Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01632904 | Randomized Switch Study From Hydroxyurea to Ruxolitinib for RELIEF of Polycythemia Vera Symptoms: The Relief Study | PHASE3 | COMPLETED | 110 | — | — | Jun 1, 2012 | May 1, 2016 | Nov 14, 2017 | 70 | United States, Belgium +5 |
| NCT01243944 | Study of Efficacy and Safety in Polycythemia Vera Subjects Who Are Resistant to or Intolerant of Hydroxyurea: JAK Inhibitor INC424 (INCB018424) Tablets Versus Best Available Care: (The RESPONSE Trial) | PHASE3 | COMPLETED | 222 | — | — | Oct 27, 2010 | Feb 9, 2018 | Mar 6, 2019 | 102 | United States, Argentina +16 |
Symptoms of polycythemia vera were assessed using a modified Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) electronic diary. Using the diary, patients rated the following symptoms on a scale from 0 (absent) to 10 (worst imaginable): tiredness, itching, muscle aches, night sweats, and sweats while awake. The total symptom score ranged from 0-50 and was calculated as the sum of the 5 symptom scores. A higher score indicates worse symptoms.
Primary response was defined as having achieved hematocrit control (the absence of phlebotomy eligibility beginning at the Week 8 visit and continuing through Week 32) and Spleen Volume Reduction (a greater than or equal to 35% reduction from baseline in spleen volume at Week 32).
| Arm | Type | Description |
|---|---|---|
| ruxolitinib and hydroxyurea (HU)-placebo | EXPERIMENTAL | - |
| HU and ruxolitinib-placebo | ACTIVE_COMPARATOR | - |
| ruxolitinib tablets | EXPERIMENTAL | Starting dose of 10 mg BID with individualized dose titration ranging from 5 mg once a day (QD) to 25 mg BID based on safety and efficacy |
| Best Available Therapy | OTHER | Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements. |
| Name | Type | Description |
|---|---|---|
| Ruxolitinib | DRUG | Ruxolitinib will be orally self-administered at a starting dose of 10 mg (two 5 mg tablets) twice a day. Dose increases of 5 mg (1 tablet) in twice-daily increments are permitted after 4 weeks and again after 8 weeks of therapy for subjects who meet prespecified criteria for inadequate efficacy. |
| Hydroxyurea (HU) | DRUG | Hydroxyurea (500 mg capsules) will be orally self-administered at the dose that the subject was receiving previously. The dose may be increased after 4 weeks and again after 8 weeks of therapy to optimize efficacy for subjects meeting prespecified criteria. |
| HU-placebo | DRUG | All placebo will be self-administered, and dosing will be the same as with the blinded dose. When adjustments are made to the ruxolitinib dose, the dose of HU-placebo will be adjusted concurrently. |
| Ruxolitinib-placebo | DRUG | All placebo will be self-administered, and dosing will be the same as with the blinded dose. When adjustments are made to the HU dose, the dose of ruxolitinib-placebo will be adjusted concurrently. |
| ruxolitinib tablets | DRUG | Starting dose of 10 mg BID with individualized dose titration ranging from 5 mg QD to 25 mg BID based on safety and efficacy |
| Best Available Therapy (BAT) | OTHER | Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements. |
Inclusion Criteria: * Subjects must currently be reporting symptoms while on a stable dose of HU monotherapy and be eligible to continue HU on study after randomization. * Before screening, the subject must have been receiving HU for at least 12 weeks AND be receiving a stable dose. * Subjects must...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Protagonist Therapeutics, Inc. | PTGX | 2 | PHASE3 | Rusfertide, Open-label rusfertide |
| Merck & Co., Inc. | MRK | 1 | PHASE3 | Bomedemstat |
| Ionis Pharmaceuticals, Inc. | IONS | 1 | PHASE2 | sapablursen |
| Disc Medicine, Inc. | IRON | 1 | PHASE2 | DISC-3405 |
| Prelude Therapeutics, Inc. | PRLD | 1 | PHASE1 | PRT12396 |
| Novartis AG Sponsored ADR | NVS | 1 | — | Undisclosed |