Study to Allow Access to Pasireotide for Patients Benefiting From Pasireotide Treatment in Novartis-sponsored Studies

An Open Label, Multi-center Pasireotide Roll-over Protocol for Patients Who Have Completed a Previous Novartis-sponsored Pasireotide Study and Are Judged by the Investigator to Benefit From Continued Pasireotide Treatment

The purpose of this study is to allow continued use of pasireotide in patients who are on pasireotide treatment in a Novartis-sponsored study and are benefiting from the treatment as judged by the investigator.

Study Overview

• Status: Completed
• Conditions: Prostate Cancer; Neuroendocrine Tumors; Acromegaly; Cushing's Disease; Dumping Syndrome; Pituitary Tumors; Ectopic ACTH Secreting (EAS) Tumors; Melanoma Negative for bRAF; Melanoma Negative for nRAS
• Intervention / Treatment: Drug: Pasireotide; Drug: Cabergoline; Drug: Pasireotide LAR

Detailed Description

This is a multi-center, open label, phase IV study to provide continued supply of pasireotide to patients being treated in a current Novartis-sponsored study and who are benefiting from treatment with pasireotide alone or in combination with another treatment for Cushing's Disease and Acromegaly . Eligible patients are to be consented and can then continue treatment with pasireotide alone or in combination with another treatment for Cushing's Disease and Acromegaly in this protocol. All patients at their scheduled visits will have drug dispensing information and reported adverse events and serious adverse events collected.

A patient will reach the end of study when pasireotide treatment is permanently discontinued and the end of treatment visit has been performed. All patients must be followed up for safety evaluations for 3 months following the last dose of pasireotide LAR treatment and for 1 month following the last dose of pasireotide s.c. treatment.

The study is expected to remain open for approximately 10 years or until such time that enrolled patients no longer need treatment with pasireotide or are able to obtain commercial supply according to local regulations for their medical condition.

• Study Type: Interventional
• Enrollment (Actual): 337
• Phase: Phase 4

Contacts and Locations

Study Locations

• Argentina
  • Buenos Aires
    • Caba, Buenos Aires, Argentina, C1405BCH
      • Recordati Investigative Site
    • Mar del Plata, Buenos Aires, Argentina, B7602CBM
      • Recordati Investigative Site
• Belgium
  • Brugge, Belgium, 8310
    • Recordati Investigative Site
  • Brussel, Belgium, 1090
    • Recordati Investigative Site
  • Bruxelles, Belgium, 1200
    • Recordati Investigative Site
  • Gent, Belgium, 9000
    • Recordati Investigative Site
  • Leuven, Belgium, 3000
    • Recordati Investigative Site
  • Liege, Belgium, 4000
    • Recordati Investigative Site
  • Wilrijk, Belgium, 2610
    • Recordati Investigative Site
  • Antwerpen
    • Edegem, Antwerpen, Belgium, 2650
      • Recordati Investigative Site
• Brazil
  • CE
    • Fortaleza, CE, Brazil, 60430 370
      • Recordati Investigative Site
  • PR
    • Curitiba, PR, Brazil, 80030-110
      • Recordati Investigative Site
  • RJ
    • Rio de Janeiro, RJ, Brazil, 21941-913
      • Recordati Investigative Site
  • RS
    • Porto Alegre, RS, Brazil, 90560 030
      • Recordati Investigative Site
  • SC
    • Joinville, SC, Brazil, 89201260
      • Recordati Investigative Site
  • SP
    • Botucatu, SP, Brazil, 18618-970
      • Recordati Investigative Site
    • Sao Paulo, SP, Brazil, 05403 000
      • Recordati Investigative Site
• Bulgaria
  • Sofia, Bulgaria, 1431
    • Recordati Investigative Site
• Canada
  • Nova Scotia
    • Halifax, Nova Scotia, Canada, B3H 1V7
      • Recordati Investigative Site
  • Ontario
    • London, Ontario, Canada, N6A 4L6
      • Recordati Investigative Site
  • Quebec
    • Montreal, Quebec, Canada, H2W 1T8
      • Recordati Investigative Site
    • Montreal, Quebec, Canada, H2L 4M1
      • Recordati Investigative Site
• France
  • Angers cedex 09, France, 49933
    • Recordati Investigative Site
  • Bron Cedex, France, 69677
    • Recordati Investigative Site
  • Le Kremlin Bicetre, France, 94275
    • Recordati Investigative Site
  • Lille Cedex, France, 59037
    • Recordati Investigative Site
  • Marseille cedex 05, France, 13385
    • Recordati Investigative Site
  • Pessac Cedex, France, 33604
    • Recordati Investigative Site
  • Pierre Benite Cedex, France, 69495
    • Recordati Investigative Site
  • Cedex 9
    • Toulouse, Cedex 9, France, 31000
      • Recordati Investigative Site
• Germany
  • Berlin, Germany, 13353
    • Recordati Investigative Site
  • Hamburg, Germany, 20246
    • Recordati Investigative Site
  • Muenchen, Germany, 81377
    • Recordati Investigative Site
  • Muenchen, Germany, 81667
    • Recordati Investigative Site
  • Tübingen, Germany, 72076
    • Recordati Investigative Site
  • Ulm, Germany, 89081
    • Recordati Investigative Site
  • Wuerzburg, Germany, 97080
    • Recordati Investigative Site
• Greece
  • GR
    • Athens, GR, Greece, 115 27
      • Recordati Investigative Site
• Hungary
  • Budapest, Hungary, 1085
    • Recordati Investigative Site
• India
  • Karnataka
    • Bangalore, Karnataka, India, 560054
      • Recordati Investigative Site
  • Tamil Nadu
    • Vellore, Tamil Nadu, India, 632004
      • Recordati Investigative Site
• Israel
  • Petach Tikva, Israel, 49100
    • Recordati Investigative Site
• Italy
  • AN
    • Ancona, AN, Italy, 60126
      • Recordati Investigative Site
  • GE
    • Genova, GE, Italy, 16132
      • Recordati Investigative Site
  • MI
    • Milano, MI, Italy, 20149
      • Milano Investigative Site
  • PD
    • Padova, PD, Italy, 35128
      • Recordati Investigative Site
  • RM
    • Roma, RM, Italy, 00168
      • Recordati Investigative Site
• Japan
  • Aichi
    • Nagoya, Aichi, Japan, 460-0001
      • Recordati Investigative Site
  • Gunma
    • Maebashi city, Gunma, Japan, 371 8511
      • Recordati Investigative Site
  • Hyogo
    • Kobe-shi, Hyogo, Japan, 650-0017
      • Recordati Investigative Site
  • Kochi
    • Nankoku city, Kochi, Japan, 783 8505
      • Recordati Investigative Site
  • Kyoto
    • Kyoto-city, Kyoto, Japan, 612-8555
      • Recordati Investigative Site
  • Osaka
    • Suita city, Osaka, Japan, 565 0871
      • Recordati Investigative Site
• Korea, Republic of
  • Seoul, Korea, Republic of, 03722
    • Recordati Investigative Site
  • Seoul, Korea, Republic of, 06351
    • Recordati Investigative Site
• Malaysia
  • Kuala Lumpur, Malaysia, 62502
    • Recordati Investigative Site
  • Pulau Pinang, Malaysia, 10990
    • Recordati Investigative Site
• Mexico
  • Durango, Mexico, 34270
    • Recordati Investigative Site
  • Mexico City, Mexico, 07760
    • Recordati Investigative Site
  • Distrito Federal
    • México, Distrito Federal, Mexico, 14269
      • Recordati Investigative Site
  • Jalisco
    • Guadalajara, Jalisco, Mexico, 44130
      • Recordati Investigative Site
• Netherlands
  • Groningen, Netherlands, 9713 GZ
    • Recordati Investigative Site
• Peru
  • Lima
    • San Isidro, Lima, Peru, 27
      • Recordati Investigative Site
• Poland
  • Gdansk, Poland, 80 952
    • Recordati Investigative Site
  • Poznan, Poland, 60-355
    • Recordati Investigative Site
  • Warszawa, Poland, 04141
    • Recordati Investigative Site
  • Wroclaw, Poland, 50 367
    • Recordati Investigative Site
• Portugal
  • Porto, Portugal, 4200-319
    • Recordati Investigative Site
• Romania
  • Bucuresti, Romania, 011863
    • Recordati Investigative Site
• Russian Federation
  • Barnaul, Russian Federation, 656024
    • Recordati Investigative Site
  • Moscow, Russian Federation, 123317
    • Recordati Investigative Site
  • Saint Petersburg, Russian Federation, 197341
    • Recordati Investigative Site
  • Tyumen, Russian Federation, 625023
    • Recordati Investigative Site
• Spain
  • Barcelona, Spain, 08041
    • Recordati Investigative Site
  • Comunidad Valenciana
    • Alicante, Comunidad Valenciana, Spain, 03010
      • Recordati Investigative Site
• Switzerland
  • Lausanne, Switzerland, 1011
    • Recordati Investigative Site
  • Zuerich, Switzerland, 8091
    • Recordati Investigative Site
• Taiwan
  • Taichung, Taiwan, 40705
    • Recordati Investigative Site
• Thailand
  • Bangkok, Thailand, 10700
    • Recordati Investigative Site
  • Bangkok, Thailand, 10400
    • Recordati Investigative Site
  • Songkla, Thailand, 90110
    • Recordati Investigative Site
• Turkey
  • Ankara, Turkey, 06500
    • Recordati Investigative Site
  • Antalya, Turkey, 07070
    • Recordati Investigative Site
  • Istanbul, Turkey, 34890
    • Recordati Investigative Site
  • Izmir, Turkey, 35340
    • Recordati Investigative Site
  • Kocaeli, Turkey, 41380
    • Recordati Investigative Site
  • TUR
    • Istanbul, TUR, Turkey, 34098
      • Recordati Investigative Site
• United States
  • California
    • La Jolla, California, United States, 92037
      • Ximed Research SC - SOM230B2412
    • Los Angeles, California, United States, 90048
      • Cedars Sinai Medical Center Cedars Sinai 4
    • Stanford, California, United States, 94304
      • Stanford Universtiy Medical Center Stanford Hospital & Clinics
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • University of Michigan Comprehensive Cancer Center SC-2
  • New York
    • New York, New York, United States, 10065
      • Memorial Sloan-Kettering Cancer Center SC
  • Virginia
    • Chesapeake, Virginia, United States, 23321
      • Virginia Endocrinology Research SC
  • Washington
    • Seattle, Washington, United States, 98104
      • Swedish Cancer Institute Swedish Cancer Institute (SC)

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Patient is currently participating in a Novartis-sponsored study receiving pasireotide (LAR and/or s.c.) on monotherapy or combination therapy (for Cushing's Disease or Acromegaly), and has fulfilled all required assessments in the parent study and patients that are benefiting from the study treatment have no other alternatives.
2. Patient is currently benefiting from the treatment with pasireotide, as determined by the investigator
3. Patient has demonstrated compliance, as assessed by the investigator, with the parent study requirements.
4. Willingness and ability to comply with scheduled visits, treatment plans and any other study procedures.

5. Written informed consent obtained prior to enrolling in roll-over study and receiving study medication.

   • If consent cannot be expressed in writing, it must be formally documented and witnessed, ideally via an independent trusted witness.

Exclusion Criteria:

1. Patient has been permanently discontinued from pasireotide study treatment in the parent study due to unacceptable toxicity, non-compliance to study procedures, withdrawal of consent or any other reason.
2. Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test.

3. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during the study treatment and for 30 days after the final dose of pasireotide s.c. and 84 days after the final dose of pasireotide LAR. Highly effective contraception is defined as either:

   • Total abstinence (when this is in line with the preferred and usual lifestyle of the patient. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception
   • Female sterilization (have had surgical bilateral oophorectomy with or without hysterectomy) or tubal ligation at least six weeks before taking study treatment. In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment.
   • Male sterilization (at least 6 months prior to enrolling). For female patients on the study the vasectomized male partner should be the sole partner for that patient.
   • Use of oral, injected or implanted hormonal methods of contraception or placement of an intrauterine device (IUD) or intrauterine system (IUS), or other forms of hormonal contraception that have comparable efficacy (failure rate <1%), for example hormone vaginal ring or transdermal hormone contraception.
   • In case of use of oral contraception women should have been stable on the same pill for a minimum of 3 months before taking study treatment.
   • Women are considered post-menopausal and not of child bearing potential if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (i.e. age appropriate, history of vasomotor symptoms) or have had surgical bilateral oophorectomy (with or without hysterectomy), total hysterectomy, or tubal ligation at least six weeks ago. In the case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment is she considered not of child-bearing potential.
   • Sexually active males, unless they use a condom during intercourse while taking drug and for 1 months after pasireotide s.c. last dose and 3 months after pasireotide LAR last dose, should not father a child in this period. A condom is required to be used also by vasectomized men in order to prevent delivery of the drug via seminal fluid.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Pasireotide subcutaneous; 0.3mg, 0.6mg and 0.9mg. Doses to be taken BID or TID, dependent on parent study guidelines. Cabergoline may be combined in this arm for Cushing's Disease and Acromegaly patients.	Drug: Pasireotide; Administered subcutaneously in strengths 0.3mg, 0.6mg and 0.9mg. Doses to be taken BID or TID, dependent on parent study guidelines.; Other Names: SOM230; Signifor; Drug: Cabergoline; Cabergoline tablet 0.5mg or 1.0mg taken by mouth once daily may be combined with subcutaneous formulation of pasireotide for Cushing's Disease or Acromegaly. Dose is dependent on parent study guidelines.; Other Names: dostinex
Experimental: Pasireotide Long Acting Release (LAR); 10mg, 20mg, 40mg and 60mg. All doses to be taken q28days. Strength is dependent on parent study guidelines.	Drug: Pasireotide LAR; Long Acting Release is administered by a single intramuscular (i.m.) monthly injection. The strengths are 10mg, 20mg, 40mg and 60mg. All doses to be taken q28days. Strength is dependent on parent study guidelines.; Other Names: SOM230; Signifor

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of Adverse Events to Evaluate Long Term Safety Data	Collect long term safety data, i.e. SAEs and AEs. SAES will be reviewed and reported as part of the regular pharmacovigilance activities.	Baseline up to approximately 10 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Patients With Clinical Benefit as Assessed by the Investigator	Efficacy was assessed by the investigator at each scheduled visit using a binary (yes/no) response confirming the investigator's judgement of clinical benefit (via the question: "Does investigator confirm that the subject continues to have clinical benefit from the study treatment"). No other measures of efficacy were used in this study.	Baseline up to approximately 10 years

Collaborators and Investigators

• Sponsor: RECORDATI GROUP
• Investigators: Study Director: Recordati, Recordati AG

Study record dates

Study Major Dates

• Study Start (Actual): June 10, 2013
• Primary Completion (Actual): July 25, 2023
• Study Completion (Actual): July 25, 2023

Study Registration Dates

• First Submitted: February 15, 2013
• First Submitted That Met QC Criteria: February 15, 2013
• First Posted (Estimated): February 20, 2013

Study Record Updates

• Last Update Posted (Actual): October 1, 2024
• Last Update Submitted That Met QC Criteria: September 16, 2024
• Last Verified: August 1, 2024

More Information

Terms related to this study

• Keywords: Acromegaly; pasireotide LAR; SOM230,; roll-over study; Cushing's disease; neuroendocrine tumorsNETs; pituitary tumors; Ectopic ACTH secreting; EAS; Dumping Syndrome; metastatic prostate cancer, metastatic melanoma; bRAF; nRAS
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Skin Diseases; Neoplasms by Histologic Type; Neoplasms; Urogenital Neoplasms; Neoplasms by Site; Neoplasms, Glandular and Epithelial; Postoperative Complications; Endocrine System Diseases; Gastrointestinal Diseases; Stomach Diseases; Endocrine Gland Neoplasms; Genital Neoplasms, Male; Prostatic Diseases; Musculoskeletal Diseases; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms, Nerve Tissue; Hypothalamic Diseases; Hypothalamic Neoplasms; Supratentorial Neoplasms; Brain Neoplasms; Central Nervous System Neoplasms; Nervous System Neoplasms; Bone Diseases; Nevi and Melanomas; Bone Diseases, Endocrine; Hyperpituitarism; Adenoma; Postgastrectomy Syndromes; Skin Neoplasms; Urogenital Diseases; Male Urogenital Diseases; Genital Diseases, Male; Genital Diseases; Prostatic Neoplasms; Pituitary Neoplasms; Pituitary Diseases; Melanoma; Acromegaly; Neuroendocrine Tumors; ACTH-Secreting Pituitary Adenoma; Pituitary ACTH Hypersecretion; Dumping Syndrome; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Dopamine Agonists; Dopamine Agents; Antiparkinson Agents; Anti-Dyskinesia Agents; Cabergoline; Somatostatin; Pasireotide
• Other Study ID Numbers: CSOM230B2412

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No