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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04910685




Registration number
NCT04910685
Ethics application status
Date submitted
17/05/2021
Date registered
2/06/2021

Titles & IDs
Public title
(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis
Scientific title
A Randomized, Double-Blind, Placebo-Controlled Phase 2/3 Study of BLU-263 in Indolent Systemic Mastocytosis
Secondary ID [1] 0 0
BLU-263-1201
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Indolent Systemic Mastocytosis 0 0
Smoldering Systemic Mastocytosis 0 0
Condition category
Condition code
Skin 0 0 0 0
Other skin conditions
Blood 0 0 0 0
Other blood disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Elenestinib
Treatment: Drugs - Placebo

Experimental: (Part 1) Elenestinib Dose 1 + SDT - Participants 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.

Experimental: (Part 1) Elenestinib Dose 2 + SDT - Participants 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.

Experimental: (Part 1) Elenestinib Dose 3 + SDT - Participants 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.

Placebo comparator: (Part 1) Placebo + SDT - Participants 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.

Experimental: (Part 2) Elenestinib Dose 1 + SDT - Participants 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.

Placebo comparator: (Part 2) Placebo + SDT - Participants 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.

Experimental: (Part 3) Elenestinib + SDT - Participants 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.

Experimental: (Part S) Elenestinib Dose 1 + SDT - Participants 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.

Experimental: (Part K) Elenestinib Dose 1 + SDT - Participants 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.

Experimental: (PK groups) Elenestinib + SDT - Participants 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.


Treatment: Drugs: Elenestinib
Elenestinib oral tablet

Treatment: Drugs: Placebo
Placebo oral tablet
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Part 1: Number of participants with Treatment-emergent Adverse Events (TEAEs)
Timepoint [1] 0 0
Up to 12 weeks
Primary outcome [2] 0 0
Part 1: Mean change from baseline in ISM-Symptom in Assessment Form (ISM-SAF) Total Symptom Score (TSS)
Timepoint [2] 0 0
Baseline, Week 13
Primary outcome [3] 0 0
Part 2: Mean change from baseline in ISM-SAF TSS
Timepoint [3] 0 0
Baseline, Week 49
Primary outcome [4] 0 0
Part 3: Number of participants with Adverse Events (AEs)
Timepoint [4] 0 0
Up to 5 years
Primary outcome [5] 0 0
Part 3: Change from baseline in ISM-SAF TSS
Timepoint [5] 0 0
Baseline up to 5 years
Secondary outcome [1] 0 0
Part 1: Change from baseline in serum tryptase
Timepoint [1] 0 0
Baseline, Week 13
Secondary outcome [2] 0 0
Part 1: Change from baseline in KIT D816V allele fraction in blood
Timepoint [2] 0 0
Baseline, Week 13
Secondary outcome [3] 0 0
Part 1: Change from baseline in Bone Marrow (BM) mast cells
Timepoint [3] 0 0
Baseline, Week 13
Secondary outcome [4] 0 0
Part 1: Mean change from baseline in ISM-SAF individual symptom scores
Timepoint [4] 0 0
Baseline, Week 13
Secondary outcome [5] 0 0
Part 1: Time to achieve 30% reduction from baseline in ISM-SAF TSS
Timepoint [5] 0 0
Baseline up to Week 13
Secondary outcome [6] 0 0
Part 1: Time to achieve 30% reduction from baseline in ISM-SAF domain scores
Timepoint [6] 0 0
Baseline up to Week 13
Secondary outcome [7] 0 0
Part 2: Proportion of participants achieving normalized tryptase
Timepoint [7] 0 0
Baseline up to Week 49
Secondary outcome [8] 0 0
Part 2: Proportion of participants who achieve an undetectable level or at least a 50% reduction in KIT D816V Variant Allele Frequency (VAF)
Timepoint [8] 0 0
Baseline up to Week 49
Secondary outcome [9] 0 0
Part 2: Proportion of participants achieving symptom control as defined by achieving mild symptoms
Timepoint [9] 0 0
Baseline up to Week 49
Secondary outcome [10] 0 0
Part 2: Mean percent change from baseline in Bone Mineral Density (BMD)
Timepoint [10] 0 0
Baseline, Week 49
Secondary outcome [11] 0 0
Part 2: Mean change from baseline in the annualized rate of anaphylaxis events
Timepoint [11] 0 0
Baseline, Weeks 25 to 48
Secondary outcome [12] 0 0
Part 2: Mean change from baseline in Quality of Life (QoL) scores
Timepoint [12] 0 0
Baseline, Week 49
Secondary outcome [13] 0 0
Part 2: Mean change from baseline in ISM-SAF domain scores
Timepoint [13] 0 0
Baseline, Week 49
Secondary outcome [14] 0 0
Part 2: Number of participants with AEs
Timepoint [14] 0 0
Up to Week 49
Secondary outcome [15] 0 0
Part 2: Proportion of participants with a 50% reduction in ISM-SAF TSS
Timepoint [15] 0 0
Baseline, Weeks 24 and 48
Secondary outcome [16] 0 0
Part 2: Proportion of participants with a 50% reduction in ISM-SAF domain scores
Timepoint [16] 0 0
Baseline, Weeks 24 and 48
Secondary outcome [17] 0 0
Part 2: Proportion of participants with a 30% reduction in ISM-SAF TSS
Timepoint [17] 0 0
Baseline, Weeks 24 and 48
Secondary outcome [18] 0 0
Part 2: Proportion of participants with a 30% reduction in ISM-SAF domain scores
Timepoint [18] 0 0
Baseline, Weeks 24 and 48
Secondary outcome [19] 0 0
Part 3: Proportion of participants achieving symptom control as defined by achieving mild symptoms
Timepoint [19] 0 0
Baseline up to 5 years
Secondary outcome [20] 0 0
Part 3: Change from baseline in ISM-SAF domain scores
Timepoint [20] 0 0
Baseline, up to 5 years
Secondary outcome [21] 0 0
Part 3: Proportion of participants achieving a normalized tryptase
Timepoint [21] 0 0
Baseline up to 5 years
Secondary outcome [22] 0 0
Part 3: Change from baseline in BMD
Timepoint [22] 0 0
Baseline up to 5 years
Secondary outcome [23] 0 0
Part 3: Change from baseline in the annualized rate of anaphylaxis events
Timepoint [23] 0 0
Baseline up to 5 years
Secondary outcome [24] 0 0
Parts 2 and 3: Change from baseline in serum tryptase
Timepoint [24] 0 0
Baseline up to 5 years
Secondary outcome [25] 0 0
Parts 2 and 3: Change from baseline in KIT D816V allele fraction in blood
Timepoint [25] 0 0
Baseline up to 5 years
Secondary outcome [26] 0 0
Parts 2 and 3: Change from baseline in Bone Marrow (BM) mast cells
Timepoint [26] 0 0
Baseline up to 5 years
Secondary outcome [27] 0 0
Parts 2 and 3: Proportion of participants achieving controlled disease
Timepoint [27] 0 0
Baseline up to 5 years
Secondary outcome [28] 0 0
Parts 2 and 3: Change from baseline in skin lesions as assessed by the fractional body surface area of the most affected skin area
Timepoint [28] 0 0
Baseline up to 5 years
Secondary outcome [29] 0 0
Parts 2 and 3: Change from baseline in the number of concomitant medications identified as SDT
Timepoint [29] 0 0
Baseline up to 5 years
Secondary outcome [30] 0 0
Parts 2 and 3: Change from baseline in ISM-SAF Individual Symptom Scores
Timepoint [30] 0 0
Baseline up to 5 years
Secondary outcome [31] 0 0
Parts 2 and 3: Change from baseline in ISM-SAF Lead (most severe) Symptom Score
Timepoint [31] 0 0
Baseline up to 5 years
Secondary outcome [32] 0 0
Parts 2 and 3: Change from baseline in QoL scores
Timepoint [32] 0 0
Baseline up to 5 years
Secondary outcome [33] 0 0
Part S: Number of participants with AEs
Timepoint [33] 0 0
Baseline up to 5 years
Secondary outcome [34] 0 0
Part S: Proportion of participants who achieve a Pure Pathologic Response (PPR)
Timepoint [34] 0 0
Baseline up to 5 years
Secondary outcome [35] 0 0
Part S: Mean change from baseline in ISM-SAF
Timepoint [35] 0 0
Baseline, Week 25
Secondary outcome [36] 0 0
Part K: Number of participants with AEs
Timepoint [36] 0 0
Baseline up to 5 years
Secondary outcome [37] 0 0
Part K: Change from baseline in serum tryptase
Timepoint [37] 0 0
Baseline up to 5 years
Secondary outcome [38] 0 0
Part K: Change from baseline in KIT D816V allele fraction in blood
Timepoint [38] 0 0
Baseline up to 5 years
Secondary outcome [39] 0 0
Part K: Mean change from baseline in ISM-SAF TSS
Timepoint [39] 0 0
Baseline up to 5 years
Secondary outcome [40] 0 0
Part K: Change from baseline in QoL scores
Timepoint [40] 0 0
Baseline up to 5 years

Eligibility
Key inclusion criteria
Key

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.
* 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

Key
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* 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.
* 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
30/11/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
30/09/2032
Actual
Sample size
Target
534
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD,VIC
Recruitment hospital [1] 0 0
Princess Alexandra Hospital - Woolloongabba
Recruitment hospital [2] 0 0
The Alfred Hospital - Melbourne
Recruitment postcode(s) [1] 0 0
- Woolloongabba
Recruitment postcode(s) [2] 0 0
3004 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Massachusetts
Country [4] 0 0
United States of America
State/province [4] 0 0
Michigan
Country [5] 0 0
United States of America
State/province [5] 0 0
Minnesota
Country [6] 0 0
United States of America
State/province [6] 0 0
New York
Country [7] 0 0
United States of America
State/province [7] 0 0
Ohio
Country [8] 0 0
United States of America
State/province [8] 0 0
Texas
Country [9] 0 0
United States of America
State/province [9] 0 0
Utah
Country [10] 0 0
Austria
State/province [10] 0 0
Linz
Country [11] 0 0
Belgium
State/province [11] 0 0
Antwerpen
Country [12] 0 0
France
State/province [12] 0 0
Amiens
Country [13] 0 0
France
State/province [13] 0 0
Caen
Country [14] 0 0
France
State/province [14] 0 0
Grenoble Cedex 9
Country [15] 0 0
France
State/province [15] 0 0
Limoges Cedex
Country [16] 0 0
France
State/province [16] 0 0
Nantes
Country [17] 0 0
France
State/province [17] 0 0
Paris
Country [18] 0 0
France
State/province [18] 0 0
Poitiers
Country [19] 0 0
France
State/province [19] 0 0
Toulouse
Country [20] 0 0
Germany
State/province [20] 0 0
Aachen
Country [21] 0 0
Germany
State/province [21] 0 0
Berlin
Country [22] 0 0
Germany
State/province [22] 0 0
Erlangen
Country [23] 0 0
Germany
State/province [23] 0 0
Hamburg
Country [24] 0 0
Germany
State/province [24] 0 0
Mannheim
Country [25] 0 0
Germany
State/province [25] 0 0
Munich
Country [26] 0 0
Italy
State/province [26] 0 0
Forli-Cesena
Country [27] 0 0
Italy
State/province [27] 0 0
Lombardia
Country [28] 0 0
Italy
State/province [28] 0 0
Toscana
Country [29] 0 0
Italy
State/province [29] 0 0
Bologna
Country [30] 0 0
Italy
State/province [30] 0 0
Pavia
Country [31] 0 0
Italy
State/province [31] 0 0
Salerno
Country [32] 0 0
Italy
State/province [32] 0 0
Verona
Country [33] 0 0
Netherlands
State/province [33] 0 0
Zuid-Holland
Country [34] 0 0
Netherlands
State/province [34] 0 0
Groningen
Country [35] 0 0
Norway
State/province [35] 0 0
Oslo
Country [36] 0 0
Portugal
State/province [36] 0 0
Lisbon
Country [37] 0 0
Portugal
State/province [37] 0 0
Porto
Country [38] 0 0
Spain
State/province [38] 0 0
Barcelona
Country [39] 0 0
Spain
State/province [39] 0 0
Madrid
Country [40] 0 0
Spain
State/province [40] 0 0
Toledo
Country [41] 0 0
Sweden
State/province [41] 0 0
Uppsala
Country [42] 0 0
Switzerland
State/province [42] 0 0
Basel
Country [43] 0 0
Switzerland
State/province [43] 0 0
Luzern
Country [44] 0 0
United Kingdom
State/province [44] 0 0
Wales
Country [45] 0 0
United Kingdom
State/province [45] 0 0
Cardiff
Country [46] 0 0
United Kingdom
State/province [46] 0 0
London
Country [47] 0 0
United Kingdom
State/province [47] 0 0
Oxford
Country [48] 0 0
United Kingdom
State/province [48] 0 0
Plymouth

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Blueprint Medicines Corporation
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Blueprint Medicines
Address 0 0
Country 0 0
Phone 0 0
617-714-6707
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT04910685