Safety and Efficacy of Atiprimod Treatment for Patients With Low to Intermediate Grade Neuroendocrine Carcinoma

Sponsor

Callisto Pharmaceuticals (Industry)

Overall Status

Completed

CT.gov ID

NCT00388063

Collaborator

(none)

Enrollment

Locations

Duration (Months)

6.9

Patients Per Site

0.6

Patients Per Site Per Month

Study Details

Study Description

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).

Condition or Disease	Intervention/Treatment	Phase
Neuroendocrine Carcinoma	Drug: Atiprimod	Phase 2

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).

Study Design

Study Type:

Interventional

Actual Enrollment :

55 participants

Allocation:

Non-Randomized

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

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 Start Date :

Oct 1, 2006

Actual Study Completion Date :

Sep 1, 2007

Outcome Measures

Primary Outcome Measures

Reduction of symptoms (diarrhea, flushing and/or wheezing) [1 year]
Progression of neuroendocrine tumor(s) [1 year]

Secondary Outcome Measures

Adverse events [1 year]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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).

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Hematology Oncology Services of Arkansas	Little Rock	Arkansas	United States	72205
2	Cedars Sinai Outpatient Cancer Center at the Samuel Oschin Comprehensive Cancer Institute	Los Angeles	California	United States	90048
3	H. Lee Moffitt Cancer Center & Research Institute	Tampa	Florida	United States	33612
4	Robert H. Lurie Comprehensive Cancer Center of Northwestern	Chicago	Illinois	United States	60611
5	Dana Farber Cancer Institute	Boston	Massachusetts	United States	02115
6	Lahey Clinic	Burlington	Massachusetts	United States	01805
7	Mount Sinai Medical Center	New York	New York	United States	10029
8	Scott and White Memorial Hospital, Scott Sherwood and Brindley Facility	Temple	Texas	United States	76508