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Indolent Systemic MastocytosisPhase 2/3NCT04910685

(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis

A Phase 2/3 study testing Elenestinib in people with Indolent Systemic Mastocytosis and Smoldering Systemic Mastocytosis, run by Blueprint Medicines Corporation at 88 sites in 25 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

This is a randomized, double-blind, placebo-controlled, Phase 2/3 study comparing the efficacy and safety of elenestinib (BLU-263) + symptom directed therapy (SDT) with placebo + SDT in participants with indolent systemic mastocytosis (ISM) whose symptoms are not adequately controlled by SDT. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Elenestinib
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
534 participants
Where
88 sitesin 25 countries
Study dates
30 Nov 2021to 30 Sep 2032
Phase
Phase 2/3
Are there results?
Not yet: the study is under way

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

  • All Participants:
  • -Participant must have an Eastern Cooperative Oncology Group Performance Status (ECOG PS) of 0 to 2.
  • Part 1 and PK groups:
  • Participant has confirmed diagnosis of ISM, confirmed by Central Pathology Review
  • Participant must have failed to achieve adequate symptom control for 1 or more Baseline symptoms, as determined by the Investigator, with at least 2 of the following symptom-directed therapies administered: H1 blockers, H2 blockers, proton-pump inhibitors, leukotriene inhibitors, cromolyn sodium, corticosteroids, or omalizumab.
  • Participants must have SDT for ISM symptom management stabilized for at least 14 days prior to starting screening procedures.
Show all 13Show fewer
  • For participants receiving corticosteroids, the dose must be ≤ 20 mg/day prednisone or equivalent, and the dose must be stable for ≥ 14 days.
  • Part K:
  • -Participant has confirmed diagnosis of ISM, confirmed by Central Pathology Review
  • Part S:
  • -Participant has confirmed diagnosis of SSM, confirmed by Central Pathology Review of BM biopsy and central review of B- and C-findings by WHO diagnostic criteria.
  • Part 2:
  • -Participant has confirmed diagnosis of ISM, confirmed by Central Pathology Review

Who cannot join

  • Participant has been diagnosed with any of the following WHO systemic mastocytosis (SM) sub-classifications: cutaneous mastocytosis only, SM with an associated hematologic neoplasm of non-MC lineage (SM-AHN), aggressive SM, mast cell leukemia, or mast cell sarcoma.
  • Participant has been diagnosed with another myeloproliferative disorder.
  • Participant has organ damage attributable to SM.
  • Participant has clinically significant, uncontrolled, cardiovascular disease
  • Participant has a QT interval corrected using Fridericia's formula (QTcF) > > 470 milliseconds (msec) (for females) or > 450 msec (for males).
  • Participant has a history of a primary malignancy that has been diagnosed or required therapy within 3 years. The following prior malignancies are not exclusionary: completely resected basal cell and squamous cell skin cancer, curatively treated localized prostate cancer, and completely resected carcinoma in situ of any site.
Show all 9Show fewer
  • Time since any cytoreductive therapy including masitinib and midostaurin should be at least 5 half-lives or 14 days (whichever is longer), and for cladribine, interferon alpha, pegylated interferon, or antibody therapy < 28 days or 5 half-lives of the drug (whichever is longer), before beginning the screening period.
  • Participant has received radiotherapy or psoralen and ultraviolet A (PUVA) therapy < 14 days before beginning the screening period.
  • Other protocol-defined criteria apply.

Trial locations and contacts

88 sites in 25 countries. Contact the study team or a site near you to ask about taking part.

Blueprint Medicines contact

617-714-6707

medinfo@blueprintmedicines.com

88 sites in 25 countries

United States15 sites, 15 recruiting

  • Michigan Medicine University of Michigan

    Ann Arbor, Michigan, 48109

    Recruiting

  • Winship Cancer Institute, Emory University

    Atlanta, Georgia, 30322

    Recruiting

  • UCHealth Blood Disorders and Cell Therapies Center - Anschutz Medical Campus

    Aurora, Colorado, 80045

    Recruiting

  • University of Alabama at Birmingham

    Birmingham, Alabama, 35294

    Recruiting

  • Brigham and Women's Hospital

    Boston, Massachusetts, 02115

    Recruiting

  • Roswell Park Cancer Institute

    Buffalo, New York, 14263

    Recruiting

  • University of Cincinnati Medical Center

    Cincinnati, Ohio, 45219

    Recruiting

  • Duke Asthma, Allergy and Airway Center

    Durham, North Carolina, 27705

    Recruiting

  • The University of Texas, MD Anderson Cancer Center

    Houston, Texas, 77030

    Recruiting

  • Orso Health, Modena Allergy and Asthma Inc

    La Jolla, California, 92037

    Recruiting

  • David Geffen School of Medicine at UCLA

    Los Angeles, California, 90095

    Recruiting

  • Columbia University Medical Center

    New York, New York, 10032

    Recruiting

  • Stanford Cancer Institute

    Palo Alto, California, 94305

    Recruiting

  • Mayo Clinic

    Rochester, Minnesota, 55902

    Recruiting

  • Huntsman Cancer Institute, University of Utah

    Salt Lake City, Utah, 84112

    Recruiting

Other trials in United States

France9 sites, 9 recruiting

  • CHU Amiens-Picardie

    Amiens, 80000

    Recruiting

  • CHU de Caen

    Caen

    Recruiting

  • CHU Grenoble

    Grenoble, 38043

    Recruiting

  • CHU de Limoges

    Limoges, 87042

    Recruiting

  • CHU de Nantes

    Nantes, 44093

    Recruiting

  • Hôpital de la Pitié Salpétrière

    Paris, 75013

    Recruiting

  • Hôpital Necker - Départementd 'HématologieA dultes

    Paris, 75015

    Recruiting

  • CHU de Poitiers

    Poitiers, 86000

    Recruiting

  • CHU Toulouse - Hopital Larrey

    Toulouse

    Recruiting

Other trials in France

Italy9 sites, 9 recruiting

  • Unita Operativa di Ematologia AOU Policlinico S. Orsola-Malpighi

    Bologna, 40138

    Recruiting

  • AOU Policlinico G.Rodolico - San Marco

    Catania, 95123

    Recruiting

  • SOD Ematologia (Ambulatori)- AOUC Azienda Ospedaliero Universitaria Careggi

    Florence, Tuscany, 50134

    Recruiting

  • UOC Ematologia

    Milan, Lombardy, 20122

    Recruiting

  • Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico

    Milan, 20122

    Recruiting

  • S.C. Ematologia Fondazione I.R.C.C.S. Policlinico San Matteo

    Pavia, 27100

    Recruiting

  • S.S.D. Immunologia Clinica e Allergologia Azienda Ospedaliera Universitaria San Giovanni di Dio e Ruggi d'Aragona

    Salerno, 84131

    Recruiting

  • Unità Operativa di Allergologia Azienda Ospedaliera Universitaria Integrata di Verona

    Verona, 37126

    Recruiting

  • Azienda Unita Locale Socia Sanitaria N8 Berica

    Vicenza, 36100

    Recruiting

Other trials in Italy

Germany8 sites, 8 recruiting

  • Universitätsklinikum RWTH Aachen Klinik für Hämatologie, Onkologie, Hämostaseologie und Stammzelltransplantation

    Aachen

    Recruiting

  • Charité - Universitätsmedizin Berlin Institute of Allergology

    Berlin, 12203

    Recruiting

  • University Clinic Erlangen

    Erlangen, 91054

    Recruiting

  • University Clinic Hamburg Eppendorf

    Hamburg, 20246

    Recruiting

  • Universitatsklinikum Schleswig-Holstein, Campus Lubeck

    Lübeck, 23562

    Recruiting

  • Universitätsmedizin Mannheim III. Medizinische Klinik Universität Heidelberg Medizinische Fakultät Mannheim

    Mannheim, 68167

    Recruiting

  • Klinikum rechts der Isar der Technischen Universitaet Muenchen

    München, 81675

    Recruiting

  • LMU Klinikum

    Munich, 80377

    Recruiting

Other trials in Germany

United Kingdom6 sites, 6 recruiting

  • Queen Elizabeth Hospital, University Hospital Birmingham

    Birmingham, B15 2GW

    Recruiting

  • University Hospital of Wales

    Cardiff, Wales, CF14 4XW

    Recruiting

  • University College London Hospitals (UCLH), Haematology Cancer Clinical Trials Unit

    London, NW1 2PG

    Recruiting

  • Guy's and St Thomas's NHS Foundation Trust

    London, SE1 7EH

    Recruiting

  • GenesisCare

    Oxford, OX4 6LB

    Recruiting

  • University Hospital Plymouth Trust - Derriford Hospital

    Plymouth, PL6 8DH

    Recruiting

Other trials in United Kingdom

Belgium4 sites, 4 recruiting

  • Unitversitair Ziekenhuis Antwerpen

    Edegem, Antwerpen

    Recruiting

  • Universitair Ziekenhuis Gent

    Ghent, 9000

    Recruiting

  • CHU Tivoli

    La Louvière, 7100

    Recruiting

  • UZ Leuven

    Leuven, 3000

    Recruiting

Other trials in Belgium

Portugal4 sites, 4 recruiting

  • Centro Hospitalar de Lisboa Central, E.P.E. - Hospital de Santo Antonio dos Capuchos

    Lisbon, 1169-050

    Recruiting

  • CHUPorto, EPE - Hospital de Santo António

    Porto, 4099-001

    Recruiting

  • Centro Hospitalar Universitario Sao Joao, E.P.E.

    Porto, 4200-139

    Recruiting

  • Unidade Local de Saude de Matosinhos, EPE - Hospital Pedro Hispanc

    Senhora Da Hor, 4464-513

    Recruiting

Other trials in Portugal

Australia3 sites, 3 recruiting

  • The Canberra Hospital

    Garran, ACT 2605

    Recruiting

  • The Alfred Hospital

    Melbourne, Victoria, 3004

    Recruiting

  • Princess Alexandra Hospital

    Woolloongabba, Queensland

    Recruiting

Other trials in Australia

Netherlands3 sites, 3 recruiting

  • University Medical Center Groningen

    Groningen, 9713 GZ

    Recruiting

  • Maastricht UMC+

    Maastricht, 6229 HX

    Recruiting

  • ErasmusMC

    Rotterdam, South Holland, 3015 GD

    Recruiting

Other trials in Netherlands

Spain3 sites, 3 recruiting

  • Hospital Universitario Vall d'Hebron

    Barcelona

    Recruiting

  • Hospital Universitario Ramon y Cajal

    Madrid, 28034

    Recruiting

  • Hospital Virgen del Valle - Instituto de Estudios de Mastocitosis de Castilla-La Mancha

    Toledo, 45071

    Recruiting

Other trials in Spain

Turkey (Türkiye)3 sites, 3 recruiting

  • Ankara Universitesi Tip Fakultesi Cebeci Hastanesi

    Ankara, 06620

    Recruiting

  • lstanbul Universitesi lstanbul Tip Fakultesi Hastanesi

    Istanbul, 34093

    Recruiting

  • Mersin VM Medikal Park Hastanesi

    Mersin, 33200

    Recruiting

Other trials in Turkey (Türkiye)

Argentina2 sites, 2 recruiting

  • CHP Centro Hematologico Pavlovsky

    Buenos Aires, 1113

    Recruiting

  • Consultorios Medicos Dr. Doreski - Fundacion Respirar

    Buenos Aires, 1426

    Recruiting

Other trials in Argentina

Czechia2 sites, 2 recruiting

  • Fakultní nemocnice Brno, Interní hematologická a onkologická klinika

    Brno, 625 00

    Recruiting

  • Fakultní nemocnice Královské Vinohrady, Hematologická klinika 3. LF UK v Praze a FNKV

    Prague, 100 34

    Recruiting

Other trials in Czechia

Greece2 sites, 2 recruiting

  • University General Hospital - General Hospital of West Attica

    Chaïdári, 12462

    Recruiting

  • General Hospital of Thessaloniki "G. Papanikolaou"

    Thessaloniki, 57010

    Recruiting

Other trials in Greece

Poland2 sites, 2 recruiting

  • Uniwersyteckie Centrum Kliniczne Klinika Alergologii

    Gdansk, 80-214

    Recruiting

  • Samodzielny Publiczny Za kład Opieki Zdrowotnej Szpital Uniwersytecki w Krakowie

    Krakow, 30-688

    Recruiting

Other trials in Poland

Romania2 sites, 2 recruiting

  • Spitalul Clinic Colentina Bucuresti

    Bucharest, 020125

    Recruiting

  • Spitalul Universitar De Urgenta Bucuresti

    Bucharest, 050098

    Recruiting

Other trials in Romania

Sweden2 sites, 2 recruiting

  • Karolinska Universitetssjukhuset Huddinge

    Stockholm, 141 57

    Recruiting

  • Uppsala University Hospital

    Uppsala, 751 85

    Recruiting

Other trials in Sweden

Switzerland2 sites, 2 recruiting

  • University Hospital Basel

    Basel, C-4031

    Recruiting

  • Luzerner Kantonsspital

    Lucerne, 6000

    Recruiting

Other trials in Switzerland

Austria1 site, 1 recruiting

  • Kepler Universitatsklinikum, Med Campus III. Clinic of Internal Medicine 3 - Hematology and Oncology

    Linz, 4021

    Recruiting

Other trials in Austria

Brazil1 site, 1 recruiting

  • Hospital das Clínicas da Faculdade de Medicina da USP - HCFMUSP

    São Paulo, 05403-010

    Recruiting

Other trials in Brazil

Chile1 site, 1 recruiting

  • Clinica Alemana de Santiago

    Santiago, 1410

    Recruiting

Other trials in Chile

Colombia1 site, 1 recruiting

  • Fundación Valle del Lili

    Cali, 760033

    Recruiting

Other trials in Colombia

Denmark1 site, 1 recruiting

  • Odense University Hospital and University of Southern Denmark

    Odense, 5000

    Recruiting

Other trials in Denmark

Ireland1 site, 1 recruiting

  • Cork University Hospital

    Cork, T12 DC4A

    Recruiting

Other trials in Ireland

Norway1 site, 1 recruiting

  • Oslo University Hospital

    Oslo, N-0424

    Recruiting

Other trials in Norway

About this study

Official title: A Randomized, Double-Blind, Placebo-Controlled Phase 2/3 Study of BLU-263 in Indolent Systemic Mastocytosis

This is a randomized, double-blind, placebo-controlled, Phase 2/3 study comparing the efficacy and safety of elenestinib (BLU-263) + symptom directed therapy (SDT) with placebo + SDT in participants with indolent systemic mastocytosis (ISM) whose symptoms are not adequately controlled by SDT. Parts 1 and 2 will enroll participants with ISM. Participants enrolled in Part 2 will roll over onto Part 3 to receive treatment with elenestinib in an open-label fashion following completion of the earlier Part. Part K will enroll participants with ISM who have previously received an approved selective KIT inhibitor. The study also includes pharmacokinetic (PK) groups that will enroll participants with ISM.

Being tested
Elenestinib, Placebo
Keywords
ISM, SSM
Medical subject headings
Mastocytosis, Systemic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ElenestinibDrug

    Also called BLU-263

    Elenestinib oral tablet

  • PlaceboDrug

    Placebo oral tablet

Study groups in NCT04910685
Study groupRoleWhat they receive
(Part 1) Elenestinib Dose 1 + SDTExperimentalParticipants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily until completion of Part 1.
Receives: Drug: Elenestinib
(Part 1) Elenestinib Dose 2 + SDTExperimentalParticipants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily until completion of Part 1.
Receives: Drug: Elenestinib
(Part 1) Elenestinib Dose 3 + SDTExperimentalParticipants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily until completion of Part 1.
Receives: Drug: Elenestinib
(Part 1) Placebo + SDTPlacebo comparatorParticipants will receive SDT and matching placebo. SDT will be determined on a per participant basis. Placebo will be administered orally, once daily until completion of Part 1.
Receives: Drug: Placebo
(Part 2) Elenestinib Dose 1 + SDTExperimentalParticipants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily for approximately 48 weeks.
Receives: Drug: Elenestinib
(Part 2) Placebo + SDTPlacebo comparatorParticipants will receive SDT and matching placebo. SDT will be determined on a per participant basis. Placebo will be administered orally, once daily for approximately 48 weeks.
Receives: Drug: Placebo
(Part 3) Elenestinib + SDTExperimentalParticipants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily for up to approximately 5 years.
Receives: Drug: Elenestinib
(Part S) Elenestinib Dose 1 + SDTExperimentalParticipants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily for up to approximately 5 years.
Receives: Drug: Elenestinib
(Part K) Elenestinib Dose 1 + SDTExperimentalParticipants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily for up to approximately 5 years.
Receives: Drug: Elenestinib
(PK groups) Elenestinib + SDTExperimentalParticipants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily for up to approximately 5 years.
Receives: Drug: Elenestinib

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Part 1: Number of participants with Treatment-emergent Adverse Events (TEAEs)

    Measured: Up to 12 weeks

  2. Part 1: Mean change from baseline in ISM-Symptom in Assessment Form (ISM-SAF) Total Symptom Score (TSS)

    Measured: Baseline, Week 13

  3. Part 2: Mean change from baseline in ISM-SAF TSS

    Measured: Baseline, Week 49

  4. Part 3: Number of participants with Adverse Events (AEs)

    Measured: Up to 5 years

  5. Part 3: Change from baseline in ISM-SAF TSS

    Measured: Baseline up to 5 years

Secondary outcomes (40)

  1. Part 1: Change from baseline in serum tryptase

    Measured: Baseline, Week 13

  2. Part 1: Change from baseline in KIT D816V allele fraction in blood

    Measured: Baseline, Week 13

  3. Part 1: Change from baseline in Bone Marrow (BM) mast cells

    Measured: Baseline, Week 13

  4. Part 1: Mean change from baseline in ISM-SAF individual symptom scores

    Measured: Baseline, Week 13

  5. Part 1: Time to achieve 30% reduction from baseline in ISM-SAF TSS

    Measured: Baseline up to Week 13

  6. Part 1: Time to achieve 30% reduction from baseline in ISM-SAF domain scores

    Measured: Baseline up to Week 13

  7. Part 2: Proportion of participants achieving normalized tryptase

    Measured: Baseline up to Week 49

  8. Part 2: Proportion of participants who achieve an undetectable level or at least a 50% reduction in KIT D816V Variant Allele Frequency (VAF)

    Measured: Baseline up to Week 49

  9. Part 2: Proportion of participants achieving symptom control as defined by achieving mild symptoms

    Measured: Baseline up to Week 49

  10. Part 2: Mean percent change from baseline in Bone Mineral Density (BMD)

    Measured: Baseline, Week 49

  11. Part 2: Mean change from baseline in the annualized rate of anaphylaxis events

    Measured: Baseline, Weeks 25 to 48

  12. Part 2: Mean change from baseline in Quality of Life (QoL) scores

    Measured: Baseline, Week 49

  13. Part 2: Mean change from baseline in ISM-SAF domain scores

    Measured: Baseline, Week 49

  14. Part 2: Number of participants with AEs

    Measured: Up to Week 49

  15. Part 2: Proportion of participants with a 50% reduction in ISM-SAF TSS

    Measured: Baseline, Weeks 24 and 48

  16. Part 2: Proportion of participants with a 50% reduction in ISM-SAF domain scores

    Measured: Baseline, Weeks 24 and 48

  17. Part 2: Proportion of participants with a 30% reduction in ISM-SAF TSS

    Measured: Baseline, Weeks 24 and 48

  18. Part 2: Proportion of participants with a 30% reduction in ISM-SAF domain scores

    Measured: Baseline, Weeks 24 and 48

  19. Part 3: Proportion of participants achieving symptom control as defined by achieving mild symptoms

    Measured: Baseline up to 5 years

  20. Part 3: Change from baseline in ISM-SAF domain scores

    Measured: Baseline, up to 5 years

  21. Part 3: Proportion of participants achieving a normalized tryptase

    Measured: Baseline up to 5 years

  22. Part 3: Change from baseline in BMD

    Measured: Baseline up to 5 years

  23. Part 3: Change from baseline in the annualized rate of anaphylaxis events

    Measured: Baseline up to 5 years

  24. Parts 2 and 3: Change from baseline in serum tryptase

    Measured: Baseline up to 5 years

  25. Parts 2 and 3: Change from baseline in KIT D816V allele fraction in blood

    Measured: Baseline up to 5 years

  26. Parts 2 and 3: Change from baseline in Bone Marrow (BM) mast cells

    Measured: Baseline up to 5 years

  27. Parts 2 and 3: Proportion of participants achieving controlled disease

    Measured: Baseline up to 5 years

  28. Parts 2 and 3: Change from baseline in skin lesions as assessed by the fractional body surface area of the most affected skin area

    Measured: Baseline up to 5 years

  29. Parts 2 and 3: Change from baseline in the number of concomitant medications identified as SDT

    Measured: Baseline up to 5 years

  30. Parts 2 and 3: Change from baseline in ISM-SAF Individual Symptom Scores

    Measured: Baseline up to 5 years

  31. Parts 2 and 3: Change from baseline in ISM-SAF Lead (most severe) Symptom Score

    Measured: Baseline up to 5 years

  32. Parts 2 and 3: Change from baseline in QoL scores

    Measured: Baseline up to 5 years

  33. Part S: Number of participants with AEs

    Measured: Baseline up to 5 years

  34. Part S: Proportion of participants who achieve a Pure Pathologic Response (PPR)

    Measured: Baseline up to 5 years

  35. Part S: Mean change from baseline in ISM-SAF

    Measured: Baseline, Week 25

  36. Part K: Number of participants with AEs

    Measured: Baseline up to 5 years

  37. Part K: Change from baseline in serum tryptase

    Measured: Baseline up to 5 years

  38. Part K: Change from baseline in KIT D816V allele fraction in blood

    Measured: Baseline up to 5 years

  39. Part K: Mean change from baseline in ISM-SAF TSS

    Measured: Baseline up to 5 years

  40. Part K: Change from baseline in QoL scores

    Measured: Baseline up to 5 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted2 Jun 2021
  2. Study start30 Nov 2021
  3. Primary completion30 Sep 2032 (estimated)
  4. Study completion30 Sep 2032 (estimated)

Resources

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
Randomised
Intervention model
Parallel groups
Model description
Part 1 and Part 2: Randomized, Blinded Part 3, Part K, Part S and PK groups: Non-randomized, Open-label
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
534 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04910685
Sponsor's protocol ID
BLU-263-1201
Status last verified
Oct 2026
First submitted
17 May 2021
Last updated
7 Oct 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing Elenestinib in people with Indolent Systemic Mastocytosis and Smoldering Systemic Mastocytosis, run by Blueprint Medicines Corporation at 88 sites in 25 countries.

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 13 inclusion and 9 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: looking for participants now, as of the sponsor's last update on 7 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 88 sites in United States, France, Italy, Germany, United Kingdom and 20 more countries. 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. Some participants in this study may receive a placebo. 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?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Blueprint Medicines (617-714-6707, medinfo@blueprintmedicines.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Part 1: Number of participants with Treatment-emergent Adverse Events (TEAEs) (time frame: Up to 12 weeks). The study lists 5 primary outcomes in all. 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 NCT04910685.

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 7 Oct 2026.

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