Clinical trial NCT00388063: Safety and Efficacy of Atiprimod Treatment for Patients With Low to Intermediate Grade Neuroendocrine Carcinoma
Official title: A Phase II Open-Label Study of the Safety and Efficacy of Atiprimod Treatment for Patients With Low to Intermediate Grade Neuroendocrine Carcinoma
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Neuroendocrine Carcinoma
Keywords: Neuroendocrine
Carcinoma
Atiprimod
Carcinoid
Lead sponsor: Callisto Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2006-10-13
Enrollment: 55
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This study will evaluate 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).

Detailed description: 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).

Eligibility criteria: Inclusion Criteria:

* Patient must have documented histologic proof of low or intermediate grade neuroendocrine carcinoma. Both carcinoid (any site; atypical/intermediate grade carcinoid is allowed) and islet cell (pancreatic endocrine tumor) will be eligible. Patient with neuroendocrine tumors associated with MEN1 syndrome will be eligible.
* Patients must have either metastatic or unresectable local-regional cancer. Patients with brain metastases are allowed on study, but they must have evaluable target lesions elsewhere.
* Patients must have measurable disease, as defined by RECIST.
* Patients must 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).

Primary outcomes:
measure: Reduction of symptoms (diarrhea, flushing and/or wheezing)
timeFrame: 1 year
measure: Progression of neuroendocrine tumor(s)
timeFrame: 1 year
Secondary outcomes:
measure: Adverse events
timeFrame: 1 year
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: Hematology Oncology Services of Arkansas
state: Arkansas
city: Los Angeles
country: United States
facility: Cedars Sinai Outpatient Cancer Center at the Samuel Oschin Comprehensive Cancer Institute
state: California
city: Tampa
country: United States
facility: H. Lee Moffitt Cancer Center & Research Institute
state: Florida
city: Chicago
country: United States
facility: Robert H. Lurie Comprehensive Cancer Center of Northwestern
state: Illinois
city: Boston
country: United States
facility: Dana Farber Cancer Institute
state: Massachusetts
city: Burlington
country: United States
facility: Lahey Clinic
state: Massachusetts
city: New York
country: United States
facility: Mount Sinai Medical Center
state: New York
city: Temple
country: United States
facility: Scott and White Memorial Hospital, Scott Sherwood and Brindley Facility
state: Texas
Study officials:
affiliation: Callisto Pharmaceuticals
name: Gary Jacob, Ph.D.
role: STUDY_DIRECTOR
References: