A Study in Leukemia Patients With Karonudib

Study Purpose

The primary objective of this study is to determine safety and tolerability of Karonudib for the treatment of hematological malignancies. Secondary objectives are to determine a recommended RP2D and schedule for further development of Karonudib, to determine the pharmacokinetics of Karonudib, to look for evidence of treatment efficacy. Overall survival will also be recorded.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.

An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.

Searching Both is inclusive of interventional and observational studies.

Eligible Ages 18 Years - 75 Years
Gender All
More Inclusion & Exclusion Criteria

Inclusion Criteria:

1. Written informed consent. 2. Age 18-75 years (may be extended to older if deemed fit). 3. AML, ALL, DLBCL, Burkitt lymphoma, multiple myeloma or high-risk MDS, according to the WHO 2016 criteria. 4. The patient has received standard of care treatments and has refractory or relapsed disease with only experimental therapies as further treatment options. 5. Life expectancy of at least 8 weeks (as per investigators clinical assessment). 6. ECOG PFS 0-2. 7. Patients must have measurable disease by blood or bone marrow or imaging examination. 8. Adequate hepatic and renal function defined as: 1. Total bilirubin < 3 x ULN (does not apply to patients with Gilberts Syndrome). 2. AST and ALT ≤ 5 x ULN. 3. The calculated GFR is at least 30 ml/min using Cockcroft-Gault method. 9. Subject must be able to take oral medication. 10. Negative pregnancy test according to CTFG guidance 2014 for females of child-producing potential.

Exclusion Criteria:

1. Age less than 18 years. 2. Less than 4 weeks since stopping previous systemic chemotherapy treatment with the exception of stable dose Hydroxyurea, Trophosphamide, oral Cyclophosphamide, ImID or Thioguanine which needs to be stopped 10 x t1/2 prior to Karonudib administration. 3. Less than 3 weeks since stopping palliative radiotherapy. 4. Less than 3 weeks after surgery except access surgical procedures. 5. Less than 6 months since a clinically significant cardiovascular event such as myocardial infarction, unstable angina, angioplasty, bypass surgery, stroke or TIA. 6. Congestive heart failure NYHA class >
  • II. 7.
History of arrhythmias or arrhythmias discovered during the screening period (apart from atrial fibrillation without ventricular tachycardia and premature extra beats. 8. Patients requiring anti-arrhythmic drugs except for stable dose beta-blocking or calcium channel blocking agents. 9. QTc interval >470 ms at baseline (Fridericia correction). 10. Use of Fentanyl (must be stopped at least 1 week prior to initiation of Karonudib). 11. Use of anti-oxidants vitamins and Acetylcysteine (must be stopped within 48 hours of starting treatment with Karonudib). 12. Use of antidepressant medications which are substrate for CYP2D6 (must be stopped at least 3 weeks prior to starting treatment with Karonudib). 13. Any severe acute or chronic medical condition that places the patient at increased risk or interferes with the interpretation of study results. 14. Intracerebral engagement (patient with previously known engagement are eligible provided that there is no evidence of disease progression for a minimum of 8 weeks prior to inclusion. 15. Known acute or chronic infection with hepatitis B or C except for DNA-negative hepatitis B with stable dose anti-viral agents. 16. Known HIV infection. 17. Pregnant or breast-feeding women. 18. Patients with reproductive potential not implementing accepted and effective means of contraception. 19. Participation in any other clinical trial with a pharmaceutical product within 10 x t½, or minimum 2 weeks, since last dosing of the IMP. 20. Acute promyelocytic leukemia (AML M3). 21. Uncontrolled ongoing systemic or localized infection. 22. Unable to comply with study procedures. 23. Peripheral neurological toxicity CTCAE grade 2 or higher.

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.


Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Phase 1
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Thomas Helleday Foundation
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

Stefan Deneberg, MD
Principal Investigator Affiliation Karolinska University Hospital
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Overall Status Recruiting
Countries Sweden

The disease, disorder, syndrome, illness, or injury that is being studied.

Arms & Interventions


Experimental: Dose escalation

Karonudib is an oral inhibitor of MTH1 and will be supplied as an oral solution to be taken every other day. There are three planned dose cohorts. Patients will be given every second day dosing.


Drug: - Karonudib

Dose escalation of administration with Karonudib. Three different dose cohorts are planned.

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

International Sites

Karolinska University Hospital, Huddinge, Sweden




Karolinska University Hospital

Huddinge, ,

Site Contact

Stefan Deneberg, MD, PhD