Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Ended
Participants planned
33
Countries
1
Sites
1
Chronic myelomonocyte leukemia
To evaluate the cost-effectiveness of a precision therapy strategy compared with standard treatment in elderly unfit patients with AML, MDS or CMML-2.
Key facts
- Sponsor
- Akershus University Hospital
- Participant type
- Patients
- Age range
- 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Neoplasms [C04], Health Care [N] - Health Care Economics and Organizations [N03]
- Trial duration
- completed 27 Nov 2025
- Decision date (initial)
- 2024-10-18
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- No
External identifiers
- EU CT number
- 2023-509092-16-00
- EudraCT number
- 2020-005023-36
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Others, Therapy
To evaluate the cost-effectiveness of a precision therapy strategy compared with standard treatment in elderly unfit patients with AML, MDS or CMML-2.
Conditions and MedDRA coding
Chronic myelomonocyte leukemia
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 8
- Patients with: a. a newly or recurrent/refractory diagnosis of AML and related precursor neoplasms according to WHO 2016 classification (excluding acute promyelocytic leukemia) including secondary AML (after an antecedent hematological disease and therapy-related AML ), or b. acute leukemias of ambiguous lineage according to WHO 2016 or c. a newly or recurrent diagnosis of myelodysplastic syndrome (MDS) with IPSS-R > 4.5.1 or d. a newly or recurrent diagnosis of chronic myelomonocytic leukemia with 10-19% blasts or blast equivalents in the bone-marrow (CMML-2).
- Patients 60 years and older.
- Patients must NOT be eligible for intensive chemotherapy or allogeneic stem cell therapy
- WBC ≤ 30 x109/L (prior hydroxyurea allowed for a maximum of 14 days, stopped before start of treatment)
- Adequate renal and hepatic functions unless clearly disease related as indicated by the following laboratory values: a. Serum creatinine ≤ 2.5 mg/dL (≤ 221.7 μmol/L), unless considered AML-related b. Serum bilirubin ≤ 2.5 x upper limit of normal (ULN), unless considered AML-related or due to Gilbert’s syndrome c. Alanine transaminase (ALT) ≤ 2.5 x ULN, unless considered AML-related
- WHO performance status 0, 1 or 2 for subjects ≥ 75 years of age OR 0 to 3 for subjects ≥ 60 to 74 years of age.
- Signed Informed Consent
- Male Subjects Only: Male subjects who are sexually active, must agree, from Study Day 1 through at least 90 days after the last dose of study drug, to practice contraception with condom. Male subjects must agree to refrain from sperm donation from initial study drug administration until at least 90 days after the last dose of study drug. If the patient is randomized to a targeted drug where the SPC recommends contraception more than 90 days after last dose, the SPC recommendation should be followed.
Exclusion criteria 14
- Acute promyelocytic leukemia.
- AML with favourable cytogenetic or genetic changes in patients who are fit for intensive chemotherapy. Favourable genetics are: t(8;21)(q22;q22.1); RUNX1-RUNX1T1; inv(16)(p13.1q22) or t(16;16)(p13.1;q22); CBFB-MYH11; mutated NPM1 without FLT3-ITD or with FLT3-ITDlow†; Biallelic mutated CEBPA.
- Patients who previously were included in the Palm-study.
- Patients where it is not possible to get bone-marrow for NGS, i.e., “dry tap”.
- Blast crisis of chronic myeloid leukemia.
- Concurrent severe and/or uncontrolled medical condition (e.g. uncontrolled diabetes, infection, hypertension, pulmonary disease etc.)
- Cardiac dysfunction as defined by: a. Unstable angina or b. New York Heart Association Class ≥ 2. Class 2 is defined as cardiac disease in which patients are comfortable at rest but ordinary physical activity results in fatigue, palpitations, dyspnea, or angina pain or c. Unstable cardiac arrhythmias
- Patients with a history of non-compliance to medical regimens or who are considered unreliable with respect to compliance with the study protocol and follow-up schedule.
- Patients who have senile dementia, mental impairment or any other psychiatric disorder that prohibits the patient from understanding and giving informed consent.
- Patients who do not understand the Written Informed Consent (e.g., language problems)
- Current concomitant chemotherapy, radiation therapy, or immunotherapy for other malignancies; other than hydroxyurea.
- Subject is known to be positive for HIV (HIV testing is not required).
- Subject is known to be positive for hepatitis B or C infection with the exception of those with an undetectable viral load within 3 months. (Hepatitis B or C testing is not required). Subjects with serologic evidence of prior vaccination to HBV [ie. HBs Ag-, and anti-HBs+] may participate.
- Subject has a malabsorption syndrome or other condition that precludes enteral route of administration.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Cost-effectiveness of precison therapy, defined by the incremental costs per quality adjusted life-years gained (QALYs) based on measurements of Health-related quality of life (HRQoL) and total costs of treatment and follow-up
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 7
PRD3045785 · Product
- Active substance
- Dabrafenib
- Substance synonyms
- GSK2118436
- Pharmaceutical form
- CAPSULE, HARD
- Route of administration
- ORAL USE
- Max daily dose
- 150 mg milligram(s)
- Max total dose
- 100000 mg milligram(s)
- Max treatment duration
- 1000 Month(s)
- Authorisation status
- Authorised
- ATC code
- L01EC02 — -
- Marketing authorisation
- EU/1/13/865/003
- MA holder
- NOVARTIS EUROPHARM LIMITED
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
PRD7506627 · Product
- Active substance
- Etoposide
- Pharmaceutical form
- CAPSULE, SOFT
- Route of administration
- ORAL
- Max daily dose
- 100 mg/m2 milligram(s)/sq. meter
- Max total dose
- 1000000 mg/m2 milligram(s)/square meter
- Max treatment duration
- 1000 Month(s)
- Authorisation status
- Authorised
- ATC code
- L01CB01 — ETOPOSIDE
- Marketing authorisation
- 7245
- MA holder
- CHEPLAPHARM ARZNEIMITTEL GMBH
- MA country
- Norway
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Hydroxyurea medac 500 mg kapsel, hard
PRD546900 · Product
- Active substance
- Hydroxycarbamide
- Substance synonyms
- HYDROXYUREA
- Pharmaceutical form
- CAPSULE, HARD
- Route of administration
- ORAL
- Max daily dose
- 5 g gram(s)
- Max total dose
- 100000 g gram(s)
- Max treatment duration
- 1000 Month(s)
- Authorisation status
- Authorised
- ATC code
- L01XX05 — HYDROXYCARBAMIDE
- Marketing authorisation
- 00-6008
- MA holder
- MEDAC GESELLSCHAFT FÜR KLINISCHE SPEZIALPRÄPARATE MBH (WEDEL)
- MA country
- Norway
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Atorvastatin Accord 10 mg filmdrasjerte tabletter
PRD5409703 · Product
- Active substance
- Atorvastatin
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL
- Max daily dose
- 80 mg milligram(s)
- Max total dose
- 10000 mg milligram(s)
- Max treatment duration
- 1000 Month(s)
- Authorisation status
- Authorised
- ATC code
- C10AA05 — ATORVASTATIN
- Marketing authorisation
- 16-11026
- MA holder
- ACCORD HEALTHCARE B.V.
- MA country
- Norway
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Nexavar 200 mg film-coated tablets
PRD3117113 · Product
- Active substance
- Sorafenib
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL
- Max daily dose
- 800 mg milligram(s)
- Max total dose
- 100000 mg milligram(s)
- Max treatment duration
- 1000 Month(s)
- Authorisation status
- Authorised
- ATC code
- L01EX02 — -
- Marketing authorisation
- EU/1/06/342/001
- MA holder
- BAYER AG
- MA country
- Iceland
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Mekinist 2 mg film-coated tablets
PRD3045799 · Product
- Active substance
- Trametinib
- Substance synonyms
- GSK1120212B
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL
- Max daily dose
- 2 mg milligram(s)
- Max total dose
- 100000 mg milligram(s)
- Max treatment duration
- 1000 Month(s)
- Authorisation status
- Authorised
- ATC code
- L01EE01 — -
- Marketing authorisation
- EU/1/14/931/005
- MA holder
- NOVARTIS EUROPHARM LIMITED
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Dacogen 50 mg powder for concentrate for solution for infusion.
PRD3349065 · Product
- Active substance
- Decitabine
- Pharmaceutical form
- SOLUTION FOR INFUSION
- Route of administration
- INTRAVENOUS
- Max daily dose
- 20 mg/m2 milligram(s)/square meter
- Max total dose
- 10000 mg/m2 milligram(s)/square meter
- Max treatment duration
- 1000 Month(s)
- Authorisation status
- Authorised
- ATC code
- L01BC08 — -
- Marketing authorisation
- EU/1/12/792/001
- MA holder
- JANSSEN-CILAG INTERNATIONAL NV
- MA country
- Iceland
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Comparator 3
Venclyxto 100 mg film-coated tablets
PRD6353834 · Product
- Active substance
- Venetoclax
- Substance synonyms
- ABT-199, GDC-0199, 4-(4-((2-(4-CHLOROPHENYL)-4,4-DIMETHYLCYCLOHEX-1-EN-1-YL)METHYL)PIPERAZIN-1-YL)-N-((3-NITRO-4-((TETRAHYDRO-2H-PYRAN-4-YLMETHYL)AMINO)PHENYL)SULFONYL)-2-(1H-PYRROLO(2,3-B)PYRIDIN-5-YLOXY)BENZAMIDE
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 400 mg milligram(s)
- Max total dose
- 1000000 mg milligram(s)
- Max treatment duration
- 1000 Month(s)
- Authorisation status
- Authorised
- ATC code
- L01XX52 — -
- Marketing authorisation
- EU/1/16/1138/005
- MA holder
- ABBVIE DEUTSCHLAND GMBH & CO. KG
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Venclyxto 50 mg film-coated tablets
PRD6353826 · Product
- Active substance
- Venetoclax
- Substance synonyms
- ABT-199, GDC-0199, 4-(4-((2-(4-CHLOROPHENYL)-4,4-DIMETHYLCYCLOHEX-1-EN-1-YL)METHYL)PIPERAZIN-1-YL)-N-((3-NITRO-4-((TETRAHYDRO-2H-PYRAN-4-YLMETHYL)AMINO)PHENYL)SULFONYL)-2-(1H-PYRROLO(2,3-B)PYRIDIN-5-YLOXY)BENZAMIDE
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 400 mg milligram(s)
- Max total dose
- 100000 mg milligram(s)
- Max treatment duration
- 1000 Month(s)
- Authorisation status
- Authorised
- ATC code
- L01XX52 — -
- Marketing authorisation
- EU/1/16/1138/003
- MA holder
- ABBVIE DEUTSCHLAND GMBH & CO. KG
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Vidaza 25 mg/ml powder for suspension for injection
PRD9244549 · Product
- Active substance
- Azacitidine
- Pharmaceutical form
- SUSPENSION FOR INJECTION
- Route of administration
- SUBCUTANEOUS
- Max daily dose
- 100 mg/m2 milligram(s)/square meter
- Max total dose
- 10000 mg/m2 milligram(s)/square meter
- Max treatment duration
- 1000 Month(s)
- Authorisation status
- Authorised
- ATC code
- L01BC07 — -
- Marketing authorisation
- EU/1/08/488/001
- MA holder
- BRISTOL-MYERS SQUIBB PHARMA EEIG
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Akershus University Hospital
- Sponsor organisation
- Akershus University Hospital
- Address
- Sykehusveien 25
- City
- Loerenskog
- Postcode
- 1474
- Country
- Norway
Scientific contact point
- Organisation
- Akershus University Hospital
- Contact name
- Anders Erik Astrup Dahm
Public contact point
- Organisation
- Akershus University Hospital
- Contact name
- Anders Erik Astrup Dahm
Locations
1 EU/EEA country · 1 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Norway | Ended | 33 | 1 |
| Rest of world | — | 0 | — |
Investigational sites
Akershus University Hospital
Haematology, Sykehusveien 25, 1474, Loerenskog
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 12 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_protocol 2023-509092-16-00 | 3.7 |
| Recruitment arrangements (for publication) | wi-lm-3-06-02-vedl-02-template-for-documents-no-longer-subject-to-publication-rules | 1 |
| Subject information and informed consent form (for publication) | Samtykke Palm v 6 240122 endringer godtatt | 6 |
| Summary of Product Characteristics (SmPC) (for publication) | G2_SmPC atorvastatin | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | G2_SmPC azacitidine | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | G2_SmPC dacogen | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | G2_SmPC hydroxyurea | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | G2_SmPC mekinist | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | G2_SmPC nexavar | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | G2_SmPC tafinlar | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | G2_SmPC venclyxto | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | G2_SmPC vepesid | 1 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-09-24 | Norway | Acceptable 2024-10-13
|
2024-10-18 |