RISE UP: Studying whether an investigational drug can increase hemoglobin and reduce pain crises
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What is this?
About the Study
The RISE UP Study seeks to determine if an investigational molecule called “mitapivat” has the potential to improve the health of red blood cells in individuals living with sickle cell disease by improving the energy and reducing the sickling of red blood cells. This could result in mitapivat potentially reducing pain crises and anemia.
This study seeks to determine mitapivat’s safety and effectiveness versus placebo in study participants with sickle cell disease over a year. the effects it has on people with sickle cell disease who take the therapy and its safety over a longer period of 1 year compared to individuals who take a placebo living with sickle cell disease.
Read moreParticipation information
Study Information
Study type
Clinical
Interventions
Mitapivat, placebo
Phase
3
Compensation
No
Am I Eligible?
Requirements
You may be eligible to participate in this study if you:- You may be eligible to enroll in this study if: A person CAN be eligible to participate in this study if:
- You are 16 years or older
- You are diagnosed with sickle cell disease (SCD)
- You have had at least two but no more than 10 pain crises in the past year. These are pain crises that required medical attention caused by acute chest syndrome, priapism, or red blood cell blockages (vaso-occlusions)
- You have a hemoglobin level between 5.5 and 10.5 g/dL
- You’re taking hydroxyurea OR if taking hydroxyurea, the dose has not changed within at least 90 days before starting this study drug
- If you or your partner are capable of becoming pregnant, you must agree to use 2 forms of contraception
- Other inclusions may apply
Restrictions
You can't participate in this study if you:- You cannot participate in this study if:
- You are pregnant or breastfeeding
- You regularly receive scheduled transfusions
- You have a hepatobiliary disorder, which may include liver or gallbladder disease(having to do with the liver, gallbladder, bile ducts, or bile) disorders, including but not limited to significant liver disease or gallbladder disease
- You have severe kidney disease
- You have undergone gene therapy or bone marrow/stem cell transplant
- You, except for hydroxyurea=, are currently taking a treatment for sickle cell disease such as voxelotor (Oxbryta), crizanlizumab (Adakveo), and L-glutamine (Endari) – hydroxyurea is okay within 90 days of starting the study drug
- You are currently receiving therapies that stimulate blood production such as erythropoietin (EPO) within 90 days of starting the study drug or plan to participate in another investigational drug trial
- Other exclusions may apply
Study point of contact
| Agios Medical Information | |
| 833-228-8474 | |
| [email protected] |
Study Sites
- Philadelphia, Pennsylvania, USA, Penn Medicine - University of Pennsylvania
- Philadelphia, Pennsylvania, USA, St. Christopher's Hospital for Children
- Los Angeles, California, USA, UCLA Health
- La Jolla, California, USA, University of California San Diego
- Ann Arbor, Michigan, USA, University of Michigan
- Boston, Massachusetts, USA, Boston Medical Center & Boston University School of Medicine
- Houston, Texas, USA, Texas Children's Hospital
- Richmond, Virginia, USA, Virginia Commonwealth University
- Bethesda, Maryland, USA, National Heart Lung and Blood Institute
- Houston, Texas, USA, University of Texas Health Science Center of Houston
- Las Vegas, Nevada, USA, Cure 4 The Kids Foundation, A Division of Roseman University of Health Sciences
- Washington, District of Columbia, USA, Children's National Hospital
- Madison, Mississippi, USA, Mississippi Center for Advanced Medicine
- Toronto, Ontario, Canada, University Health Network
- Montreal, Quebec, Canada, McGill University Health Center
- Montreal, Quebec, Canada, CHU Montreal
- Muscat, Musqal, Oman, Sultan Qaboos University Hospital, Hematology Department, COM&HS
- Utrecht, Netherlands, Universitair Medisch Centrum Utrecht
- Rotterdam, Zuid-Holland, Netherlands, Erasmus MC
- London, London, UK, Hammersmith Hospita
- London, London, UK, Evelina Children's Hospital
- Cambridge, UK, Cambridge University Hospitals NHS Foundation Trust
- Manchester, Manchester, UK, Manchester Royal Infirmary, Manchester University NHS Foundation Trust
- Edegem, Brussels, Belgium, Universitair Ziekenhuis Antwerpen
- Anderlecht, Brussels, Belgium, Hôpital Erasme
- Antwerpen, Brussels, Belgium, ZNA Stuivenberg
- Liège, Belgium, CHR de la Citadelle
- Liège, Belgium, Clinique CHC MontLégia
- Créteil, Val-de-Marne, France, CHU Hôpital Henri Mondor
- Marseille, Bouches-du-Rhône, France, Hopitaux de La Timone
- Toulouse, Haute-Garonne, France, Institut Universitaire du Cancer de Toulouse - Oncopole
- Guadeloupe, France, CHU Guadeloupe
- Paris, Ile De France, France, Hôpital Européen Georges Pompidou
- Bordeaux, Gironde, France, Hôpital Pellegrin, CHU de Bordeaux
- Essen, Germany, Universitätsklinikum Essen
- Regensburg, Germany, Universitätsklinikum Regensburg
- Modena, Emilia-Romagna, Italy, Azienda Ospedaliero Universitaria Di Modena Policlinico
- Kisumu, Western, Kenya, Kondele Children's Hospital