Iron OverloadPhase 4NCT01546415
Safety and Efficacy of Desferasirox in Chinese Patients With Iron Overload and Aplastic Anemia
A Phase 4 study testing Desferasirox in people with Iron Overload, run by Novartis Pharmaceuticals at 7 sites in China.
Trial summary
The study is designed to collect safety and efficacy of Desferasirox in Chinese patients with Iron Overload and Aplastic Anemia. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Iron Overload
- What is being tested?
- Desferasirox
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 64 participants
- Where
- 7 sitesChina
- Study dates
- Oct 2011to Nov 2013
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients presenting with transfusion-related IOL as shown by a SF level of ≥ 1000 ng/mL at start of study
- History of transfusion >20 international units or 100 mL/kg of red blood cells
- Underlying transfusion-dependent illness:
- AA: Clinically diagnosed AA with bone marrow confirmation of the diagnosis
- Age ≥ 18 years, male or female
- Written informed consent obtained from patient prior to any screening procedures.
Who cannot join
- Patients with serum creatinine > ULN or with significant proteinuria as indicated by a urinary protein/creatinine ratio (UPCR) ≥ 1.0 mg/mg in a non-first void urine sample at baseline. If serum creatinine is found to be > ULN or UPCR is found to be ≥ 1 mg/mg the test can be repeated after 1 month.
- Creatinine Clearance <40 ml/min
- Patients with other than AA transfusion-dependent underlying illnesses
- Patients with a previous history of clinically relevant ocular and/or auditory toxicity related to iron chelation
- Any other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of any drug. The investigator should be guided by evidence of any of the following:
- history of inflammatory bowel syndrome, gastritis, ulcers, gastrointestinal or rectal bleeding
Show all 11Show fewer
- history of major gastrointestinal tract surgery such as gastrectomy, gastroenterostomy, or bowel resection
- history of pancreatic injury or pancreatitis; indications of impaired pancreatic function/injury as indicated by abnormal lipase or amylase
- history or presence of impaired renal function as indicated by creatinine or blood urea nitrogen (BUN) values equal or above ULN
- history of urinary obstruction or difficulty in voiding
- Other protocol-defined inclusion/exclusion criteria may apply
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
China7 sites
Novartis Investigative Site
Beijing
Novartis Investigative Site
Chengdu, Sichuan, 610041
Novartis Investigative Site
Chengdu
Novartis Investigative Site
Shanghai, Shanghai Municipality, 200437
Novartis Investigative Site
Shanghai
Novartis Investigative Site
Tianjin, Tianjin Municipality, 300020
Novartis Investigative Site
Tianjin
About this study
Official title: A Single Arm, Multicenter, Open Label Study of Desferasirox in Chinese Patients With Iron Overload and Aplastic Anemia
The study is designed to collect safety and efficacy of Desferasirox in Chinese patients with Iron Overload and Aplastic Anemia.
- Conditions
- Iron Overload
- Being tested
- Desferasirox
- Keywords
- Phase 4, Desferasirox, Iron Overload, Aplastic Anemia
- Medical subject headings
- Iron Overload, Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DesferasiroxDrug
Initial Dose: 20 mg/kg/d. Dose modification according to protocol.
| Study group | Role | What they receive |
|---|---|---|
| Desferasirox | Experimental | Not described Receives: Drug: Desferasirox |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in serum ferritin from baseline to 52 weeks with desferasirox treatment in Chinese patients with AA presenting with transfusional hemosiderosis and pre-existing serum ferritin levels of ≥ 1000 ng/mL.
Difference in serum ferritin from baseline vs. 52 weeks of treatment.
Measured: Every 4 Weeks for 52 weeks of treatment
Secondary outcomes (4)
Correlation between serum ferritin (SF) and transferrin saturation (TFS)
Study will compare SF with TFS level to find degree of relationship measured by correlation.
Measured: Every 4 Weeks for 52 weeks of treatment
Number of dispensed, used, partially used or unused packages of study medication to assess drug usage compliance
Measured: Every 4 Weeks for 52 weeks of treatment
Correlation between dose adjustment (increase or decrease) regimens and transfusional burden
Evaluation of the relationship between dose adjustment regimens (as dictated by both efficacy and safety parameters) and transfusional burden will be undertaken.
Measured: Every 3 months for 52 Weeks of treatment
Number of patients with adverse events, serious adverse events and death
Measured: Every 4 weeks for 52 Weeks of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Mar 2012 (estimated)
- Study startOct 2011
- Primary completionNov 2013
- Study completionNov 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Iron OverloadStudies 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
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 64 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
- NCT01546415
- Sponsor's protocol ID
- CICL670ACN01
- Status last verified
- Oct 2014
- First submitted
- 10 Nov 2011
- Last updated
- 28 Oct 2014
- Results posted
- No
Publications
Shi J, Chang H, Zhang L, Shao Y, Nie N, Zhang J, Huang J, Zhang L, Tang X, Quan R, Zheng C, Xiao H, Hu D, Hu L, Liu F, Zhou Y, Zheng Y, Zhang F. [Efficacy and safety of deferasirox in aplastic anemia patients with iron overload: a single arm, multi-center,prospective study in China]. Zhonghua Xue Ye Xue Za Zhi. 2016 Jan;37(1):1-6. doi: 10.3760/cma.j.issn.0253-2727.2016.01.001. Chinese.
PubMed 26876245
Study at a glance
From the registry record, last updated by the sponsor on 28 Oct 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single Arm, Multicenter, Open Label Study of Desferasirox in Chinese Patients With Iron Overload and Aplastic Anemia
- ClinicalTrials.gov ID
- NCT01546415
- Acronym / sponsor ID
- CICL670ACN01
- Conditions
- Iron Overload
- Keywords
- Phase 4, Desferasirox, Iron Overload, Aplastic Anemia
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 64 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Oct 2011
- Primary completion
- Nov 2013 (actual)
- Study completion
- Nov 2013 (actual)
- First posted
- 7 Mar 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.
The study is designed to collect safety and efficacy of Desferasirox in Chinese patients with Iron Overload and Aplastic Anemia.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Desferasirox | Experimental | Drug: Desferasirox |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Patients presenting with transfusion-related IOL as shown by a SF level of ≥ 1000 ng/mL at start of study
- History of transfusion >20 international units or 100 mL/kg of red blood cells
- Underlying transfusion-dependent illness:
- Age ≥ 18 years, male or female
- Written informed consent obtained from patient prior to any screening procedures.
Key exclusion criteria (11 listed)
- Patients with serum creatinine > ULN or with significant proteinuria as indicated by a urinary protein/creatinine ratio (UPCR) ≥ 1.0 mg/mg in a non-first void urine sample at baseline. If serum creatinine is found to be > ULN or UPCR is found to be ≥ 1 mg/mg the test can be repeated after 1 month.
- Creatinine Clearance <40 ml/min
- Patients with other than AA transfusion-dependent underlying illnesses
- Patients with a previous history of clinically relevant ocular and/or auditory toxicity related to iron chelation
- Any other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of any drug. The investigator should be guided by evidence of any of the following:
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 |
|---|---|---|
| DesferasiroxInitial Dose: 20 mg/kg/d. Dose modification according to protocol. | Drug | Desferasirox |
Primary outcome
Change in serum ferritin from baseline to 52 weeks with desferasirox treatment in Chinese patients with AA presenting with transfusional hemosiderosis and pre-existing serum ferritin levels of ≥ 1000 ng/mL.
Time frame: Every 4 Weeks for 52 weeks of treatment
Difference in serum ferritin from baseline vs. 52 weeks of treatment.
4 secondary outcomes
Correlation between serum ferritin (SF) and transferrin saturation (TFS)
Time frame: Every 4 Weeks for 52 weeks of treatment
Number of dispensed, used, partially used or unused packages of study medication to assess drug usage compliance
Time frame: Every 4 Weeks for 52 weeks of treatment
Correlation between dose adjustment (increase or decrease) regimens and transfusional burden
Time frame: Every 3 months for 52 Weeks of treatment
Number of patients with adverse events, serious adverse events and death
Time frame: Every 4 weeks for 52 Weeks of treatment
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Oct 2014.
- Derived from this study: PubMed 26876245Shi J, Chang H, Zhang L, Shao Y, Nie N, Zhang J, Huang J, Zhang L, Tang X, Quan R, Zheng C, Xiao H, Hu D, Hu L, Liu F, Zhou Y, Zheng Y, Zhang F. [Efficacy and safety of deferasirox in aplastic anemia patients with iron overload: a single arm, multi-center,prospective study in China]. Zhonghua Xue Ye Xue Za Zhi. 2016 Jan;37(1):1-6. doi: 10.3760/cma.j.issn.0253-2727.2016.01.001. Chinese.
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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
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Desferasirox in people with Iron Overload, run by Novartis Pharmaceuticals at 7 sites in China.
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 6 inclusion and 11 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: finished, as of the sponsor's last update on 28 Oct 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Change in serum ferritin from baseline to 52 weeks with desferasirox treatment in Chinese patients with AA presenting with transfusional hemosiderosis and pre-existing serum ferritin levels of ≥ 1000 ng/mL. (time frame: Every 4 Weeks for 52 weeks of treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01546415, which also lists 1 linked publication.
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 28 Oct 2014.