A Phase 3 interventional study of fitusiran and factor concentrates in Hemophilia A and Hemophilia B, sponsored by Genzyme, a Sanofi Company. Completed at 64 sites in 19 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 2nd 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 120 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:
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 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).
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 without inhibitors.
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:
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.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
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.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
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 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.
Drug: factor concentrates
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.
Drug: fitusiran · Drug: factor concentrates
by SC 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 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).
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 \<=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).
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 \<=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).
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 \<=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).
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 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).
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 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).
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) 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).
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) 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).
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 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.
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). 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
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).
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 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.
Time frame: From Baseline (Day 1) up to 15 months (i.e., 9 months treatment period + 6-month follow-up)
The study was conducted at 64 centers in 19 countries. A total of 177 participants were screened between 1 March 2018 to 22 May 2020, of which 57 participants were screen failure. Screen failures were mainly due to presence of a co-existing thrombophilic disorder. In total, 120 participants were enrolled in the study.
| Milestone | Factor On-demand | Fitusiran 80 mg Prophylaxis |
|---|---|---|
| Started | 40 | 80 |
| Treated | 40 | 79 |
| Completed | 37 | 79 |
| Not completed | 3 | 1 |
| Withdrew: Adverse event | 1 | 0 |
| Withdrew: Protocol violation | 1 | 0 |
| Withdrew: Withdrawal by subject | 1 | 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 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).
| episodes per participant per year | Factor On-demand | Fitusiran 80 mg Prophylaxis |
|---|---|---|
| Estimated Annualized Bleeding Rate (ABR) for Treated Bleeds During the Efficacy Period | 30.991 (21.114 to 45.487) | 3.133 (2.269 to 4.326) |
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).
| episodes per participant per year | Factor On-demand | Fitusiran 80 mg Prophylaxis |
|---|---|---|
| Observed Annualized Bleeding Rate (ABR) for Treated Bleeds During the Efficacy Period | 21.8 (8.4 to 41.0) | 0.0 (0.0 to 3.4) |
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).
| episodes per participant per year | Factor On-demand | Fitusiran 80 mg Prophylaxis |
|---|---|---|
| Estimated Annualized Bleeding Rate (ABR) for Treated Bleeds During the Treatment Period | 31.444 (22.995 to 42.999) | 4.092 (3.143 to 5.328) |
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).
| episodes per participant per year | Factor On-demand | Fitusiran 80 mg Prophylaxis |
|---|---|---|
| Observed Annualized Bleeding Rate (ABR) for Treated Bleeds During the Treatment Period | 25.2 (11.9 to 43.8) | 1.8 (0.0 to 4.5) |
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).
| episodes per participant per year | Factor On-demand | Fitusiran 80 mg Prophylaxis |
|---|---|---|
| Estimated Annualized Spontaneous Bleeding Rate for Treated Bleeds During the Efficacy Period | 22.036 (14.159 to 34.295) | 1.825 (1.237 to 2.692) |
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).
| episodes per participant per year | Factor On-demand | Fitusiran 80 mg Prophylaxis |
|---|---|---|
| Observed Annualized Spontaneous Bleeding Rate for Treated Bleeds During the Efficacy Period | 16.1 (3.4 to 27.6) | 0.0 (0.0 to 1.7) |
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).
| episodes per participant per year | Factor On-demand | Fitusiran 80 mg Prophylaxis |
|---|---|---|
| Estimated Annualized Joint Bleeding Rate for Treated Bleeds During the Efficacy Period | 23.413 (15.363 to 35.680) | 2.282 (1.594 to 3.268) |
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).
| episodes per participant per year | Factor On-demand | Fitusiran 80 mg Prophylaxis |
|---|---|---|
| Observed Annualized Joint Bleeding Rate for Treated Bleeds During the Efficacy Period | 15.9 (4.2 to 33.5) | 0.0 (0.0 to 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). 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.
| scores on a scale | Factor 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 Score at Month 9 | -3.32 (-9.67 to 3.04) | -23.07 (-28.00 to -18.14) |
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.
| scores on a scale | Factor On-demand | Fitusiran 80 mg Prophylaxis |
|---|---|---|
| Health-related Quality of Life (HRQOL): Change From Baseline in Haem-A-QOL Total Score at Month 9 | -2.62 (-6.27 to 1.03) | -9.68 (-12.51 to -6.86) |
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).
| episodes per participant per year | Factor On-demand | Fitusiran 80 mg Prophylaxis |
|---|---|---|
| Estimated Annualized Bleeding Rate (ABR) for Treated Bleeds During the Onset Period | 33.389 (25.619 to 43.517) | 10.805 (8.143 to 14.337) |
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.
| Participants | Factor On-demand | Fitusiran 80 mg Prophylaxis |
|---|---|---|
| Any TEAE | 18 | 62 |
| Any TESAE | 5 | 5 |
Collected over From Baseline (Day 1) up to 15 months (i.e., 9 months treatment period + 6-month follow-up).. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Factor On-demand | 0/40 (0%) | 5/40 (12.5%) | 8/40 (20%) |
| Fitusiran 80 mg Prophylaxis | 0/79 (0%) | 5/79 (6.3%) | 45/79 (57%) |
| Event | Factor On-demand | Fitusiran 80 mg Prophylaxis |
|---|---|---|
| CholelithiasisHepatobiliary disorders | 0/40 | 2/79 |
| Epidural HaemorrhageInjury, poisoning and procedural complications | 1/40 | 0/79 |
| Humerus FractureInjury, poisoning and procedural complications | 1/40 | 0/79 |
| Subdural HaemorrhageInjury, poisoning and procedural complications | 1/40 | 0/79 |
| Tibia FractureInjury, poisoning and procedural complications | 1/40 | 0/79 |
| DiplopiaEye disorders | 1/40 | 0/79 |
| OsteoarthritisMusculoskeletal and connective tissue disorders | 1/40 | 0/79 |
| GastroenteritisInfections and infestations | 1/40 | 0/79 |
| PneumoniaInfections and infestations | 1/40 | 0/79 |
| Suicidal IdeationPsychiatric disorders | 1/40 | 0/79 |
| Event | Factor On-demand | Fitusiran 80 mg Prophylaxis |
|---|---|---|
| Alanine Aminotransferase IncreasedInvestigations | 1/40 | 18/79 |
| Upper Respiratory Tract InfectionInfections and infestations | 0/40 | 9/79 |
| HypertensionVascular disorders | 4/40 | 3/79 |
| NasopharyngitisInfections and infestations | 3/40 | 7/79 |
| Aspartate Aminotransferase IncreasedInvestigations | 2/40 | 6/79 |
| Abdominal PainGastrointestinal disorders | 1/40 | 6/79 |
| CoughRespiratory, thoracic and mediastinal disorders | 0/40 | 6/79 |
| HeadacheNervous system disorders | 0/40 | 5/79 |
| GastritisGastrointestinal disorders | 1/40 | 5/79 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 1/40 | 5/79 |
Analysis was performed on intent-to-treat (ITT) population which included all randomized participants analyzed according to the randomized arm.
| Age, Continuous(years) | Factor On-demand | Fitusiran 80 mg Prophylaxis | Total Title |
|---|---|---|---|
| Mean | 33.6 ± 13.6 | 33.9 ± 14.6 | 33.8 ± 14.2 |
| Sex: Female, Male(Participants) | Factor On-demand | Fitusiran 80 mg Prophylaxis | Total Title |
|---|---|---|---|
| Female | 0 | 0 | 0 |
| Male | 40 | 80 | 120 |
| Race (NIH/OMB)(Participants) | Factor On-demand | Fitusiran 80 mg Prophylaxis | Total Title |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 28 | 43 | 71 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 2 | 2 |
| White | 12 | 33 | 45 |
| More than one race | 0 | 2 | 2 |
| 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 Mar 2018 to primary completion in Jan 2021, 34 months.
Results were first posted in Feb 2022, 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 34 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.
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Studies of the drug tested here: Fitusiran