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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04059406


Additional trial details provided through ANZCTR are available at the end of this record.


Registration number
NCT04059406
Ethics application status
Date submitted
14/08/2019
Date registered
16/08/2019

Titles & IDs
Public title
Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx)
Scientific title
A Phase 2a, Randomized, Open-Label Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Patients With Non-Transfusion Dependent ß-Thalassemia Intermedia
Secondary ID [1] 0 0
2019-003505-96
Secondary ID [2] 0 0
ISIS 702843-CS2
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Beta Thalassemia Intermedia 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

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

Experimental: Sapablursen Dose Level 1 (Cohort A) - A single injection of sapablursen at multiple dose levels, administered subcutaneously every 4 weeks.

Experimental: Sapablursen Dose Level 1 (Cohort B) - A single injection of sapablursen at multiple dose levels, administered subcutaneously every 4 weeks.

Experimental: Sapablursen Dose Level 1 (Cohort C) - A single injection of sapablursen at multiple dose levels, administered subcutaneously every 4 weeks.


Treatment: Drugs: sapablursen
sapablursen administered subcutaneously
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percentage of participants with a = 1.0 grams per deciliter (g/dL) increase from Baseline in hemoglobin (Hb) at Week 27
Timepoint [1] 0 0
Baseline and Week 27
Secondary outcome [1] 0 0
Percentage of participants with a = 1.5 grams per deciliter (g/dL) increase from Baseline in hemoglobin (Hb) at Week 53
Timepoint [1] 0 0
Baseline and Week 53
Secondary outcome [2] 0 0
Percentage of participants with a = 1.0 milligram of iron per gram of dry weight of liver (mg Fe/g) decrease from Baseline in liver iron concentration (LIC) at Week 53
Timepoint [2] 0 0
Baseline and Week 53

Eligibility
Key inclusion criteria
* Willingness to comply with study procedures
* Clinical diagnosis of Beta-Thalassemia Intermedia with genotypic confirmation
* Non-transfusion dependent, as defined by: no more than 6 transfusions in the past 12-month period, and no transfusions in the 8-week period prior to Day 1
* Mean Hb within the range of 6.0-10.0 g/dL, inclusive at Screening
* LIC within the range of 3.0-20.0 mg Fe/g dry weight, inclusive
* If using chelators, must be on a stable dose for at least 3 months with LIC > 5.0 mg Fe/g dry weight and serum ferritin > 300 nanograms per milliliter (ng/mL)
* Females must be non-pregnant and non-lactating, and either surgically sterile or postmenopausal
* Males must be surgically sterile, abstinent or using an acceptable contraceptive method
Minimum age
18 Years
Maximum age
65 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Clinically significant abnormalities in lab values, medical history, or physical examination
* a-globin gene triplication
* Symptomatic splenomegaly
* Platelet count < lower limit of normal (LLN) or > 1,000 x 10^9/L
* Significant concurrent/recent coagulopathy, history of non-traumatic significant bleeding; history of immune thrombocytopenic purpura (ITP); current use of SC anti-coagulants; history of thrombotic events, including stroke or DVT
* Clinically significant renal, liver or cardiac dysfunction
* Uncontrolled hypertension (> 140 mm Hg systolic or > 90 mm Hg diastolic)
* Fasting blood glucose > 2.0 × upper limit of normal (ULN)
* Inability to have a magnetic resonance imaging (MRI) scan
* Known history or positive test for human immunodeficiency virus (HIV), hepatitis C (HCV), or hepatitis B (HBV)
* Active infection requiring systemic antiviral or antimicrobial therapy
* Regular excessive use of alcohol
* Recent start of hydroxyurea (6 months prior to Day 1)
* Treatment with or recent exposure to another investigational drug, biological agent, ASO, small interfering ribonucleic acid (siRNA), or device within 1 month of Screening, or 5 half-lives of investigational agent, whichever is longer; or treatment with or exposure to:

* sotatercept (ACE-011), luspatercept (ACE-536), or ruxolitinib within 4 months of Screening
* hematopoietic stimulating agents or any hypoxia-inducible factor prolyl hydroxylase inhibitors within 8 weeks of Day 1
* prior bone marrow transplant, stem cell transplant, or gene therapy
* Surgery associated with significant blood loss within 4 months of Screening, splenectomy within 12 months of Screening, or splenectomy scheduled during treatment

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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
24/09/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
28/03/2023
Sample size
Target
Accrual to date
Final
29
Recruitment in Australia
Recruitment state(s)
NSW,VIC,WA
Recruitment hospital [1] 0 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [2] 0 0
Monash Medical Centre - Clayton
Recruitment hospital [3] 0 0
Royal Perth Hospital - Perth
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
3168 - Clayton
Recruitment postcode(s) [3] 0 0
6000 - Perth
Recruitment outside Australia
Country [1] 0 0
Greece
State/province [1] 0 0
Attica
Country [2] 0 0
Greece
State/province [2] 0 0
Peloponnese
Country [3] 0 0
Greece
State/province [3] 0 0
Thessaly
Country [4] 0 0
Lebanon
State/province [4] 0 0
Hazmiyeh
Country [5] 0 0
Thailand
State/province [5] 0 0
Bangkok
Country [6] 0 0
Thailand
State/province [6] 0 0
Chiang Mai
Country [7] 0 0
Thailand
State/province [7] 0 0
Khon Kaen
Country [8] 0 0
Thailand
State/province [8] 0 0
Pathum Thani
Country [9] 0 0
Thailand
State/province [9] 0 0
Pathum Wan
Country [10] 0 0
Thailand
State/province [10] 0 0
Phitsanulok
Country [11] 0 0
Thailand
State/province [11] 0 0
Songkhla
Country [12] 0 0
Turkey
State/province [12] 0 0
Adana
Country [13] 0 0
Turkey
State/province [13] 0 0
Ankara
Country [14] 0 0
Turkey
State/province [14] 0 0
Antalya
Country [15] 0 0
Turkey
State/province [15] 0 0
Izmir
Country [16] 0 0
Turkey
State/province [16] 0 0
Topkapi

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Ionis Pharmaceuticals, Inc.
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
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


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/NCT04059406

Additional trial details provided through ANZCTR
Accrual to date
Recruitment state(s)
Funding & Sponsors
Primary sponsor
Commercial sector/Industry
Primary sponsor name
Ionis Pharmaceuticals
Primary sponsor address
2855 Gazelle Court
Carlsbad, CA 92010
Primary sponsor country
United States of America
Ethics approval
Ethics application status
Approved
 
Public notes

Contacts
Principal investigator
Title 93 0
Name 93 0
Address 93 0
Country 93 0
Phone 93 0
Fax 93 0
Email 93 0
Contact person for public queries
Title 94 0
Name 94 0
Ionis Pharmaceuticals
Address 94 0
2855 Gazelle Court Carlsbad, CA 92010
Country 94 0
United States of America
Phone 94 0
1-800-679-4747
Fax 94 0
Email 94 0
[email protected]
Contact person for scientific queries
Title 95 0
Name 95 0
Ionis Pharmaceuticals
Address 95 0
2855 Gazelle Court Carlsbad, CA 92010
Country 95 0
United States of America
Phone 95 0
1-800-679-4747
Fax 95 0
Email 95 0
[email protected]