For Medical Professionals

The MOMENTUM STUDY for Patients with Sickle Cell Disease

ID: NCT02186418

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Purpose This open-label, Phase 1/2 study will determine the safety, efficacy and feasibility of ARU-1801 in patients with severe sickle cell disease (SCD).
Investigational Product ARU-1801 is autologous CD34+ cells transduced ex vivo with a lentiviral vector containing a modified gamma-globin gene.
Mechanism of Action ARU-1801 is designed to increase production of fetal hemoglobin (HbF) in order to reduce vaso-occlusive crises, hospitalizations and cumulative organ damage. The modified γ-globin gene contains a point mutation at codon 16 that improves the affinity of γ-globin to α-globin, which promotes the preferential formation of a modified HbF tetramer.
Reduced Intensity Conditioning The proprietary modification of the gamma globin allows engraftment with only reduced intensity conditioning, and does not require fully myeloablative conditioning prior to treatment.

For Medical Professionals

Study Population

  • Ages 18 to 45 years.
  • Confirmed diagnosis of severe SCD (HbSS, HbS-b0 or Hbs-b+ thalassemia).
  • Severe SCD is defined as one or more of the following:
    • At least two severe acute sickle pain events per year requiring treatment at a medical facility outside of home, or
    • Two or more lifetime episodes of acute chest syndrome (ACS), or
    • One episode of ACS requiring intensive care unit (ICU) admission, or
    • Chronic transfusions required for management of disease symptoms.
  • Adequate functional status and organ function.

For Medical Professionals

Trial Sites

  • 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.

For Medical Professionals

Duration and Follow-Up

After ARU-1801 infusion, patients are monitored to evaluate the efficacy, safety and durability of ARU-1801. The follow-up schedule is as follows:

  • Once per month for the first year
  • Every three months in Year 2
  • Every 6 months years 3-5
  • Every year in years 6-15 

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View the study brochure to share with a potential participant.