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Summary
EudraCT Number:2018-002880-25
Sponsor's Protocol Code Number:LTE15174
National Competent Authority:Denmark - DHMA
Clinical Trial Type:EEA CTA
Trial Status:Temporarily Halted
Date on which this record was first entered in the EudraCT database:2019-04-26
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
A. Protocol Information
A.1Member State ConcernedDenmark - DHMA
A.2EudraCT number2018-002880-25
A.3Full title of the trial
ATLAS-OLE: An Open-label, Long-term Safety and Efficacy Study of Fitusiran in Patients with Hemophilia A or B, with or without Inhibitory Antibodies to Factor VIII or IX
A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
Long-term Safety and Efficacy Study of Fitusiran in Patients with Hemophilia A or B, with or without Inhibitory Antibodies to Factor VIII or IX
A.3.2Name or abbreviated title of the trial where available
ATLAS-OLE
A.4.1Sponsor's protocol code numberLTE15174
A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1210-0018
A.5.4Other Identifiers
Name:INDNumber:125632
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 SponsorGenzyme 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 supportGenzyme Corporation
B.4.2CountryUnited States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1Name of organisationPPD Global
B.5.2Functional name of contact pointProject Management
B.5.3 Address:
B.5.3.1Street Address929 North Front Street
B.5.3.2Town/ cityWilmington
B.5.3.3Post code28401
B.5.3.4CountryUnited States
B.5.4Telephone number+49 89 578 77 131
B.5.5Fax number+49 89 578 77 400
B.5.6E-mailSaban.Musoski@ppdi.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 Yes
D.2.5.1Orphan drug designation numberEU/3/14/1297; EU/3/14/1298
D.3 Description of the IMP
D.3.1Product nameFitusiran
D.3.2Product code SAR439774
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 INNfitusiran
D.3.9.1CAS number 1609016-97-8
D.3.9.2Current sponsor codeSAR439774
D.3.9.4EV Substance CodeSUB130859
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 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 Yes
D.2.5.1Orphan drug designation numberEU/3/14/1297; EU/3/14/1298
D.3 Description of the IMP
D.3.1Product nameFitusiran
D.3.2Product code SAR439774
D.3.4Pharmaceutical form Solution for injection in pre-filled syringe
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 INNfitusiran
D.3.9.1CAS number 1609016-97-8
D.3.9.2Current sponsor codeSAR439774
D.3.9.4EV Substance CodeSUB130859
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 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
Hemophilia A or Hemophilia B
E.1.1.1Medical condition in easily understood language
Hemophilia is an inherited bleeding disorder in which the blood does not clot normally and can result in internal bleeding into the muscles and joints.
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 10060613
E.1.2Term Hemophilia A (Factor VIII)
E.1.2System Organ Class 100000004850
E.1.2 Medical condition or disease under investigation
E.1.2Version 20.0
E.1.2Level LLT
E.1.2Classification code 10060614
E.1.2Term Hemophilia B (Factor IX)
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 characterize the long-term safety and tolerability of fitusiran.
E.2.2Secondary objectives of the trial
To characterize the long-term efficacy of fitusiran as assessed by the frequency of:
- Bleeding episodes,
- Spontaneous bleeding episodes,
- Target joint bleeding episodes.
To characterize the effects of fitusiran on health-related quality of life measures in participants ≥17 years of age. In Denmark
only in patients ≥ 18 years of age.
E.2.3Trial contains a sub-study No
E.3Principal inclusion criteria
Participants are eligible to be included in the study only if all of the following criteria apply:

Age
01. Participant must be at least 12 years of age inclusive, at the time of signing the informed consent. In Denmark only males ≥ 18 years of age.

Type of participant and disease characteristics
02. Participants with severe hemophilia A or B who have completed a Phase 3 fitusiran clinical trial

Sex
03. Male
A) There are no contraceptive requirements for this study except where required by local regulations.

Informed Consent
04. Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in
this protocol. In countries where legal age of majority is above 18 years, a specific ICF must also be signed by the participant’s legally authorized representative.
E.4Principal exclusion criteria
Participants are excluded from the study if any of the following criteria apply:

Medical conditions
01. Completion of a surgical procedure within 14 days prior to screening, or currently receiving additional factor concentrate or BPA infusion for postoperative hemostasis

Prior/concomitant therapy
02. Current participation in ITI
03. Current use of factor concentrates or BPAs as regularly administered prophylaxis designed to prevent spontaneous bleeding episodes
04. Use of compounds other than factor concentrates or BPAs for hemophilia treatment

Prior/concurrent clinical study experience
05. Received an investigational drug or device, other than fitusiran, within 30 days of anticipated IMP administration or 5 half-lives of the IMP, whichever is longer
06. Current or prior participation in a gene therapy trial

Diagnostic assessments
07. ALT and/or AST >1.5× upper limit of normal reference range (ULN) for patients who are naïve to fitusiran at study start; ALT and/or AST >5× ULN for patients who were in the fitusiran arm in the parent study.

Other exclusions
08. Individuals accommodated in an institution because of regulatory or legal order; prisoners or participants who are legally institutionalized
09. Any country-related specific regulation that would prevent the participant from entering the study
10. Participant not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures
11. Participants who are dependent on the Sponsor or Investigator (as defined in section 1.61 of the International Council for Harmonisation (ICH)-Good Clinical Practice (GCP) E6)
12. Participants are employees of the clinical study site or other individuals directly involved in the conduct of the study, or immediate family members of such individuals
13. Any specific situation during study implementation/course that may rise ethics considerations
14. Sensitivity to any of the study interventions, or components thereof, or drug or other allergy that, in the opinion of the Investigator, contraindicates participation in the study
E.5 End points
E.5.1Primary end point(s)
Incidence, severity, relatedness, and seriousness of AEs, and laboratory assessments.
E.5.1.1Timepoint(s) of evaluation of this end point
Through duration of the study (up to 55 months)
E.5.2Secondary end point(s)
Annualized bleeding rate in the treatment period.
Annualized spontaneous bleeding rate in the treatment period.
Annualized joint bleeding rate in the treatment period.
Change in haemophilia quality of life questionnaire for adults physical health score and total score in the treatment period (in participants ≥17 years of age, however in Denmark only in participants ≥18 years of age).
E.5.2.1Timepoint(s) of evaluation of this end point
Through duration of treatment (up to 48 months)
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 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) No
E.7.3Therapeutic confirmatory (Phase III) Yes
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 Yes
E.8.4 The trial involves multiple sites in the Member State concerned Information not present in EudraCT
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 EEA40
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
Australia
Bulgaria
Canada
China
Denmark
France
Germany
Hungary
India
Ireland
Israel
Italy
Japan
Korea, Republic of
Malaysia
Portugal
Russian Federation
South Africa
Spain
Taiwan
Turkey
Ukraine
United Kingdom
United States
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
Last Patient Last Visit (LPLV)
E.8.9 Initial estimate of the duration of the trial
E.8.9.1In the Member State concerned years5
E.8.9.1In the Member State concerned months11
E.8.9.1In the Member State concerned days16
E.8.9.2In all countries concerned by the trial years6
E.8.9.2In all countries concerned by the trial months7
E.8.9.2In all countries concerned by the trial days3
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: 22
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: 22
F.1.2Adults (18-64 years) Yes
F.1.2.1Number of subjects for this age range: 210
F.1.3Elderly (>=65 years) Yes
F.1.3.1Number of subjects for this age range: 12
F.2 Gender
F.2.1Female No
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 state13
F.4.2 For a multinational trial
F.4.2.1In the EEA 100
F.4.2.2In the whole clinical trial 244
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
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 Decision2019-05-14
N.Ethics Committee Opinion of the trial applicationFavourable
N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
N.Date of Ethics Committee Opinion2019-09-02
P. End of Trial
P.End of Trial StatusTemporarily Halted

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