The MOMENTUM Study: Gene Transfer for Patients With Sickle Cell Disease
The Sickle Cell 101 research team in collaboration with study investigator have reviewed this information to ensure it is patient-friendly and accurate.
About the study
The MOMENTUM study is determining if an investigational gene therapy allows the body to produce more fetal hemoglobin. Fetal hemoglobin has anti-sickling properties and higher levels are known to reduce vaso-occlusive crises.
The purpose of this study is to see if a one-time investigational drug is a safe and effective way to reduce or eliminate vaso-occlusive crises in sickle cell disease by producing more fetal hemoglobin.
MORE ABOUT THIS STUDY
Current treatment options for sickle cell disease have limitations; they may not work for everyone and may also need to be taken for a lifetime to maintain benefits.
Fetal hemoglobin is present in the red blood cells before birth. Typically, people stop producing it shortly after birth and transition to either sickle or non-sickle hemoglobin. Higher levels of fetal hemoglobin are known to reduce vaso-occlusive crises that can cause pain episodes in people living with sickle cell disease. This study aims to utilize an investigational gene therapy to allow the body to produce more fetal hemoglobin in sickle cell disease.
Confirmed severe sickle cell disease diagnosis with at least one of the following:
Minimum of two episodes of acute chest syndrome (ACS) requiring hospital admission, one life threatening episode of ACS requiring Intensive Care Unit (ICU) admission for exchange transfusion and/or intubation, or frequent ACS episodes which necessitate treatment with long term transfusion therapy
Two or more severe acute sickle pain events per year requiring additional treatment at a medical facility outside of home pain management over the preceding 2-year period prior to study enrollment, or that necessitate long term transfusion therapy.
Severe disease symptoms other than those listed above, and which interfere with normal life activities and require long term transfusion therapy.
Has failed or is unable or unwilling to take hydroxyurea therapy.
Has adequate functional status and organ function as determined at the initial screening.
Female who is breastfeeding or pregnant.
Refusal to practice effective birth control methods for 1 year after receiving treatment or who are not surgically sterile or postmenopausal (females).
Currently receiving treatment for most types of cancer.
Currently diagnosed with or has a history of hepatitis B, Hepatitis C, or HIV.
Has received another study drug within 30 days or 5 half-lives of the last dose prior to screening.
Severe obstruction, restriction, or diffusion defect on pulmonary function tests.
Uncontrolled bacterial, viral, or fungal infection within one month prior to study
History of stroke or at moderate to high risk of stroke.
Previous liver biopsy showing cirrhosis, bridging hepatic fibrosis, or active hepatitis, or has received chronic transfusions with previous evidence of iron overload and liver fibrosis.
Alpha thalassemia sickle cell disease.
Matched and feasible sibling donor.
Hypersensitivity to any study treatments.
Additional eligibility criteria will apply, and a study doctor will explain this information to you
Cincinnati, Ohio, Cincinnati Children’s Hospital Medical Center
Charlotte, North Carolina, Atrium Health, Levine Cancer Institute
Kingston, Jamaica, University of the West Indies Hospital
Philadelphia, Pennsylvania, University of Pennsylvania