NCT01663116

Cx611-0101, eASCs Intravenous Administration to Refractory Rheumatoid Arthritis Patients

"Phase Ib/IIa, Escalating Dose, Single Blind, Clinical Trial to Assess the Safety of the i.v Administration of Allogeneic Adipose-derived Mesenchymal Cells (eASCs) to Refractory Rheumatoid Arthritis (RA) Patients".

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 53 (actual)
• Sponsor: Tigenix S.A.U. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

Phase Ib/IIa clinical trial of a new medicinal product of the somatic cell therapy class (eASCs). This study is designed as a multicenter, single blind, fixed dose escalation, with three treatment groups, controlled with placebo (randomization 3:1) whose target population are patients with rheumatoid arthritis refractory to at least two biologic.

Detailed description

Traditionally, RA has been treated with non-steroidal anti-inflammatory drugs, glucocorticoids and non-biologics-DMARDs. Only non-biologics-DMARDs and, at a lesser extent, glucocorticoids have shown to be able to prevent or interrupt the inflammatory and destructive disease processes. Mesenchymal stem cells (MSCs) are nonhematopoietic stromal cells that are able to differentiate into mesenchymal tissues such as bone, cartilage, muscle, ligament, tendon, and adipose. MSCs can be easily isolated from bone marrow or adipose tissue and rapidly expanded in culture. MSCs have also been shown to have immuno-suppressive and healing capacities, improve angiogenesis and prevent fibrosis. These properties could be used for novel therapeutic applications in various disorders, including rheumatoid arthritis, osteoarthritis (OA), genetic bone and cartilage disorders and bone metastasis. MSCs can potently modulate immune responses, showing antiproliferative and anti-inflammatory capacities. This study is a multicenter phase Ib/IIa, escalating dose, single blind clinical trial to assess the safety of the intravenous administration of expanded allogeneic adipose-derived mesenchymal stem cells (eASCs) to refractory rheumatoid arthritis (RA) patients. The primary objective of the study is to determine the safety, feasibility and tolerance, and to identify, if possible, the dose limiting toxicity (DLT) and the dose for future clinical trials on efficacy of the intravenous infusion of allogeneic eASCs for patients suffering rheumatoid arthritis (RA) under treatment with at least one non-biologic-Disease modifying antirheumatoid drug (DMARD) who have previously failed to treatment with at least two biologics. The secondary objective is to obtain information on the clinical and functional effects of the intravenous infusion of allogeneic eASCs in patients with RA and to explore pharmacodynamics parameters. 53 patients (i.e. patients having received at least one dose of study treatment) in three different cohorts are planned to be included in this clinical trial. Expansion will start after acute toxicity assessment of the first three patients of each cohort. Dose and intervals for the trial consist of the following active groups: a) first cohort: 1 million cells/kg administered at days 1, 8 and 15; b) second cohort: 2 million cells / kg administered at days 1, 8 and 15; c) third cohort: 4 million cells / kg administered at days 1, 8 and 15.

Conditions

• Rheumatoid Arthritis Aggravated

Interventions

Type	Name	Description
GENETIC	Stem cells	1. first cohort: 1 million stem cells/kg administered at days 1, 8 and 15 2. second cohort: 2 million stem cells / kg administered at days 1, 8 and 15 3. third cohort: 4 million stem cells / kg administered at days 1, 8 and 15
GENETIC	Placebo	1. first cohort: 20 ml administered at days 1, 8 and 15 2. second cohort: 40 ml administered at days 1, 8 and 15 3. third cohort: 80 ml administered at days 1, 8 and 15

Timeline

• Start date: 2011-03-01
• Primary completion: 2013-01-01
• Completion: 2013-01-01
• First posted: 2012-08-13
• Last updated: 2019-04-12

Locations

23 sites across 1 country: Spain