Phase 3 Haemophilia Trial, Completed NCT01662531 Sponsor: CSL Behring Condition: Haemophilia
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Trial results

AI generated results summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

According to the results reported on ClinicalTrials.gov, this trial (NCT01662531) enrolled 27 children with haemophilia B (a condition where the blood lacks enough of a clotting protein called Factor IX). All 27 participants completed the study — none dropped out. The trial was investigating how the body processed a medicine called rIX-FP, a laboratory-made form of Factor IX. Researchers looked at things like how quickly the medicine entered the bloodstream, how long it stayed active in the body, and whether the children's immune systems produced substances (called inhibitors) that could block the medicine from working. The reported data shows that after a single dose of rIX-FP, the medicine remained active in the blood for an average of around 91 hours (roughly 3.8 days), compared to approximately 19 hours for the children's previous Factor IX product. The reported incremental recovery — a measure of how much the clotting protein level in the blood rose per unit of medicine given — was approximately 1.01 for rIX-FP overall, compared to about 0.74 for the previous product. These figures were broadly similar across the two age groups tested (children under 6, and children aged 6 to under 12). The reported data also shows that zero out of 27 participants developed inhibitors to Factor IX during the study. Regarding side effects, the data reports that 26 out of 27 participants experienced at least one adverse event (an unwanted health event recorded during the trial); however, zero participants experienced an adverse event that was considered treatment-related, according to the reported results. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

These are the results as reported to ClinicalTrials.gov, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.

Phase 3 Haemophilia Trial, Completed

NCT01662531
Completed Phase 3 🇦🇺 Australian site

Who may be able to join

AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

Who might be able to join this trial:

  • The participant must be male and younger than 12 years old.
  • They must have severe Hemophilia B, meaning their Factor IX (a blood clotting protein) works at 2% or less of normal levels.
  • They must weigh at least 10 kilograms (about 22 pounds).
  • They must have already been treated with Factor IX products a certain number of times — more than 150 treatment sessions if aged 6 to under 12, or more than 50 treatment sessions if under 6 years old.
  • They must have no personal history of developing antibodies (called "inhibitors") that fight against Factor IX, no detectable inhibitors at the screening appointment, and no family history of such inhibitors.
  • A parent or guardian must provide written consent for the child to take part in the study.

Who may not be able to join:

  • Anyone with a known allergic reaction to any Factor IX product or to hamster proteins.
  • Anyone who has another blood clotting disorder, either one they were born with or one they developed, aside from Hemophilia B.
  • Anyone with a known kidney or liver disease.
  • Anyone who has recently had a life-threatening bleeding episode.

Important: Always verify eligibility with the trial site directly before applying.

Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.

This is a simplified plain English summary of the eligibility criteria. Full criteria are set by the trial investigators and may include additional requirements not shown here. Never self-exclude from a trial based on this summary. Contact the trial site directly to confirm your eligibility.
Last synced 28 July 2026
Phase 3: approximately ~65% of drugs entering this phase reach regulatory approval, based on published industry-wide historical data. This is not specific to this trial.
Phase success rates shown are historical industry-wide averages based on published data. They are not a prediction for this specific trial or your individual situation.

Contact this trial

Principal Investigator: Program Director, CSL Behring

Australian sites

The Royal Children's Hospital, Melbourne, Parkville, Victoria
The Children's Hospital at Westmead, Westmead,

Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.

GP referral letter

Print a one-page summary to share with your doctor.

Trial details

Status
Completed
Phase
Phase 3
Sponsor
CSL Behring
Registry
ClinicalTrials.gov
Start date
1 January 2013
Est. completion
1 October 2014

Where this trial is recruiting

🇦🇺 Australia 🇦🇹 Austria 🇨🇦 Canada 🇨🇿 Czechia 🇫🇷 France 🇩🇪 Germany 🇮🇱 Israel 🇮🇹 Italy 🇷🇺 Russia 🇪🇸 Spain

2 site(s) in Australia. Confirm current status and contact details directly with the trial site.

Primary endpoints

Incremental Recovery Following a Single Intravenous Dose of 50 IU/kg rIX-FP or Previous FIX Product; Half-life (t1/2) Following a Single Intravenous Dose of 50 IU/kg rIX-FP or Previous FIX Product; Area Under the Concentration Versus Time Curve From Time Point Zero to the Last Sample With Quantifiable Drug Concentration (AUClast); Clearance for FIX Activity Following a Single Intravenous Dose of 50 IU/kg rIX-FP or Previous FIX Product; Number of Subjects Developing Inhibitors to Factor IX (FIX)

Can't join this trial?

Expanded access pathways

If this trial is not available to you, other access pathways may exist. In Australia, the TGA Special Access Scheme allows access to unapproved therapeutic goods for individual patients.

TGA Special Access Scheme information

Find other recruiting trials on ClinicalTrials.gov

Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.

Trial recruitment status can change without notice between our nightly data updates. Always contact the trial site directly to confirm current recruitment status before making any decisions or travel arrangements.

View original record on ClinicalTrials.gov