Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Ongoing, recruitment ended
Participants planned
30
Countries
1
Sites
24
Patients with very low, low or intermediate risk myelodysplastic syndromes (MDS) presenting with anemia, transfusion independence (NTD) and naive towards ESA treatment
To evaluate the erythroid response (hematologic improvement erythroid, HI-E) rate of luspatercept (LUS) for the treatment of anemia due to very low-, low-, or intermediate-risk MDS in subjects who require no RBC transfusions.
Key facts
- Sponsor
- Universitaet Leipzig
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Immune System Diseases [C20]
- Trial duration
- 13 Sep 2022 → ongoing
- Decision date (initial)
- 2024-08-16
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- No
External identifiers
- EU CT number
- 2024-516438-36-00
- EudraCT number
- 2020-005219-30
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Therapy, Safety
To evaluate the erythroid response (hematologic improvement erythroid, HI-E) rate of luspatercept (LUS) for the treatment of
anemia due to very low-, low-, or intermediate-risk MDS in subjects who require no RBC transfusions.
Secondary objectives 9
- To evaluate HI-E response duration on a time horizon of 18 months after response
- To evaluate time to HI-E
- To evaluate hemoglobin changes from baseline
- To evaluate red blood cell transfusion (RBC) independence in 8 and 12 weeks
- To evaluate neutrophil and platelet (HI-N and HI-P) responses
- To assess time course changes in sEPO levels
- To assess the impact of luspatercept treatment on quality of life (QoL) over 24 weeks
- To evaluate safety and toxicity profile of luspatercept
- Safety endpoints of particular interest are thrombosis und cardiovascular events
Conditions and MedDRA coding
Patients with very low, low or intermediate risk myelodysplastic syndromes (MDS) presenting with anemia, transfusion independence (NTD) and naive towards ESA treatment
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 4
- Diagnosis of MDS according to WHO classification
- Very low-, low-, or intermediate-risk disease with up to 3.5 score points according to revised International Prognostic Scoring System (IPSS-R) classification
- Non-transfusion dependence (NTD) according to IWG 2018
- Symptomatic anemia: mean baseline Hb < 10 g/dL
Exclusion criteria 5
- Secondary MDS
- Known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or gastrointestinal bleeding
- Prior allogeneic or autologous stem cell transplant
- ECOG > 2
- Prior ESA treatment
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- The primary efficacy endpoint is erythroid response (HI-E) according to IWG 2018 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) and will be determined after 24 weeks of LUS treatment.
Secondary endpoints 1
- To evaluate HI-E response duration on a time horizon of 18 months after response
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 2
Reblozyl 75 mg powder for solution for injection
PRD9257437 · Product
- Active substance
- Luspatercept
- Substance synonyms
- RECOMBINANT FUSION PROTEIN CONSISTING OF A MODIFIED FORM OF THE EXTRACELLULAR DOMAIN OF HUMAN ACTIVIN RECEPTOR IIB LINKED TO THE HUMAN IGG1 FC DOMAIN, ACE-536
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS INJECTION
- Max daily dose
- 1.75 mg/kg milligram(s)/kilogram
- Max total dose
- 1.75 mg/kg milligram(s)/kilogram
- Max treatment duration
- 3 Week(s)
- Authorisation status
- Authorised
- ATC code
- B03XA06 — -
- Marketing authorisation
- EU/1/20/1452/002
- MA holder
- BRISTOL-MYERS SQUIBB PHARMA EEIG
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Reblozyl 25 mg powder for solution for injection
PRD9257430 · Product
- Active substance
- Luspatercept
- Substance synonyms
- RECOMBINANT FUSION PROTEIN CONSISTING OF A MODIFIED FORM OF THE EXTRACELLULAR DOMAIN OF HUMAN ACTIVIN RECEPTOR IIB LINKED TO THE HUMAN IGG1 FC DOMAIN, ACE-536
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS INJECTION
- Max daily dose
- 1.75 mg/kg milligram(s)/kilogram
- Max total dose
- 1.75 mg/kg milligram(s)/kilogram
- Max treatment duration
- 3 Week(s)
- Authorisation status
- Authorised
- ATC code
- B03XA06 — -
- Marketing authorisation
- EU/1/20/1452/001
- 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
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 · 24 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Germany | Ongoing, recruitment ended | 30 | 24 |
| Rest of world | — | 0 | — |
Investigational sites
OncoResearch Lerchenfeld GmbH
Praxis, Lerchenfeld 14, Uhlenhorst, Hamburg
Klinikum rechts der Isar der TU Muenchen AöR
III. Medizinische Klinik - Hämatologie und Onkologie, Ismaninger Strasse 22, Au-Haidhausen, Munich
St. Franziskus Hospital Flensburg
Abt. für Hämatologie, Onkologie, Waldstraße 17, 24939, Flensburg
Kliniken Maria Hilf GmbH Moenchengladbach
Klinik für Hämatologie, Onkologie und Gastroenterologie, Viersener Strasse 450, Windberg, Moenchengladbach
Universitaetsklinikum Magdeburg AöR
Zentrum für Innere Medizin, Klinik für Hämatologie/Onkologie, Leipziger Strasse 44, 39120, Magdeburg
Klinikum Kassel GmbH
Klinik für Hämatologie, Onkologie und Immunologie, Moenchebergstrasse 41-43, Fasanenhof, Kassel
Praxis für Hämatologie und Onkologie
Praxis für Hämatologie und Onkologie, Europaallee 5, 66113, Saarbrücken
Klinikum Hochsauerland GmbH
Klinik f. Hämatologie, Onkologie, Palliativmedizin, Stammzelltransplantation, Schederweg 12, 59870, Meschede
Onkologie/Haematologie Rhein Ruhr Dr. med. Steiniger und Schneider Partnerschaft von Aerzten
Praxis für Hämatologie/Onkologie Oberhausen, Bahnhofstrasse 64, Sterkrade Mitte, Oberhausen
VK&K Studien GbR
VK&K Studien GbR, Achdorfer Weg 5,, 84036, Landshut
Universitaetsklinikum Bonn AöR
Med. Klinik III / ZIM, Hämatologie/Onkologie, Venusberg-Campus 1, Venusberg, Bonn
OncoSearch Institut für klinische Studien GbR
Institut für Klin. Studien GbR, Naegelsbachstr. 49 c, 91052, Erlangen
Klinikum Mutterhaus der Borromaeerinnen gGmbH
Abteilung für Innere Medizin I (Onkologie), Feldstrasse 16, Innenstadt, Trier
Onkologie am Raschplatz Dr. Ingo Zander & Dr. Eyck von der Heyde
Praxis, Rundestraße 10, 30161, Hannover
Universitaetsmedizin Greifswald KöR
Klinik & Poliklinik für Innere Medizin C, Ferdinand-Sauerbruch-Strasse, 17489, Greifswald
Carl-Thiem-Klinikum Cottbus gGmbH
2. Med. Klinik, Thiemstrasse 111, Spremberger Vorstadt, Cottbus
Haematologie und Onkologie Muenchen-Pasing MVZ GmbH
Hämatologie und Onkologie, Baeckerstrasse 4, Pasing-Obermenzing, Munich
Universitaetsmedizin Goettingen
Klinik für Hämatologie/Med. Onkologie, Robert-Koch-Strasse 40, Weende, Goettingen
Mannheimer Onkologie Praxis
Onkologie Praxis, Q5, 14-22, Mannheim
Universitaetsklinikum Mannheim GmbH
III. Medizinische Klinik - Hämatologie und Onkologie, Theodor-Kutzer-Ufer 1-3, Wohlgelegen, Mannheim
Universitaetsklinikum Muenster AöR
Innere Medizin A/Hämatologie-Onkologie, Albert-Schweitzer-Campus 1, Sentrup, Muenster
Universitaet Leipzig
Klinik und Poliklinik für Hämatologie, Zelltherapie und Hämostaseologie, Liebigstrasse 22, Zentrum-Suedost, Leipzig
Universitaetsklinikum Tuebingen AöR
Medizinische Klinik II, Hämatologie/Onkologie, Otfried-Mueller-Strasse 10, Nordstadt, Tuebingen
Zentrum Fuer Innere Medizin
Klinik für Innere Medizin III, Hämatologie, Onkologie, Palliativmedizin, Ernst-Heydemann-Strasse 6, Hansaviertel, Rostock
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Germany | 2022-09-13 | 2022-11-02 | 2025-12-31 |
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.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | LENNON_trial protocol_p | 5 |
| Recruitment arrangements (for publication) | LENNON_recruitment | 1 |
| Subject information and informed consent form (for publication) | Lennon_informed consent_optional | 1 |
| Subject information and informed consent form (for publication) | LENNON_informed consent_short form | 2 |
| Subject information and informed consent form (for publication) | LENNON_informed consent_short form_new | 3 |
| Subject information and informed consent form (for publication) | LENNON_informed consent_trial_new | 5 |
| Summary of Product Characteristics (SmPC) (for publication) | LENNON_SmPC | 1 |
Application history
4 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-08-05 | Germany | Acceptable 2024-08-09
|
2024-08-16 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2024-12-16 | Germany | Acceptable | 2025-01-09 |
| 3 | SUBSTANTIAL MODIFICATION | SM-3 | 2025-05-06 | Germany | Acceptable 2025-06-23
|
2025-06-27 |
| 4 | SUBSTANTIAL MODIFICATION | SM-4 | 2025-07-04 | Germany | Acceptable | 2025-07-17 |