β-thalassemia MajorPhase 4MACS2163NCT01724138
An Open Label Study to Evaluate the Pharmacokinetics, Safety, Tolerability and Efficacy of Deferasirox Administered to Chinese Patients With β-thalassemia Major Aged From 2 to Less Than 6 Years Old
A Phase 4 study testing Deferasirox in people with β-thalassemia Major, run by Novartis Pharmaceuticals.
Trial summary
To characterize the PK of deferasirox in pediatric β-thalassemia major patients aged from 2 to less than 6 years old, when administrated with a fixed starting dose of 20 mg/kg/day. Read more about this study
- Who is it for?
- Ages 2 to 71 years
- Conditions the study is for
- β-thalassemia Major
- What is being tested?
- Deferasirox
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Jun 2013to Oct 2014
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 71 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Pediatric patients aged from 2 to less than 6 years old.
- Patients with transfusion dependent β-thalassemia major.
- Serum ferritin values ≥ 1000 ng/ml at screening.
- Written informed consent must be obtained from the patient's legal guardian in accordance with local law and regulation prior to any screening procedures.
Who cannot join
- Non-transfusion-dependent thalassemia.
- Systemic diseases which would prevent study treatment (e.g. uncontrolled hypertension, cardiovascular, renal, hepatic, metabolic, etc.)
- Serum creatinine > age adjusted ULN.
- Significant proteinuria as indicated by a urinary protein/creatinine ratio (UPCR) ≥ 0.5mg/mg in a non-first void urine sample at screening. If UPCR is found to be ≥ 0.5 mg/mg the test can be repeated after 1 month.
- ALT/AST > 2.5xULN and total bilirubin > 1×ULN.
- Left ventricular ejection fraction < 56% by echocardiography.
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- Patient has a known history of HIV seropositivity or history of active/treated hepatitis B or C (a test for screening is not required).
- A history of clinically relevant ocular and/or auditory toxicity related to iron chelation therapy
- Any surgical or medical conditions which will significantly alter the absorption, distribution, metabolism or excretion of the drug(e.g. ulcerative disease, uncontrolled nausea, vomiting, malabsorption syndrome, obstruction, or stomach and/or small bowel resection).
- Other conditions which investigator deems potential harm to patients if participate the study.
About this study
To characterize the PK of deferasirox in pediatric β-thalassemia major patients aged from 2 to less than 6 years old, when administrated with a fixed starting dose of 20 mg/kg/day.
- Conditions
- β-thalassemia Major
- Being tested
- Deferasirox
- Keywords
- deferasirox
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DeferasiroxDrug
Patients will start their deferasirox treatment with a dose of 20 mg/kg/day.
| Study group | Role | What they receive |
|---|---|---|
| Deferasirox | Experimental | The study will provide PK, safety, tolerability and efficacy data collected during 48 weeks of treatment with deferasirox in Chinese pediatric patients with transfusion dependent -thalassemia major, aged 2 to <6 years at enrollment. The target patient pool consists of 20 patients with evidence of iron overload measured by serum ferritin level at the start of study. Patients will start their deferasirox treatment with a dose of 20 mg/kg/day. Serum ferritin will be monitored every month and the dose of deferasirox will be adjusted if necessary every 3 months based on the trends in serum ferritin. Other possible dose adjustments will be based on the patient's safety assessments. Receives: Drug: Deferasirox |
What is being measured
The primary outcome is the main question the study is designed to answer.
the PK profile of deferasirox in pediatric β-thalassemia major patients aged from 2 to less than 6 years old.
Area under the plasma concentration-time curve from time zero to the end of the dosing interval.
Measured: PK sampling times are 0.5, 2.5, 6, 10h in Day 1 postdose; Day 2, 3, 4 and 5 predose and 0.5, 2.5, 6, 10h post dose on Day 4, then pre-dose at each subsequent visit until Week 49. Day -1 PK sample will be treated as Day 1 predose sample.
the PK profile of deferasirox in pediatric β-thalassemia major patients aged from 2 to less than 6 years old: Cmax
The maximum plasma concentration of study medication.
Measured: PK sampling times are 0.5, 2.5, 6, 10h in Day 1 postdose; Day 2, 3, 4 and 5 predose and 0.5, 2.5, 6, 10h post dose on Day 4, then pre-dose at each subsequent visit until Week 49. Day -1 PK sample will be treated as Day 1 predose sample.
the PK profile of deferasirox in pediatric β-thalassemia major patients aged from 2 to less than 6 years old: Tmax
Tmax was directly determined from the raw plasma concentration-time data.
Measured: PK sampling times are 0.5, 2.5, 6, 10h in Day 1 postdose; Day 2, 3, 4 and 5 predose and 0.5, 2.5, 6, 10h post dose on Day 4, then pre-dose at each subsequent visit until Week 49. Day -1 PK sample will be treated as Day 1 predose sample.
Secondary outcomes (2)
The safety and tolerability of deferasirox following multiple dosing in pediatric β-thalassemia major patients.
The adverse events and abnormal measurements of hematology, blood chemistry, urinalysis, urinary protein/creatinine ratio, physical examination, auditory and ocular examinations, ECG, ECHO, and growth development are collected for the measurement of safety and tolerability.
Measured: Baseline, every 4 weeks until 48 weeks after taking the drug
The efficacy of deferasirox in pediatric β-thalassemia patients as measured by change of serum ferritin.
Changes in serum ferritin from baseline to every 4 weeks are collected for the measurement of efficacy.
Measured: Baseline, every 4 weeks until 48 weeks after taking the drug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Nov 2012 (estimated)
- Study startJun 2013
- Primary completionApr 2014 (estimated)
- Study completionOct 2014 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for β-thalassemia MajorStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01724138
- Sponsor's protocol ID
- CICL670ACN02
- Acronym
- MACS2163
- Status last verified
- Aug 2014
- First submitted
- 24 Oct 2012
- Last updated
- 20 Apr 2017
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Apr 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label Study to Evaluate the Pharmacokinetics, Safety, Tolerability and Efficacy of Deferasirox Administered to Chinese Patients With β-thalassemia Major Aged From 2 to Less Than 6 Years Old
- ClinicalTrials.gov ID
- NCT01724138
- Acronym / sponsor ID
- MACS2163 / CICL670ACN02
- Conditions
- β-thalassemia Major
- Keywords
- deferasirox
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Withdrawn
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Jun 2013
- Primary completion
- Apr 2014 (estimated)
- Study completion
- Oct 2014 (estimated)
- First posted
- 9 Nov 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To characterize the PK of deferasirox in pediatric β-thalassemia major patients aged from 2 to less than 6 years old, when administrated with a fixed starting dose of 20 mg/kg/day.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Deferasirox | Experimental | Drug: DeferasiroxThe study will provide PK, safety, tolerability and efficacy data collected during 48 weeks of treatment with deferasirox in Chinese pediatric patients with transfusion dependent -thalassemia major, aged 2 to <6 years at enrollment. The target patient pool consists of 20 patients with evidence of iron overload measured by serum ferritin level at the start of study. Patients will start their deferasirox treatment with a dose of 20 mg/kg/day. Serum ferritin will be monitored every month and the dose of deferasirox will be adjusted if necessary every 3 months based on the trends in serum ferritin. Other possible dose adjustments will be based on the patient's safety assessments. |
Who may qualify
- Ages
- 2 to 71 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Pediatric patients aged from 2 to less than 6 years old.
- Patients with transfusion dependent β-thalassemia major.
- Serum ferritin values ≥ 1000 ng/ml at screening.
- Written informed consent must be obtained from the patient's legal guardian in accordance with local law and regulation prior to any screening procedures.
Key exclusion criteria (10 listed)
- Non-transfusion-dependent thalassemia.
- Systemic diseases which would prevent study treatment (e.g. uncontrolled hypertension, cardiovascular, renal, hepatic, metabolic, etc.)
- Serum creatinine > age adjusted ULN.
- Significant proteinuria as indicated by a urinary protein/creatinine ratio (UPCR) ≥ 0.5mg/mg in a non-first void urine sample at screening. If UPCR is found to be ≥ 0.5 mg/mg the test can be repeated after 1 month.
- ALT/AST > 2.5xULN and total bilirubin > 1×ULN.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DeferasiroxPatients will start their deferasirox treatment with a dose of 20 mg/kg/day. | Drug | Deferasirox |
Primary outcome
the PK profile of deferasirox in pediatric β-thalassemia major patients aged from 2 to less than 6 years old.
Time frame: PK sampling times are 0.5, 2.5, 6, 10h in Day 1 postdose; Day 2, 3, 4 and 5 predose and 0.5, 2.5, 6, 10h post dose on Day 4, then pre-dose at each subsequent visit until Week 49. Day -1 PK sample will be treated as Day 1 predose sample.
Area under the plasma concentration-time curve from time zero to the end of the dosing interval.
the PK profile of deferasirox in pediatric β-thalassemia major patients aged from 2 to less than 6 years old: Cmax
Time frame: PK sampling times are 0.5, 2.5, 6, 10h in Day 1 postdose; Day 2, 3, 4 and 5 predose and 0.5, 2.5, 6, 10h post dose on Day 4, then pre-dose at each subsequent visit until Week 49. Day -1 PK sample will be treated as Day 1 predose sample.
The maximum plasma concentration of study medication.
the PK profile of deferasirox in pediatric β-thalassemia major patients aged from 2 to less than 6 years old: Tmax
Time frame: PK sampling times are 0.5, 2.5, 6, 10h in Day 1 postdose; Day 2, 3, 4 and 5 predose and 0.5, 2.5, 6, 10h post dose on Day 4, then pre-dose at each subsequent visit until Week 49. Day -1 PK sample will be treated as Day 1 predose sample.
Tmax was directly determined from the raw plasma concentration-time data.
2 secondary outcomes
The safety and tolerability of deferasirox following multiple dosing in pediatric β-thalassemia major patients.
Time frame: Baseline, every 4 weeks until 48 weeks after taking the drug
The efficacy of deferasirox in pediatric β-thalassemia patients as measured by change of serum ferritin.
Time frame: Baseline, every 4 weeks until 48 weeks after taking the drug
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Apr 2017.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Deferasirox in people with β-thalassemia Major, run by Novartis Pharmaceuticals.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 20 Apr 2017. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
the PK profile of deferasirox in pediatric β-thalassemia major patients aged from 2 to less than 6 years old. (time frame: PK sampling times are 0.5, 2.5, 6, 10h in Day 1 postdose; Day 2, 3, 4 and 5 predose and 0.5, 2.5, 6, 10h post dose on Day 4, then pre-dose at each subsequent visit until Week 49. Day -1 PK sample will be treated as Day 1 predose sample.). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Apr 2017.