Iron OverloadPhase 4NCT01335035
Open-Label Single-Arm Pilot Study in Adult Allogeneic Hematopoietic Stem Cell Transplant Recipients With Transfusional Iron Overload
A Phase 4 study testing deferasirox in people with Iron Overload, run by Novartis Pharmaceuticals at 9 sites in Spain.
Trial summary
This study has been designed to establish the efficacy and safety of deferasirox in patients with iron overload from 6 to 18 months after allogeneic hematopoietic stem cell transplant (HSCT). 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?
- deferasirox
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 9 sitesSpain
- Study dates
- Dec 2008to May 2011
- 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
- Male or female patients 18 years of age and older
- Patients to undergo allogeneic HSCT between 6 and 18 months prior to inclusion.
- Patients with screening ANC > 1000/mm3 and ferritin values ≥ 1000 ng/mL (ferritin values must be measured in two measurements at one-week intervals).
- Patients receiving at least 20 RBC concentrate units or 100mL/kg RBC during their lives.
- Patients giving their informed consent (prior to performing any study procedure)
Who cannot join
- Haemosiderosis not related to transfusion.
- Patients with concomitant active malignancy.
- Active known viral hepatitis or known HIV-positive.
- Mean levels of alanine aminotransferase (ALT) > 5x ULN
- Treatment with any iron chelating agent after allogeneic HSCT.
- Uncontrolled hypertension.
- Other protocol-defined inclusion/exclusion criteria may app
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
Spain9 sites
Novartis Investigative Site
Andalucia
Novartis Investigative Site
Asturias
Novartis Investigative Site
Canarias
Novartis Investigative Site
Castilla Y Leon
Novartis Investigative Site
Cataluna
Novartis Investigative Site
Comunidad Valenciana
Novartis Investigative Site
Islas Baleares
Novartis Investigative Site
Madrid
Novartis Investigative Site
Murcia
About this study
Official title: Open-Label Single-Arm Pilot Study of Deferasirox (Exjade®) in Adult Allogeneic Hematopoietic Stem Cell Transplant Recipients With Transfusional Iron Overload
This study has been designed to establish the efficacy and safety of deferasirox in patients with iron overload from 6 to 18 months after allogeneic hematopoietic stem cell transplant (HSCT).
The purpose of this study is to assess the mean change in serum ferritin after 52 weeks of treatment with deferasirox, in patients with iron overload (defined with serum ferritin levels ≥ 1000 ng/ml and receiving > 20 RBC concentrates) after allogeneic HSCT.
- Conditions
- Iron Overload
- Being tested
- deferasirox
- Keywords
- Iron metabolism,, Allogenetic Hematopoietic Stem Cell Transplant,, haematological diseases
- Medical subject headings
- Iron Overload
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
deferasiroxDrug
Also called ICL670
| Study group | Role | What they receive |
|---|---|---|
| ICL670 | Experimental | Not described Receives: Drug: deferasirox |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean change in serum ferritin
Measured: after 52 weeks of treatment with deferasirox
Secondary outcomes (5)
Mean change in the no. of sideroblasts, assessed by Perls staining
Measured: after 52 weeks of treatment with deferasirox
Mean change in liver iron concentration (LIC), assessed by liver MRI.
Measured: after 52 weeks of treatment with deferasirox
Incidence of chronic graft-versus-host disease ("limited" or "extensive", according to Shulman criteria)
Measured: up to 52 weeks of study
Incidence of infections (bacterial, viral, or fungal)
Measured: up to 52 weeks of study
Incidence of venous occlusive disease during the study
Measured: up to 52 weeks of study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Apr 2011 (estimated)
- Study startDec 2008
- Primary completionMay 2011
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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 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
- NCT01335035
- Sponsor's protocol ID
- CICL670AES04
- Other IDs
- EudraCT: 2008-003207-30
- Status last verified
- Apr 2012
- First submitted
- 30 Mar 2011
- Last updated
- 1 May 2012
- Results posted
- No
Publications
Vallejo C, Batlle M, Vazquez L, Solano C, Sampol A, Duarte R, Hernandez D, Lopez J, Rovira M, Jimenez S, Valcarcel D, Belloch V, Jimenez M, Jarque I; Subcommittee of Non-Infectious Complications of the Grupo Espanol de Trasplante Hematopoyetico (GETH). Phase IV open-label study of the efficacy and safety of deferasirox after allogeneic stem cell transplantation. Haematologica. 2014 Oct;99(10):1632-7. doi: 10.3324/haematol.2014.105908. Epub 2014 Jul 4.
PubMed 24997153
Study at a glance
From the registry record, last updated by the sponsor on 1 May 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open-Label Single-Arm Pilot Study of Deferasirox (Exjade®) in Adult Allogeneic Hematopoietic Stem Cell Transplant Recipients With Transfusional Iron Overload
- ClinicalTrials.gov ID
- NCT01335035
- Acronym / sponsor ID
- CICL670AES04
- Conditions
- Iron Overload
- Keywords
- Iron metabolism,, Allogenetic Hematopoietic Stem Cell Transplant,, haematological diseases
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Dec 2008
- Primary completion
- May 2011 (actual)
- First posted
- 13 Apr 2011 (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.
This study has been designed to establish the efficacy and safety of deferasirox in patients with iron overload from 6 to 18 months after allogeneic hematopoietic stem cell transplant (HSCT).
The purpose of this study is to assess the mean change in serum ferritin after 52 weeks of treatment with deferasirox, in patients with iron overload (defined with serum ferritin levels ≥ 1000 ng/ml and receiving > 20 RBC concentrates) after allogeneic HSCT.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ICL670 | Experimental | Drug: deferasirox |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Male or female patients 18 years of age and older
- Patients to undergo allogeneic HSCT between 6 and 18 months prior to inclusion.
- Patients with screening ANC > 1000/mm3 and ferritin values ≥ 1000 ng/mL (ferritin values must be measured in two measurements at one-week intervals).
- Patients receiving at least 20 RBC concentrate units or 100mL/kg RBC during their lives.
- Patients giving their informed consent (prior to performing any study procedure)
Key exclusion criteria (7 listed)
- Haemosiderosis not related to transfusion.
- Patients with concomitant active malignancy.
- Active known viral hepatitis or known HIV-positive.
- Mean levels of alanine aminotransferase (ALT) > 5x ULN
- Treatment with any iron chelating agent after allogeneic HSCT.
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 |
|---|---|---|
| deferasiroxAlso: ICL670 | Drug | ICL670 |
Primary outcome
Mean change in serum ferritin
Time frame: after 52 weeks of treatment with deferasirox
5 secondary outcomes
Mean change in the no. of sideroblasts, assessed by Perls staining
Time frame: after 52 weeks of treatment with deferasirox
Mean change in liver iron concentration (LIC), assessed by liver MRI.
Time frame: after 52 weeks of treatment with deferasirox
Incidence of chronic graft-versus-host disease ("limited" or "extensive", according to Shulman criteria)
Time frame: up to 52 weeks of study
Incidence of infections (bacterial, viral, or fungal)
Time frame: up to 52 weeks of study
Incidence of venous occlusive disease during the study
Time frame: up to 52 weeks of study
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 1 May 2012.
- Derived from this study: PubMed 24997153Vallejo C, Batlle M, Vazquez L, Solano C, Sampol A, Duarte R, Hernandez D, Lopez J, Rovira M, Jimenez S, Valcarcel D, Belloch V, Jimenez M, Jarque I; Subcommittee of Non-Infectious Complications of the Grupo Espanol de Trasplante Hematopoyetico (GETH). Phase IV open-label study of the efficacy and safety of deferasirox after allogeneic stem cell transplantation. Haematologica. 2014 Oct;99(10):1632-7. doi: 10.3324/haematol.2014.105908. Epub 2014 Jul 4.
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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
9 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 deferasirox in people with Iron Overload, run by Novartis Pharmaceuticals at 9 sites in Spain.
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 5 inclusion and 7 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 1 May 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in Spain. 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?
Mean change in serum ferritin (time frame: after 52 weeks of treatment with deferasirox). 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 NCT01335035, 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 1 May 2012.