A Phase III, Open-label, Single Arm, Prospective, Multicenter Study to Assess Efficacy and Safety of Kedrion Intravenous Human Normal Immunoglobulin (IVIg) 10% in Adult Patients with Chronic Immune Thrombocytopenia (ITP)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Kedrion S.p.A.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Trial duration

3 Nov 2025 → ongoing

Decision date (initial)

2025-04-30

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy, Others, Dose response

To assess the responder rate of KIg10 in the treatment of adults with chronic ITP by measuring platelet count increase according to the Response (R) definition

Secondary objectives 7

1. • To assess the clinical response rate of KIg10 platelet count increase and absence of bleeding according to the Complete Response (CR), Loss of R/CR, Nonresponders definitions.
2. • To assess the time to platelet count response.
3. • To assess the duration of response.
4. • To assess the time to stop bleeding.
5. • To assess the maximum platelet count.
6. • To assess time to achieve maximum platelet count.
7. • To assess the reduction of bleeding symptoms

Conditions and MedDRA coding

Chronic Immune Thrombocytopenia (ITP)

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 8

1. 1. Male or female, 18-70 years of age.
2. 2. Patient and/or legal authorized representative has signed the ICF.
3. 3. Diagnosis of chronic (> 12 months duration) ITP as defined by the International Working Group.
4. 4. Mean screening platelet count of < 30 × 109/L from two qualifying counts measured at least one calendar day apart. The first qualifying count can be from historical data if measured within 7 days prior to the screening. The second qualifying count will be measured within 7 days before the first KIg10 infusion.
5. 5. Platelet count of < 30 × 109/L at the Baseline Visit.
6. 6. Patient is willing to comply with all requirements of the protocol.
7. 7. Women of childbearing potential must have a negative urine pregnancy test at screening and agree to employ adequate birth control measures during the study.
8. 8. Authorization to access personal health information.

Exclusion criteria 21

1. 1. Patients with secondary ITP
2. 10. Received platelets within 7days of Visit 1 and any other blood/plasma product within 1 month of the Baseline Visit (BV)
3. 11. Received recombinant activated factor VII within 7 days rituximab within 6 months of the BV
4. 12 Received live attenuated virus vaccines within 3 months of the BV
5. 13. Use of loop diuretics w/i 1 week of the BV
6. 14. Uncontrolled hypertension
7. 15. Congestive heart failure as per New York Heart Association III/IV, cardiomyopathy, cardiac arrhythmia associated with thromboembolic events , unstable or advanced ischemic heart disease, hyperviscosity
8. 16. Patients with significant protein losing enteropathy, nephrotic syndrome, lymphangiectasia, hyperproteinemia, increased serum viscosity and/or hyponatremia
9. 17. Severe liver or kidney disease
10. 18. Signs of severe anemia
11. 19. BMI > 40 kg/m2 or an IVIg dose that puts the patient at risk of fluid overload
12. 2. Patients with Evans Syndrome, inherited thrombocytopenia, myelodysplastic syndrome
13. 20. History of a malignant disease within 3 years of the Baseline Visit other than treated carcinoma in situ of the cervix or basal cell or squamous cell carcinoma of the skin
14. 21. Patient has participated in an interventional, investigational clinical study within 30 days of the Baseline Visit
15. 3. Patients infected with HBV, HCV or HIV
16. 4. Patients with a history of thrombotic events and/or at high risk of thrombotic events
17. 5. History of hypersensitivity to IVIg or to any of the excipients
18. 6. Patients unresponsive previously to IVIg or anti-D Ig treatment
19. 7. Patients with immunoglobulin A deficiency and Ab against IgA
20. 8. Splenectomy within 4 weeks of the Baseline Visit or planned throughout the study
21. 9. Administration of IVIg, anti-D Ig, Mercaptopurine, Vinca alkaloid, or platelet enhancing drugs within 3 weeks of the Baseline Visit unless patients are on stable treatment as defined. Treatment with any other products licensed for primary chronic ITP is also exclusive.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The rate of participants with response (R), defined as participants with a platelet count > 30 × 109/L and at least a 2-fold increase of the baseline count, confirmed during the evaluation period on at least 2 separate occasions at least 7 days apart, and absence of bleeding during the evaluation period.

Secondary endpoints 7

1. 1.Number and rate of participants with: a.CR: PLT > 100x109/L on 2 occasions at least 7 days apart and no bleeding b. loss of CR or R: PLT <100x109/L or bleeding ( CR) or PLT <30x109/L or < 2-fold increase of baseline or bleeding (R) c.Nonresponders (NR)
2. 2.Time to response
3. 3. Response Duration
4. 4. Hemorrhages regression of
5. 5. maximum PLT count achieved
6. 6.Time to maximum PLT count
7. 7.Bleedings (timing/grade)

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Kedrion S.p.A.

Sponsor organisation

Kedrion S.p.A.

Address

Castelvecchio Pascoli, Localita Ai Conti Snc Localita Ai Conti Snc

City

Barga

Postcode

55051

Country

Italy

Scientific contact point

Organisation

Kedrion S.p.A.

Contact name

Miranda Norton

Public contact point

Organisation

Kedrion S.p.A.

Contact name

Miranda Norton

Third parties 4

Locations

5 EU/EEA countries · 14 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 73 files

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

Application history

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