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

• Register: ClinicalTrials.gov
• Last refreshed on: 6 December 2021
• Main ID: NCT05135442
• Date of registration: 06/11/2021
• Prospective Registration: Yes
• Primary sponsor: Peking Union Medical College Hospital
• Public title: Efficacy and Safety of Bortezomib as First-line Treatment of Acquired TTP
• Scientific title: The Efficacy and Safety of Bortezomib as First-line Treatment for Acquired Thrombotic Thrombocytopenic Purpura
• Date of first enrolment: December 1, 2021
• Target sample size: 30
• Recruitment status: Not yet recruiting
• URL: https://clinicaltrials.gov/show/NCT05135442
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 4

Countries of recruitment

China

Contacts

• Name: jing yang

• Name: tienan zhu
• Affiliation: Peking Union Medical College Hospital

• Name: jing yang

Key inclusion & exclusion 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.



Age minimum: 18 Years
Age maximum: 80 Years
Gender: All

Health Condition(s) or Problem(s) studied

Thrombotic Thrombocytopenic Purpura, Acquired

Intervention(s)

Drug: Bortezomib Injection

Primary Outcome(s)

Time to clinical remission and the number of plasma exchanges required [Time Frame: one month]

Secondary Outcome(s)

ICU hospitalization time and total hospitalization time; [Time Frame: 6 months]

Mortality rate [Time Frame: 6 months]

Secondary ID(s)

FTTPB

Source(s) of Monetary Support

Please refer to primary and secondary sponsors