Systemic MastocytosisPhase 2/3NCT01602939
Cladribine Plus Pegylated Interpheron Alfa-2a in Systemic Mastocytosis
A Phase 2/3 study testing Cladribine and pegylated interpheron alpha-2a in people with Systemic Mastocytosis, run by Hospital Virgen de la Salud at 1 site in Spain.
Trial summary
The aim of this study is to evaluate the efficacy in terms of clinical and biological response rates of Cladribine plus Pegylated Interpheron alpha-2a therapy in patients with advanced systemic mastocytosis carrying D816V or other exon 17 KIT mutations. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Systemic Mastocytosis
- What is being tested?
- Cladribine and pegylated interpheron alpha-2a
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteSpain
- Study dates
- May 2012to Jun 2017
- Phase
- Phase 2/3
- 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
- Age older than 18 years.
- Diagnosis of advanced systemic mastocytosis (aggressive systemic mastocytosis or proggressing systemic mastocytosis) with D816V or other exon 17 KIT mutations.
- ECOG ≤ 3.
- Signed informed consent.
Who cannot join
- Impaired liver function (total bilirubin ≥ 2.0 mg/dl, AST or ALT > 3 x upper limit of normal)not related to mastocytosis.
- Impaired renal function (≥ 2.0 mg/dL)not related to mastocytosis.
- Grade III-IV cytopenias not related to mastocytosis. Severe cardiopathy (grade III/IV of NYHA, or left ventricular ejection fraction < 50%).
- Pregnancy or breastfeeding.
- Female patients who do not use contraceptive methods.
Trial locations and contacts
1 site in 1 country.
Spain1 site, 1 recruiting
- Recruiting
Instituto de Estudios de Mastocitosis de Castilla La Mancha; Hospital Virgen del Valle
Toledo, Toledo, 45071
Luis Escribano, MD, PhD, +34925269335, lescribanom@sescam.jccm.es
Iván Alvarez-Twose, MD, +34925269336, ivana@sescam.jccm.es
About this study
Official title: Subcutaneous Cladribine Plus Pegylated Interpheron Alfa-2a in Advanced Systemic Mastocytosis With D816V and Other Exon 17 KIT Mutations.
The aim of this study is to evaluate the efficacy in terms of clinical and biological response rates of Cladribine plus Pegylated Interpheron alpha-2a therapy in patients with advanced systemic mastocytosis carrying D816V or other exon 17 KIT mutations.
- Conditions
- Systemic Mastocytosis
- Being tested
- Cladribine and pegylated interpheron alpha-2a
- Keywords
- Mast cell, Mastocytosis, Mast cell disease
- Medical subject headings
- Mastocytosis, Systemic, Mastocytosis, Mast Cell Activation Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cladribine and pegylated interpheron alpha-2aDrug
Cladribine (0.07 mg/Kg/day) s.c for 5 consecutive days each month for a total of 6 months.Cladribine daily doses could be increased up to 0.14 mg/Kg in the fourth, fifth and sixth cycles of therapy if no objetive response is achieved after the third cycle.
Pegylated Interpheron alpha-2a (1 mcgr/Kg) s.c weekly for a total of 6 months.
| Study group | Role | What they receive |
|---|---|---|
| 2CDA+IFN | Experimental | Not described Receives: Drug: Cladribine and pegylated interpheron alpha-2a |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the effect of therapy on bone marrow mast cell infiltration.
Evaluation of bone marrow response will be assessed by immunohistochemestry, citology, flow cytometry and molecular analyses of bone marrow samples.
Measured: 6 months
Secondary outcomes (6)
To determine the effect of therapy on serum tryptase levels and other altered peripheral blood parameters due to mastocytosis.
Serum tryptase and any other mastocytosis-related altered biochemical parameter at diagnosis will be measured monthly until the end of therapy.
Measured: 6 months
To evaluate the effect of therapy on mast cell-mediator release symptoms: pruritus, flushing, gastrointestinal symptoms or anaphylaxis).
Specific questionnaires regarding mast cell-mediator release symptoms will be filled monthly by each patient until the end of therapy.
Measured: 6 months
To determine de safety of combined therapy with low doses of cladribine plus pegylated interpheron alpha-2a.
Potentially drugs-related adverse events will be recorded in each case following accepted criteria (NIH CTCAE).
Measured: 6 months
To evaluate the effect of therapy on mastocytosis skin lesions.
Evaluation of cutaneous response will be assessed by macroscopic inspection including photographs and by skin immunohistochemestry.
Measured: 6 moths
To evaluate the effect of therapy on mastocytosis-related organomegalies.
Evaluation of organomegalies response will be assessed by abdominal ultrasound and/or computerized tomography.
Measured: 6 months
To evaluate the effect of therapy on mastocytosis-related bone alterations.
Evaluation of bone response will be assessed by X-ray survey and/or computerized tomography.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 May 2012 (estimated)
- Study startMay 2012
- Primary completionFeb 2017 (estimated)
- Study completionJun 2017 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Systemic MastocytosisStudies 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 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hospital Virgen de la Salud
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, LUIS ESCRIBANO, Hospital Virgen de la Salud
- Investigators
- Luis Escribano, MD, PhD, Principal investigator, Instituto de Estudios de Mastocitosis de Castilla La Mancha
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01602939
- Sponsor's protocol ID
- EC11-187
- Status last verified
- Aug 2016
- First submitted
- 16 May 2012
- Last updated
- 29 Aug 2016
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 29 Aug 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Subcutaneous Cladribine Plus Pegylated Interpheron Alfa-2a in Advanced Systemic Mastocytosis With D816V and Other Exon 17 KIT Mutations.
- ClinicalTrials.gov ID
- NCT01602939
- Acronym / sponsor ID
- EC11-187
- Conditions
- Systemic Mastocytosis
- Keywords
- Mast cell, Mastocytosis, Mast cell disease
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Hospital Virgen de la SaludUniversity, hospital or other
- Study start
- May 2012
- Primary completion
- Feb 2017 (estimated)
- Study completion
- Jun 2017 (estimated)
- First posted
- 21 May 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 aim of this study is to evaluate the efficacy in terms of clinical and biological response rates of Cladribine plus Pegylated Interpheron alpha-2a therapy in patients with advanced systemic mastocytosis carrying D816V or other exon 17 KIT mutations.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 2CDA+IFN | Experimental | Drug: Cladribine and pegylated interpheron alpha-2a |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Age older than 18 years.
- Diagnosis of advanced systemic mastocytosis (aggressive systemic mastocytosis or proggressing systemic mastocytosis) with D816V or other exon 17 KIT mutations.
- ECOG ≤ 3.
- Signed informed consent.
Key exclusion criteria (5 listed)
- Impaired liver function (total bilirubin ≥ 2.0 mg/dl, AST or ALT > 3 x upper limit of normal)not related to mastocytosis.
- Impaired renal function (≥ 2.0 mg/dL)not related to mastocytosis.
- Grade III-IV cytopenias not related to mastocytosis. Severe cardiopathy (grade III/IV of NYHA, or left ventricular ejection fraction < 50%).
- Pregnancy or breastfeeding.
- Female patients who do not use contraceptive methods.
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 |
|---|---|---|
| Cladribine and pegylated interpheron alpha-2aCladribine (0.07 mg/Kg/day) s.c for 5 consecutive days each month for a total of 6 months.Cladribine daily doses could be increased up to 0.14 mg/Kg in the fourth, fifth and sixth cycles of therapy if no objetive response is achieved after the third cycle. Pegylated Interpheron alpha-2a (1 mcgr/Kg) s.c weekly for a total of 6 months. | Drug | 2CDA+IFN |
Primary outcome
To evaluate the effect of therapy on bone marrow mast cell infiltration.
Time frame: 6 months
Evaluation of bone marrow response will be assessed by immunohistochemestry, citology, flow cytometry and molecular analyses of bone marrow samples.
6 secondary outcomes
To determine the effect of therapy on serum tryptase levels and other altered peripheral blood parameters due to mastocytosis.
Time frame: 6 months
To evaluate the effect of therapy on mast cell-mediator release symptoms: pruritus, flushing, gastrointestinal symptoms or anaphylaxis).
Time frame: 6 months
To determine de safety of combined therapy with low doses of cladribine plus pegylated interpheron alpha-2a.
Time frame: 6 months
To evaluate the effect of therapy on mastocytosis skin lesions.
Time frame: 6 moths
To evaluate the effect of therapy on mastocytosis-related organomegalies.
Time frame: 6 months
To evaluate the effect of therapy on mastocytosis-related bone alterations.
Time frame: 6 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Aug 2016.
- Individual participant dataSharing planned: Undecided
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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.
Luis Escribano, MD, PhD principal investigator
Instituto de Estudios de Mastocitosis de Castilla La Mancha
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Cladribine and pegylated interpheron alpha-2a in people with Systemic Mastocytosis, run by Hospital Virgen de la Salud at 1 site 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 4 inclusion and 5 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: status not confirmed, as of the sponsor's last update on 29 Aug 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 combined 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.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Luis Escribano, MD, PhD (+34925269335, lescribanom@sescam.jccm.es), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To evaluate the effect of therapy on bone marrow mast cell infiltration. (time frame: 6 months). 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 29 Aug 2016.