ISIS 702843 in patients with Phlebotomy Dependent Polycythemia Vera
Research type
Research Study
Full title
A Phase 2a, Randomized, Open-Label Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 702843 Administered to Patients with Phlebotomy Dependent Polycythemia Vera (PD-PV)
IRAS ID
1004740
Contact name
Terry Barrett
Contact email
Sponsor organisation
Ionis Pharmaceuticals, Inc.
Eudract number
2021-003704-40
Clinicaltrials.gov Identifier
Research summary
Patients with Polycythemia Vera (PV) have too many RBCs in their blood. They required to have phlebotomies (blood samples collected from a vein) to control their symptoms. This type of PV is called Phlebotomy-Dependent Polycythemia Vera (PD-PV).
This clinical study is testing a new treatment for this medical condition. The new treatment is called ISIS 702843. The study medication is designed to suppress the production of a protein called TMPRSS6. When this happens, the body increases the production of a different protein called hepcidin. Increased hepcidin limits how much iron is available for the body to create haemoglobin and red blood cells (RBCs).
The purpose of this clinical study is to check if suppressing the production of TMPRSS6 protein and allowing hepcidin production to increase can reduce the number of RBCs in the blood and improve patients’ disease.
The study medication is given as one or two subcutaneous (under the skin) injection(s) in the abdomen (stomach area, avoiding the belly button area), thigh, or upper arm. Participants will be assigned to receive one of 2 Dosing Levels – a higher or a lower level. Participants will have an equal chance of being assigned to either Dosing Level.
Participants will be asked to visit the study clinic 1 or 2 times every month for about 13 months for a total of 16 visits. Procedures to be performed include physical examinations, collection of vital signs, blood and urine samples, electrocardiogram (ECG)s, ultrasound of the spleen and completion of questionnaires.
REC name
London - Central Research Ethics Committee
REC reference
22/LO/0144
Date of REC Opinion
19 Apr 2022
REC opinion
Further Information Favourable Opinion