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A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors

Primary Purpose

Hemophilia A, Hemophilia B

Status

Completed

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

fitusiran

factor concentrates

About this trial

This is an interventional treatment trial for Hemophilia A focused on measuring RNAi therapeutic, Hemophilia A, Hemophilia B, Hemophilia A, Severe, Hemophilia B, Severe, Blood Coagulation Disorders, Inherited, Blood Coagulation Disorders, Hematologic Diseases, Coagulation Protein Disorders, Hemorrhagic Disorders, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, Factor VIII, Factor IX, Coagulants, Fitusiran

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

Inclusion Criteria:

Males, >=12 years of age.
Severe hemophilia A or B without inhibitors.
- Severity confirmed by a central laboratory where FVIII level was less than (<) 1 percent (%) or Factor IX (FIX) level was less than or equal to (<=) 2% at Screening.
- On-demand use of factor concentrate to manage 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 Bethesda units per milliliter (BU/mL) at Screening.
  - No use of Bypassing agents to treat bleeding episodes for at least the last 6 months prior to Screening.
  - No history of immune tolerance induction therapy within the last 3 years prior to Screening.
A minimum of 6 bleeding episodes requiring factor concentrate treatment within the last 6 months prior to Screening.
Willing and complied 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, i.e., Von Willebrand's disease, additional factor deficiencies, or platelet disorders.
Antithrombin (AT) activity <60% at Screening.
Co-existing thrombophilic disorder.
Clinically significant liver disease.
Active hepatitis C virus infection.
HIV positive with a cluster of differentiation-4 count of <200 cells/microliter.
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 participant unsuitable for enrollment or could interfere with participation in or completion of the study, per Investigator judgment.

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Arm Label

Factor On-demand

Fitusiran 80 mg Prophylaxis

Arm Description

Participants received on-demand factor concentrates (as needed, for episodic bleeding episodes, and not on a regular regimen intended to prevent spontaneous bleeding) per Investigator discretion for the treatment of breakthrough bleeding episodes from Day 1 up to a total of 9 months.

Participants received open-label fitusiran 80 milligram (mg) administered subcutaneously (SC) as prophylaxis once monthly from Day 1 up to a total of 9 months. Participants received on-demand factor concentrates (per investigator's discretion and within bleeding dosing guidelines) for the treatment of breakthrough bleeding episodes.

Outcomes

Primary Outcome Measures

Estimated Annualized Bleeding Rate (ABR) for Treated Bleeds During the Efficacy Period

ABR for a participant during efficacy period (EP) was defined as annualized number of bleeding episodes during EP annualized to a 1-year interval of time. Treated Bleeding episode: any occurrence of hemorrhage that required administration of by-passing agents (BPA) or factor. It started from first sign of a bleed and ended no more than 72 hours after last treatment for bleed, within which any symptoms of bleeding at same location or injections less than or equal to (<=) 72 hours apart were considered the same bleeding episode. EP was defined as time duration starting from Day 29 when antithrombin (AT) lowering capacity of fitusiran had achieved therapeutic target range to the earliest of Day 246 or last day of bleeding follow up (maximum duration of EP: from Day 29 to Day 246). This outcome measure (OM) presents estimated results (i.e., results received by applying negative binomial [NB] regression model on data collected during EP).

Observed Annualized Bleeding Rate (ABR) for Treated Bleeds During the Efficacy Period

ABR for a participant during EP was defined as annualized number of bleeding episodes during EP annualized to a 1-year interval of time. ABR= number of bleeding episodes during EP divided by total number of days during EP*365.25. A bleeding episode was defined as any occurrence of hemorrhage that required administration of BPA or factor. It started from the first sign of a bleed and ended no more than 72 hours after the last treatment for the bleed, within which any symptoms of bleeding at the same location or injections <=72 hours apart were considered the same bleeding episode. EP was defined as time duration starting from Day 29 when the AT lowering capacity of fitusiran had achieved therapeutic target range to the earliest of Day 246 or the last day of bleeding follow up (maximum duration of EP: from Day 29 to Day 246). This OM presents observed results (i.e., descriptive statistics values based on the data collected during EP).

Secondary Outcome Measures

Estimated Annualized Bleeding Rate (ABR) for Treated Bleeds During the Treatment Period

ABR for a participant during treatment period (TP) was defined as annualized number of bleeding episodes during TP annualized to a 1-year interval of time. Bleeding episode: any occurrence of hemorrhage that required administration of BPA or factor. It started from first sign of a bleed and ended no more than 72 hours after last treatment for bleed, within which any symptoms of bleeding at same location or injections <=72 hours apart were considered the same bleeding episode. TP was sum of onset period (first 28 days after first dose of fitusiran) and EP (starting on Day 29 when AT lowering capacity of fitusiran had achieved therapeutic target range to earliest of Day 246 or last day of bleeding follow up (maximum duration of TP: from Day 1 to Day 246). This OM presents estimated results (i.e., results received by applying NB regression model on data collected during TP).

Observed Annualized Bleeding Rate (ABR) for Treated Bleeds During the Treatment Period

ABR for a participant during TP was defined as annualized number of bleeding episodes during TP annualized to a 1- year interval of time. ABR= number of bleeding episodes during TP divided by total number of days during TP*365.25. Bleeding episode: any occurrence of hemorrhage that required administration of BPA or factor. It started from the first sign of a bleed and ended no more than 72 hours after last treatment for bleed, within which any symptoms of bleeding at the same location or injections <=72 hours apart were considered the same bleeding episode. TP was sum of onset period (first 28 days after the first dose of fitusiran) and EP (starting on Day 29 when AT lowering capacity of fitusiran had achieved therapeutic target range to the earliest of Day 246 or the last day of bleeding follow up (maximum duration of TP: from Day 1 to Day 246). This OM presents observed results (i.e., descriptive statistics values based on data collected during TP).

Estimated Annualized Spontaneous Bleeding Rate for Treated Bleeds During the Efficacy Period

Annualized spontaneous bleeding rate for a participant during EP was defined as annualized number of spontaneous bleeding episodes during EP annualized to a 1-year interval of time. Spontaneous bleeding episode was bleeding event that occurred for no apparent or known reason, particularly into joints, muscles, and soft tissues. It started from first sign of a bleed and ended no more than 72 hours after last treatment for the bleed, within which any symptoms of bleeding at the same location or injections <=72 hours apart were considered the same bleeding episode. EP was defined as time duration starting from Day 29 when AT lowering capacity of fitusiran had achieved therapeutic target range to the earliest of Day 246 or last day of bleeding follow up (maximum duration of EP: from Day 29 to Day 246). This OM presents estimated results (i.e., results received by applying NB regression model on data collected during EP).

Observed Annualized Spontaneous Bleeding Rate for Treated Bleeds During the Efficacy Period

ABR for participant during EP was defined as annualized number of spontaneous bleeding episodes during EP annualized to a 1-year interval of time. ABR=number of treated spontaneous bleeding episodes during EP divided by total number of days during EP*365.25. Spontaneous bleeding episode was bleeding event that occurred for no apparent or known reason, into joints, muscles, and soft tissues. It started from the first sign of a bleed and ended no more than 72 hours after the last treatment for the bleed, within which any symptoms of bleeding at the same location or injections <=72 hours apart were considered the same bleeding episode. EP: time duration starting from Day 29 when the AT lowering capacity of fitusiran had achieved therapeutic target range to earliest of Day 246 or the last day of bleeding follow up (maximum duration of EP: from Day 29 to Day 246).This OM presents observed results (i.e., descriptive statistics values based on data collected during EP).

Estimated Annualized Joint Bleeding Rate for Treated Bleeds During the Efficacy Period

Annualized joint bleeding rate for a participant during EP was defined as annualized number of bleeding episodes during EP annualized to a 1-year interval of time. Joint bleeding episode was characterized by an unusual sensation in the joint ("aura") in combination with 1) increasing swelling or warmth over the skin, joint, 2) increasing pain, or 3) progressive loss of range of motion or difficulty in using the limb as compared with Baseline. It started from first sign of a bleed and ended no more than 72 hours after last treatment for the bleed. EP was defined as time duration starting from Day 29 when the AT lowering capacity of fitusiran had achieved therapeutic target range to the earliest of Day 246 or the last day of bleeding follow up (maximum duration of EP: from Day 29 to Day 246). This OM presents estimated results (i.e., results received by applying NB regression model on data collected during EP).

Observed Annualized Joint Bleeding Rate for Treated Bleeds During the Efficacy Period

Annualized joint bleeding rate for participant during EP was defined as annualized number of joint bleeding episodes during EP annualized to 1-year interval of time. ABR= number of treated joint bleeding episodes during EP divided by total number of days during EP*365.25. A joint bleeding episode was characterized by an unusual sensation in joint ("aura") in combination with 1) increasing swelling or warmth over skin, joint, 2) increasing pain, or 3) progressive loss of range of motion or difficulty in using limb as compared with Baseline. It started from first sign of a bleed and ended no more than 72 hours after the last treatment for the bleed. EP: time duration starting from Day 29 when the AT lowering capacity of fitusiran had achieved therapeutic target range to the earliest of Day 246 or last day of bleeding follow up (maximum duration of EP: from Day 29 to Day 246). This OM presents observed results (i.e., descriptive statistics values based on data collected during EP).

Health-related Quality of Life (HRQOL): Change From Baseline in Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QOL) Physical Health Score at Month 9

Haem-A-QoL: participant-reported questionnaire designed for adult participants (>=17 years of age) with hemophilia; and consisted of 46 items comprising 10 domains (physical health, feelings, view of yourself, sports and leisure, work and school, dealing with hemophilia, treatment, future, family planning, partnership and sexuality). Scoring for each item was based on a 5-point Likert scale (1= never, 2= rarely, 3= sometimes, 4= often, and 5=all the time), and higher scores represented greater impairment. Change from baseline in physical health domain score was reported in this outcome measure. Raw score for physical health domain were transformed to a scale ranged from 0 (better health outcome) to 100 (worst health outcome), where lower scores denoted better physical health.

Health-related Quality of Life (HRQOL): Change From Baseline in Haem-A-QOL Total Score at Month 9

Haem-A-QoL: participant-reported questionnaire designed for adult participants (>=17 years of age) with hemophilia; and consisted of 46 items comprising 10 domains (physical health, feelings, view of yourself, sports and leisure, work and school, dealing with hemophilia, treatment, future, family planning, partnership and sexuality). Scoring for each item was based on a 5-point Likert scale (1= never, 2= rarely, 3= sometimes, 4= often, and 5=all the time), and higher scores represent greater impairment. Raw score for each domain were transformed to a scale ranged between 0 and 100, where lower scores denoted better health. Haem-A-QoL total score was average of all domain scores and ranged from 0 (better health outcome) to 100 (worst health outcome), where lower scores denoted better physical health.

Estimated Annualized Bleeding Rate (ABR) for Treated Bleeds During the Onset Period

ABR was annualized number of bleeding episodes during onset period per participant annualized to a 1-year interval of time. A treated bleeding episode was defined as any occurrence of hemorrhage that required administration of BPA or factor. It started from the first sign of a bleed and ended no more than 72 hours after the last treatment for the bleed, within which any symptoms of bleeding at the same location or injections <=72 hours apart were considered the same bleeding episode. The onset period was defined as time interval from Day 1 to the earlier of Day 28 or the last day of bleeding follow up. This OM presents estimated results (i.e., results received by applying NB regression model on data collected during onset period).

Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs)

An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug which did not necessarily have a causal relationship with the treatment. Treatment-emergent AEs were defined as any AE with onset date after first dose of fitusiran in the fitusiran prophylaxis arm or after Day 1 visit in the factor on-demand arm. A serious AE (SAE) was defined as any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability or incapacity, was a congenital anomaly or birth defect, or was a medically important event.

Full Information

NCT ID

NCT03417245

First Posted

January 25, 2018

Last Updated

March 15, 2022

Sponsor

Genzyme, a Sanofi Company

1. Study Identification

Unique Protocol Identification Number

NCT03417245

Brief Title

A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors

Official Title

ATLAS-A/B: A Phase 3 Study to Evaluate the Efficacy and Safety of Fitusiran in Patients With Hemophilia A or B, Without Inhibitory Antibodies to Factor VIII or IX

Study Type

Interventional

2. Study Status

Record Verification Date

March 2022

Overall Recruitment Status

Completed

Study Start Date

March 1, 2018 (Actual)

Primary Completion Date

January 26, 2021 (Actual)

Study Completion Date

July 14, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Genzyme, a Sanofi Company

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

Primary Objective: -To evaluate the efficacy of fitusiran compared to on-demand treatment with factor concentrates, as determined by the frequency of bleeding episodes. Secondary Objectives: To evaluate the efficacy of fitusiran compared to on-demand treatment with factor concentrates, as determined by: The frequency of spontaneous bleeding episodes. The frequency of joint bleeding episodes. Health-related quality of life (HRQOL) in participants >=17 years of age. To determine the frequency of bleeding episodes during the onset period. To determine the safety and tolerability of fitusiran.

Detailed Description

The duration of treatment with fitusiran was 9 months. The estimated total time on study, inclusive of screening, was up to 11 months for all participants in the factor on-demand arm and for participants in the fitusiran arm who enrolled in the extension study (LTE15174). The estimated total time on the study was up to 17 months for participants in the fitusiran treatment arm who did not enroll in the extension study due to the requirement for up to an additional 6 months of follow-up monitoring for antithrombin levels. Participants who completed the study will be eligible for an open-label extension study LTE15174 (NCT03754790).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Hemophilia A, Hemophilia B

Keywords

RNAi therapeutic, Hemophilia A, Hemophilia B, Hemophilia A, Severe, Hemophilia B, Severe, Blood Coagulation Disorders, Inherited, Blood Coagulation Disorders, Hematologic Diseases, Coagulation Protein Disorders, Hemorrhagic Disorders, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, Factor VIII, Factor IX, Coagulants, Fitusiran

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Randomized

Enrollment

120 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Factor On-demand

Arm Type

Experimental

Arm Description

Arm Title

Fitusiran 80 mg Prophylaxis

Arm Type

Experimental

Arm Description

Intervention Type

Drug

Intervention Name(s)

fitusiran

Intervention Description

by SC injection

Intervention Type

Drug

Intervention Name(s)

factor concentrates

Intervention Description

by intravenous (IV) injection

Primary Outcome Measure Information:

Title

Estimated Annualized Bleeding Rate (ABR) for Treated Bleeds During the Efficacy Period

Description

Time Frame

From Day 29 up to Day 246 or up to the last day of bleeding follow up (any day up to Day 246), whichever was the earliest

Title

Observed Annualized Bleeding Rate (ABR) for Treated Bleeds During the Efficacy Period

Description

Time Frame

From Day 29 up to Day 246 or up to the last day of bleeding follow up (any day up to Day 246), whichever was the earliest

Secondary Outcome Measure Information:

Title

Estimated Annualized Bleeding Rate (ABR) for Treated Bleeds During the Treatment Period

Description

Time Frame

From Day 1 up to Day 246 or up to the last day of bleeding follow up (any day up to Day 246), whichever was the earliest

Title

Observed Annualized Bleeding Rate (ABR) for Treated Bleeds During the Treatment Period

Description

Time Frame

From Day 1 up to Day 246 or up to the last day of bleeding follow up (any day up to Day 246), whichever was the earliest

Title

Estimated Annualized Spontaneous Bleeding Rate for Treated Bleeds During the Efficacy Period

Description

Time Frame

From Day 29 up to Day 246 or up to the last day of bleeding follow up (any day up to Day 246), whichever was the earliest

Title

Observed Annualized Spontaneous Bleeding Rate for Treated Bleeds During the Efficacy Period

Description

Time Frame

From Day 29 up to Day 246 or up to the last day of bleeding follow up (any day up to Day 246), whichever was the earliest

Title

Estimated Annualized Joint Bleeding Rate for Treated Bleeds During the Efficacy Period

Description

Time Frame

From Day 29 up to Day 246 or up to the last day of bleeding follow up (any day up to Day 246), whichever was the earliest

Title

Observed Annualized Joint Bleeding Rate for Treated Bleeds During the Efficacy Period

Description

Time Frame

From Day 29 up to Day 246 or up to the last day of bleeding follow up (any day up to Day 246), whichever was the earliest

Title

Health-related Quality of Life (HRQOL): Change From Baseline in Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QOL) Physical Health Score at Month 9

Description

Time Frame

Baseline (Day 1), Month 9

Title

Health-related Quality of Life (HRQOL): Change From Baseline in Haem-A-QOL Total Score at Month 9

Description

Haem-A-QoL: participant-reported questionnaire designed for adult participants (>=17 years of age) with hemophilia; and consisted of 46 items comprising 10 domains (physical health, feelings, view of yourself, sports and leisure, work and school, dealing with hemophilia, treatment, future, family planning, partnership and sexuality). Scoring for each item was based on a 5-point Likert scale (1= never, 2= rarely, 3= sometimes, 4= often, and 5=all the time), and higher scores represent greater impairment. Raw score for each domain were transformed to a scale ranged between 0 and 100, where lower scores denoted better health. Haem-A-QoL total score was average of all domain scores and ranged from 0 (better health outcome) to 100 (worst health outcome), where lower scores denoted better physical health.

Time Frame

Baseline (Day 1), Month 9

Title

Estimated Annualized Bleeding Rate (ABR) for Treated Bleeds During the Onset Period

Description

Time Frame

From Day 1 up to Day 28 or up to the last day of bleeding follow up (any day up to Day 28), whichever was the earliest

Title

Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs)

Description

Time Frame

From Baseline (Day 1) up to 15 months (i.e., 9 months treatment period + 6-month follow-up)

10. Eligibility

Sex

Male

Minimum Age & Unit of Time

12 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Males, >=12 years of age. Severe hemophilia A or B without inhibitors. Severity confirmed by a central laboratory where FVIII level was less than (<) 1 percent (%) or Factor IX (FIX) level was less than or equal to (<=) 2% at Screening. On-demand use of factor concentrate to manage 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 Bethesda units per milliliter (BU/mL) at Screening. No use of Bypassing agents to treat bleeding episodes for at least the last 6 months prior to Screening. No history of immune tolerance induction therapy within the last 3 years prior to Screening. A minimum of 6 bleeding episodes requiring factor concentrate treatment within the last 6 months prior to Screening. Willing and complied 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, i.e., Von Willebrand's disease, additional factor deficiencies, or platelet disorders. Antithrombin (AT) activity <60% at Screening. Co-existing thrombophilic disorder. Clinically significant liver disease. Active hepatitis C virus infection. HIV positive with a cluster of differentiation-4 count of <200 cells/microliter. 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 participant unsuitable for enrollment or could interfere with participation in or completion of the study, per Investigator judgment.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Clinical Sciences & Operations, MD

Organizational Affiliation

Sanofi

Official's Role

Study Director

Facility Information:

Facility Name

Investigational Site Number 0140

City

Little Rock

State/Province

Arkansas

ZIP/Postal Code

72202

Country

United States

Facility Name

Investigational Site Number 128

City

Gainesville

State/Province

Florida

ZIP/Postal Code

32610

Country

United States

Facility Name

Investigational Site Number 103

City

Tampa

State/Province

Florida

ZIP/Postal Code

33607

Country

United States

Facility Name

Investigational Site Number 102

City

Chicago

State/Province

Illinois

ZIP/Postal Code

60612-3833

Country

United States

Facility Name

Investigational Site Number 119

City

New Orleans

State/Province

Louisiana

ZIP/Postal Code

70112

Country

United States

Facility Name

Investigational Site Number 125

City

Ann Arbor

State/Province

Michigan

ZIP/Postal Code

48109

Country

United States

Facility Name

Investigational Site Number 111

City

Las Vegas

State/Province

Nevada

ZIP/Postal Code

89135

Country

United States

Facility Name

Investigational Site Number 110

City

Akron

State/Province

Ohio

ZIP/Postal Code

44308

Country

United States

Facility Name

Investigational Site Number 6101

City

Camperdown

ZIP/Postal Code

2050

Country

Australia

Facility Name

Investigational Site Number 6103

City

Murdoch

ZIP/Postal Code

6961

Country

Australia

Facility Name

Investigational Site Number 6104

City

Prahran

ZIP/Postal Code

3181

Country

Australia

Facility Name

Investigational Site Number 8604

City

Beijing

ZIP/Postal Code

100045

Country

China

Facility Name

Investigational Site Number 8602

City

Guangzhou

ZIP/Postal Code

510515

Country

China

Facility Name

Investigational Site Number 8605

City

Hangzhou

ZIP/Postal Code

89147

Country

China

Facility Name

Investigational Site Number 8603

City

Shanghai

ZIP/Postal Code

200025

Country

China

Facility Name

Investigational Site Number 8601

City

Tianjin

ZIP/Postal Code

300020

Country

China

Facility Name

Investigational Site Number 4501

City

Copenhagen

ZIP/Postal Code

2100

Country

Denmark

Facility Name

Investigational Site Number 3303

City

Lyon

ZIP/Postal Code

69677

Country

France

Facility Name

Investigational Site Number 3305

City

Paris

ZIP/Postal Code

75015

Country

France

Facility Name

Investigational Site Number 3301

City

Rouen

ZIP/Postal Code

76038

Country

France

Facility Name

Investigational Site Number 4904

City

Berlin

ZIP/Postal Code

10249

Country

Germany

Facility Name

Investigational Site Number 4905

City

Frankfurt Am Main

ZIP/Postal Code

60590

Country

Germany

Facility Name

Investigational Site Number 4906

City

Leipzig

ZIP/Postal Code

4103

Country

Germany

Facility Name

Investigational Site Number 3602

City

Budapest

ZIP/Postal Code

1134

Country

Hungary

Facility Name

Investigational Site Number 9102

City

Bangalore

ZIP/Postal Code

560034

Country

India

Facility Name

Investigational Site Number 9104

City

Jaipur

ZIP/Postal Code

302017

Country

India

Facility Name

Investigational Site Number 9106

City

Lucknow

ZIP/Postal Code

226003

Country

India

Facility Name

Investigational Site Number 9109

City

Mumbai

ZIP/Postal Code

400012

Country

India

Facility Name

Investigational Site Number 9108

City

Mumbai

ZIP/Postal Code

400022

Country

India

Facility Name

Investigational Site Number 9111

City

Mumbai

Country

India

Facility Name

Investigational Site Number 9103

City

Pune

ZIP/Postal Code

411001

Country

India

Facility Name

Investigational Site Number 9105

City

Vellore

ZIP/Postal Code

632004

Country

India

Facility Name

Investigational Site Number 9701

City

Ramat-Gan

ZIP/Postal Code

52621

Country

Israel

Facility Name

Investigational Site Number 3904

City

Padova

ZIP/Postal Code

35128

Country

Italy

Facility Name

Investigational Site Number 8105

City

Isehara

Country

Japan

Facility Name

Investigational Site Number 8104

City

Saitama

Country

Japan

Facility Name

Investigational Site Number 8201

City

Busan

ZIP/Postal Code

602-739

Country

Korea, Republic of

Facility Name

Investigational Site Number 8202

City

Daejeon

ZIP/Postal Code

35233

Country

Korea, Republic of

Facility Name

Investigational Site Number 8204

City

Seoul

ZIP/Postal Code

3722

Country

Korea, Republic of

Facility Name

Investigational Site Number 6004

City

Ampang

ZIP/Postal Code

68000

Country

Malaysia

Facility Name

Investigational Site Number 6002

City

Johor Bahru

ZIP/Postal Code

80100

Country

Malaysia

Facility Name

Investigational Site Number 6003

City

Kota Kinabalu

ZIP/Postal Code

88586

Country

Malaysia

Facility Name

Investigational Site Number 2701

City

Parktown

ZIP/Postal Code

2193

Country

South Africa

Facility Name

Investigational Site Number 2702

City

Port Elizabeth

ZIP/Postal Code

6001

Country

South Africa

Facility Name

Investigational Site Number 3402

City

Madrid

ZIP/Postal Code

28046

Country

Spain

Facility Name

Investigational Site Number 8807

City

Taichung

ZIP/Postal Code

40447

Country

Taiwan

Facility Name

Investigational Site Number 8805

City

Taichung

ZIP/Postal Code

40705

Country

Taiwan

Facility Name

Investigational Site Number 8804

City

Taipei

ZIP/Postal Code

100

Country

Taiwan

Facility Name

Investigational Site Number 8801

City

Taipei

ZIP/Postal Code

110

Country

Taiwan

Facility Name

Investigational Site Number 8808

City

Taoyuan

ZIP/Postal Code

33305

Country

Taiwan

Facility Name

Investigational Site Number 9002

City

Adana

ZIP/Postal Code

?01130

Country

Turkey

Facility Name

Investigational Site Number 9004

City

Antalya

ZIP/Postal Code

07059

Country

Turkey

Facility Name

Investigational Site Number 9008

City

Gaziantep

ZIP/Postal Code

27100

Country

Turkey

Facility Name

Investigational Site Number 9005

City

Istanbul

ZIP/Postal Code

34093

Country

Turkey

Facility Name

Investigational Site Number 9003

City

Izmir

ZIP/Postal Code

TR-35100

Country

Turkey

Facility Name

Investigational Site Number 9009

City

Kayseri

ZIP/Postal Code

38039

Country

Turkey

Facility Name

Investigational Site Number 9006

City

Samsun

ZIP/Postal Code

55200

Country

Turkey

Facility Name

Investigational Site Number 8001

City

Kyiv

ZIP/Postal Code

04060

Country

Ukraine

Facility Name

Investigational Site Number 8003

City

Kyiv

ZIP/Postal Code

?01135

Country

Ukraine

Facility Name

Investigational Site Number 8002

City

Lviv

ZIP/Postal Code

79044

Country

Ukraine

Facility Name

Investigational Site Number 8005

City

Mykolaiv

ZIP/Postal Code

54058

Country

Ukraine

Facility Name

Investigational Site Number 4402

City

Glasgow

ZIP/Postal Code

G4 0SF

Country

United Kingdom

Facility Name

Investigational Site Number 4407

City

London

ZIP/Postal Code

E1 2ES

Country

United Kingdom

Facility Name

Investigational Site Number 4401

City

London

ZIP/Postal Code

SE1 9RT

Country

United Kingdom

12. IPD Sharing Statement

Plan to Share IPD

Yes

IPD Sharing 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

Citations:

PubMed Identifier

34922648

Citation

Del Pozo Martin Y. 2021 ASH annual meeting. Lancet Haematol. 2022 Feb;9(2):e92-e93. doi: 10.1016/S2352-3026(21)00384-7. Epub 2021 Dec 16. No abstract available.

Results Reference

derived

Learn more about this trial

A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors

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A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors

Hemophilia A, Hemophilia B

About this trial

Eligibility Criteria

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

1. Study Identification

2. Study Status

3. Sponsor/Collaborators

4. Oversight

5. Study Description

6. Conditions and Keywords

7. Study Design

8. Arms, Groups, and Interventions

10. Eligibility

12. IPD Sharing Statement

Learn more about this trial