Recruiting
Phase 1
A Phase 1 Study of ATORM-C in Patients With Crohn's Disease
Condition(s) studied
Crohn's DiseasesCrohn's Disease
Investigational drug(s) / intervention(s)
Autologous Adult Stem Cell-Derived Intestinal Organoid
Autologous Adult Stem Cell-Derived Intestinal Organoid: ATORM-C is an investigational product consisting of autologous adult stem cell-derived intestinal organoids. Participants will receive a single administration of ATORM-C applied directly to the target intestinal ulcer during colonoscopy. ATORM-C is administered with fibrin glue (Greenplast-Q) to facilitate local application to the target ulcer.
Study summary
This Phase 1 study evaluates the safety and tolerability of ATORM-C in patients with Crohn's disease. ATORM-C is an investigational product consisting of autologous adult stem cell-derived intestinal organoids. Participants will receive a single dose of ATORM-C applied directly to a target intestinal ulcer during colonoscopy. The study will also explore the effect of ATORM-C on intestinal ulcer healing.
Eligibility
Inclusion Criteria:
1. Aged 19 years or older and younger than 80 years at the time of screening (male or female).
2. Diagnosed with Crohn's disease at least 6 months prior to screening, confirmed by imaging, tissue sampling (biopsy), and/or colonoscopy.
3. Have had an inadequate response to at least 6 months of standard treatment for Crohn's disease (such as anti-inflammatory drugs, oral corticosteroids, immunosuppressants, or biologic agents). Inadequate response means failure to achieve ulcer-free endoscopy, defined as an SES-CD Ulcerated Surface Score of 0 or 1.
4. Have active daily symptoms at screening and right before treatment, defined as either an average of 4 or more loose/liquid stools per day or an average daily abdominal pain score of 2 or more, based on the corresponding CDAI components.
5. Meet all of the following colonoscopy findings at screening:
* Have at least one active ulcer in an accessible area of the intestine (such as the colon or ileocecal region).
* Have at least one ulcer with a longest diameter of 5 mm or larger.
* Have an active ulcer segment with an endoscopic severity score (SES-CD) of 6 or higher, with an ulcer size score of 2 or higher and no narrowing or blockage (stricture score of 0) in that area.
6. Meet all required blood test standards for cell therapy donor eligibility.
7. Voluntarily agree to participate and sign the written informed consent form after receiving a full explanation of the study.
Exclusion Criteria:
1. Other Inflammatory Bowel Conditions: Diagnosed with ulcerative colitis or unclassified colitis.
2. Surgical Needs: Require, or are expected to require, surgery for Crohn's disease or its complications.
3. Severe Co-existing Conditions: Have a severe autoimmune disease other than Crohn's disease, or an active abdominal/perianal condition (such as an undrained abscess or active fistula).
4. Recent Surgeries: Had bowel resection within 6 months or intra-abdominal surgery within 3 months prior to screening.
5. Procedure Inability: Unable to safely undergo bowel preparation, colonoscopy, or sedation, or have a known allergy/contraindication to medications used during these procedures.
6. Uncontrolled Medical Issues: Have a clinically significant or poorly controlled medical condition, such as severe heart, bleeding, kidney, or liver disease, or uncontrolled diabetes.
7. Active Severe Infection: Have an active, severe infection requiring ongoing medical treatment.
8. Cancer History: Have a history of cancer (malignancy) within 5 years before screening, except for specific low-risk cancers permitted by the protocol.
9. Allergies to Study Treatment: Have a known severe allergy (hypersensitivity) to ATORM-C, its ingredients, or materials used in its manufacturing.
10. Prohibited Therapies or Transplants: Are taking prohibited medications, have previously received any cell therapy product, or have received an organ transplant (except corneal transplant).
11. Significant Blood Test Abnormalities: Have severe laboratory test abnormalities, including liver function (AST or ALT ≥3 times the upper limit of normal), kidney function (eGFR \<30 mL/min/1.73 m²), bleeding markers (PT INR \>1.5), or low blood platelets (\<50,000/mm³).
12. Pregnancy, Breastfeeding, or Contraception: Are pregnant, breastfeeding, or unwilling to follow the protocol-specified contraception requirements during the study.
13. Recent Clinical Trials: Received another investigational drug or device within 4 weeks prior to screening.
14. Investigator Judgment: Deemed unsuitable for participation by the study investigator for any other safety reason.
Primary outcome measure(s)
- Dose-Limiting Toxicities (DLTs) — From ATORM-C administration through 4 weeks post-dose
Occurrence of dose-limiting toxicities (DLTs). A DLT is defined as any Grade 3 or higher adverse reaction according to NCI CTCAE v5.0 that is assessed as possibly, probably, or certainly related to ATORM-C. CTCAE grades range from Grade 1 (mild) to Grade 5 (death related to an adverse event), with higher grades indicating greater severity.
- Determination of Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D) — From ATORM-C administration through 24 weeks post-dose
The MTD and RP2D of ATORM-C will be determined based on the occurrence of dose-limiting toxicities (DLTs) during the dose-escalation period and the overall safety and tolerability of ATORM-C.
Trial sites (1)
| Facility | City | Region | Status |
| Asan Medical Center |
Seoul |
South Korea |
Recruiting |
Other trials for the same condition