干细胞治疗临床试验的全球趋势：2007–2016十年回顾（英文版）_23页

Summary of Global Trends in Stem Cell Therapy Clinical Trials (2007-2016)

Core Content

This report provides a 10-year retrospective analysis of global stem cell therapy clinical trials initiated between 2007 and 2016. It highlights key trends in the number of trials, their phases, sponsor types, therapy areas, indications, locations, and the leading sponsors and interventions involved.

Main Features of the Report

Total Number of Trials

• A total of 584 stem cell therapy clinical trials were analyzed.
• The number of trials increased steadily from 2007 to 2012, peaking at 70 trials in 2012.
• A slight decrease occurred in 2013 (67 trials), followed by a continuous rise to 74 trials in 2015.
• In 2016, the number of trials slightly dropped to 69.

Trial Phases

• Phase I trials increased significantly over the 10 years, reaching a 10-year peak of 38% in 2016.
• Phase II trials had the highest contribution in 2012 and 2013, at 71% and 75%, respectively, but declined to 48% in 2016.
• Phase III trials peaked at 32% in 2007, then declined to 6% in 2010, with fluctuations thereafter.
• Phase IV trials were minimal, with only 3% in 2016 and no trials registered between 2007 and 2010.

Sponsor Types

• Industry sponsors dominated the majority of trials, with 53 trials in 2014 being the highest number.
• Non-industry sponsors saw an increase from 13 to 29 trials between 2007 and 2012.
• Mesoblast was the top industry sponsor.
• Nanfang Hospital was the top non-industry sponsor.

Therapy Areas

• Oncology had the largest number of trials (269), followed by Cardiovascular (245), Central Nervous System (137), Musculoskeletal Disorders (88), and Immunology (87).
• Oncology, Musculoskeletal Disorders, Cardiovascular, and Central Nervous System areas featured more industry-sponsored trials than non-industry ones.
• Immunology had a higher proportion of non-industry-sponsored trials.

Top Indications

• The top indications were myocardial infarction, graft versus host disease, congestive heart failure, critical limb ischemia, and osteoarthritis.
• Industry sponsors were more prominent than non-industry sponsors for all indications except graft versus host disease, where both were approximately equal.
• Osteoarthritis had the highest ratio of industry to non-industry trials (11.5 times).

Trial Status

• 26% of the trials reported results.
• 88.2% of trials achieved primary endpoints, 5.9% did not achieve them, 3.3% were inconclusive, and 2.6% were partially achieved.
• Completed, planned, not recruiting, and S/T/W (suspended, terminated, withdrawn) trials were mainly sponsored by industry sponsors.
• Recruiting and recruiting by invitation trials were more common among non-industry sponsors.

Trial Locations

• Single-country trials were 7.1 times more common than multinational trials.
• The US hosted the most trials with 180, followed by South Korea (63) and China (55).
• Multinational trials peaked in 2014 (12 trials) and reached a low in 2009 (2 trials).

Trial Size

• The average number of subjects in patient trials decreased by 59%, from 92 to 38.
• The average number of healthy participants increased by 429%, from 17 to 90.
• 2015 had the highest number of average healthy participants, due to a large multinational trial by Mesoblast with Quintiles.
• Single-country trials saw a significant decrease in locations from 2015 to 2016, with the average number of locations dropping to 2.8 in 2016.

Top Interventions

• The top drugs investigated in stem cell therapy clinical trials were:
  1. Remestemcel-L (13 trials)
  2. Stempeucel (12 trials)
  3. MA09-Hrpe, HuCNS-SC, and ALLO-ASC (each with 11 trials)
  4. PLX-PAD, NSI-566, PF-05285401, Cenplacecl-L, and AdipoCell (each with 10 trials)
• Phase II trials were the most common across all drugs.
• Phase IV trials were absent for all drugs until 2016, when myocardial infarction had one.

Key Sponsors and CROs

• Mesoblast was the leading industry sponsor.
• Nanfang Hospital was the leading non-industry sponsor.
• Reliance Life Sciences was the most common CRO used, with 5.8% of trials involving its services.

Key Organizations Mentioned

• Aderans Research Institute
• Anterogen
• Celgene
• City of Hope
• Great Ormond Street Hospital
• MediPost
• Mesoblast
• Microbot Medical
• Nanfang Hospital
• Neuralstem
• Pluristem Therapeutics
• Samsung Medical Center
• Stempeutics Research
• TiGenix
• University of California and Los Angeles
• Universiti of Kebangsaan Malaysia
• University of Miami
• University of Texas
• US Stem Cell

Country Distribution

• The US was the most active country for stem cell therapy trials with 180 trials.
• South Korea and China followed with 63 and 55 trials, respectively.
• Other notable countries included India, Spain, UK, Australia, Germany, Japan, and Iran.

Conclusion

Over the 10-year period (2007-2016), stem cell therapy clinical trials showed a steady increase, with Phase I and Phase II trials being the most common. Industry sponsors dominated the landscape, with Mesoblast leading the pack. Oncology was the largest therapy area, while myocardial infarction and osteoarthritis were the most frequently studied indications. The US hosted the most trials, and single-country trials were more prevalent than multinational ones. The average size of trials decreased for patient studies but increased for healthy participant studies, especially in 2015. CROs played a supporting role, with Reliance Life Sciences being the most frequently used.