A Phase II Open-Label Study of the Safety and Efficacy of Atiprimod Treatment for Patients With Low to Intermediate Grade Neuroendocrine Carcinoma
For carcinoid, despite the many cytotoxic chemotherapy trials that have been conducted, no
regimen has demonstrated a response rate of more than 20% using the criterion of a 50%
reduction of bidimensionally measurable disease. In the more recently reported ECOG phase
III study of chemotherapy in carcinoid tumors (E1281), patients were randomly assigned to
treatment with 5-fluorouracil (5FU) plus doxorubicin or 5FU plus streptozocin. The median
progression free survival durations were disappointing. They were 4.5 months in the 5FU
plus doxorubicin arm and 5.3 months in the 5FU plus streptozocin arm. Overall survival
durations recorded in the trial were also suboptimal at 15 and 24 months respectively.
There is no clear survival benefit for cytotoxic chemotherapy.
This is a phase II, multi-center, open-label study of the safety and efficacy of atiprimod
treatment in patients with low to intermediate grade neuroendocrine carcinoma who have
metastatic or unresectable local-regional cancer and who have either symptoms (diarrhea,
flushing and/or wheezing) despite standard therapy (octreotide) or progression of
neuroendocrine tumor(s) (defined as the appearance of one or more new lesions or a 20%
increase in the sum of the longest diameter of target lesions during the 6 months prior to
enrollment). A maximum of 40 evaluable patients will be enrolled in this study. Atiprimod
will be administered orally as a single daily dose of 120 mg/day for 14 days, followed by a
14-day treatment-free period (i.e., 1 treatment cycle = 28 days).
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Reduction of symptoms (diarrhea, flushing and/or wheezing)
Gary Jacob, Ph.D.
United States: Food and Drug Administration
|Mount Sinai Medical Center||New York, New York 10029|
|Hematology Oncology Services of Arkansas||Little Rock, Arkansas 72205|
|H. Lee Moffitt Cancer Center & Research Institute||Tampa, Florida 33612|
|Dana Farber Cancer Institute||Boston, Massachusetts 02115|
|Lahey Clinic||Burlington, Massachusetts 01805|
|Cedars Sinai Outpatient Cancer Center at the Samuel Oschin Comprehensive Cancer Institute||Los Angeles, California 90048|
|Robert H. Lurie Comprehensive Cancer Center of Northwestern||Chicago, Illinois 60611|
|Scott and White Memorial Hospital, Scott Sherwood and Brindley Facility||Temple, Texas 76508|