For International Sponsors – Powered by GCP ClinPlus

1. What is a CGT IIT in China?

In China, CGT IITs are investigator-initiated trials typically conducted at leading hospitals. They are often used for early-stage exploration of new indications or treatment regimens using stem cells, CAR-T, or other CGT products. These trials fall under the regulatory purview of the National Health Commission (NHC) and local ethics committees, not the NMPA (drug authority), although data can later support NMPA IND filings.

2. Are CGT IITs allowed for unapproved products?

Yes. While most IITs in China focus on marketed drugs, CGT IITs are an exception due to unmet medical needs and urgent clinical demand. This includes:

· CAR-T for relapsed/refractory cancers

· Stem cells for degenerative or autoimmune diseases

These are governed by special regulations such as the Stem Cell Clinical Research Guidelines (Trial) and Technical Guidance for Immune Cell Therapy Clinical Trials.

3. What are the benefits of running CGT IITs in China?

· Faster early-phase clinical data from real-world settings

· Lower costs compared to global Phase I/II studies

· Opportunity to generate pre-IND safety/efficacy data

· Access to large patient populations for rare or advanced diseases

· Potential to shorten time to registration by 1.5 years

4. What is the regulatory and ethics review process?

Key steps include:

· Submit proposal via hospital CTMS

· Scientific & ethical review

· Approval by hospital’s IIT office

· MOH/NHC registration in the Medical Research Registry System

· Human genetic resources filing (if applicable)

· ClinicalTrials.gov registration

5. Are there restrictions on who can run CGT IITs?

Only Tier 1 hospitals with specific credentials may host CGT IITs:

· Must hold NHC certification for stem cell/immune cell therapy research.

· Require on-site GMP-compliant cell processing facilities or contracted NMPA-licensed CDMOs.

· Must demonstrate expertise in managing CGT-related risks (e.g., CRS, neurotoxicity).

Trials must have:

· Institutional certification

· Clear patient risk controls

· Pre-defined stopping rules for severe adverse events

6. Can CGT IIT data support later IND or registration studies?

Yes. IIT data—especially long-term safety and early efficacy signals—can be used as:

· Supporting evidence for IND submission

· Design reference for pivotal trials

· Bridging data for conditional approvals

7. Do I need a local sponsor or biotech partner?

Not necessarily. Foreign sponsors are no longer required to partner with a local biotech. However:

· Using a local CDMO simplifies approvals

· Working with an experienced CRO like GCP ClinPlus ensures quality, compliance, and site engagement

8. Why choose GCP ClinPlus as your CRO partner in China?

· 22+ years’ experience, with 50+ CGT trials completed

· Proven track record across CAR-T, stem cells, oncolytic virus, and gene therapy

· Executed 19 IITs in China in diseases including glioblastoma, MM, NSCLC, HCC, β-thalassemia, ALS, and more

· Deep relationships with top CGT investigators and elite hospitals

· Full-service capabilities from protocol development to site management and data submission

9. What are the future trends in CGT IIT in China?

· More real-world data integration into regulatory filings

· Regulatory upgrades to differentiate gene-editing vs. non-editing therapies

· Increasing emphasis on ethics and long-term safety

· IITs as a strategic path for market entry

10. How is intellectual property (IP) managed in IITs?

· Sponsor-owned IP: Default if sponsor funds the trial (defined in the hospital contract).

· Joint IP: Possible for investigator-initiated discoveries; rights negotiated upfront.

· Publication rights: Hospitals typically require manuscript review

10. How do I get started?

Contact GCP ClinPlus for a feasibility consultation, hospital connection, or local regulatory assessment. We are ready to help international sponsors conduct high-quality, compliant, and efficient CGT IITs in China.

�� Email: suling.zhang@gcp-clinplus.com �� Website: http://en.gcp-clinplus.com �� Locations: China (Beijing, Shanghai, Guangzhou & more), US