Trial · NCT03569891
HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients
NCT03569891, Phase 3, Completed, CSL Behring; results first posted 2022-10-10
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients. Trials, record NCT03569891. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03569891
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients
- NCT number
- NCT03569891
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 67 participants, actual
- Lead sponsor
- CSL Behring
- Conditions
- Hemophilia B
- Interventions
- AAV5-hFIXco-Padua, Factor IX (FIX)
- First posted
- 2018-06-26
- Last updated
- 2026-03-27
- Other registries' IDs
- eudract:2017-004305-40
- Results first posted
- 2022-10-10
- Limitations and caveats
- This is an 18-month post-dose interim analysis due to the completion of the primary endpoint. The study will complete approximately 5 years after post-dose.
Source ClinicalTrials.gov
Registered outcomes26
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Annualized Bleeding Rate (ABR) for All Bleeding Episodes | Lead-in period and months 7-18 post-treatment of AMT-061 (CSL222) | Adjusted ABR for Lead-in and Post-Treatment period was estimated from a repeated measures generalized estimating equations negative binomial regression model accounting for the paired design of the trial with an offset parameter to account for the differential collection periods. Treatment period was included as a categorical covariate. |
| Secondary | Factor IX Activity Levels After AMT-061 (CSL222) Dosing | At Baseline, 6, 12, and 18 months after AMT-061 (CSL222) dosing | One-stage activated partial thromboplastin time (aPTT) based endogenous FIX activity levels from central laboratory assay. |
| Secondary | Annualized Exogenous FIX Consumption | Lead-in period and months 0-6, 7-12, and 13-18 after AMT-061 (CSL222) dosing | Annualized consumption of FIX replacement therapy. |
| Secondary | Adjusted Annualized Infusion Rate of FIX Replacement Therapy | Lead-in period and months 7-18 after AMT-061 (CSL222) dosing | |
| Secondary | Percent of Participants Who Discontinued FIX Prophylaxis and Remained Free of Routine FIX Prophylaxis After AMT-061 (CSL222) Dosing | Months 7-18 after AMT-061 (CSL222) dosing | |
| Secondary | Percentage of Participants With Trough FIX Activity <12% of Normal | Lead-in and 3, 12, and 18 months after AMT-061 (CSL222) dosing | |
| Secondary | ABR for FIX-treated Bleeding Episodes | Lead-in and Months 7-18 after AMT-061 (CSL222) dosing | Adjusted ABR for Lead-In and Post-Treatment period was estimated from a repeated measures generalized estimating equations negative binomial regression model accounting for the paired design of the trial with an offset parameter to account for the differential collection periods. Treatment period was included as a categorical covariate. |
| Secondary | Annualized Rate of Spontaneous Bleeding Episodes | Lead-in period and months 7-18 after AMT-061 (CSL222) dosing | Adjusted ABR for Lead-In and Post-Treatment period was estimated from a repeated measures generalized estimating equations negative binomial regression model accounting for the paired design of the trial with an offset parameter to account for the differential collection periods. Treatment period was included as a categorical covariate. |
| Secondary | Annualized Rate of Joint Bleeding Episodes | Lead-in period and months 7-18 after AMT-061 (CSL222) dosing | Adjusted ABR for Lead-In and Post-Treatment period was estimated from a repeated measures generalized estimating equations negative binomial regression model accounting for the paired design of the trial with an offset parameter to account for the differential collection periods. Treatment period was included as a categorical covariate. |
| Secondary | Mean FIX Activity (%) in Participants With Pre-Existing Neutralizing Antibodies to AAV5 After AMT-061 (CSL222) Dosing | At Baseline, 6, 12, and 18 months after AMT-061 (CSL222) dosing | One-stage aPTT-based endogenous FIX activity levels from central laboratory assay. |
1 to 10 of 26Source ClinicalTrials.gov
See all 26 registered outcomesOutcomes16
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Annualized Bleeding Rate (ABR) for All Bleeding Episodes | Lead-in period and months 7-18 post-treatment of AMT-061 | Full Analysis Set (FAS) included all subjects who were enrolled, entered the lead-in phase, were dosed with AMT-061, and provided at least 1 efficacy endpoint assessment for any efficacy endpoint subsequent to AMT-061 dosing. Subjects who were Lead-in Discontinuers were not included in the FAS. | bleeds/year/subject | MEAN |
| SECONDARY | Factor IX Activity Levels After AMT-061 Dosing | Baseline and 6,12, and 18 months after AMT-061 dosing | FAS. | Factor IX activity (%) | MEAN |
| SECONDARY | Annualized Exogenous Factor IX Consumption | Lead-in period and months 0-6, 7-12, and 13-18 after AMT-061 dosing | FAS | IU/year | MEAN |
| SECONDARY | Adjusted Annualized Infusion Rate of FIX Replacement Therapy | Lead-in period and months 7-18 after AMT-061 dosing | FAS. | Infusions/year | MEAN |
| SECONDARY | Percent of Subjects Who Discontinued FIX Prophylaxis and Remained Free of Routine FIX Prophylaxis After AMT-061 Dosing | Months 7-18 after AMT-061 dosing | FAS | percentage of participants | NUMBER |
| SECONDARY | Percentage of Subjects With Trough FIX Activity <12% of Normal | Lead-in and 3, 12, and 18 months after AMT-061 dosing | FAS | percentage of participants | NUMBER |
| SECONDARY | ABR for FIX-treated Bleeding Episodes | Lead-in and Months 7-18 after AMT-061 dosing | FAS | bleeds/year/subject | MEAN |
| SECONDARY | Number of Spontaneous Bleeding Episodes | Lead-in period and months 7-18 after AMT-061 dosing | FAS | Number of bleeds | NUMBER |
| SECONDARY | Number of Joint Bleeding Episodes | Lead-in period and months 7-18 after AMT-061 dosing | FAS | Number of bleeds | NUMBER |
| SECONDARY | Mean FIX Activity (%) in Subjects With Pre-Existing Neutralizing Antibodies to AAV5 After AMT-061 Dosing | Baseline and 6,12, and 18 months after AMT-061 dosing | FAS | Factor IX activity (%) | MEAN |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 outcomesResults by group39
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Annualized Bleeding Rate (ABR) for All Bleeding Episodes | FIX (Lead-in) | 4.19 | 95% Confidence Interval 3.22 to 5.45 | bleeds/year/subject | |
| Annualized Bleeding Rate (ABR) for All Bleeding Episodes | AMT-061 | 1.51 | 95% Confidence Interval 0.81 to 2.82 | bleeds/year/subject | |
| Factor IX Activity Levels After AMT-061 Dosing | AMT-061 | Baseline | 1.19 | Standard Deviation 0.39 | Factor IX activity (%) |
| Factor IX Activity Levels After AMT-061 Dosing | AMT-061 | 6 months | 38.95 | Standard Deviation 18.72 | Factor IX activity (%) |
| Factor IX Activity Levels After AMT-061 Dosing | AMT-061 | 12 months | 41.48 | Standard Deviation 21.71 | Factor IX activity (%) |
| Factor IX Activity Levels After AMT-061 Dosing | AMT-061 | 18 months | 36.90 | Standard Deviation 21.40 | Factor IX activity (%) |
| Annualized Exogenous Factor IX Consumption | FIX (Lead-in) | Lead-in | 257,338.8 | Standard Deviation 149,013.1 | IU/year |
| Annualized Exogenous Factor IX Consumption | AMT-061 | Months 0-6 | 12,912.9 | Standard Deviation 37,093.1 | IU/year |
| Annualized Exogenous Factor IX Consumption | AMT-061 | Months 7-12 | 8399.1 | Standard Deviation 29,720.9 | IU/year |
| Annualized Exogenous Factor IX Consumption | AMT-061 | Months 13-18 | 8486.6 | Standard Deviation 28,770.2 | IU/year |
1 to 10 of 39Source ClinicalTrials.gov
See all 39 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Lead-in | STARTED | Lead-in Safety Population | 67 | |
| Lead-in | COMPLETED | Lead-in Safety Population | 54 | |
| Lead-in | NOT COMPLETED | Lead-in Safety Population | 13 | |
| AMT-061 | STARTED | Lead-in Safety Population | 54 | |
| AMT-061 | COMPLETED | Lead-in Safety Population | 53 | |
| AMT-061 | NOT COMPLETED | Lead-in Safety Population | 1 |
Source ClinicalTrials.gov
Adverse events168
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 | Arthralgia | Musculoskeletal and connective tissue disorders | Lead-In (Including Discontinuers) | 5 | 67 | 5 |
| other | Arthralgia | Musculoskeletal and connective tissue disorders | Lead-In (Not Including Discontinuers) | 4 | 54 | 4 |
| other | Arthralgia | Musculoskeletal and connective tissue disorders | AMT-061 | 18 | 54 | 31 |
| other | Back pain | Musculoskeletal and connective tissue disorders | Lead-In (Including Discontinuers) | 1 | 67 | 1 |
| other | Back pain | Musculoskeletal and connective tissue disorders | Lead-In (Not Including Discontinuers) | 1 | 54 | 1 |
| other | Back pain | Musculoskeletal and connective tissue disorders | AMT-061 | 7 | 54 | 10 |
| other | Pain in extremity | Musculoskeletal and connective tissue disorders | Lead-In (Including Discontinuers) | 1 | 67 | 1 |
| other | Pain in extremity | Musculoskeletal and connective tissue disorders | Lead-In (Not Including Discontinuers) | 1 | 54 | 1 |
| other | Pain in extremity | Musculoskeletal and connective tissue disorders | AMT-061 | 6 | 54 | 7 |
| other | Myalgia | Musculoskeletal and connective tissue disorders | Lead-In (Including Discontinuers) | 1 | 67 | 1 |
1 to 10 of 168Source ClinicalTrials.gov
See all 168 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Lead-In (Including Discontinuers) | 0 | 67 |
| Total, all-cause mortality | Lead-In (Not Including Discontinuers) | 0 | 54 |
| Total, all-cause mortality | AMT-061 | 1 | 54 |
| Total, other adverse events | Lead-In (Including Discontinuers) | 27 | 67 |
| Total, other adverse events | Lead-In (Not Including Discontinuers) | 24 | 54 |
| Total, other adverse events | AMT-061 | 53 | 54 |
| Total, serious adverse events | Lead-In (Including Discontinuers) | 5 | 67 |
| Total, serious adverse events | Lead-In (Not Including Discontinuers) | 4 | 54 |
| Total, serious adverse events | AMT-061 | 13 | 54 |
Source ClinicalTrials.gov
Papers5
The papers the trial lists on ClinicalTrials.gov.
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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/NCT03569891
GET https://conductscience.org/api/trials/v1/NCT03569891/registered?page=2