- ICH GCP
- US Clinical Trials Registry
- Klinisk forsøg NCT07649096
A Safety Trial to Evaluate an Orally Ingested Yeast Modified to Express a Tetra-specific Anti-toxin for Clostridioides Difficile
A Phase 1 Trial to Evaluate an Orally Ingested Probiotic Yeast Genetically Modified to Express a Tetra-specific Anti-toxin for Clostridioides Difficile
This is a Phase 1, first-in-human, double-blind, placebo-controlled, adaptive-design, single-site study, involving dose exploration cohorts.
During Part A, up to 4 cohorts, each consisting of approximately 14 healthy adult study participants, will be randomly allocated to receive either FZ002 or placebo. Part A is designed to begin with Cohort 1 evaluating the highest proposed dose of study product. This is intended to represent the "ceiling" (highest level) of the ranges of doses to be evaluated in the study. In the event of a safety or tolerability concern in Cohort 1, a dose de-escalation will be evaluated in Cohorts 2-4 until a safe and well-tolerated dose is identified. If no safety or tolerability concerns are identified at the completion of a cohort, the study will proceed to Part B with the approval of the independent Safety Monitoring Committee (SMC). This adaptive design allows for the progression of the study from Part A to Part B without necessarily progressing through all 4 cohorts in Part A if there is a lack of safety or tolerability concerns.
During Part B, an initial 'sentinel' Cohort 5 "some-risk" adult study participants will be randomly allocated to receive a single daily dose of FZ002 or placebo for 28 consecutive days. This is intended to represent the "floor" (lowest level) of doses to be evaluated in the study. A Protocol Safety Review Team (PSRT) will review the available safety data from the first 7 days of Cohort 5. If there is a lack of safety or tolerability concerns over the first 7-days of dosing for Cohort 5, then Cohort 6 will be opened for enrollment. Cohort 6 "some-risk" adult study participants will evaluate the "floor" and "ceiling" dose of FZ002 versus placebo.
The primary objective is to evaluate safety and clinical tolerability of oral doses of FZ002 or placebo when taken for up to 28 days.
Studieoversigt
Status
Betingelser
Intervention / Behandling
Detaljeret beskrivelse
This is a Phase 1, first-in-human, double-blind, placebo-controlled, adaptive-design, single-site study, involving dose exploration cohorts.
During Part A, up to 4 cohorts, each consisting of approximately 14 healthy adult study participants, will be randomly allocated to receive either FZ002 or placebo. Part A is designed to begin with Cohort 1 evaluating the highest proposed dose of study product. This is intended to represent the "ceiling" (highest level) of the ranges of doses to be evaluated in the study. In the event of a safety or tolerability concern in Cohort 1, a dose de-escalation will be evaluated in Cohorts 2-4 until a safe and well-tolerated dose is identified. If no safety or tolerability concerns are identified at the completion of a cohort, the study will proceed to Part B with the approval of the independent Safety Monitoring Committee (SMC). This adaptive design allows for the progression of the study from Part A to Part B without necessarily progressing through all 4 cohorts in Part A if there is a lack of safety or tolerability concerns.
During Part B, an initial 'sentinel' Cohort 5 "some-risk" adult study participants will be randomly allocated to receive a single daily dose of FZ002 or placebo for 28 consecutive days. This is intended to represent the "floor" (lowest level) of doses to be evaluated in the study. A Protocol Safety Review Team (PSRT) will review the available safety data from the first 7 days of Cohort 5. If there is a lack of safety or tolerability concerns over the first 7-days of dosing for Cohort 5, then Cohort 6 will be opened for enrollment. Cohort 6 "some-risk" adult study participants will evaluate the "floor" and "ceiling" dose of FZ002 versus placebo.
The primary objective is to evaluate safety and clinical tolerability of oral doses of FZ002 or placebo when taken for up to 28 days. The secondary objective is to evaluate laboratory abnormalities with FZ002 or placebo when taken for up to 28 days.
Undersøgelsestype
Tilmelding (Anslået)
Fase
- Fase 1
Kontakter og lokationer
Studiekontakt
- Navn: William B Smith
- Telefonnummer: 18663059100
- E-mail: william.smith@amr-clinical.com
Deltagelseskriterier
Berettigelseskriterier
Aldre berettiget til at studere
- Voksen
- Ældre voksen
Tager imod sunde frivillige
Beskrivelse
Inclusion Criteria:
To be eligible to participate in Part A of this trial, an individual must meet all of the following criteria:
- Provides written informed consent prior to the initiation of any trial procedures.
Is able to understand and agrees to comply with all planned trial procedures and be available for all study visits, including:
- Receiving 28 days of blinded oral study product
- Providing blood and self-collected stool samples
- Is a non-pregnant individual, aged 18-75 years, inclusive, at the time of enrollment.
- Has no more than 1 single Grade 1 screening laboratory abnormality that is not clinically significant. If the participant has more than 1 Grade 1 abnormality, it must be approved by the Division of Microbiology and Infectious Diseases [DMID] Medical Monitor (MM).
- Participants of childbearing potential: use of adequate contraception for at least 4 weeks prior to enrollment and agreement to use such a method during trial participation and for an additional 4 weeks after the last dose of blinded study product. Note: Definitions of participants of childbearing potential and adequate contraception are provided in Section 13.1.
Agrees to refrain from ingestion of probiotics* or fermented foods** from 7 days prior to study product administration, while on blinded study product, and through Day 36 (Visit 6).
*E.g., probiotic nutritional Lactobacillus or Bifidobacterium supplements, yogurt, kefir, any "live and active culture" nutritional product, etc.
**E.g., kombucha, fermented pickled vegetables, etc.
Has good health by medical history, vital signs, physical examination, and concomitant medication review*. Participants should be stable based on their condition over the last 3 months prior to enrollment.
*As defined by not requiring a change in therapy (including dose or frequency) or medical care for worsening disease for at least 3 months prior to enrollment. Vital signs must not meet Grade 1 or higher.
Not using medications daily that may affect gut motility, gastric acidity, or baseline gut function* within 3 months of enrollment and through Day 36 (Visit 6).
*E.g., proton pump inhibitors, opioids, anti-diarrheal agents, anti-constipation agents, daily Over-the-counter [OTC] "heartburn" relief medications.
- Not using glucagon-like peptide-1 (GLP-1) receptor agonists within 6 weeks of enrollment.
- Any elective surgery (including dental) that required preoperative or postoperative antibiotics must have been completed at least 3 months prior to enrollment.
No history of Clostridioides difficile infection [CDI] (suspected or proven), no recent hospital admissions (within 3 years), and no receipt of systemic antibiotics known to increase risk of CDI* (within 6 months).
*Systemic antibiotics that are known to increase the risk of CDI include lincosamides (e.g., clindamycin), monobactams (e.g., aztreonam), extended-spectrum penicillin combinations with beta-lactamase inhibitors (e.g., piperacillin-tazobactam), carbapenems (e.g., imipenem, meropenem, ertapenem), third generation or higher cephalosporins, and fluoroquinolones. Common oral antibiotics that would be allowable or are not known to significantly increase risk of CDI include amoxicillin, azithromycin, cephalexin, doxycycline, metronidazole, and trimethoprim/sulfamethoxazole.
- No history of other enteric infections or diarrheal illness within 3 months of enrollment.
In order to be eligible to participate in Part B of this trial, an individual must meet all of the following criteria:
- Provides written informed consent prior to the initiation of any trial procedures.
Is able to understand and agrees to comply with all planned trial procedures and be available for all study visits, including:
- Receiving 28 days of blinded oral study product
- Providing blood and self-collected stool samples
- Is non-pregnant individual, aged 18-75 years, inclusive, at the time of enrollment.
- Has no more than 1 single Grade 1 hematology result and no more than 1 Grade 1 metabolic panel result. If the participant has more than 1 Grade 1 abnormality, it must be approved by the MM.
- Participants of childbearing potential: use of adequate contraception for at least 4 weeks prior to enrollment through 4 weeks after the last dose of blinded study product. Note: Definitions of participants of childbearing potential and adequate contraception are provided in Section 13.1.
To rule out live microbial products and factors that may actively modulate the gut microbiome, participant agrees to refrain from ingestion of probiotics* or fermented foods** from 7 days prior to study product administration, while on blinded study product, and through Day 36 (Visit 6).
* E.g., probiotic nutritional Lactobacillus or Bifidobacterium supplements, yogurt, kefir, any "live and active culture" nutritional product, etc.
** E.g., kombucha, fermented pickled vegetables, etc.
- Stable health by medical history, vital signs, physical examination, concomitant medication review, not requiring a change in therapy or medical care for worsening disease within 1 month of enrollment.
Not using medications daily that may affect gut motility, gastric acidity, or baseline gut function* within 1 month of enrollment.
*E.g., proton pump inhibitors, opioids, anti-diarrheal agents, anti-constipation agents, daily OTC "heartburn" relief medications.
- Not using GLP-1 receptor agonists within 6 weeks of enrollment.
- Any elective surgery (including dental) that required preoperative or postoperative antibiotics must have been completed at least 1 month prior to enrollment.
Must have had at least 1 of these 3 conditions:
- History of CDI (suspected or proven) within the past 3 years, with "cure" or no symptoms for at least 1 month prior to enrollment
- History of a hospitalization within the past 3 years, but discharge not sooner than 1 month prior to enrollment
- History of receiving systemic antibiotics known to increase the risk of CDI* within the past 3 years, but last dose not sooner than 1 month prior to enrollment *Systemic antibiotics that are known to increase risk of CDI include lincosamides (e.g., clindamycin), monobactams (e.g., aztreonam), extended-spectrum penicillin combinations with beta-lactamase inhibitors (e.g., piperacillin-tazobactam), carbapenems (e.g., imipenem, meropenem, ertapenem), third generation or higher cephalosporins, and fluoroquinolones. Common oral antibiotics that would be allowable or are not known to significantly increase the risk of CDI include amoxicillin, azithromycin, cephalexin, doxycycline, metronidazole, and trimethoprim/sulfamethoxazole.
- No history of other enteric infections or diarrheal illness within 1 month of enrollment.
Exclusion Criteria:
- Dwells in a long-term care or skilled nursing facility (living independently with limited nursing care is allowable).
- Known to be pregnant or has a positive pregnancy test at screening or enrollment.
- Currently breastfeeding a child.
Known to have significant hypersensitivity to any components of the study product; including S. boulardii (or any S. boulardii-based nutritional supplement), hydroxypropyl methylcellulose, and Microcrystalline cellulose [MCC].
Hydroxypropyl methylcellulose is the major component of the capsule shell and MCC is the placebo component.
- Known to have significant hypersensitivity to a first-line antifungal therapy (i.e., fluconazole or amphotericin B) against Saccharomyces.
- Known to be immunocompromised or have known or suspected congenital or acquired immunodeficiency, as determined by the investigator.
- Receipt of immunosuppressive therapy such as anti-cancer chemotherapy or radiation therapy within 3 years of enrollment.
Receipt of chronic (>/=14 days) immunosuppressive corticosteroids at a dose of >/=20 mg prednisone daily or prednisone equivalent within 30 days of enrollment, including oral, parenteral, or high-dose inhaled* corticosteroids.
*High-dose inhaled corticosteroid is defined as >800 mcg/day of beclomethasone dipropionate CFC or equivalent. Intra-articular, intranasal, and topical steroids are allowable.
- Active malignancy or history of malignancy in the past 3 years (non-melanoma, excised, cured skin cancers are allowed).
- History of or testing positive for human immunodeficiency virus (HIV), hepatitis B, or active hepatitis C at screening (positive hepatitis C antibody and detectable hepatitis C virus RNA).
- Anticipated or current receipt of kidney dialysis treatment.
- Concurrent, acutely life-threatening disease or condition, or any unstable medical history, as determined by the investigator.
Significant chronic gastrointestinal [GI] condition(s) or disease*.
*Includes diagnosis of inflammatory bowel disease or active Rome IV irritable bowel syndrome, poorly controlled Celiac disease, active gastroparesis, toxic megacolon, colostomy, intestinal resection (except uncomplicated appendectomy), ileus, short gut syndrome, or recent (within 6 months of enrollment) diverticular bleeding requiring surgical intervention or transfusion.
- Recent (within 6 months of enrollment) history of difficulty with swallowing food, liquids, or pills.
- Prosthetic heart valve or indwelling foreign structure within vascular supply (e.g., no central line or indwelling catheter) or known to have moderate to severe valvular disease.
- History of alcohol abuse or drug addiction within 5 years of enrollment or that might interfere with the ability to comply with trial procedures.
- Diagnosis of schizophrenia, bipolar disease, or other significant psychiatric disease in the opinion of the investigator that may interfere with or pose a problem with compliance with the study or the welfare of the participant.
- Presence of mild, acute, or self-limited condition, such as fever or cough or other symptoms of upper respiratory tract infection within 7 days prior to planned randomization.
- Any chronic illness that, in the opinion of the investigator, is at a stage where it might interfere with trial conduct or completion or participant safety.
- Identified as a study site or contract research organization employee or family member who is involved in the protocol and may have direct access to trial-related data.
- Participating in another clinical trial investigating a vaccine, drug, medical device, or medical procedure within 4 weeks of enrollment through the last visit of the study.
Studieplan
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Behandling
- Tildeling: Randomiseret
- Interventionel model: Sekventiel tildeling
- Maskning: Tredobbelt
Våben og indgreb
Deltagergruppe / Arm |
Intervention / Behandling |
|---|---|
|
Eksperimentel: Part A Cohort 1 Arm FZ002
Healthy adults administered 4 capsules of FZ002 orally QH12 for 28 days.
N=12
|
A recombinant live biotherapeutic product (rLBP) for oral administration, bioengineered from the parental S. boulardii (Sb) strain to constitutively produce ABAB, an anti-toxin against TcdA and TcdB of CDiff, intended to reduce the risk of CDI recurrence.
|
|
Placebo komparator: Part A Cohort 1 Arm Placebo
Healthy adults administered 4 capsules of placebo orally QH12 for 28 days.
N=2
|
The placebo is a capsule filled with commercially sourced pharmaceutical-grade microcrystalline cellulose (MCC), an inert substance.
No anticipated risk related to the study product.
|
|
Eksperimentel: Part A Cohort 2 Arm FZ002
Healthy adults administered 2 capsules of FZ002 orally QH12 for 28 days.
N=12
|
A recombinant live biotherapeutic product (rLBP) for oral administration, bioengineered from the parental S. boulardii (Sb) strain to constitutively produce ABAB, an anti-toxin against TcdA and TcdB of CDiff, intended to reduce the risk of CDI recurrence.
|
|
Placebo komparator: Part A Cohort 2 Arm Placebo
Healthy adults administered 2 capsules of placebo orally QH12 for 28 days.
N=2
|
The placebo is a capsule filled with commercially sourced pharmaceutical-grade microcrystalline cellulose (MCC), an inert substance.
No anticipated risk related to the study product.
|
|
Eksperimentel: Part A Cohort 3 Arm FZ002
Healthy adults administered 1 capsule of FZ002 orally QH12 for 28 days.
N=12
|
A recombinant live biotherapeutic product (rLBP) for oral administration, bioengineered from the parental S. boulardii (Sb) strain to constitutively produce ABAB, an anti-toxin against TcdA and TcdB of CDiff, intended to reduce the risk of CDI recurrence.
|
|
Placebo komparator: Part A Cohort 3 Arm Placebo
Healthy adults administered 1 capsule of placebo orally QH12 for 28 days.
N=2
|
The placebo is a capsule filled with commercially sourced pharmaceutical-grade microcrystalline cellulose (MCC), an inert substance.
No anticipated risk related to the study product.
|
|
Eksperimentel: Part A Cohort 4 Arm FZ002
Healthy adults administered with 1 capsule of FZ002 orally once daily for 28 days.
N=12
|
A recombinant live biotherapeutic product (rLBP) for oral administration, bioengineered from the parental S. boulardii (Sb) strain to constitutively produce ABAB, an anti-toxin against TcdA and TcdB of CDiff, intended to reduce the risk of CDI recurrence.
|
|
Placebo komparator: Part A Cohort 4 Arm Placebo
Healthy adults administered with 1 capsule of placebo orally once daily for 28 days.
N=2
|
The placebo is a capsule filled with commercially sourced pharmaceutical-grade microcrystalline cellulose (MCC), an inert substance.
No anticipated risk related to the study product.
|
|
Eksperimentel: Part B Cohort 5 Arm FZ002
"some-risk" adults administered with 1 capsule of FZ002 orally once daily for 28 days.
N=4
|
A recombinant live biotherapeutic product (rLBP) for oral administration, bioengineered from the parental S. boulardii (Sb) strain to constitutively produce ABAB, an anti-toxin against TcdA and TcdB of CDiff, intended to reduce the risk of CDI recurrence.
|
|
Placebo komparator: Part B Cohort 5 Arm Placebo
"some-risk" adults administered with 1 capsule of placebo orally once daily for 28 days.
N=1
|
The placebo is a capsule filled with commercially sourced pharmaceutical-grade microcrystalline cellulose (MCC), an inert substance.
No anticipated risk related to the study product.
|
|
Eksperimentel: Part B Cohort 6 Group 1 Arm FZ002
"some-risk" adults administered with 4 capsules of FZ002 orally QH12 for 28 days.
N=12
|
A recombinant live biotherapeutic product (rLBP) for oral administration, bioengineered from the parental S. boulardii (Sb) strain to constitutively produce ABAB, an anti-toxin against TcdA and TcdB of CDiff, intended to reduce the risk of CDI recurrence.
|
|
Placebo komparator: Part B Cohort 6 Group 1 Arm Placebo
"some-risk" adults administered with 4 capsules of placebo orally QH12 for 28 days.
N=3
|
The placebo is a capsule filled with commercially sourced pharmaceutical-grade microcrystalline cellulose (MCC), an inert substance.
No anticipated risk related to the study product.
|
|
Eksperimentel: Part B Cohort 6 Group 2 Arm FZ002
"some-risk" adults administered with 1 capsule of FZ002 orally once daily for 28 days.
N=8
|
A recombinant live biotherapeutic product (rLBP) for oral administration, bioengineered from the parental S. boulardii (Sb) strain to constitutively produce ABAB, an anti-toxin against TcdA and TcdB of CDiff, intended to reduce the risk of CDI recurrence.
|
|
Placebo komparator: Part B Cohort 6 Group 2 Arm Placebo
"some-risk" adults administered with 1 capsule of placebo orally once daily for 28 days.
N=2
|
The placebo is a capsule filled with commercially sourced pharmaceutical-grade microcrystalline cellulose (MCC), an inert substance.
No anticipated risk related to the study product.
|
Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Occurrence of serious adverse events (SAEs)
Tidsramme: Through Day 208
|
Through Day 208
|
|
|
Occurrence of solicited adverse events (AEs)
Tidsramme: Through Day 35
|
Solicited AEs will include headache, fever, nausea, abdominal pain, diarrhea, malaise, constipation, fatigue, and vomiting.
Oral temperatures will be measured daily.
|
Through Day 35
|
|
Occurrence of unsolicited AEs
Tidsramme: Through Day 57
|
Through Day 57
|
|
|
The occurrence of any positive yeast blood culture for Saccharomyces
Tidsramme: Days 8 and 29
|
Days 8 and 29
|
Sekundære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
The occurrence of abnormalities in clinical safety laboratory parameters based on change from baseline
Tidsramme: Days 8 and 29
|
laboratory parameters include White blood cell [WBC], Absolute neutrophil count [ANC], Hemoglobin [Hg], Platelets [PLT], creatinine, Alanine aminotransferase [ALT], Aspartate aminotransferase [AST], and total bilirubin
|
Days 8 and 29
|
Samarbejdspartnere og efterforskere
Samarbejdspartnere
Datoer for undersøgelser
Studer store datoer
Studiestart (Anslået)
Primær færdiggørelse (Anslået)
Studieafslutning (Anslået)
Datoer for studieregistrering
Først indsendt
Først indsendt, der opfyldte QC-kriterier
Først opslået (Faktiske)
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
Sidst verificeret
Mere information
Begreber relateret til denne undersøgelse
Nøgleord
Yderligere relevante MeSH-vilkår
Andre undersøgelses-id-numre
- 24-0034
- 75N93022D00018
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