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Myelodysplastic SyndromesPhase 2LUCASNCT05178342

Treatment of Anemia in Patients With Very Low, Low or Intermediate Risk Myelodysplastic Syndromes With CA-4948

A Phase 2 study testing CA-4948 in people with Myelodysplastic Syndromes and Anemia, run by University of Leipzig at 15 sites in Germany.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

Anemia in LR-MDS patients Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Myelodysplastic Syndromes, Anemia
What is being tested?
CA-4948
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
38 participants
Where
15 sitesGermany
Study dates
1 Jan 2022to 31 Jul 2024
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Diagnosis of de novo myelodysplastic syndrome (MDS) OR de novo myelodysplastic/myeloproliferative neoplasias (MDS/MPN) including MDS/MPN-RS-T, MDS/MPNu, aCML or CMML
  • Very low/low/intermediate risk disease: IPSS-R up to 3.5 for MDS; MDS/MPN < 10% bone marrow blasts; for CMML low or intermediate risk according to CPSS-Score
  • Symptomatic anemia (based on valid and complete hemoglobin and transfusion history):
  • NTD (non transfusion dependent): < 3 RBC transfusions and mean hemoglobin level <10 g/dl within the last 16 weeks
  • LTB (low transfusion burden): 3-7 RBC transfusions within the last 16 weeks in at least two transfusion episodes, maximum 3 in 8 weeks
  • HTB (high transfusion burden): ≥ 8 RBC transfusions within the last 16 weeks, ≥ 4 in 8 weeks
Show all 10Show fewer
  • Defined transfusion strategy
  • No available option of an approved MDS therapy and classification of prior erythropoiesis-stimulating agent (ESA) treatment as follows:
  • Cohort A: ESA exposed (and refractory or intolerant)
  • Cohort B: ESA naive AND serum erythropoietin level >200 U/L

Who cannot join

  • Compliance with major study procedures
  • Inability to swallow and retain oral medications (> 10 pills)
  • Patient does not accept bone marrow sampling during screening and after the treatment
  • Patient does not accept up to weekly peripheral blood sampling during screening and treatment
  • Safety
  • ECOG performance status ≥ 3
Show all 29Show fewer
  • Inacceptable organ function
  • Serum creatinine > 2 × ULN or calculated creatinine clearance < 30 ml/min
  • AST > 2 × ULN or ALT > 2 × ULN
  • total bilirubin > 2 × ULN (exception >3 × ULN in patients with documented Gilbert's syndrome)
  • Interfering treatments
  • Prior treatment with azacitidine or decitabine
  • Treatment with erythropoiesis stimulating agent (ESA), G-CSF, GM-CSF, lenalidomide, luspatercept and/or another investigational drug or device up to 14 days before registration
  • Treatment with iron chelation therapy 56 days before registration, except for subjects on a stable or decreasing dose for at least eight weeks prior to inclusion and during study treatment
  • Major surgery within 28 days prior to registration
  • Concomitant diseases
  • Known human immunodeficiency virus infection (HIV)
  • Active infectious hepatitis (HBV or HCV)
  • Hepatitis virus detectable within 6 months before registration in patients with a history of hepatitis
  • History of other invasive malignancy, unless definitively treated with curative intent, provided it is deemed to be at low risk for recurrence by the treating physician
  • Presence of an acute or chronic toxicity resulting from prior anti-cancer therapy that has not resolved to Grade ≤ 1 (except anemia and alopecia)
  • Known allergy or hypersensitivity to any component of the formulation of CA-494824
  • Severe cardiovascular disease (e.g. myocardial infarction within 6 months registration, unstable angina within 6 months registration, NYHA Class III or greater congestive heart failure, serious arrhythmias uncontrolled on treatment, clinically significant pericardial disease, known QTc abnormality > 450 msec on ECG
  • Formal requirements
  • Positive serum pregnancy test in women of childbearing potential
  • Women of childbearing potential and men who partner with a woman of childbearing potential unwilling to use highly effective contraceptive methods for the duration of the study and for 90 days after the last dose of CA-4948
  • Age under 18 years at registration
  • Inability to provide written informed consent
  • Simultaneous participation in another interventional clinical trial or participation in any clinical trial involving administration of an investigational medicinal product within 28 days prior registration

Trial locations and contacts

15 sites in 1 country.

15 sites in 1 country

Germany15 sites

  • Charité Berlin - Campus Benjamin Franklin, Med. Klinik m. S. Hämatologie, Onkologie, Tumorimmunologie

    Berlin, 12200

  • Carl-Thiem-Klinikum Cottbus gGmbH, 2. Med. Klinik

    Cottbus, 03048

  • Gemeinschaftspraxis Dr. Jacobasch Dresden, Hämatologie Onkologie

    Dresden, 01307

  • Marienhospital Düsseldorf, Klinik für Onkologie und Hämatologie, Palliativmedizin

    Düsseldorf, 40479

  • ONCOSEARCH, Institut für Klinische Studien GbR

    Erlangen, 91052

  • InVO-Institut für Versorgungsforschung in der Onkologie

    Koblenz, 56068

  • VK & K Studien GbR, Studienzentrum

    Landshut, 84036

  • University Leipzig, Medizinische Klinik und Poliklinik I - Hämatologie und Zelltherapie, Hämostaseologie

    Leipzig, 04103

  • Universitätsklinikum Schleswig-Holstein, Klinik für Hämatologie und Onkologie Campus Lübeck

    Lübeck, 23538

  • Universitätsklinikum Mainz, III. Medizinische Klinik und Poliklinik - Hämatologie, Internistische Onkologie und Pneumologie

    Mainz, 55131

  • Universitätsklinikum Mannheim, III. Medizinische Klinik - Hämatologie und Onkologie

    Mannheim, 68167

  • Klinikum Hochsauerland GmbH, Klinik f. Hämatologie, Onkologie, Palliativmedizin, Stammzelltransplantation

    Meschede, 59872

  • Klinikum rechts der Isar der TU München III. Medizinische Klinik - Hämatologie und Onkologie

    München, 81675

  • Friedrich-Ebert-Krankenhaus GmbH, Klinik für Hämatologie, Onkologie und Nephrologie

    Neumünster, 24534

  • Rems-Murr-Kliniken gGmbH, Hämatologie, Onkologie und Palliativmedizin

    Winnenden, 71364

Other trials in Germany

About this study

Official title: A Phase II, Open-Label, Multicenter Study of Orally Administered CA-4948 for the Treatment of Anemia in Patients With Very Low, Low or Intermediate Risk Myelodysplastic Syndromes (MDS)

Anemia in LR-MDS patients

Read the detailed description

Anemia in non-transfusion dependent (NTD) or transfusion dependent (low or high transfusion burden, LTB/HTB) patients with very low, low or intermediate risk myelodysplastic syndromes

Being tested
CA-4948
Keywords
MDS, Anemia, Myelodysplastic Syndrome, IRAK4
Medical subject headings
Myelodysplastic Syndromes, Anemia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CA-4948Drug

    Also called Emavusertib

    Patients will be treated orally with CA-4948 at 300 mg BID (2x200mg) over 4 cycles. One cycle consists of 28 days, 21 of which are treatment days, followed by 7 days off.

    Patients with erythroid response (HI-E) after 4 cycles who tolerate CA-4948 may continue to receive CA-4948 until loss of HI-E response.

Study groups in NCT05178342
Study groupRoleWhat they receive
CA-4948 treatmentOtherSingle-arm design. all patients are treated with IMP
Receives: Drug: CA-4948

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Erythroid response (HI-E)

    To evaluate the proportion of patients who have an erythroid response (HI-E) according to the modified IWG 2018 criteria separately for both independent substudies.

    Measured: At the end of cycle 4 (each cycle is 28 days).

Secondary outcomes (9)

  1. HI-E response (erythroid response) duration

    To evaluate HI-E response from the first day of response until loss of response.

    Measured: From the date of treatment start until date of documented loss of response, assessed up to 30 months.

  2. Time to HI-E (erythroid response)

    To evaluate the time between start of treatment and first day of response.

    Measured: From the date of treatment start until first day of response, assessed up to end of cycle 4 (each cycle is 28 days).

  3. Red blood cell (RBC) transfusions

    To evaluate frequency of red blood cell transfusions in transfusion dependent patients

    Measured: From the date of treatment start until the date of end of treatment, assessed up to 30 months.

  4. Neutrophil (HI-N) responses

    Neutrophil (HI-N) responses according to IWG 2018 criteria

    Measured: At the end of cycle 4 (each cycle is 28 days).

  5. Platelet (HI-P) responses

    Platelet (HI-P) responses according to IWG 2018 criteria

    Measured: At the end of cycle 4 (each cycle is 28 days).

  6. Safety of CA-4948 (toxicities and adverse events)

    Assessments will include characterization of toxicities; characterization of AEs including type, incidence, severity, seriousness, and relationship to treatment

    Measured: From the date of treatment start until the end of study, assessed up to 30 months.

  7. Number of participants with clinically significant changes of selected laborotory parameters (parameters listed in detailed description)

    To ensure patient safety, close monitoring is carried and includes the analysis of: transaminases, bilirubin, amylase, lipase, troponin, lactate dehydrogenase, creatine kinase, uric acid, TSH, FT4, urine analysis.

    Measured: From the date of treatment start until the end of study, assessed up to 30 months.

  8. Impact of treatment assessed by using the validated European Organisation for Research and Treatment of Cancer Core Quality of Life questionnaire (EORTC QLQ-C30)

    To assess patient-reported quality of life during CA-4948 treatment: 30 questions assessing the quality of life of oncology patients across 10 subscales will be analyzed. All subscales have a score range from 0 to 100 points.

    Function subscales: a higher score represents a higher quality of life. Symptoms subscales: higher score represents higher level of symptoms/problems, i.e., represents lower quality of life.

    Measured: From the date of treatment start until the end of study, assessed up to 30 months.

  9. Impact of treatment assessed by using the validated European Organisation for Research and Treatment of Cancer cancer related fatigue questionnaire (EORTC QLQ- FA12)

    To assess patient-reported quality of life during CA-4948 treatment: 12 items, with four response categories for each item (coded with values from 1 to 4) will be analyzed. FA12 scores are transformed to the range 0-100: Higher levels indicate greater degrees of fatigue.

    Measured: From the date of treatment start until the end of study, assessed up to 30 months.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted5 Jan 2022
  2. Study start1 Jan 2022
  3. Primary completion31 Jul 2024
  4. Study completion31 Jul 2024

Why it stopped: Prematurely according to trial protocol

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
38 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05178342
Sponsor's protocol ID
LUCAS
Acronym
LUCAS
Status last verified
Aug 2024
First submitted
2 Nov 2021
Last updated
13 Aug 2024
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Platzbecker AS, Zoldan K, Brauer D, Kiewe P, Sasca D, Sockel K, Illmer T, Giagounidis A, Nowak D, Rieger K, Wortmann F, Gotze K, Schmidt-Hieber M, Mayer K, Vehling-Kaiser U, Weide R, Mahlmann S, Przybilla J, Melzer S, Cross M, Schneider M, Hasenclever D, Metzeler KH, Platzbecker U. Canakinumab or emavusertib for anemia in lower-risk MDS: results of the CANFIRE and LUCAS phase 2, open-label, multicenter trials. Commun Med (Lond). 2026 Sep 16;6(1):486. doi: 10.1038/s43856-026-01885-z.

    PubMed 42749863

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing CA-4948 in people with Myelodysplastic Syndromes and Anemia, run by University of Leipzig at 15 sites in Germany.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 10 inclusion and 29 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 13 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 15 sites in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early (reason given: Prematurely according to trial protocol). Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Erythroid response (HI-E) (time frame: At the end of cycle 4 (each cycle is 28 days).). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05178342, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Aug 2024.

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