A Phase 1, Open-Label, Dose Escalation, Safety, Pharmacokinetic, and Pharmacodynamic Study of Intravenously Administered CX-5461 in Patients with Advanced Haematologic Malignancies

Phase 1

• Conditions: Any advanced Haematologic Malignancy; Cancer - Myeloma; Cancer - Lymphoma (non Hodgkin's lymphoma) - High grade lymphoma; Cancer - Leukaemia - Acute leukaemia; Cancer - Leukaemia - Chronic leukaemia

• Registration Number: ACTRN12613001061729
• Lead Sponsor: Peter MacCallum Cancer Centre

Brief Summary

Seventeen patients with advanced blood cancers were treated with CX-5461 with escalating doses on this first-in-human study and a maximum tolerated dose of 170 mg/ sqm was defined. The most common side-effect observed was photosensitivity in about half the patients. One patient with T-cell lymphoma had a good disease response and received treatment for more than 1 year. The second phase of the study was terminated early after 3 patients due to inadequate recruitment, with no new safety or efficacy findings noted.

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2013-09-23
• Study Completion: 2023-06-16
• Last Update Posted: 6 November 2023

Recruitment & Eligibility

• Status: Stopped early
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

Patients with any histologically confirmed relapsed or refractory advanced haematologic malignancies for which no effective standard therapies are available. These patients must have progressed following at least one prior treatment regimen. There must be evidence of measurable disease.

Exclusion Criteria

Pregnant or lactating women, uncontrolled intercurrent illness, history of other malignancy within 2 years of study entry (excluding treated nonmelanotic skin cancer and in-situ carcinoma), known CNS involvement unless previously treated and well controlled for a period of >=3 months and does not require the use of steroids.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
To determine the maximum tolerated dose (MTD) and dose-limiting toxicities (DLTs) of intravenously administered CX-5461 when used intravenously in haematologic cancers. DLTs will be assessed by physical exam and clinical laboratory blood tests.[ Dose limiting toxicities will be assessed during the first cycle only, on Days 1, 2, 3, 4, 8, 15 and at the end of the 3 week cycle.<br>]