Who is sponsoring NCT02216084?

NCT02216084 is sponsored by Baxalta now part of Shire.

What drugs are being tested in NCT02216084?

Interventions in NCT02216084: Recombinant ADAMTS13.

What condition does NCT02216084 study?

NCT02216084 studies Hereditary Thrombotic Thrombocytopenic Purpura (TTP).

Is NCT02216084 still recruiting?

NCT02216084 is currently completed. Last updated 5 May 2021.

Last reviewed 2021-05-05 · How we verify

NCT02216084

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Phase 1 Dose Escalation, Single Dose Study to Assess Safety and Pharmacokinetics of BAX930 in Hereditary Thrombotic Thrombocytopenic Purpura (TTP)

Completed Phase 1 Last updated 5 May 2021

What this trial tests

Phase 1 trial testing Recombinant ADAMTS13 in Hereditary Thrombotic Thrombocytopenic Purpura (TTP) in 16 participants. Completed in 22 February 2016.

Timeline

30 September 2014

Primary endpoint
22 February 2016

22 February 2016

Quick facts

Lead sponsor	Baxalta now part of Shire
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	16
Start date	30 September 2014
Primary completion	22 February 2016
Estimated completion	22 February 2016
Sites	11 locations across Japan, Austria, United Kingdom, Germany, Poland, Switzerland, United States

Drugs / interventions tested

Recombinant ADAMTS13 — full drug profile →

Conditions studied

Hereditary Thrombotic Thrombocytopenic Purpura (TTP) — all drugs for Hereditary Thrombotic Thrombocytopenic Purpura (TTP) →

Sponsor

Baxalta now part of Shire — full company profile →

Who can join

Adults 12 to 65, any sex, with Hereditary Thrombotic Thrombocytopenic Purpura (TTP). Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

The purpose of this Phase 1, prospective, uncontrolled, open-label, multicenter, dose-escalation study is to evaluate the safety, including immunogenicity, and pharmacokinetics of BAX930 (rADAMTS13) in a total of 14 evaluable subjects diagnosed with severe hereditary thrombotic thrombocytopenic purpura (TTP) (plasma ADAMTS13 activity \<6%) who are assigned to one of three dose cohorts.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

Mechanisms of Autoantibody-Induced Pathology.
Ludwig RJ, Vanhoorelbeke K, Leypoldt F, Kaya Z, et al · · 2017 · cited 331× · PMID 28620373 · DOI 10.3389/fimmu.2017.00603
Recombinant ADAMTS-13: first-in-human pharmacokinetics and safety in congenital thrombotic thrombocytopenic purpura.
Scully M, Knöbl P, Kentouche K, Rice L, et al · · 2017 · cited 178× · PMID 28912376 · DOI 10.1182/blood-2017-06-788026
Pathophysiology of thrombotic thrombocytopenic purpura.
Sadler JE. · · 2017 · cited 169× · PMID 28768626 · DOI 10.1182/blood-2017-04-636431
Recombinant ADAMTS13 reduces abnormally up-regulated von Willebrand factor in plasma from patients with severe COVID-19.
Turecek PL, Peck RC, Rangarajan S, Reilly-Stitt C, et al · · 2021 · cited 39× · PMID 33662796 · DOI 10.1016/j.thromres.2021.02.012
Thrombocytopenia-Associated Multiple Organ Failure and Acute Kidney Injury.
Nguyen TC, Cruz MA, Carcillo JA. · · 2015 · cited 35× · PMID 26410136 · DOI 10.1016/j.ccc.2015.06.004
Advances in Clinical and Basic Science of Coagulation: Illustrated abstracts of the 9th Chapel Hill Symposium on Hemostasis.
Bergmeier W, Antoniak S, Conway EM, Denis CV, et al · · 2018 · cited 5× · PMID 30046746 · DOI 10.1002/rth2.12095
The history of thrombotic thrombocytopenic purpura research: a narrative review.
Halkidis K, Lämmle B, Zheng XL. · · 2024 · cited 2× · PMID 39049905 · DOI 10.21037/aob-23-46
Quantitative Systems Pharmacology Modeling of Platelet Responses to Recombinant ADAMTS13 in Patients With Congenital Thrombotic Thrombocytopenic Purpura.
McBride C, Jiang J, Zhang Z, Tolsma J, et al · · 2025 · cited 1× · PMID 40614125 · DOI 10.1002/psp4.70063

Verify or expand the search:

Other trials of Recombinant ADAMTS13

Trials testing the same drug.

NCT06441578 — A Survey of Recombinant ADAMTS13 in Participants With Congenital Thrombotic Thrombocytopenic Purpura · recruiting

Other Baxalta now part of Shire trials

Trials by the same sponsor.

NCT04985682 — A Study of ADVATE in People With Hemophilia A in India · Phase 4 · completed
NCT04578535 — A Study to Assess the Tolerability, Safety, and Pharmacokinetics of Subcutaneous Immune Globulin Infusion 10% (Human) Wi · Phase 1 · completed
NCT04346108 — A Study of Immune Globulin Subcutaneous (Human), 20% Solution (IGSC, 20%) in Japanese Participants With Primary Immunode · Phase 3 · completed
NCT04158934 — A Long-term Study of ADYNOVI/ADYNOVATE in Participants With Haemophilia A · active not recruiting
NCT04394286 — A Phase 1/2 Study of SHP648, an Adeno-Associated Viral Vector for Gene Transfer in Hemophilia B Subjects · Phase 1, PHASE2 · terminated

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT02216084 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Baxalta now part of Shire
Last refreshed: 5 May 2021

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02216084.

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