Hypereosinophilic SyndromePhase 2NILG-HES1-03NCT00787384
Efficacy of Imatinib Mesylate in Hypereosinophilic Syndromes
A Phase 2 study testing Imatinib in people with Hypereosinophilic Syndrome and Chronic Eosinophilic Leukemia, run by Northern Italy Leukemia Group at 4 sites in Italy.
Trial summary
The study was performed to assess: 1) clinical activity of Imatinib in patients with HES, CEL and CIH; 2) correlation between Imatinib activity and specific disease subtype; 3) long-term outcome of HES, CEL and CIH patients treated with Imatinib; 4) safety and tolerability of Imatinib administration. Read more about this study
- Who is it for?
- Ages 15 years and older
- Conditions the study is for
- Hypereosinophilic Syndrome, Chronic Eosinophilic Leukemia, Chronic Idiopathic Hypereosinophilia
- What is being tested?
- Imatinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 4 sitesItaly
- Study dates
- Oct 2004to Dec 2007
- Phase
- Phase 2
- 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 15 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- patients with a diagnosis of HES, CEL and CIH, who are either previously untreated or have been treated with corticosteroids, cytotoxic drugs, and IFN.
- age > 15 years.
- signature of a written informed consent(by parents/tutors for patients aged < 18 years).
Who cannot join
- patients with a diagnosis of secondary hypereosinophilia
- age < 15 years
Trial locations and contacts
4 sites in 1 country.
Italy4 sites
USC Ematologia Ospedali Riuniti di Bergamo
Bergamo, Bergamo, 24128
Divisione di Ematologia Spedali Civili di Brescia
Brescia, Brescia
USC Ematologia Azienda Ospedaliera Università Careggi
Florence, Firenze, 50134
UO Ematologia, Azienda Ospedaliera ULSS6
Vicenza, Vicenza, 36100
About this study
Official title: Therapeutic and Biological Effects of Imatinib Mesylate in Primary Hypereosinophilic Syndromes
The study was performed to assess: 1) clinical activity of Imatinib in patients with HES, CEL and CIH; 2) correlation between Imatinib activity and specific disease subtype; 3) long-term outcome of HES, CEL and CIH patients treated with Imatinib; 4) safety and tolerability of Imatinib administration.
Read the detailed description
Hypereosinophilic syndrome (HES), chronic eosinophilic leukaemia (CEL) and chronic idiopathic hypereosinophilia (CIH) are rare disorders characterized by chronic hypereosinophilia with possible damage to various organs due to eosinophilic infiltration and release of cytokines. The therapies of these diseases are largely unsatisfactory and based on the use of a variety of antiproliferative drugs such as corticosteroids, interferon-alfa, cyclosporine, vincristine or hydroxyurea. More often the responses are transient and patients need numerous treatment lines.
In 2001 Schaller et al reported the first case of a patient with HES resistant to conventional treatment that responded to imatinib mesylate. (Schaller, MGM 2001). After that, many authors described cases with hypereosinophilia that achieve a rapid response to Imatinib and in 2003 Cools et al identified a novel tyrosine kinase generated from the fusion of the Fip1-like 1 (FIP1L1) gene to the PDGFRalfa gene associated to hypereosinophilia.
The optimal dose of Imatinib in this setting of patients is still unknown; however, the demonstration of effective and safe clinical doses in a variety of currently studied malignant diseases, suggests that a dose of 100 mg/day increasing weekly of 100 mg/day (maximum dose 400 mg/day), may be employed.
We designed a phase II trial to investigate the clinical anti-proliferative activity, safety and tolerability of escalating doses of Imatinib (entry dose 100 mg/d)administered for 12 total weeks in HES, CEL and CIH patients.
- Conditions
- Hypereosinophilic Syndrome, Chronic Eosinophilic Leukemia, Chronic Idiopathic Hypereosinophilia
- Being tested
- Imatinib
- Keywords
- Hypereosinophilic syndrome (HES), chronic eosinophilic leukaemia (CEL), chronic idiopathic hypereosinophilia (CIH), Imatinib, Response, Timing to response
- Medical subject headings
- Hypereosinophilic Syndrome, Pdgfra-Associated Chronic Eosinophilic Leukemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ImatinibDrug
Also called Gleevec
Patients received oral imatinib 100 mg/d; in case of unsatisfactory response (less than complete) Imatinib could be increased by 100 mg/die on a weekly basis and up to a maximum of 400 mg/die. Imatinib wsa discontinued after 12 total weeks of therapy.
| Study group | Role | What they receive |
|---|---|---|
| Imatinib | Experimental | Patients received oral imatinib 100 mg/d; in case of unsatisfactory response (less than complete) Imatinib could be increased by 100 mg/die on a weekly basis and up to a maximum of 400 mg/die. Imatinib was discontinued after 12 total weeks of therapy. Receives: Drug: Imatinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Response rate
Secondary outcomes (4)
Safety: Adverse events and serious adverse events
Time to response
Diagnostic profile of Imatinib-responsive cases
Duration of responses following drug withdrawal after 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Nov 2008 (estimated)
- Study startOct 2004
- Primary completionDec 2007
- Study completionDec 2007
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 Hypereosinophilic SyndromeStudies of the same condition, open and under way.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Northern Italy Leukemia Group
- Sponsor type
- University, hospital or other
- Investigators
- Renato Bassan, MD, Principal investigator, USC Ematologia Ospedali Riuniti di Bergamo
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00787384
- Sponsor's protocol ID
- NILG-HES1-03
- Other IDs
- EUDRACT 2004-002280-24
- Acronym
- NILG-HES1-03
- Status last verified
- Dec 2010
- First submitted
- 6 Nov 2008
- Last updated
- 29 Dec 2010
- Results posted
- No
- Data monitoring committee
- No
Publications
Intermesoli T, Delaini F, Acerboni S, Salmoiraghi S, Spinelli O, Guerini V, Vannucchi AM, Mappa S, Rossi G, Rossi V, Di Bona E, Paratore S, Carobbio A, Rambaldi A, Barbui T, Bassan R. A short low-dose imatinib trial allows rapid identification of responsive patients in hypereosinophilic syndromes. Br J Haematol. 2009 Dec;147(5):681-5. doi: 10.1111/j.1365-2141.2009.07893.x. Epub 2009 Sep 4.
PubMed 19735261
Study at a glance
From the registry record, last updated by the sponsor on 29 Dec 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Therapeutic and Biological Effects of Imatinib Mesylate in Primary Hypereosinophilic Syndromes
- ClinicalTrials.gov ID
- NCT00787384
- Acronym / sponsor ID
- NILG-HES1-03 / NILG-HES1-03
- Conditions
- Hypereosinophilic Syndrome, Chronic Eosinophilic Leukemia, Chronic Idiopathic Hypereosinophilia
- Keywords
- Hypereosinophilic syndrome (HES), chronic eosinophilic leukaemia (CEL), chronic idiopathic hypereosinophilia (CIH), Imatinib, Response, Timing to response
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Northern Italy Leukemia GroupUniversity, hospital or other
- Study start
- Oct 2004
- Primary completion
- Dec 2007 (actual)
- Study completion
- Dec 2007 (actual)
- First posted
- 7 Nov 2008 (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 was performed to assess: 1) clinical activity of Imatinib in patients with HES, CEL and CIH; 2) correlation between Imatinib activity and specific disease subtype; 3) long-term outcome of HES, CEL and CIH patients treated with Imatinib; 4) safety and tolerability of Imatinib administration.
Detailed description
Hypereosinophilic syndrome (HES), chronic eosinophilic leukaemia (CEL) and chronic idiopathic hypereosinophilia (CIH) are rare disorders characterized by chronic hypereosinophilia with possible damage to various organs due to eosinophilic infiltration and release of cytokines. The therapies of these diseases are largely unsatisfactory and based on the use of a variety of antiproliferative drugs such as corticosteroids, interferon-alfa, cyclosporine, vincristine or hydroxyurea. More often the responses are transient and patients need numerous treatment lines.
In 2001 Schaller et al reported the first case of a patient with HES resistant to conventional treatment that responded to imatinib mesylate. (Schaller, MGM 2001). After that, many authors described cases with hypereosinophilia that achieve a rapid response to Imatinib and in 2003 Cools et al identified a novel tyrosine kinase generated from the fusion of the Fip1-like 1 (FIP1L1) gene to the PDGFRalfa gene associated to hypereosinophilia.
The optimal dose of Imatinib in this setting of patients is still unknown; however, the demonstration of effective and safe clinical doses in a variety of currently studied malignant diseases, suggests that a dose of 100 mg/day increasing weekly of 100 mg/day (maximum dose 400 mg/day), may be employed.
We designed a phase II trial to investigate the clinical anti-proliferative activity, safety and tolerability of escalating doses of Imatinib (entry dose 100 mg/d)administered for 12 total weeks in HES, CEL and CIH patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Imatinib | Experimental | Drug: ImatinibPatients received oral imatinib 100 mg/d; in case of unsatisfactory response (less than complete) Imatinib could be increased by 100 mg/die on a weekly basis and up to a maximum of 400 mg/die. Imatinib was discontinued after 12 total weeks of therapy. |
Who may qualify
- Ages
- 15 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- patients with a diagnosis of HES, CEL and CIH, who are either previously untreated or have been treated with corticosteroids, cytotoxic drugs, and IFN.
- age > 15 years.
- signature of a written informed consent(by parents/tutors for patients aged < 18 years).
Key exclusion criteria (2 listed)
- patients with a diagnosis of secondary hypereosinophilia
- age < 15 years
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 |
|---|---|---|
| ImatinibAlso: GleevecPatients received oral imatinib 100 mg/d; in case of unsatisfactory response (less than complete) Imatinib could be increased by 100 mg/die on a weekly basis and up to a maximum of 400 mg/die. Imatinib wsa discontinued after 12 total weeks of therapy. | Drug | Imatinib |
Primary outcome
Response rate
4 secondary outcomes
Safety: Adverse events and serious adverse events
Time to response
Diagnostic profile of Imatinib-responsive cases
Duration of responses following drug withdrawal after 12 weeks
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 29 Dec 2010.
- Results publication: PubMed 19735261Intermesoli T, Delaini F, Acerboni S, Salmoiraghi S, Spinelli O, Guerini V, Vannucchi AM, Mappa S, Rossi G, Rossi V, Di Bona E, Paratore S, Carobbio A, Rambaldi A, Barbui T, Bassan R. A short low-dose imatinib trial allows rapid identification of responsive patients in hypereosinophilic syndromes. Br J Haematol. 2009 Dec;147(5):681-5. doi: 10.1111/j.1365-2141.2009.07893.x. Epub 2009 Sep 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.
Renato Bassan, MD principal investigator
USC Ematologia Ospedali Riuniti di Bergamo
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Imatinib in people with Hypereosinophilic Syndrome and Chronic Eosinophilic Leukemia, run by Northern Italy Leukemia Group at 4 sites in Italy.
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 3 inclusion and 2 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 29 Dec 2010. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Italy. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Response rate. 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 NCT00787384, 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 29 Dec 2010.