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    EudraCT Number:2015-003943-20
    Sponsor's Protocol Code Number:DX-2930-03
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2018-04-20
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2015-003943-20
    A.3Full title of the trial
    HELP Study™: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study to Evaluate DX 2930 For Long-Term Prophylaxis Against Acute Attacks of Hereditary Angioedema (HAE)
    HELP StudyTM: Studio di efficacia e sicurezza multicentrico, randomizzato, in doppio cieco, controllato verso placebo, per la valutazione di DX 2930 nella profilassi a lungo termine contro gli attacchi acuti di angioedema ereditario (AEE)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Efficacy and safety study to evaluate DX-2930 in preventing agioedema attacks in patients with Type I and Type II heriditary angioedema (HAE)
    Studio di efficacia e sicurezza per la valutazione di DX-2930 nella profilassi degli attacchi acuti di angioedema ereditario in pazienti affetti da AEE di tipo I e II.
    A.3.2Name or abbreviated title of the trial where available
    A.4.1Sponsor's protocol code numberDX-2930-03
    A.5.4Other Identifiers
    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 SponsorDYAX CORP.
    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 supportDyax Corp.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationVoisin Consulting
    B.5.2Functional name of contact pointClinical Trial Operations
    B.5.3 Address:
    B.5.3.1Street Address3 rue des Longs Prés
    B.5.3.2Town/ cityBoulogne-Billancourt
    B.5.3.3Post code92100
    B.5.4Telephone number+ 33 1 41 31 83 00
    B.5.5Fax number+33 1 46 20 53 38
    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 numberEU/3/15/1551
    D.3 Description of the IMP
    D.3.1Product nameDX-2930
    D.3.2Product code DX-2930
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMP
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.1CAS number 1426055-14-2
    D.3.9.2Current sponsor codeDX-2930
    D.3.9.4EV Substance CodeSUB130835
    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 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. 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 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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution 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
    Hereditary angioedema (HAE)
    Angioedema ereditario (AAE)
    E.1.1.1Medical condition in easily understood language
    Long-term, debilitating and life-threatening disease that manifests clinically as unpredictable, intermittent attacks of edema of the face, larynx, gastrointestinal tract, limbs and/or genitalia
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10075280
    E.1.2Term Hereditary angioedema attack
    E.1.2System Organ Class 100000004850
    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 DX-2930 in preventing HAE attacks
    Valutare l'efficacia di DX-2930 nella prevenzione degli attacchi di AEE
    E.2.2Secondary objectives of the trial
    To evaluate the safety of repeated subcutaneous administrations of DX-2930
    Valutare la sicurezza di somministrazioni sottocutanee ripetute di DX-2930
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Males and females 12 years of age or older at the time of screening
    2. Documented diagnosis of HAE (Type I or II) based upon all of the following :
    - Documented clinical history consistent with HAE (subcutaneous or mucosal nonpruritic swelling episodes without accompanying urticaria)
    - Diagnostic testing results obtained during screening (or a prior DX-2930 study) that confirm HAE Type I or II :
    C1 inhibitor (C1-INH) functional level < 40% of the normal level.
    Subjects with functional C1-INH level 40-50% of the normal level may be enrolled if they also have a C4 level below the normal range.
    Subjects may begin participating in the run-in period before these diagnostic results are available.
    Subjects may be retested if results are incongruent with clinical history or believed by the Investigator to be confounded by recent LTP use.
    - At least one of the following : age at reported onset of first angioedema symptoms ≤ 30 years, a family history consistent with HAE Type I or II, or C1q within normal range.
    3. Experiencing a baseline rate of at least 1 Investigator-confirmed HAE attack per 4 weeks as confirmed during the run-in period.
    4. Adult subjects and caregivers of subjects under the age of 18 are willing and able to read, understand, and sign an informed consent form. Subjects age to 12 to 17, whose caregiver provides informed consent, are willing and able to read, understand and sign an assent form.
    5. Males and females who are fertile and sexually active must adhere to contraception requirements for the duration of the study as follows:
    - Females of childbearing potential must agree to be abstinent or else use any two of the following medically acceptable forms of contraception from screening through 30 days after the final study visit : progestin-only oral contraceptive, condom with or without spermicidal jelly, diaphragm or cevical cap with spermicidal jelly, or intra-uterine device (IUD, all types). A female whose male partner has had a vasectomy must agree to use one additionnal form of medically acceptable contraception.
    - Females of non-childbearing potential, defined as surgically sterile (status post hysterectomy, bilateral oophorectomy, or bilateral tubal ligation) or post-menopausal for at least 12 months do not require contraception during the study.
    - Males, including males who are surgically sterile (post vasectomy), with female partners of childbearing potential must agree to be abstinent or else use a medically acceptable form of contraception from screening through 60 days after the final study visit.
    1. Soggetti di entrambi i sessi di età pari o superiore ai 12 anni.
    2. Diagnosi documentata di AEE (tipo I o II), basata su tutti i seguenti fattori:
    - anamnesi clinica documentata coerente con AEE (episodi di gonfiore non pruriginoso, sottocutaneo o mucosale, con assenza di orticaria concomitante);
    - risultati dei test diagnostici acquisiti durante lo screening (o un precedente studio DX-2930) che confermano la presenza di AEE di tipo I o II: livello funzionale dell’inibitore della C1 esterasi (C1 INH) < 40% del livello normale. I soggetti con livello funzionale di C1 INH pari al 40 50% del livello normale possono essere arruolati se presentano anche un livello C4 inferiore ai limiti della norma. I soggetti possono iniziare la partecipazione al periodo di run in prima che questi risultati diagnostici siano disponibili. I test possono essere ripetuti nei soggetti se i risultati sono incoerenti con l’anamnesi clinica o se lo Sperimentatore ritiene che sia presente un certo grado di confondimento dovuto all’uso recente della LTP;
    - almeno uno dei seguenti fattori: età ≤ 30 anni al momento della segnalazione dell’esordio dei primi sintomi di angioedema, anamnesi familiare coerente con AEE di tipo I o II o C1q entro i limiti della norma.
    3. Soggetti che presentano un tasso basale di almeno un attacco di AEE confermato dallo Sperimentatore in 4 settimane, come attestato durante il periodo di run in.
    4. Soggetti adulti e caregiver dei soggetti di età inferiore a 18 anni disposti e in grado di leggere, comprendere e firmare una dichiarazione di consenso informato. Soggetti di età compresa tra 12 e 17 anni, i cui caregiver accordano il consenso informato, disposti e in grado di firmare una dichiarazione di consenso informato.
    5. I soggetti maschi e femmine fertili e sessualmente attivi devono rispettare i requisiti di contraccezione per l’intera durata dello studio come indicato di seguito:
    - le donne in età fertile devono accettare di astenersi dai rapporti sessuali, oppure di far uso di due dei seguenti metodi anticoncezionali, accettabili sotto il profilo medico, a partire dallo screening fino a 30 giorni dopo la visita finale dello studio: contraccettivo orale a base di solo progestinico, profilattico con o senza gel spermicida, diaframma o cappuccio cervicale con gel spermicida o dispositivo intrauterino (IUD di qualsiasi tipo); una donna il cui partner è stato sottoposto a vasectomia deve accettare di far uso di un metodo contraccettivo addizionale, accettabile sotto il profilo medico;
    - le donne non potenzialmente fertili, definite come chirurgicamente sterili (status post-isterectomia, ooforectomia bilaterale o legatura tubarica bilaterale) o in postmenopausa da almeno 12 mesi non necessitano di contraccezione durante lo studio;
    - gli uomini, compresi i soggetti chirurgicamente sterili (vasectomizzati) con partner femmine potenzialmente fertili, devono accettare di astenersi dai rapporti sessuali, oppure di far uso di un metodo anticoncezionale, accettabile sotto il profilo medico, a partire dallo screening fino a 60 giorni dopo la visita finale.
    E.4Principal exclusion criteria
    1. Concomitant diagnosis of another form of chronic, recurrent angioedema, such as acquired angioedema (AAE), HAE with normal C1-INH (also known as HAE Type III), idiopathic angioedema, or recurrent angioedema associated with urticaria.
    2. Dosing with an investigational drug or exposure to an investigational device within 4 weeks prior to screening.
    3. Exposure to angiotensin-converting enzyme (ACE) inhibitors or any estrogen-containing medications with systemic absorption (such as oral contraceptives or hormonal replacement therapy) within 4 weeks prior to screening.
    4. Exposure to androgens (e.g. stanozolol, danazol, oxandrolone, methyltestosterone, testosterone) within 2 weeks prior to entering the run-in period.
    5. Use of long-term prophylaxis for HAE (C1-INH, attenuated androgens, anti-fibrinolytics) within 2 weeks prior to entering the run-in period.
    6. Use of short-term prophylaxis for HAE within 7 days prior to entering the run-in period. Short-term prophylaxis is defined as C1-INH, attenuated androgens, or anti-fibrinolytics used to avoid angioedema complications from medically indicated procedures.
    7. Any of the following liver function test abnormalities : alanine aminotransferase (ALT) > 3x upper limit of normal, or aspartate aminotransferase (AST) >3x upper limit of normal, or total bilirubin > 2x upper limit of normal (unless the bilirubin elevation is a result of Gilbert's Syndrome).
    8. Pregnancy or breastfeeding.
    9. Subject has any condition that, in the opinion of the Investigator or Sponsor, may compromise their safety or compliance, preclude successful conduct of the study, or interfere with interpretation of the results (e.g. history of substance abuse or dependence, significant pre-existing illness or other major comorbidity that the Investigator considers may confound the interpretation of study results).

    1. Diagnosi concomitante di un’altra forma di angioedema cronico, recidivante, come angioedema acquisito (AEA), AEE con C1 INH normale (chiamato anche AEE di tipo III), angioedema idiopatico o angioedema recidivante associato a orticaria.
    2. Somministrazione di un farmaco sperimentale o esposizione a un dispositivo sperimentale nelle 4 settimane precedenti allo screening.
    3. Esposizione agli inibitori dell’enzima di conversione dell’angiotensina (ACE) o a qualsiasi medicinale contenente estrogeni con assorbimento sistemico (come contraccettivi orali o terapia ormonale sostitutiva) nelle 4 settimane precedenti allo screening.
    4. Esposizione ad androgeni (ad es. stanozololo, danazolo, oxandrolone, metiltestosterone, testosterone) nelle 2 settimane precedenti all’ammissione nel periodo di run in.
    5. Uso di terapia profilattica a lungo termine contro l’AEE (C1 INH, androgeni attenuati o antifibrinolitici) nelle 2 settimane precedenti all’ammissione nel periodo di run in.
    6. Uso di profilassi a breve termine contro l’AEE nei 7 giorni precedenti all’ammissione nel periodo di run in. Per profilassi a breve termine si intendono C1 INH, androgeni attenuati o antifibrinolitici utilizzati per evitare le complicanze dell’angioedema provocate dalle procedure clinicamente indicate.
    7. Uno qualsiasi dei seguenti risultati anomali dei test di funzionalità epatica: alanina aminotransferasi (ALT) > 3 volte il limite superiore della norma, o aspartato aminotransferasi (AST) > 3 volte il limite superiore della norma, o bilirubina totale > 2 volte il limite superiore della norma (a meno che l’innalzamento dei valori di bilirubina sia una conseguenza della sindrome di Gilbert).
    8. Gravidanza o allattamento.
    9. Soggetti che presentano una qualsiasi patologia che, a giudizio dello Sperimentatore o del Promotore, possa compromettere la loro sicurezza o compliance, precludere il successo nell’esecuzione dello studio o interferire con l’interpretazione dei risultati (ad es. anamnesi di abuso o dipendenza da sostanze, importante malattia preesistente o altra comorbilità maggiore che lo Sperimentatore ritiene possa essere un fattore confondente nell’interpretazione dei risultati dello studio).
    E.5 End points
    E.5.1Primary end point(s)
    Number of HAE attacks per week
    E.5.1.1Timepoint(s) of evaluation of this end point
    Day 14 (Visit 2) to Day 182 (Visit 14)
    E.5.2Secondary end point(s)
    1. Time to first attack, i.e. duration that a subject is attack-free until their first attack
    2. Number per week of HAE attacks requiring acute attack therapy use
    3. Number per week of moderate or severe HAE attacks
    4. Number per week of high-morbidity HAE attacks; a high-morbidity HAE attack is defined as any attack that has at least one of the following characteristics: severe, results in hospitalization (except hospitalization for observation < 24 hours), hemodynamically significant (systolic blood pressure < 90, requires IV hydratation, or associated with syncope or near-syncope) or laryngeal.
    1. Tempo al primo attacco, ossia per quanto tempo il soggetto non accusa attacchi fino al primo attacco.
    2. Numero di attacchi di AEE per settimana che necessitano l’utilizzo di terapia per l’ attacco acuto.
    3. Numero di attacchi di AEE da moderati a severi alla settimana.
    4. Numero di attacchi di AEE ad alta morbilità alla settimana. Per attacco di AEE ad alta morbilità si intende qualsiasi attacco che presenta almeno una delle seguenti caratteristiche: severo, necessitante di ricovero in ospedale (con l’eccezione dei ricoveri per osservazione < 24 ore), emodinamicamente importante (pressione sistolica < 90, che necessita di idratazione e.v. o associato a sincope o presincope) o laringeo.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Day 14 (Visit 2) to Day 182 (Visit 14)
    Day 14 (Visit 2) to Day 182 (Visit 14)
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis Yes
    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 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. 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 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 trial4
    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.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA4
    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
    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
    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 months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months0
    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 Yes
    F.1.1Number of subjects for this age range: 1
    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. of subjects for this age range: 12
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 103
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 5
    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 Yes
    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 state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 33
    F.4.2.2In the whole clinical trial 120
    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)
    Subjects who complete the treatment period will be offered the option of enrolling in the DX-2930-04 Open Label Extension (OLE) study for continued treatment and safety follow-up. Subjects who do not roll over to the OLE study will be followed for an additional 8 weeks (visits 15 and 16).
    Ai soggetti che hanno completato il periodo di trattamento verrà offerta la possibilità di arruolarsi nello studio di estensione in aperto DX-2930-04 (OLE) per la continuazione del trattamento e le valutazioni di sicurezza durante il follow-up. I soggetti che non proseguiranno nello studio OLE saranno seguiti per ulteriori 8 settimane (visite 15 e 16).
    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 Decision2016-01-14
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2017-04-13
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