Phase IIA Open Label Study to Evaluate Efficacy and Safety of BL-8040 Followed by (hATG), Cyclosporine and Methyprednisolone in Adult Subjects With Aplastic Anemia or Hypoplastic Myelodysplastic Syndrome | Aplastic Anemia and MDS International Foundation

Clinical Trial: NCT02462252

Phase IIA Open Label Study to Evaluate Efficacy and Safety of BL-8040 Followed by (hATG), Cyclosporine and Methyprednisolone in Adult Subjects With Aplastic Anemia or Hypoplastic Myelodysplastic Syndrome
For more details on this clinical trial, including contact information, please see this trial’s listing on clinicaltrials.gov:
Purpose: 

This will be an open-label, single arm, phase IIa study in subjects with AA or Hypoplastic MDS.

Eligible subjects will receive subcutaneous (SC) injections of BL-8040 monotherapy over 10 days. From Day 11 through Day 14, subjects will receive hATG, Methylprednisolone and Cyclosporine. From Day 15 until Day 30 (of the first month), subjects will continue treatment only with Methylprednisolone and Cyclosporine. Cyclosporine will continue daily through Month 6/Day 30 (M6/D30) (end of study treatment). Beginning on M2/D1, BL-8040 will be administered daily as part of the maintenance period for the first 5 days of each month through M6. All BL-8040 injection courses will be given at the site, as either an inpatient or outpatient per the treating physician's decision.

The primary objective of the study is to determine the efficacy of the treatment with BL-8040 on top of the standard immunotherapy regimen of: hATG, cyclosporine, and steroids in patients with Hypoplastic MDS and AA. Safety and efficacy will be assessed at defined time-points throughout the study. Duration of response and overall survival will be assessed as a part of the long term FU. A maximum of 25 patients will be enrolled in the study. Subjects will be equally distributed between the disease populations.

Status: 
Recruiting
Study Date: 
Mon, 05/25/2015 to Sat, 07/01/2017
Bone Marrow Disease(s): 
aplastic anemia
Associated Drug(s): 
Intervention: 
Drug: BL-8040 Subjects will receive subcutaneous (SC) injections of BL-8040 monotherapy on days 1-10 of the study. Beginning on Month 2 day 1, (M2/D1), and continuing monthly through Month 6, BL-8040 will be administered daily as part of the maintenance period for the first 5 days of each month. Drug: horse anti-thymocyte globulin (hATG) From Day 11 through Day 14 of first month, subjects will receive hATG infusion over 6-8 hours each day. Other Name: ATGAM Drug: Methylprednisolone From Day 11-14 of first month, subjects receive infusion of methylprednisolone 30 minutes prior to hATG infusion. Treatment with methylprednisolone will continue for 30 days. (After day 14, subjects may receive oral prednisone dose equivalent to IV methylprednisolone dose. The oral dose will be tapered off over 30 days. Other Name: Medrol, Solu-Medrol, Depo-Medrol Drug: Cyclosporine From Day 11 through end of treatment (Month 6 Day 30), subjects will receive oral dose of cyclosporine. Other Name: Sandimmune, Cyclosporine A