Status:

RECRUITING

A Study to Investigate Treatment of HU and VPA, or 6-MP and VPA in Unfit AML/HR-MDS Patients

Lead Sponsor:

Haukeland University Hospital

Conditions:

Acute Myeloid Leukemia, Adult

Myelodysplastic Syndromes, Adult

Eligibility:

All Genders

18+ years

Phase:

PHASE1

PHASE2

Brief Summary

The purpose of this study is to investigate the safety, tolerability, and preliminary efficacy of the combination treatment of hydroxyurea capsules and valproic acid capsules, or the combination treat...

Detailed Description

This a two-part, open-label phase 1/2 study that will include clinical sites in Norway and other Nordic countries. The study consists of part A and part B. Part A will run in Norway only. Part B will...

Eligibility Criteria

Inclusion

  • Participants are eligible for the study only if all of the following criteria apply:
  • o Female or male, age 18 years or older
  • Written informed consent
  • Patients with Newly diagnosed AML, as defined by ELN 2022 criteria, or relapsed/refractory AML who: - are unfit, defined as HCT-CI ≥ 3, or - in the opinion of the investigator are not candidates for standard therapy or unlikely to tolerate or derive significant clinical benefit from standard therapy, or
  • the patient has declined standard therapy
  • Newly diagnosed HR-MDS, or relapsed/refractory HR-MDS who:
  • are unfit, defined as HCT-CI ≥ 3, or
  • in the opinion of the investigator are not candidates for standard therapy or unlikely to tolerate or derive significant clinical benefit from standard therapy, or
  • has declined standard therapy
  • Secondary AML (MDS-related/ therapy- induced), or
  • Acute promyelocytic leukemia not eligible for standard therapy and/or specific therapy.
  • Adequate renal and hepatic functions unless clearly disease related as indicated by the following laboratory values:
  • Serum creatinine ≤1.5 x ULN;
  • Estimated creatinine clearance ≥ 40 mL/min (Cockcroft-Gault equation);
  • Hepatic function;
  • i. Serum bilirubin ≤ 1.5 x upper limit of normal (ULN); ii. Aspartate aminotransferase (AST)
  • ≤2.5 × ULN
  • ≤5 × ULN for patients with liver metastases
  • iii. Alanine aminotransferase (ALT)
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  • ≤2.5 × ULN
  • ≤5 × ULN for patients with liver metastases
  • iv. Alkaline phosphatase (ALP)
  • 1\. ≤2.5 × ULN
  • European Cooperative Oncology Group (ECOG) performance status 0, 1, 2 or 3
  • Female patients of childbearing potential must have a negative serum pregnancy test within 3 days prior to taking their first dose of study medication. Male patients and female patients of reproductive potential must agree to practice highly effective methods of contraception (such as hormonal implants, combined oral contraceptives, injectable contraceptives, intrauterine device with hormone spirals, total sexual abstinence, vasectomy) throughout the study and for \>3 months after the last dose of study medication. Female patients are considered NOT of childbearing potential if they have a history of surgical sterility or evidence of post-menopausal status defined as any of the following:
  • Natural menopause with last menses \>1 year ago
  • Radiation induced oophorectomy with last menses \>1 year ago
  • Chemotherapy induced menopause with last menses \>1 year ago

Exclusion

  • Participants are excluded from the study if any of the following criteria apply:
  • Patients on treatment for AML (any anti-leukemic therapy including investigational agents) or treated less than 2 weeks before inclusion.
  • Concurrent history of active malignancy in the past six months prior to diagnosis except for
  • basal and squamous cell carcinoma of the skin
  • in situ carcinoma of the cervix
  • Concurrent severe and/or uncontrolled medical condition (e.g. uncontrolled diabetes, infection, hypertension, pulmonary disease et cetera) at the investigators discretion.
  • Breastfeeding women
  • Cardiac dysfunction as defined by:
  • myocardial infarction within the last 3 months of study entry, or
  • congestive heart failure NYHA class IV or
  • unstable angina, or
  • unstable cardiac arrhythmias
  • SARS-CoV-2 infection \< 7 days or Covid-19-vaccine \< 7 days from study onset
  • Patients with a history of non-compliance to medical regimens or who are considered unreliable with respect to compliance.
  • Patients with any serious concomitant medical condition that could, in the opinion of the investigator, compromise participation in the study.
  • Patients with senile dementia, mental impairment or any other psychiatric disorder that prohibits the patient from understanding and giving informed consent.
  • Current concomitant chemotherapy, radiation therapy, or immunotherapy other than as specified in the protocol.
  • Known hypersensitivity to study medications or its excipients.
  • Any psychological, familial, sociological, and geographical condition potentially hampering compliance with the study protocol and follow-up schedule.

Key Trial Info

Start Date :

May 23 2024

Trial Type :

INTERVENTIONAL

Allocation :

ESTIMATED

End Date :

September 30 2029

Estimated Enrollment :

48 Patients enrolled

Trial Details

Trial ID

NCT06199557

Start Date

May 23 2024

End Date

September 30 2029

Last Update

June 29 2025

Active Locations (1)

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Haukeland University Hospital

Bergen, Bergen, Norway, 5021