Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Ongoing, recruiting
Participants planned
200
Countries
4
Sites
11
Myelodysplastic syndrome
To prevent clinical events (relapse or non-relapse death) in minimal residual disease (MRD) positive subjects
Key facts
- Sponsor
- Karolinska University Hospital
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Hemic and Lymphatic Diseases [C15]
- Trial duration
- 21 Sep 2021 → ongoing
- Decision date (initial)
- 2025-01-07
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- Yes
External identifiers
- EU CT number
- 2024-511651-17-00
- EudraCT number
- 2021-003809-22
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Therapy, Efficacy, Pharmacodynamic, Others, Safety, Dose response
To prevent clinical events (relapse or non-relapse death) in minimal residual disease (MRD) positive subjects
Secondary objectives 6
- MRD positive cohort: To induce molecular remission
- MRD positive cohort: To evaluate the incidence and severity of graft-versus-host disease (GVHD)
- MRD positive cohort: To evaluate safety during pre-emptive treatment
- Whole study cohort: To prevent relapse or non-relapse death
- Whole study cohort: To evaluate the incidence and severity of GVHD
- Whole study cohort: To prolong overall survival
Conditions and MedDRA coding
Myelodysplastic syndrome
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 5
- Signed informed consent
- Age ≥ 18 years
- Subjects eligible for stem cell transplantation
- Subjects having the disease myelodysplastic syndrome (MDS), mixed myelodysplastic/myeloproliferative syndrome or acute myeloid leukemia (AML) with myelodysplasia related dysplasia and 20-29% marrow blasts
- All female subjects of childbearing potential have to have negative pregnancy test within 2 weeks prior to inclusion to the study
Exclusion criteria 3
- No traceable genetic aberration identified either in screening next generation sequencing panel or next generation sequencing panel performed at diagnosis
- Uncontrolled hypertension, heart, liver, kidney related or other uncontrolled medical or psychiatric disorders
- Mental inability, reluctance or language difficulties that results in difficulty understanding the meaning of study participation
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- The percentage of clinical events defined as relapse or non-relapse death at 12 months from verified MRD positivity (NMDSG14B Part 2 compared to Part 1)
Secondary endpoints 9
- MRD positive cohort: Relapse-free survival
- MRD positive cohort: GVHD and relapse-free survival
- MRD positive cohort: Proportion of MRD positive subjects achieving MRD-negativity
- MRD positive cohort: Incidence and severity of GVHD
- MRD positive cohort: Safety in MRD positive subjects subjected to MRD-guided clinical intervention
- Whole study cohort: Relapse-free survival
- Whole study cohort: GVHD and relapse-free survival
- Whole study cohort: Incidence and severity of GVHD
- Whole study cohort: Overall survival
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
SUB05624MIG · Substance
- Active substance
- Azacitidine
- Pharmaceutical form
- POWDER FOR SUSPENSION FOR INJECTION
- Route of administration
- SUBCUTANEOUS
- Max daily dose
- 75 mg/m2 milligram(s)/square meter
- Max total dose
- 75 mg/m2 milligram(s)/square meter
- Max treatment duration
- 630 Day(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Karolinska University Hospital
- Sponsor organisation
- Karolinska University Hospital
- Address
- Halsovagen, Flemingsberg Flemingsberg
- City
- Huddinge
- Postcode
- 141 86
- Country
- Sweden
Scientific contact point
- Organisation
- Karolinska University Hospital
- Contact name
- Magnus Tobiasson
Public contact point
- Organisation
- Karolinska University Hospital
- Contact name
- Magnus Tobiasson
Locations
4 EU/EEA countries · 11 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Denmark | Ongoing, recruiting | 25 | 2 |
| Finland | Ongoing, recruiting | 25 | 1 |
| Norway | Ongoing, recruiting | 50 | 2 |
| Sweden | Ongoing, recruiting | 100 | 6 |
| Rest of world | — | 0 | — |
Investigational sites
Aarhus University Hospital
Clinical Medicine, Palle Juul-Jensens Boulevard 99, 8200, Aarhus N
Rigshospitalet
Hematology, Blegdamsvej 9, 2100, Copenhagen Oe
Helse Bergen HF
Hematology, Haukelandsveien 22, 5021, Bergen
Oslo University Hospital HF
Hematology, Taarnbygget, Kirkeveien 166, Oslo
Sahlgrenska University Hospital-Vaestra Goetalandsregionen
Hematology, Bla Straket 5, 413 46, Goteborg
Region Skane Skanes Universitetssjukhus
Hematology, Entregatan 7, 222 42, Lund
Norrlands University Hospital
Hematology, Daniel Naezens Vag, 907 37, Umea
Uppsala University Hospital
Hematology, Akademiska Sjukhuset, 751 85, Uppsala
Linkoping University Hospital Region Ostergotland
Hematology, Universitetssjukhuset I Linkoping, 581 85, Linkoping
Karolinska University Hospital
Hematology, Halsovagen, Flemingsberg, Huddinge
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 |
|---|---|---|---|---|---|
| Denmark | 2025-06-04 | 2025-06-26 | |||
| Finland | 2021-09-21 | 2025-09-02 | |||
| Norway | 2021-09-21 | 2025-09-02 | |||
| Sweden | 2021-09-21 | 2025-09-02 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 19 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol_2024-511651-17-00_v 4_1_1_20240630_tc | 6.0 |
| Protocol (for publication) | Protocol 2024-511651-17-00 | 6.0 |
| Recruitment arrangements (for publication) | K1_Recruitement arrangements_Denmark_v1_20240516 | 2.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_Finland | 2.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_Norway | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_Sweden | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Denmark_v1_20240322 | 2.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Finland_v4_2024_tc | 4.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Finland_v4_20240823 | 4.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Norway_v4_20240630_tc | 4.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Sweden_v2_2_20250331_tc | 2.2 |
| Subject information and informed consent form (for publication) | SIS and ICF Finland | 3.0 |
| Subject information and informed consent form (for publication) | SIS and ICF Norway | 4.0 |
| Subject information and informed consent form (for publication) | SIS and ICF Sweden | 2.2 |
| Summary of Product Characteristics (SmPC) (for publication) | SmPC_Azacitidin | N/A |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis MS Norwegian_2024-511651-17-00_v4_1_1_20240630 | 6.0 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis MS Norwegian_2024-511651-17-00_v6_0_20260305_tc | 6.0 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis MS Swedish 2024-511651-17-00 | 6.0 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis MS Swedish_2024-511651-17-00_v6_0_20260305_tc | 6.0 |
Application history
5 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-02-13 | Sweden | Acceptable 2024-03-15
|
2024-03-15 |
| 2 | SUBSTANTIAL MODIFICATION | SM-2 | 2024-07-08 | Sweden | Acceptable 2024-08-26
|
2024-08-26 |
| 3 | SUBSEQUENT ADDITION OF MSC | APP-3 | 2024-11-07 | 2025-01-07 | ||
| 4 | SUBSTANTIAL MODIFICATION | SM-4 | 2025-04-08 | Sweden | No conclusion 2025-06-02
|
2025-06-03 |
| 5 | SUBSTANTIAL MODIFICATION | SM-5 | 2026-03-05 | Sweden | Acceptable 2026-05-25
|
2026-05-25 |