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Study to Assess SLN124 in Patients With Polycythemia Vera

Study Purpose

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.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Interventional
Eligible Ages 18 Years and Over
Gender All
More Inclusion & Exclusion 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%.

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT05499013
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Phase 1/Phase 2
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Silence Therapeutics plc
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

N/A
Principal Investigator Affiliation N/A
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Industry
Overall Status Recruiting
Countries Australia, Bulgaria, Malaysia, Poland, United States
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Polycythemia Vera
Arms & Interventions

Arms

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

Interventions

Drug: - SLN124

SLN124 is a double-stranded small interfering ribonucleic acid (siRNA) targeting transmembrane protease, serine 6 (TMPRSS6) messenger ribonucleic acid (mRNA).

Drug: - Placebo

sodium chloride, solution for injection

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

University of Michigan, Ann Arbor, Michigan

Status

Recruiting

Address

University of Michigan

Ann Arbor, Michigan, 48109

Site Contact

Moshe Talpaz, Dr

[email protected]

+44 (0) 20 3457 6900

Mount Sinai Hospital, New York, New York

Status

Recruiting

Address

Mount Sinai Hospital

New York, New York, 10029

Site Contact

Marina Kremyanskaya, Dr

[email protected]

+44 (0) 20 3457 6900

Duke Cancer Institute, Durham, North Carolina

Status

Not yet recruiting

Address

Duke Cancer Institute

Durham, North Carolina, 27705

Site Contact

Lindsay Anne Magura Rein, Dr

[email protected]

+44 (0) 20 3457 6900

North Houston Cancer Clinics, Huntsville, Texas

Status

Completed

Address

North Houston Cancer Clinics

Huntsville, Texas, 77340

Renovatio Clinical, The Woodlands, Texas

Status

Recruiting

Address

Renovatio Clinical

The Woodlands, Texas, 77380

Site Contact

Maya Fleyhan

[email protected]

713-703-2398

International Sites

Pindara Private Hospital, Benowa, Queensland, Australia

Status

Recruiting

Address

Pindara Private Hospital

Benowa, Queensland, 4217

Site Contact

Hanlon Sia, Dr

[email protected]

07 5597 1211

Ashford Cancer Centre Research, Kurralta Park, South Australia, Australia

Status

Recruiting

Address

Ashford Cancer Centre Research

Kurralta Park, South Australia, 5037

Site Contact

Sue Yeend

[email protected]

+44 (0) 20 3457 6900

Peter MacCallum Cancer Centre, Melbourne, Victoria, Australia

Status

Recruiting

Address

Peter MacCallum Cancer Centre

Melbourne, Victoria, 3000

Site Contact

Ashley Ng, Dr

[email protected]

(03) 8559 7456

Alfred Health, Melbourne, Victoria, Australia

Status

Recruiting

Address

Alfred Health

Melbourne, Victoria, 3004

Site Contact

Andrew Perkins, MD

[email protected]

(03) 9076 3451

Epworth HealthCare, Richmond, Victoria, Australia

Status

Recruiting

Address

Epworth HealthCare

Richmond, Victoria, 3121

Site Contact

Indu Raman, Dr

[email protected]

(03) 9936 8216

Linear Clinical Research, Nedlands, Western Australia, Australia

Status

Recruiting

Address

Linear Clinical Research

Nedlands, Western Australia, 6009

Site Contact

Carolyn Grove, Dr

[email protected]

+61 1300 54 6327

MHAT Dr Nikola Vasiliev AD, Kyustendil, Bulgaria

Status

Recruiting

Address

MHAT Dr Nikola Vasiliev AD

Kyustendil, , 2500

Site Contact

Desislava Ilieva-Chiviyska, Dr

[email protected]

+35 9888474560

Medical Centre Leo Clinic EOOD, Plovdiv, Bulgaria

Status

Recruiting

Address

Medical Centre Leo Clinic EOOD

Plovdiv, , 4003

Site Contact

Anna Filipova

[email protected]

+359 878955662

Hospital Sultanah Aminah, Johor Bahru, Johor, Malaysia

Status

Recruiting

Address

Hospital Sultanah Aminah

Johor Bahru, Johor, 80100

Site Contact

Azizan Sharif, Dr

[email protected]

6012 758-0827

Hospital Tengku Ampuan Afzan, Kuantan, Pahang, Malaysia

Status

Recruiting

Address

Hospital Tengku Ampuan Afzan

Kuantan, Pahang, 25100

Site Contact

Ahlam Naila Kori, Dr

[email protected]

6013-930 7777

Hospital Umum Sarawak, Kuching, Sarawak, Malaysia

Status

Recruiting

Address

Hospital Umum Sarawak

Kuching, Sarawak, 93586

Site Contact

Chew Lee Ping, Dr

[email protected]

+44 (0) 20 3457 6900

Hospital Ampang, Ampang, Selangor, Malaysia

Status

Recruiting

Address

Hospital Ampang

Ampang, Selangor, 68000

Site Contact

V Selvaratnam, Dr

[email protected]

+44 (0) 20 3457 6900

Hospital Sultanah Nur Zahirah, Kuala Terengganu, Terengganu, Malaysia

Status

Recruiting

Address

Hospital Sultanah Nur Zahirah

Kuala Terengganu, Terengganu, 20400

Site Contact

Ganesh A/L Kasinathan, Dr

[email protected]

6012-6238014

Uniwersyteckie Centrum Klinlczne, Gdańsk, Poland

Status

Recruiting

Address

Uniwersyteckie Centrum Klinlczne

Gdańsk, ,

Site Contact

Witold Prejzner, Dr

[email protected]

+44 (0) 20 3457 6900

PRATIA Hematologia Sp. z o. o., Katowice, Poland

Status

Recruiting

Address

PRATIA Hematologia Sp. z o. o.

Katowice, ,

Site Contact

Sebastian Grosicki, Dr

[email protected]

+44 (0) 20 3457 6900

Lublin, Poland

Status

Recruiting

Address

Samodzielny Publiczny Szpital Kliniczny Nr 1 w Lublinie

Lublin, ,

Site Contact

Michal Mielnik

[email protected]

608033811

Skorzewo, Poland

Status

Recruiting

Address

Centrum Medyczne Pratia Poznan Sp. z o. o.

Skorzewo, ,

Site Contact

Roza Jerzewska

[email protected]

+48 794 006 988

Specjalistyczny Szpital Im Dra. A., Wałbrzych, Poland

Status

Recruiting

Address

Specjalistyczny Szpital Im Dra. A.

Wałbrzych, , 58-300

Site Contact

Aleksandra Butrym, MD

[email protected]

+44 (0) 20 3457 6900