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Ruxolitinib

Phase 3

Myelofibrosis (MF) | Small molecule | Hematology |AbbVie Inc.|Last Updated: Mar 23, 2026

Target and mechanism

Molecular targetJAK1, TYK2, JAK2, JAK3
Target classInhibitor
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindCONTROLLEDDMC
Total Trials2
Total Enrollment443

FDA Designations

No designations recorded

Clinical trial landscape

Ruxolitinib · 2 trials · 1 indication

Phase 3 1Phase 2 1
NCT04472598Study of Oral Navitoclax Tablet In Combination With Oral Ruxolitinib Tablet When Compared With Oral Ruxolitinib Tablet To Assess Change In Spleen Volume In Adult Participants With MyelofibrosisMyelofibrosis (MF)
COMPLETED252 Analytics
PHASE3COMPLETED
Study of Oral Navitoclax Tablet In Combination With Oral Ruxolitinib Tablet When Compared With Oral Ruxolitinib Tablet To Assess Change In Spleen Volume In Adult Participants With Myelofibrosis
Myelofibrosis (MF)Unlock trial analytics

Study Endpoints

Primary Endpoints

Percentage of Participants With ≥ 35% Reduction From Baseline in Spleen Volume at Week 24 (SVR35W24)
Baseline, Week 24

Reduction in spleen volume is measured by magnetic resonance imaging (MRI) or computed tomography (CT), per International Working Group (IWG) criteria.

Percentage of Participants With ≥ 35% Reduction From Baseline in Spleen Volume at Week 24
Baseline, Week 24

Reduction in spleen volume is measured by magnetic resonance imaging/computerized tomography (MRI/CT).

Secondary Endpoints

Change From Baseline in Total Symptom Score (TSS) at Week 24 as Measured by Myelofibrosis Symptom Assessment Form (MFSAF) v4.0
Baseline, Week 24
Percentage of Participants With ≥ 35% Reduction From Baseline in Spleen Volume (SVR35) at Any Time
Up to Week 97
Duration of 35% Spleen Volume Reduction (SVR35)
Baseline (Week 0) Up to Month 48
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Placebo for Navitoclax + RuxolitinibACTIVE_COMPARATORPlacebo for navitoclax tablets were administered orally once daily (QD) per Baseline platelet count (\>150 × 10\^9/L starting dose of 200 mg; ≤150 × 10\^9/L starting dose of 100 mg, which could be increased to 200 mg QD after 7 days provided platelet count is ≥75 × 10\^9/L). Placebo for navitoclax didn't exceed 200 mg QD for first 24 weeks of treatment. After Week 25, Day 1 visit, placebo for navitoclax dose may be increased to 300 mg QD at Investigator's discretion for those with suboptimal spleen response defined as failure to achieve spleen volume reduction of at least 10% per imaging. Ruxolitinib tablets were administered orally twice daily (BID) per Baseline platelet count (\>200 × 10\^9/L starting dose of 20 mg; 100-200 × 10\^9/L starting dose of 15 mg). Participants will continue their treatment until end of clinical benefit, unacceptable toxicity, or they meet other protocol criteria for discontinuation (whichever occurs first).
Navitoclax + RuxolitinibEXPERIMENTALNavitoclax tablets were administered orally once daily (QD) per Baseline platelet count (\>150 × 10\^9/L starting dose of 200 mg; ≤150 × 10\^9/L starting dose of 100 mg, which could be increased to 200 mg QD after 7 days provided platelet count is ≥75 × 10\^9/L). Navitoclax didn't exceed 200 mg QD for first 24 weeks of treatment. After Week 25, Day 1 visit, navitoclax dose may be increased to 300 mg QD at Investigator's discretion for those with suboptimal spleen response defined as failure to achieve spleen volume reduction of at least 10% per imaging. Ruxolitinib tablets were administered orally twice daily (BID) per Baseline platelet count (\>200 × 10\^9/L starting dose of 20 mg; 100-200 × 10\^9/L starting dose of 15 mg). Participants will continue their treatment until end of clinical benefit, unacceptable toxicity, or they meet other protocol criteria for discontinuation (whichever occurs first).
Navitoclax + ruxolitinib (Cohort 1a)EXPERIMENTALParticipants must have received ruxolitinib for at least 12 weeks and on stable dose of ≥10 mg tablets orally twice daily (BID) for ≥8 weeks prior to the 1st dose of navitoclax. Navitoclax tablets are administered once daily (QD) at a starting dose of 50 mg. This was increased after approximately ≥7 days to next dose level if platelet count is ≥75 × 10\^9/L up to a maximum dose of navitoclax 300 mg QD. Participants continued their treatment until the end of clinical benefit, unacceptable toxicity, or they met other protocol criteria for discontinuation (whichever occurred first).
Navitoclax + ruxolitinib (Cohort 1b)EXPERIMENTALThose receiving ruxolitinib at Screening must be on a stable dose ≥10 mg tablets orally twice daily (BID) for ≥ 4 weeks prior to 1st dose of navitoclax. Those not receiving ruxolitinib at Screening received 10 mg ruxolitinib BID starting on Day 1. Navitoclax tablets were administered orally once daily (QD) per Baseline platelet count (\>150 × 10\^9/L starting dose of 200 mg; ≤150 × 10\^9/L starting dose of 100 mg, which could be increased to 200 mg QD after 7 days provided platelet count is ≥75 × 10\^9/L). Navitoclax didn't exceed 200 mg QD for first 24 weeks of treatment. After Week 24 disease assessment, navitoclax dose was increased to 300 mg QD at discretion of the Investigator for those with suboptimal spleen response defined as failure to achieve spleen volume reduction of at least 10% per imaging. Participants continued their treatment until the end of clinical benefit, unacceptable toxicity, or they met other protocol criteria for discontinuation (whichever occurred first).
Navitoclax (Cohort 2)EXPERIMENTALParticipants must have received prior treatment with a Janus Kinase 2 (JAK-2) inhibitor. Those with Baseline platelet count \>150 × 10\^9/L initiated navitoclax film-coated tablets orally once daily (QD) at the starting dose of 200 mg. Those with a Baseline platelet count ≤150 × 10\^9/L initiated navitoclax film-coated tablets orally once daily (QD) at the starting dose of 100 mg, which could be increased to 200 mg once daily after 7 days provided the platelet count is ≥75 × 10\^9/L. Navitoclax didn't exceed 200 mg QD for the first 24 weeks of treatment. After Week 24 disease assessment, navitoclax dose may be increased to 300 mg QD at discretion of the Investigator for those with suboptimal spleen response defined as failure to achieve spleen volume reduction of at least 10% per imaging. Participants continued their treatment until the end of clinical benefit, unacceptable toxicity, or they met other protocol criteria for discontinuation (whichever occurred first).
Navitoclax + ruxolitinib (Cohort 3)EXPERIMENTALPrior treatment with a Janus Kinase 2 (JAK-2) or Bromodomain and Extra-Terminal motif (BET) proteins inhibitor was prohibited. Ruxolitinib tablets administered orally twice daily (BID) based on Baseline platelet count as per the local approved label. Navitoclax tablets were administered orally once daily (QD) per Baseline platelet count (\>150 × 10\^9/L starting dose of 200 mg; ≤150 × 10\^9/L starting dose of 100 mg, which could be increased to 200 mg QD after 7 days provided platelet count is ≥75 × 10\^9/L). Navitoclax didn't exceed 200 mg QD for first 24 weeks of treatment. After Week 24 disease assessment, navitoclax dose may be increased to 300 mg QD at discretion of Investigator for those with suboptimal spleen response defined as failure to achieve spleen volume reduction of at least 10% per imaging. Participants continued their treatment until the end of clinical benefit, unacceptable toxicity, or they met other protocol criteria for discontinuation (whichever occurred first).

Interventions

NameTypeDescription
Placebo for NavitoclaxDRUGFilm-coated tablet; Oral
RuxolitinibDRUGTablet; Oral
NavitoclaxDRUGFilm-coated tablet; Oral
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites194

Inclusion Criteria: * Documented diagnosis of Primary MyeloFibrosis (MF) as defined by World Health Organization (WHO) classification or Secondary MF (post polycythemia vera \[PPV\] - MF or Post Essential Thrombocythemia \[PET\] - MF) . * Must be able to complete the MF Symptom Assessment Form (MFS...

Countries:United StatesAustraliaAustriaBelgiumBulgariaCanadaCroatiaFranceGermanyGreeceIsraelItalyJapanNetherlandsNew ZealandRussiaSerbiaSouth AfricaSouth KoreaSpainSwedenTaiwanTurkey (Türkiye)UkraineUnited KingdomHungaryPuerto Rico
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Frequently asked questions about Ruxolitinib

What is Ruxolitinib used for in Myelofibrosis (MF)?

Ruxolitinib is an investigational small molecule being studied for the treatment of Myelofibrosis (MF), a type of myeloproliferative neoplasm. It is being evaluated in combination with navitoclax in clinical trials to assess its effect on spleen volume and overall tolerability in adult patients with this condition.

What does Ruxolitinib target?

Ruxolitinib targets Janus kinases, specifically JAK1, JAK2, JAK3, and TYK2. As an inhibitor of these enzymes, it is designed to interfere with signaling pathways involved in the development of Myelofibrosis. This mechanism is being explored in clinical trials for the treatment of this hematologic condition.

Who makes Ruxolitinib?

Ruxolitinib is being developed by AbbVie Inc., a biopharmaceutical company traded on the New York Stock Exchange under the ticker ABBV. AbbVie is conducting clinical trials to evaluate the safety and efficacy of Ruxolitinib in combination with navitoclax for patients with Myelofibrosis.

What phase is Ruxolitinib in?

Ruxolitinib is in Phase 3 clinical development for Myelofibrosis. It is being studied in a randomized, double-blind, controlled trial comparing the combination of Ruxolitinib and navitoclax against Ruxolitinib alone. The drug is investigational and has not been approved by regulatory authorities for this indication.

What clinical trials is Ruxolitinib in?

Ruxolitinib has been studied in two completed clinical trials for Myelofibrosis. NCT03222609 was a Phase 2 trial evaluating navitoclax alone or with Ruxolitinib, enrolling 191 participants. NCT04472598 was a Phase 3 trial comparing Ruxolitinib plus navitoclax to Ruxolitinib alone, enrolling 252 participants to assess spleen volume changes.

Is Ruxolitinib the same as navitoclax?

No, Ruxolitinib is not the same as navitoclax. Ruxolitinib is a JAK inhibitor targeting JAK1, JAK2, JAK3, and TYK2, while navitoclax is a separate investigational drug. In clinical trials for Myelofibrosis, Ruxolitinib is being studied in combination with navitoclax, not as an alternative name for it.