Efficacy and Safety of Bortezomib as First-line Treatment of Acquired TTP

NCT ID: NCT05135442

Last Updated: 2021-11-26

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE4
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-12-01
• Study Completion Date: 2025-11-30

Brief Summary

To evaluate the efficacy and safety of bortezomib in the first-line treatment of patients with acquired TTP，we design this prospective, multi-center, single-arm interventional study.All enrolled TTP patients were given bortezomib 1.3 mg/m2 intravenous injection d1, 4, 8, on the basis of standard single membrane plasma exchange (2L/d) and hormone therapy (1mg/kg prednisone or equivalent methylprednisolone). 11 (4 doses in total). Bortezomib should be administered immediately after each plasma exchange, and the interval between the next plasma exchange is> 24h. Plasma exchange continued until the patient's platelet count was >100×109/L for 2 consecutive days, and then changed to once every other day for a total of two times and then stopped.

Detailed Description

To evaluate the efficacy and safety of bortezomib in the first-line treatment of patients with acquired TTP，we design this prospective, multi-center, single-arm interventional study.All enrolled TTP patients were given bortezomib 1.3 mg/m2 intravenous injection d1, 4, 8, on the basis of standard single membrane plasma exchange (2L/d) and hormone therapy (1mg/kg prednisone or equivalent methylprednisolone). 11 (4 doses in total). Bortezomib should be administered immediately after each plasma exchange, and the interval between the next plasma exchange is> 24h. Plasma exchange continued until the patient's platelet count was >100×109/L for 2 consecutive days, and then changed to once every other day for a total of two times and then stopped.

Conditions

Thrombotic Thrombocytopenic Purpura, Acquired

Keywords

thrombotic thrombocytopenic purpura; bortezomib; plasma exchange

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

bortezomib group

On the basis of standard single membrane plasma exchange (2L/d) and hormone therapy (1mg/kg prednisone or equivalent methylprednisolone), bortezomib was given intravenous injection of 1.3mg/m2 d1, 4, 8, 11 (total 4 doses).

Group Type: EXPERIMENTAL

Bortezomib Injection

Intervention Type: DRUG

Bortezomib should be administered immediately after each plasma exchange, and the interval between the next plasma exchange is\> 24h. Plasma exchange continued until the patient's platelet count was \>100×109/L for 2 consecutive days, and then changed to once every other day for a total of two times and then stopped.

Interventions

Bortezomib Injection

Bortezomib should be administered immediately after each plasma exchange, and the interval between the next plasma exchange is\> 24h. Plasma exchange continued until the patient's platelet count was \>100×109/L for 2 consecutive days, and then changed to once every other day for a total of two times and then stopped.

Intervention Type: DRUG

Other Intervention Names

plasma exchange; hormone therapy

Eligibility Criteria

Inclusion Criteria

• clinically diagnosed as TTP (thrombocytopenia + MAHA± clinical evidence of related organ damage， a significant reduction in ADAMTS13 activity level and/or positive antibody screening）
• elder than 18 years old;
• informed consent is required;

Exclusion Criteria

• Uncontrollable systemic infection;
• Known allergy to bortezomib;
• Expected survival time <1 week;
• Pregnant or lactating women (women of childbearing age have a positive pregnancy test at baseline or have not received a pregnancy test. Postmenopausal women must be at least 12 months after menopause);
• If the creatinine level is ≥200μmol/l (1.5mg/dl), the levels of transaminase and bilirubin are 2 times higher than the upper limit of normal (except due to the primary disease);
• Known congenital TTP or a clear family history of TTP;
• Other diseases that cause microangiopathic hemolysis and thrombocytopenia, such as DIC, APS, HUS, malignant hypertension, transplantation-related microangiopathy;
• active malignant tumors (except skin basal cell carcinoma or cervical carcinoma in situ) ( have not been treated or recurred within 5 years before signing the informed consent);
• peripheral neuropathy;
• Patients or family members cannot understand the conditions and goals of this study;
• The investigator believes that the patient should not participate in any other situations in this trial.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Peking Union Medical College Hospital

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

tienan zhu

Role: STUDY_CHAIR

Peking Union Medical College Hospital

Locations

Peking Union Medical College Hospital

Beijing, (Select), China

Countries

China

Central Contacts

jing yang

Role: CONTACT

Phone: 1069159146

Email: yangbujing@126.com

huacong cai

Role: CONTACT

Email: caihc@pumch.cn

Facility Contacts

jing yang

Role: primary

References

Scully M, Cataland S, Coppo P, de la Rubia J, Friedman KD, Kremer Hovinga J, Lammle B, Matsumoto M, Pavenski K, Sadler E, Sarode R, Wu H; International Working Group for Thrombotic Thrombocytopenic Purpura. Consensus on the standardization of terminology in thrombotic thrombocytopenic purpura and related thrombotic microangiopathies. J Thromb Haemost. 2017 Feb;15(2):312-322. doi: 10.1111/jth.13571. Epub 2017 Jan 30.

Reference Type: BACKGROUND

PMID: 27868334 (View on PubMed)

Patriquin CJ, Thomas MR, Dutt T, McGuckin S, Blombery PA, Cranfield T, Westwood JP, Scully M. Bortezomib in the treatment of refractory thrombotic thrombocytopenic purpura. Br J Haematol. 2016 Jun;173(5):779-85. doi: 10.1111/bjh.13993. Epub 2016 Mar 24.

Reference Type: BACKGROUND

PMID: 27009919 (View on PubMed)

Shortt J, Oh DH, Opat SS. ADAMTS13 antibody depletion by bortezomib in thrombotic thrombocytopenic purpura. N Engl J Med. 2013 Jan 3;368(1):90-2. doi: 10.1056/NEJMc1213206. No abstract available.

Reference Type: BACKGROUND

PMID: 23281998 (View on PubMed)

Other Identifiers

FTTPB

Identifier Type: -

Identifier Source: org_study_id