SOM230 Ectopic ACTH-producing Tumors

NCT02780882 | Withdrawn Phase 2 DMC

• 1 other identifier: Pro34493
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this prospective open-label phase II study, is to evaluate the efficacy of pasireotide twice daily subcutaneous injections for normalizing 24 hour urine free cortisol in patients with ectopic ACTH-producing tumors as measured by the proportion of patients achieving normal UFC at the end of the study period.

Conditions

Ectopic ACTH Syndrome

Keywords

Pasireotide; Ectopic ACTH; SOM230; ectopic ACTH-dependent Cushing's syndrome

Outcome Measures

Primary Outcomes (1)

• Evaluate the efficacy of pasireotide twice daily subcutaneous injections for normalizing 24 hour urine free cortisol in patients with ectopic ACTH-producing tumors

  Effectiveness of pasireotide as measured by 24 hour urine free cortisol

  6 months

Secondary Outcomes (14)

• Number of participants with abnormal laboratory values for urine free cortisol

  6 months

• Number of participants with abnormal laboratory values for serum cortisol levels

  6 months

• Number of participants with abnormal laboratory values for salivary cortisol levels

  6 months

• Number of participants with abnormal laboratory values for Hemoglobin A1C (HbA1C)

  6 months

• Number of participants with abnormal laboratory values for fasting blood glucose

  6 months

Study Arms (1)

Pasireotide

EXPERIMENTAL

Each patient will be treated with pasireotide at an initial dose of 600 μg twice daily for one month. The dose will be further increased to 900 μg twice daily for month 2 and 3. After month 3, patients who continue to meet the inclusion and exclusion criteria will be entered into an additional 3 months of treatment.

Drug: Pasireotide

Interventions

Pasireotide DRUG

Also known as: SOM230

Pasireotide

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Written informed consent obtained prior to any screening procedures
• Male or female patients aged 18 years or greater
• Confirmed non-pituitary ectopic-ACTH secreting tumor
• Well differentiated, and low or intermediate grade (WHO classification G1-2) neuroendocrine tumor
• Tumor size increase \< 10% in 6 months prior to screening on CT or MRI
• Mean 24-hour urinary free cortisol level of at least 1.5 x the upper limit of the normal range, and a morning plasma ACTH level of \> 5 ng/L

You may not qualify if:

• Patients with highly malignant ACTH-secreting tumors, i.e. small-cell lung carcinomas, medullary thyroid carcinomas, and pheochromocytomas
• Patients with poorly differentiated neuroendocrine tumors (WHO classification G3)
• Patients with \>10% increase of tumor size in 6 months prior to screening by CT or MRI
• Patients with Cushing's syndrome due to pituitary ACTH secretion
• Patients with hypercortisolism secondary to adrenal tumors or nodular (primary) bilateral adrenal hyperplasia
• Patients who have a known inherited syndrome as the cause for hormone over secretion (i.e. Carney Complex, McCune-Albright syndrome, MEN-1)
• Patients with a diagnosis of glucocorticoid-remedial aldosteronism (GRA)
• Patients who have undergone major surgery within 1 month prior to screening
• Patients with known gallbladder or bile duct disease, acute or chronic pancreatitis (patients with asymptomatic cholelithiasis and asymptomatic bile duct dilation can be included)
• Diabetic patients whose blood glucose is poorly controlled as evidenced by HbA1C \>8%
• Patients who have clinically significant impairment in cardiovascular function or are at risk thereof, as evidenced by
• Congestive heart failure (NYHA Class III or IV), unstable angina, sustained ventricular tachycardia, clinically significant bradycardia, high grade AV block, history of acute MI less than one year prior to study entry
• QTcF \>450 msec at screening
• History of syncope or family history of idiopathic sudden death
• Risk factors for Torsades de Pointes such as uncorrected hypokalemia, uncorrected hypomagnesemia, cardiac failure

Sponsors & Collaborators

• Cedars-Sinai Medical Center: lead
• Novartis: collaborator

Study Sites (1)

Cedars-Sinai Medical Center

Los Angeles, California, 90048, United States

Location

MeSH Terms

Conditions

ACTH Syndrome, Ectopic

Interventions

pasireotide

Condition Hierarchy (Ancestors)

Paraneoplastic Endocrine Syndromes; Paraneoplastic Syndromes; Neoplasms

Study Officials

• Ning-Ai Liu, MD, PhD

  Cedars-Sinai Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD

Study Record Dates

• First Submitted: April 26, 2016
• First Posted: May 24, 2016
• Study Start: December 1, 2015
• Primary Completion: December 1, 2018
• Study Completion: June 1, 2019
• Last Updated: January 26, 2018

Record last verified: 2018-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)