A Phase II, Single-Arm, Open-Label Study to Assess the Efficacy and Safety of Canakinumab for the Treatment of Anemia in Patients with IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes or MDS/MPN

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Universitaet Leipzig

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Not possible to specify

Trial duration

23 Mar 2022 → ongoing

Decision date (initial)

2024-07-09

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

External identifiers

EU CT number

2024-515886-33-00

EudraCT number

2020-002246-16

ClinicalTrials.gov

NCT05237713

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

To study the erythroid response rate (HI-E) of canakinumab in patients with IPSS-R very low, low, or intermediate risk MDS or MDS/MPN.

Secondary objectives 4

1. To study the HI-E response duration
2. To study the safety and toxicity of single agent canakinumab
3. To study the proportion of disease progression
4. To assess the impact of canakinumab on quality of life (QoL) over 6 months post screening

Conditions and MedDRA coding

Lower-risk MDS or MDS/MPN

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Cytomorphology based confirmed diagnosis of lower-risk MDS or MDS/MPN including MDS/MPN-RS-T, MDS/MPNu, aCML or CMML (as per the World Health Organization [WHO] 2016 classification)
2. IPSS-R up to 3.5: very low/low/intermediate risk disease (MDS); MDS/MPN < 10% bone marrow blasts; CPSS-Score low/intermediate for CMML
3. Symptomatic anemia (based on complete Hb time course and transfusion history)
4. Relapsed / refractory / intolerant / ineligible (sEPO ≥ 200 U/L) to ESA treatment
5. Age ≥ 18 years
6. Written informed consent

Exclusion criteria 12

1. Known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or gastrointestinal bleeding
2. Prior allogeneic or autologous stem cell transplant
3. Known history of diagnosis of AML
4. Severe neutropenia (ANC  0.5 Gpt/L)
5. Severe thrombocytopenia (PLT <30 Gpt/L)
6. Serum creatinine > 1.5x ULN OR measured or calculated creatinine clearance < 40 ml/min
7. AST or ALT ≥ 3.0 x ULN
8. ECOG > 2
9. Total bilirubin ≥ 2.0 x ULN
10. Active second malignancy
11. Prior or concomitant treatment with anticancer medications or disease modifying agents
12. Previously treated with canakinumab

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The primary clinical endpoint “documented achievement of erythroid response (HI-E)” is based on internationally accepted and validated IWG 2018 response criteria and defined as an increase in hemoglobin levels by at least 1.5 g/dL, persistent for at least 8 weeks over the baseline hemoglobin level (mean over 16 weeks prior to inclusion) for NTD patients or no transfusions within a 8 week-window for LTB and HTB (major response), and will be determined after 8 cycles of canakinumab treatment.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Universitaet Leipzig

Sponsor organisation

Universitaet Leipzig

Address

Ritterstrasse 26, Zentrum Zentrum

City

Leipzig

Postcode

04109

Country

Germany

Scientific contact point

Organisation

Universitaet Leipzig

Contact name

Susanne Melzer

Public contact point

Organisation

Universitaet Leipzig

Contact name

Susanne Melzer

Locations

1 EU/EEA country · 2 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 7 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

4 submissions — initial application plus substantial / non-substantial modifications