Acquired Thrombotic Thrombocytopenic Purpura
Conditions
Brief summary
This is a multicenter, randomized, double-blind, parallel, placebo-controlled phase II clinical study. It is planned to recruit 74 patients with acquired thrombotic thrombocytopenic purpura (TTP). To evaluate the efficacy and safety of Anfibatide as an adjuvant therapy for plasma exchange in patients with acquired TTP.
Detailed description
The study will consist of 3 periods: screening(up to 7 days), treatment and follow-up(up to 3 months). Subjects are assigned in a 1:1 ratio to Anfibatide or placebo during plasma exchange with the use of a computerized randomization schedule. The administration is 5 IU/60kg intravenous infusion and 0.002 IU/kg/h continuous intravenous infusion. Plasma exchange as the main treatment method is performed daily on the 5th day of solstice and then adjusted to every other day. Fresh frozen plasma（FFP）and cryoprecipitate-reduced plasma are preferred. Subjects will receive the study drug immediately after daily plasma exchange.
Interventions
DRUGAnfibatide
5 IU/60kg intravenous infusion follow by 0.002 IU/kg/h continuous intravenous infusion
DRUGPlacebos
5 IU/60kg intravenous infusion follow by 0.002 IU/kg/h continuous intravenous infusion
Sponsors
Lee's Pharmaceutical Limited
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Female and male subjects with 18 years of age or older. 2. Subjects with diagnosis of TTP. 3. Necessitating plasma exchange. 4. Obtained, signed and dated informed consent.
Exclusion criteria
1. Platelet count greater or equal to 100\*10\^9/μL. 2. Severe heart, liver and kidney dysfunction, including those with glutamic-pyruvic transaminase ≥ 5xULN，glomerular filtration rate \<30ml/min. 3. Uncontrolled severe active infection. 4. Known congenital TTP. 5. Subjects with malignant tumors in the past 5 years. 6. Other diseases that cause microangiopathy hemolytic anemia and thrombocytopenia, such as disseminated intravascular coagulation (DIC), antiphospholipid antibody syndrome, hemolytic uremic syndrome, malignant hypertension, and transplant-related microangiopathy. 7. Pregnant or lactating women. Subjects of reproductive age, are unable to use effective contraceptive methods during the study period. 8. Severe active bleeding or progressive aggravation of bleeding symptoms. 9. Subjects who have received plasmapheresis during the treatment of the onset of the disease. 10. Subject is participating in other clinical stuy or is less than 3 months away from the end of previous clinical study. 11. Subject who have participated in other clinical trials related to Anfibatide. 12. Severe or life threatening clinical condition other than TTP that would impair participation in the trial. 13. Life expectation less than 1 week. 14. Known to be allergic to the drugs or ingredients in the study. 15. Inability to follow programme requirements and procedures. 16. Subjects who are not eligible to participate in this clinical study as determined by the investigator.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Time to response of treatment | up to 21days | Time to response of treatment ,defined by a recovery of platelets ≥100\*10\^9/l |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Remission rate | up to 21days | Number of subjects with remission |
Other
| Measure | Time frame | Description |
|---|---|---|
| Number of plasma exchange | up to 21 days | The number of plasma exchange to achieved remission. |
| Volume of plasma | up to 21 days | The volume of plasma to achieved remission. |
| Number of subjects relapsing of TTP for maximum of 3 months and time to first relapse of TTP. | up to 3 months | Relapse is defined as novo event of TTP that occurs later than 30 days after the last plasma exchange. |
| Total mortality | up to 21days and the ensuing 30 days | Total mortality within the plasma exchange treatment period and the ensuing 30 days. |
| Number of exacerbations of TTP and time to first exacerbation of TTP. | up to 1 month | Exacerbation is defined as recurrent thrombocytopenia following a response and requiring a reinitiation of daily plasma exchange treatment after ≥ 1 day but ≤ 30 days of no plasma exchange treatment. |
| Time to achieve threshold values of biological markers. | up to 3 months | The biological markers include platelets，LDH and creatinine. |
| Complete remission rate | up to 21 days | Number of subjects with complete remission |
Countries
China
Contacts
Primary ContactWeili Zhao, MD
Outcome results
None listed