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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT05499013
Registration number
NCT05499013
Ethics application status
Date submitted
29/07/2022
Date registered
12/08/2022
Date last updated
22/04/2024
Titles & IDs
Public title
Study to Assess SLN124 in Patients With Polycythemia Vera
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Scientific title
Phase 1/2 Study With an Open-label Dose Escalation Phase Followed by a Randomized, Double-blind Phase of SLN124 in Patients With Polycythemia Vera
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Secondary ID [1]
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SANRECO
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Secondary ID [2]
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SLN124-004
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Universal Trial Number (UTN)
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Trial acronym
SLN
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Polycythemia Vera
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Condition category
Condition code
Blood
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Haematological diseases
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Blood
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Other blood disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - SLN124
Treatment: Drugs - Placebo
Experimental: Phase 1 open-label SLN124 - SLN124 for subcutaneous (s.c.) injection
Experimental: Phase 2 Blinded SLN124 - SLN124 for subcutaneous (s.c.) injection
Placebo Comparator: Phase 2 Blinded Placebo - Sodium chloride for s.c. injection
Treatment: Drugs: SLN124
SLN124 is a double-stranded small interfering ribonucleic acid (siRNA) targeting transmembrane protease, serine 6 (TMPRSS6) messenger ribonucleic acid (mRNA).
Treatment: Drugs: Placebo
sodium chloride, solution for injection
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Incidence of treatment-emergent adverse events (AEs)
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Assessment method [1]
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Safety and tolerability will be reported separately following open-label dose escalation phase and double-blind phase
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Timepoint [1]
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Day 239
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Primary outcome [2]
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Assessment of the number of phlebotomies at intervals
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Assessment method [2]
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Timepoint [2]
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6 months prior to dosing to Day 239
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Secondary outcome [1]
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Pharmacokinetic: area under the plasma concentration (AUC)
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Assessment method [1]
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Timepoint [1]
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Day 127
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Secondary outcome [2]
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Pharmacokinetic: peak plasma concentration (Cmax)
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Assessment method [2]
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Timepoint [2]
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Day 127
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Secondary outcome [3]
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Pharmacodynamic: change in haematocrit
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Assessment method [3]
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Timepoint [3]
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Day 1 to Day 239
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Secondary outcome [4]
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Pharmacodynamic: Change in Transferrin saturation (TSAT)
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Assessment method [4]
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Timepoint [4]
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Day 1 to Day 239
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Secondary outcome [5]
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Pharmacodynamic: Change in Hepcidin
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Assessment method [5]
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Timepoint [5]
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Day 1 to Day 239
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Eligibility
Key inclusion criteria
- Male and female patients aged 18 years or older.
- A confirmed diagnosis of PV according to the revised 2016 World Health Organization
criteria:
- Suitable phlebotomy history
- Must agree to adhere to appropriate contraception requirements
- Patients who are not receiving cytoreductive therapy must have been discontinued from
any prior cytoreductive therapy for at least 24 weeks before dosing and have recovered
from any adverse events due to cytoreductive therapy.
- Patients receiving cytoreductive therapy with hydroxyurea, interferon, busulfan or
ruxolitinib must have received a stable dose of cytoreductive therapy for at least 12
weeks before dosing and with no planned change in dose.
- Patients must have had a dermatological examination within 6 months prior to
screening.
- Must have an Eastern Cooperative Oncology Group score of 0, 1, or 2.
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
- Drug intolerance:
1. History of intolerance to oligonucleotides, or GalNAc, or any component of
SLN124.
2. History of intolerance to s.c. injections.
- Clinically significant thrombosis (e.g., deep vein thrombosis or splenic vein
thrombosis) within 12 weeks of screening.
- History of major bleeding events and/or a requirement for blood transfusion therapy
owing to bleeding in the last 6 months prior to screening.
- Meets the criteria for post-PV myelofibrosis as defined by the International Working
Group-Myeloproliferative Neoplasms Research and Treatment
- Any investigational drug less than 6 weeks prior to the first dose of study drug or
not recovered from effects of prior administration of any investigational agent.
- Any investigational or marketed product using GalNAc targeting less than 48 weeks
prior to administration of any investigational agent.
- Clinically significant co-morbidities
- Biochemical and hematological parameters:
1. Biochemical evidence of significant liver disease during screening
2. Hematological parameters at screening as follows: platelets 1,000,000/µL; or
white blood cell (WBC) count > 25,000/µL; or peripheral blasts < 1%.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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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
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 1/Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
26/01/2023
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/06/2025
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Actual
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Sample size
Target
65
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
QLD,SA,VIC,WA
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Recruitment hospital [1]
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Pindara Private Hospital - Benowa
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Recruitment hospital [2]
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Ashford Cancer Centre Research - Kurralta Park
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Recruitment hospital [3]
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Peter MacCallum Cancer Centre - Melbourne
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Recruitment hospital [4]
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Alfred Health - Melbourne
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Recruitment hospital [5]
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Epworth HealthCare - Richmond
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Recruitment hospital [6]
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Linear Clinical Research - Nedlands
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Recruitment postcode(s) [1]
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4217 - Benowa
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Recruitment postcode(s) [2]
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5037 - Kurralta Park
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Recruitment postcode(s) [3]
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3000 - Melbourne
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Recruitment postcode(s) [4]
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3004 - Melbourne
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Recruitment postcode(s) [5]
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3121 - Richmond
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Recruitment postcode(s) [6]
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6009 - Nedlands
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Michigan
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United States of America
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State/province [2]
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New York
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United States of America
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North Carolina
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United States of America
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Texas
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Country [5]
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Bulgaria
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State/province [5]
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Kyustendil
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Country [6]
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Bulgaria
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State/province [6]
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Plovdiv
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Country [7]
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Malaysia
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State/province [7]
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Johor
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Country [8]
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Malaysia
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State/province [8]
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Pahang
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Country [9]
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Malaysia
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State/province [9]
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Sarawak
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Country [10]
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Malaysia
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State/province [10]
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Selangor
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Malaysia
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State/province [11]
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Terengganu
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Poland
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State/province [12]
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Gdansk
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Poland
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Katowice
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Poland
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State/province [14]
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Lublin
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Poland
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State/province [15]
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Skorzewo
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Poland
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State/province [16]
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Walbrzych
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Silence Therapeutics plc
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Address
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Ethics approval
Ethics application status
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Summary
Brief summary
This is a Phase 1/2, multicenter study with an open-label dose escalation followed by a
randomized placebo controlled and double-blind phase of SLN124 in adult patients with
Polycythemia Vera (PV) to assess the safety, tolerability, efficacy, pharmacokinetic (PK),
and Pharmacodynamic (PD) response of SLN124.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT05499013
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Address
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Silence Therapeutics Patient Information
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Address
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Country
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Phone
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+44 (0) 20 3457 6900
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Fax
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Email
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[email protected]
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT05499013
Download to PDF