Instructions
If you are interested in learning more about your possible participation in this clinical trial, please complete the form. Your information will be forwarded directly to the sponsoring company.
Status:
Recruiting
Phase:
Phase 1
Phase 2
Gender:
Female
Male
Age Group:
18 years and older
Accepts Healthy Volunteers:
No
Inclusion Criteria:
- SAA that is refractory to or has relapsed while on standard of care IST, as defined in the protocol
- Hematopoietic stem cell transplantation (HSCT) is not available or suitable as a treatment option or has been refused by the patient
- Adequate hepatic and renal function as defined in the protocol
Exclusion Criteria:
- Diagnosis of Fanconi anemia as defined in the protocol
- Evidence of myelodysplastic syndrome as defined in the protocol
- Paroxysmal nocturnal hemoglobinuria (PNH) with evidence of significant hemolysis or history of PNH-associated thrombosis
- Treatment with a T cell-depleting agent (eg, ATG or alemtuzumab) within 6 months prior to dosing
- Treatment with a calcineurin inhibitor (eg, cyclosporine) within 4 weeks prior to dosing
- Treatment with eltrombopag or investigational thrombopoietin receptor agonist, Granulocyte Colony-Stimulating Factor (G-CSF), or an androgen (eg, danazol), within 2 weeks prior to dosing
- HIV, hepatitis B or hepatitis C positive by serological testing at the screening visit
- Active tuberculosis, latent tuberculosis infection (LTBI) or history incompletely-treated tuberculosis or LTBI
Note: Other protocol-defined inclusion/ exclusion criteria apply.