3d PNH Type 3 Cells) on PNH RBCs at Week 12
Description
Baseline was defined as the last nonmissing value prior to first dose of study intervention. LS mean and SE were produced using MMRM.
Time Frame
Baseline, Week 12
Title
Change From Baseline in Lactate Dehydrogenase at Week 12
Description
Baseline was defined as the average of all available assessments prior to the first dose of study intervention. LS mean and SE were produced using MMRM.
Time Frame
Baseline, Week 12
Title
Percentage of Participants With Hgb Normalization at Week 12
Description
Hgb normalization was defined as Hgb value above lower limit of normal (LLN) reference range. For male, the LLN was 125 grams (g)/liter (L), for female, the LLN was 110 g/L. Participants with transfusions within 4 weeks prior to Week 12 were considered as not meeting Hgb normalization regardless of actual value observed at Week 12.
Time Frame
Week 12
Title
Percentage of Participants With Hgb Normalization at Week 24
Time Frame
Week 24

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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
Facility Information:
Facility Name
Clinical Trial Site
City
Los Angeles
State/Province
California
ZIP/Postal Code
90033
Country
United States
Facility Name
Clinical Trial Site
City
Weston
State/Province
Florida
ZIP/Postal Code
33331
Country
United States
Facility Name
Clinical Trial Site
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
Clinical Trial Site
City
Kalamazoo
State/Province
Michigan
ZIP/Postal Code
49001
Country
United States
Facility Name
Clinical Trial Site
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64111
Country
United States
Facility Name
Clinical Trial Site
City
New York
State/Province
New York
ZIP/Postal Code
10065
Country
United States
Facility Name
Clinical Trial Site
City
Dallas
State/Province
Texas
ZIP/Postal Code
75231
Country
United States
Facility Name
Clinical Trial Site
City
Salvador
State/Province
Bahia
ZIP/Postal Code
40170-010
Country
Brazil
Facility Name
Clinical Trial Site
City
Fortaleza
State/Province
Ceara
ZIP/Postal Code
60430-370
Country
Brazil
Facility Name
Clinical Trial Site
City
Goiania
State/Province
Goias
ZIP/Postal Code
74605-020
Country
Brazil
Facility Name
Clinical Trial Site
City
Curitiba
State/Province
Parana
ZIP/Postal Code
80810-050
Country
Brazil
Facility Name
Clinical Trial Site
City
Belem
State/Province
Para
ZIP/Postal Code
66053-000
Country
Brazil
Facility Name
Clinical Trial Site
City
Porto Alegre
State/Province
Rio Grande Do Sul
ZIP/Postal Code
90110-270
Country
Brazil
Facility Name
Clinical Trial Site
City
Rio de Janeiro
ZIP/Postal Code
20211-030
Country
Brazil
Facility Name
Clinical Trial Site
City
Edmonton
State/Province
Alberta
ZIP/Postal Code
T6G 2G3
Country
Canada
Facility Name
Clinical Trial Site
City
Toronto
State/Province
Ontario
ZIP/Postal Code
MSG 2C4
Country
Canada
Facility Name
Clinical Trial Site
City
Brno
ZIP/Postal Code
62500
Country
Czechia
Facility Name
Clinical Trial Site
City
Nice
State/Province
Alpes Maritimes
ZIP/Postal Code
06200
Country
France
Facility Name
Clinical Trial Site
City
Marseille Cedex 9
State/Province
Bouches-du-Rhone
ZIP/Postal Code
13273
Country
France
Facility Name
Clinical Trial Site
City
Lille cedex
State/Province
Nord
ZIP/Postal Code
59037
Country
France
Facility Name
Clinical Trial Site
City
Paris cedex 10
State/Province
Paris
ZIP/Postal Code
75475
Country
France
Facility Name
Clinical Trial Site
City
Pierre-Bénite
State/Province
Rhone
ZIP/Postal Code
69495
Country
France
Facility Name
Clinical Trial Site
City
Aachen
ZIP/Postal Code
52074
Country
Germany
Facility Name
Clinical Trial Site
City
Ulm
ZIP/Postal Code
89081
Country
Germany
Facility Name
Clinical Trial Site
City
Athens
ZIP/Postal Code
11527
Country
Greece
Facility Name
Clinical Trial Site
City
Thessaloniki
ZIP/Postal Code
57010
Country
Greece
Facility Name
Clinical Trial Site
City
Haifa
ZIP/Postal Code
31048
Country
Israel
Facility Name
Clinical Trial Site
City
Jerusalem
ZIP/Postal Code
91120
Country
Israel
Facility Name
Clinical Trial Site
City
Reggio Calabria
State/Province
Calabria
ZIP/Postal Code
89214
Country
Italy
Facility Name
Clinical Trial Site
City
Avellino
State/Province
Campagnia
ZIP/Postal Code
83100
Country
Italy
Facility Name
Clinical Trial Site
City
Roma
State/Province
Lazio
ZIP/Postal Code
00161
Country
Italy
Facility Name
Clinical Trial Site
City
Milano
State/Province
Lombardia
ZIP/Postal Code
20122
Country
Italy
Facility Name
Clinical Trial Site
City
Firenze
State/Province
Toscana
ZIP/Postal Code
50134
Country
Italy
Facility Name
Clinical Trial Site
City
Bassano Del Grappa
State/Province
Vicenza
ZIP/Postal Code
36061
Country
Italy
Facility Name
Clinical Trial Site
City
Nagakute-shi
State/Province
Aichi
ZIP/Postal Code
480-1195
Country
Japan
Facility Name
Clinical Trial Site
City
Toyoake
State/Province
Aichi
ZIP/Postal Code
470-1192
Country
Japan
Facility Name
Clinical Trial Site
City
Kashiwa-shi
State/Province
Chiba
ZIP/Postal Code
277-8567
Country
Japan
Facility Name
Clinical Trial Site
City
Fukuoka-shi
State/Province
Fukuoka
ZIP/Postal Code
812-8582
Country
Japan
Facility Name
Clinical Trial Site
City
Fukushima-shi
State/Province
Fukushima-ken
ZIP/Postal Code
960-1295
Country
Japan
Facility Name
Clinical Trial Site
City
Ogaki-shi
State/Province
Gifu
ZIP/Postal Code
503-8502
Country
Japan
Facility Name
Clinical Trial Site
City
Tsukuba-shi
State/Province
Ibaraki
ZIP/Postal Code
305-8576
Country
Japan
Facility Name
Clinical Trial Site
City
Kyoto-shi
State/Province
Kyoto-Fu
ZIP/Postal Code
605-0981
Country
Japan
Facility Name
Clinical Trial Site
City
Osaka-shi
State/Province
Osaka
ZIP/Postal Code
530-8480
Country
Japan
Facility Name
Clinical Trial Site
City
Osakasayama-shi
State/Province
Osaka
ZIP/Postal Code
589-8511
Country
Japan
Facility Name
Clinical Trial Site
City
Suita-shi
State/Province
Osaka
ZIP/Postal Code
565-0871
Country
Japan
Facility Name
Clinical Trial Site
City
Bunkyo-ku
State/Province
Tokyo
ZIP/Postal Code
113-8603
Country
Japan
Facility Name
Clinical Trial Site
City
Shibuya-ku
State/Province
Tokyo
ZIP/Postal Code
150-8935
Country
Japan
Facility Name
Clinical Trial Site
City
Shinjuku-Ku
State/Province
Tokyo
ZIP/Postal Code
160-0023
Country
Japan
Facility Name
Clinical Trial Site
City
Tanabe-shi
State/Province
Wakayama
ZIP/Postal Code
646-8588
Country
Japan
Facility Name
Clinical Trial Site
City
Suwon-si
State/Province
Gyeonggi-do
ZIP/Postal Code
16247
Country
Korea, Republic of
Facility Name
Clinical Trial Site
City
Daejeon
ZIP/Postal Code
35015
Country
Korea, Republic of
Facility Name
Clinical Trial Site
City
Seoul
ZIP/Postal Code
03080
Country
Korea, Republic of
Facility Name
Clinical Trial Site
City
Seoul
ZIP/Postal Code
03722
Country
Korea, Republic of
Facility Name
Clinical Trial Site
City
Seoul
ZIP/Postal Code
05505
Country
Korea, Republic of
Facility Name
Clinical Trial Site
City
Seoul
ZIP/Postal Code
06351
Country
Korea, Republic of
Facility Name
Clinical Trial Site
City
Seoul
ZIP/Postal Code
06591
Country
Korea, Republic of
Facility Name
Clinical Trial Site
City
Kota Kinabalu
State/Province
Sabah
ZIP/Postal Code
88586
Country
Malaysia
Facility Name
Clinical Trial Site
City
Kuching
State/Province
Sarawak
ZIP/Postal Code
93588
Country
Malaysia
Facility Name
Clinical Trial Site
City
Miri
State/Province
Sarawak
ZIP/Postal Code
98000
Country
Malaysia
Facility Name
Clinical Trial Site
City
Maastricht
State/Province
Limburg
ZIP/Postal Code
6229 HX
Country
Netherlands
Facility Name
Clinical Trial Site
City
Gdansk
ZIP/Postal Code
80-214
Country
Poland
Facility Name
Clinical Trial Site
City
Badalona
State/Province
Barcelona
ZIP/Postal Code
08916
Country
Spain
Facility Name
Clinical Trial Site
City
L'Hospitalet de Llobregat
State/Province
Barcelona
ZIP/Postal Code
08908
Country
Spain
Facility Name
Clinical Trial Site
City
Las Palmas
State/Province
Las Palmas De Gran Canaria
ZIP/Postal Code
35019
Country
Spain
Facility Name
Clinical Trial Site
City
Majadahonda
State/Province
Madrid
ZIP/Postal Code
28222
Country
Spain
Facility Name
Clinical Trial Site
City
Málaga
State/Province
Malaga
ZIP/Postal Code
29010
Country
Spain
Facility Name
Clinical Trial Site
City
Barcelona
ZIP/Postal Code
08035
Country
Spain
Facility Name
Clinical Trial Site
City
Barcelona
ZIP/Postal Code
08036
Country
Spain
Facility Name
Clinical Trial Site
City
Madrid
ZIP/Postal Code
28040
Country
Spain
Facility Name
Clinical Trial Site
City
Sevilla
ZIP/Postal Code
41013
Country
Spain
Facility Name
Clinical Trial Site
City
Taipei
ZIP/Postal Code
100
Country
Taiwan
Facility Name
Clinical Trial Site
City
Pathum Wan
State/Province
Bangkok
ZIP/Postal Code
10330
Country
Thailand
Facility Name
Clinical Trial Site
City
London
State/Province
Greater London
ZIP/Postal Code
SE5 9NU
Country
United Kingdom
Facility Name
Clinical Trial Site
City
Airdrie
State/Province
North Lanarkshire
ZIP/Postal Code
ML6 0JS
Country
United Kingdom
Facility Name
Clinical Trial Site
City
Leeds
State/Province
West Yorkshire
ZIP/Postal Code
LS9 7TF
Country
United Kingdom

12. IPD Sharing Statement

Links:
URL
https://doi.org/10.1182/blood-2019-124837
Description
Mechanistic Evaluation of Efficacy Using Biomarkers of the Oral, Small Molecule Factor D Inhibitor, Danicopan (ACH-4471), in Untreated Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)
URL
https://doi.org/10.1182/blood-2019-124748
Description
A Phase 2 Open-Label Study of Danicopan (ACH-0144471) in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have an Inadequate Response to Eculizumab Monotherapy
URL
https://library.ehaweb.org/eha/2019/24th/267447/peter.browett.a.phase.2.open
Description
Danicopan (ACH-4471) in Untreated Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Phase 2, Open-label, Proof of Concept Study of the Oral, Small Molecule Factor D Inhibitor

Learn more about this trial

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

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