RAD001 in Pheochromocytoma or Nonfunctioning Carcinoid

NCT01152827 | Completed Phase 2 DMC

• 1 other identifier: H-0710-049-223
• Study Type: interventional
• Target: 33
• Locations: 1 country
• Sites: 1

Brief Summary

• According to Martin F et al, AKT is highly phosphorylated in phenochromocytoma but not in benign adrenocortical tumors.
• In nonfunctioning carcinoid, the PI3K/AKT/mTOR pathway is activated.
• Although mTOR is clearly an attractive therapeutic target in tumor, no clinical study on mTOR inhibition by RAD001 have been conducted in pheochromocytoma or extra-adrenal paraganglioma or non-functioning carcinoid.
• So we design this phase II study of RAD001 in pheochromocytoma or extra-adrenal paraganglioma or non-functioning carcinoid to evaluate the efficacy of RAD001 in this orphan disease.

Conditions

Pheochromocytoma; Extra-adrenal Paraganglioma; Non-functioning Carcinoid

Keywords

Pheochromocytoma; extra-adrenal paraganglioma; non-functioning carcinoid; RAD001

Outcome Measures

Primary Outcomes (1)

• progression-free survival rate at 4 months

  proportion of patients who are alive and progression-free at the time of 4 months of treatment among all patients

  10 months

Secondary Outcomes (4)

• time to progression (TTP)

  10 months

• overall survival (OS)

  2 years

• response rate (RR)

  6 months

• metabolic response rate by PET-CT

  2 months

Study Arms (1)

RAD001

EXPERIMENTAL

RAD001 10 mg daily po medication

Drug: RAD001

Interventions

RAD001 DRUG

RAD001 10 mg daily po medication. Treatments will be continued until any of the following events occur * progression of disease * the subject develops unacceptable toxicity * consent to participate in the study is withdrawn

RAD001

Eligibility Criteria

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

You may qualify if:

• \. Histologically or cytologically confirmed pheochromocytoma or extra-adrenal paraganglioma or carcinoid
• \. Local, locally-advanced or metastatic disease documented as having shown progression on a scan (CT, MRI, MIBI scan) taken 2 to 12 months prior to baseline compared to a previous scan taken at any time in the past. Progression must be documented according to RECIST criteria.
• \. Disease that is not amenable to surgery, radiation or combined modality therapy with curative intent.
• \. Presence of at least one measurable target lesion for further evaluation according to RECIST criteria
• \. 18 years or older
• \. ECOG performance status 0, 1
• \. Previous treatment with chemotherapy, loco-regional therapy (e.g chemoembolization) are permitted providing that toxicity has resolved to ≤grade 1 at study entry and that last treatment was at least 4 weeks prior to baseline assessment.
• \. Adequate organ function
• \. A patient with the willingness to comply with the study protocol during the study period and capable of complying with it
• \. A patient who signed the informed consent prior to the participation of the study and who understands that he/she has a right to withdrawal from participation in the study at any time without any disadvantages.

You may not qualify if:

• \. A patient with no measurable disease
• \. Prior chemotherapy, radiation therapy or surgery within 4 weeks prior to study entry except palliative radiotherapy to non-target lesions (within 2 weeks prior to study entry)
• \. A patient with functioning carcinoid
• \. A patient with previous active or passive immunotherapy
• \. A patient with intestinal obstruction or impending obstruction, recent active upper GI bleeding
• \. A pregnant or lactating patient
• \. A patient of childbearing potential without being tested for pregnancy at baseline or with being tested for positive. (A postmenopausal woman with the amenorrhea period of at least 12 months or longer is considered to have non-childbearing potential)
• \. A man or woman of childbearing potential who has no willingness to use a contraceptive measure during the study
• \. A patient with history of another malignant disease within past 5 years, except curatively treated basal cell carcinoma of skin and cervical carcinoma in situ.
• \. A patient with history of uncontrolled seizures, central nervous system disorder or psychiatric disorders that are considered clinically significant by the investigator that would prohibit the understanding of informed consent or that may be considered to interfere with the compliance of the administration of the study medications.
• \. A patient with clinically significant heart disease (e.g. congestive heart failure, symptomatic coronary artery diseases, cardiac arrhythmia, etc) or myocardial infarction within past 12 months.
• \. Ongoing cardiac arrhythmia of grade ≥2, atrial fibrillation of any grade, or QTc interval\>450msec for males or \>470msec for female.
• \. A patient with interstitial pneumonia or diffuse symptomatic fibrosis of the lungs
• \. A patient with organ transplantation requiring immunosuppressive therapy

Sponsors & Collaborators

• Seoul National University Hospital: lead

Study Sites (1)

Seoul National University Hospital

Seoul, 110-744, South Korea

Location

MeSH Terms

Conditions

Pheochromocytoma; Paraganglioma, Extra-Adrenal

Interventions

Everolimus

Condition Hierarchy (Ancestors)

Paraganglioma; Neuroendocrine Tumors; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Nerve Tissue

Intervention Hierarchy (Ancestors)

Sirolimus; Macrolides; Lactones; Organic Chemicals

Study Officials

• Yung-Jue Bang, MD, PhD

  Seoul National University Hospital

  PRINCIPAL INVESTIGATOR

• Do-Youn Oh, MD, PhD

  Seoul National University Hospital

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 31, 2010
• First Posted: June 29, 2010
• Study Start: July 1, 2008
• Primary Completion: May 1, 2011
• Study Completion: December 1, 2011
• Last Updated: May 20, 2015

Record last verified: 2010-06

Locations

KR (1)