A Study of Fitusiran in Severe Hemophilia A and B Patients Previously Receiving Factor or Bypassing Agent Prophylaxis (ATLAS-PPX)
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ClinicalTrials.gov Identifier: NCT03549871 |
Recruitment Status :
Completed
First Posted : June 8, 2018
Last Update Posted : May 12, 2022
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Primary Objective:
To characterize the frequency of bleeding episodes while receiving fitusiran treatment, relative to the frequency of bleeding episodes while receiving factor or bypassing agent (BPA) prophylaxis
Secondary Objectives:
- To characterize the following while receiving fitusiran treatment, relative to receiving factor or BPA prophylaxis:
- the frequency of spontaneous bleeding episodes
- the frequency of joint bleeding episodes
- health related quality of life (HRQOL) in patients ≥17 years of age
- To characterize the frequency of bleeding episodes during the onset and treatment periods in patients receiving fitusiran
- To characterize the safety and tolerability of fitusiran
- Annualized weight-adjusted consumption of factor/BPA
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Hemophilia | Drug: Fitusiran | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 80 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | ATLAS-PPX: an Open-label, Multinational, Switching Study to Describe the Efficacy and Safety of Fitusiran Prophylaxis in Patients With Hemophilia A and B Previously Receiving Factor or Bypassing Agent Prophylaxis. |
Actual Study Start Date : | July 25, 2018 |
Actual Primary Completion Date : | January 20, 2022 |
Actual Study Completion Date : | March 25, 2022 |

Arm | Intervention/treatment |
---|---|
Experimental: Fitusiran
Fitusiran sub-cutaneous injection for 7 months
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Drug: Fitusiran
Pharmaceutical form: solution for injection Route of administration: subcutaneous |
- Annualized bleeding rate (ABR) [ Time Frame: 13 months (6 months in factor/BPA prophylaxis period and fitusiran baseline to end of study which includes one month onset period and 6 months efficacy period) ]Annualized Bleeding Rate (ABR) in the fitusiran efficacy period and the factor or BPA prophylaxis period
- Annualized spontaneous bleeding rate [ Time Frame: 13 months (6 months in factor/BPA prophylaxis period and fitusiran baseline to end of study which includes one month onset period and 6 months efficacy period) ]Annualized spontaneous bleeding rate in the fitusiran efficacy period and the factor or BPA prophylaxis period
- Annualized joint bleeding rate [ Time Frame: 13 months (6 months in factor/BPA prophylaxis period and fitusiran baseline to end of study which includes one month onset period and 6 months efficacy period) ]Annualized joint bleeding rate in the fitusiran efficacy period and the factor or BPA prophylaxis period
- Quality of Life (QOL) as measured by Haem-A-QOL Questionnaire score on a scale of 0-100 with higher scores representing greater impairment [ Time Frame: 7 months ]Change in Haem-A-QOL physical health score and total score in the fitusiran treatment period
- ABR in the onset period [ Time Frame: 1 months ]ABR in the fitusiran onset period
- ABR in the treatment period [ Time Frame: 7 months ]ABR in the fitusiran treatment period
- Annualized weight-adjusted consumption of factor/BPA [ Time Frame: 6 months ]
- Number of patients reported with treatment emergent adverse events [ Time Frame: Up to 19 months (including up to 6 months of Antithrombin (AT) follow-up) ]

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Ages Eligible for Study: | 12 Years and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Males, ≥12 years of age
- Severe hemophilia A or B (as evidenced by a central laboratory measurement at screening or documented medical record evidence of FVIII <1% or FIX level ≤2%)
- A minimum of 2 bleeding episodes requiring BPA treatment within the last 6 months prior to Screening for patients with inhibitory antibodies to factor VIII or factor IX (Cohort A). A minimum of 1 bleeding episode requiring factor treatment within the last 12 months prior to Screening for patients without inhibitory antibodies to factor VIII or factor IX (Cohort B).
- Must meet either the definition of inhibitor or non-inhibitor patient as below:
- Inhibitor:Use of BPAs for prophylaxis and for any bleeding episodes for at least the last 6 months prior to Screening, and meet one of the following Nijmegen-modified Bethesda assay results criteria:
- Inhibitor titer of ≥0.6 BU/mL at Screening, or
- Inhibitor titer of <0.6 BU/mL at Screening with medical record evidence of 2 consecutive titers ≥0.6 BU/mL, or
- Inhibitor titer of <0.6 BU/mL at Screening with medical record evidence of anamnestic response
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The subgroup of patients in Cohort A patients must additionally meet the following criteria to be eligible to start treatment with fitusiran directly after the screening period:
- Hemophilia B with inhibitory antibody to Factor IX as defined above
- Not responding adequately to BPA treatment (historical ABR ≥20) prior to enrollment
- In the opinion of the Investigator, with approval of Sponsor Medical Monitor, 6-month BPA prophylaxis period should be omitted.
- Non-inhibitor:Use of factor concentrates for prophylaxis and for any bleeding episodes for at least the last 6 months prior to Screening, and meet each of the following criterion:
- Nijmegen-modified Bethesda assay inhibitor titer of <0.6 BU/mL at Screening and
- No use of bypassing agents to treat bleeding episodes for at least the last 6 months prior to Screening and
- No history of immune tolerance induction therapy within the past 3 years prior to Screening.
- Documented prophylactic treatment with factor concentrates or bypassing agents for the treatment of hemophilia A or B for at least 6 months prior to Screening
- Adherent to the prescribed prophylactic therapy for at least 6 months prior to Screening per Investigator assessment
- Willing and able to comply with the study requirements and to provide written informed consent and assent
Exclusion Criteria:
- Known co-existing bleeding disorders other than hemophilia A or B
- AT activity <60% at Screening
- Co-existing thrombophilic disorder
- Clinically significant liver disease
- Active Hepatitis C virus infection
- Acute or chronic Hepatitis B virus infection
- HIV positive with a CD4 count of <200 cells/μL
- History of arterial or venous thromboembolism
- Inadequate renal function
- History of multiple drug allergies or history of allergic reaction to an oligonucleotide or N-Acetylgalactosamine (GalNAc)
- History of intolerance to SC injection(s)
- Any other conditions or comorbidities that would make the patient unsuitable for enrollment or could interfere with participation in or completion of the study, per Investigator judgment

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03549871

Study Director: | Clinical Sciences & Operations | Sanofi |
Responsible Party: | Genzyme, a Sanofi Company |
ClinicalTrials.gov Identifier: | NCT03549871 |
Other Study ID Numbers: |
EFC15110 2016-004087-19 ( EudraCT Number ) ALN-AT3SC-009 ( Other Identifier: Alnylam ) U1111-1217-3270 ( Registry Identifier: WHO Universal Trial Number (UTN) ) |
First Posted: | June 8, 2018 Key Record Dates |
Last Update Posted: | May 12, 2022 |
Last Verified: | April 2022 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Yes |
Plan Description: | Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Product Manufactured in and Exported from the U.S.: | Yes |
Hemophilia A Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hematologic Diseases |
Coagulation Protein Disorders Hemorrhagic Disorders Genetic Diseases, Inborn |