Description Module
The Description Module contains narrative descriptions of the clinical trial, including a brief summary and detailed description. These descriptions provide important information about the study's purpose, methodology, and key details in language accessible to both researchers and the general public.
Description Module path is as follows:
Study -> Protocol Section -> Description Module
Description Module
Ignite Creation Date:
2025-12-24 @ 11:04 PM
Ignite Modification Date:
2025-12-24 @ 11:04 PM
NCT ID:
NCT01916369
Brief Summary:
The primary objective of the Phase I ascending dose trial is to investigate the safety and tolerability of intramuscular (gastrocnemius) injections of human neural stem cell product, CTX, in patients with peripheral arterial disease (Fontaine Stage II through IV). This trial is based on independent preclinical data from a leading academic research institution that has been submitted for publication. Inclusion of patients with Fontaine Stage II is justified as these patients have a lower incidence of background events and will facilitate distinction between events which are possibly intervention-related versus spontaneous events associated with underlying advanced atherosclerosis. The trial is designed to treat 9-18 patients and evaluate safety measures over a 12 month follow-up period.
Detailed Description:
Design: The trial is a multi-centre open label, non-comparative, ascending dose safety study, using CTX cells to treat patients with lower limb ischaemia, with follow-up over 12 months.
Pre-treatment selection of patients: Men and women, aged \>50 years with peripheral arterial occlusive disease (Fontaine Stage II through IV) of one leg or both, unsuitable for surgical re-vascularisation, an ankle/brachial pressure index (ABPI) of \<0.9 or a toe/brachial index of \<0.7. Women must be surgically sterile or more than 2 years past their last menstrual period and test negative for pregnancy.
Treatment: One patient will be treated at one time with a single dose of CTX cells. Three ascending doses (20, 50 or 80 million cells), allocated sequentially, will be tested in 9-18 patients (3 dose cohorts of 3-6 patients. All trial patients will receive 10 intramuscular injections of CTX DP into the gastrocnemius muscle of their ischaemic leg on a single occasion. In the event that both legs have peripheral arterial disease, the injections will be administered to the leg determined by the Investigator to have the more advanced/severe disease provided that leg has not already had surgical amputation of toes or above.
A minimum interval of 28 days will be observed between dosing the first and second patient and 7 days between dosing subsequent patients in a given cohort in order to assess the safety and tolerability of CTX DP at that dose. A further 3 patients may be added to any cohort (to a maximum of 18 of total treated patients) at the discretion of the Data Safety Monitoring Board (DSMB) if required to further clarify the safety profile). A minimum interval of 28 days will be observed between dosing the first and second patient and 7 days between dosing subsequent patients in a given cohort in order to assess the safety and tolerability of CTX DP at that dose.
Post-treatment follow-up: There will be 8 scheduled visits to the clinic for monitoring over the 12 month follow-up period.
End-points: The primary endpoint of the trial is safety, measured by numbers of relevant adverse events, health screening, physical examination (overall and of the treated limb), immunological response, amputation and concomitant medications in the first year after treatment.
Post-trial follow-up: Life-long annual follow-up to record any new cancer via national cancer registry or similar system or by Investigator or family practitioner (as permitted by competent authorities, ethics committees and informed consent).
Study:
NCT01916369
Study Brief:
Protocol Section:
NCT01916369