Acromegaly is a chronic disorder caused by GH hypersecretion, most commonly as a result of a GH-secreting pituitary adenoma. MedDRA version: 20.0 Level: PT Classification code 10000599 Term: Acromegaly System Organ Class: 10014698 - Endocrine disorders
Conditions
Interventions
Product Name: ISIS 766720
Product Code: ISIS 766720
Pharmaceutical Form: Solution for injection
INN or Proposed INN: not available yet
CAS Number: 2131025-83-5
Current Sponsor code: ISIS 766720
Other
Sponsors
Ionis Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Must have given written informed consent (signed and dated) and any authorizations required by local law and be able to comply with all study requirements 2. Randomized in index trial ISIS 766720-CS2 and completed the entire study, or completion of the Treatment Period for ISIS 766720-CS2 and completed or plan to complete PTWK5 visit with an acceptable safety profile, per Investigator judgment 3. Patients with confirmed stable monthly regimen of SRL for 3 months prior to Screening 4. Able and willing to participate in a 53-week Treatment and 14-week Post-Treatment study 5. Females must be non-pregnant and non-lactating, and either a. surgically sterile (e.g., tubal occlusion, hysterectomy, bilateral salpingectomy, bilateral oophorectomy) b. post-menopausal (defined as 12 months of spontaneous amenorrhea in females > 55 years of age or, in females = 55 years, 12 months of spontaneous amenorrhea without an alternative medical cause and follicle stimulating hormone (FSH) levels in the postmenopausal range for the laboratory involved) c. abstinent or d. Women of childbearing potential (WOCBP) should agree to taking all precaution to avoid pregnancy during the Trial Period (including Post-Treatment), including agreeing to receive pregnancy testing before each monthly dose, using 1 highly effective method of birth control from the time of signing the informed consent form until 14 weeks after the last dose of ISIS 766720 administration 5. Males must be e. surgically sterile f. abstinent (defined in Protocol) g. if engaged in sexual relations with a female of child-bearing potential, the patient must be using 1 highly effective contraceptive method from the time of signing the informed consent form until 14 weeks after the last dose of ISIS 766720. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 52 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 8
Exclusion criteria
Exclusion criteria: 1. Have any new condition or worsening of existing condition which in the opinion of the Investigator would make the patient unsuitable for enrollment or could interfere with the patient participating in or completing the study 2. Treatment with any investigational drug, biological agent or device other than ISIS 766720 within 1 month of screening, or 5 half-lives of the investigational agent; whichever is longer. 3. Treatment with any other acromegaly medications taken prior to Day 1 within the time period listed below: bromocriptine: 2 weeks carbergoline: 4 weeks quinagolide: 4 weeks pegvisomant: 4 weeks pasireotide: 4 months 4. Patients who received surgery for pituitary adenoma in the last 3 months prior to screening and patients needing and/or planning to receive surgery for the pituitary adenoma during the trial 5. Unwilling to comply with required study procedures during the Treatment and Post-Treatment Periods 6. Screening laboratory assessments for chemistry, urinalysis, or hematology that have been confirmed and meet the study-specified monitoring or stopping rules. Results approaching monitoring rules will need to be reviewed by the Investigator and Sponsor Medical Monitor
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety of extended dosing with ISIS 766720 in patients with Acromegaly.;Secondary Objective: To evaluate the efficacy of extended dosing of ISIS 766720;Primary end point(s): The primary safety endpoint is the incidence of adverse events.;Timepoint(s) of evaluation of this end point: Incidence of adverse events throughout the study | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Baseline is defined as the Day 1 for ISIS 766720-CS2 for those treated with ISIS 766720 in ISIS 766720-CS2 and Day 1 for ISIS 766720-CS3 for those treated with placebo in ISIS 766720-CS2. The following secondary endpoints will be analyzed for efficacy: • Percent change from Baseline on IGF-1 levels at 6 months/Week 26 (Day 169) and 12 months/Week 53 (Day 365) • Proportion of patients who achieve normalized IGF-1 levels to within 1.2 times of gender and age adjusted upper limits at Week 26 (Day 169) and at 28 days after last dose Week 53 (Day 365) • Proportion of patients who achieve normalized IGF-1 levels to within 1.0 times of gender and age adjusted upper limits at Week 26 (Day 169) and at 28 days after last dose Week 53 (Day 365) • Proportion of patients who begin other acromegaly medication • Time from first dose of ISIS 766720 in ISIS 766720-CS3 to date of initiation of other acromegaly medications;Timepoint(s) of evaluation of this end point: • Percent change on IGF-1 levels: from Baselineat 6 months/Week 26 (Day 169) and 12 months/Week 53 (Day 365) • Proportion of patients who achieve normalized IGF-1 levels to within 1.2 times of gender and age adjusted upper limits: at Week 26 (Day 169) and at 28 days after last dose Week 53 (Day 365) • Proportion of patients who achieve normalized IGF-1 levels to within 1.0 times of gender and age adjusted upper limits: at Week 26 (Day 169) and at 28 days after last dose Week 53 (Day 365) • Proportion of patients who begin other acromegaly medication: at any timepoint of the study • Other acromegaly medications: Time from first dose of ISIS 766720 in ISIS 766720-CS3 to date of initiation of the other medication | — |
Countries
Hungary, Lithuania, Poland, Romania, Russian Federation, Serbia, United States
Contacts
Public ContactIonis Clinical Trial Information
Ionis Pharmaceuticals, Inc.
Outcome results
None listed