NCT00175812

Brief Summary

Hypothesis: Differentiation induction therapy in acute myelogenous leukemia (AML) can be used to achieve disease control and stabilize peripheral blood counts in patients with acute myelogenous leukemia. Adult patients (\<18 years of age) who can be included: Elderly patients (\>60 years of age) with newly diagnosed AML who cannot achieve standard chemotherapy, patients with relapsed or resistant AML. Patients with relapsed or resistant AML who cannot receive intensive chemotherapy. Treatment: Patients will be treated with all-trans retinoic acid (oral administration), valproic acid (7 days intravenous administration and later oral administration)and theophyllamine (7 days intravenous administration and later oral administration). Duration of treatment at least 2 months or until disease progression. Maximal duration of treatment 2 years. Followup: Clinical evaluation, peripheral blood samples, bone marrow samples.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
24

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Nov 2004

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

November 1, 2004

Completed
10 months until next milestone

First Submitted

Initial submission to the registry

September 9, 2005

Completed
6 days until next milestone

First Posted

Study publicly available on registry

September 15, 2005

Completed
2.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 1, 2008

Completed
1.5 years until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2009

Completed
Last Updated

June 24, 2015

Status Verified

June 1, 2015

Enrollment Period

3.5 years

First QC Date

September 9, 2005

Last Update Submit

June 23, 2015

Conditions

Keywords

Acute myelogenous leukemiaDifferentiationAll trans retinoic acidValproic acidTheophyllamin

Outcome Measures

Primary Outcomes (1)

  • Survival

    2008

Secondary Outcomes (3)

  • Disease stabilisation

    2008

  • Disease complications

    2008

  • Side effects of therapy

    2008

Study Arms (1)

ATRA plus valproic acid plus theophyllin

EXPERIMENTAL

ATRA for 14 days, continuous treatment with valproic acid and theophyllin

Drug: all-trans retinoic acid (ATRA)Drug: Valproic acidDrug: Theophyllin

Interventions

All-trans retinoic acid 22.5 mg/square meter twice daily days 1-14

ATRA plus valproic acid plus theophyllin

Valproic acid, highest dose without side effects from day 3 until progression

ATRA plus valproic acid plus theophyllin

Theophyllin, targetted serum level 50-100 from day 3 until progression

ATRA plus valproic acid plus theophyllin

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Recently diagnosed acute myelogenous leukemia (AML)
  • Patients above 60 years of age
  • Patients who cannot receive conventional chemotherapy
  • Patients with relapsed or refractory AML independent of age

You may not qualify if:

  • Chronic myelogenous leukemia in blast phase
  • Intolerance to the study drugs
  • Serious liver disease
  • No informed consent

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Haukeland University Hospital, University of Bergen

Bergen, N-5021, Norway

Location

Related Publications (5)

  • Ryningen A, Stapnes C, Lassalle P, Corbascio M, Gjertsen BT, Bruserud O. A subset of patients with high-risk acute myelogenous leukemia shows improved peripheral blood cell counts when treated with the combination of valproic acid, theophylline and all-trans retinoic acid. Leuk Res. 2009 Jun;33(6):779-87. doi: 10.1016/j.leukres.2008.10.005. Epub 2008 Nov 12.

  • Ryningen A, Stapnes C, Paulsen K, Lassalle P, Gjertsen BT, Bruserud O. In vivo biological effects of ATRA in the treatment of AML. Expert Opin Investig Drugs. 2008 Nov;17(11):1623-33. doi: 10.1517/13543784.17.11.1623.

  • Haaland I, Hjelle SM, Reikvam H, Sulen A, Ryningen A, McCormack E, Bruserud O, Gjertsen BT. p53 Protein Isoform Profiles in AML: Correlation with Distinct Differentiation Stages and Response to Epigenetic Differentiation Therapy. Cells. 2021 Apr 7;10(4):833. doi: 10.3390/cells10040833.

  • Reikvam H, Hovland R, Forthun RB, Erdal S, Gjertsen BT, Fredly H, Bruserud O. Disease-stabilizing treatment based on all-trans retinoic acid and valproic acid in acute myeloid leukemia - identification of responders by gene expression profiling of pretreatment leukemic cells. BMC Cancer. 2017 Sep 6;17(1):630. doi: 10.1186/s12885-017-3620-y.

  • Skavland J, Jorgensen KM, Hadziavdic K, Hovland R, Jonassen I, Bruserud O, Gjertsen BT. Specific cellular signal-transduction responses to in vivo combination therapy with ATRA, valproic acid and theophylline in acute myeloid leukemia. Blood Cancer J. 2011 Feb;1(2):e4. doi: 10.1038/bcj.2011.2. Epub 2011 Feb 11.

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Interventions

TretinoinValproic AcidTheophylline

Condition Hierarchy (Ancestors)

Leukemia, MyeloidLeukemiaNeoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Vitamin ARetinoidsCarotenoidsPolyenesAlkenesHydrocarbons, AcyclicHydrocarbonsOrganic ChemicalsCyclohexenesCyclohexanesCycloparaffinsHydrocarbons, AlicyclicHydrocarbons, CyclicTerpenesDiterpenesPigments, BiologicalBiological FactorsPentanoic AcidsValeratesAcids, AcyclicCarboxylic AcidsFatty Acids, VolatileFatty AcidsLipidsXanthinesAlkaloidsHeterocyclic CompoundsPurinonesPurinesHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-Ring

Study Officials

  • Oystein Bruserud, MD

    University of Bergen

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor

Study Record Dates

First Submitted

September 9, 2005

First Posted

September 15, 2005

Study Start

November 1, 2004

Primary Completion

May 1, 2008

Study Completion

November 1, 2009

Last Updated

June 24, 2015

Record last verified: 2015-06

Locations