An Open-label, Multi-center, Expanded Access Study of Pasireotide s.c. in Patients With Cushing's Disease (MACS1599)

This study is currently recruiting participants. (see Contacts and Locations)
Verified July 2014 by Novartis
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01582061
First received: April 12, 2012
Last updated: July 8, 2014
Last verified: July 2014
  Purpose

This study will document the safety and efficacy of pasireotide s.c. in patients with Cushing's disease.


Condition Intervention Phase
Cushing's Disease
Drug: Pasireotide sub-cutaneous formulation
Phase 3

Novartis Pharmaceuticals has indicated that access to an investigational treatment associated with this study is available outside the clinical trial.  

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open-label, Multi-center, Expanded Access Study of Pasireotide s.c. in Patients With Cushing's Disease (Seascape)

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • Proportion of patients having a drug-related adverse event that is recorded as grade 3 or 4 or as a serious adverse event [ Time Frame: Week 0, 1, 2, 3, 4, 8, 12, 16, 20, 24, 36, 48 ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Proportion of patients with mean Urinary Free Cortisol (UFC) ≤ Upper Limit of Normal (ULN) [ Time Frame: Baseline, week 12, 24 and 48 ] [ Designated as safety issue: No ]
  • Proportion of patients achieving a reduction of mean UFC ≥ 50% [ Time Frame: Baseline, week 12, 24 and 48 ] [ Designated as safety issue: No ]
  • Change in Cushing's disease clinical signs and symptoms [ Time Frame: Baseline, week 12, 24 and 48 ] [ Designated as safety issue: No ]
  • Change in Cushing Quality of Life and Work Productivity and Activity Impairment-General Health (WPAI-GH) scores [ Time Frame: Baseline, week 12, 24 and 48 ] [ Designated as safety issue: No ]
  • Change in GH and IGF-I values [ Time Frame: Baseline, week 12, 24 and 48 ] [ Designated as safety issue: No ]

Estimated Enrollment: 200
Study Start Date: August 2011
Estimated Study Completion Date: October 2015
Estimated Primary Completion Date: October 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Pasireotide sub-cutaneous formulation
Pasireotide will be administered twice a day until pasireotide is approved and reimbursed for commercial use in each respective country or until 31DEC15, whichever comes first
Drug: Pasireotide sub-cutaneous formulation
Pasireotide will be administered twice a day until pasireotide is approved and reimbursed for commercial use in each respective country or until 31DEC15, whichever comes first
Other Name: SOM230 sub-cutaneous formulation

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion criteria:

  1. Patients with confirmed diagnosis of Cushing's disease as evidenced by

    • mean urinary free cortisol of three 24-hour urine samples collected during the 2-week screening period above the upper limit of the laboratory normal range
    • morning plasma adrenocorticotropic hormone (ACTH) within the normal or above normal range
    • either Magnetic Resonance Imaging (MRI) confirmation of pituitary adenoma (greater than or equal to 0.6 cm), or inferior petrosal sinus gradient >3 after corticotrophin-releasing hormone (CRH) stimulation for those patients with a microadenoma less than 0.6 cm, or for patients who have had prior pituitary surgery, histopathology confirming an ACTH staining adenoma.
  2. Patients with de novo Cushing's disease must not be considered as candidates for pituitary surgery (i.e. poor surgical candidates, surgically unapproachable tumors, patients with no visible pituitary tumor, patients who refuse to have surgical treatment)
  3. Karnofsky performance status >60 (i.e. requires occasional assistance, but is able to care for most of his personal needs)

Exclusion criteria:

  1. Radiotherapy of the pituitary <4 weeks before screening or patient who has not recovered from side effects
  2. Patients with compression of the optic chiasm causing acute clinically significant visual field defect
  3. Patients with Cushing's syndrome due to ectopic ACTH secretion
  4. Patients with hypercortisolism secondary to adrenal tumors or nodular (primary) bilateral adrenal hyperplasia
  5. Patients who have undergone major surgery within 1 month prior to screening
  6. Patients with symptomatic cholelithiasis
  7. Patients who have clinically significant impairment in cardiovascular function or are at risk thereof.

Other protocol-defined inclusion/exclusion criteria may apply

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01582061

Contacts
Contact: Novartis Pharmaceuticals 1-888-669-6682
Contact: Novartis Pharmaceuticals

  Show 121 Study Locations
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

No publications provided

Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01582061     History of Changes
Other Study ID Numbers: CSOM230B2406
Study First Received: April 12, 2012
Last Updated: July 8, 2014
Health Authority: United States: Food and Drug Administration
Brazil: Ethics Committee
Brazil: Ministry of Health
Czech Republic: Ethics Committee
Czech Republic: State Institute for Drug Control
Egypt: Institutional Review Board
Germany: Ethics Commission
Germany: Ministry of Health
Greece: Ethics Committee
Greece: Ministry of Health and Welfare
Lebanon: Institutional Review Board
Netherlands: Independent Ethics Committee
Netherlands: Medicines Evaluation Board (MEB)
Romania: National Medicines Agency
Russia: Ethics Committee
Russia: Ministry of Health of the Russian Federation
South Korea: Institutional Review Board
South Korea: Korea Food and Drug Administration (KFDA)
Spain: Ethics Committee
Spain: Ministry of Health
Thailand: Ethical Committee
Thailand: Food and Drug Administration

Keywords provided by Novartis:
Cushing's disease
Hormone disorder
Cortisol
Adrenocorticotropic hormone
Pituitary tumor

Additional relevant MeSH terms:
Cushing Syndrome
Pituitary ACTH Hypersecretion
Adrenocortical Hyperfunction
Adrenal Gland Diseases
Endocrine System Diseases
Hyperpituitarism
Pituitary Diseases
Hypothalamic Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Adrenocorticotropic Hormone
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on July 31, 2014