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    Summary
    EudraCT Number:2021-003764-27
    Sponsor's Protocol Code Number:HS-20-677
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2022-05-13
    Trial 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 ConcernedGermany - BfArM
    A.2EudraCT number2021-003764-27
    A.3Full title of the trial
    A randomized, placebo-controlled, double-blind, multi-center trial to assess
    efficacy and safety of octreotide subcutaneous depot (CAM2029) in
    patients with symptomatic polycystic liver disease
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    This study is being conducted to evaluate efficacy and safety of Octreotide compared with Placebo via subcutaneous depot route in subjects with polycystic liver disease


    A.3.2Name or abbreviated title of the trial where available
    POSITANO (POlycystic liver Safety and effIcacy TriAl with subcutaNeous Octreotide
    A.4.1Sponsor's protocol code numberHS-20-677
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05281328
    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 SponsorCamurus AB
    B.1.3.4CountrySweden
    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 supportCamurus AB
    B.4.2CountrySweden
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCamurus AB
    B.5.2Functional name of contact pointRegulatory Affairs
    B.5.3 Address:
    B.5.3.1Street AddressIdeon Science Park
    B.5.3.2Town/ cityLund
    B.5.3.3Post codeSE- 223 70
    B.5.3.4CountrySweden
    B.5.6E-mailregulatory@camurus.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 nameCAM2029 (Octreotide subcutaneous depot)
    D.3.2Product code CAM2029
    D.3.4Pharmaceutical form Prolonged-release 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 INNOCTREOTIDE
    D.3.9.1CAS number 83150-76-9
    D.3.9.2Current sponsor codeCAM2029
    D.3.9.4EV Substance CodeSUB09417MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    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 typeDosage form of IMP or Placebo Solution for injection in a pre-filled pen
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboProlonged-release solution for injection
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Polycystic liver disease
    E.1.1.1Medical condition in easily understood language
    Multiple fluid filled sac in liver
    E.1.1.2Therapeutic area Diseases [C] - Digestive System Diseases [C06]
    MedDRA Classification
    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 treatment effect of CAM2029 compared to placebo on liver volume in patients with polycystic liver disease (PLD)
    E.2.2Secondary objectives of the trial
    • To evaluate the treatment effect of CAM2029:
    o Compared to placebo on patient-reported PLD-related symptoms
    o On liver volume over time in patients with PLD
    o On patient-reported PLD-related symptoms over time
    o Over time on kidney volume in patients with presence of kidney cysts
    o Over time on renal function in patients with presence of kidney cysts
    o Over time on patient-reported PLD-related impact on functioning and well-being
    o Over time on PLD-related symptoms
    o Over time on functioning and well-being
    o Over time on PLD-related symptoms
    • To evaluate the safety and tolerability of CAM2029
    • To assess the PK of octreotide after administration of CAM2029
    Exploratory Objectives
    • To evaluate treatment effect of CAM2029 over time on
    o Total liver cyst volume
    o Change in liver volume compared to the pre-treatment growth rate
    o IGF-1
    • Assess patients’ satisfaction with CAM2029 T/T & self- administration












    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Male or female patient, ≥18 years at screening
    • Diagnosis of PLD (associated with ADPKD or isolated as in ADPLD) with htTLV ≥1800 mL/m at screening
    • Presence of at least 1 of the following PLD-related symptoms within 2 weeks before screening: bloating, fullness in abdomen, lack of appetite, feeling full quickly after beginning to eat, acid reflux, nausea, rib cage pain or pressure, pain in side, abdominal pain, back pain, shortness of breath after physical exertion, limited in mobility, concern about abdomen getting larger, dissatisfied by the size of abdomen
    • Not a candidate for, or not willing to undergo, surgical intervention for hepatic cysts during the trial
    E.4Principal exclusion criteria
    • Surgical intervention for PLD within 3 months before screening. For sclerotherapy patients, at least 6 months before screening
    • Treatment with a somatostatin analogue (SSA) within 3 months before screening
    • Non-responsive to previous treatment of PLD with an SSA as per the Investigator’s assessment
    • Symptomatic cholelithiasis within 3 months before screening or previous medical history of cholelithiasis induced by SSAs unless treated with cholecystectomy
    • Presence of extrahepatic cysts that, in the Investigator’s opinion, may prevent the patient from safely participating in the trial
    • Severe kidney disease, as defined by eGFR <30 mL/min/1.73 m2
    • Severe liver disease defined as liver cirrhosis of Child-Pugh class C
    • Use of oral contraceptives or estrogen supplementation within 3 months before screening
    • Poorly controlled diabetes (hemoglobin A1c ≥10%) at screening
    • Patients with a known history of hypothyroidism, unless they have been on adequate and stable replacement thyroid hormone therapy for at least 3 months before the first dose of the IMP.
    • Uncontrolled hypertension defined by a systolic blood pressure of >160 mmHg and/or diastolic blood pressure of >100 mmHg at screening
    • History of significant cardiac disease or current diagnosis of cardiac disease indicating significant risk of safety for patients participating in the trial, such as uncontrolled or significant cardiac disease, including any of the following:
    a. History of myocardial infarction, angina pectoris or coronary artery bypass graft within 6 months before screening
    b. Clinically significant cardiac arrhythmias (e.g. ventricular tachycardia), complete left bundle branch block or high-grade atrioventricular block (e.g. bifascicular block, Mobitz type II and third-degree atrioventricular block)
    c. Long QT syndrome, family history of idiopathic sudden death or congenital long QT syndrome, or any of the following:
    i. Risk factors for Torsades de Pointes including uncorrected hypokalemia or hypomagnesemia, history of cardiac failure or history of clinically significant/symptomatic bradycardia
    ii. Treatment with concomitant medication(s) with a "Known risk of Torsades de Pointes" that cannot be discontinued or replaced by safe alternative medication at least 5 half-lives or 7 days (whichever is longer) before the first dose of IMP
    iii. Patients with a baseline QTc interval corrected by Fridericia's formula >450 msec for males and >470 msec for females at screening
    • Patients with vascular compromise, including, but not limited to, mesenteric thrombosis, portal hypertension and thrombocytopenia (platelet counts less than 100x109/L)
    • Pregnant, lactating or planning to be pregnant during the trial
    • History of solid organ transplantation. Exception for kidney transplant patients
    • Contraindications to, or interference with, MRI assessments, as dictated by local hospital regulations
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline to Week 53 in height-adjusted total liver volume (htTLV) as determined by magnetic resonance imaging (MRI) volumetry

    • Change from baseline in total liver cyst volume determined by MRI volumetry
    • Rate of change in htTLV (calculated from up to the last 5 years prior to trial entry) as compared to htTLV change from baseline
    • Change from baseline in serum Insulin-like growth factor-1 (IGF-1) levels
    • Treatment Satisfaction Questionnaire for Medication (TSQM) scores using all 4 domains of TSQM v1.4 (effectiveness, side effects, convenience and satisfaction)
    • Change from PRE module to the POST module in Self-Injection Assessment Questionnaire (SIAQ) scores
    • Data collected in the pre-defined Validation Battery to be used for psychometric analyses to confirm measurement properties, reliability, validity and interpretability
    • Qualitative analysis of patients’ entry and exit interviews
    • Semi-quantitative analysis of anti-octreotide antibodies
    E.5.1.1Timepoint(s) of evaluation of this end point
    Timepoints:
    Before trail period
    1. During Screening period (Week -12 to -1)
    During trial phase
    2. Week 1
    3. Week 13
    4. Week 21
    5. Week 25
    6. Week 39
    7. Week 53
    8. Week 77

    If the MRI of the liver and kidneys performed at screening and Week
    77/End-of-Treatment include satisfactory images of the gallbladder,
    these scans may also be used for the safety assessment by the
    Investigator.
    E.5.2Secondary end point(s)
    • Change from baseline to Week 25 in the Polycystic Liver Disease Symptoms (PLD-S) measure score
    • Change from baseline in htTLV as determined by MRI volumetry
    • Change from baseline in the PLD-S measure score
    • Change from baseline in height-adjusted total kidney volume (htTKV) as measured by MRI volumetry
    • Change from baseline in estimated glomerular filtration rate (eGFR), assessed by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) cystatin C equation using serum concentrations of creatinine and cystatin C
    • Change from baseline in the Polycystic Liver Disease Impact (PLD-I) measure score
    Change from baseline in the Clinical Global Impression of Severity (CGI-S) score
    • Change from baseline in the Patient Global Impression of Severity (PGI-S) score
    • Change from baseline in the Patient Global Impression of Change (PGI-C) score
    • Change from baseline in the Short Form-36 (SF-36) scores
    • Change from baseline in the Polycystic Liver Disease Questionnaire (PLD-Q) score
    • Incidence of adverse events (AEs)
    • Changes from baseline in laboratory values, vital signs and electrocardiogram (ECG) readings
    • Octreotide plasma concentrations over time
    E.5.2.1Timepoint(s) of evaluation of this end point
    Before Trial start
    1. During the screening period (week -12 to -1)
    During trial phase
    2. Week 1
    3. Week 13
    4. Week 21
    5. Week 25
    6. Week 39
    7. Week 53
    8. Week 77
    Adverse events are evaluated through put the trial period
    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 No
    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) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial3
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA6
    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
    United States
    Belgium
    Germany
    Netherlands
    E.8.7Trial has a data monitoring committee Yes
    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
    A Safety Follow-up Visit will be performed 4 weeks after the Week 77/End-of-Treatment Visit.

    This is consider as last patient last visit for the trial, end of trial.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months8
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months8
    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: 69
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 3
    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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 41
    F.4.2.2In the whole clinical trial 69
    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)
    After the end of trial, Sponsor will not provide any additional treatment
    to patients
    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 Decision2022-06-22
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-07-19
    P. End of Trial
    P.End of Trial StatusOngoing
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