Clinical Trials List
2019-10-30 - 2027-10-27
Phase III
Not yet recruiting1
Recruiting3
Terminated3
ICD-10D68.1
Hereditary factor XI deficiency
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
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Trial Applicant
PPD DEVELOPMENT (HK) LIMITED
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Sponsor
Genzyme Corporation
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Trial scale
Multi-Regional Multi-Center
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Update
2025/08/20
Investigators and Locations
The Actual Total Number of Participants Enrolled
0 Recruiting
The Actual Total Number of Participants Enrolled
0 Recruiting
Co-Principal Investigator
- Yen-Lin Liu Division of Pediatrics
The Actual Total Number of Participants Enrolled
0 Recruiting
Co-Principal Investigator
- Po-Shen Ko Division of Hematology & Oncology
- 顏秀如 Division of Hematology & Oncology
- Jyh-Pyng Gau Division of Hematology & Oncology
- Giun-Yi Hung Division of Hematology & Oncology
The Actual Total Number of Participants Enrolled
0 Stop recruiting
The Actual Total Number of Participants Enrolled
0 Stop recruiting
Co-Principal Investigator
The Actual Total Number of Participants Enrolled
0 Stop recruiting
Co-Principal Investigator
- Shih-Hsiang Chen Division of Pediatrics
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
Condition/Disease
Objectives
Test Drug
Active Ingredient
Dosage Form
Dosage
Endpoints
Incidence, severity, relatedness, and seriousness of AEs, and
laboratory assessments
Secondary:
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)
Inclution Criteria
I 01. Participant must be at least 12 years of age inclusive, at the time of signing the informed
consent
Type of participant and disease characteristics
I 02. Participants with severe hemophilia A or B who have completed a Phase 3 fitusiran
clinical trial
Sex
I 03. Male
A) There are no contraceptive requirements for this study except where required by local
regulations.
Informed Consent
I 04. Capable of giving signed informed consent as described in Appendix 1 (Section 10.1.3)
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.
Exclusion Criteria
E 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
E 02. Current participation in ITI
E 03. Current use of factor concentrates or BPAs as regularly administered prophylaxis designed
to prevent spontaneous bleeding episodes
E 04. Use of compounds other than factor concentrates or BPAs for hemophilia treatment
Prior/concurrent clinical study experience
E 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
E 06. Current or prior participation in a gene therapy trial
Diagnostic assessments
E 07. ALT and/or AST >1.5× upper limit of normal reference range (ULN) for participants who
are naïve to fitusiran at study start; ALT and/or AST >5× ULN for participants who were
in the fitusiran arm in the parent study (Section 5.4).
Other exclusions
E 08. Individuals accommodated in an institution because of regulatory or legal order; prisoners
or participants who are legally institutionalized
E 09. Any country-related specific regulation that would prevent the participant from entering
the study – see Appendix 8 (Section 10.8) (country-specific requirements)
E 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
E 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)
E 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
E 13. Any specific situation during study implementation/course that may rise ethics
considerations
E 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
The Estimated Number of Participants
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Taiwan
7 participants
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Global
244 participants
