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A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)

Primary Purpose

Sickle Cell Disease

Status

Active

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

Mitapivat

Mitapivat-matching placebo

About this trial

This is an interventional treatment trial for Sickle Cell Disease

Eligibility Criteria

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

Inclusion Criteria:

Age 16 years or older (18 years or older [France and Germany]); participants age 16 or 17 years must physically have completed puberty;
Documented diagnosis of sickle cell disease (SCD) (HbSS, HbSC [combined heterozygosity for hemoglobins S and C], HbS/beta 0- thalassemia, HbS/ beta plus thalassemia, or other sickle cell syndrome variants);
At least 2 sickle cell pain crises (SCPCs) and no more than 10 SCPCs in the past 12 months;
Hemoglobin at least 5.5 and 10.5 gram per deciliter (g/dL) at the most. Hemoglobin concentration must be based on an average of at least 2 Hb concentration measurements (separated by ≥7 days) collected during the Screening Period;
If taking hydroxyurea, the hydroxyurea dose must be stable for at least 90 days before starting study drug;
Women capable of becoming pregnant and men with partners who are women that are capable of becoming pregnant must agree to use 2 forms of contraception.

Exclusion Criteria:

Pregnant, breastfeeding, or parturient;
Receiving regularly scheduled transfusions;
Hepatobiliary disorders including but not limited to significant liver disease or gallbladder disease;
Severe kidney disease;
Prior exposure to gene therapy or prior bone marrow or stem cell transplantation;
Currently receiving treatment for SCD (eg, voxelotor, crizanlizumab, L-glutamine), with the exception of hydroxyurea. The last dose of such therapies must have been administered at least 90 days before starting study drug;
Currently receiving treatment with hematopoietic stimulating agents; the last dose must have been administered at least 90 days before starting study drug;
Received treatment on another investigational trial within 90 days prior to start of study drug or plans to participate in another investigational drug trial;
Taking medications that are strong inhibitors of CYP3A4/5 or strong inducers of CYP3A4 that cannot be stopped in an acceptable timeframe before starting study drug (timeframe will be discussed with your doctor).

Sites / Locations

University of California San Diego
UCLA Health
Children's Hospital Oakland
Children's National Hospital
Kaiser Permanente - Largo Medical Center
Massachusetts General Hospital
Boston Medical Center & Boston University School of Medicine
University of Michigan
Children's Hospital of Michigan
Mississippi Center for Advanced Medicine
Cure 4 The Kids Foundation, A Division of Roseman University of Health Sciences
East Carolina University - Brody School of Medicine
Penn State Health Milton S. Hershey Medical Center
Penn Medicine - University of Pennsylvania Health System
St. Christopher's Hospital for Children
University of Texas Health Science Center of Houston
Seattle Cancer Care Alliance, University of Washington
Hôpital Erasme
ZNA Stuivenberg
Universitair Ziekenhuis Antwerpen
CHR de la Citadelle
Clinique CHC MontLégia
Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul (PUCRS)
Hospital de Clínicas da Unicamp
Hospital Das Clínicas da Faculdade de Medicina de Ribeirão Preto - USP
Praxis Pesquisa Medica
HEMORIO Instituto Nacional de Hematologia
Instituto do Cancer do Estado de São Paulo, Hospital das Clínicas da Faculdade de Medicina da Universidad de São Paulo
CHU Montreal
McGill University Health Center
Hopitaux de La Timone
Hôpital Pellegrin, CHU de Bordeaux
Institut Universitaire du Cancer de Toulouse - Oncopole
Hôpital Européen Georges Pompidou
CHU Hôpital Henri Mondor
HaEmek Medical Center
Rambam Medical Center
Ziv Medical Center
A.O.R.N. "A. Cardarelli"
AOU dell'Università degli Studi della Campania Luigi Vanvitelli
Ente Ospedaliero Ospedali Galliera
Kenya Medical Research Institute (KEMRI) Centre for Clinical Research Butere County Hospital
American University of Beirut Medical Center
Nini Hospital
Hammoud Hospital University Medical Center
Erasmus MC
National Hospital Abuja
University of Abuja Teaching Hospital
Lagos University Teaching Hospital
Sultan Qaboos University Hospital, Hematology Department, COM&HS
King Abdullah International Medical Research Center
Hacettepe University
Acibadem Adana Hospital
Evelina Children's Hospital
Hammersmith Hospital
Manchester Royal Infirmary, Manchester University NHS Foundation Trust

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm 6

Arm 7

Arm Type

Experimental

Placebo Comparator

Experimental

Placebo Comparator

Experimental

Arm Label

Phase 2: Mitapivat 50 mg BID

Phase 2: Mitapivat 100 mg BID

Phase 2: Placebo

Phase 2: Open-Label Extension Period

Phase 3: Mitapivat selected Phase 3 dose

Phase 3: Placebo

Phase 3: Open-Label Extension Period

Arm Description

Double-blind Period: Mitapivat 50 milligrams (mg) twice daily (BID) for 12 weeks.

Double-blind Period: Mitapivat 100 mg BID for 12 weeks.

Double-blind Period: Mitapivat-matching placebo for 12 weeks.

Participants who received mitapivat 50mg BID in the double-blind period may choose to receive mitapivat 50mg BID for 216 weeks after. Participants who received mitapivat 100mg BID in the double-blind period may choose to receive mitapivat 100 mg BID for 216 weeks after. Participants who received mitapivat-matching placebo in the double-blind period, may be randomized to receive either mitapivat 50 mg or 100 mg BID for 216 weeks after.

Double-blind Period: Mitapivat selected Phase 3 dose (50 mg or 100 mg BID) for 52 weeks.

Double-blind Period: Mitapivat-matching placebo selected Phase 3 dose (which can be either 50 mg or 100 mg BID based on the phase 2 results) for 52 weeks.

Participants may choose to receive the selected Phase 3 mitapivat dose (which can be either 50 mg or 100 mg BID based on the phase 2 results) for 216 weeks after the Double-blind Period.

Outcomes

Primary Outcome Measures

Phase 2: Percentage of Participants With Hemoglobin (Hb) Response

Phase 2: Percentage of Participants With Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious AEs (SAEs)

Phase 3: Percentage of Participants With Hb Response

Phase 3: Annualized Rate of Sickle Cell Pain Crises (SCPCs)

Secondary Outcome Measures

Phase 2: Change From Baseline in Hb Concentration

Phase 2: Change From Baseline in Indirect Bilirubin

Phase 2: Change From Baseline in Lactate Dehydrogenase (LDH)

Phase 2: Change From Baseline in Absolute Reticulocytes Count

Phase 2: Change From Baseline in Percent Reticulocytes

Phase 2: Change From Baseline in Erythropoietin

Phase 2: Change From Baseline in Patient-Reported Outcomes Measurement Information System® (PROMIS®) Fatigue 13a Short Form (SF) Score

Phase 2: Annualized Rate of SCPCs

Phase 2: Pharmacokinetic/Pharmacodynamic Relationship: Evaluate the Exposure of Mitapivat to the Change in Adenosine Triphosphate (ATP) and 2,3-Diphosphoglycerate (2,3-DPG)

Phase 2: Mitapivat Concentration Over Time

Phase 2: Mitapivat Area Under the Concentration

Phase 2: Mitapivat Maximum (Peak) Concentration

Phase 3: Change From Baseline in Hb Concentration

Phase 3: Change From Baseline in Indirect Bilirubin

Phase 3: Change From Baseline in Percent Reticulocytes

Phase 3: Change From Baseline in PROMIS® Fatigue 13a SF Scores

Phase 3: Annualized Frequency of Hospitalizations for SCPC

Phase 3: Change From Baseline in LDH Concentration

Phase 3: Change From Baseline in Absolute Reticulocytes

Phase 3: Change From Baseline in Erythropoietin

Phase 3: Percentage of Participants With Improvement in the Patient Global Impression of Severity (PGIS) -Fatigue

Phase 3: Percentage of Participants With Improvement in the Patient Global Impression of Change (PGIC) -Fatigue

Phase 3: Time to First SCPC

Phase 3: Time to Second SCPC

Phase 3: Annualized Rate of Hospitalization Days for SCPC

Phase 3: Annualized Rate of Emergency Room Visits for SCPC

Phase 3: Change From Baseline in 6-Minute Walk Test (6MWT)

Phase 3: Change From Baseline in PROMIS Pain Intensity

Phase 3: Change From Baseline in Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me) Pain Impact

Phase 3: PGIC of Pain

Phase 3: Change From Baseline in PGIS of Pain

Phase 3: Percentage of Participants With Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious AEs (SAEs)

Phase 3: Pharmacokinetic/Pharmacodynamic Relationship: Evaluate the Exposure of Mitapivat to the Change in ATP and 2,3-DPG Levels

Phase 3: Mitapivat Concentration Over Time

Phase 3: Mitapivat Area Under the Concentration Curve

Phase 3: Mitapivat Maximum (Peak) Concentration

Full Information

NCT ID

NCT05031780

First Posted

July 29, 2021

Last Updated

October 11, 2023

Sponsor

Agios Pharmaceuticals, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT05031780

Brief Title

A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)

Official Title

A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Mitapivat in Subjects With Sickle Cell Disease

Study Type

Interventional

2. Study Status

Record Verification Date

October 2023

Overall Recruitment Status

Active, not recruiting

Study Start Date

February 11, 2022 (Actual)

Primary Completion Date

December 2025 (Anticipated)

Study Completion Date

February 2030 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Agios Pharmaceuticals, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

This clinical trial is a Phase 2/3 study that will determine the recommended dose of mitapivat and evaluate the efficacy and safety of mitapivat in sickle cell disease by testing how well mitapivat works compared to placebo to increase the amount of hemoglobin in the blood and to reduce or prevent the occurrence of sickle cell pain crises. In addition, the long-term effect of mitapivat on efficacy and safety will be explored in an open-label extension portion.

Detailed Description

Mitapivat is a small molecule, oral activator of pyruvate kinase R (PKR). PKR is involved with maintaining health, energy, and longevity of red blood cells (RBCs). The study aims to evaluate the efficacy and safety of treatment with mitapivat in participants with sickle cell disease. The study is a Phase 2/3 study in which the recommended dose of mitapivat will be selected and further evaluated. The Phase 2 portion includes a 12-week randomized, placebo-controlled period in which participants will be randomized in a 1:1:1 ratio to receive 2 dose levels of mitapivat or placebo. The Phase 3 portion includes a 52-week randomized, placebo-controlled period in which participants will be randomized in a 2:1 ratio to receive the recommended mitapivat dose level or placebo. Participants who complete either the Phase 2 or Phase 3 portion will have the option to move into a 216-week open label extension period to receive mitapivat.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Sickle Cell Disease

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2, Phase 3

Interventional Study Model

Sequential Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

277 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Phase 2: Mitapivat 50 mg BID

Arm Type

Experimental

Arm Description

Double-blind Period: Mitapivat 50 milligrams (mg) twice daily (BID) for 12 weeks.

Arm Title

Phase 2: Mitapivat 100 mg BID

Arm Type

Experimental

Arm Description

Double-blind Period: Mitapivat 100 mg BID for 12 weeks.

Arm Title

Phase 2: Placebo

Arm Type

Placebo Comparator

Arm Description

Double-blind Period: Mitapivat-matching placebo for 12 weeks.

Arm Title

Phase 2: Open-Label Extension Period

Arm Type

Experimental

Arm Description

Arm Title

Phase 3: Mitapivat selected Phase 3 dose

Arm Type

Experimental

Arm Description

Double-blind Period: Mitapivat selected Phase 3 dose (50 mg or 100 mg BID) for 52 weeks.

Arm Title

Phase 3: Placebo

Arm Type

Placebo Comparator

Arm Description

Double-blind Period: Mitapivat-matching placebo selected Phase 3 dose (which can be either 50 mg or 100 mg BID based on the phase 2 results) for 52 weeks.

Arm Title

Phase 3: Open-Label Extension Period

Arm Type

Experimental

Arm Description

Participants may choose to receive the selected Phase 3 mitapivat dose (which can be either 50 mg or 100 mg BID based on the phase 2 results) for 216 weeks after the Double-blind Period.

Intervention Type

Drug

Intervention Name(s)

Mitapivat

Other Intervention Name(s)

AG-348, Mitapivat Sulfate

Intervention Description

Mitapivat tablets

Intervention Type

Other

Intervention Name(s)

Mitapivat-matching placebo

Intervention Description

Placebo to match 50 mg or 100 mg tablets

Intervention Type

Other

Intervention Name(s)

Mitapivat-matching placebo

Intervention Description

Placebo to match selected Phase 3 dose tablets

Primary Outcome Measure Information:

Title

Phase 2: Percentage of Participants With Hemoglobin (Hb) Response

Time Frame

Week 12

Title

Phase 2: Percentage of Participants With Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious AEs (SAEs)

Time Frame

Up to Week 12

Title

Phase 3: Percentage of Participants With Hb Response

Time Frame

Week 52

Title

Phase 3: Annualized Rate of Sickle Cell Pain Crises (SCPCs)

Time Frame

Up to Week 52

Secondary Outcome Measure Information:

Title

Phase 2: Change From Baseline in Hb Concentration

Time Frame

Baseline, Week 10 up to Week 12

Title

Phase 2: Change From Baseline in Indirect Bilirubin

Time Frame

Baseline, Week 10 up to Week 12

Title

Phase 2: Change From Baseline in Lactate Dehydrogenase (LDH)

Time Frame

Baseline, Week 10 up to Week 12

Title

Phase 2: Change From Baseline in Absolute Reticulocytes Count

Time Frame

Baseline, Week 10 up to Week 12

Title

Phase 2: Change From Baseline in Percent Reticulocytes

Time Frame

Baseline, Week 10 up to Week 12

Title

Phase 2: Change From Baseline in Erythropoietin

Time Frame

Baseline, Week 10 up to Week 12

Title

Phase 2: Change From Baseline in Patient-Reported Outcomes Measurement Information System® (PROMIS®) Fatigue 13a Short Form (SF) Score

Time Frame

Baseline, Week 10 up to Week 12

Title

Phase 2: Annualized Rate of SCPCs

Time Frame

Up to Week 12

Title

Phase 2: Pharmacokinetic/Pharmacodynamic Relationship: Evaluate the Exposure of Mitapivat to the Change in Adenosine Triphosphate (ATP) and 2,3-Diphosphoglycerate (2,3-DPG)

Time Frame

Day 1 up to Week 8

Title

Phase 2: Mitapivat Concentration Over Time

Time Frame

Day 1 up to Week 8

Title

Phase 2: Mitapivat Area Under the Concentration

Time Frame

Day 1 up to Week 8

Title

Phase 2: Mitapivat Maximum (Peak) Concentration

Time Frame

Day 1 up to Week 8

Title

Phase 3: Change From Baseline in Hb Concentration

Time Frame

Baseline, Week 24 up to Week 52

Title

Phase 3: Change From Baseline in Indirect Bilirubin

Time Frame

Baseline, Week 24 up to Week 52

Title

Phase 3: Change From Baseline in Percent Reticulocytes

Time Frame

Baseline, Week 24 up to Week 52

Title

Phase 3: Change From Baseline in PROMIS® Fatigue 13a SF Scores

Time Frame

Baseline, Week 24 up to Week 52

Title

Phase 3: Annualized Frequency of Hospitalizations for SCPC

Time Frame

Up to Week 52

Title

Phase 3: Change From Baseline in LDH Concentration

Time Frame

Baseline, Week 24 up to Week 52

Title

Phase 3: Change From Baseline in Absolute Reticulocytes

Time Frame

Baseline, Week 24 up to Week 52

Title

Phase 3: Change From Baseline in Erythropoietin

Time Frame

Baseline, Week 24 up to Week 52

Title

Phase 3: Percentage of Participants With Improvement in the Patient Global Impression of Severity (PGIS) -Fatigue

Time Frame

Baseline, Weeks 24, 28, 40, and 52

Title

Phase 3: Percentage of Participants With Improvement in the Patient Global Impression of Change (PGIC) -Fatigue

Time Frame

Baseline, Weeks 24, 28, 40, and 52

Title

Phase 3: Time to First SCPC

Time Frame

Up to Week 52

Title

Phase 3: Time to Second SCPC

Time Frame

Up to Week 52

Title

Phase 3: Annualized Rate of Hospitalization Days for SCPC

Time Frame

Up to Week 52

Title

Phase 3: Annualized Rate of Emergency Room Visits for SCPC

Time Frame

Up to Week 52

Title

Phase 3: Change From Baseline in 6-Minute Walk Test (6MWT)

Time Frame

Baseline, Week 52

Title

Phase 3: Change From Baseline in PROMIS Pain Intensity

Time Frame

Baseline, Week 24 and 52

Title

Phase 3: Change From Baseline in Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me) Pain Impact

Time Frame

Baseline, Week 24 and 52

Title

Phase 3: PGIC of Pain

Time Frame

Baseline, Week 52

Title

Phase 3: Change From Baseline in PGIS of Pain

Time Frame

Baseline, Week 52

Title

Phase 3: Percentage of Participants With Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious AEs (SAEs)

Time Frame

Up to 56 weeks

Title

Phase 3: Pharmacokinetic/Pharmacodynamic Relationship: Evaluate the Exposure of Mitapivat to the Change in ATP and 2,3-DPG Levels

Time Frame

Day 1 up to Week 40

Title

Phase 3: Mitapivat Concentration Over Time

Time Frame

Day 1 up to Week 40

Title

Phase 3: Mitapivat Area Under the Concentration Curve

Time Frame

Day 1 up to Week 40

Title

Phase 3: Mitapivat Maximum (Peak) Concentration

Time Frame

Day 1 up to Week 40

10. Eligibility

Sex

All

Minimum Age & Unit of Time

16 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Age 16 years or older (18 years or older [France and Germany]); participants age 16 or 17 years must physically have completed puberty; Documented diagnosis of sickle cell disease (SCD) (HbSS, HbSC [combined heterozygosity for hemoglobins S and C], HbS/beta 0- thalassemia, HbS/ beta plus thalassemia, or other sickle cell syndrome variants); At least 2 sickle cell pain crises (SCPCs) and no more than 10 SCPCs in the past 12 months; Hemoglobin at least 5.5 and 10.5 gram per deciliter (g/dL) at the most. Hemoglobin concentration must be based on an average of at least 2 Hb concentration measurements (separated by ≥7 days) collected during the Screening Period; If taking hydroxyurea, the hydroxyurea dose must be stable for at least 90 days before starting study drug; Women capable of becoming pregnant and men with partners who are women that are capable of becoming pregnant must agree to use 2 forms of contraception. Exclusion Criteria: Pregnant, breastfeeding, or parturient; Receiving regularly scheduled transfusions; Hepatobiliary disorders including but not limited to significant liver disease or gallbladder disease; Severe kidney disease; Prior exposure to gene therapy or prior bone marrow or stem cell transplantation; Currently receiving treatment for SCD (eg, voxelotor, crizanlizumab, L-glutamine), with the exception of hydroxyurea. The last dose of such therapies must have been administered at least 90 days before starting study drug; Currently receiving treatment with hematopoietic stimulating agents; the last dose must have been administered at least 90 days before starting study drug; Received treatment on another investigational trial within 90 days prior to start of study drug or plans to participate in another investigational drug trial; Taking medications that are strong inhibitors of CYP3A4/5 or strong inducers of CYP3A4 that cannot be stopped in an acceptable timeframe before starting study drug (timeframe will be discussed with your doctor).

Facility Information:

Facility Name

University of California San Diego

City

La Jolla

State/Province

California

ZIP/Postal Code

92037-1337

Country

United States

Facility Name

UCLA Health

City

Los Angeles

State/Province

California

ZIP/Postal Code

90095-1678

Country

United States

Facility Name

Children's Hospital Oakland

City

Oakland

State/Province

California

ZIP/Postal Code

94609

Country

United States

Facility Name

Children's National Hospital

City

Washington

State/Province

District of Columbia

ZIP/Postal Code

20010-2916

Country

United States

Facility Name

Kaiser Permanente - Largo Medical Center

City

Largo

State/Province

Maryland

ZIP/Postal Code

20774-5374

Country

United States

Facility Name

Massachusetts General Hospital

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02114-2621

Country

United States

Facility Name

Boston Medical Center & Boston University School of Medicine

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02118

Country

United States

Facility Name

University of Michigan

City

Ann Arbor

State/Province

Michigan

ZIP/Postal Code

48109-5000

Country

United States

Facility Name

Children's Hospital of Michigan

City

Detroit

State/Province

Michigan

ZIP/Postal Code

48201

Country

United States

Facility Name

Mississippi Center for Advanced Medicine

City

Madison

State/Province

Mississippi

ZIP/Postal Code

39110-6115

Country

United States

Facility Name

Cure 4 The Kids Foundation, A Division of Roseman University of Health Sciences

City

Las Vegas

State/Province

Nevada

ZIP/Postal Code

89106

Country

United States

Facility Name

East Carolina University - Brody School of Medicine

City

Greenville

State/Province

North Carolina

ZIP/Postal Code

27834

Country

United States

Facility Name

Penn State Health Milton S. Hershey Medical Center

City

Hershey

State/Province

Pennsylvania

ZIP/Postal Code

17033

Country

United States

Facility Name

Penn Medicine - University of Pennsylvania Health System

City

Philadelphia

State/Province

Pennsylvania

ZIP/Postal Code

19104-5127

Country

United States

Facility Name

St. Christopher's Hospital for Children

City

Philadelphia

State/Province

Pennsylvania

ZIP/Postal Code

19134-1011

Country

United States

Facility Name

University of Texas Health Science Center of Houston

City

Houston

State/Province

Texas

ZIP/Postal Code

77030-1501

Country

United States

Facility Name

Seattle Cancer Care Alliance, University of Washington

City

Seattle

State/Province

Washington

ZIP/Postal Code

98195

Country

United States

Facility Name

Hôpital Erasme

City

Anderlecht

State/Province

Brussels

ZIP/Postal Code

1070

Country

Belgium

Facility Name

ZNA Stuivenberg

City

Antwerpen

State/Province

Brussels

ZIP/Postal Code

2060

Country

Belgium

Facility Name

Universitair Ziekenhuis Antwerpen

City

Edegem

State/Province

Brussels

ZIP/Postal Code

2650

Country

Belgium

Facility Name

CHR de la Citadelle

City

Liège

ZIP/Postal Code

4000

Country

Belgium

Facility Name

Clinique CHC MontLégia

City

Liège

ZIP/Postal Code

4000

Country

Belgium

Facility Name

Hospital de Clinicas de Porto Alegre (HCPA) - PPDS

City

Porto Alegre

State/Province

Rio Grande Do Sul

ZIP/Postal Code

90035-903

Country

Brazil

Facility Name

Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul (PUCRS)

City

Porto Alegre

State/Province

Rio Grande Do Sul

ZIP/Postal Code

90619-900

Country

Brazil

Facility Name

Hospital de Clínicas da Unicamp

City

Campinas

State/Province

São Paulo

ZIP/Postal Code

13083-878

Country

Brazil

Facility Name

Hospital Das Clínicas da Faculdade de Medicina de Ribeirão Preto - USP

City

Ribeirão Preto

State/Province

São Paulo

ZIP/Postal Code

14051-140

Country

Brazil

Facility Name

Praxis Pesquisa Medica

City

Santo Andre

State/Province

São Paulo

Country

Brazil

Facility Name

HEMORIO Instituto Nacional de Hematologia

City

Rio De Janeiro

ZIP/Postal Code

20211-030

Country

Brazil

Facility Name

Instituto do Cancer do Estado de São Paulo, Hospital das Clínicas da Faculdade de Medicina da Universidad de São Paulo

City

São Paulo

ZIP/Postal Code

05403-010

Country

Brazil

Facility Name

CHU Montreal

City

Montreal

State/Province

Quebec

ZIP/Postal Code

H2X 3E4

Country

Canada

Facility Name

McGill University Health Center

City

Montreal

State/Province

Quebec

ZIP/Postal Code

H3A 2B4

Country

Canada

Facility Name

Hopitaux de La Timone

City

Marseille

State/Province

Bouches-du-Rhône

ZIP/Postal Code

13005

Country

France

Facility Name

Hôpital Pellegrin, CHU de Bordeaux

City

Bordeaux

State/Province

Gironde

ZIP/Postal Code

33000

Country

France

Facility Name

Institut Universitaire du Cancer de Toulouse - Oncopole

City

Toulouse

State/Province

Haute-Garonne

ZIP/Postal Code

31100

Country

France

Facility Name

Hôpital Européen Georges Pompidou

City

Paris

State/Province

Ile De France

ZIP/Postal Code

75015

Country

France

Facility Name

CHU Hôpital Henri Mondor

City

Créteil

State/Province

Val-de-Marne

ZIP/Postal Code

94000

Country

France

Facility Name

HaEmek Medical Center

City

Afula

ZIP/Postal Code

1834111

Country

Israel

Facility Name

Rambam Medical Center

City

Haifa

State/Province

Ḥeifā

ZIP/Postal Code

31096

Country

Israel

Facility Name

Ziv Medical Center

City

Safed

State/Province

Ḥeifā

ZIP/Postal Code

13100

Country

Israel

Facility Name

A.O.R.N. "A. Cardarelli"

City

Napoli

State/Province

Campania

ZIP/Postal Code

80131

Country

Italy

Facility Name

AOU dell'Università degli Studi della Campania Luigi Vanvitelli

City

Napoli

State/Province

Campania

ZIP/Postal Code

80138

Country

Italy

Facility Name

Ente Ospedaliero Ospedali Galliera

City

Genova

State/Province

Liguria

ZIP/Postal Code

16128

Country

Italy

Facility Name

Kenya Medical Research Institute (KEMRI) Centre for Clinical Research Butere County Hospital

City

Butere

State/Province

Western Kenya

Country

Kenya

Facility Name

American University of Beirut Medical Center

City

Beirut

State/Province

Beyrouth

Country

Lebanon

Facility Name

Nini Hospital

City

Tarablus

State/Province

Liban Nord

Country

Lebanon

Facility Name

Hammoud Hospital University Medical Center

City

Sidon

Country

Lebanon

Facility Name

Erasmus MC

City

Rotterdam

State/Province

Zuid-Holland

ZIP/Postal Code

3015 GD

Country

Netherlands

Facility Name

National Hospital Abuja

City

Abuja

State/Province

Abuja Capital Territory

Country

Nigeria

Facility Name

University of Abuja Teaching Hospital

City

Abuja

State/Province

Abuja Capital Territory

Country

Nigeria

Facility Name

Lagos University Teaching Hospital

City

Suru-Lere

State/Province

Lagos

ZIP/Postal Code

101014

Country

Nigeria

Facility Name

Sultan Qaboos University Hospital, Hematology Department, COM&HS

City

Muscat

State/Province

Musqal

Country

Oman

Facility Name

King Abdullah International Medical Research Center

City

Riyadh

ZIP/Postal Code

1515 (KAIMRC)

Country

Saudi Arabia

Facility Name

Hacettepe University

City

Ankara

State/Province

Adana

Country

Turkey

Facility Name

Acibadem Adana Hospital

City

Seyhan

State/Province

Adana

ZIP/Postal Code

01130

Country

Turkey

Facility Name

Evelina Children's Hospital

City

London

State/Province

City Of London

ZIP/Postal Code

SE1 7EH

Country

United Kingdom

Facility Name

Hammersmith Hospital

City

London

ZIP/Postal Code

W12 0HS

Country

United Kingdom

Facility Name

Manchester Royal Infirmary, Manchester University NHS Foundation Trust

City

Manchester

ZIP/Postal Code

M13 9WL

Country

United Kingdom

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)

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