Trial · NCT00749515
Pilot Study for Patients With Poor Response to Deferasirox
NCT00749515, Phase 4, Completed, Boston Children's Hospital; results first posted 2019-02-06
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Pilot Study for Patients With Poor Response to Deferasirox. Trials, record NCT00749515. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00749515
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Pilot Study for Patients With Poor Response to Deferasirox
- NCT number
- NCT00749515
- Status
- Completed
- Phases
- Phase 4
- Enrollment
- 15 participants, actual
- Lead sponsor
- Boston Children's Hospital
- Conditions
- Transfusion-dependent Hemachromatosis, Thalassemia Major, Sickle Cell Disease
- Interventions
- Deferoxamine, Deferasirox, HIDA
- First posted
- 2008-09-09
- Last updated
- 2024-02-12
- Results first posted
- 2019-02-06
Source ClinicalTrials.gov
Registered outcomes6
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Area Under the Curve of Deferasirox After a Dose of 35 mg/kg | 0, 1, 2, 4, 6, 8, 12, and 24 hours post dose | Area Under the Curve (AUC) 0 to 24 hours post dose |
| Primary | Half-Life of Deferasirox | 0, 1, 2, 4, 6, 8, 12, and 24 hours post dose. | All patients received the same interventions of deferoxamine challenge, deferasirox dose with pharmacokinetic monitoring. Then we compared responses between patients who were known to be slow responders to deferasirox and those who were known to be rapid responders (chelated well). Deferoxamine: After a 3-day washout period from all chelation, all patients have a 12 hour infusion of 50mg/kg of deferoxamine with urine collection and pre and post blood sampling to assess iron and Total Iron Binding Capacity (TIBC) by atomic absorption. |
| Primary | Volume of Distribution/Bioavailability of Deferasirox After a Dose of 35 mg/kg | 0, 1, 2, 4, 6, 8, 12, and 24 hours post dose | Volume of distribution/bioavailability (Vd/F) |
| Primary | Volume of Distribution/Bioavailability of Deferasirox After a Dose of 35 mg/kg | 0, 1, 2, 4, 6, 8, 12, and 24 hours post dose | Volume of distribution/bioavailability (Vd/F), adjusted per kilogram body weight |
| Primary | Clearance/Bioavailability of Deferasirox in Patients With Poor Response to Deferasirox Compared to Patients With Good Response After a Dose of 35 mg/kg | 0, 1, 2, 4, 6, 8, 12, and 24 hours post dose. | Clearance/bioavailability (CL/F) |
| Secondary | Number of Participants With Polymorphisms in Genes Known to be, or Potentially Involved, in Deferasirox Disposition | 3 months | Polymorphisms in genes known to be, or potentially involved, in deferasirox disposition: UGT1a1 (including the Gilbert syndrome promoter polymorphism, (TA)nTAA),UGT1a3, BRCP/ABCG2, MRP2/ABCC2. These genes were chosen because deferasirox is primarily eliminated by glucuronidation and subsequent biliary excretion. |
Source ClinicalTrials.gov
Outcomes6
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Area Under the Curve of Deferasirox After a Dose of 35 mg/kg | 0, 1, 2, 4, 6, 8, 12, and 24 hours post dose | micromole/liter*hour | MEAN | |
| PRIMARY | Half-Life of Deferasirox | 0, 1, 2, 4, 6, 8, 12, and 24 hours post dose. | hour | MEAN | |
| PRIMARY | Volume of Distribution/Bioavailability of Deferasirox After a Dose of 35 mg/kg | 0, 1, 2, 4, 6, 8, 12, and 24 hours post dose | liter | MEAN | |
| PRIMARY | Volume of Distribution/Bioavailability of Deferasirox After a Dose of 35 mg/kg | 0, 1, 2, 4, 6, 8, 12, and 24 hours post dose | liter/kilogram | MEAN | |
| PRIMARY | Clearance/Bioavailability of Deferasirox in Patients With Poor Response to Deferasirox Compared to Patients With Good Response After a Dose of 35 mg/kg | 0, 1, 2, 4, 6, 8, 12, and 24 hours post dose. | liter/hour | MEAN | |
| SECONDARY | Number of Participants With Polymorphisms in Genes Known to be, or Potentially Involved, in Deferasirox Disposition | 3 months | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group18
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Area Under the Curve of Deferasirox After a Dose of 35 mg/kg | Inadequate Responders | 479.59 | Standard Deviation 259.42 | micromole/liter*hour | |
| Area Under the Curve of Deferasirox After a Dose of 35 mg/kg | Adequate Responders (Control) | 1123.11 | Standard Deviation 63.4 | micromole/liter*hour | |
| Half-Life of Deferasirox | Inadequate Responders | 6.08 | Standard Deviation 2.01 | hour | |
| Half-Life of Deferasirox | Adequate Responders (Control) | 7.83 | Standard Deviation 2.95 | hour | |
| Volume of Distribution/Bioavailability of Deferasirox After a Dose of 35 mg/kg | Inadequate Responders | 10.33 | Standard Deviation 7.42 | liter | |
| Volume of Distribution/Bioavailability of Deferasirox After a Dose of 35 mg/kg | Adequate Responders (Control) | 6.5 | Standard Deviation 2.67 | liter | |
| Volume of Distribution/Bioavailability of Deferasirox After a Dose of 35 mg/kg | Inadequate Responders | 0.32 | Standard Deviation 0.25 | liter/kilogram | |
| Volume of Distribution/Bioavailability of Deferasirox After a Dose of 35 mg/kg | Adequate Responders (Control) | 0.13 | Standard Deviation 0.05 | liter/kilogram | |
| Clearance/Bioavailability of Deferasirox in Patients With Poor Response to Deferasirox Compared to Patients With Good Response After a Dose of 35 mg/kg | Inadequate Responders | 1.30 | Standard Deviation .18 | liter/hour | |
| Clearance/Bioavailability of Deferasirox in Patients With Poor Response to Deferasirox Compared to Patients With Good Response After a Dose of 35 mg/kg | Adequate Responders (Control) | 0.61 | Standard Deviation 0.22 | liter/hour |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Inadequate Responders | 10 | |
| Overall Study | STARTED | Adequate Responders (Control) | 5 | |
| Overall Study | COMPLETED | Inadequate Responders | 10 | |
| Overall Study | COMPLETED | Adequate Responders (Control) | 5 | |
| Overall Study | NOT COMPLETED | Inadequate Responders | 0 | |
| Overall Study | NOT COMPLETED | Adequate Responders (Control) | 0 |
Source ClinicalTrials.gov
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Inadequate Responders | 0 | 10 |
| Total, all-cause mortality | Adequate Responders (Control) | 0 | 5 |
| Total, other adverse events | Inadequate Responders | 0 | 10 |
| Total, other adverse events | Adequate Responders (Control) | 0 | 5 |
| Total, serious adverse events | Inadequate Responders | 0 | 10 |
| Total, serious adverse events | Adequate Responders (Control) | 0 | 5 |
Source ClinicalTrials.gov
Papers2
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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Inadequate Responders vs Adequate Responders (Control): deaths from any cause | Trial comparisons |
| Inadequate Responders vs Adequate Responders (Control): serious adverse events | Trial comparisons |
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/NCT00749515
GET https://conductscience.org/api/trials/v1/NCT00749515/registered