Phase 2 Rare Disease Trial, Completed NCT00303329 Sponsor: Novartis Pharmaceuticals Condition: Rare Disease
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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 enrolled 184 people in total — 85 with a condition called beta-thalassemia (a blood disorder affecting haemoglobin) and 99 with other rare anaemias (conditions where the blood cannot carry enough oxygen). The trial was measuring the safety of a medicine called deferasirox, which is used to remove excess iron from the body, as well as tracking how iron levels in the liver and blood changed over the course of the study. Not everyone finished the trial: 50 of the beta-thalassemia group and 37 of the rare anaemias group completed it. The reported data shows that, for the primary outcome — counting how many participants experienced any unwanted medical events (called adverse events) or serious adverse events, or died — all 85 beta-thalassemia participants and all 99 rare anaemias participants had at least one adverse event recorded. Serious adverse events (those involving hospitalisation, life-threatening situations, lasting disability, or death) were reported in 36 of the 85 beta-thalassemia participants and 50 of the 99 rare anaemias participants. Deaths were reported for 2 participants in the beta-thalassemia group and 12 in the rare anaemias group. For the secondary outcomes, the reported data shows that iron levels in the liver (measured by biopsy) fell by an average of 5.17 units in the beta-thalassemia group and 5.10 units in the rare anaemias group. A different liver iron measurement method showed a small average increase of 0.19 units in the beta-thalassemia group and a decrease of 2.04 units in the rare anaemias group. A blood marker of iron build-up called serum ferritin also changed over the study — starting at around 4,321 in the beta-thalassemia group and 3,269 in the rare anaemias group, and ending with average decreases of 612 and 382 units respectively. 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 2 Rare Disease Trial, Completed

NCT00303329
Completed Phase 2

voxsanity.com.au · Eligibility summary from public government registries · 18 August 2026 · not medical advice

Who may and may not 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 may be able to join

  • You must have already completed the full 12-month main part of this study.
  • Female patients who have started their periods and are sexually active must be using two forms of contraception (such as two barrier methods, or a barrier method plus the pill), or must have had a hysterectomy, removal of the ovaries, or tubal ligation ("tubes tied").
  • You (and/or your parent or legal guardian, if applicable) must have signed a written consent form agreeing to take part, following the rules of the country where the trial is taking place.

Who may not be able to join

Each point below is a reason the trial team may not be able to accept someone. It is not a list of requirements to meet.

  • You cannot take part if you are currently pregnant.
  • You cannot take part if you are currently breastfeeding.

Important: There may be additional eligibility requirements not listed here that the trial team will need to check — 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 13 August 2026

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Trial details

Status
Completed
Phase
Phase 2
Registry
ClinicalTrials.gov
Start date
1 March 2004
Est. completion
1 October 2008

Where this trial was run

🇧🇪 Belgium 🇨🇦 Canada 🇫🇷 France 🇩🇪 Germany 🇮🇹 Italy 🇬🇧 United Kingdom 🇺🇸 United States

These are the locations recorded on the registry, and they are a historical record: this trial is not enrolling, so they are not places you can join it.

Primary endpoints

The Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) or Deaths

Other options

Expanded access pathways

This trial is not enrolling. Other access pathways exist in general: in Australia, the TGA Special Access Scheme allows access to unapproved therapeutic goods for individual patients. Whether it applies to any treatment studied here is a question for your doctor — nothing on this page says it does.

TGA Special Access Scheme information

Find other recruiting trials on ClinicalTrials.gov

Data last synced from ClinicalTrials.gov: 13 August 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