NCT01246102

AT13387 in Adults With Refractory Solid Tumors

Completed Phase 1 Last updated 5 July 2018

What this trial tests

Phase 1 trial testing AT13387 in Solid Tumors in 31 participants. Completed in 27 October 2017.

Timeline

19 November 2010

Primary endpoint
30 December 2013

27 October 2017

Quick facts

Lead sponsor	National Cancer Institute (NCI)
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	non randomized
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	31
Start date	19 November 2010
Primary completion	30 December 2013
Estimated completion	27 October 2017
Sites	1 location across United States

Drugs / interventions tested

• AT13387 — full drug profile →

Conditions studied

• Solid Tumors — all drugs for Solid Tumors →
• Breast Cancer — all drugs for Breast Cancer →

Sponsor

National Cancer Institute (NCI)

Who can join

Adults 18 to 120, any sex, with Solid Tumors or Breast Cancer. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Background:

\- The experimental drug AT13387 has been shown to have some anticancer effects against tumor cells by blocking a protein that affects other proteins inside certain cancer cells, and helps to prevent the cancer cells from reproducing and spreading. AT13387 has not been tested in humans, and researchers are interested in investigating whether it can be used to treat solid tumors that have not responded to standard treatments.

Objectives:

\- To investigate the safety and effectiveness of AT13387 in individuals with solid tumors.

Eligibility:

\- Individuals at least 18 years of age who have solid tumors that have not responded to standard treatments.

Design:

* Participants will be screened with a physical examination and medical history, as well as blood tests and tumor imaging studies.
* AT13387 will be given in 28-day cycles of treatment. Participants will receive AT13387 twice a week (2 days in a row) for the first 3 weeks of the cycle, followed by a fourth week without the drug.
* Participants will have regular blood and urine samples, imaging studies, eye examinations, and tumor biopsies to monitor the effects of the treatment.
* Participants will continue treatment with AT13387 unless serious side effects develop or the tumor stops responding to treatment.

Publications & conference data

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

1. Targeting Heat Shock Proteins in Cancer: A Promising Therapeutic Approach.
   Chatterjee S, Burns TF. · · 2017 · cited 337× · PMID 28914774 · DOI 10.3390/ijms18091978
2. Heat Shock Proteins: Agents of Cancer Development and Therapeutic Targets in Anti-Cancer Therapy.
   Yun CW, Kim HJ, Lim JH, Lee SH. · · 2019 · cited 204× · PMID 31878360 · DOI 10.3390/cells9010060
3. Heat shock protein 90: biological functions, diseases, and therapeutic targets.
   Wei H, Zhang Y, Jia Y, Chen X, et al · · 2024 · cited 54× · PMID 38283176 · DOI 10.1002/mco2.470
4. Heat shock protein 90 inhibition: rationale and clinical potential.
   Den RB, Lu B. · · 2012 · cited 44× · PMID 22754594 · DOI 10.1177/1758834012445574
5. Phase I study of the heat shock protein 90 (Hsp90) inhibitor onalespib (AT13387) administered on a daily for 2 consecutive days per week dosing schedule in patients with advanced solid tumors.
   Do K, Speranza G, Chang LC, Polley EC, et al · · 2015 · cited 39× · PMID 26082332 · DOI 10.1007/s10637-015-0255-1
6. HSP90 multi-functionality in cancer.
   Albakova Z. · · 2024 · cited 32× · PMID 39148727 · DOI 10.3389/fimmu.2024.1436973
7. Advances in the structures, mechanisms and targeting of molecular chaperones.
   Gu J, He Y, He C, Zhang Q, et al · · 2025 · cited 30× · PMID 40069202 · DOI 10.1038/s41392-025-02166-2
8. Chaperone-assisted E3 ligase CHIP: A double agent in cancer.
   Kumar S, Basu M, Ghosh MK. · · 2022 · cited 29× · PMID 36157498 · DOI 10.1016/j.gendis.2021.08.003