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    Summary
    EudraCT Number:2019-000591-42
    Sponsor's Protocol Code Number:ISIS766720-CS3
    National Competent Authority:Lithuania - SMCA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2019-04-24
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedLithuania - SMCA
    A.2EudraCT number2019-000591-42
    A.3Full title of the trial
    An Open-Label Extension Trial of IONIS GHR-LRX, an Antisense Inhibitor of the Growth Hormone Receptor Administered Monthly Subcutaneously to Patients with Acromegaly Being Treated with Long-Acting Somatostatin Receptor Ligands (SRL)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An open label study to assess the long term safety of the study drug, ISIS 766720, in patients with acromegaly (a hormonal disorder that results from too much growth hormone in the body)
    A.4.1Sponsor's protocol code numberISIS766720-CS3
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorIonis Pharmaceuticals, Inc.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportIonis Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationIonis Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointIonis Clinical Trial Information
    B.5.3 Address:
    B.5.3.1Street Address2855 Gazelle Court
    B.5.3.2Town/ cityCarlsbad
    B.5.3.3Post codeCA 92010
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1760603-3804
    B.5.5Fax number+1760603-2504
    B.5.6E-mailClinicalTrials@ionisph.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameISIS 766720
    D.3.2Product code ISIS 766720
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNnot available yet
    D.3.9.1CAS number 2131025-83-5
    D.3.9.2Current sponsor codeISIS 766720
    D.3.9.3Other descriptive nameISIS 766720
    D.3.9.4EV Substance CodeSUB191641
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product Yes
    D.3.11.13.1Other medicinal product type2'-MOE antisense oligonucleotide
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Acromegaly is a chronic disorder caused by GH hypersecretion, most commonly as a result of a GH-secreting pituitary adenoma.
    E.1.1.1Medical condition in easily understood language
    Acromegaly is a hormonal disorder that results from too much growth hormone (GH) in the body. Most cases the overproduction of GH is due to a benign tumor of the pituitary gland called an adenoma.
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10000599
    E.1.2Term Acromegaly
    E.1.2System Organ Class 10014698 - Endocrine disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the safety of extended dosing with ISIS 766720 in patients with Acromegaly.
    E.2.2Secondary objectives of the trial
    To evaluate the efficacy of extended dosing of ISIS 766720
    E.2.3Trial contains a sub-study No
    E.3Principal 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.
    E.4Principal 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
    E.5 End points
    E.5.1Primary end point(s)
    The primary safety endpoint is the incidence of adverse events.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Incidence of adverse events throughout the study
    E.5.2Secondary 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
    E.5.2.1Timepoint(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
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    extension study of ISIS 766720-CS2
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA18
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Hungary
    Lithuania
    Poland
    Romania
    Russian Federation
    Serbia
    United States
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months10
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months10
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 52
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 8
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state14
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 45
    F.4.2.2In the whole clinical trial 60
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    None
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2019-06-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-05-29
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2022-07-07
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