NCT00540722

A Phase 2 Study of R-(-)-Gossypol (Ascenta's AT-101) in Recurrent Glioblastoma Multiforme

Completed Phase 2 Results posted Last updated 12 July 2017

What this trial tests

Phase 2 trial testing R-(-)-gossypol acetic acid in Adult Giant Cell Glioblastoma in 56 participants. Completed in 1 June 2012.

Timeline

1 January 2008

Primary endpoint
1 June 2012

1 June 2012

Quick facts

Lead sponsor	National Cancer Institute (NCI)
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	56
Start date	1 January 2008
Primary completion	1 June 2012
Estimated completion	1 June 2012
Sites	8 locations across United States

Drugs / interventions tested

• R-(-)-gossypol acetic acid — full drug profile →
• laboratory biomarker analysis — full drug profile →

Conditions studied

• Adult Giant Cell Glioblastoma — all drugs for Adult Giant Cell Glioblastoma →
• Adult Glioblastoma — all drugs for Adult Glioblastoma →
• Adult Gliosarcoma — all drugs for Adult Gliosarcoma →
• Recurrent Adult Brain Tumor — all drugs for Recurrent Adult Brain Tumor →

Sponsor

National Cancer Institute (NCI)

Who can join

18 and older, any sex, with Adult Giant Cell Glioblastoma or Adult Glioblastoma. 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.

• Overall Survival
  Time frame: 4.5 years
  The overall failure rate will be estimated along with 95% confidence intervals. A median time of survival will be estimated using standard methods.

Sponsor's own description

This phase II trial is studying how well gossypol works in treating patients with progressive or recurrent glioblastoma multiforme. Gossypol may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Publications & conference data

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

1. Glioblastoma multiforme (GBM): An overview of current therapies and mechanisms of resistance.
   Wu W, Klockow JL, Zhang M, Lafortune F, et al · · 2021 · cited 532× · PMID 34302977 · DOI 10.1016/j.phrs.2021.105780
2. The Regulation and Function of Lactate Dehydrogenase A: Therapeutic Potential in Brain Tumor.
   Valvona CJ, Fillmore HL, Nunn PB, Pilkington GJ. · · 2016 · cited 409× · PMID 26269128 · DOI 10.1111/bpa.12299
3. Altered metabolism in cancer: insights into energy pathways and therapeutic targets.
   Tufail M, Jiang CH, Li N. · · 2024 · cited 265× · PMID 39294640 · DOI 10.1186/s12943-024-02119-3
4. Warburg effect in colorectal cancer: the emerging roles in tumor microenvironment and therapeutic implications.
   Zhong X, He X, Wang Y, Hu Z, et al · · 2022 · cited 223× · PMID 36319992 · DOI 10.1186/s13045-022-01358-5
5. Survival signalling and apoptosis resistance in glioblastomas: opportunities for targeted therapeutics.
   Krakstad C, Chekenya M. · · 2010 · cited 214× · PMID 20515495 · DOI 10.1186/1476-4598-9-135
6. Lactate in the Tumor Microenvironment: An Essential Molecule in Cancer Progression and Treatment.
   Pérez-Tomás R, Pérez-Guillén I. · · 2020 · cited 206× · PMID 33153193 · DOI 10.3390/cancers12113244
7. Lactate and lactylation in cancer.
   Chen J, Huang Z, Chen Y, Tian H, et al · · 2025 · cited 185× · PMID 39934144 · DOI 10.1038/s41392-024-02082-x
8. ROS homeostasis and metabolism: a critical liaison for cancer therapy.
   Kim J, Kim J, Bae JS. · · 2016 · cited 183× · PMID 27811934 · DOI 10.1038/emm.2016.119