The MOMENTUM study is evaluating an investigational therapy (given only one time) that allows the body to produce fetal hemoglobin.
- Current treatment options for sickle cell disease have limitations: they may not work for everyone and need to be taken forever to maintain benefits.
- The MOMENTUM study is evaluating an investigational therapy (given only one time) that allows the body to produce fetal hemoglobin.
- Fetal hemoglobin is an “anti-sickling” hemoglobin that is present in the red blood cells before birth; however, most people stop producing it shortly after birth, which causes it to lose its “anti-sickling” effect.
- Higher levels of fetal hemoglobin are known to reduce the vaso-occlusive crises that can cause acute pain episodes in people with sickle cell disease.
- The purpose of this study is to see if this one-time investigational drug is a safe and effective way to reduce or eliminate vaso-occlusive crises in sickle cell disease.
About the Investigational Drug
- This investigational drug is called ARU-1801.
- ARU-1801 is designed to add a new fetal hemoglobin-generating gene into a person’s own blood-making stem cells.
- The new gene is intended to transform those cells and to allow them to make more fetal hemoglobin.
- If more fetal hemoglobin is made, there may be less sickle–shaped red blood cells. This could lead to less red blood cell clumping and help ease other symptoms of SCD.
A Phase 1/2 Trial Investigating ARU-1801 in Adults with SCD
- The MOMENTUM study is a Phase 1/2 trial that will assess the safety and efficacy of ARU-1801 in adults (age 18 to 45) with SCD.
- Momentum has already started treating patients with ARU-1801.
- All eligible participants who complete preparation will receive ARU-1801.
- The study requires only one administration of ARU-1801.
- ARU-1801 is designed to permanently add an extra fetal hemoglobin gene into a person’s blood-making stem cells. The goal is to increase production of fetal hemoglobin and reduce the sickle–shaped adult hemoglobin in red blood cells. This could lead to less red blood cell clumping and help ease other symptoms of SCD.
- Current trial sites
- Cincinnati Children’s Hospital Medical Center, Cincinnati, Ohio.
- Atrium Health, Levine Cancer Institute, Charlotte, North Carolina.
- University of the West Indies Hospital, Kingston, Jamaica.
- University of Pennsylvania, Philadelphia, Pennsylvania.
- Study participation requires two trips to Cincinnati (for apheresis and product administration) with all other visits taking place at site.
- All travel expenses for patient and one family member are reimbursed.
Who can Participate?
Have severe SCD as defined by having had one of the following:
- Two or more painful crises requiring treatment at a medical facility in your life, or
- Two or more episodes of acute chest syndrome in your life, or
- One episode of life-threatening acute chest syndrome in your life, or
- On chronic transfusion therapy for disease symptoms.
Mikiyah gives her perspective on participating in a gene therapy clinical trial. She talks about a range of topics from the challenges of growing up with sickle cell to why she decided to participate in the trial.
Key MOMENTUM Information
- Researchers are examining the safety and efficacy of a one-time investigational drug that is designed to be curative.
- All participants receive access to investigational drug.
- Participants are paid for their time and reimbursed for travel to take part in this study.
- All research procedures will be covered by the study.
What are the benefits and risks?
- You will be able to try an investigational new therapy that might help you reduce your SCD symptoms.
- You will have access to this experimental therapy before it is otherwise available.
- The experimental therapy may or may not help your symptoms, but what we learn may help other people with SCD.
The full detailed risks and potential benefits of this trial will be reviewed with you by the study team and investigator, for you to make an informed decision about whether to participate.
To learn more about our study, download the study brochure here.
Sickle Cell Disease Resources
- National Human Genome Research Institute: About Sickle Cell Disease
- U.S. National Library of Medicine: Sickle Cell Disease
- National Heart, Lung, and Blood Institute: Sickle Cell Disease
- U.S. National Library of Medicine: Help Me Understand Genetics: Gene Therapy
- FDA: What Is Gene Therapy? How Does It Work?
- American Society of Gene and Cell Therapy: Gene Therapy Basics
Patient Advocacy and Support
- Cure Sickle Cell
- Raremark: Sickle Cell Disease
- Sickle Cell Alliance Foundation
- Sickle Cell Disease Association of America
- Sickle Cell Information Center
- Sickle Cell Society