Clinical Trials

Date: 2016-12-05

Type of information: Results

phase: 2

Announcement: results

Company: Reneuron (UK)

Product: CTX DP (human neural stem cell product)

Action mechanism: stem cell therapy.

Disease: acute stroke

Therapeutic area: Cerebrovascular diseases

Country: UK

Trial details: The primary aim of the PISCES II trial is to determine whether it is sufficiently likely that CTX DP treatment at a dose level of 20 million cells improves the recovery in the use of the paretic arm in acute stroke patients to justify a subsequent larger prospectively controlled study. This study will evaluate the safety and efficacy of intracerebral CTX DP at a dose level of 20 million cells in patients with paresis of an arm following an ischaemic middle cerebral artery (MCA) stroke. Eligible patients will have no useful function of the paretic arm a minimum of 28 days after the ischaemic stroke (a modified NIH Stroke Scale (NIHSS) Motor Arm Score of 2, 3 or 4 for the affected arm). (NCT02117635)

Latest news:

• • On December 5, 2016, ReNeuron announced positive data from the company's Phase II clinical trial (PISCES II) of its CTX cell therapy candidate for stroke disability. The study's primary endpoint was for two patients to reach a minimum two-point improvement in the grasping and lifting test, sub-test number 2, of the Action Research Arm Test ("ARAT"), at three months post-treatment. Three of the 21 patients achieved this at three, six or twelve months respectively after treatment and were within a group of four responders who also showed clinically relevant improvements on the total ARAT score of arm motor performance. Although the ARAT sub-test number 2 study endpoint was not met as some responses came later than the three-month target, the result is nonetheless highly encouraging.Strongly positive results were also seen in the other endpoints of the study, with seven patients (33%) showing a clinically relevant improvement on the Modified Rankin Scale (a measure of disability and dependence) and eight patients (38%) showing a clinically relevant improvement on the Barthel Index (a measure of performance in activities of daily living). In total, 15 out of 21 patients had a clinically significant response on at least one efficacy measure. Improvements in the ARAT scores, Modified Rankin Scale and Barthel Index were all sustained throughout the follow up period.
• All 21 patients in the study have completed three-month follow-up, with ten patients followed for six months and three for twelve months. Further data will continue to be collected until all patients have reached 12 months post treatment.
• As a result of the positive data from the PISCES II study, Reneuron intends to apply to the US and European regulatory authorities to commence a randomised, placebo-controlled, pivotal clinical trial in patients who are living with disability post-stroke. The PISCES II study also demonstrated that the CTX treatment was well tolerated. The most common adverse events were transitory and related to the surgical procedure, such as headache and nausea. Safety and efficacy data from the study will be presented at forthcoming stroke and rehabilitation medical conferences.
• • On June 13, 2016, ReNeuron provided an update on progress with its stroke programme. The company has completed patient recruitment in the Phase II clinical trial (PISCES II) of its CTX cell therapy candidate in patients with motor disability as a result of ischaemic stroke. The three-month follow-up data from this study are expected to be available in the fourth quarter of this year. ReNeuron has commenced formal interactions with regulatory authorities regarding plans for a randomised, controlled, pivotal Phase II/III clinical trial with CTX in stroke disability. In particular, representatives from the company recently attended a face-to-face pre-IND meeting with the FDA to discuss plans for conducting the study in the US. Reneuron is incorporating the advice received in this meeting into the design and planning of the pivotal study. Similar discussions are taking place with the equivalent European regulatory body, the European Medicines Agency (EMA). Subject to the results of the Phase II study, the company expects to file an application in the first quarter of 2017 to commence a Phase II/III clinical trial.

Is general: Yes