Treatment of Moderate to Severe Refractory Crohn's Disease

NCT ID: NCT06721962

Last Updated: 2025-12-10

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

39 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-05-05

Study Completion Date

2027-01-30

Brief Summary

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This research study is testing an investigational research product called TRX103 as a possible treatment for individuals suffering from Crohn's Disease (CD). The primary purpose of this study is to learn how safe and effective different doses of TRX103 are when administered to individuals with CD.

Detailed Description

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Please see our study website at https://www.cdclinicaltrial.com

Conditions

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Crohns Disease

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cohort 1

Dose level 1

Group Type EXPERIMENTAL

TRX103

Intervention Type BIOLOGICAL

TRX103 is an investigational research product that may treat and provide long term relief to individuals suffering from Crohn's Disease.

Cohort 2

Dose level 2

Group Type EXPERIMENTAL

TRX103

Intervention Type BIOLOGICAL

TRX103 is an investigational research product that may treat and provide long term relief to individuals suffering from Crohn's Disease.

Cohort 2A

Dose level 2 with conditioning

Group Type EXPERIMENTAL

TRX103

Intervention Type BIOLOGICAL

TRX103 is an investigational research product that may treat and provide long term relief to individuals suffering from Crohn's Disease.

Cyclophosphamide

Intervention Type DRUG

Low dose cyclophosphamide conditioning.

Cohort 3

Dose level 3

Group Type EXPERIMENTAL

TRX103

Intervention Type BIOLOGICAL

TRX103 is an investigational research product that may treat and provide long term relief to individuals suffering from Crohn's Disease.

Cohort 3A

Dose level 3 with conditioning

Group Type EXPERIMENTAL

TRX103

Intervention Type BIOLOGICAL

TRX103 is an investigational research product that may treat and provide long term relief to individuals suffering from Crohn's Disease.

Cyclophosphamide

Intervention Type DRUG

Low dose cyclophosphamide conditioning.

Interventions

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TRX103

TRX103 is an investigational research product that may treat and provide long term relief to individuals suffering from Crohn's Disease.

Intervention Type BIOLOGICAL

Cyclophosphamide

Low dose cyclophosphamide conditioning.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Male and females ≥ 18 and ≤ 65 years of age at time of consent.
2. Weight of ≥ 40 kg.
3. Medical history and biological evidence of active bowel inflammation documented by:

* Minimum of approximate 1 year of Crohn's disease diagnosis confirmed through Endoscopy, and;
* Endoscopic evidence of CD diagnosis at least 3 months prior to and/or at Screening (SES-CD ≥ 6 for ilealcolonic or ≥ 4 isolated ileal disease by central reader)
4. Active disease defined as moderate to severe active CD at Screening defined by all of the following:

* Evidence of mucosal inflammation, defined as SES-CD ≥ 6 (≥ 4 for subjects with isolated ileal disease), and;
* CDAI total scores ≥ 220
5. Subject on treatment with corticosteroids may be included if they meet the following:

* prednisone or equivalent dose ≤ 20 mg/day; or
* budesonide ≤ 9 mg/day; or
* has been on a stable dose for at least 7 days prior to TRX103 dose.
6. Advanced therapy-refractory disease defined by:

Failure of two or more advanced approved therapies. Prior therapies may be inclusive of any combination of the following:
* TNF-alpha inhibitors
* IL-12/23 inhibitors
* Anti-integrins
* JAK inhibitors Failure of therapies are defined as either non-response (primary failure), complete loss of response (secondary failure) or intolerant to therapy at a dose indicated for CD.
* Primary failure is defined as:

* When a subject does not achieve a response after having received the induction doses of a CD approved drug per prescribing information. Induction period is defined as 12-weeks.
* A response is defined as CDAI score that reduces by ≥ 100-point from Baseline or by Physician assessment.
* Secondary failure, or relapse defined as:

* Subjects who respond to the therapy or achieves remission after an induction regimen per prescribing information, but subsequently lose response or relapses during maintenance treatment.
* Relapse is defined as an increase in the CDAI score from maintenance of ≥ 100 points and a CDAI score \> 220, and/or SES-CD score ≥ 6 (or ≥ 4 if isolated ileal disease) or by Physician assessment.
* Intolerant to therapy, defined as:

* When a subject is unable to cope with the side effects or mechanism of actions of the treatment and/or experiences unacceptable side effects when dosed at appropriate therapeutic levels.
7. Absence of uncontrolled bacterial, viral, or fungal infection at time of enrollment.
8. Subjects must be able to understand and sign informed consent and be willing and able to complete all specified procedures and visits.

Exclusion Criteria

1. Prior organ transplant, or allogeneic bone marrow, peripheral blood, or cord blood stem cell transplant. Note: Blood transfusion are not exclusionary if it occurred ≥ 3 years (- 3 months) of TRX103 infusion.
2. Received another investigational agent or therapy, within 28 days of planned TRX103 infusion (or 5 half-lives, whichever is longer) and/or have not recovered from treatment related toxicities.
3. Received any approved treatment for CD within the designated washout period (including off-label use of approved therapies) as per the protocol.
4. Strictures, active fistulae (including perianal), or abscess by computed tomography (CT) or magnetic resonance enterography (MRE) or Endoscopy within 6 months of Screening.
5. Positive serology for HIV.
6. Positive hepatitis-B surface antigen. Subject may be included if they are HBV PCR negative.
7. Hepatitis C virus (HCV RNA detectable in any subject with anti-HCV antibodies).
8. Subject with active or chronic recurring infections or untreated latent Tuberculosis (TB).
9. Current diagnosis of ulcerative colitis (UC), indeterminate colitis or CD isolated to colon only (the colitis must be related to CD and inclusive of ileal with or without colonic involvement).
10. Subjects with the following known complications of Crohn's Disease

* active diverticulitis,
* active fistulae or abscess,
* abscess (abdominal or perianal) - abscess with no evidence of pus when pressed upon are permitted,
* impassable fibrotic strictures - Patients with strictures passable by dilation are permitted,
* symptomatic bowel strictures - must be confirmed via endoscopy and/or radiologically,
* fulminant colitis,
* toxic megacolon,
* ostomy or ileoanal pouch - previous temporary ostomy pouch, followed by a reversal is permitted,
* diagnosed with short gut or short bowel syndrome,
* or any other manifestation that might require surgery while enrolled in the study.
11. Subject with surgical bowel resection within the past 3 months prior to Screening, or a history of \> 2 bowel resections. Note: Surgery for temporary use of an ostomy bag or reconnection of the gut post colostomy use is not considered exclusionary, nor considered as part of the surgical resection if during the reconnection removal of tissue is required to facilitate reattachment.
12. Subjects that are pregnant, breast feeding, or aim to become pregnant during the 12 month study period. (Subjects, males and females, must agree to use a highly effective method of contraception).
13. Screening laboratory and other analyses show any of the following abnormal results:

* Serum aspartate transaminase or alanine transaminase \> 3.0 × upper limit of normal;
* Total white blood cell count \< 2,000/μL;
* Estimated glomerular filtration rate by simplified 4-variable Modification of Diet in Renal Disease (MDRD) formula or by Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation \< 40 mL/min/1.73 m2;
* Hemoglobin \< 8 g/dL;
* Bilirubin ≥ 2 x ULN;
* Platelet count \< 100,000/μL;
* Absolute neutrophil count \< 1,200/μL;
* Absolute lymphocytes count \< 750/μL.
14. Any subject with a history of significant renal, hepatic, pulmonary, or cardiac dysfunction, or on treatment to support cardiac dysfunction.
15. Any serious illness, uncontrolled inter-current illness, psychiatric illness, active or uncontrolled infection, or other medical condition or history, including laboratory results, which, in the Investigator's opinion:

* places the subject at increased risk during participation in the study, and/or;
* interferes with the subject's capacity to provide informed consent and their participation in the study, and/or;
* interferes with the interpretation of the results.
Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Tr1X, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Mayo Clinic Arizona

Scottsdale, Arizona, United States

Site Status RECRUITING

University of California, Davis

Sacramento, California, United States

Site Status RECRUITING

University of California, San Francisco

San Francisco, California, United States

Site Status RECRUITING

University of Florida

Gainesville, Florida, United States

Site Status RECRUITING

Northwestern

Chicago, Illinois, United States

Site Status RECRUITING

University of Chicago

Chicago, Illinois, United States

Site Status RECRUITING

University of Iowa

Iowa City, Iowa, United States

Site Status RECRUITING

University of Louisville

Louisville, Kentucky, United States

Site Status RECRUITING

University of Michigan

Ann Arbor, Michigan, United States

Site Status RECRUITING

Mayo Clinic Rochester

Rochester, Minnesota, United States

Site Status RECRUITING

Washington University in St. Louis

St Louis, Missouri, United States

Site Status RECRUITING

Mount Sinai Health Systems

New York, New York, United States

Site Status RECRUITING

Cleveland Clinic

Cleveland, Ohio, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Tr1X Clinical Trials Tr1X Clinical Trials

Role: CONTACT

858-283-7879

Study Team

Role: CONTACT

Facility Contacts

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Study Coordinator

Role: primary

480-301-6373

Clinical Research Supervisor

Role: primary

(916) 734-0753

Study Coordinator

Role: primary

(415) 514-1170

Study Coordinator

Role: primary

352-273-9483

Study Coordinator

Role: primary

312-695-5878

Clinical Trial Manager

Role: primary

773-834-7414

Study Coordinator

Role: primary

319-467-4169

Study Coordinator

Role: primary

502-852-6993

Clinical Trial Manager

Role: primary

734-615-4843

Study Coordinator

Role: primary

507-266-4728

Study Coordinator

Role: primary

314-273-0301

Study Coordinator

Role: primary

(929) 641-0917

Study Coordinator

Role: primary

440-523-8503

Other Identifiers

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RESTORE Study

Identifier Type: OTHER

Identifier Source: secondary_id

TRX103-02

Identifier Type: -

Identifier Source: org_study_id

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