131I-apamistamab-based Conditioning for Hematopoietic Stem Cell Transplant (HSCT) in Advanced Sickle Cell Disease (SCD)

Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age12-50
SponsorColumbia University

About this trial

The purpose of this study is to find the smallest amount of the 131 I-apamistamab needed for preparing patients with severe sickle cell disease (SCD) for a bone marrow transplant. This is the first time 131 I-apamistamab is being used for advanced Sickle Cell Disease (SCD) in the setting of allogeneic stem cell transplant. 131 I-apamistamab is an investigational product. This means that 131 I-apamistamab has not been approved by the Food and Drug Administration (FDA) for medical use in patients.

The study treatment that is given before the transplant is called the conditioning regimen. In this study, the investigators are adding a drug called 131 I-apamistamab instead of the conditioning regimen typically given before a stem cell transplant.

Eligibility criteria

Qualifiers

Availability of an HLA-matched sibling donor

Clinically significant neurologic event (stroke) or any neurological deficit lasting at least 24 hours. Stroke will be defined as a clinically significant neurologic event that is accompanied by an infarct on cerebral MRI or cerebral arteriopathy requiring chronic transfusion therapy.

History of two or more episodes of ACS in the 2-year period preceding enrollment despite supportive care measures (i.e. asthma therapy and/or hydroxyurea).

History of three or more severe vaso-occlusive pain crises per year in the 2-year period preceding enrollment despite the institution of supportive care measures (i.e. a pain management plan and/or treatment with hydroxyurea).

Disqualifiers

Pulmonary dysfunction defined as DLCO (corrected for hemoglobin and alveolar volume) < 40% of predicted OR baseline oxygen saturation of <85% or PaO2 <70.

Severe cardiac dysfunction defined as ejection fraction <35%.

Impaired renal function defined as GFR <40.

Hepatic dysfunction defined as bridging (portal to portal) fibrosis or cirrhosis of the liver OR transaminases >5x ULN for age.

Trial design

Treatments tested in this trial

  • 131I-apamistmab
  • Sirolimus
  • Campath
  • Total Body Irradiation
  • Exchange Transfusion
  • Planar gamma imaging

Treatment groups

24 Participants
are divided into 1 treatment group

Sponsors and collaborators

Columbia University

Lead sponsor

Actinium Pharmaceuticals

Collaborator