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The purpose of this study is to characterize the intestinal flora in subjects with systemic sclerosis-related constipation and/or significant bloating and to determine safety and trends in improvements in the diversity of colonic microbiome and patient symptoms following the administration of either frozen or lyophilized PRIM-DJ2727 microbiota.
The purpose of this study is to evaluate the tolerability and safety of frozen and lyophilized microbiome transplant product (PRIM- DJ2727), to characterize the microbiome (α-diversity and β-diversity) and metabolome in patients with Systemic Sclerosis (SSc)-related constipation and/or significant bloating, to examine improvement in constipation and/or significant bloating after microbiome transplant, to examine improved colonic transit after microbiome transplant, to examine subjective global improvement and improvement in SSc disease characteristics, to monitor change in concentration of systemic and fecal inflammatory markers, and to monitor for change in fecal short-chain fatty acids and metabolome.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| PRIM-DJ2727 | Experimental |
| |
| Placebo | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| PRIM-DJ2727 | Drug | For oral administration, each dose will contain the number of capsules needed to deliver the lyophilized intestinal bacteria powder derived after lyophilization of 60 g of filtered stool in 300 mL 0.85% NaCl and 2% mannitol. Participants will take the capsules (5-6 capsules twice weekly) from week 2 through week 10. Capsules will be taken over a period of up to one hour to maximize tolerance and ensure the capsules are not expelled by vomiting. For administration via retention enema, which will be done at baseline and week 11, 50 g of filtered, frozen stool in 250 mL of 0.85% sodium chloride (NaCl) will be used. |
| Measure | Description | Time Frame |
|---|---|---|
| Change in symptoms and signs of constipation and/or significant bloating as assessed by total score on the University of California, Los Angeles Gastrointestinal Tract Questionnaire, version 2.0 (UCLA GIT 2.0) | This consists of 34 questions covering domains such as diarrhea, constipation, abdominal pain, nausea, bloating, gastroesophageal reflux, fecal incontinence, and emotional well-being related to GI symptoms. Each item is scored on a Likert scale ranging from 0 to 3 (0 = none, 1 = mild, 2 = moderate, 3 = severe), with higher scores indicating greater symptom severity. Domain scores are calculated as the sum of responses to items within the domain, and a total score is calculated by summing all domain scores, representing the overall GI symptom burden. Total score will be reported, and the total score ranges from 0-102. | pre-transplant, post-treatment within weeks 11 to 12 |
| Change in Pelvic Floor Distress as Assessed by the Pelvic Floor Distress Inventory Questionnaire-20 (PFDI-20 summary score) | The Pelvic Floor Distress Inventory Questionnaire-20 (PFDI-20) is composed of 3 subscales: the Urinary Distress Inventory-6 (UDI-6), the Pelvic Organ Prolapse Distress Inventory-6 (POPDI-6), and the Colorectal-Anal Distress Inventory-8 (CRADI-8). For each question, the participant must indicate how much bowl, bladder, or pelvic symptoms have been bothering them in the past 3 months on a 4-point scale that ranges from "not at all" (0) to "quite a bit" (4). Total score for each subscale ranges from 0 to 100, with a higher score indicating worse distress. The sum of the 3 subscales are added together to get the PFDI-20 summary score, which ranges from 0 to 300, with a higher score indicating worse distress. | pre-transplant, post-treatment within weeks 11 to 12 |
| Change in Pelvic Floor Distress as Assessed by the Pelvic Floor Distress Inventory Questionnaire-20 (UDI-6 subscale score) | The Pelvic Floor Distress Inventory Questionnaire-20 (PFDI-20) is composed of 3 subscales: the Urinary Distress Inventory-6 (UDI-6), the Pelvic Organ Prolapse Distress Inventory-6 (POPDI-6), and the Colorectal-Anal Distress Inventory-8 (CRADI-8). For each question, the participant must indicate how much bowl, bladder, or pelvic symptoms have been bothering them in the past 3 months on a 4-point scale that ranges from "not at all" (0) to "quite a bit" (4). Total score for each subscale ranges from 0 to 100, with a higher score indicating worse distress. The sum of the 3 subscales are added together to get the PFDI-20 summary score, which ranges from 0 to 300, with a higher score indicating worse distress. |
| Measure | Description | Time Frame |
|---|---|---|
| Change in alpha diversity of the microbiome | Species richness and evenness within a single sample will be measured. | pre-transplant, post-treatment within weeks 11 to 12 |
| Change in beta diversity of the microbiome |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Zsuzsanna McMahan, MD, MHS | Contact | 713-500-7531 | Zsuzsanna.H.McMahan@uth.tmc.edu | |
| Ashish P Balar, MBBS, MPH | Contact | 713-500-4409 | Ashish.P.Balar@uth.tmc.edu |
| Name | Affiliation | Role |
|---|---|---|
| Zsuzsanna McMahan, MD, MHS | The University of Texas Health Science Center, Houston | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The University of Texas Health Science Center at Houston | Recruiting | Houston | Texas | 77030 | United States |
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| Placebo | Drug | Placebo will be administered as capsules from week 2-10 and as enema at baseline and week 11. |
|
| pre-transplant, post-treatment within weeks 11 to 12 |
| Change in Pelvic Floor Distress as Assessed by the Pelvic Floor Distress Inventory Questionnaire-20 (POPDI-6 subscale score) | The Pelvic Floor Distress Inventory Questionnaire-20 (PFDI-20) is composed of 3 subscales: the Urinary Distress Inventory-6 (UDI-6), the Pelvic Organ Prolapse Distress Inventory-6 (POPDI-6), and the Colorectal-Anal Distress Inventory-8 (CRADI-8). For each question, the participant must indicate how much bowl, bladder, or pelvic symptoms have been bothering them in the past 3 months on a 4-point scale that ranges from "not at all" (0) to "quite a bit" (4). Total score for each subscale ranges from 0 to 100, with a higher score indicating worse distress. The sum of the 3 subscales are added together to get the PFDI-20 summary score, which ranges from 0 to 300, with a higher score indicating worse distress. | pre-transplant, post-treatment within weeks 11 to 12 |
| Change in Pelvic Floor Distress as Assessed by the Pelvic Floor Distress Inventory Questionnaire-20 (CRADI-8 subscale score) | The Pelvic Floor Distress Inventory Questionnaire-20 (PFDI-20) is composed of 3 subscales: the Urinary Distress Inventory-6 (UDI-6), the Pelvic Organ Prolapse Distress Inventory-6 (POPDI-6), and the Colorectal-Anal Distress Inventory-8 (CRADI-8). For each question, the participant must indicate how much bowl, bladder, or pelvic symptoms have been bothering them in the past 3 months on a 4-point scale that ranges from "not at all" (0) to "quite a bit" (4). Total score for each subscale ranges from 0 to 100, with a higher score indicating worse distress. The sum of the 3 subscales are added together to get the PFDI-20 summary score, which ranges from 0 to 300, with a higher score indicating worse distress. | pre-transplant, post-treatment within weeks 11 to 12 |
| Change in symptoms and signs of constipation as assessed by score on the Patient Assessment of Constipation Symptoms (PAC-SYM) scale | The PAC-SYM is a 12-item questionnaire that assesses abdominal, rectal, and stool symptoms, with each item scored from 0 (No symptoms) to 4 (Very severe symptoms), for a total score range of 0-48, with a higher score indicating worse constipation severity. | pre-transplant, post-treatment within weeks 11 to 12 |
| Change in Gastrointestinal Symptom Severity as Assessed by the Patient Assessment of Gastrointestinal Disorders-Symptom Severity Index (PAGI-SYM) | Total score ranges from 0 (none or absent) to 5 (very severe), with a higher score indicating a worse outcome. | pre-transplant, post-treatment within weeks 11 to 12 |
| Number of participants who show an improvement in constipation and/or significant bloating symptoms as indicated by the stool diary | from baseline to end of study (week 12) |
| Change in gut transit times (assessed by Sitz markers and X-ray) | pre-transplant, post-treatment within weeks 11 to 12 |
| pre-transplant, post-treatment within weeks 11 to 12 |
| ID | Term |
|---|---|
| D003248 | Constipation |
| D012595 | Scleroderma, Systemic |
| ID | Term |
|---|---|
| D012817 | Signs and Symptoms, Digestive |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D012871 | Skin Diseases |
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