NCT02763644

Efficacy and Safety of LFG316 in Transplant Associated Microangiopathy (TAM) Patients

A Randomized, Open Label, Controlled, Multiple Dose Study to Evaluate the Clinical Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of LFG316 in Patients With Transplant Associated Microangiopathy After Hematopoietic Precursor Cell Transplantation

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 7 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This was a randomized, SoC-controlled, open-label, multi-center study in patients with TAM after hematopoietic precursor cell transplantation (HPCT) . Study consisted of up to 28 days of screening period, 16 weeks treatment period that can be extended to 45 weeks.Approximately 40 patients was to be randomized to receive SoC or LFG316 plus SoC. Patients was included in the study if they have diagnosis of TAM and poor prognostic markers. This trial was terminated: LFG316, a monoclonal antibody inhibitor of complement factor 5 (C5), had been studied in seven patients with transplantation-associated microangiopathy (TAM). Due to low confidence of clinical benefit, this study was closed

Detailed description

This was a randomized, SoC-controlled, open-label, multi-center study in patients with TAM after hematopoietic precursor cell transplantation (HPCT). Study consisted of up to 28 days of screening period, 16 weeks treatment period that can be extended to 45 weeks, 36 weeks follow up, and end of study visit (EOS) at week 52. Duration of follow up depended on duration of treatment. Patients who are treated for more than 41 weeks will proceed directly to EOS visit. Approximately 40 patients was to be randomized to receive SoC or LFG316 plus SoC. Patients was included in the study if they have diagnosis of TAM and poor prognostic markers. Patients showing worsening of disease after two weeks of treatment or showing no response at week 4 or any time after will be considered failures and can be switched to receive the alternative treatment (SoC or LFG316). Patients can only switch treatment arms once.

Conditions

• Transplant Associated Microangiopathy TAM

Interventions

Type	Name	Description
DRUG	LFG316 active drug	LFG316
OTHER	Standard of care treatment	SoC (site specific)

Timeline

• Start date: 2016-04-22
• Primary completion: 2017-02-10
• Completion: 2017-06-30
• First posted: 2016-05-05
• Last updated: 2021-01-05
• Results posted: 2019-04-17

Locations

6 sites across 3 countries: United States, France, Germany

Regulatory

• FDA-regulated drug study