18 and older, any sex, with Polycythemia Vera, Post-Polycythemic Myelofibrosis Phase or Primary Myelofibrosis. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Number of Participants With a ResponsePrimary· After 3 courses of Therapy
Will be defined as complete remission (CR), partial remission (PR), or clinical improvement (CI) after 3 courses of treatment according to International Working Group (IWG) consensus criteria for myelofibrosis. Complete remission (CR): bone marrow blasts \<5%, hemoglobin \>/= 10, absolute neutrophil count (ANC) \>/= 1000, platelets \>/= 100, \<2% immature myeloid cell, spleen and liver not palpable. Partial Response (PR): CR plus one or more of the following: ANC \>/= 1000, decreased platelets by 50%, hemoglobin \>/= 8.5 but \< 10, \<2% immature myeloid cells. Clinical improvement (CI): hemogl
Group
Value
95% CI
Treatment (SMAC Mimetic LCL161)
15
Duration of ResponseSecondary· Up to 7 years 5 Months
duration of response is defined as the date at which the subject's objective status is first noted to be a CR or PR to the date progression is documented (if one has occurred) or to the date of last follow-up (for those subjects who have not progressed).
Group
Value
95% CI
Treatment (SMAC Mimetic LCL161)
31.5
3.6 – 59.2
Time to ResponseSecondary· Up to 7 years 5 months
The time to response is defined as the time from study registration to the first date at which the subject's objective status was classified as a response (CR or PR). In subjects who do not achieve a response, time to response will be censored at the subject's last evaluation date. The distribution for each of these event-time variables (duration of response and time to response) will be estimated by Kaplan-Meier curves.
Group
Value
95% CI
Treatment (SMAC Mimetic LCL161)
1.4
0.9 – 9.1
Overall SurvivalSecondary· Up to 7 years 5 months
Time from date of treatment start until date of death due to any cause or last Follow-up.
Group
Value
95% CI
Treatment (SMAC Mimetic LCL161)
34
2.2 – 60.1
Adverse events — posted to ClinicalTrials.gov
Time frame: Up to 7 years 5 months.
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Treatment (SMAC Mimetic LCL161)
Serious: 33/50 (66%)
Deaths: 2/50
Serious adverse events (37 terms)
Reaction
System
Treatment (SMAC Mimetic LC…
Lung infection
Infections and infestations
—
Anemia
Blood and lymphatic system disorders
—
Fever
General disorders
—
Blood and lymphatic system disorders
Blood and lymphatic system disorders
—
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
This phase II trial studies how well second mitochondrial-derived activator of caspases (SMAC) mimetic LCL161 (LCL161) works in treating patients with primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocytosis myelofibrosis. SMAC mimetic LCL161 may help control the growth of abnormal cells by promoting apoptosis (programmed cell death).
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by M.D. Anderson Cancer Center
Last refreshed: 6 June 2023
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02098161.