Phase III Study Investigating the Efficacy and Safety of Ruxolitinib in Early Myelofibrosis Patients With High Molecular Risk Mutations. (ReTHINK)

Novartis

Status and phase: Terminated; Phase 3

Conditions: Myelofibrosis With High Molecular Risk Mutations

Treatments: Drug: Ruxolitinib; Drug: Ruxolitinib Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT02598297

CINC424A2353

2014-004928-21 (EudraCT Number)

Details and patient eligibility

About

Myelofibrosis patients with high molecular risk mutations have an intrinsically aggressive disease with increased risk of leukemic transformation and reduced overall survival. As there are no therapies currently established in the subset of high molecular risk patients with early myelofibrosis, the study aimed to evaluate ruxolitinib in this patient population.

Enrollment: 49 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Confirmed diagnosis of MF with bone marrow fibrosis of at least Grade 1; irrespective of JAK2 mutational status
• Patients with at least one mutation in one of the five HMR genes (ASXL1, EZH2, SRSF2 and IDH1/2)
• Patients with non-palpable spleen or spleen palpable ≤ 5 cm from the left costal margin to the point of greatest splenic protrusion
• Patients with MF-7 score of ≤ 15, with each individual symptom score of ≤ 3

Exclusion criteria

• Patients with prior treatment with ruxolitinib or other JAK inhibitors.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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49 participants in 2 patient groups, including a placebo group

Ruxolitinib

Active Comparator group

Description:

Two tablets of ruxolitinib 5 mg were administered orally twice per day.

Treatment:

Drug: Ruxolitinib

Ruxolitinib Placebo

Placebo Comparator group

Description:

Two tablets of 5mg placebo were administered orally twice per day.

Treatment:

Drug: Ruxolitinib Placebo