E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Moderate/severe to severe hemophilia B |
|
E.1.1.1 | Medical condition in easily understood language |
Moderate/severe to severe inherited blood coagulation disorder |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Blood and lymphatic diseases [C15] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10060614 |
E.1.2 | Term | Hemophilia B (Factor IX) |
E.1.2 | System Organ Class | 100000004850 |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
1. To determine the long-term safety and efficacy of DTX101 following a single IV infusion in adults with moderate/severe to severe hemophilia B |
|
E.2.2 | Secondary objectives of the trial |
1. To evaluate the long-term kinetics, duration, and magnitude of plasma FIX activity, by dose, after a single IV infusion of DTX101 in adults with hemophilia B
2. To assess the long-term impact of DTX101 on the number of bleeding episodes that require FIX replacement therapy
3. To assess the long-term impact of DTX101 on the frequency of FIX replacement therapy use during the study
4. To assess the long-term immune response to FIX after IV administration of DTX101
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Willing and able to provide written informed consent
2. Completed the Cohort 1/Week 52 or Cohort 2/week 44 visit in Study 101HEMB01
3. Willing to stop prophylactic treatment with recombinant FIX at specified time points during the study if medically acceptable
4. Willing, able, and committed to comply with scheduled study site visits, study procedures, and requirements |
|
E.4 | Principal exclusion criteria |
1. Planned or current participation in another interventional clinical study that may confound the safety or efficacy evaluation of DTX101 during the duration of this study
2. Any clinically significant medical condition that, in the opinion of the investigator, would pose a risk to subject safety or would impede the study |
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E.5 End points |
E.5.1 | Primary end point(s) |
1. The incidence of AEs and SAEs
2. The change from baseline (predose on Day 0 for Study 101HEMB01) in FIX activity at Week 208/216 (±14 days) as determined by the aPTT clot-based assay |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
1. From the time the subject signs the ICF until their exit from the study
2. Week 208/216 (±14 days) |
|
E.5.2 | Secondary end point(s) |
1. The time course of FIX activity, as determined by aPTT
2. The annualized bleeding rate through Week 208/216 (±14 days)
3. The annualized and average weekly use of FIX replacement therapy through Week 208/216 (±14 days)
4. Development of FIX inhibitor
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|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
1. Day 0, Weeks 26-156, Unscheduled visit, Week 208/216 (±14 days)
2. Through Week 208/216 (±14 days)
3. Through Week 208/216 (±14 days)
4. Day 0, Weeks 26-156, Unscheduled visit, Week 208/216 (±14 days)
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | Yes |
E.7.1.1 | First administration to humans | Yes |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | Yes |
E.7.1.3.1 | Other trial type description |
Phase 1/2 long-term follow-up study |
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E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
Long-term follow-up study; no IMP administered |
|
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 2 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
United Kingdom |
United States |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 5 |
E.8.9.1 | In the Member State concerned months | 1 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 5 |
E.8.9.2 | In all countries concerned by the trial months | 1 |
E.8.9.2 | In all countries concerned by the trial days | 0 |