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    EudraCT Number:2018-001301-90
    Sponsor's Protocol Code Number:SNFCT2017-06
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-06-04
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2018-001301-90
    A.3Full title of the trial
    A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of SNF472 When Added to Background Care for the Treatment of Calciphylaxis
    Studio di fase 3, randomizzato, in doppio cieco, controllato con placebo per valutare l’efficacia e la sicurezza di SNF472 quando aggiunto alla terapia di fondo per il trattamento della calcifilassi
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to assess how safe and effective the study drug, SNF472, is when administered with background care for the treatment of calciphylaxis
    Uno studio per valutare quanto sia sicuro ed efficace il farmaco in studio, SNF472, che è somministrato con la terapia di fondo per il trattamento della calcifilassi.
    A.3.2Name or abbreviated title of the trial where available
    A.4.1Sponsor's protocol code numberSNFCT2017-06
    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 SponsorSANIFIT THERAPEUTICS S.A.
    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 supportSanifit Therapeutics S.A.
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSanifit Therapeutics S. A.
    B.5.2Functional name of contact pointSVP Development Operations
    B.5.3 Address:
    B.5.3.1Street Address3655 Nobel Dr. Suite 540
    B.5.3.2Town/ citySan Diego
    B.5.3.3Post codeCA 92122
    B.5.3.4CountryUnited States
    B.5.4Telephone number9456542
    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 numberEU3/12/1026
    D.3 Description of the IMP
    D.3.1Product nameNA
    D.3.2Product code [SNF472]
    D.3.4Pharmaceutical form Solution for injection/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.3.9.1CAS number 34367-89-0
    D.3.9.2Current sponsor codeNA
    D.3.9.4EV Substance CodeSUB166954
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number30
    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. cell therapy medicinal product Information not present in EudraCT
    D. therapy medical product Information not present in EudraCT
    D. Engineered Product Information not present in EudraCT
    D. ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D. on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 placeboSolution for injection/infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Calciphylaxis (calcific uremic arteriolopathy [CUA])
    calcifilassi (arteriolopatia calcifica uremica [CUA])
    E.1.1.1Medical condition in easily understood language
    Rare disease of calcification of the small blood vessels in the fatty tissue and deeper layers of the skin.
    Malattia rara di calcificazione dei piccoli vasi sanguigni nel tessuto adiposo e negli strati profondi della pelle
    E.1.1.2Therapeutic area Body processes [G] - Circulatory and Respiratory Physiological Phenomena [G09]
    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 efficacy of SNF472 compared with placebo when added to background care for the treatment of CUA
    • To evaluate the safety and tolerability of SNF472 compared with placebo when added to background care for the treatment of CUA
    - Valutare l’efficacia di SNF472 rispetto a placebo quando aggiunto alla terapia di fondo per il trattamento della CUA
    • Valutare la sicurezza e la tollerabilità di SNF472 rispetto a placebo quando aggiunto alla terapia di fondo per il trattamento della CUA
    E.2.2Secondary objectives of the trial
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. =18 years of age
    2. Receiving maintenance HD in a clinical setting for at least 2 weeks prior to screening
    3. Clinical diagnosis of CUA by the Investigator including =1 CUA lesion with ulceration of
    the epithelial surface. A central wound rating group will review wound images to
    confirm the primary lesion is due to CUA.
    4. CUA wound-related pain shown by a Pain VAS score =50 out of 100
    5. Primary lesion that can be clearly photographed for the purpose of protocol-specified
    wound healing assessments
    6. Willing and able to understand and sign the informed consent form and willing to
    comply with all aspects of the protocol
    1. Età =18 anni
    2. Aver ricevuto HD di mantenimento in un contesto clinico per almeno 2 settimane prima dello screening
    3. Diagnosi clinica di CUA eseguita dallo Sperimentatore, comprese =1 lesioni da CUA con ulcerazione della superficie epiteliale. Un gruppo di valutazione delle ferite a livello centrale esaminerà le immagini delle ferite per confermare che la lesione primaria sia dovuta a CUA.
    4. Dolore correlato alla ferita da CUA dimostrato tramite un punteggio VAS del dolore =50 su 100
    5. Lesione primaria che può essere fotografata chiaramente ai fini delle valutazioni della guarigione della ferita specifiche del protocollo
    6. Soggetto disposto a, e in grado di, comprendere e firmare il modulo di consenso informato, oltre che disposto a rispettare tutti gli aspetti del protocollo
    E.4Principal exclusion criteria
    1. Subjects whose primary lesion is due to causes other than CUA
    2. History of treatment with bisphosphonates within 3 months of baseline (Week 1 Day 1)
    3. Severely ill subjects without a reasonable expectation of survival for at least 6 months based on the assessment of the Investigator
    4. Subjects with a scheduled parathyroidectomy during the study period
    5. Expectation for kidney transplant within the next 6 months based on Investigator assessment or identification of a known living donor
    6. Pregnant or trying to become pregnant, currently breastfeeding, or of childbearing potential (including perimenopausal women who have had a menstrual period within one year) and not willing to either completely avoid sexual intercourse with a person of the opposite sex or use a highly effective method of birth control from screening through at least 30 days after last dose of study drug
    7. Significant noncompliance with dialysis treatment evidenced by repeated missed dialysis sessions (including if due to hospitalizations where dialysis treatment is unavailable) or significant noncompliance with medication regimen, in the judgment of the Investigator
    8. Any history of active malignancy within the last year (history of localized basal cell or squamous cell carcinoma that has been excised/appropriately treated or a fully excised malignant lesion with a low probability of recurrence will not be considered exclusionary)
    9. Clinically significant illness other than CUA within 30 days prior to screening that, in the judgment of the Investigator, could interfere with interpretation of study results, impair compliance with study procedures, or impact the safety of the subject (e.g., unstable angina, unstable heart failure, stroke, uncontrolled hypertension, or other illness requiring hospitalization)
    10. Participation in an investigational study and receipt of an investigational drug or investigational use of a licensed drug (with the exception of intravenous STS) within 30 days prior to screening. If participating in an investigational study of intravenous STS, all visits of that study must be completed prior to screening for this study. Note: Off-label use of intravenous STS outside of an investigational study is not restricted.
    11. Past or current participation in another clinical study with SNF472
    12. History or presence of active alcoholism or drug abuse as determined by the Investigator within 6 months before screening or concurrent social conditions that, in the opinion of the Investigator, would potentially interfere with the subject's study compliance
    13. Mental impairment or history of or current significant psychiatric disease that, in the opinion of the Investigator, may impair ability to provide informed consent or impact compliance with study procedures
    14. Any other condition or circumstance that, in the opinion of the Investigator, may make the subject unlikely to complete the study or comply with study procedures and requirements, or may pose a risk to the subject's safety and well-being
    15. Subjects whose CUA lesions exhibit significant improvement, in the opinion of the Investigator, between the first and second screening visit
    1. Soggetti la cui lesione primaria è dovuta a cause diverse dalla CUA
    2. Anamnesi di trattamento con bisfosfonati entro 3 mesi dal basale (Settimana 1, Giorno 1)
    3. Soggetti affetti da malattia grave, senza una ragionevole aspettativa di sopravvivenza di almeno 6 mesi in base alla valutazione dello Sperimentatore
    4. Soggetti con paratiroidectomia programmata durante il periodo dello studio
    5. Aspettativa di trapianto di rene nei 6 mesi successivi in base alla valutazione dello Sperimentatore o all’individuazione di un donatore vivente noto
    6. Soggetti di sesso femminile in gravidanza, o che stanno tentando una gravidanza, che sono attualmente in fase di allattamento o in età potenzialmente fertile (comprese le donne in premenopausa che hanno avuto un ciclo mestruale in un anno), o che non sono disposti a evitare del tutto i rapporti sessuali con una persona del sesso opposto o ad usare un metodo contraccettivo altamente efficace dallo screening fino ad almeno 30 giorni dopo l’ultima dose di farmaco dello studio
    7. Assenza significativa di aderenza al trattamento di dialisi, evidenziata da ripetute assenze alle sessioni di dialisi (anche se a causa di ricoveri laddove il trattamento di dialisi non fosse disponibile) oppure, secondo il parere dello Sperimentatore, assenza significativa di aderenza al regime posologico del farmaco
    8. Qualsiasi anamnesi di tumore maligno attivo nell’ultimo anno (un’anamnesi di carcinoma basocellulare localizzato o di carcinoma a cellule squamose che è stato escisso/adeguatamente trattato, o di lesione maligna completamente asportata con una bassa probabilità di recidiva, non sarà considerata criterio di esclusione)
    9. Malattia clinicamente significativa diversa da CUA nei 30 giorni precedenti allo screening che, a giudizio dello Sperimentatore, potrebbe interferire con l’interpretazione dei risultati dello studio, compromettere l’aderenza alle procedure dello studio o influire sulla sicurezza del soggetto (ad es. angina instabile, insufficienza cardiaca instabile, ictus, ipertensione non controllata o altra malattia che richiede ricovero)
    10. Partecipazione a uno studio sperimentale e ricezione di un farmaco sperimentale o utilizzo sperimentale di un farmaco approvato (ad eccezione dell’STS somministrato per via endovenosa) nei 30 giorni precedenti lo screening. Se il soggetto sta partecipando a uno studio sperimentale con STS somministrato per via endovenosa, tutte le visite di tale studio dovranno essere completate prima della visita di screening per questo studio. Nota: l’uso off-label di STS somministrato per via endovenosa al di fuori di uno studio sperimentale non è soggetto a limitazioni.
    11. Partecipazione precedente o attuale a un altro studio clinico con SNF472
    12. Anamnesi o presenza di alcolismo attivo o abuso di farmaci, come determinato dallo Sperimentatore, nei 6 mesi prima dello screening o in condizioni sociali concomitanti che, a giudizio dello Sperimentatore, potrebbero potenzialmente interferire con l’aderenza allo studio da parte del soggetto
    13. Compromissione mentale o anamnesi di attuale o pregressa di malattia psichiatrica significativa che, a giudizio dello Sperimentatore, potrebbe compromettere la capacità di fornire il consenso informato o avere un impatto sull’aderenza alle procedure dello studio
    14. Qualsiasi altra condizione o circostanza che, a giudizio dello Sperimentatore, renda improbabile il completamento dello studio da parte del soggetto o l’aderenza di quest’ultimo alle procedure e ai requisiti dello stesso, oppure che potrebbe rappresentare un rischio per la sicurezza e il benessere del soggetto
    15. Soggetti le cui lesioni da CUA presentano un miglioramento significativo, a giudizio dello Sperimentatore, tra la prima e la seconda visita di screening (VS)
    E.5 End points
    E.5.1Primary end point(s)
    Efficacy Endpoints; Applies to part 1.
    These endpoints will compare the placebo and SNF472 groups as follows;

    Alternate Primary Efficacy Endpoints:
    • Absolute change from baseline to Week 12 in the BWAT-CUA score for the primary lesion
    • Absolute change from baseline to Week 12 in Pain VAS score

    Safety Endpoints; Applies throughout part 1 and part 2
    • Proportion of subjects with AEs, SAEs, and deaths
    • Changes from baseline in the following:
    o Laboratory parameters
    o QTc interval and other ECG parameters
    o Holter monitoring results
    o Vital signs
    • Proportion of subjects with a CUA wound-related infection, sepsis, hospitalization, or any CUA wound-related complication
    Endpoint di efficacia:
    Nella Parte 1, gli endpoint di efficacia primari, secondari ed esplorativi confronteranno i gruppi di placebo e di SNF472 così come segue.
    Endpoint primari di efficacia alternativi:
    • Variazione assoluta dal basale alla Settimana 12 nel punteggio BWAT-CUA per la lesione primaria
    • Variazione assoluta dal basale alla Settimana 12 nel punteggio VAS del dolore

    Endpoint secondari di efficacia (valutati in maniera gerarchica):
    • Variazione assoluta dal basale alla Settimana 12 nel punteggio VAS del dolore
    • Variazione assoluta dal basale alla Settimana 12 nel punteggio totale BWAT per la lesione primaria
    • Valutazione qualitativa delle immagini della ferita per la lesione primaria alla Settimana 12 (peggiorata, uguale a o migliorata rispetto al basale)
    • Tasso di variazione nell’uso degli oppiacei misurato in milligrammi equivalenti di morfina (MME), dal basale alla Settimana 12
    E.5.1.1Timepoint(s) of evaluation of this end point
    Efficacy (Part 1): Various timepoints from baseline to week 12
    Efficacia (parte 1): vari timepoint dal basale alla settimana 12
    E.5.2Secondary end point(s)
    Secondary Efficacy Endpoints (assessed hierarchically):
    • Absolute change from baseline to Week 12 in the Wound-QoL score
    • Absolute change from baseline to Week 12 in the BWAT total score for the primary lesion
    • Qualitative wound image evaluation for the primary lesion (worsened, equal to, or improved relative to baseline) at Week 12
    • Rate of change in opioid use as measured in morphine milligram equivalents (MME) from baseline to Week 12
    Endpoint secondari di efficacia (valutati in maniera gerarchica):
    • Variazione assoluta dal basale alla Settimana 12 nel punteggio VAS del dolore
    • Variazione assoluta dal basale alla Settimana 12 nel punteggio totale BWAT per la lesione primaria
    • Valutazione qualitativa delle immagini della ferita per la lesione primaria alla Settimana 12 (peggiorata, uguale a o migliorata rispetto al basale)
    • Tasso di variazione nell’uso degli oppiacei misurato in milligrammi equivalenti di morfina (MME), dal basale alla Settimana 12
    E.5.2.1Timepoint(s) of evaluation of this end point
    Various timepoints from baseline to week 12
    vari timepoints dal basale alla settimana 12
    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 No
    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. trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    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 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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA16
    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 Information not present in EudraCT
    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
    United Kingdom
    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
    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 months3
    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 months3
    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: 36
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 30
    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 No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 20
    F.4.2.2In the whole clinical trial 66
    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)
    Follow-Up Period/ Early Termination: Subjects completing the study will return for a follow-up visit 4 weeks after the last dose of study drug. Subjects should continue on stable background care during the 4-week follow-up period, including pain medication, with no changes unless medically indicated in the opinion of the Investigator.
    Periodo di follow-up / interruzione anticipata: i soggetti che completano lo studio torneranno per una visita di follow-up 4 settimane dopo l'ultima dose del farmaco in studio. I soggetti devono continuare con un'assistenza di base stabile durante il periodo di follow-up di 4 settimane, inclusi i farmaci antidolorifici, senza modifiche a meno che non sia indicato dal punto di vista medico secondo l'opinione dello sperimentatore.
    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 Decision2020-11-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-10-21
    P. End of Trial
    P.End of Trial StatusOngoing
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