A Study of TAK-755 (rADAMTS13) With Little to No Plasma Exchange (PEX) Treatment in Adults With Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP)
- Conditions
- Thrombotic Thrombocytopenic Purpura (TTP)
- Interventions
- Biological: TAK-755
- Registration Number
- NCT05714969
- Lead Sponsor
- Takeda
- Brief Summary
This is a study of TAK-755 in adults with immune-mediated thrombotic thrombocytopenic purpura (iTTP). The main aim of this study is to determine the percentage of participants with a clinical response without plasma exchange during the study. Participants who have an acute attack of iTTP will receive TAK-755 and immunosuppressive therapy during their stay at the hospital until they achieve a clinical response. Participants will also be treated with TAK-755 for an additional time of up to 6 weeks after the acute phase. In total, participants will stay in the study for approximately 3 months.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 40
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description TAK-755 Dose 2 in Both Acute and Post-Acute Phase TAK-755 TAK-755 Dose 2, IV infusion, in the acute phase until clinical response is achieved. All participants achieving clinical response will receive TAK-755 at Dose 2, for up to 6 weeks during the post-acute phase. TAK-755 Dose 1 in Acute Phase and Dose 2 in Post-acute Phase TAK-755 TAK-755 Dose 1, IV infusion, in the acute phase until clinical response is achieved. All participants achieving clinical response will receive TAK-755 at Dose 2, for up to 6 weeks during the post-acute phase.
- Primary Outcome Measures
Name Time Method Incidence of Adverse Events (AEs), Serious Adverse Events (SAEs), and Adverse Event of Special Interest (AESIs) After Receiving any Dose of Investigational Product (IP) Through study completion, approximately 12 weeks An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (e.g., a clinically significant abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, whether or not it is considered related to the drug. SAE: Signs, symptoms or outcomes which results in death, requires inpatient hospitalization or prolongation of hospitalization, results in persistent or significant disability/incapacity, results in a congenital abnormality/birth defect, or is an important medical event. Adverse events of special interest include thromboembolic events and treatment-related bleeding events.
- Secondary Outcome Measures
Name Time Method Occurrence of Refractoriness (Acute Phase) Through study completion, approximately 12 weeks Occurrence of Treatment Failure Through study completion, approximately 12 weeks Treatment failure is defined as failure to achieve or maintain clinical response, including refractory iTTP and recurrent iTTP.
Time to Occurrence of Any One of the Following Events: Clinical Recurrence, iTTP-Related Death, or Major Thromboembolic Event From Time of First IP Administration Through Study Completion Through study completion, approximately 12 weeks ADAMTS13 Activity Level Resulting From TAK-755 Administration in Acute and Post-Acute Phases Through study completion, approximately 12 weeks Von Willebrand Factor (VWF) Antigen Level Resulting From TAK-755 Administration in Acute and Post-Acute Phases Through study completion, approximately 12 weeks Achievement of Clinical Response Without On-Study Plasma Exchange (PEX) Through study completion, approximately 12 weeks Clinical response is defined as normalization of platelets and no clinical evidence of new or progressive ischemic organ injury. Normalization of platelets: First occurrence of normal platelet count (≥150,000/microliter \[μL\]) that is followed by a confirmatory platelet count of ≥150,000/μL and a lactate dehydrogenase (LDH) \<1.5×upper limit of normal (ULN) at 48±12 hours after the first occurrence.
Occurrence of Immune-Mediated Thrombotic Thrombocytopenic Purpura (iTTP) Recurrence, Exacerbation, or Relapse (Post-acute Phase) Through study completion, approximately 12 weeks iTTP recurrence comprised of exacerbation or relapse. Clinical exacerbation: Occurs \<30 days after achieving initial clinical response (i.e., before clinical remission) and recurrent thrombocytopenia (platelet levels \<150,000/μL), with or without clinical evidence of new or progressive ischemic organ damage, requiring daily PEX or rescue therapy. Clinical relapses: Occurs ≥30 days after achieving initial clinical response (i.e., after clinical remission) and recurrent thrombocytopenia (platelet levels \<150,000/μL), with or without clinical evidence of new or progressive ischemic organ damage, requiring daily PEX or rescue therapy.
Change From Baseline in Troponin Levels at Clinical Response and Study Completion Through study completion, approximately 12 weeks Achievement of Clinical Remission Through study completion, approximately 12 weeks Clinical remission is defined as achieving and maintaining clinical response for ≥30 days.
VWF Activity Level Resulting From TAK-755 Administration in Acute and Post-Acute Phases Through study completion, approximately 12 weeks Achievement of Clinical Response With Zero or Minimal on-Study PEX Through study completion, approximately 12 weeks The number of PEX administered considered Zero when no PEX is administered and considered Minimal when 1 to 3 PEX are administered.
Achievement of Clinical Response Overall Through study completion, approximately 12 weeks Overall indicates clinical response regardless of whether on-study PEX is administered, or the number of PEX administered.
Time to Clinical Response (Acute Phase) Through study completion, approximately 12 weeks Total Volume of Plasma Administered (Acute Phase) Through study completion, approximately 12 weeks Occurrence of any one of the following events: Clinical Recurrence, iTTP-Related Death, or Major Thromboembolic Event From Time of First IP Administration Through Study Completion Through study completion, approximately 12 weeks Change From Baseline in Lactate Dehydrogenase [LDH] Levels at Clinical Response and Study Completion Through study completion, approximately 12 weeks Time to First On-Study PEX to Achieve Clinical Response Through study completion, approximately 12 weeks Number of Days of On-study PEX in Participants Who Achieved Clinical Response (Acute Phase) Through study completion, approximately 12 weeks Time to iTTP Recurrence, Exacerbation, or Relapse Through study completion, approximately 12 weeks A Disintegrin and Metalloproteinase With Thrombospondin Motifs 13 (ADAMTS13) Antigen Level Resulting From TAK-755 Administration in Acute and Post-Acute Phases Through study completion, approximately 12 weeks
Trial Locations
- Locations (28)
University of Florida Shands
🇺🇸Gainesville, Florida, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
Beth Israel Deaconess Medical Center
🇺🇸Boston, Massachusetts, United States
Brigham and Women's Hospital
🇺🇸Boston, Massachusetts, United States
University if Minnesota Med CAR
🇺🇸Minneapolis, Minnesota, United States
Rutgers University
🇺🇸New Brunswick, New Jersey, United States
Weill Cornell Medical College New York Presbyterian Hospital
🇺🇸New York, New York, United States
University of Minnesota
🇺🇸Minneapolis, Minnesota, United States
Duke University Medical Center
🇺🇸Durham, North Carolina, United States
Leo Jenkins Cancer Center/ECU School of Medicine
🇺🇸Greenville, North Carolina, United States
Ohio State University
🇺🇸Columbus, Ohio, United States
University of Utah
🇺🇸Salt Lake City, Utah, United States
Versiti Clinical Trials and Research Office
🇺🇸Milwaukee, Wisconsin, United States
Clinica Zabala
🇦🇷Buenos Aires, Argentina
Hospital Universitario Austral
🇦🇷Buenos Aires, Argentina
AKH- Medizinische Universitat Wien
🇦🇹Vienna, Austria
General Hospital Of Athens Laiko
🇬🇷Athens, Greece
University Hospital of Patra
🇬🇷Patra, Greece
General Hospital of Thessaloniki "G. Papanikolaou"
🇬🇷Thessaloniki, Greece
Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico
🇮🇹Milan, Italy
Azienda Ospedaliero-Universitaria Citta della Salute e della Scienza di Torino
🇮🇹Torino, Italy
Instytut Hematologii i Transfuzjologii
🇵🇱Warszawa, Poland
Hospital de Cruces
🇪🇸Barakaldo, Spain
Hospital General Universitario Gregorio Maranon
🇪🇸Madrid, Spain
Hospital Universitario Virgen del Rocio
🇪🇸Sevilla, Spain
Hospital Universitari i Politecnic La Fe
🇪🇸Valencia, Spain
Hospital Universitario Dr. Peset
🇪🇸Valencia, Spain
Royal Liverpool University Hospital
🇬🇧Liverpool, United Kingdom