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    Summary
    EudraCT Number:2016-001275-80
    Sponsor's Protocol Code Number:X358605
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2016-04-11
    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 ConcernedGermany - PEI
    A.2EudraCT number2016-001275-80
    A.3Full title of the trial
    An Open-Label Study of XOMA 358, with Optional Dose Escalation, in Patients with Congenital Hyperinsulinism
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A non-blinded study of XOMA 358, with a possibility to increase the dose, in patients with abnormal levels of insulin due to a genetic dysfunction
    A.4.1Sponsor's protocol code numberX358605
    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 SponsorXOMA (US) LLC
    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 supportXOMA (US) LLC
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationKF2 / Kornelia Fiering Klinische Forschung
    B.5.2Functional name of contact pointKlinische Forschung
    B.5.3 Address:
    B.5.3.1Street AddressOldenburgallee 48
    B.5.3.2Town/ cityBerlin
    B.5.3.3Post code14052
    B.5.3.4CountryGermany
    B.5.4Telephone number+493089749646
    B.5.5Fax number+493089749645
    B.5.6E-mailmail@kf2-services.de
    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 Yes
    D.2.5.1Orphan drug designation numberEMA/OD/040/16
    D.3 Description of the IMP
    D.3.2Product code XOMA 358
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNXOMA 358
    D.3.9.2Current sponsor codeXOMA 358
    D.3.9.3Other descriptive namefully human IgG2 monoclonal antibody
    D.3.10 Strength
    D.3.10.1Concentration unit mg/l milligram(s)/litre
    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 No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    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 No
    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
    Hypoglycemia associated with congenital hyperinsulinism
    E.1.1.1Medical condition in easily understood language
    Low blood glucose levels associated with abnormally high level of insulin due to a specific birth defect
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.0
    E.1.2Level LLT
    E.1.2Classification code 10077227
    E.1.2Term Hyperinsulinemic hypoglycemia
    E.1.2System Organ Class 10027433 - Metabolism and nutrition disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.0
    E.1.2Level LLT
    E.1.2Classification code 10020644
    E.1.2Term Hyperinsulinism NOS
    E.1.2System Organ Class 10027433 - Metabolism and nutrition disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.0
    E.1.2Level PT
    E.1.2Classification code 10061211
    E.1.2Term Hyperinsulinism
    E.1.2System Organ Class 10027433 - Metabolism and nutrition disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.0
    E.1.2Level LLT
    E.1.2Classification code 10022484
    E.1.2Term Insulin hypoglycaemia
    E.1.2System Organ Class 10027433 - Metabolism and nutrition disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the safety and clinical pharmacology of XOMA 358 in patients with hypoglycemia associated with congenital hyperinsulinism (HI).
    E.2.2Secondary objectives of the trial
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Provide written informed consent and, as applicable, assent, before any study specific procedures are performed
    2. Aged 12 to 65 years at Screening
    3. Body mass index < 35 kg/m2 for patients aged 18 years and above and, for patients aged less than 18 years, BMI < 95% percentile for age
    4. An established clinical diagnosis, with or without a genetic diagnosis, of congenital hyperinsulinism
    5. Documented hypoglycemia (blood glucose < 60 mg/dL [< 3.33 mM]) during 2 monitored fasts during screening. Patients with blood glucose levels ≥ 60 mg/dL (≥ 3.33 mM) but who demonstrate a clinically meaningful decrease in blood glucose in conjunction with symptoms during the monitored fasts may be enrolled upon discussion and agreement between the Investigator and the Sponsor.
    6. In the opinion of the Investigator, can be safely washed out of background medications used to treat HI
    7. Hepatic ultrasound at Screening without clinically significant findings or evidence of peliosis hepatis
    8. For female patients of childbearing potential, a negative serum pregnancy test
    9. For female patients with reproductive potential (ie, fertile, following menarche and until becoming post-menopausal unless permanently sterile), a willingness to use highly effective* contraceptive measures adequate to prevent a new pregnancy for at least 3 months after administration of study drug. For women with reproductive potential who use a hormonal method of contraception, concurrent use of a second (barrier) method is recommended.

    (*Highly effective methods of birth control are defined as those that alone or in combination result in a low failure rate [ie, less than 1% per year] when used consistently and correctly [eg, implants, injectables, oral contraceptives, some intrauterine devices, bilateral tubal occlusion, true sexual abstinence in line with the preferred and usual lifestyle of the patient, or vasectomized partner.])
    E.4Principal exclusion criteria
    1. Any out-of-range laboratory value at Screening that has not been reviewed, approved, and documented as not clinically significant by the Investigator, except for the following liver function test exclusions:
    - total bilirubin ≥ 1.5X upper limit of normal
    - ALT, AST, or ALP ≥ 2X upper limit of normal
    2. Use of any agent, such as diazoxide, octreotide, chronic systemic glucocorticoids, or β agonists, that may affect glucose metabolism. Such agents will be washed out before dosing. Topical, inhaled, and intranasal corticosteroids at doses that are unlikely to affect glucose homeostasis and corticosteroids for ophthalmic use are permitted.
    3. Use of any long-acting somatostatin analogs or glucose-affecting medications that require > 72 hour washout
    Note: Patients who are on a stable dose and regimen of a long-acting somatostatin analog and meet inclusion criterion number 5 for hypoglycemia during Screening may remain on such treatment and be enrolled into the study upon discussion and agreement between the Investigator and the XOMA Medical Monitor.
    4. History of malignancy within 3 years before Screening other than carcinoma in situ of the cervix or adequately treated nonmetastatic squamous or basal cell carcinoma of the skin.
    5. History of seropositivity for HIV antibody, hepatitis B, or hepatitis C antibody
    6. Major general surgery within 3 months before Screening or anticipated during the study period
    7. Known allergy or sensitivity to XOMA 358 or any component of the study drug
    8. Treatment with an investigational drug or device within 30 days or 5 half-lives of the investigational drug before Day 1, whichever is longer. Participation in registries and purely diagnostic studies is allowed.
    9. Female patients who are pregnant, planning to become pregnant during the course of the study, have recently delivered (within 3 months before Screening), or are breast-feeding
    10. Male patients who are planning a pregnancy with a female partner during the course of the study or within 4 months after administration of study drug
    11. Any organ condition, concomitant disease (eg, psychiatric illness, severe alcoholism, or drug abuse, cardiac, hepatic, or kidney disease), or other abnormality that itself, or the treatment of which, could interfere with the conduct of the study (eg, may affect absorption, distribution, metabolism, or elimination of the study drug) or that, in the opinion of the Investigator and/or Sponsor’s medical monitor, would pose an unacceptable risk to the patient in the study.
    E.5 End points
    E.5.1Primary end point(s)
    Biological (analyses for each dose):
    • Glucose AUC24
    • Cumulative time blood glucose < 70 mg/dL (< 3.89 mM)
    • Fasting and postprandial glucose, insulin, c-peptide
    • Time to hypoglycemia during provocation (< 60 mg/dL [< 3.33 mM])
    • Frequency of hypoglycemic events (< 60 mg/dL [< 3.33 mM])
    • Concomitant treatment/medications used during rescue
    Additional analyses will be performed, as appropriate.
    Safety:
    Safety will be assessed through an examination of the incidence, severity, and type of treatment-emergent adverse events and changes in vital signs, physical examination results, laboratory test results, ECG, hepatic ultrasound, glucose monitoring via a bedside glucometer, infusion site reaction, and use of concomitant medications and concomitant treatment from baseline to specified time points throughout the trial.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Measured at various time points (see schedule of events)
    E.5.2Secondary end point(s)
    Not applicable
    E.5.2.1Timepoint(s) of evaluation of this end point
    Not applicable
    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 Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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 No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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
    LVLS
    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 months
    E.8.9.1In the Member State concerned days
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 10
    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) Yes
    F.1.1.6.1Number of subjects for this age range: 10
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 2
    F.1.3Elderly (>=65 years) No
    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 state12
    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)
    The single dose treatment in this study (with an optional second dose at a higher dose level depending in response) will inform the dosing regimen for next studies and patients will have the option to participate in any future studies.
    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 Decision2016-08-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-06-16
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2017-01-20
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