Trial · NCT02554773
An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B
NCT02554773, Phase 1, Phase 2, Completed, Genzyme, a Sanofi Company; results first posted 2024-06-05
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B. Trials, record NCT02554773. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02554773
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B
- NCT number
- NCT02554773
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 34 participants, actual
- Lead sponsor
- Genzyme, a Sanofi Company
- Conditions
- Hemophilia A, Hemophilia B
- Interventions
- Fitusiran (SAR439774)
- First posted
- 2015-09-18
- Last updated
- 2024-06-05
- Other registries' IDs
- eudract:2015-001395-21
- Results first posted
- 2024-06-05
Source ClinicalTrials.gov
Registered outcomes26
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs) | From first dose of study drug (Day 1) up to end of the study (SAS 1: up to 76 months and SAS 2: up to 40 months) | An adverse event (AE) is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An SAE is any untoward medical occurrence that results: death or life-threatening or inpatient hospitalization or prolongation of existing hospitalization or persistent or significant disability or congenital anomaly or medically important event. All AEs collected in LTE14762 were considered TEAE because all participants received dose in the parent study. AEs of special interest (AESI) are alanine aminotransferase (ALT) or aspartate aminotransferase (AST) elevations \>3× upper limit of normal (ULN) or suspected or confirmed thromboembolic events or severe or serious injection site reactions or systemic injection associated reactions or cholecystitis or cholelithiasis. |
| Primary | Number of Participants With Potentially Clinically Significant Abnormality (PCSA): Hematology | From first dose of study drug (Day 1) up to end of the study (SAS 1: up to 76 months and SAS 2: up to 40 months) | Blood samples were collected to determine the hematology laboratory significant abnormalities. Here, DFB = decrease from baseline, NB = non-black, and B = black. |
| Primary | Number of Participants With Potentially Clinically Significant Abnormality: Clinical Chemistry | From first dose of study drug (Day 1) up to end of the study (SAS 1: up to 76 months and SAS 2: up to 40 months) | Blood samples were collected to determine the clinical chemistry laboratory abnormalities. Here, mmol/L = millimoles per liter, LLN = lower limit of normal, mg/L = milligram per liter, umol/L = micromoles per liter, mL/min = milliliter per minute, m\^2 = meter square, CB = conjugated bilirubin, and DB = direct bilirubin. |
| Primary | Number of Participants With Potentially Clinically Significant Abnormality: Urinalysis | From first dose of study drug (Day 1) up to end of the study (SAS 1: up to 76 months and SAS 2: up to 40 months) | Urine samples collected to determine the significant abnormalities in urine. |
| Primary | Number of Participants With Potentially Clinically Significant Abnormality: Vital Signs | From first dose of study drug (Day 1) up to end of the study (SAS 1: up to 76 months and SAS 2: up to 40 months) | Participants vital signs were examined to determine the abnormalities. Vital signs included weight, supine systolic blood pressure (SSBP) and supine diastolic blood pressure (SDBP). Here, mmHg = millimeter of mercury, and IFB = increase from baseline. |
| Primary | Number of Participants With Potentially Clinically Significant Abnormality: Electrocardiogram (ECG) | From first dose of study drug (Day 1) up to end of the study (SAS 1: up to 76 months and SAS 2: up to 40 months) | Standard 12-lead ECGs were recorded after at least 15 minutes in the supine position using an electrocardiographic device. The following were assessed: heart rate, rhythm, interval between the peaks of successive QRS complexes (RR), interval from the beginning of the P wave until the beginning of the QRS complex (PR), interval from start of the Q wave to the end of the S wave (QRS), interval between the start of the Q wave and the end of the T wave (QT), QT interval corrected for heart rate (QTc) automatic correction evaluation, QRS axis, left ventricular hypertrophy criteria, right ventricular hypertrophy criteria, repolarization charges, and overall cardiac impression for each participant. Here, msec = milliseconds. |
| Primary | Number of Participants With Potentially Clinically Significant Abnormality: Physical Examination | From first dose of study drug (Day 1) up to end of the study (SAS 1: up to 76 months and SAS 2: up to 40 months) | Physical examination included, at a minimum, an assessment of the participant's general appearance; skin; head, eyes, ears, nose, and throat; examinations of lymph nodes, abdomen, extremities/joints, neurological and mental status; heart and respiratory auscultation; peripheral arterial pulse; and pupil, knee, achilles, and plantar reflexes. |
| Secondary | Annualized Bleeding Rate (ABR) During the Efficacy Period | From Day 29 up to end of the study (SAS 1: up to 76 months and SAS 2: up to 40 months) | The ABR was annualized for each participant using the following formula: ABR = total number of bleeding events/total number of days in the respective period x 365.25. The efficacy period was defined as treatment Day 29 to earlier of end of study date before the dose pause or the last fitusiran administration date before the dose pause + 28 days, whichever comes first for original dose regimen (SAS 1) and the dose re-start Day 169 to the end of study visit for AT-based dose regimen (SAS 2). |
| Secondary | Annualized Spontaneous Bleeding Rate During the Efficacy Period | From Day 29 up to end of the study (SAS 1: up to 76 months and SAS 2: up to 40 months) | A spontaneous bleeding episode was defined as a bleeding event that occurred for no apparent or known reason, particularly into the joints, muscles, and soft tissues. The efficacy period was defined as treatment Day 29 to earlier of end of study date before the dose pause or the last fitusiran administration date before the dose pause + 28 days, whichever comes first for original dose regimen (SAS 1) and the dose re-start Day 169 to the end of study visit for AT-based dose regimen (SAS 2). |
| Secondary | Annualized Joint Bleeding Rate During the Efficacy Period | From Day 29 up to end of the study (SAS 1: up to 76 months and SAS 2: up to 40 months) | A joint bleeding episode was defined as an event that is characterized by an unusual sensation in the joint ("aura") in combination with 1) increasing swelling or warmth over the skin over the joint; 2) increasing pain; or 3) progressive loss of range of motion or difficulty in using the limb as compared with baseline. The efficacy period was defined as treatment Day 29 to earlier of end of study date before the dose pause or the last fitusiran administration date before the dose pause + 28 days, whichever comes first for original dose regimen (SAS 1) and the dose re-start Day 169 to the end of study visit for AT-based dose regimen (SAS 2). |
1 to 10 of 26Source ClinicalTrials.gov
See all 26 registered outcomesOutcomes26
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs) | From first dose of study drug (Day 1) up to end of the study (SAS 1: up to 76 months and SAS 2: up to 40 months) | Safety analysis set (SAS) included all participants who received at least a partial dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Potentially Clinically Significant Abnormality (PCSA): Hematology | From first dose of study drug (Day 1) up to end of the study (SAS 1: up to 76 months and SAS 2: up to 40 months) | The SAS included all participants who received at least a partial dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Potentially Clinically Significant Abnormality: Clinical Chemistry | From first dose of study drug (Day 1) up to end of the study (SAS 1: up to 76 months and SAS 2: up to 40 months) | The SAS included all participants who received at least a partial dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Potentially Clinically Significant Abnormality: Urinalysis | From first dose of study drug (Day 1) up to end of the study (SAS 1: up to 76 months and SAS 2: up to 40 months) | The SAS included all participants who received at least a partial dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Potentially Clinically Significant Abnormality: Vital Signs | From first dose of study drug (Day 1) up to end of the study (SAS 1: up to 76 months and SAS 2: up to 40 months) | The SAS included all participants who received at least a partial dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Potentially Clinically Significant Abnormality: Electrocardiogram (ECG) | From first dose of study drug (Day 1) up to end of the study (SAS 1: up to 76 months and SAS 2: up to 40 months) | The SAS included all participants who received at least a partial dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Potentially Clinically Significant Abnormality: Physical Examination | From first dose of study drug (Day 1) up to end of the study (SAS 1: up to 76 months and SAS 2: up to 40 months) | The SAS included all participants who received at least a partial dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Annualized Bleeding Rate (ABR) During the Efficacy Period | From Day 29 up to end of the study (SAS 1: up to 76 months and SAS 2: up to 40 months) | Full analysis set (FAS) included all participants in SAS. | bleeding events per year | MEAN |
| SECONDARY | Annualized Spontaneous Bleeding Rate During the Efficacy Period | From Day 29 up to end of the study (SAS 1: up to 76 months and SAS 2: up to 40 months) | The FAS included all participants in SAS. | bleeding events per year | MEAN |
| SECONDARY | Annualized Joint Bleeding Rate During the Efficacy Period | From Day 29 up to end of the study (SAS 1: up to 76 months and SAS 2: up to 40 months) | The FAS included all participants in SAS. | bleeding events per year | MEAN |
1 to 10 of 26Source ClinicalTrials.gov
See all 26 outcomesResults by group205
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs) | Original Dose Regimen (SAS 1) | Any TEAE | 33 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs) | AT-Based Dose Regimen (SAS 2) | Any TEAE | 14 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs) | Original Dose Regimen (SAS 1) | Any Treatment-emergent SAE | 13 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs) | AT-Based Dose Regimen (SAS 2) | Any Treatment-emergent SAE | 1 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs) | Original Dose Regimen (SAS 1) | Any Treatment-emergent AESI | 11 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs) | AT-Based Dose Regimen (SAS 2) | Any Treatment-emergent AESI | 1 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs) | Original Dose Regimen (SAS 1) | Any TEAE leading to study drug discontinuation | 5 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs) | AT-Based Dose Regimen (SAS 2) | Any TEAE leading to study drug discontinuation | 1 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs) | Original Dose Regimen (SAS 1) | Any TEAE leading to death | 1 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs) | AT-Based Dose Regimen (SAS 2) | Any TEAE leading to death | 0 | Participants |
1 to 10 of 205Source ClinicalTrials.gov
See all 205 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Original Dose Regimen (SAS 1) | STARTED | Original Dose Regimen [Safety Analysis Set 1 (SAS 1)] | 34 | |
| Original Dose Regimen (SAS 1) | STARTED | Antithrombin (AT)-Based Dose Regimen [Safety Analysis Set 2 (SAS 2)] | 0 | |
| Original Dose Regimen (SAS 1) | COMPLETED | Original Dose Regimen [Safety Analysis Set 1 (SAS 1)] | 18 | |
| Original Dose Regimen (SAS 1) | COMPLETED | Antithrombin (AT)-Based Dose Regimen [Safety Analysis Set 2 (SAS 2)] | 0 | |
| Original Dose Regimen (SAS 1) | NOT COMPLETED | Original Dose Regimen [Safety Analysis Set 1 (SAS 1)] | 16 | |
| Original Dose Regimen (SAS 1) | NOT COMPLETED | Antithrombin (AT)-Based Dose Regimen [Safety Analysis Set 2 (SAS 2)] | 0 | |
| AT-Based Dose Regimen (SAS 2) | STARTED | Original Dose Regimen [Safety Analysis Set 1 (SAS 1)] | 0 | |
| AT-Based Dose Regimen (SAS 2) | STARTED | Antithrombin (AT)-Based Dose Regimen [Safety Analysis Set 2 (SAS 2)] | 18 | |
| AT-Based Dose Regimen (SAS 2) | COMPLETED | Original Dose Regimen [Safety Analysis Set 1 (SAS 1)] | 0 | |
| AT-Based Dose Regimen (SAS 2) | COMPLETED | Antithrombin (AT)-Based Dose Regimen [Safety Analysis Set 2 (SAS 2)] | 12 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events406
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Blood Loss Anaemia | Blood and lymphatic system disorders | Original Dose Regimen (SAS 1) | 2 | 34 | 6 |
| other | Blood Loss Anaemia | Blood and lymphatic system disorders | AT-Based Dose Regimen (SAS 2) | 0 | 18 | 0 |
| other | Iron Deficiency Anaemia | Blood and lymphatic system disorders | Original Dose Regimen (SAS 1) | 0 | 34 | 0 |
| other | Iron Deficiency Anaemia | Blood and lymphatic system disorders | AT-Based Dose Regimen (SAS 2) | 1 | 18 | 1 |
| other | Neutropenia | Blood and lymphatic system disorders | Original Dose Regimen (SAS 1) | 1 | 34 | 1 |
| other | Neutropenia | Blood and lymphatic system disorders | AT-Based Dose Regimen (SAS 2) | 0 | 18 | 0 |
| other | Bundle Branch Block Right | Cardiac disorders | Original Dose Regimen (SAS 1) | 1 | 34 | 1 |
| other | Bundle Branch Block Right | Cardiac disorders | AT-Based Dose Regimen (SAS 2) | 0 | 18 | 0 |
| other | Conjunctivitis Allergic | Eye disorders | Original Dose Regimen (SAS 1) | 1 | 34 | 1 |
| other | Conjunctivitis Allergic | Eye disorders | AT-Based Dose Regimen (SAS 2) | 0 | 18 | 0 |
1 to 10 of 406Source ClinicalTrials.gov
See all 406 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Original Dose Regimen (SAS 1) | 1 | 34 |
| Total, all-cause mortality | AT-Based Dose Regimen (SAS 2) | 0 | 18 |
| Total, other adverse events | Original Dose Regimen (SAS 1) | 33 | 34 |
| Total, other adverse events | AT-Based Dose Regimen (SAS 2) | 13 | 18 |
| Total, serious adverse events | Original Dose Regimen (SAS 1) | 13 | 34 |
| Total, serious adverse events | AT-Based Dose Regimen (SAS 2) | 1 | 18 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 39642315 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons4
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Original Dose Regimen (SAS 1) vs AT-Based Dose Regimen (SAS 2): Number of Participants With Potentially Clinically Significant Abnormality: Physical Examination | Trial comparisons |
| Original Dose Regimen (SAS 1) vs AT-Based Dose Regimen (SAS 2): deaths from any cause | Trial comparisons |
| Original Dose Regimen (SAS 1) vs AT-Based Dose Regimen (SAS 2): Number of Participants With Potentially Clinically Significant Abnormality: Urinalysis | Trial comparisons |
| Original Dose Regimen (SAS 1) vs AT-Based Dose Regimen (SAS 2): serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02554773
GET https://conductscience.org/api/trials/v1/NCT02554773/registered?page=2