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
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|ClinicalTrials.gov Identifier: NCT02462252|
Recruitment Status : Unknown
Verified August 2017 by BioLineRx, Ltd..
Recruitment status was: Recruiting
First Posted : June 4, 2015
Last Update Posted : August 18, 2017
|Condition or disease||Intervention/treatment||Phase|
|Aplastic Anemia Hypoplastic Myelodysplastic Syndrome||Drug: BL-8040 Drug: horse anti-thymocyte globulin (hATG) Drug: Methylprednisolone Drug: Cyclosporine||Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||25 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase IIA, Open-label Study Designed to Evaluate Efficacy and Safety of BL-8040 Followed by Anti-Thymocyte Globulin (hATG), Cyclosporine and Methylprednisolone in Adult Subjects With Aplastic Anemia (AA) or Hypoplastic Myelodysplastic Syndrome (MDS)|
|Study Start Date :||October 2015|
|Estimated Primary Completion Date :||January 2018|
|Estimated Study Completion Date :||April 2018|
Experimental: BL-8040 plus hATG, methylprednisolone, cyclosporine
BL-8040 0.75mg/kg will be administered Subcutaneously (SC ) on Days 1-10, then on first 5 days of months 2-6.
Standard therapy: hATG: Days 11-14: 40mg/kg/day IV over 6-8 hours, in all AA subjects, or in MDS subjects less than 55 years old. 35mg/kg/day IV over 6-8 hours in MDS subjects 55 years and older.
Standard therapy: methylprednisolone: Days 11-14: 40mg/kg/day IV over 6-8 hours, in all AA subjects, or in MDS subjects less than 55 years old. 35mg/kg/day IV over 6-8 hours in MDS subjects 55 years and older.
Standard therapy: cyclosporine: 5mg/kg/day orally, given in 2 divided doses, starting on day 11 and continuing through Month 6 Day 30 (end of treatment)
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
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
From Day 11 through end of treatment (Month 6 Day 30), subjects will receive oral dose of cyclosporine.
Other Name: Sandimmune, Cyclosporine A
- Response Rate [ Time Frame: up to 6 months (180 days) ]95% confidence interval (CI) will be calculated for subjects who achieve Complete response (CR), Partial response (PR), for all AA and MDS subjects, and hematological improvement (HI) for MDS subjects only. Measurement in percentages (%)
- Toxicity [ Time Frame: study treatment duration (180 days) plus 30 days following last dose of last treatment ]Safety and tolerability parameter monitored according to CTCAE version 4.03 criteria for adverse events and clinical laboratory parameters. Adverse events will be summarized by MedDRA dictionary and by patient population (AA /MDS)
- general safety [ Time Frame: up to end of study treatment (180 days) ]measurement of vital signs, ECG, and physical exam
- tolerability [ Time Frame: up to end of end of study treatment (180 days) ]number of subjects who discontinued study treatment early for any reason or due to toxicity
- Time to response [ Time Frame: up to 5 years ]Interval between treatment start and date of best response following treatment with the BL-8040 in addition to hATG, cyclosporine, and methylprednisolone.
- Response duration [ Time Frame: up to 5 years ]Time interval between the date of complete response (CR) and the date of first sign of disease recurrence or last follow-up or to the date of last follow-up if patients are alive without disease recurrence, within those patients who have achieved CR.
- Overall survival [ Time Frame: up to 5 years ]Time from treatment start until the death or last follow-up time (visit or call).
- Change in blood product requirements compared to Baseline [ Time Frame: up to 6 months (180 days) ]number of transfusions per month compared to Baseline
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02462252
|Contact: Anne Goren, B.Sc.Pharm||972-8-642-9100 ext email@example.com|
|Contact: Dana Keinan, PhD||972-8-642-9100 ext firstname.lastname@example.org|
|United States, Texas|
|MD Anderson Cancer Center||Recruiting|
|Houston, Texas, United States, 77030|
|Contact: Sheri Rivera 713-563-4553|
|Principal Investigator: Tapan Kadia, MD|
|Principal Investigator:||Tapan Kadia, MD||M.D. Anderson Cancer Center|