Clinical study in order to compare the effectiveness and safety of two different treatments in patients with newly diagnosed primary immune thrombocytopenia

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Fundacion Publica Andaluza Para La Gestion De La Investigacion En Salud De Sevilla

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

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

Trial duration

2 Dec 2022 → ongoing

Decision date (initial)

2024-07-23

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

External identifiers

EU CT number

2024-514147-28-00

EudraCT number

2021-006970-22

ClinicalTrials.gov

NCT05325593

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety, Therapy

To evaluate the superiority of romiplostim plus dexamethasone versus dexamethasone alone after 6 months (≥180 days) from treatment cessation in patients with newly diagnosed primary immune thrombocytopenia (ITP) in terms of sustained response off any ITP treatment (6mSROT-50) and without WHO grade 2 or more bleeding.

Secondary objectives 1

1. - To evaluate the difference between study arms in the proportion of patients with newly diagnosed of ITP achieving platelets >= than 30x109/L (SROT-30) or 50 x109/L (SROT-50) in the absence of any ITP treatment including any rescue treatment for at least 6 consecutive months (≥180 days) or 12 consecutive months from treatment cessation and without WHO grade 2 or more bleeding. - To evaluate the difference between study arms in the proportion of patients with newly diagnosed of ITP with complete response, response, global response and targeted range. Also with early response and initial response - To compare the time to loss of response(LoR)in patients who achieved response in both arms. - To compare the difference between study arms in the proportion of patients requiring any rescue treatment along the study period. - To compare the difference between study arms in the proportion of patients with adverse events, including serious adverse events and laboratory safety parameters.

Conditions and MedDRA coding

Primary immune thrombocytopenia

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. 1.Age ≥ 18 years of age at the time of signing informed consent. 2.Newly diagnosis of primary ITP according to the International Working Group assessment [1] and previously untreated for ITP. 3.Platelet counts <30x109/L or ITP with platelet counts <50x109/L and concomitant bleeding symptoms. 4.Serum creatinine concentration ≤1.5 mg/dL.

Exclusion criteria 1

1. 1.WHO performance status >2. 2.Previous therapy with rituximab (within 3 months previous of study enrollment), corticosteroids, therapy with other immunomodulating agents within 1 month of enrolment, hematopoietic analogs and fostamatinib for any other reason than ITP. 3.Previous use of romiplostim, PEG-recombinant human (rHu) megakaryocyte growth and development factor, eltrombopag, recombinant human anti-thrombopoietin (rHuTPO), or any plateletproducing agent for three months prior to enrolment. 4.Alkylating agents within 8 weeks before the screening visit or anticipated use during the time of the proposed study. 5.Splenectomy within 3 months of the screening visit or planned splenectomy during study period. 6.Abnormal renal function (serum creatinine > 1.5 mg/dL). 7.Active hepatic disease (alanine aminotransferase [ALT] or aspartate aminotransferase [AST] levels >5 times the upper limit of normal). 8.Severe chronic liver disease as evidenced by, but not limited to, any of the following: International Normalized Ratio (INR) > 1.4, hypoalbuminemia, portal vein hypertension including presence of otherwise unexplained splenomegaly and history of esophageal varices. 9.Pregnancy or lactation. 10.Patients with known IgM seropositive tests for cytomegalovirus and/or Epstein-Barr virus in the previous month. 11.Patients with known serum-positivity and a positive test for an active viral infection at screening with: Hepatitis B Virus (HBV), Hepatitis C Virus (HCV), detectable virus charge of HIV. 12.Intolerance to dexamethasone or romiplostim. 13.History of a bone marrow stem cell disorder. 14.Active or prior malignancy except adequately treated (ie, complete surgical excision with negative margins) basal cell carcinoma in the last 5 years.. 15.History of Heliobacter pylori by urea breath test or stool antigen test within 6 months of enrollment, if available. 16.History of myelodysplastic syndrome, systemic lupus erythematosus, or autoimmune cytopenia. 17.History of antiphospholipid antibody syndrome. 18.History of disseminated intravascular coagulation, hemolytic uremic syndrome, or thrombotic thrombocytopenic purpura. 19.History of deep or superficial venous thromboembolism in the last 12 months or stroke, acute ischaemic heart disease or acute peripheral vascular disese in the last 6 months. 20.Hypersensitivity to any recombinant Escherichia coli-derived product (eg, Infergen, Neupogen, Somatropin, and Actimmune) or known sensitivity to any of the products to be administered during dosing 21.Currently enrolled in another investigational device or drug study or < 30 days since ending another investigational device or drug studies, or receiving other investigational agents. 22.Will have any other investigational procedures performed while enrolled in this clinical study. 23.Pregnant or breastfeeding, or planning to become pregnant or breastfeed during treatment or within 1 month after the end of treatment. 24.Female subject of childbearing potential is not willing to use, in combination with her partner, an acceptable method of effective contraception during treatment and for 1 month after the end of treatment (see annex 5 for additional contraception information). Females of childbearing potential should only be included after a negative, highly sensitive urine pregnancy test. 25.Will not be available for protocol-required study visits, to the best of the subject's and investigator's knowledge. 26.Any kind of disorder that, in the opinion of the investigator, may compromise the ability of the subject to give written informed consent and/or to comply with all required study procedures. 27.Other serious comorbidities at investigator criteria.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. To evaluate the difference between study arms in the proportion of patients achieving 6mSROT-50 at 6 months (180 days) from treatment cessation. Definition of 6mSROT-50: platelets higher or equal than 50x109/L in the absence of any ITP treatment including any rescue treatment for at least 6 consecutive months (≥180 days) from treatment cessation and without WHO grade 2 or more bleeding.

Secondary endpoints 1

1. -To evaluate the difference between study arms in the proportion of patients achieving 6mSROT-30 at 6 months (180 days) from treatment cessation. -To evaluate the difference between study arms in the proportion of patients achieving 12mSROT-50 at 12 months (365 days) from treatment cessation. -To evaluate the difference between study arms in the proportion of patients achieving 12mSROT-30 at 12 months (365 days) from treatment cessation

Investigational products

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

Test 2

Comparator 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Fundacion Publica Andaluza Para La Gestion De La Investigacion En Salud De Sevilla

Sponsor organisation

Fundacion Publica Andaluza Para La Gestion De La Investigacion En Salud De Sevilla

Address

Avenida De Manuel Siurot Sn

City

Sevilla

Postcode

41013

Country

Spain

Scientific contact point

Organisation

Fundacion Publica Andaluza Para La Gestion De La Investigacion En Salud De Sevilla

Contact name

Clinical Trial Unit IBIS/University Hospital Virgen del Rocío

Public contact point

Organisation

Fundacion Publica Andaluza Para La Gestion De La Investigacion En Salud De Sevilla

Contact name

Clinical Trial Unit IBIS/University Hospital Virgen del Rocío

Locations

2 EU/EEA countries · 21 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

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

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