A Phase 1 Study of ATORM-C in Patients With Crohn's Disease

An Open, Sequential Dose-Escalation, Single-Center, Phase 1 Clinical Trial to Evaluate Tolerability, Safety, and Exploratory Efficacy of ATORM-C in Patients With Crohn's Disease

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.

Study Overview

• Status: Recruiting
• Conditions: Crohn's Disease; Crohn's Diseases
• Intervention / Treatment: Biological: Autologous Adult Stem Cell-Derived Intestinal Organoid

Detailed Description

This is an open-label, sequential dose-escalation Phase 1 study to evaluate the safety, tolerability, and exploratory efficacy of ATORM-C across three dose levels in patients with Crohn's disease. ATORM-C is manufactured by culturing and expanding autologous adult stem cell-derived intestinal organoids isolated from each participant's intestinal tissue. Participants will receive a single administration of ATORM-C directly applied to a target intestinal ulcer during colonoscopy. Primary evaluations will focus on safety and tolerability, with exploratory assessments of ATORM-C's effect on intestinal ulcer healing.

• Study Type: Interventional
• Enrollment (Estimated): 18
• Phase: Phase 1

Contacts and Locations

Study Locations

• South Korea
  • Seoul, South Korea, 05505
    • Recruiting
    • Asan Medical Center
    • Contact: Byong Duk Ye, MD; Phone Number: +82-70-4105-0960; Email: ey_lee@organoidrx.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Low-Dose ATORM-C; 16,330 organoids (0.2 mL) per cm² of ulcer area	Biological: 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.
Experimental: Medium-Dose ATORM-C; 81,650 organoids (0.2 mL) per cm² of ulcer area	Biological: 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.
Experimental: High-Dose ATORM-C; 163,300 organoids (0.2 mL) per cm² of ulcer area	Biological: 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Dose-Limiting Toxicities (DLTs)	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.	From ATORM-C administration through 4 weeks post-dose
Determination of Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D)	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.	From ATORM-C administration through 24 weeks post-dose

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of Adverse Events (AEs) and Adverse Events of Special Interest (AESIs)	Occurrence of treatment-emergent adverse events (TEAEs), adverse drug reactions (ADRs), serious adverse events (SAEs), serious adverse drug reactions (SADRs), adverse events of special interest (AESIs), and administration site local adverse events. Adverse events leading to discontinuation of study treatment or death will also be evaluated.	From ATORM-C administration through 24 weeks post-dose
Changes in Clinical Laboratory Tests, Vital Signs, Physical Examinations, and 12-Lead Electrocardiograms (ECGs)	Changes from baseline in continuous safety parameters, including clinical laboratory tests and vital signs, evaluated at protocol-specified visits. Occurrence of clinically significant abnormalities (Abnormal CS) in clinical laboratory tests, physical examinations, and 12-lead electrocardiograms (ECGs) post-dose will also be evaluated.	Baseline through 24 weeks post-dose, at protocol-specified visits
Change from Baseline in Target Ulcer Size	Absolute change and percentage change from baseline in the size of the target ulcer, evaluated based on the longest diameter, shortest diameter, and total surface area measured during colonoscopy at protocol-specified time points.	Baseline and Weeks 4, 12, and 24 post-dose
Change from Baseline in Modified Global Histologic Disease Activity Score (Modified GHAS) of the Target Ulcer	Change from baseline in the Modified Global Histologic Disease Activity Score (Modified GHAS) based on histologic evaluation of mucosal biopsy specimens collected from the target ulcer. The regional Modified GHAS ranges from 0 to 12, with higher scores indicating greater histologic disease activity.	Baseline and Week 24 post-dose
Change from Baseline in Simple Endoscopic Score for Crohn's Disease (SES-CD) of the Target Ulcer	Change from baseline in the Simple Endoscopic Score for Crohn's Disease (SES-CD) total score and ulcer size subscore evaluated specifically for the target ulcer via colonoscopy. The SES-CD assessed for the target ulcer evaluates 4 endoscopic variables, each scored from 0 to 3, yielding a target ulcer regional score ranging from 0 to 12. Higher scores indicate greater endoscopic disease activity.	Baseline and Weeks 4, 12, and 24 post-dose
Proportion of Participants Achieving ≥50% Reduction from Baseline in SES-CD Score of the Target Ulcer	Percentage of participants who achieve a 50% or greater reduction from baseline in the Simple Endoscopic Score for Crohn's Disease (SES-CD) total score for the target ulcer at Week 24 post-dose. The regional target ulcer SES-CD ranges from 0 to 12, with higher scores indicating greater endoscopic disease activity.	Baseline and Week 24 post-dose
Proportion of Participants Achieving Endoscopic Remission of the Target Ulcer	Percentage of participants achieving endoscopic remission of the target ulcer at Week 24 post-dose. Endoscopic remission is defined as a Simple Endoscopic Score for Crohn's Disease (SES-CD) total score of less than 2 (<2) for the target ulcer. The regional target ulcer SES-CD ranges from 0 to 12, with higher scores indicating greater endoscopic disease activity.	Baseline and Weeks 4, 12, and 24 post-dose

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of Participants Achieving a ≥100-Point Reduction from Baseline in Crohn's Disease Activity Index (CDAI)	Percentage of participants who achieve a reduction of 100 points or greater (≥100 points) from baseline in the Crohn's Disease Activity Index (CDAI) total score at Week 24 post-dose. CDAI total scores range from 0 to over 600, with higher scores indicating greater disease activity. Frequencies and percentages will be summarized for each dose cohort and overall.	Baseline and Week 24 post-dose
Proportion of Participants Achieving Clinical Remission Based on Crohn's Disease Activity Index (CDAI)	Percentage of participants who achieve clinical remission, defined as a Crohn's Disease Activity Index (CDAI) total score of less than 150 (<150) at protocol-specified time points post-dose. Higher CDAI scores indicate greater Crohn's disease activity.	Weeks 4, 12, and 24 post-dose
Proportion of Participants Achieving Clinical Response Based on Patient-Reported Outcomes (PRO2)	Percentage of participants who achieve a clinical response based on patient-reported outcomes (PRO2), defined as a reduction of 30% or greater (≥30%) from baseline in either the 7-day average Stool Frequency (SF) subscore or Abdominal Pain (AP) subscore, with neither subscore worsening from baseline. The SF subscore ranges from 0 to 11+ (higher scores reflect greater stool frequency), and the AP subscore ranges from 0 to 3 (higher scores indicate more severe pain).	Baseline, and Weeks 4, 12, and 24 post-dose
Proportion of Participants Achieving Clinical Remission Based on Patient-Reported Outcomes (PRO), Defined as SF ≤2.8 and AP ≤1.0	Percentage of participants who achieve clinical remission based on patient-reported outcomes (PRO2), defined as a 7-day average daily Stool Frequency (SF) of 2.8 or less (≤2.8) and a 7-day average Abdominal Pain (AP) score of 1.0 or less (≤1.0). Higher SF values indicate greater stool frequency, and higher AP scores indicate more severe abdominal pain.	Weeks 4, 12, and 24 post-dose
Change and Percentage Change from Baseline in Fecal Calprotectin Levels	Absolute change and percentage change from baseline in fecal calprotectin levels (measured in µg/g) at protocol-specified time points post-dose.	Baseline, and Weeks 4, 12, and 24 post-dose
Change and Percentage Change from Baseline in C-Reactive Protein (CRP) Levels	Absolute change and percentage change from baseline in C-reactive protein (CRP) levels (measured in mg/L) at protocol-specified time points post-dose.	Baseline, and Weeks 4, 12, and 24 post-dose
Use of Rescue Medication During the Study	Percentage of participants who use rescue medication and the amount of rescue medication used at protocol-specified visits during the study period.	From ATORM-C administration through 24 weeks post-dose

Collaborators and Investigators

• Sponsor: ORGANOIDSCIENCES LTD.

Study record dates

Study Major Dates

• Study Start (Estimated): September 1, 2026
• Primary Completion (Estimated): August 1, 2028
• Study Completion (Estimated): August 1, 2028

Study Registration Dates

• First Submitted: September 10, 2026
• First Submitted That Met QC Criteria: September 10, 2026
• First Posted (Actual): September 16, 2026

Study Record Updates

• Last Update Posted (Actual): September 16, 2026
• Last Update Submitted That Met QC Criteria: September 10, 2026
• Last Verified: September 1, 2026

More Information

Terms related to this study

• Keywords: Crohn's disease; Crohn Disease; Dose Escalation; Intestinal Organoid; Autologous Organoid; Intestinal Ulcer; ATORM-C; Autologous Adult Stem Cell-Derived Intestinal Organoid
• Additional Relevant MeSH Terms: Intestinal Diseases; Digestive System Diseases; Gastrointestinal Diseases; Gastroenteritis; Inflammatory Bowel Diseases; Crohn Disease
• Other Study ID Numbers: ATORM-C_CD-101

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No