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    Summary
    EudraCT Number:2021-003157-27
    Sponsor's Protocol Code Number:Sobi.PEGCET-201
    National Competent Authority:Greece - EOF
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-10-05
    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 ConcernedGreece - EOF
    A.2EudraCT number2021-003157-27
    A.3Full title of the trial
    An Open-label, Single-arm, Multicenter Pilot Study to Evaluate the Pharmacokinetics, Pharmacodynamics and Safety of Pegcetacoplan in Patients with Transplant-associated Thrombotic Microangiopathy (TA-TMA) After Hematopoietic Stem Cell Transplantation (HSCT)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An Open-label, Single-arm, Study to Evaluate Pegcetacoplan in Patients with TA-TMA after HSCT
    A.4.1Sponsor's protocol code numberSobi.PEGCET-201
    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 SponsorSwedish Orphan Biovitrum AB
    B.1.3.4CountrySweden
    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 supportSwedish Orphan Biovitrum AB
    B.4.2CountrySweden
    B.4.1Name of organisation providing support Apellis Pharmaceuticals, Inc (and subsidiaries).
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSwedish Orphan Biovitrum AB
    B.5.2Functional name of contact pointRegulatory Affairs
    B.5.3 Address:
    B.5.3.1Street AddressTomtebodavägen 23A
    B.5.3.2Town/ citySolna
    B.5.3.3Post codeSE-112 76
    B.5.3.4CountrySweden
    B.5.4Telephone number004179 362 97 99
    B.5.6E-mailAnke.Arnold@sobi.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 namePegcetacoplan
    D.3.2Product code APL-2
    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.3.8INN - Proposed INNpegcetacoplan
    D.3.9.1CAS number 2019171-69-6
    D.3.9.2Current sponsor codeAPL-2
    D.3.9.3Other descriptive nameAPL-2
    D.3.9.4EV Substance CodeSUB192794
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number54
    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.IMP: 2
    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 namePegcetacoplan
    D.3.2Product code APL-2
    D.3.4Pharmaceutical form Solution for injection/infusion
    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 INNpegcetacoplan
    D.3.9.1CAS number 2019171-69-6
    D.3.9.2Current sponsor codeAPL-2
    D.3.9.3Other descriptive nameAPL-2
    D.3.9.4EV Substance CodeSUB192794
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number54
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Transplant-Associated Thrombotic Microangiopathy (TA-TMA)
    E.1.1.1Medical condition in easily understood language
    Inflammatory and thrombotic diseases of the system of tiny blood vessels
    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 PT
    E.1.2Classification code 10043645
    E.1.2Term Thrombotic microangiopathy
    E.1.2System Organ Class 10005329 - Blood and lymphatic system disorders
    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 pharmacokinetics (PK), safety and tolerability of pegcetacoplan in patients with TA-TMA.
    E.2.2Secondary objectives of the trial
    • To evaluate the pharmacodynamics (PD) of pegcetacoplan in patients with TA-TMA.
    • To evaluate the clinical response of pegcetacoplan in patients with TA TMA.
    • To evaluate the overall survival with pegcetacoplan in patients with TA TMA.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male and female patients aged ≥ 18 years at the time of informed consent form (ICF) signature.
    2. Received allogeneic HSCT from a related or unrelated, human leukocyte antigen-matched or mismatched donor. Patients having received any of the following stem cell sources are eligible: granulocyte colony stimulating factor mobilized peripheral blood stem cells, bone marrow, umbilical cord blood.
    3. Diagnosis of TA-TMA established by histologic evidence of microangiopathy in any biopsied organ OR, as per the laboratory markers below, indicating TMA:
    a. De novo or progressing thrombocytopenia (platelet count < 50 x 109/L or > 50 % decrease in platelet count from the highest value achieved after transplantation). AND
    b. Elevated LDH (> 1.5 x ULN).
    AND at least 1 additional laboratory criteria among the following:
    c. Schistocytes on the peripheral blood smear (≥ 2 per hpf). OR
    d. De novo anemia (hemoglobin < LLN or anemia requiring PRBC transfusion support as per local institutional standard). OR
    e. Proteinuria (rUPCR ≥ 2 mg/mg). OR
    f. Elevated plasma concentration of sC5b-9 above ULN.
    4. Have a diagnosis of TA-TMA that persists despite initial management of any triggering condition.
    5. Have at least 1 sign/symptom of organ dysfunction:
    a. Kidney: doubling of serum creatinine compared with pre-HSCT level or patient receiving renal replacement therapy or proteinuria ≥ 30 mg/dL AND rUPCR ≥ 2 mg/mg.
    b. Lungs: hypoxemia or any need for noninvasive or invasive positive pressure ventilation.
    c. Cardiovascular: pulmonary hypertension diagnosed by a cardiologist using cardiac catheterization, or pulmonary hypertension criteria on echocardiography or arterial hypertension, defined by systolic blood pressure (BP) ≥ 140 mmHg and/or diastolic BP ≥ 90 mmHg at baseline or hypertension requiring > 2 medications (excluding diuretics).
    d. Serositis: clinically significant pleural effusion or pericardial effusion requiring surgical therapy (e.g., pericardiocentesis/ thoracocentesis).
    e. CNS: seizures attributable to posterior reversible encephalopathy syndrome.
    f. GI tract: presence of biopsy-proven GI TA-TMA. Patients with GI bleeding (hematemesis or hematochezia) will be excluded.
    6. Women of childbearing potential, defined as any women who have experienced menarche and who are NOT permanently sterile or postmenopausal, must have a negative serum pregnancy test at screening and agree to use protocol-defined methods of contraception for the duration of the study and 8 weeks after their last IMP dose.
    Note: Postmenopausal is defined as having had 12 consecutive months with no menses without an alternative medical cause.
    7. Men must agree to the following for the duration of the study and 8 weeks after their last dose of IMP:
    a. Avoid fathering a child.
    b. Use protocol-defined methods of contraception.
    c. Refrain from donating sperm.
    8. Patient and/or legally authorized representative must be capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF.
    E.4Principal exclusion criteria
    1. Positive direct Coombs test.
    2. Known familial or acquired ADAMTS13 deficiency.
    3. Known Shiga toxin‐related hemolytic uremic syndrome.
    4. Known bone marrow or graft failure.
    5. Diagnosis of disseminated intravascular coagulation.
    6. Diagnosis of veno-occlusive disease (VOD).
    7. Active GI bleeding (hematemesis or hematochezia) at baseline.
    8. Body weight < 30 kg and > 100 kg.
    9. Uncontrolled systemic bacterial or fungal infection, presence or suspicion of sepsis.
    10. Previously or currently treated with a complement inhibitor (approved or investigational).
    11. Pregnancy or breastfeeding.
    12. Positive human immunodeficiency virus antibody at screening or documented in pre-HSCT medical record.
    13. Hepatitis C virus detectable by polymerase chain reaction at screening or documented in pre-HSCT medical record.
    14. Chronic inactive hepatitis B virus with viral loads > 1000 IU/mL (> 5000 copies/mL) at screening or documented in pre-HSCT medical record. Eligible patients who are chronic active carriers (≤ 1000 IU/mL) must receive prophylactic antiviral treatment (e.g., entecavir, tenofovir, lamivudine) according to local country guidelines.
    15. Inability to cooperate with study procedures or any condition that, in the opinion of the investigator, could increase the patient’s risk by participating in the study or confound the outcome of the study.
    E.5 End points
    E.5.1Primary end point(s)
    • Pegcetacoplan PK parameters:
    o AUC0-tau, Cmax, Tmax and Ctrough.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Days 1, 3 and 5, and, from Week 2 onwards, at all other visits
    E.5.2Secondary end point(s)
    • PD endpoints:
    o Absolute levels, change from baseline, and % change from baseline to Week 24 in biomarkers of complement activation: sC5b-9, C3a, C3, Bb, C4a, functional assays for classical and alternative complement pathways.
    • Clinical response at Week 24 from treatment start, defined as improvement in laboratory markers and improvement in clinical status as follows:
    Laboratory markers:
    o Lactate dehydrogenase (LDH) less than 1.5 x upper limit of normal (ULN) and
    o Platelet count ≥ 50 000/ mm3 without transfusion support during the prior 7 days.
    Clinical status: at least 1 of the following (without deterioration in other organs attributable to TA-TMA):
    o Renal response – requires > 40 % reduction in creatinine, or normalization of creatinine, or discontinuation of renal replacement therapy, or reduction of proteinuria < 30 mg/dL or random urine protein/creatinine ratio (rUPCR) < 2 mg/mg.
    o Pulmonary response – requires extubation and discontinuation of positive pressure ventilation.
    o Gastrointestinal (GI) response – applicable only to patients with biopsy proven GI TA-TMA and requires improvement in GI function as determined by the Mount Sinai Acute Graft-versus-host disease International Consortium (MAGIC) criteria (no or intermittent nausea, vomiting or anorexia attributed to TA-TMA for upper GI; stool output/day for lower GI as follows: < 500 mL/day or < 3 episodes/day.
    o Neurological response – requires improvement in reversible neurological conditions (e.g., cessation of seizures or controlled under medication, resolution of mental alteration; residual radiologic signs are acceptable without clinical symptomatology), or stabilization of irreversible. neurological conditions (e.g., stability of neurological deficits following stroke without further deterioration or subsequent strokes).
    o Freedom from transfusion – requires absence of platelet or packed red blood cells (PRBC) transfusions attributed to TA TMA during the prior 7 days (only applicable if patient was undergoing platelet or PRBC transfusion at baseline).
    o Cardiovascular response – requires resolution of pulmonary hypertension (may receive anti-pulmonary hypertension medications if still on maintenance therapy), or hypertension control on no more than 2 medications excluding diuretics (applicable only to patients with severe hypertension at baseline).
    o Serositis response – requires no evidence of clinically significant pericardial or pleural effusion requiring surgical therapy (e.g., pericardiocentesis/ thoracocentesis).
    Patients meeting intercurrent events, including use of prohibited medication or study withdrawal before Week 24 (with the exception of non-TA-TMA related death), will be considered as failures/nonresponders.
    • Overall survival at Day 100 from date of TA-TMA diagnosis.
    E.5.2.1Timepoint(s) of evaluation of this end point
    as per the protocol
    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 No
    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.8.2.4Number of treatment arms in the trial1
    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 EEA8
    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 No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    France
    Greece
    Italy
    Spain
    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
    LVLS
    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 months1
    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 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: 7
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    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
    Unconscious patients unable to provide consent for themselves will be included
    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 6
    F.4.2.2In the whole clinical trial 9
    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)
    None. Expected normal treatment of the condition.
    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 Decision2021-11-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-10-21
    P. End of Trial
    P.End of Trial StatusOngoing
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