An open-label, single-arm, safety and efficacy study of recombinant human factor IX in children less than 6 years of age with severe haemophilia B
| DEC-NET Serial number GB259 | |
| Published online | 29/06/2004 14.23.00 |
| Last updated | 02/08/2005 13.06.33 |
| Other protocol ID number | N0012120170 |
| This trial has been approved by an ethics committee | |
| Current trial status | Open (actively recruiting new participants) |
| Major Disease (ICD9 class) | CONGENITAL FACTOR IX DISORDER |
| Experimental drug | |
| COAGULATION FACTOR IX | |
| Gender | Both |
| Age (range) | < 6 years |
Eligibility criteria | |
| Inclusion criteria | |
| Children with Severe Haemophilia B | |
| Exclusion criteria | |
| Informed consent not been given | |
Trial design/methodology | |
| Phase | 1 |
| Kind of study | Efficacy Safety |
| Design | Controlled Cohort Observation |
| Purpose of study | |
| To undertake a systematic observation of treatment in children aged less than 6 years with severe haemophillia B, regardless of prior treatment, to make recommendations regarding dosing and treatment of these children using the factor IX | |
| Primary outcomes | |
| To measure incremental recovery in children following a 75iu/kg dose, observing for allergic reactions, inhibitor development and thrombotic events | |
| Summary of study design, objectives, and ongoing research findings | |
| Cohort observation. To undertake a systematic observation of treatment in children aged less than 6 years with severe haemophillia B, regardless of prior treatment, to make recommendations regarding dosing and treatment of these children using the factor IX. Outcome measure description: To measure incremental recovery in children following a 75iu/kg dose, observing for allergic reactions, inhibitor development and thrombotic events | |
| Sponsor name |
| Commercial Funder (Industry) |
| ISRCTN | EudraCT |
