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    Summary
    EudraCT Number:2019-001059-37
    Sponsor's Protocol Code Number:AKST4290-221
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2019-06-11
    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 - BfArM
    A.2EudraCT number2019-001059-37
    A.3Full title of the trial
    Double-Blind, Randomized, Placebo-Controlled Trial of AKST4290 for Adjunctive Treatment of Mild to Moderate Bullous Pemphigoid
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Double-Blind, Randomized, Placebo-Controlled Trial of AKST4290 for Adjunctive Treatment of Mild to Moderate Bullous Pemphigoid
    A.4.1Sponsor's protocol code numberAKST4290-221
    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 SponsorAlkahest, 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 supportAlkahest, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAlkahest, Inc.
    B.5.2Functional name of contact pointJonas Hannestadt, MD, PhD
    B.5.3 Address:
    B.5.3.1Street Address125 Shoreway Road, Suite D,
    B.5.3.2Town/ city San Carlos
    B.5.3.3Post codeCA 94070
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1650801-0469
    B.5.5Fax number+1650801-0480
    B.5.6E-mailjhannestad@alkahest.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 nameAKST4290
    D.3.2Product code AKST4290
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNAKST4290
    D.3.9.1CAS number 1251528-23-0
    D.3.9.2Current sponsor codeAKST4290
    D.3.9.3Other descriptive name2-[[[(2R)-1-[1-[(4-CHLORO-3-METHYLPHENYL)METHYL]-4- PIPERIDINYL]-5-OXO-2-PYRROLIDINYL]CARBONYL]AMINO]- N,N,6-TRIMETHYL-4-PYRIDINECARBOXAMIDE,DIHYDROCHLORIDE
    D.3.9.4EV Substance CodeSUB191358
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number400
    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 No
    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 placeboFilm-coated tablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Mild to Moderate Bullous Pemphigoid
    E.1.1.1Medical condition in easily understood language
    Mild to Moderate Bullous Pemphigoid
    E.1.1.2Therapeutic area Diseases [C] - Skin and Connective Tissue Diseases [C17]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level LLT
    E.1.2Classification code 10006567
    E.1.2Term Bullous pemphigoid
    E.1.2System Organ Class 100000004858
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To investigate the proportion of subjects who achieve disease control (defined as ≤ 3 new blisters/day and healing of existing blisters) following topical steroid treatment with adjunctive AKST4290 without receiving rescue therapy.
    E.2.2Secondary objectives of the trial
    To assess treatment safety of the proposed dosing regimen. Additional secondary endpoints include assessment of time to disease control; time to rescue therapy; change in BP Disease Area Index (BPDAI) score by treatment week and at disease control; and change in pruritis as assessed by the BPDAI-Visual Analog Scale (BPDAI-VAS) by treatment week and at disease control. In addition, change in skin (biopsy) eosinophil counts and overall steroid use required to achieve disease control will be assessed.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Age 60-95 years, inclusive at screening.
    2. Clinical diagnosis of mild or moderate BP at screening:
    o Mild BP is defined as BPDAI ≤ 10 OR < 10% affected body surface
    o Moderate BP is defined as BPDAI ≥ 10 and ≤ 55 OR 10-30% affected body surface
    3. Treatment naïve or initiation of whole-body high potency topical steroid treatment < 7 days of screening (lesion-only treatment for any amount of time with any topical steroids prior to screening is allowed without restriction).
    4. Female subjects must not be pregnant or breastfeeding. Women of childbearing potential (WOCBP) must have a negative pregnancy test at screening. WOCBP and men must agree to use highly effective contraception (Clinical Trial Facilitation Group 2014) prior to study entry. A woman is considered of childbearing potential following menarche and until becoming postmenopausal (no menses for at least 1 year without an alternative cause). Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in the study, she should inform her treating physician immediately. Male subjects must be willing to use a barrier method contraception while participating in the study.
    5. The subject must be able to follow the study procedures and receive the treatment in the established timeframe.
    6. The subject must be able to understand the procedures and agree to complete the required assessments.
    7. Provide a signed and dated informed consent form in accordance with local regulations and/or IRB/IEC guidelines.
    E.4Principal exclusion criteria
    1. Severe BP (BPDAI score ≥ 56 OR > 30 new blisters per day OR > 30% affected body surface).
    2. Initiation of gliptins and other treatments (e.g., etanercept, sulfasalazine, furosemide, penicillin) that can trigger BP if this treatment was started within 4 weeks prior to screening and is considered possibly related to the onset of BP.
    3. Initiation of any concomitant medication in the last 3 months prior to screening and assessed by the investigator as possibly related to the development of BP.
    4. Planned use of intravenous immunoglobulin or other concomitant treatments for BP (i.e., doxycycline, dapsone) during the study period.
    5. Life expectancy of < 6 months (as assessed by the investigator).
    6. Use of systemic immunosuppressants (i.e., mycophenolate, azathioprine, methotrexate) within 4 weeks prior to screening.
    7. Treatment with rituximab within 1 year prior to screening.
    8. Medical history of:
    • Myocardial infarction or stroke within 6 months of screening
    • Active bleeding disorder
    • Major surgery within 1 month of screening or planned within the study period
    • Active liver disease
    • Positive screening test result for hepatitis B virus (HBV), hepatitis C virus (HCV), human immunodeficiency virus (HIV), or tuberculosis (TB)(by QuantiFERON testing)
    9. Prior treatment within 2 weeks or planned use of strong/potent cytochrome (CYP) P450 3A4/5 (CYP3A4/5) or P-glycoprotein (P-gp) inhibitors or inducers during the study (see Section 17.6.1).
    10. Current or planned concomitant use of drugs that are P-gp sensitive substrates and that have a narrow therapeutic index (NTI) (e.g., some factor Xa inhibitors) (see Section 17.6.1).
    11. Subjects taking warfarin (see Section 17.6.1).
    12. Renal function as defined by estimated glomerular filtration rate (eGFR) < 45 mL/min/1.73 m2 using the Modification of Diet in Renal Disease (MDRD) study equation (see
    Section 17.5).
    13. Use of systemic steroids (>10 mg prednisone or equivalent/day) within 14 days of first dose of study agent or known diseases (other than BP) that could require the use of systemic steroids within the study period. Subjects who have received a single high-dose of steroids (intravenous or oral) 14 days or more before the administration of the first dose of study drug are eligible for enrollment.
    14. Clinically relevant abnormal laboratory value at screening, including hematology, blood chemistry, or urinalysis (laboratory testing may be repeated once during the screening phase).
    15. Significant alcohol or drug abuse within past 2 years.
    16. Based on ECG reading, subjects with a risk of QT prolongation including:
    • A baseline prolongation of QTc (using Fridericia’s formula: ≥ 450 ms in men and ≥ 470 ms in women) with confirmation on a repeat ECG.
    • A history of additional risk factors for Torsades de pointes arrhythmia (e.g., heart failure, hypokalemia, family history of Long QT Syndrome, etc.).
    17. The use of concomitant medications known to prolong the QT/QTc interval.
    18. Significant medical conditions (as determined by medical history, examination, and clinical investigations at screening) that may, in the opinion of the investigator, result in the any of the following:
    • Put the subject at risk because of participation in the study.
    • Influence the results of the study.
    • Cause concern regarding the subject’s ability to participate in the study.
    19. Malignancy for which the subject is currently undergoing resection, radiation, or chemotherapy.
    20. Participation in studies of investigational drugs must have been discontinued within 30 days or 5 half-lives of the drug (whichever was longer) prior to screening.
    E.5 End points
    E.5.1Primary end point(s)
    The proportion of subjects who achieve disease control (≤ 3 new blisters/day and healing of existing blisters) without requiring rescue therapy.
    E.5.1.1Timepoint(s) of evaluation of this end point
    At 1-3 weeks depending on when subjects reach disease control.
    E.5.2Secondary end point(s)
    • Safety as assessed by the incidence, seriousness, and severity of adverse events (AEs).
    • Time to disease control by treatment Day/Week.
    • Time to rescue therapy by treatment Day/Week.
    • Change from baseline in BPDAI score by treatment Week and at disease control.
    • Change from baseline in pruritus as evaluated by the BPDAI-VAS by treatment week and at disease control.
    • Change from baseline in skin biopsy eosinophil levels at disease control.
    • Evaluation of total cumulative steroid exposure at baseline, by treatment Week and at disease control.
    • Evaluation of maximum daily steroid dose at baseline, by treatment Week and at disease control.
    E.5.2.1Timepoint(s) of evaluation of this end point
    At 1-3 weeks depending on when subjects reach disease control.
    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 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) No
    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 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 Yes
    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 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 concerned8
    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 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 years
    E.8.9.1In the Member State concerned months15
    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 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: 10
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 20
    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 state30
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 30
    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 participation in the trial has ended, further treatment will be at the discretion of the investigator and/or treating physician.
    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 Decision2019-06-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-06-18
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2021-04-15
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