Trial Title:
Study to Assess SLN124 in Patients With Polycythemia Vera
NCT ID:
NCT05499013
Condition:
Polycythemia Vera
Conditions: Official terms:
Polycythemia Vera
Polycythemia
Conditions: Keywords:
PV
Study type:
Interventional
Study phase:
Phase 1/Phase 2
Overall status:
Recruiting
Study design:
Allocation:
Randomized
Intervention model:
Parallel Assignment
Intervention model description:
Phase 1 is an open-label, dose-finding study. Phase 2 is a randomized, double-blind,
placebo-controlled study.
Primary purpose:
Treatment
Masking:
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Intervention:
Intervention type:
Drug
Intervention name:
SLN124
Description:
SLN124 is a double-stranded small interfering ribonucleic acid (siRNA) targeting
transmembrane protease, serine 6 (TMPRSS6) messenger ribonucleic acid (mRNA).
Arm group label:
Phase 1 open-label SLN124
Arm group label:
Phase 2 Blinded SLN124
Intervention type:
Drug
Intervention name:
Placebo
Description:
sodium chloride, solution for injection
Arm group label:
Phase 2 Blinded Placebo
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.
Criteria for eligibility:
Criteria:
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.
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%.
Gender:
All
Minimum age:
18 Years
Maximum age:
N/A
Healthy volunteers:
No
Locations:
Facility:
Name:
University of Michigan
Address:
City:
Ann Arbor
Zip:
48109
Country:
United States
Status:
Recruiting
Contact:
Last name:
Moshe Talpaz, Dr
Investigator:
Last name:
Moshe Talpaz, Dr
Email:
Principal Investigator
Facility:
Name:
Mount Sinai Hospital
Address:
City:
New York
Zip:
10029
Country:
United States
Status:
Recruiting
Contact:
Last name:
Marina Kremyanskaya, Dr
Email:
marina.kremyanskaya@mssm.edu
Investigator:
Last name:
Marina Kremyanskaya, Dr
Email:
Principal Investigator
Facility:
Name:
Duke Cancer Institute
Address:
City:
Durham
Zip:
27705
Country:
United States
Status:
Not yet recruiting
Contact:
Last name:
Lindsay Anne Magura Rein, Dr
Investigator:
Last name:
Lindsay Anne Magura Rein, Dr
Email:
Principal Investigator
Facility:
Name:
North Houston Cancer Clinics
Address:
City:
Huntsville
Zip:
77340
Country:
United States
Status:
Completed
Facility:
Name:
Renovatio Clinical
Address:
City:
The Woodlands
Zip:
77380
Country:
United States
Status:
Recruiting
Contact:
Last name:
Maya Fleyhan
Phone:
713-703-2398
Email:
maya.fleyhan@renovatioclinical.com
Investigator:
Last name:
Jonathan Lu, Dr
Email:
Principal Investigator
Facility:
Name:
Pindara Private Hospital
Address:
City:
Benowa
Zip:
4217
Country:
Australia
Status:
Recruiting
Contact:
Last name:
Hanlon Sia, Dr
Phone:
07 5597 1211
Investigator:
Last name:
Hanlon Sia, Dr
Email:
Principal Investigator
Facility:
Name:
Ashford Cancer Centre Research
Address:
City:
Kurralta Park
Zip:
5037
Country:
Australia
Status:
Recruiting
Contact:
Last name:
Sue Yeend
Email:
sue.yeend@icon.team
Investigator:
Last name:
Akash Kalro, Dr
Email:
Principal Investigator
Facility:
Name:
Peter MacCallum Cancer Centre
Address:
City:
Melbourne
Zip:
3000
Country:
Australia
Status:
Recruiting
Contact:
Last name:
Ashley Ng, Dr
Phone:
(03) 8559 7456
Email:
PCCTU.HaemC@petermac.org
Investigator:
Last name:
Ashley Ng, Dr
Email:
Principal Investigator
Facility:
Name:
Alfred Health
Address:
City:
Melbourne
Zip:
3004
Country:
Australia
Status:
Recruiting
Contact:
Last name:
Andrew Perkins, MD
Phone:
(03) 9076 3451
Email:
Andrew.perkins@monash.edu
Investigator:
Last name:
Andrew Perkins, Prof
Email:
Principal Investigator
Facility:
Name:
Epworth HealthCare
Address:
City:
Richmond
Zip:
3121
Country:
Australia
Status:
Recruiting
Contact:
Last name:
Indu Raman, Dr
Phone:
(03) 9936 8216
Email:
Indu.Raman2@epworth.org.au
Investigator:
Last name:
Indu Raman, Dr
Email:
Principal Investigator
Facility:
Name:
Linear Clinical Research
Address:
City:
Nedlands
Zip:
6009
Country:
Australia
Status:
Recruiting
Contact:
Last name:
Carolyn Grove, Dr
Phone:
+61 1300 54 6327
Email:
contactus@linear.org.au
Investigator:
Last name:
Carolyn Grove, Dr
Email:
Principal Investigator
Facility:
Name:
MHAT Dr Nikola Vasiliev AD
Address:
City:
Kyustendil
Zip:
2500
Country:
Bulgaria
Status:
Recruiting
Contact:
Last name:
Desislava Ilieva-Chiviyska, Dr
Phone:
+35 9888474560
Email:
dr.desislava.ilieva-chiviyska@gmail.com
Investigator:
Last name:
Desislava Ilieva-Chiviyska, Dr
Email:
Principal Investigator
Facility:
Name:
Medical Centre Leo Clinic EOOD
Address:
City:
Plovdiv
Zip:
4003
Country:
Bulgaria
Status:
Recruiting
Contact:
Last name:
Anna Filipova
Phone:
+359 878955662
Email:
Anna.filipova@pratia.com
Contact backup:
Last name:
Ivelina Mladenova
Phone:
+359 888 887 625
Email:
ivelina.mladenova@pratia.com
Investigator:
Last name:
Boyan Semov, Dr
Email:
Principal Investigator
Facility:
Name:
Hospital Sultanah Aminah
Address:
City:
Johor Bahru
Zip:
80100
Country:
Malaysia
Status:
Recruiting
Contact:
Last name:
Azizan Sharif, Dr
Phone:
6012 758-0827
Email:
azinar@gmail.com
Contact backup:
Last name:
Wong Yih Seong, Dr
Phone:
6012 783-1871
Email:
yihseong@gmail.com
Investigator:
Last name:
Azizan Sharif, Dr
Email:
Principal Investigator
Facility:
Name:
Hospital Tengku Ampuan Afzan
Address:
City:
Kuantan
Zip:
25100
Country:
Malaysia
Status:
Recruiting
Contact:
Last name:
Ahlam Naila Kori, Dr
Phone:
6013-930 7777
Email:
ahlamnaila@gmail.com
Contact backup:
Last name:
Normala Binti Nordin, Dr
Phone:
6019-664 8644
Email:
normalanordin80@gmail.com
Investigator:
Last name:
Ahlam Naila Kori, Dr
Email:
Principal Investigator
Facility:
Name:
Hospital Umum Sarawak
Address:
City:
Kuching
Zip:
93586
Country:
Malaysia
Status:
Recruiting
Contact:
Last name:
Chew Lee Ping, Dr
Email:
leepingc@gmail.com.my
Contact backup:
Last name:
Tiong Xun Ting, Dr
Email:
tiongxt@gmail.com
Investigator:
Last name:
Chew Lee Ping, Dr
Email:
Principal Investigator
Facility:
Name:
Hospital Ampang
Address:
City:
Ampang
Zip:
68000
Country:
Malaysia
Status:
Recruiting
Contact:
Last name:
V Selvaratnam, Dr
Email:
veena_263@yahoo.com
Contact backup:
Last name:
Ong Jiun Jyh, Dr
Email:
jynx87@yahoo.com.my
Investigator:
Last name:
Veena Selvaratnam, Dr
Email:
Principal Investigator
Facility:
Name:
Hospital Sultanah Nur Zahirah
Address:
City:
Kuala Terengganu
Zip:
20400
Country:
Malaysia
Status:
Recruiting
Contact:
Last name:
Ganesh A/L Kasinathan, Dr
Phone:
6012-6238014
Email:
ganeshkasinathan11@hotmail.com
Contact backup:
Last name:
Nur Aina Binti Abdul Rohim
Phone:
6019-3141698
Email:
nur.aina@clinicalresearch.my
Investigator:
Last name:
Ganesh Kasinathan, Dr
Email:
Principal Investigator
Facility:
Name:
Uniwersyteckie Centrum Klinlczne
Address:
City:
Gdańsk
Country:
Poland
Status:
Recruiting
Contact:
Last name:
Witold Prejzner, Dr
Email:
wprejzner@uck.gda.pl
Investigator:
Last name:
Witold Prejzner, Dr
Email:
Principal Investigator
Facility:
Name:
PRATIA Hematologia Sp. z o. o.
Address:
City:
Katowice
Country:
Poland
Status:
Recruiting
Contact:
Last name:
Sebastian Grosicki, Dr
Email:
Sebastian.grosicki@pratia.com
Investigator:
Last name:
Sebastian Grosicki, Dr
Email:
Principal Investigator
Facility:
Name:
Samodzielny Publiczny Szpital Kliniczny Nr 1 w Lublinie
Address:
City:
Lublin
Country:
Poland
Status:
Recruiting
Contact:
Last name:
Michal Mielnik
Phone:
608033811
Investigator:
Last name:
Marek Hus, Dr
Email:
Principal Investigator
Facility:
Name:
Centrum Medyczne Pratia Poznan Sp. z o. o.
Address:
City:
Skorzewo
Country:
Poland
Status:
Recruiting
Contact:
Last name:
Roza Jerzewska
Phone:
+48 794 006 988
Email:
roza.jerzewska@pratia.com
Contact backup:
Last name:
Kinga Kuzdak
Phone:
+48 506 969 916
Investigator:
Last name:
Michal Kwiatek, Dr
Email:
Principal Investigator
Facility:
Name:
Specjalistyczny Szpital Im Dra. A.
Address:
City:
Wałbrzych
Zip:
58-300
Country:
Poland
Status:
Recruiting
Contact:
Last name:
Aleksandra Butrym, MD
Email:
aleksandra.butrym@gmail.com
Investigator:
Last name:
Aleksandra Butrym, MD
Email:
Principal Investigator
Start date:
January 26, 2023
Completion date:
June 2025
Lead sponsor:
Agency:
Silence Therapeutics plc
Agency class:
Industry
Source:
Silence Therapeutics plc
Record processing date:
ClinicalTrials.gov processed this data on November 12, 2024
Source: ClinicalTrials.gov page:
https://clinicaltrials.gov/ct2/show/NCT05499013
