Indolent Systemic MastocytosisPhase 2NCT01920204
Midostaurin in Indolent Systemic Mastocytosis
A Phase 2 study testing Midostaurin, in people with Indolent Systemic Mastocytosis, run by University Medical Center Groningen at 1 site in Netherlands.
Trial summary
Rationale: Patients with indolent or smoldering systemic mastocytosis can have severe disabling symptoms. Almost all patients have fatigue, a compromised quality of life, hampering normal functioning. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Indolent Systemic Mastocytosis
- What is being tested?
- Midostaurin,
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteNetherlands
- Study dates
- Aug 2013to May 2015
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients with Indolent Systemic Mastocytosis (ISM) or Smouldering Systemic Mastocytosis (SSM) according to the WHO criteria
- Presence of the D816V c-KIT mutation
- Serum tryptase > 20 mg/l
- Serious mediator-related symptoms that cannot be controlled by H1 and H2 blocking drugs. Symptoms will be scored by an adapted MSAF (mastocytosis symptom assessment form) with at least:
- a pre-study score of 4 or more on 3 non-related items,
- or a pre-study score of 5 or more on 2 non-related items.
Show all 10Show fewer
- one item from the scoring list can be replaced by flushes 7 or more per week or anaphylactic attacks 1 or more per week.
- Age >18 years
- Willingness to apply optimal contraceptive measures (double barrier method, both men and women) for women below the age of 55, men at all ages; for both: if sexually active.
- Written informed consent
Who cannot join
- Aggressive systemic mastocytosis, mast cell leukemia, or ASM with or without accompanying non-clonal related non-mast cell disorder (SM-ANHMD).
- Any known other present malignancy, non-melanoma skin cancers excluded
- History of malignancy within the last 5 years, non-melanoma skin cancers excluded
- Any serious comorbidity interfering with therapy compliance and follow-up compliance
- Pregnancy
- Patients not willing or who are not able to comply with contraceptive measures
Trial locations and contacts
1 site in 1 country.
Netherlands1 site
University Medical Center Groningen
Groningen, Provincie Groningen, 9700RB
About this study
Official title: Phase II Single Arm Open Pilot Study to Demonstrate the Efficacy of Midostaurin in Symptom Improvement and Decrease of Mast Cell Burden in Patients With Indolent or Smoldering Systemic Mastocytosis.
Rationale: Patients with indolent or smoldering systemic mastocytosis can have severe disabling symptoms. Almost all patients have fatigue, a compromised quality of life, hampering normal functioning. Because this form of mastocytosis is not considered life-threatening, mast cell eradication has never been applied and patients receive only symptomatic therapy with histamine blockers. Midostaurin, a c-KIT inhibitor has shown activity regarding symptom control and decrease of malignant mast cells in patients with aggressive systemic mastocytosis (ASM) or mast cell leukemia
Read the detailed description
Objective:
Primary: To study in a pilot phase II trial the efficacy of midostaurin administered at an oral dose of 100 mg twice daily in patients with indolent or smoldering systemic mastocytosis on mediator symptom reduction, documented by the Mastocytosis Symptom Assessment Questionnaire, measured at 3 months.
Secondary:
- To study whether symptom improvement persists at 6 months, and whether midostaurin can reduce mast cell infiltration in the skin and bone marrow, documented by decrease of serum tryptase, decrease of urticaria pigmentosa and decrease of bone marrow mast cells.
- To assess safety and tolerability of midostaurin in the above mentioned settings
Study design: Single arm, open label pilot phase II study.
Study population: Adult patients (n=20) with histologically documented systemic mastocytosis, indolent or smoldering subtype, with severe symptoms, not controlled by histamine 1 and 2 blockers.
Intervention: treatment with Midostaurin, twice daily 100 mg orally for 6 months continuously.
- Conditions
- Indolent Systemic Mastocytosis
- Being tested
- Midostaurin,
- Keywords
- Mastocytosis, ISM, SSM
- Medical subject headings
- Mastocytosis, Systemic, Mastocytosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Midostaurin,Drug
Also called PKC412
Midostaurin, twice daily 100 mg orally, continuously for 6 months
| Study group | Role | What they receive |
|---|---|---|
| Midostaurin | Experimental | Treatment with Midostaurin, twice daily 100 mg orally for 6 months continuously. Receives: Drug: Midostaurin, |
What is being measured
The primary outcome is the main question the study is designed to answer.
Symptom Scoring
Percent change in the total score ("Sumscore") of all symptoms assessed by the Mastocytosis Symptom Assessment Form (MSAF) after 12 weeks.
Measured: 12 weeks
Secondary outcomes (3)
Persistence of improvements
persistence of improvement symptom score at 6 months.
Measured: 6 months
Mast cell burden
Percent change in the mast cell burden (bone marrow infiltrate, skin infiltrate, serum tryptase levels) after 6 months.
Measured: 6 months
Adverse events
Number and grading of Common Terminology Criteria adverse events during the 6 months of therapy.
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 posted9 Aug 2013 (estimated)
- Study startAug 2013
- Primary completionMar 2015 (estimated)
- Study completionMay 2015 (estimated)
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 Indolent Systemic MastocytosisStudies of the same condition, open and under way.
- More trials run by University Medical Center GroningenThe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Medical Center Groningen
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Prof.dr. J.C. Kluin-Nelemans, University Medical Center Groningen
- Investigators
- J.C. Kluin-Nelemans, MD, PhD, Principal investigator, University Medical Center Groningen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01920204
- Sponsor's protocol ID
- UMCG41973
- Status last verified
- Jan 2015
- First submitted
- 7 Aug 2013
- Last updated
- 19 Jan 2015
- Results posted
- No
- Data monitoring committee
- Yes
Publications
van Anrooij B, Oude Elberink JNG, Span LFR, de Monchy JGR, Rosati S, Mulder AB, Kluin-Nelemans JC. Midostaurin in patients with indolent systemic mastocytosis: An open-label phase 2 trial. J Allergy Clin Immunol. 2018 Sep;142(3):1006-1008.e7. doi: 10.1016/j.jaci.2018.06.003. Epub 2018 Jun 8. No abstract available.
PubMed 29890238
Study at a glance
From the registry record, last updated by the sponsor on 19 Jan 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Single Arm Open Pilot Study to Demonstrate the Efficacy of Midostaurin in Symptom Improvement and Decrease of Mast Cell Burden in Patients With Indolent or Smoldering Systemic Mastocytosis.
- ClinicalTrials.gov ID
- NCT01920204
- Acronym / sponsor ID
- UMCG41973
- Conditions
- Indolent Systemic Mastocytosis
- Keywords
- Mastocytosis, ISM, SSM
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- University Medical Center GroningenUniversity, hospital or other
- Study start
- Aug 2013
- Primary completion
- Mar 2015 (estimated)
- Study completion
- May 2015 (estimated)
- First posted
- 9 Aug 2013 (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.
Rationale: Patients with indolent or smoldering systemic mastocytosis can have severe disabling symptoms. Almost all patients have fatigue, a compromised quality of life, hampering normal functioning. Because this form of mastocytosis is not considered life-threatening, mast cell eradication has never been applied and patients receive only symptomatic therapy with histamine blockers. Midostaurin, a c-KIT inhibitor has shown activity regarding symptom control and decrease of malignant mast cells in patients with aggressive systemic mastocytosis (ASM) or mast cell leukemia
Detailed description
Objective:
Primary: To study in a pilot phase II trial the efficacy of midostaurin administered at an oral dose of 100 mg twice daily in patients with indolent or smoldering systemic mastocytosis on mediator symptom reduction, documented by the Mastocytosis Symptom Assessment Questionnaire, measured at 3 months.
Secondary:
- To study whether symptom improvement persists at 6 months, and whether midostaurin can reduce mast cell infiltration in the skin and bone marrow, documented by decrease of serum tryptase, decrease of urticaria pigmentosa and decrease of bone marrow mast cells.
- To assess safety and tolerability of midostaurin in the above mentioned settings
Study design: Single arm, open label pilot phase II study.
Study population: Adult patients (n=20) with histologically documented systemic mastocytosis, indolent or smoldering subtype, with severe symptoms, not controlled by histamine 1 and 2 blockers.
Intervention: treatment with Midostaurin, twice daily 100 mg orally for 6 months continuously.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Midostaurin | Experimental | Drug: Midostaurin,Treatment with Midostaurin, twice daily 100 mg orally for 6 months continuously. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Patients with Indolent Systemic Mastocytosis (ISM) or Smouldering Systemic Mastocytosis (SSM) according to the WHO criteria
- Presence of the D816V c-KIT mutation
- Serum tryptase > 20 mg/l
- Serious mediator-related symptoms that cannot be controlled by H1 and H2 blocking drugs. Symptoms will be scored by an adapted MSAF (mastocytosis symptom assessment form) with at least:
- Age >18 years
Key exclusion criteria (6 listed)
- Aggressive systemic mastocytosis, mast cell leukemia, or ASM with or without accompanying non-clonal related non-mast cell disorder (SM-ANHMD).
- Any known other present malignancy, non-melanoma skin cancers excluded
- History of malignancy within the last 5 years, non-melanoma skin cancers excluded
- Any serious comorbidity interfering with therapy compliance and follow-up compliance
- Pregnancy
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 |
|---|---|---|
| Midostaurin,Also: PKC412Midostaurin, twice daily 100 mg orally, continuously for 6 months | Drug | Midostaurin |
Primary outcome
Symptom Scoring
Time frame: 12 weeks
Percent change in the total score ("Sumscore") of all symptoms assessed by the Mastocytosis Symptom Assessment Form (MSAF) after 12 weeks.
3 secondary outcomes
Persistence of improvements
Time frame: 6 months
Mast cell burden
Time frame: 6 months
Adverse events
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 19 Jan 2015.
- Derived from this study: PubMed 29890238van Anrooij B, Oude Elberink JNG, Span LFR, de Monchy JGR, Rosati S, Mulder AB, Kluin-Nelemans JC. Midostaurin in patients with indolent systemic mastocytosis: An open-label phase 2 trial. J Allergy Clin Immunol. 2018 Sep;142(3):1006-1008.e7. doi: 10.1016/j.jaci.2018.06.003. Epub 2018 Jun 8. No abstract available.
Related trials for Indolent Systemic Mastocytosis
Active, not recruitingPhase 2
(Summit) A Study to Evaluate the Efficacy and Safety of CGT9486 Versus Placebo in Patients With Indolent or Smoldering Systemic Mastocytosis
SSM, Mastocytosis, Indolent
RecruitingPhase 2/3
(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis
Indolent Systemic Mastocytosis, Smoldering Systemic Mastocytosis
RecruitingObservational study
A Non-Interventional Study in Participants With Indolent Systemic Mastocytosis (ISM) in Germany
Indolent Systemic Mastocytosis
Active, not recruitingPhase 2
(PIONEER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, Versus Placebo in Patients With Indolent Systemic Mastocytosis
Indolent Systemic Mastocytosis
RecruitingObservational study
An Observational Study in Participants With Indolent Systemic Mastocytosis (ISM)
Indolent Systemic Mastocytosis
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.
J.C. Kluin-Nelemans, MD, PhD principal investigator
University Medical Center Groningen
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Midostaurin, in people with Indolent Systemic Mastocytosis, run by University Medical Center Groningen at 1 site in Netherlands.
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 10 inclusion and 6 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 19 Jan 2015. 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 Netherlands. 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?
Results are not available in the source record.
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?
Symptom Scoring (time frame: 12 weeks). 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 NCT01920204, 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 19 Jan 2015; AzTrials last refreshed its copy on 1 Nov 2026.