Combination Therapy with Interferon-Alpha2a and Ruxolitinib in Newly Diagnosed Patients with Polycythemia Vera. A Danish Safety and Efficacy Study.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Region Sjaelland

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Hemic and Lymphatic Diseases [C15]

Decision date (initial)

2024-11-04

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

External identifiers

EU CT number

2024-518216-39-00

EudraCT number

2018-004150-13

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety, Therapy

Safety profile of COMBI therapy in patients with polycythemia Vera after 24 months

Secondary objectives 1

1. 1. Safety profile of COMBI therapy in patients with polycythemia Vera after 12 months as assessed by AE’s, SAE’s and withdrawal AE’s/SAE’s. 2. Efficacy Profile as assessed by normalization of red blood cell values, white blood cell counts (WBC) and platelet counts (PLA); response rates (see below)*; changes in the JAK2V617F allele burden; proportion of patients suffering thrombosis and/or hemorrhage; proportion of patients achieving no need of phlebotomies; proportion of patients achieving normal spleen size; proportion of patients with progression of disease*; progression free survival.

Conditions and MedDRA coding

Polycythemia

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. The participant must meet criteria 1–3 AND at least one of 4–8 1. Age ≥ 18 years AND 2. A confirmed diagnosis of PV according to the WHO 2016 criteria AND 3. A negative test for active or latent tuberculosis (from within the last month) 4. Biochemical evidence of active disease as defined by a) Elevated hematocrit and/or red cell count and/or b) Leucocyte count > 10 x 109/L and/or c) Platelet count > 400 x 109/L. OR 5. Hypermetabolic symptoms such as weight loss (> 10% within 6 months), night sweats and subfebrilia (temperature> 38 º C for more than 2 weeks without signs of infection OR 6. Pruritus OR 7. Symptomatic splenomegaly OR 8. Presence of previous thrombosis

Exclusion criteria 1

1. 1. Patients of childbearing potential without a negative pregnancy test prior to initiation of study drug. Participants must use contraceptives during the entire study period*. 2. Other active malignancy within the past 5 years (not including non-melanoma skin cancer and prostate cancer without need for treatment). 3. ECOG function scores> / = 3 4. Serum creatinine more than 2 x ULN 5. Total serum bilirubin greater than 1.5 x ULN 6. Plasma ALAT more than 3 x ULN 7. Former psychiatric disorder (depression diagnosed by a psychiatrist) 8. Uncontrolled metabolic (endocrinological) disease. 9. Severe heart disease (heart failure NYHA class 3-4). 10. Severe myelosuppression. (WBC<1.5 Mill/L, PLA<100) 11. Chronic hepatitis with decompensated cirrhosis. 12. Chronic hepatitis in patients who have been or recently (within 6 months) has been treated with immunosuppressive drugs except for corticosteroid treatment. 13. Epilepsy and / or other serious CNS disorders. *Spiral, birth control pills, implants, transdermal patch, vaginal ring or transdermal injection. Sterile / infertile subjects are exempt from the use of contraception. To be considered sterile or infertile, one must generally be surgically sterilized (vasectomy, bilateral tubectomy, hysterectomy or ovariectomy) or be postmenopausal, defined as absent menstruation for at least 12 months before study enrollment.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Safety Profile: relevant adverse events (AE), serious AEs (SAE), withdrawal due to AEs/SAE

Secondary endpoints 1

1. 1. Safety Profile: adverse events (AE), serious AEs (SAE), withdrawal, AEs/SAEs 2. Efficacy Profile: a) Proportion of patients achieving complete hematological remission (HCT < 45, PLA ≤400 and WBC < 10) after 3, 6, 8, 9, 12, 18 and 24 months. b) Remission rates at 12 and 24 months. c) JAK2V617F allele burden measured at baseline and after 3,6,12 and 24 months. d) Proportion of patients suffering thrombosis and/or hemorrhage at 12 at 24 months. with more e)-j)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Region Sjaelland

Sponsor organisation

Region Sjaelland

Address

Sygehusvej 10

City

Roskilde

Postcode

4000

Country

Denmark

Scientific contact point

Organisation

Region Sjaelland

Contact name

Sponsor/PI Hans Hasselbalch

Public contact point

Organisation

Region Sjaelland

Contact name

Sjællands Universitetshospital - Hæmatologisk afdeling

Third parties 1

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 5 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications