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Crohns DiseasePhase 1/2NCT06721962

Treatment of Moderate to Severe Refractory Crohn's Disease

A Phase 1/2 study testing TRX103 and Cyclophosphamide in people with Crohns Disease, run by Tr1X, Inc. at 13 sites in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

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. Read more about this study

Who is it for?
Ages 18 to 65 years
Conditions the study is for
Crohns Disease
What is being tested?
TRX103 and Cyclophosphamide
Could I receive a placebo?
No, everyone receives an active treatment
How many people
39 participants
Where
13 sitesUnited States
Study dates
5 May 2025to 30 Jan 2027
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 65 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Male and females ≥ 18 and ≤ 65 years of age at time of consent.
  • Weight of ≥ 40 kg.
  • 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)
  • Active disease defined as moderate to severe active CD at Screening defined by all of the following:
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  • Evidence of mucosal inflammation, defined as SES-CD ≥ 6 (≥ 4 for subjects with isolated ileal disease), and;
  • CDAI total scores ≥ 220
  • 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.
  • 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.
  • Absence of uncontrolled bacterial, viral, or fungal infection at time of enrollment.
  • Subjects must be able to understand and sign informed consent and be willing and able to complete all specified procedures and visits.

Who cannot join

  • 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.
  • 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.
  • Received any approved treatment for CD within the designated washout period (including off-label use of approved therapies) as per the protocol.
  • Strictures, active fistulae (including perianal), or abscess by computed tomography (CT) or magnetic resonance enterography (MRE) or Endoscopy within 6 months of Screening.
  • Positive serology for HIV.
  • Positive hepatitis-B surface antigen. Subject may be included if they are HBV PCR negative.
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  • Hepatitis C virus (HCV RNA detectable in any subject with anti-HCV antibodies).
  • Subject with active or chronic recurring infections or untreated latent Tuberculosis (TB).
  • 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).
  • 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.
  • 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.
  • 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).
  • 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.
  • Any subject with a history of significant renal, hepatic, pulmonary, or cardiac dysfunction, or on treatment to support cardiac dysfunction.
  • 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.

Trial locations and contacts

13 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Tr1X Clinical Trials Tr1X Clinical Trials contact

858-283-7879

Tr1xClinicalTrials@Tr1x.bio

Study Team contact

Tr1xClinicalTrials@Tr1x.bio

13 sites in 1 country

United States13 sites, 13 recruiting

  • University of Michigan

    Ann Arbor, Michigan, 48109

    Clinical Trial Manager, 734-615-4843, harrisnl@med.umich.edu

    Recruiting

  • Northwestern

    Chicago, Illinois, 60611

    Study Coordinator, 312-695-5878, rarrieta@northwestern.edu

    Recruiting

  • University of Chicago

    Chicago, Illinois, 60637

    Clinical Trial Manager, 773-834-7414, Kristi.Kearney@uchicagomedicine.org

    Recruiting

  • Cleveland Clinic

    Cleveland, Ohio, 44195

    Study Coordinator, 440-523-8503, dussanl@ccf.org

    Recruiting

  • University of Florida

    Gainesville, Florida, 32608

    Study Coordinator, 352-273-9483, Valeria.BlancoBorges@medicine.ufl.edu

    Recruiting

  • University of Iowa

    Iowa City, Iowa, 52242

    Study Coordinator, 319-467-4169, megan-sharer@uiowa.edu

    Recruiting

  • University of Louisville

    Louisville, Kentucky, 40202

    Study Coordinator, 502-852-6993, Kelsey.small@louisville.edu

    Recruiting

  • Mount Sinai Health Systems

    New York, New York, 10029

    Study Coordinator, (929) 641-0917, Phyu.mar@mssm.edu

    Recruiting

  • Mayo Clinic Rochester

    Rochester, Minnesota, 55905

    Study Coordinator, 507-266-4728, IBDRESEARCH@mayo.edu

    Recruiting

  • University of California, Davis

    Sacramento, California, 95817

    Clinical Research Supervisor, (916) 734-0753, tyassear@health.ucdavis.edu

    Recruiting

  • University of California, San Francisco

    San Francisco, California, 94158

    Study Coordinator, (415) 514-1170, paige.shave@ucsf.edu

    Recruiting

  • Mayo Clinic Arizona

    Scottsdale, Arizona, 85259

    Study Coordinator, 480-301-6373, Hall.Megan@mayo.edu

    Recruiting

  • Washington University in St. Louis

    St Louis, Missouri, 63110

    Study Coordinator, 314-273-0301, luluhuang@wustl.edu

    Recruiting

Other trials in United States

About this study

Official title: A Phase 1/2a, Open Label, Dose Escalation Study to Evaluate the Safety and Preliminary Efficacy of TRX103 in Subjects With Moderate to Severe Treatment-Refractory Crohn's Disease

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.

Read the detailed description

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

Conditions
Crohns Disease
Being tested
TRX103, Cyclophosphamide
Keywords
Tr1X, RESTORE, Crohns, Crohns Disease, autoimmune, IBD, CD, chronic inflammation, gastrointestinal tract, chronic diarrhea, intestinal ulcers, abdominal pain
Medical subject headings
Crohn Disease, Abdominal Pain

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • TRX103Biological

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

  • CyclophosphamideDrug

    Low dose cyclophosphamide conditioning.

Study groups in NCT06721962
Study groupRoleWhat they receive
Cohort 1ExperimentalDose level 1
Receives: Biological: TRX103
Cohort 2ExperimentalDose level 2
Receives: Biological: TRX103
Cohort 2AExperimentalDose level 2 with conditioning
Receives: Biological: TRX103; Drug: Cyclophosphamide
Cohort 3ExperimentalDose level 3
Receives: Biological: TRX103
Cohort 3AExperimentalDose level 3 with conditioning
Receives: Biological: TRX103; Drug: Cyclophosphamide

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. To assess the safety and tolerability of TRX103 infusion in subjects with moderate to severe treatment-refractory Crohn's Disease.

    As measured by:

    • Incidence and severity of treatment-emergent adverse events (TEAEs) and treatment-emergent serious adverse events (TESAEs), including CD flares (perforations, abscesses).
    • Incidence of infections, either bacterial, fungal or viral.
    • The safety of TRX103 determined by negative Replication Competent Lentivirus (RCL) at approximately 3-months, 6-months, and 12-months.

    Measured: From baseline until 12 months post TRX103 infusion.

Secondary outcomes (7)

  1. Improvement in Simple Endoscopic Score for Crohn's Disease (SES-CD Score).

    Endoscopic remission defined as:

    • Participants with ileocolonic or isolated ileal CD: SES-CD ≤ 4 points and no sub-score > 1 point in any individual variable, as scored by central reviewer at Week 12.

    OR

    • Participants with isolated ileal disease: SES-CD of 0 - 2 points, as scored by central reviewer at Week 12.

    Endoscopic response, defined as: Decrease in SES-CD ≥ 50% from Baseline at Week 12.

    Measured: From baseline to Week 12 post TRX103 infusion.

  2. Improvement of CD status.

    • Clinical remission, defined as CD Activity Index (CDAI) ≤ 150 at Week 12.
    • Clinical response, defined as ≥ 100-point reduction in CDAI score from Baseline at Week 12.
    • Proportion of subjects who discontinue corticosteroid use and achieve clinical remission at all subsequent timepoints, in subjects taking corticosteroids at Baseline.

    Measured: From baseline to Week 12 post TRX103 infusion.

  3. Reduction in Fecal Calprotectin.

    • Change from Baseline in fecal calprotectin at each timepoint.
    • Proportion of subjects with fecal calprotectin < 250 μg/mg at Week 12 or a decrease of 50% from Baseline.

    Measured: From baseline to Week 12 post TRX103 infusion.

  4. Reduction of highly sensitive C-reactive protein (hs-CRP).

    Change from Baseline in hs-CRP at each timepoint.

    Measured: From baseline until 12 months post TRX103 infusion.

  5. Improvement in Patient Reported Outcomes (PRO).

    Change from Baseline in Health-Related Quality-of-Life (HRQOL) Questionnaires scores:

    • Inflammatory Bowel Disease Questionnaire (IBDQ)
    • EuroQol Group - 5 Dimension - 5 Level (EQ-5D-5L) Questionnaire

    Measured: From baseline until 12 months post TRX103 infusion.

  6. Pharmacokinetics (PK) of TRX103.

    TRX103 kinetics post infusion.

    Measured: From baseline until 12 months post TRX103 infusion.

  7. Level of Inflammation and mucosal damage

    Change from baseline in histopathological scores by Global Histologic Disease Activity Score (GHAS) and Robarts Histopathology Index (RHI).

    Measured: From baseline until 12 months post TRX103 infusion.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted6 Dec 2024
  2. Study start5 May 2025
  3. Primary completion30 Jan 2026 (estimated)
  4. Study completion30 Jan 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
39 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06721962
Sponsor's protocol ID
TRX103-02
Other IDs
RESTORE Study
Status last verified
Dec 2025
First submitted
25 Nov 2024
Last updated
10 Dec 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing TRX103 and Cyclophosphamide in people with Crohns Disease, run by Tr1X, Inc. at 13 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 28 inclusion and 36 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 10 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 13 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Tr1X Clinical Trials Tr1X Clinical Trials (858-283-7879, Tr1xClinicalTrials@Tr1x.bio), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

To assess the safety and tolerability of TRX103 infusion in subjects with moderate to severe treatment-refractory Crohn's Disease. (time frame: From baseline until 12 months post TRX103 infusion.). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06721962.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Dec 2025.

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