A Phase 3 interventional study of fitusiran and Bypassing agents in Hemophilia A and Hemophilia B, sponsored by Genzyme, a Sanofi Company. Completed at 58 sites in 17 countries. Open to male participants aged 12 Years and older. Per ClinicalTrials.gov, last updated 2022-03-28.
Sponsored by Genzyme, a Sanofi Company (part of Sanofi) · Phase 3, Interventional, and Treatment
Among 10 studies testing fitusiran in Hemophilia A — 2 open to participants now, 3 under way, 2 of them phase 3. fitusiran · Hemophilia A.
Among the 10 studies testing fitusiran in Hemophilia A, this one is the 6th largest by enrollment, is one of 3 that started in 2018, is one of 6 in phase 3, and was the 3rd registered.
844 studies on the registry are indexed under Hemophilia A; 138 are open to participants now.
This study's enrollment of 60 is above the median of 30 across 499 interventional studies indexed under Hemophilia A.
Browse Hemophilia A studies →2 of the 10 studies testing fitusiran in Hemophilia A are open now, in 12 countries. Each count lists the studies.
fitusiran: first FDA approval 2025 (QFITLIA). What it is approved for, and how studies give it, is on its page →
Of the 88 Hemophilia A studies recruiting now, 52 (59%) set no upper age limit and 30 (34%) stop at 75 or younger; 54 take children under 18; 10 take healthy volunteers.
This study: 12 Years and older · male · no healthy volunteers.
The full table on the hub →10 studies; the count open now beside each. This study's row is marked.
First in 2014; this study’s year is marked. 3 started in 2018.
Median enrollment 70 across the 10 interventional studies. Of the 7 that completed or were terminated, 4 (57%) have results posted.
10 of 10 have an industry lead sponsor. The 2 most frequent:
Genzyme, a Sanofi Company is the lead sponsor of 308 studies on the registry; 5 are open to participants now.
Of its 25 completed or terminated interventional studies of FDA-regulated products, 19 (76%) have results posted.
Genzyme, a Sanofi Company has 6 studies filed under Hemophilia A — fewer than ten, so there is no comparison to draw.
1 of the studies testing fitusiran in Hemophilia A that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT05662319Phase 3SanofiA Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia | 2027-03-25 |
Among the 10 studies testing fitusiran in Hemophilia A, from their registry updates:
The purpose of this study was to determine the frequency of bleeding episodes in participants receiving fitusiran as prophylactic treatment of hemophilia compared to participants who were assigned to continue with their regular medication. In addition, the study assessed safety, quality of life, pharmacodynamics (PD), and pharmacokinetics (PK).
The duration of treatment with fitusiran was 9 months. The estimated total time on study, inclusive of screening, for each participant was up to 11 months for all participants who enroll in the extension study and participants in the on-demand arm who did not enroll in the extension study. The estimated total time on study was up to 17 months in fitusiran treatment arm participants 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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Severe hemophilia A or B with inhibitors.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants received On-demand BPAs (use of these agents, as needed, for episodic bleeding episodes, and not on a regular regimen intended to prevent spontaneous bleeding) per Investigator discretion from Day 1 for treatment of breakthrough bleeding episodes, up to a total of 9 months.
Drug: Bypassing agents
Participants received Fitusiran 80 mg subcutaneously (SC) as prophylaxis once monthly from Day 1, along with the on-demand BPAs (per investigator's discretion and within bleeding dosing guidelines) for treatment of breakthrough bleeding episodes, up to a total of 9 months.
Drug: fitusiran
solution for injection; by subcutaneous (SC) injection
solution for injection; by intravenous (IV) injection
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 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) represents estimated results (i.e., results received by applying negative binomial \[NB\] regression model on data collected during EP).
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
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 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 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 represents observed results (i.e., descriptive statistics values based on the data which was collected during EP).
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
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 less than or equal to 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 represents estimated results (i.e., results received by applying NB regression model on data collected during TP).
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
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 less than or equal to 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 represents observed results (i.e., descriptive statistics values based on data collected during TP).
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
Estimated Annualized Spontaneous Bleeding Rate for Treated Bleeds During 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 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 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 represents estimated results (i.e., results received by applying NB regression model on data collected during EP).
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
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 bleed, within which any symptoms of bleeding at the same location or injections less than or equal to 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 represents observed results (i.e., descriptive statistics values based on data collected during EP).
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
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) increased 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).
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
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) increased 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).
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
Health-related Quality of Life (HRQOL): Change From Baseline in Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QOL) Physical Health Domain 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). Items were rated along five response options: never, rarely, sometimes, often, or all the time. 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 to 100, where lower scores denoted better physical health.
Time frame: Baseline (Day 1), Month 9
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). Items were rated along five response options: never, rarely, sometimes, often, or all the time. Raw score for each domain were transformed to a scale ranged from 0 to 100, where lower scores denoted better health. Haem-A-QoL Total Score was average of all domain scores and ranged from 0 to 100, where lower scores denoted better quality of life.
Time frame: Baseline (Day 1), Month 9
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 may 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 less than or equal to 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 represents estimated results (i.e., results received by applying NB regression model on data collected during onset period).
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
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 BPA 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.
Time frame: From Baseline (Day 1) up to 15 months (i.e. 9 months treatment period + 6-months follow-up)
The study was conducted at 58 centers in 17 countries. A total of 85 participants were screened between 14 February 2018 to 19-Mar-2021, of which 25 participants were screen failure. Screen failures were mainly due to the presence of clinically significant liver disease. A total of 60 participants were enrolled in the study.
| Milestone | Bypassing Agents (BPA) On-demand | Fitusiran 80 mg Prophylaxis |
|---|---|---|
| Started | 19 | 38 |
| Safety analysis set | 19 | 41 |
| Completed | 19 | 33 |
| Not completed | 0 | 5 |
| Withdrew: Adverse event | 0 | 1 |
| Withdrew: Related to coronavirus pandemic (covid-19) | 0 | 3 |
| Withdrew: Other | 0 | 1 |
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 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) represents estimated results (i.e., results received by applying negative binomial \[NB\] regression model on data collected during EP).
| episodes per participant per year | Bypassing Agents (BPA) On-demand | Fitusiran 80 mg Prophylaxis |
|---|---|---|
| Estimated Annualized Bleeding Rate (ABR) for Treated Bleeds During the Efficacy Period | 18.071 (10.598 to 30.812) | 1.666 (1.014 to 2.736) |
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 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 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 represents observed results (i.e., descriptive statistics values based on the data which was collected during EP).
| episodes per participant per year | Bypassing Agents (BPA) On-demand | Fitusiran 80 mg Prophylaxis |
|---|---|---|
| Observed Annualized Bleeding Rate (ABR) for Treated Bleeds During the Efficacy Period | 18.1 ± 14.9 | 1.7 ± 3.8 |
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 less than or equal to 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 represents estimated results (i.e., results received by applying NB regression model on data collected during TP).
| episodes per participant per year | Bypassing Agents (BPA) On-demand | Fitusiran 80 mg Prophylaxis |
|---|---|---|
| Estimated Annualized Bleeding Rate (ABR) for Treated Bleeds During the Treatment Period | 18.819 (11.541 to 30.687) | 2.024 (1.306 to 3.138) |
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 less than or equal to 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 represents observed results (i.e., descriptive statistics values based on data collected during TP).
| episodes per participant per year | Bypassing Agents (BPA) On-demand | Fitusiran 80 mg Prophylaxis |
|---|---|---|
| Observed Annualized Bleeding Rate (ABR) for Treated Bleeds During the Treatment Period | 18.8 ± 15.4 | 2.0 ± 3.7 |
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 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 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 represents estimated results (i.e., results received by applying NB regression model on data collected during EP).
| episodes per participant per year | Bypassing Agents (BPA) On-demand | Fitusiran 80 mg Prophylaxis |
|---|---|---|
| Estimated Annualized Spontaneous Bleeding Rate for Treated Bleeds During Efficacy Period | 15.675 (9.281 to 26.471) | 0.872 (0.491 to 1.551) |
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 bleed, within which any symptoms of bleeding at the same location or injections less than or equal to 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 represents observed results (i.e., descriptive statistics values based on data collected during EP).
| episodes per participant per year | Bypassing Agents (BPA) On-demand | Fitusiran 80 mg Prophylaxis |
|---|---|---|
| Observed Annualized Spontaneous Bleeding Rate for Treated Bleeds During the Efficacy Period | 15.6 ± 14.9 | 0.9 ± 2.0 |
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) increased 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).
| episodes per participant per year | Bypassing Agents (BPA) On-demand | Fitusiran 80 mg Prophylaxis |
|---|---|---|
| Estimated Annualized Joint Bleeding Rate for Treated Bleeds During the Efficacy Period | 13.759 (7.950 to 23.811) | 1.349 (0.798 to 2.280) |
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) increased 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).
| episodes per participant per year | Bypassing Agents (BPA) On-demand | Fitusiran 80 mg Prophylaxis |
|---|---|---|
| Observed Annualized Joint Bleeding Rate for Treated Bleeds During the Efficacy Period | 13.8 ± 12.2 | 1.4 ± 3.4 |
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). Items were rated along five response options: never, rarely, sometimes, often, or all the time. 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 to 100, where lower scores denoted better physical health.
| score on a scale | Bypassing Agents (BPA) On-demand | Fitusiran 80 mg Prophylaxis |
|---|---|---|
| Health-related Quality of Life (HRQOL): Change From Baseline in Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QOL) Physical Health Domain Score at Month 9 | -1.94 (-10.32 to 6.43) | -30.67 (-36.90 to -24.43) |
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). Items were rated along five response options: never, rarely, sometimes, often, or all the time. Raw score for each domain were transformed to a scale ranged from 0 to 100, where lower scores denoted better health. Haem-A-QoL Total Score was average of all domain scores and ranged from 0 to 100, where lower scores denoted better quality of life.
| score on a scale | Bypassing Agents (BPA) On-demand | Fitusiran 80 mg Prophylaxis |
|---|---|---|
| Health-related Quality of Life (HRQOL): Change From Baseline in Haem-A-QOL Total Score at Month 9 | -0.42 (-5.65 to 4.80) | -15.27 (-19.30 to -11.24) |
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 may 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 less than or equal to 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 represents estimated results (i.e., results received by applying NB regression model on data collected during onset period).
| episodes per participant per year | Bypassing Agents (BPA) On-demand | Fitusiran 80 mg Prophylaxis |
|---|---|---|
| Estimated Annualized Bleeding Rate (ABR) for Treated Bleeds During the Onset Period | 25.149 (15.819 to 39.981) | 4.426 (2.439 to 8.030) |
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 BPA 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.
| Participants | Bypassing Agents (BPA) On-demand | Fitusiran 80 mg Prophylaxis |
|---|---|---|
| Any TEAE | 11 | 38 |
| Any TESAE | 5 | 7 |
Collected over AE data were collected from Baseline (Day 1) up to 15 months (i.e., 9 months treatment period + 6-months follow-up).. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Bypassing Agents (BPA) On-demand | 0/19 (0%) | 5/19 (26.3%) | 8/19 (42.1%) |
| Fitusiran 80 mg Prophylaxis | 0/41 (0%) | 7/41 (17.1%) | 32/41 (78%) |
| Event | Bypassing Agents (BPA) On-demand | Fitusiran 80 mg Prophylaxis |
|---|---|---|
| Tooth FractureInjury, poisoning and procedural complications | 1/19 | 0/41 |
| Traumatic HaemorrhageInjury, poisoning and procedural complications | 1/19 | 0/41 |
| HaematuriaRenal and urinary disorders | 1/19 | 1/41 |
| HaemarthrosisMusculoskeletal and connective tissue disorders | 1/19 | 0/41 |
| Muscle HaemorrhageMusculoskeletal and connective tissue disorders | 1/19 | 0/41 |
| HaemorrhageVascular disorders | 1/19 | 0/41 |
| AnaemiaBlood and lymphatic system disorders | 0/19 | 1/41 |
| Spinal Vascular DisorderNervous system disorders | 0/19 | 1/41 |
| Asymptomatic Covid-19Infections and infestations | 0/19 | 1/41 |
| Device Related InfectionInfections and infestations | 0/19 | 1/41 |
| Event | Bypassing Agents (BPA) On-demand | Fitusiran 80 mg Prophylaxis |
|---|---|---|
| Alanine Aminotransferase IncreasedInvestigations | 0/19 | 13/41 |
| Aspartate Aminotransferase IncreasedInvestigations | 0/19 | 8/41 |
| Gamma-Glutamyltransferase IncreasedInvestigations | 0/19 | 6/41 |
| HeadacheNervous system disorders | 1/19 | 6/41 |
| Abdominal Pain UpperGastrointestinal disorders | 0/19 | 6/41 |
| Upper Respiratory Tract InfectionInfections and infestations | 1/19 | 6/41 |
| Blood Alkaline Phosphatase IncreasedInvestigations | 0/19 | 5/41 |
| Transaminases IncreasedInvestigations | 0/19 | 5/41 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 0/19 | 5/41 |
| Fibrin D Dimer IncreasedInvestigations | 0/19 | 3/41 |
Analysis was performed on Intent-to-Treat (ITT) population which included all randomized participants who were analyzed according to the randomized treatment arm.
| Age, Continuous(years) | Bypassing Agents (BPA) On-demand | Fitusiran 80 mg Prophylaxis | Total Title |
|---|---|---|---|
| Mean | 31.5 ± 13.1 | 26.8 ± 9.8 | 28.4 ± 11.1 |
| Sex: Female, Male(Participants) | Bypassing Agents (BPA) On-demand | Fitusiran 80 mg Prophylaxis | Total Title |
|---|---|---|---|
| Female | 0 | 0 | 0 |
| Male | 19 | 38 | 57 |
| Race/Ethnicity, Customized(Participants) | Bypassing Agents (BPA) On-demand | Fitusiran 80 mg Prophylaxis | Total Title |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 13 | 26 | 39 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 |
| White | 6 | 10 | 16 |
| Other | 0 | 1 | 1 |
| Multiple | 0 | 1 | 1 |
| Unknown or Not Reported | 0 | 0 | 0 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Yes — 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
Ran from Feb 2018 to primary completion in Nov 2020, 33 months.
Results were first posted in Dec 2021, 1 year after primary completion.
Of 89 industry-sponsored phase 3 interventional studies under Hemophilia A, half reached primary completion within 32 months of starting (the median), counting those still running; a quarter within 18 and three quarters within 56. This study's 33 months is close to that.
Counting every sponsor, half of 91 studies reached it within 33 months; a quarter within 20 and three quarters within 56.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Mar 2022. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Studies of the drug tested here: Fitusiran