Trial · NCT03203850
Study to Evaluate the Efficacy and Safety of Deferasirox Film-coated Tablet Versus Phlebotomy in Patients With Hereditary Hemochromatosis (HH)
NCT03203850, Phase 2, Terminated, Novartis Pharmaceuticals; results first posted 2024-06-07
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study to Evaluate the Efficacy and Safety of Deferasirox Film-coated Tablet Versus Phlebotomy in Patients With Hereditary Hemochromatosis (HH). Trials, record NCT03203850. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03203850
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Evaluate the Efficacy and Safety of Deferasirox Film-coated Tablet Versus Phlebotomy in Patients With Hereditary Hemochromatosis (HH)
- NCT number
- NCT03203850
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 45 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Hereditary Hemochromatosis
- Interventions
- Deferasirox FCT, Phlebotomy
- First posted
- 2017-06-29
- Last updated
- 2024-10-09
- Other registries' IDs
- eudract:2016-002529-12
- Results first posted
- 2024-06-07
- Limitations and caveats
- The results should be interpreted with caution due to low number of participants enrolled in both arms.
Source ClinicalTrials.gov
Registered outcomes13
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Proportion of Patients Achieving Target SF ≤ 100 μg/L for the First Time | Up to Month 24 | Proportion of participants achieving target serum ferritin (SF) ≤ 100 μg/L on or before Month 24. Participants were considered responders if they met response criteria (target SF ≤100 µg/L) on or before Month 24 (Week 104) during the treatment phase. Any participant who discontinued treatment prematurely before meeting such criterion and participants with unknown or missing SF by Month 24 were counted as non-responder. |
| Secondary | Number of Participants With Ocular Treatment Emergent Adverse Events (AEs) | Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days post treatment, up to a maximum duration of approximately 108 weeks. | Number of participants with at least one ocular treatment emergent adverse event (new or worsening from baseline). |
| Secondary | Number of Participants With Ocular Treatment Emergent Adverse Events (AEs) by Preferred Term | Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days post treatment, up to a maximum duration of approximately 108 weeks. | Number of participants with at least one ocular treatment emergent adverse event (new or worsening from baseline). Preferred terms are based on Medical Dictionary of Regulatory Activities (MedDRA) version 26.0. |
| Secondary | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days post treatment, up to a maximum duration of approximately 108 weeks. | Number of participants with treatment emergent AEs (any AE regardless of seriousness), AEs leading to study treatment discontinuation, and SAEs. |
| Secondary | Number of Adverse Events in Participants Who Had Study Treatment Interrupted Due to SF ≤ 100 μg/L and Re-initiated Study Treatment When ≥ 300 μg/L | Up to 24 months | Number of participants with Adverse Events who interrupt deferasirox FCT at least once due to SF level ≤ 100 μg/L and re-initiate therapy at SF level ≥ 300 μg/L. There were no participants that re-initiated therapy when reached 300 ug/L. |
| Secondary | Categorical Analysis of logMAR Score Changes From Baseline to Best Post-baseline Changes in One Eye With More Extreme Change | Baseline, Weeks 24, 52, 76 and 104. | Visual acuity was measured using an Early Treatment Diabetic Retinopathy Study (ETDRS) chart. A letter score was calculated based on the number of letters that could correctly be identified from specified distances. For low luminance and standard acuity measures, visual acuity was described on a logMAR scale for all measures. For including acuity obtained with the ETDRS letter score, the values were converted to a logMAR scale, using the following formula: logMAR = 1.7-0.02\*ETDRS score. With this conversion, a difference from baseline of 0.1 logMAR = 5-letter difference in visual acuity, 0.2 logMAR = 10-letter difference, 0.3 logMAR = 15-letter difference, 0.4 logMAR = 20-letter difference, 0.5 logMAR = 25-letter difference and 0.6 logMAR = 30-letter difference. Decrease in logMAR score category from baseline indicates improvement in visual acuity. The best change from baseline amongst all post baseline visit is presented. |
| Secondary | Categorical Analysis of logMAR Score Changes From Baseline to Worst Post-baseline Changes in One Eye With More Extreme Change | Baseline, Weeks 24, 52, 76 and 104. | Visual acuity was measured using an Early Treatment Diabetic Retinopathy Study (ETDRS) chart. A letter score was calculated based on the number of letters that could correctly be identified from specified distances. For low luminance and standard acuity measures, visual acuity was described on a logMAR scale for all measures. For including acuity obtained with the ETDRS letter score, the values were converted to a logMAR scale, using the following formula: logMAR = 1.7-0.02\*ETDRS score. With this conversion, a difference from baseline of 0.1 logMAR = 5-letter difference in visual acuity, 0.2 logMAR = 10-letter difference, 0.3 logMAR = 15-letter difference, 0.4 logMAR = 20-letter difference, 0.5 logMAR = 25-letter difference and 0.6 logMAR = 30-letter difference. Increase in logMAR score from baseline indicates worsening in visual acuity. The worst change from baseline amongst all post baseline visit is presented. |
| Secondary | Categorical Analysis of Worst Post-baseline Values of Intraocular Pressure in One Eye With More Extreme Change | Weeks 24, 52, 76 and 104 | Intraocular pressure was measured by tonometry. Intraocular pressure values \>5 to ≤21 mmHg were considered normal. The worst post-baseline value corresponds to the worst outcome amongst all post baseline visit |
| Secondary | Categorical Analysis of Changes in Intraocular Pressure From Baseline to Best/Worst Post-baseline Changes in One Eye With More Extreme Change | Baseline, up to Week 104 | Intraocular pressure was measured by tonometry. A decrease in intraocular pressure from baseline indicated improvement. |
| Secondary | Number of Participants With Slit Lamp Results for Any Evaluation and Worst Eye | Baseline, up to Week 104 | Slit lamp examination was used to evaluate lids, cornea, conjunctiva, iris, anterior chamber, aqueous flare, aqueous inflammatory cells and lens. Any post-baseline abnormalities (not present at baseline) in slit lamp examination were assesses by the investigator and classified as insignificant or clinically significant. Number of participants with slit lamp results (normal, insignificant, significant, missing) for any evaluation and worst eye are reported. |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 registered outcomesOutcomes13
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Proportion of Patients Achieving Target SF ≤ 100 μg/L for the First Time | Up to Month 24 | The Full Analysis Set (FAS) comprised all subjects to whom study treatment had been assigned by randomization. | percentage of participants | NUMBER |
| SECONDARY | Number of Participants With Ocular Treatment Emergent Adverse Events (AEs) | Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days post treatment, up to a maximum duration of approximately 108 weeks. | The Safety Set (SS) included all subjects who received at least one administration of study treatment. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Ocular Treatment Emergent Adverse Events (AEs) by Preferred Term | Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days post treatment, up to a maximum duration of approximately 108 weeks. | The Safety Set (SS) included all subjects who received at least one administration of study treatment. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days post treatment, up to a maximum duration of approximately 108 weeks. | The Safety Set (SS) included all subjects who received at least one administration of study treatment. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Adverse Events in Participants Who Had Study Treatment Interrupted Due to SF ≤ 100 μg/L and Re-initiated Study Treatment When ≥ 300 μg/L | Up to 24 months | Participants in the Safety Set who had study treatment interrupted due to SF ≤ 100 μg/L and re-initiated study treatment when ≥ 300 μg/L. There were no patients who re-initiated therapy when reached SF 300 μg/L. | ||
| SECONDARY | Categorical Analysis of logMAR Score Changes From Baseline to Best Post-baseline Changes in One Eye With More Extreme Change | Baseline, Weeks 24, 52, 76 and 104. | The Safety Set (SS) included all subjects who received at least one administration of study treatment. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Categorical Analysis of logMAR Score Changes From Baseline to Worst Post-baseline Changes in One Eye With More Extreme Change | Baseline, Weeks 24, 52, 76 and 104. | The Safety Set (SS) included all subjects who received at least one administration of study treatment. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Categorical Analysis of Worst Post-baseline Values of Intraocular Pressure in One Eye With More Extreme Change | Weeks 24, 52, 76 and 104 | The Safety Set (SS) included all subjects who received at least one administration of study treatment. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Categorical Analysis of Changes in Intraocular Pressure From Baseline to Best/Worst Post-baseline Changes in One Eye With More Extreme Change | Baseline, up to Week 104 | The Safety Set (SS) included all subjects who received at least one administration of study treatment. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Slit Lamp Results for Any Evaluation and Worst Eye | Baseline, up to Week 104 | The Safety Set (SS) included all subjects who received at least one administration of study treatment. | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 outcomesResults by group132
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Proportion of Patients Achieving Target SF ≤ 100 μg/L for the First Time | Deferasirox FCT 7mg/kg | Responder | 40 | 95% Confidence Interval 22.7 to 59.4 | percentage of participants |
| Proportion of Patients Achieving Target SF ≤ 100 μg/L for the First Time | Phlebotomy | Responder | 80 | 95% Confidence Interval 51.9 to 95.7 | percentage of participants |
| Proportion of Patients Achieving Target SF ≤ 100 μg/L for the First Time | Deferasirox FCT 7mg/kg | Non-Responder | 60 | 95% Confidence Interval 40.6 to 77.3 | percentage of participants |
| Proportion of Patients Achieving Target SF ≤ 100 μg/L for the First Time | Phlebotomy | Non-Responder | 20 | 95% Confidence Interval 4.3 to 48.1 | percentage of participants |
| Number of Participants With Ocular Treatment Emergent Adverse Events (AEs) | Deferasirox FCT 7mg/kg | At least one ocular AE | 9 | Participants | |
| Number of Participants With Ocular Treatment Emergent Adverse Events (AEs) | Phlebotomy | At least one ocular AE | 0 | Participants | |
| Number of Participants With Ocular Treatment Emergent Adverse Events (AEs) | Deferasirox FCT 7mg/kg | Treatment-related ocular AEs | 2 | Participants | |
| Number of Participants With Ocular Treatment Emergent Adverse Events (AEs) | Phlebotomy | Treatment-related ocular AEs | 0 | Participants | |
| Number of Participants With Ocular Treatment Emergent Adverse Events (AEs) by Preferred Term | Deferasirox FCT 7mg/kg | Cataract nuclear | 2 | Participants | |
| Number of Participants With Ocular Treatment Emergent Adverse Events (AEs) by Preferred Term | Phlebotomy | Cataract nuclear | 0 | Participants |
1 to 10 of 132Source ClinicalTrials.gov
See all 132 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Deferasirox FCT 7mg/kg | 30 | |
| Overall Study | STARTED | Phlebotomy | 15 | |
| Overall Study | COMPLETED | Deferasirox FCT 7mg/kg | 22 | |
| Overall Study | COMPLETED | Phlebotomy | 12 | |
| Overall Study | NOT COMPLETED | Deferasirox FCT 7mg/kg | 8 | |
| Overall Study | NOT COMPLETED | Phlebotomy | 3 |
Source ClinicalTrials.gov
Adverse events150
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anaemia | Blood and lymphatic system disorders | Deferasirox FCT 7mg/kg | 1 | 30 | |
| other | Anaemia | Blood and lymphatic system disorders | Phlebotomy | 2 | 15 | |
| other | Sinus bradycardia | Cardiac disorders | Deferasirox FCT 7mg/kg | 1 | 30 | |
| other | Sinus bradycardia | Cardiac disorders | Phlebotomy | 1 | 15 | |
| other | Deafness unilateral | Ear and labyrinth disorders | Deferasirox FCT 7mg/kg | 1 | 30 | |
| other | Deafness unilateral | Ear and labyrinth disorders | Phlebotomy | 1 | 15 | |
| other | Ear pain | Ear and labyrinth disorders | Deferasirox FCT 7mg/kg | 0 | 30 | |
| other | Ear pain | Ear and labyrinth disorders | Phlebotomy | 1 | 15 | |
| other | Middle ear inflammation | Ear and labyrinth disorders | Deferasirox FCT 7mg/kg | 0 | 30 | |
| other | Middle ear inflammation | Ear and labyrinth disorders | Phlebotomy | 1 | 15 |
1 to 10 of 150Source ClinicalTrials.gov
See all 150 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Deferasirox FCT 7mg/kg | 1 | 30 |
| Total, all-cause mortality | Phlebotomy | 0 | 15 |
| Total, other adverse events | Deferasirox FCT 7mg/kg | 23 | 30 |
| Total, other adverse events | Phlebotomy | 12 | 15 |
| Total, serious adverse events | Deferasirox FCT 7mg/kg | 7 | 30 |
| Total, serious adverse events | Phlebotomy | 0 | 15 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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 |
|---|---|
| Deferasirox FCT 7mg/kg vs Phlebotomy: deaths from any cause | Trial comparisons |
| Deferasirox FCT 7mg/kg vs Phlebotomy: 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/NCT03203850
GET https://conductscience.org/api/trials/v1/NCT03203850/results?page=2