Clinical trial NCT06976918: Registry Platform Myelofibrosis and Anemia
Official title: Clinical Research Platform on Treatment, Quality of Life and Outcome of Patients With Primary and Secondary Myelofibrosis and Anemia Who Are JAK Inhibitor Treatment-naïve or JAK Inhibitor Treatment-experienced (RHODOLITE)
Study type: OBSERVATIONAL
Overall status: NOT_YET_RECRUITING
Conditions: Primary Myelofibrosis
Secondary Myelofibrosis
Post-polycythemia Vera Myelofibrosis
Post-essential Thrombocythemia Myelofibrosis
Anemia
Myelofibrosis; Anemia
Myelofibrosis
Lead sponsor: iOMEDICO AG
Sponsor class: INDUSTRY
Collaborators: name: German Study Group for Myeloproliferative Neoplasms (GSG-MPN)
First posted: 2025-05-16
Enrollment: 200
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Adult patients with primary myelofibrosis or post-polycythemia vera or post-essential thrombocythemia myelofibrosis (MF) with anemia

Brief summary: The purpose of the project is to set up a national, prospective, longitudinal, multicenter cohort study, a tumor registry platform, to document uniform data on characteristics, molecular diagnostics, treatment and course of disease and to collect patient-reported outcomes for patients with primary and secondary myelofibrosis and anemia in Germany.

Detailed description: RHODOLITE is a national, prospective, open-label, longitudinal, non-interventional multicenter cohort study (tumour registry platform) to describe treatment in routine clinical practice of myelofibrosis patients in routine care in Germany.

The registry will follow patients for up to three years with the aim to identify common therapeutic sequences and changes in the treatment of the disease. At inclusion, data in patient characteristics, comorbidities, tumor characteristics and previous treatments are collected. During the course of observation data on all systemic treatments and outcome are documented.

Health-related quality of life (HRQoL) will be evaluated for up to three years.

The RHODOLITE clinical registry is a joint project with the German Study Group for Myeloproliferative Neoplasms (GSG-MPN) and its GSG-MPN Bioregistry (NCT03125707). Details on treatment and outcome will be collected in RHODOLITE clinical registry for up to three years, while long-term follow-up for a minimum of additional five years will be performed in the GSG-MPN Bioregistry.

Eligibility criteria: Inclusion Criteria:

* Confirmed diagnosis of primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis (MF) (Note: diagnosis according to WHO-2017, ICC-2022 or WHO-2022 or IWG-MRT criteria, respectively).
* Diagnosis of anemia at the time of enrollment as per individual, clinical assessment by the local physician.
* Start of first or subsequent systemic treatment for MF.
* Informed consent and registration for the GSG-MPN Bioregistry.
* Willingness and capability to participate in PRO assessment.
* Signed and dated informed consent form for RHODOLITE at the latest six weeks after start of the respective systemic MF treatment.

Exclusion Criteria:

* No systemic therapy for diagnosed primary or secondary MF.
* Planned allogenic stem cell transplantation (allo-SCT) or active participation in an interventional clinical trial.

Primary outcomes:
measure: Documentation of systemic treatment for MF and therapy sequences
timeFrame: 3 years per patient
Secondary outcomes:
measure: Hematologic response
timeFrame: 3 years per patient
measure: Splenic response
timeFrame: 3 years per patient
measure: Overall response rate (ORR)
timeFrame: 3 years per patient
measure: Progression free survival (PFS)
timeFrame: 3 years per patient
measure: Overall survival
timeFrame: 3 years per patient
measure: Health-related quality of life (Patient-reported outcome, PRO)
timeFrame: 3 years per patient
measure: Assessment of Myelofibrosis-related symptoms
timeFrame: 3 years per patient
Other outcomes:
Locations:
city: Multiple Locations
country: Germany
facility: Multiple sites all over Germany
Study officials:
affiliation: University Hospital Ulm, Germany
name: Konstanze Döhner, Prof. Dr. med.
role: STUDY_CHAIR
affiliation: University Hospital Aachen, Germany
name: Steffen Koschmieder, Prof. Dr. med.
role: STUDY_CHAIR
References: