Recent Updates
Recently added Catalysts

LBH589

Phase 2

Waldenstrom's Macroglobulinemia | Small molecule | Other |Novartis AG|Last Updated: Jan 28, 2021

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
Premium
Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
Premium
Trial Design
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment39
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00936611LBH589 in Relapsed or Relapsed and Refractory Waldenstrom's MacroglobulinemiaPHASE2 COMPLETED 39 — — Jul 1, 2009Nov 1, 2012Jan 28, 20212 United States
Unlock Drug Trial Details
Study Endpoints
Primary Endpoints
Overall Response Rate
Assessed after 2nd cycle and then every subsequent cycle for 6 cycles. The median number of completed cycles of therapy was 5 (0- 32). As such observed up to ~32 months.

Overall response rate is percentage of participants with complete (CR), very good partial (VGPR), partial (PR), or minimal response (MR) as best response during treatment. CR * Disappearance of monoclonal protein by immunofixation * No histologic evidence of bone marrow involvement * Resolution of any adenopathy/organomegaly (confirmed by CT scan), or signs or symptoms attributable to WM. * Second immunofixation required for confirmation. VGPR -At least 90% reduction of serum monoclonal IgM concentration on protein electrophoresis. PR * At least 50% reduction of serum monoclonal IgM concentration on protein electrophoresis and at least 50% decrease in adenopathy/organomegaly on physical examination or on CT scan. * No new symptoms or signs of active disease. MR * At least 25% but less than 50% reduction of serum monoclonal IgM by protein electrophoresis. * No new symptoms or signs of active disease.

Secondary Endpoints
Median Progression Free Survival
Assessed after 2nd cycle and then every subsequent cycle for 6 cycles and then every 3 months. If taken off treatment at cycle 2 for disease progression, assessed every 3 months. The median (range) follow up from treatment start is 7.7 month (0.9 - 29.7).
Median Time to Progression
Assessed after 2nd cycle and then every subsequent cycle for 6 cycles and then every 3 months. If taken off treatment at cycle 2 for disease progression, assessed every 3 months. The median (range) follow up from treatment start is 7.7 month (0.9 - 29.7).
Median Duration of Response
Assessed after 2nd cycle and then every subsequent cycle for 6 cycles and then every 3 months. If taken off treatment at cycle 2 for disease progression, assessed every 3 months. The median (range) follow up from treatment start is 7.7 month (0.9 - 29.7).
Unlock Study Endpoints
Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
LBH589EXPERIMENTAL30 mg three days a week (Mondays, Wednesdays and Fridays). 1 cycle was 28 days Dose modifications for attributable toxicities allowed for reduction to: * 25 mg, 20 mg three times a week every week * Or 20 mg three times a week every other week. No dose re-escalation was allowed. * The protocol was amended on 6/15/2010 because of concerns of toxicity to allow a starting dose of 25 mg; 12/36 (33%) patients were enrolled on the 25 mg dose.
Interventions
NameTypeDescription
LBH589DRUG -
Unlock Study Design Details
Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites2

Inclusion Criteria: * Male or female patients aged 18 years or older * Must have received prior therapy for their WM, any number of prior therapies is allowed * Must have symptomatic relapsed or refractory WM * Measurable monoclonal IgM protein in the blood and presence of lymphoplasmacytic cells i...

Countries:United States
Unlock Eligibility Criteria