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    Summary
    EudraCT Number:2016-004539-18
    Sponsor's Protocol Code Number:PGCN-001
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2017-07-03
    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 ConcernedFrance - ANSM
    A.2EudraCT number2016-004539-18
    A.3Full title of the trial
    A Phase 2, Open-Label, Multicenter, Efficacy, Safety, Tolerability, and Pharmacokinetic Study of ANF–Rho™ in Patients with Chronic Neutropenia
    Étude de phase 2, en ouvert, multicentrique, portant sur l’efficacité, la sécurité d’emploi, la tolérabilité et la pharmacocinétique d’ANF–Rho™ chez les patients souffrant de neutropénie chronique
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of the efficacy, safety, tolerability and drug blood levlrs of ANF-RHO to treat patients with low white blood cells count
    portant sur l’efficacité, la sécurité d’emploi, la tolérabilité et la pharmacocinétique d’ANF–Rho™ chez les patients souffrant d'une baisse de nombre de globules blanches.
    A.4.1Sponsor's protocol code numberPGCN-001
    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 SponsorProlong Pharmaceuticals, 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 supportProlong Pharmaceuticals, LLC
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCLINACT
    B.5.2Functional name of contact pointHEIDET-PIANELLI Véronique
    B.5.3 Address:
    B.5.3.1Street AddressCLINACT, INOVEL PARC NORD , Vélizy Espace, Bâtiment SANTOS-DUMONT, 13 avenue Morane-Saulnier
    B.5.3.2Town/ cityVélizy-Villacoublay
    B.5.3.3Post code78140
    B.5.3.4CountryFrance
    B.5.4Telephone number0033180 13 14 70
    B.5.5Fax number0033146 23 01 17
    B.5.6E-mailveronique.heidet-pianelli@multihealthgroup.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 nameANF RhoTM (pegfilgrastim ANF)
    D.3.2Product code ANF RhoTM
    D.3.4Pharmaceutical form 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 INNPEGFILGRASTIM
    D.3.9.1CAS number 208265-92-3
    D.3.9.2Current sponsor codeANF-RHO TM
    D.3.9.4EV Substance CodeSUB16451MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2
    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
    Chronic neutropenia
    Neutropénie chronique
    E.1.1.1Medical condition in easily understood language
    chronic decrease of white blood cells
    Baisse chronique des globules blanches
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10066702
    E.1.2Term Chronic neutropenia
    E.1.2System Organ Class 100000013471
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Primary:
    • To assess the response rate to ANF-Rho following a 6-month treatment period (induction and maintenance periods) in patients with Chronic Neutropenia

    o Complete response is defined as the achievement of Median ANC of ≥1.0 x 109/L over 6 months total treatment period

    o Partial response is defined as at least 100% increase in ANC relative to baseline and achievement of Median ANC ≥ 0.5 x 109/L and ANC < 1 x 109/L over 6 months total treatment period
    Évaluer le taux de réponse à ANF-Rho après une période de traitement de 6 mois (périodes d’induction et d’entretien) chez des patients souffrant de neutropénie chronique

    Une réponse complète est définie comme l’obtention d’un ANC médian de ≥ 1,0 x 109/l sur la période de traitement totale de 6 mois

    Une réponse partielle est définie comme une augmentation minimale de 100 % de l’ANC par rapport à la valeur initiale et l’obtention d’un ANC médian≥ 0,5 x 109/l et <1 x 109/l sur la période de traitement totale de 6 mois
    E.2.2Secondary objectives of the trial
    Secondary:
    • To determine the pharmacokinetic profile of ANF-Rho in patients with Chronic Neutropenia (CN)
    • To determine the rate of subject reported infection-related morbidities (infections, hospitalizations and antibiotic use) in CN patients treated with ANF-Rho

    • To evaluate the effect of ANF-Rho on the Quality of Life and Bone Pain in CN patients

    Safety
    • To evaluate safety and tolerability of subcutaneous injections of ANF-Rho in CN patients
    Déterminer le profil pharmacocinétique d’ANF-Rho chez les patients souffrant de neutropénie chronique (NC)

    Déterminer le taux de morbidités liées à une infection signalées par les patients (infections, hospitalisations et usage des antibiotiques) chez les patients souffrant de NC traités par ANF-Rho

    Evaluer l’effet de l’ANF-Rho sur la qualité de vie et la douleur osseuse chez les patients atteints de neutropénie chronique

    Sécurité d’emploi :
    Évaluer la sécurité d’emploi et la tolérabilité des injections sous-cutanées d’ANF-Rho chez les patients souffrant de NC
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1) Patients 12 years of age or older
    2) Patients with established Chronic, cyclic or idiopathic severe neutropenia defined as Median ANC < 0.5 x 109/L at diagnosis (both with and without demonstrated genetic lesion) having an indication for treatment by GCSF. Patients on GCSF & PEG-GCSF treatment are still eligible to participate after a washout period of 7 days (after GCSF) or 14 days after PEG-GSCF, after the stopping of the drug
    3) Signed and dated informed written consent/assent by the patient/parent
    4) Women of childbearing potential with a negative serum pregnancy test and using a reliable method of contraception during the study period. Male study participants also agreeing to use contraception for the study period
    1) Patients âgés de 12 ans ou plus
    2) Patients présentant une neutropénie chronique, cyclique ou idiopathique sévère établie, définie comme un ANC médian
    < 0,5 x 109/l (avec ou sans lésion génétique démontrée) avec une indication de traitement par G-CSF.
    Les patients sous traitement par GCSF ou PEG-GCSF sont éligibles à une participation à l’étude après une période de
    « wash-out » de 7 jours (pour le G-CSF) ou 14 jours (pour le PEG-GCSF), après l’arrêt du médicament
    3) Consentement/assentiment éclairé écrit daté et signé par le patient/parent
    4) Femmes en âge de procréer avec un test de grossesse sérique négatif et utilisant une méthode de contraception fiable pendant la période de l’étude. Les participants de sexe masculin acceptent également d’utiliser une méthode de contraception pendant la période de l’étude
    E.4Principal exclusion criteria
    A subject will not be enrolled if they meet any of the following criteria:
    1) Evidence of chromosomal abnormalities, myelodysplasia, hematologic malignancy, aplastic anemia, systemic lupus erythematosus, or rheumatoid arthritis (Felty’s syndrome) or if the neutropenia was drug-induced
    2) Progressive malignant disease or malignancy history
    3) Presence of macrophage activation syndrome before the diagnosis of neutropenia
    4) Presence of a permanent proteinuria before the diagnosis of neutropenia
    5) Clinical Significant Abnormal Renal, Cardiac, Hepatic Function or Blood Coagulation
    6) Patient has a chronic infection such as hepatitis B virus(HBV), hepatitis C Virus (HCV) or Human immunodeficiency virus( HIV) or history of tuberculosis
    7) Association with anemia <8 g/dL or thrombocytopenia (except iron deficiency anemia inflammatory except Shwachman syndrome) before the diagnosis of neutropenia
    8) Drug abuse, substance abuse, or alcohol abuse
    9) Use of any other investigational drug at the time of enrollment, or within 5 half-lives prior to enrollment, whichever is longer
    10) Patients unwilling and/or who are not capable of ensuring compliance with the provisions of the study protocol
    11) Pregnant or breastfeeding women where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive serum hCG laboratory test
    12) Women of childbearing potential, defined as all women physiologically capable of becoming pregnant, UNLESS they are using a highly effective method of birth control (i.e., one that results in a less than 1% per year failure rate when used consistently and correctly, such as implants, injectables, combined oral contraceptives and intrauterine devices (IUDs)). Periodic abstinence (e.g., calendar, ovulation, symptothermal, post ovulation methods) is not acceptable
    13) For patients with glycogen storage disease and G6PC3 neutropenia, Pulmonary hypertension should be determined by ECHO and if positive patient is not eligible for the study
    Les sujets ne pourront pas être recrutés s’ils remplissent l’un ou l’autre des critères suivants :
    1) Signes d’anomalies chromosomiques, myélodysplasie, tumeur hématologique, anémie aplastique, lupus érythémateux aigu disséminé ou polyarthrite rhumatoïde (syndrome de Felty) ou cas de neutropénie d’origine médicamenteuse
    2) Tumeur maligne évolutive ou antécédents de tumeur maligne
    3) Présence de syndrome d’activation macrophagique avant le diagnostic de neutropénie
    4) Présence d’une protéinurie permanente avant le diagnostic de neutropénie
    5) Fonction rénale, cardiaque ou hépatique, ou coagulation sanguine significativement anormales au plan clinique
    6) Patient présentant une infection chronique telle qu’une infection au virus de l’hépatite B (VHB), au virus de l’hépatite C (VHC) ou au virus de l’immunodéficience humaine (VIH), ou antécédents de tuberculose
    7) Association d’une anémie < 8 g/dl ou d’une thrombopénie (à l’exception d’une anémie ferriprive inflammatoire, sauf syndrome de Shwachman) avant le diagnostic de neutropénie
    8) Toxicomanie, pharmacodépendance ou consommation abusive d’alcool
    9) Utilisation d’un autre médicament expérimental à la date du recrutement, ou dans les 5 demi-vies précédant le recrutement, en fonction de la période la plus longue
    10) Patients non désireux et/ou incapables de garantir le respect des dispositions du protocole de l’étude
    11) Femmes enceintes ou allaitantes, la grossesse étant définie commel’état d’une femme après la conception et jusqu’à la fin de la gestation, confirmé par un test positif de mesure de hCG sérique en laboratoire
    12) Femmes en âge de procréer, définies comme toutes les femmes physiologiquement capables d’être enceintes, SAUF si elles recourent à une méthode de contraception extrêmement efficace (à savoir, une méthode dont le taux d’échec est inférieur à 1 % par an lorsqu’elle est utilisée de façon systématique et correcte, telle que des implants, des contraceptifs injectables, des contraceptifs oraux combinés et des dispositifs intra-utérins [DIU]). L’abstinence périodique (par ex., méthodes ogino, de l’ovulation, symptothermique, post-ovulatoire) n’est pas acceptable
    13) Pour les patients atteints de glycogénose et de neutropénie due à une mutation du gène G6PC3, l’hypertension pulmonaire doit être déterminée par ECHO ; en cas de résultat positif, le patient ne sera pas admissible pour cette étude
    E.5 End points
    E.5.1Primary end point(s)
    Primary Endpoint
    • Response rate to ANF-Rho following a 6-month treatment period in patients with Chronic Neutropenia
    o Complete response: Achievement of Median ANC of
    ≥1.0 x 109/L over 6-month total treatment period
    o Partial response: At least 100% increase in Median ANC relative to baseline ANC value and achievement of Median ANC ≥0.5 x 109/L and < 1 x 109/L over 6 months total treatment period
    Critère d’évaluation principale
    • Taux de réponse à ANF-Rho après une période de traitement de 6 mois chez des patients souffrant de neutropénie chronique
    o Réponse complète : Obtention d’un ANC médian
    ≥ 1,0 x 109/l sur la période de traitement totale de 6 mois
    o Réponse partielle : Augmentation minimale de 100 % de l’ANC médian par rapport à la valeur initiale et obtention d’un ANC médian ≥ 0,5 x 109/l et <1 x 109/l sur la période de traitement totale de 6 mois
    E.5.1.1Timepoint(s) of evaluation of this end point
    6 months
    6 mois
    E.5.2Secondary end point(s)
    Secondary Endpoints
    • Pharmacokinetic profile as determined from blood plasma concentrations over time
    • Type, Incidence and duration of infection-related events, including hospitalization and use of antibiotic medications
    • Quality of life and bone pain measured by SF-36 and bone pain questionnaire
    Safety
    • Changes in vital signs, electrocardiographic assessments, clinical signs, and bio-analytical measures (e.g., blood chemistry, hematology), and reported adverse events
    Critères d’évaluation secondaires
    • Profil pharmacocinétique, déterminé par les concentrations dans le plasma sanguin au fil du temps
    • Type, incidence et durée des événements liés à des infections, notamment l’hospitalisation et l’utilisation d’antibiotiques
    • Qualité de vie et douleur osseuse mesurées par le questionnaire SF-36 et le questionnaire de douleur osseuse

    Sécurité d’emploi
    • Évolution des signes vitaux, évaluations électrocardiographiques, signes cliniques et mesures bio- analytiques (par ex., chimie sanguine, hématologie) et événements indésirables signalés
    E.5.2.1Timepoint(s) of evaluation of this end point
    6 months
    6 mois
    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 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 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.8.3 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 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
    date de la dernière visite de la dernière personne participant à l’essai
    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
    E.8.9.2In all countries concerned by the trial years1
    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: 3
    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: 3
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 6
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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 state10
    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 patient's participation to the trial, the patient will be treated according to the site usual health care procedure
    Une fois que le sujet a terminé sa participation à l’étude, il sera pris en charge par son médecin selon la pratique habituelle.
    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 Decision2017-06-29
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-08-27
    P. End of Trial
    P.End of Trial StatusOngoing
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