Ignite Creation Date:
2025-12-26 @ 10:19 PM
Ignite Modification Date:
2025-12-26 @ 10:19 PM
Study NCT ID:
NCT02223312
Status:
WITHDRAWN
Last Update Posted:
2018-08-28
First Post:
2014-08-11
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Therapy for Progressive and/or Refractory Hematologic Malignancies
Sponsor:
Kiromic BioPharma Inc.
Organization:
Study Overview
Official Title:
Phase I/II Study of Low-Dose Cyclophosphamide, Tumor Associated Peptide Antigen-Pulsed Dendritic Cell Therapy and Low Dose GM-CSF, in Patients With Progressive and/or Refractory Hematologic Malignancies
Status:
WITHDRAWN
Status Verified Date:
2018-01
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Sponsor decision to discontinue study.
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The purpose of this study is to evaluate the safety and effectiveness of Tumor Associated Peptide Antigen (TAPA) pulsed dendritic cell (DC) vaccines in the treatment of progressive and/or refractory hematologic malignancies (HM). We hypothesize that treatment of patients with relapsed and/or refractory HM, without available potentially curative treatment options, and whose neoplastic cells express at least one (1) TAPA of a defined panel of TAPAs, using low-dose cyclophosphamide (CYP) followed by an autologous, monocyte-derived, TAPA-pulsed DC vaccine and low-dose granulocyte macrophage colony stimulating factor (GM-CSF), will result in TAPA-specific T-cell responses without significant toxicities. We also hypothesize CD4+ T-cell and CD8+ T-cell responses generated against specific TAPAs may translate into clinical antitumor activity.
Detailed Description:
Patients diagnosed with progressive and/or refractory hematologic malignancies, who have failed conventional therapy and have no potentially curative therapeutic options available, will be candidates for this Phase I/II study. Following confirmation of disease progression and/or refractoriness, eligible patients who agree to participate and sign a consent form will have their neoplastic cells and/or blood analyzed for the expression of a specific panel of Tumor Associated Peptide Antigens (TAPAs), including SP17, Ropporin, AKAP4, PTTG1 and Span-xb. Patients whose tumors express one (1) or more of these TAPAs will receive three (3) days of subcutaneous Granulocyte Macrophage Colony Stimulating Factor (GM-CSF) to increase bone marrow production of monocytes and dendritic cell (DC) precursors, and peripheral blood mononuclear cells will be obtained by phlebotomy and/or leukapheresis for generation of autologous DCs. Patient's DCs will be generated at Kiromic's Cell Processing GMP facility, according to established Standard Operating Procedures, and activated by pulsing/loading them with the TAPA(s) relevant for each particular patient. Patients will receive five (5) days of low-dose cyclophosphamide prior to each vaccination with TAPA-pulsed DCs to decrease Treg activity. TAPA-pulsed DCs will be administered at a fixed dose of up to 1 x 107 DCs at least two (2) days following cyclophosphamide administration. DC vaccination schedule will be once every fourteen (14) days via subcutaneous (SC) and intradermal (ID) injections for a total of 6 vaccinations. Low dose GM-CSF will also be administered SC for five (5) consecutive days, starting three (3) to six (6) hours after each TAPA-pulsed DC treatment, to optimize immune response and DC viability in vivo. Patients will be followed on a weekly basis (or more frequently if required) to evaluate treatment-related toxicity. Immune responses and anti-tumor responses will be evaluated per protocol specifications. Continuation and stopping rules for the study will be defined based on toxicity/tolerability (Phase I) and/or immune efficacy (Phase II).
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| BSK01 Dendritic cell vaccine | OTHER | Kiromic | View |