Rituximab in Patients With Relapsed or Refractory TTP-HUS

Hamilton Health Sciences (HHS)

Status and phase: Unknown; Phase 2

Conditions: Hemolytic Uremic Syndrome; Thrombotic Thrombocytopenic Purpura

Treatments: Drug: Rituximab

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT00531089

CAG-1

Details and patient eligibility

About

The general objective of this study is to assess the efficacy and safety of Rituximab in the management of patients with refractory or relapsed thrombotic thrombocytopenic purpura-hemolytic uremic syndrome (TTP-HUS). There have been several case reports and case series describing the use of Rituximab in patients with TTP-HUS; however its use has not been studied in a large trial. It is hypothesized that Rituximab may ameliorate the severity of certain cases of TTP-HUS by decreasing the number of activity of B-cells which may result in decreased production of the ADAMTS13 protease inhibitor. Patients with TTP-HUS not responding to standard therapy or patients with relapsed disease may have particular benefit. Treatments that decrease the frequency of relapse or shorten the time to remission of TTP-HUS will be of benefit by decreasing the need for blood product support.

Enrollment: 60 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• any patient 18 years or older diagnosed with relapsed or refractory TTP-HUS requiring therapy

Exclusion criteria

• alternate cause of hemolytic microangiopathy (evidence of DIC, malignant hypertension, vasculitis, anti-phospholipid antibody syndrome, post-partum acute renal failure)
• congenital or familial TTP
• TTP occuring post-stem cell, bone marrow, or solid organ transplant
• drug-induced TTP
• pregnancy or breast-feeding
• history of hepatitis B or C infection
• prior rituximab treatment
• active or metastatic cancer
• other causes of thrombocytopenia such as ITP, myelodysplastic syndrome, confirmed or suspected drug-induced thrombocytopenia
• refusal to receive blood products
• hypersensitivity to blood products, plasma products, murine proteins, or any component of the Rituximab formulation
• geographic inaccessibility
• co-morbid illness limiting life expectancy to less than 2 months independent of TTP
• failure to provide written informed consent

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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60 participants in 1 patient group

Study group

Experimental group

Description:

All patients in the study will be in the study group and will receive rituximab. There is no "control" arm.

Treatment:

Drug: Rituximab