Tolerability of trichostatin A for people with relapsed or refractory blood cancers

Tolerability Study of Trichostatin A In Subjects With Relapsed or Refractory Hematologic Malignancies

This trial will test whether trichostatin A is safe and tolerable for adults whose blood cancers have come back or stopped responding to treatment.

Quick facts

• Phase: PHASE1
• Study type: Interventional
• Enrollment: 42 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Vanda Pharmaceuticals (industry)
• Locations: 11 sites (Washington D.C., District of Columbia and 10 other locations)
• Trial ID: NCT03838926 on ClinicalTrials.gov

What this trial studies

This is a Phase 1, open-label, single-agent trial giving trichostatin A to adults with relapsed or refractory hematologic malignancies to characterize safety and tolerability. Participants must have measurable or evaluable disease, an ECOG performance status of 0–2, and have exhausted available standard therapies. Study procedures include regular clinical exams, laboratory monitoring, cardiac monitoring, and disease assessments to track adverse events and any signs of response. The trial is conducted at Vanda investigational sites and will follow participants through dosing and predefined safety follow-up periods.

Who should consider this trial

Good fit: Adults (≥18 years) with relapsed or refractory hematologic malignancies who have measurable disease, ECOG ≤2, have exhausted standard therapies, and are not receiving other anti-cancer treatments are the intended candidates.

Not a fit: Patients with active graft-versus-host disease requiring immunosuppression, uncontrolled medical conditions, recent major surgery, bleeding, significant cardiac impairment, or those receiving other anti-cancer therapies are unlikely to benefit from participation.

Why it matters

Potential benefit: If tolerated and safe, this drug could offer a new treatment option or lead to later trials testing its effectiveness in certain blood cancers.

How similar studies have performed: Other histone deacetylase inhibitors have shown activity and have approvals for some hematologic cancers, but trichostatin A itself is largely preclinical with limited clinical data, making this an early-stage effort.

Eligibility criteria

Inclusion Criteria:

* Subject is ≥ 18 years at the time of signing informed consent;
* Confirmed malignant hematologic disease or lymphoid malignancy that has relapsed or is refractory to standard therapy and has exhausted all available therapies;
* Presence of measurable or evaluable disease;
* Eastern Cooperative Oncology Group (ECOG) performance status of ≤ 2;
* Contraceptives or other approved avoidance of pregnancy measures

Exclusion Criteria:

* Allogeneic stem cell transplant recipient presenting with graft versus host disease (GVHD) either active or requiring immunosuppression;
* Concomitantly taking anti-cancer therapy (bisphosphonates are permitted);
* Undergone major surgery ≤ 2 weeks prior to starting study drug;
* Evidence of mucosal or internal bleeding;
* Impaired cardiac function or conduction defect;
* Concurrent severe and/or uncontrolled medical conditions

Where this trial is running

Washington D.C., District of Columbia and 10 other locations

• Vanda Investigational Site — Washington D.C., District of Columbia, United States (WITHDRAWN)
• Vanda Investigational Site — Lafayette, Indiana, United States (COMPLETED)
• Vanda Investigational Site — Hackensack, New Jersey, United States (WITHDRAWN)
• Vanda Investigational Site — Seattle, Washington, United States (COMPLETED)
• Vanda Investigational Site — Biała Podlaska, Poland (RECRUITING)
• Vanda Investigational Site — Katowice, Poland (RECRUITING)
• Vanda Investigational Site — Krakow, Poland (COMPLETED)
• Vanda Investigational Site — Opole, Poland (RECRUITING)
• Vanda Investigational Site — Skorzewo, Poland (COMPLETED)
• Vanda Investigational Site — Warsaw, Poland (RECRUITING)
• Vanda Investigational Site — Wroclaw, Poland (COMPLETED)

Study contacts

• Study coordinator: Vanda Pharmaceuticals

Conditions: Relapsed or Refractory Hematologic Malignancies, Hematologic, Malignancies, Cancer, Blood