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    Summary
    EudraCT Number:2013-000144-26
    Sponsor's Protocol Code Number:ABI-007-PST-001
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2017-05-24
    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
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2013-000144-26
    A.3Full title of the trial
    A Phase 1/2, multicenter, open-label, dose-finding study to assess the safety, tolerability, and preliminary efficacy of weekly nab-paclitaxel in pediatric patients with recurrent or refractory
    solid tumors.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    clinical study for pediatric patients with solid tumors that have come back after disappearing or that don't respond to any treatment, designed to find the safest and most tolerable dose of nab®-paclitaxel and to asses the preliminary efficacy of the treatment.
    A.4.1Sponsor's protocol code numberABI-007-PST-001
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/116/2013
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorCelgene Corporation
    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 supportCelgene Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCelgene Corporation
    B.5.2Functional name of contact pointClinicalTrialDisclosure
    B.5.3 Address:
    B.5.3.1Street Address9225 Indian Creek Parkway, Suite 900
    B.5.3.2Town/ cityOverland Park, Kansas
    B.5.3.3Post code66210
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1-888-260-1599
    B.5.5Fax number+1 913-266-0394
    B.5.6E-mailClinicalTrialDisclosure@celgene.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 Yes
    D.2.1.1.1Trade name Abraxane
    D.2.1.1.2Name of the Marketing Authorisation holderCelgene Europe Ltd
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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.3.2Product code nab-paclitaxel, ABI-007
    D.3.4Pharmaceutical form Powder for suspension for injection
    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 INNPACLITAXEL
    D.3.9.1CAS number 33069-62-4
    D.3.9.2Current sponsor codeABI-007
    D.3.9.4EV Substance CodeSUB09583MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    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 Yes
    D.3.11.13.1Other medicinal product typeThe IMP contains an excipient of biological origin, albumin, a non-active stabilising agent. It is derived from human blood subject to approved donor screening and product manufacturing processes.
    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
    recurrent or refractory solid tumor
    E.1.1.1Medical condition in easily understood language
    subjects with solid tumors that have come back after disappearing or that don't respond to any treatment
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10065147
    E.1.2Term Malignant solid tumor
    E.1.2System Organ Class 100000004864
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Phase I:
    determine the pediatric MTD/RP2D, safety and tolerability of nab-paclitaxel administered intravenously over 30min on Days 1, 8, and 15 of a 28-day cycle in patients or = 6 months and < 18 years old with recurrent or refractory solid tumors

    Phase II:
    assess the antitumor activity by the overall response rate (ORR) of nab-paclitaxel given at the RP2D in patients > or = 6 months and < or = 24 years old with several discrete recurrent or refractory solid tumor types stypes including neuroblastoma, rhabdomyosarcoma and Ewing's sarcoma
    E.2.2Secondary objectives of the trial
    Phase I:
    - To evaluate pharmacokinetics (PK).
    - To characterize the ORR

    Phase II:
    - To characterize duration of response (DOR).
    - To characterize the disease control rate (DCR).
    - To characterize progression-free survival (PFS).
    - To characterize 1-year survival.
    - To confirm safety.
    - To evaluate PK.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patient is male or female, meeting the following age requirements at the time the informed consent document (and assent form, if applicable) is signed.
    a. Phase 1: patient is >= 6 months to < 18 years of age
    b. Phase 2: patient is >= 6 months to <= 24 years of age

    2. Patient has a confirmed solid tumor diagnosis according to the following:
    a. Phase 1: patient has a recurrent or refractory solid tumor that has progressed or did not respond to standard therapy, or for which no standard anticancer therapy exists
    b. Phase 2: patient has radiologically documented measurable disease by RECIST 1.1 (for neuroblastoma, evaluable disease by MIBG/Curie score is also acceptable) in one of the following tumor types and has failed up to three lines of treatment
    i. Group 1: neuroblastoma (patients with bone marrow disease only are not permitted)
    ii. Group 2: rhabdomyosarcoma
    iii. Group 3: Ewing's sarcoma

    3. The patient has a Lansky/ Karnofsky performance status score of >= 70%

    4. The patient has adequate serum chemistry levels, evidenced by the following laboratory values
    a. AST (SGOT), ALT (SGPT) <= 2.5 × upper limit of normal range (ULN)
    b. Total bilirubin <= 1.5 × ULN
    c. Creatinine <= 1.5 × ULN

    5. The patient has adequate bone marrow function, evidenced by the following:
    a. Absolute neutrophil count >= 1.0 × 10^9 cells/L
    b. Platelets >= 80 × 10^9 cells/L (transfusion independent, defined as not receiving platelet transfusions within 7 days prior to laboratory sample). In the Phase 2
    portion, for patients with known bone marrow involvement, platelets ? 50 × 109 cells/L.
    c. Hemoglobin >= 8 g/dL (transfusion is permitted to fulfill this criterion)

    6. The patient (when applicable) or patient's parent(s) or legal guardian(s) understand(s) and voluntarily signed an informed consent document prior to any study-related assessments/ procedures being conducted. Where locally applicable, the patient also understands and voluntarily provides his/her assent prior to any study-related assessments/procedures being conducted.

    7. Male patients of childbearing potential must use a condom during sexual intercourse and shall not father a child during the study and for 6 months after the last dose of study medication.

    8. Female patients of childbearing potential [defined as all female patients >= 12 years old or who have reached menarche, whichever occurs first] must have both of the following:
    a. Agree to the use of two physician-approved contraceptive methods simultaneously or practice complete abstinence while on study medication medication or for a longer period if
    required by local regulations
    i. True abstinence: When this is in line with the preferred and usual lifestyle of the patient. Periodic abstinence (e.g., calendar, ovulation, symptothermal, postovulation methods) and withdrawal are not acceptable methods of contraception.
    ii. Acceptable contraceptive methods include: oral, injectable, or implantable hormonal contraceptive; tubal ligation; intra-uterine device; barrier contraceptive with spermicide; or vasectomized partner) including at least one barrier method.

    b. Have negative serum pregnancy test result at screening confirmed by negative urine pregnancy dipstick within 72 hours prior to first dose of IP (if serum test occurred > 72 hours from first dose); pregnancy test with sensitivity of at least 25 mIU/mL
    E.4Principal exclusion criteria
    1. The patient has a primary brain tumor(s) or brain metastasis (unless metastasis is treated and stable for > 28 days). In patients who are symptomatic, a brain scan is required to exclude metastasis.

    2. The patient has >= Grade 2 peripheral neuropathy by National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) at screening.

    3. The patient has received therapeutic dose chemotherapy or radiotherapy ≤ 21 days prior to start of IP.

    4. The patient has received maintenance dose chemotherapy (e.g., low dose cyclophosphamide) <= 7 days from the first dose of IP.

    5. The patient has received any investigational therapy <= 28 days prior to start of IP.
    Investigational therapy is defined as any medicinal product that is not approved in the country of treatment for any indication, adult or pediatric

    6. The patient has received any biological therapy <= 7 days prior to the start of IP, or monoclonal antibody <= 3 half-lives or 28 days, whichever is shorter, prior to the first dose of IP.

    7. The patient has received any allogeneic hematopoietic stem cell transplantation (HSCT) <=3 months or autologous HSCT <= 21 days prior to start IP.

    8. The patient has had major surgery or significant trauma <= 14 days prior to start of IP.

    9. The patient has not recovered from the acute toxic effects of prior chemotherapy, radiation, or major surgery/significant trauma.

    10. The patient has had minor surgery <= 7 days from the start of study treatment (excluding the placement of central/ peripheral lines, skin biopsy).

    11. The patient has a known history of stroke, myocardial infarction, peripheral vascular disease, or recent (within 3 months) uncontrolled deep venous thrombosis.

    12. The patient has a known history or current diagnosis of HIV infection, regardless of treatment status.

    13. The patient has an uncontrolled intercurrent illness including but not limited to ongoing or active infection requiring antibiotic, antifungal, or antiviral therapy, symptomatic heart failure, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.

    14. The patient has any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the patient from participating in the study.

    15. The patient has any condition, including the presence of laboratory abnormalities, that places him/her at unacceptable risk if he/she were to participate in the study.

    16. The patient has any condition that confounds the ability to interpret data from the study.

    17. The patient or parent(s)/guardian(s) is/are unable to comply with the study visit schedule and other protocol requirements, in the opinion of the investigator.
    E.5 End points
    E.5.1Primary end point(s)
    Phase I:
    Incidence of DLTs and the incidence of treatment-emergent adverse events (TEAEs).

    Phase II:
    ORR (CR + PR) confirmed no less than 4 weeks after the criteria for response are first met, based on RECIST version 1.1 criteria. In the
    neuroblastoma group the ORR will be determined by RECIST and/or the Curie scale (MIBG response).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Phase I:
    Initial 1-2 cycles of treatment

    Phase II:
    Expected 12-16 weeks after starting treatment; longer depending on the patient
    E.5.2Secondary end point(s)
    Phase I:
    1) PK parameters including the maximum observed concentration in blood (Cmax), area under the blood concentration-time curve (AUC), clearance and volume of distribution (Vss).
    2) ORR

    Phase II:
    1) DoR in patients with a confirmed objective CR or partial response (PR). DCR is the percentage of patients with a confirmed objective CR or PR, or stable disease for at least 16 weeks.
    2) PFS based on investigator assessment of response using RECIST 1.1 guidelines. In the neuroblastoma group the PFS will be determined by RECIST and/or the Curie scale (MIBG response)
    3) Survival at 1 year
    4) The incidence of TEAEs
    5) Population PK parameters
    E.5.2.1Timepoint(s) of evaluation of this end point
    Phase I:
    1) and 2) Up to 4 days after the first treatment.

    Phase II:
    1)Until progression according to RECIST 1.
    Expected 24 weeks after treatment starts; longer depending on the patient.
    2)Until PFS event
    3)1 Year after starting treatment
    4)Up to 28 days after stopping study treatment
    5)Up to 4 days after the first treatment.
    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 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) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    Phase I / II
    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 Information not present in EudraCT
    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 Yes
    E.8.1.7.1Other trial design description
    phI: rolling-6 dose escalation; phII: treatment with MTD/RP2D
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 Will this trial be conducted at a single site globally? No
    E.8.4 Will this trial be conducted at multiple sites globally? Yes
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    Canada
    France
    Italy
    Spain
    Switzerland
    United Kingdom
    United States
    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
    After the EOT visit, all patients will be followed for survival until death, lost to follow-up, or one year after EOT, whichever occurs first.
    The study will end when all patients have completed the posttreatment follow-up (max 1 year) or have died, whichever occurs first
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months7
    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: 121
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 1
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 60
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 60
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 13
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Minors (< 18 years old) will not sign an informed consent but they will be asked for assent
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 134
    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)
    Patients may remain on treatment until disease progression, unacceptable toxicity, until they begin a new anticancer therapy, withdrawal of consent, parent/guardian/patient refusal, physician decision or death. Upon treatment discontinuation from any of these reasons, patients will be treated at the investigator decision
    G. Investigator Networks to be involved in the Trial
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: United States
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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