THERAPY DOSSIER · th-d1ce8f6e883b

Recombinant ADAMTS-13 (rADAMTS-13); lactadherin as comparator intervention

Biologic · A disintegrin and metalloproteinase with thrombospondin motifs 13 exogenous protein

Review required. This record is flagged as borderline.

Evidence & regulatory context

Mechanism: A disintegrin and metalloproteinase with thrombospondin motifs 13 exogenous protein
Target: CHEMBL2346492
Injury models: TBI
Reported indication: Apadamtase alfa (Adzynma) - recombinant ADAMTS13 enzyme-replacement for prophylactic or on-demand treatment of congenital thrombotic thrombocytopenic purpura (cTTP); FDA-approved Nov 2023, Japan 2024
First approval: 2023
Development stage: Approved for congenital TTP; acute ischaemic stroke / acquired TTP still investigational
ChEMBL record: Not recorded
ChEMBL max phase: 4 / 4 (development scale, not regulatory approval)
Synonyms: rADAMTS13; Recombinant human ADAMTS13; Adzynma; BAX 930; SHP655; TAK-755; Apadamtase alfa
Note: Not recorded

Clinical trials 9

0 linked trials carry an injury-indication flag. These flags support record-level exploration and sit alongside the stringent aggregate counts described in Methods.

NCT04683003 ↗PHASE3active not recruiting

Phase 3b continuation study of TAK-755 (rADAMTS13) for prophylactic and on-demand treatment of severe congenital TTP

Thrombotic Thrombocytopenic Purpura

Enrolment: 77 · Results not recorded

NCT07392450 ↗PHASE2recruiting

Phase 2 randomized placebo-controlled trial of TAK-755 in acute ischemic stroke

Acute Ischemic Stroke

Enrolment: 222 · Results not recorded

NCT05714969 ↗PHASE2completed

Phase 2b study of TAK-755 (rADAMTS13) with minimal to no plasma exchange in immune-mediated TTP

Thrombotic Thrombocytopenic Purpura

Enrolment: 29 · Results not recorded

NCT03922308 ↗PHASE2completed

Phase 2 study of rADAMTS-13 (SHP655) added to standard of care in acquired TTP

Acquired Thrombotic Thrombocytopenic Purpura

Enrolment: 28 · Results not recorded

NCT03997760 ↗PHASE1completed

Phase 1 ascending dose safety and PK/PD study of SHP655 (rADAMTS13) in sickle cell disease

Sickle Cell Disease

Enrolment: 19 · Results not recorded

NCT02216084 ↗PHASE1completed

BAX930 (rADAMTS13): phase 1 dose-escalation safety and PK study in hereditary TTP

Hereditary Thrombotic Thrombocytopenic Purpura

Enrolment: 16 · Results not recorded

NCT05770219 ↗NAapproved for marketing

Expanded access programme: TAK-755 (rADAMTS13) for severe congenital or hereditary TTP

Thrombotic Thrombocytopenic Purpura

Enrolment not recorded · Results not recorded

NCT06441578 ↗NArecruiting

Special drug use surveillance of ADZYNMA intravenous 1500 (all-case investigation)

Thrombotic Thrombocytopenic Purpura

Enrolment: 40 · Results not recorded

NCT07353099 ↗NArecruiting

Post-authorization safety study of Adzynma in congenital TTP

Thrombotic Thrombocytopenic Purpura

Enrolment: 50 · Results not recorded

Preclinical evidence 1

1 studies reported at therapy level; 1 evidence entries itemised.

PMID 29941674 ↗2018Reported benefit

von Willebrand factor enhances microvesicle-induced vascular leakage and coagulopathy in mice with traumatic brain injury.

MouseTBIFluid percussionMaleGiven either before or after traumatic brain injury

Model: Fluid percussion traumatic brain injury

Recombinant ADAMTS-13 given before or after injury reduced VWF reactivity with minimal effect on VWF secretion, protected endothelial barrier integrity and prevented injury-induced coagulopathy by enhancing VWF cleavage without impairing basal haemostasis. Promoting microvesicle clearance with lact…

Extraction: extract · Subtype source: rule · Supplied extraction confidence: 0.55. Machine-extracted fields require source verification.

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