NCT01902381

A Pilot Study of CPI-613 in Patients With Myelodysplastic Syndrome Who Have Failed Previous Therapy

Terminated Phase 2 Results posted Last updated 15 October 2024

What this trial tests

Phase 2 trial testing 6,8-bis(benzylthio)octanoic acid in Previously Treated Myelodysplastic Syndromes in 12 participants. Terminated before completion.

Timeline

1 August 2013

Primary endpoint
26 November 2018

26 November 2018

Quick facts

Lead sponsor	Wake Forest University Health Sciences
Phase	Phase 2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	12
Start date	1 August 2013
Primary completion	26 November 2018
Estimated completion	26 November 2018
Sites	1 location across United States

Drugs / interventions tested

• 6,8-bis(benzylthio)octanoic acid — full drug profile →

Conditions studied

• Previously Treated Myelodysplastic Syndromes — all drugs for Previously Treated Myelodysplastic Syndromes →

Sponsor

Wake Forest University Health Sciences

Who can join

18 and older, any sex, with Previously Treated Myelodysplastic Syndromes. 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.

• Response Rate (RR), Defined as the Combined Rate of Complete Remission (CR), Marrow CR, Partial Remission (PR), or Stable Disease (SD), as Described by Cheson, et al. (2006)
  Time frame: Up to 5 years
  Response rate (RR), defined as the combined of complete remission (CR), marrow CR, partial remission (PR), or stable disease (SD), as described by Cheson, et al. (2006). The number of patients achieving RR will be presented. Complete remission - Bone marrow: ≤ 5% myeloblasts with normal maturation of all cell lines; Marrow CR - Bone marrow: ≤ 5% myeloblasts and decrease by ≥ 50% over pretreatment

Sponsor's own description

This pilot clinical trial studies 6, 8-bis (benzylthio) octanoic acid (CPI-613) in treating patients with myelodysplastic syndromes who failed previous therapy. Sometimes when chemotherapy or biological therapy is given, it does not stop the growth of tumor cells. The tumor is said to be resistant to treatment. 6, 8-bis (benzylthio) octanoic acid may interfere with the growth of tumor cells and may be an effective treatment for myelodysplastic syndromes that did not respond to previous therapy.

Publications & conference data

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

1. The emerging role and targetability of the TCA cycle in cancer metabolism.
   Anderson NM, Mucka P, Kern JG, Feng H. · · 2018 · cited 404× · PMID 28748451 · DOI 10.1007/s13238-017-0451-1
2. Communication in the Cancer Microenvironment as a Target for Therapeutic Interventions.
   Dominiak A, Chełstowska B, Olejarz W, Nowicka G. · · 2020 · cited 134× · PMID 32422889 · DOI 10.3390/cancers12051232
3. Treatment options for patients with myelodysplastic syndromes after hypomethylating agent failure.
   Carraway HE. · · 2016 · cited 7× · PMID 27913518 · DOI 10.1182/asheducation-2016.1.470
4. A Single-Center Retrospective Study of Acute Kidney Injury Incidence in Patients With Advanced Malignancies Treated With Antimitochondrial Targeted Drug.
   Anderson EM, Zhang J, Russell G, Bowline IG, et al · · 2019 · cited 1× · PMID 30775628 · DOI 10.1016/j.ekir.2018.10.021