NCT02484391

An Open Label Study to Evaluate the Feasibility of CPI-613 Given With High Dose Cytarabine and Mitoxantrone in Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML)

Completed Phase 1 Results posted Last updated 20 August 2024

What this trial tests

Phase 1 trial testing 6,8-Bis(benzylthio)octanoic Acid in Granulocytic Sarcoma in 48 participants. Completed in 2 February 2022.

Timeline

1 September 2015

Primary endpoint
1 October 2018

2 February 2022

Quick facts

Lead sponsor	Wake Forest University Health Sciences
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	48
Start date	1 September 2015
Primary completion	1 October 2018
Estimated completion	2 February 2022
Sites	1 location across United States

Drugs / interventions tested

• 6,8-Bis(benzylthio)octanoic Acid — full drug profile →
• Cytarabine — full drug profile →
• Hematopoietic Cell Transplantation — full drug profile →
• Mitoxantrone Hydrochloride — full drug profile →

Conditions studied

• Granulocytic Sarcoma — all drugs for Granulocytic Sarcoma →
• Recurrent Adult Acute Myeloid Leukemia — all drugs for Recurrent Adult Acute Myeloid Leukemia →

Sponsor

Wake Forest University Health Sciences

Who can join

18 and older, any sex, with Granulocytic Sarcoma or Recurrent Adult Acute Myeloid Leukemia. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Feasibility of Administering CPI-613 in Combination With High Dose Cytarabine and Mitoxantrone During Induction, Consolidation and Maintenance Therapies, Defined as Percentage of Patients Eligible for Maintenance Therapy Who Complete at Least 3 Courses
  Time frame: Up to 12 weeks of maintenance therapy
  Feasibility is determined by the percentage of patients eligible for maintenance therapy who complete at least 3 cycles, if ≥50% of eligible patients complete 3 cycles of maintenance therapy we will consider this regimen feasible for future study.

Sponsor's own description

This pilot phase I trial studies how well CPI-613 (6,8-bis\[benzylthio\]octanoic acid), cytarabine, and mitoxantrone hydrochloride work in treating patients with acute myeloid leukemia or granulocytic sarcoma (a malignant, green-colored tumor of myeloid cells \[a type of immature white blood cell\]) that has returned (relapsed) or that does not respond to treatment (refractory). 6,8-bis(benzylthio)octanoic acid is thought to kill cancer cells by turning off their mitochondria. Mitochondria are used by cancer cells to produce energy and are the building blocks needed to make more cancer cells. By shutting off these mitochondria, 6,8-bis(benzylthio)octanoic acid deprives the cancer cells of energy and other supplies that they need to survive and grow in the body. Drugs used in chemotherapy, such as cytarabine and mitoxantrone hydrochloride, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving 6,8-bis(benzylthio)octanoic acid together with cytarabine and mitoxantrone hydrochloride may kill more cancer cells.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Reactive Oxygen Species in the Tumor Microenvironment: An Overview.
   Weinberg F, Ramnath N, Nagrath D. · · 2019 · cited 301× · PMID 31426364 · DOI 10.3390/cancers11081191
2. Mitochondria in cancer metabolism, an organelle whose time has come?
   Anderson RG, Ghiraldeli LP, Pardee TS. · · 2018 · cited 49× · PMID 29807044 · DOI 10.1016/j.bbcan.2018.05.005
3. Mitochondrial metabolism and cancer therapeutic innovation.
   Du H, Xu T, Yu S, Wu S, et al · · 2025 · cited 43× · PMID 40754534 · DOI 10.1038/s41392-025-02311-x
4. Phase II trial of cytarabine and mitoxantrone with devimistat in acute myeloid leukemia.
   Anderson R, Miller LD, Isom S, Chou JW, et al · · 2022 · cited 32× · PMID 35354808 · DOI 10.1038/s41467-022-29039-4
5. The Tricarboxylic Acid Cycle Metabolites for Cancer: Friend or Enemy.
   Wu J, Liu N, Chen J, Tao Q, et al · · 2024 · cited 29× · PMID 38867720 · DOI 10.34133/research.0351
6. Role of mitochondria in physiological activities, diseases, and therapy.
   Wang L, Zhou X, Lu T. · · 2025 · cited 23× · PMID 40536597 · DOI 10.1186/s43556-025-00284-5
7. Targeting membrane contact sites to mediate lipid dynamics: innovative cancer therapies.
   Wang J, Wang M, Zeng X, Li Y, et al · · 2025 · cited 5× · PMID 39955542 · DOI 10.1186/s12964-025-02089-z
8. Feasibility and Safety of Targeting Mitochondria Function and Metabolism in Acute Myeloid Leukemia.
   Firmanty P, Chomczyk M, Dash S, Konopleva M, et al · · 2024 · cited 3× · PMID 40756330 · DOI 10.1007/s40495-024-00378-8