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SMAC Mimetic

Smac Mimetic LCL161 for Polycythemia Vera

Phase 2
Waitlist Available
Led By Naveen Pemmaraju
Research Sponsored by M.D. Anderson Cancer Center
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase [SGOT]) and alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) =< 2.5 x ULN, except for patients with MF involvement of the liver who must have AST and ALT =< 5 x ULN
Serum creatinine =< 1.5 x ULN
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 7 years 5 months
Awards & highlights

Study Summary

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).

Eligible Conditions
  • Polycythemia Vera
  • Primary Myelofibrosis
  • Myelofibrosis

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 7 years 5 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 7 years 5 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of Participants With a Response
Secondary outcome measures
Duration of Response
Overall Survival
Time to Response

Side effects data

From 2016 Phase 2 trial • 25 Patients • NCT01955434
96%
Anemia
96%
Fatigue
76%
Nausea
72%
Platelet count decreased
68%
Peripheral sensory neuropathy
68%
Neutrophil count decreased
56%
Vomiting
56%
Lymphocyte count decreased
48%
Pruritus
48%
White blood cell decreased
44%
Diarrhea
44%
Cough
44%
Dyspnea
44%
Constipation
40%
Anorexia
36%
Rash maculo-papular
32%
Fever
28%
Edema limbs
24%
Abdominal pain
16%
Peripheral motor neuropathy
12%
Lymphocyte count increased
12%
Hyperglycemia
8%
Cytokine release syndrome
8%
Syncope
4%
Hypotension
4%
Presyncope
4%
Personality change
4%
Pneumonitis
4%
Dyspepsia
4%
Gastritis
4%
Malaise
4%
Sinusitis
4%
Blood bilirubin increased
4%
Myalgia
4%
Lung infection
4%
Sepsis
4%
Hyperuricemia
4%
Pain in extremity
4%
Urticaria
100%
80%
60%
40%
20%
0%
Study treatment Arm
Treatment (SMAC Mimetic LCL161 and Cyclophosphamide)

Trial Design

1Treatment groups
Experimental Treatment
Group I: Treatment (SMAC mimetic LCL161)Experimental Treatment3 Interventions
Patients receive SMAC mimetic LCL161 PO on days 1, 8, 15, and 22. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Smac Mimetic LCL161
2013
Completed Phase 2
~80

Find a Location

Who is running the clinical trial?

Novartis PharmaceuticalsIndustry Sponsor
2,855 Previous Clinical Trials
4,197,300 Total Patients Enrolled
22 Trials studying Polycythemia Vera
5,563 Patients Enrolled for Polycythemia Vera
National Cancer Institute (NCI)NIH
13,654 Previous Clinical Trials
40,932,273 Total Patients Enrolled
39 Trials studying Polycythemia Vera
4,123 Patients Enrolled for Polycythemia Vera
M.D. Anderson Cancer CenterLead Sponsor
2,966 Previous Clinical Trials
1,804,731 Total Patients Enrolled
10 Trials studying Polycythemia Vera
507 Patients Enrolled for Polycythemia Vera

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
clinicaltrials.govStudy
LCL161 in Treating Patients With Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocytosis Myelofibrosis
2014. "LCL161 in Treating Patients With Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocytosis Myelofibrosis". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02098161.
~5 spots leftby Apr 2025
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