Treatment of Moderate to Severe Refractory Crohn's Disease
ConditionCrohns Disease
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18-65
SponsorTr1X, Inc.
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Male and females ≥ 18 and ≤ 65 years of age at time of consent.
Weight of ≥ 40 kg.
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)
Disqualifiers
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.
Trial design
Design model
Sequential
Treatments tested in this trial
TRX103
Biological/VaccineTRX103 is an investigational research product that may treat and provide long term relief to individuals suffering from Crohn's Disease.
Cyclophosphamide
DrugLow dose cyclophosphamide conditioning.
Treatment groups
39 Participants
are divided into 5 treatment groupsGroup A: Cohort 1Experimental treatment 1 intervention
Group B: Cohort 2Experimental treatment 1 intervention
Group C: Cohort 2AExperimental treatment 2 interventions
Group D: Cohort 3Experimental treatment 1 intervention
Group E: Cohort 3AExperimental treatment 2 interventions
Trial outcomes
Primary outcomes
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.
Time frame
From baseline until 12 months post TRX103 infusion.Secondary outcomes
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.
Time frame
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.
Time frame
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.
Time frame
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.
Time frame
From baseline until 12 months post TRX103 infusion. Source ClinicalTrials.gov