Crohns Disease
Conditions
Keywords
Crohn's disease, Fistula, Diarrhea, Colon, Stem cells
Brief summary
To assess the safety and efficacy of intravenous (IV) PDA001 infused every two weeks for up to 5 total infusions in subjects with Crohn's disease who are refractory to one or more standard Crohn's disease therapies.
Detailed description
This is a randomized, double-blind, placebo-controlled, dose-escalation study to study 3 cohorts of subjects with Crohn's Disease including (but not limited to) those with colonic involvement. Each cohort (n = 9) will include PDA001 treated subjects (n = 6) as well as placebo (vehicle control) treated subjects (n = 3). Cohorts will be enrolled sequentially, beginning with the lowest dose cohort (1/4 unit PDA001) and progressing until the maximum tolerated dose of IV PDA001 is determined.
Interventions
BIOLOGICALPDA001
Cohort 1 Dose Level 1: ¼ unit Human Placenta-Derived Cells PDA001 infused a total of 5 times 2 weeks apart. Cohort 2 Dose Level 2: ½ unit Human Placenta-Derived Cells PDA001 infused a total of 5 times 2 weeks apart. Cohort 3 Dose Level 3: 1 unit Human Placenta-Derived Cells PDA001 infused a total of 5 times 2 weeks apart.
Cohort 1 Dose Level 1: ¼ unit vehicle controlled placebo infused a total of 5 times 2 weeks apart. Cohort 2 Dose Level 2: ½ unit vehicle controlled placebo infused a total of 5 times 2 weeks apart. Cohort 3 Dose Level 3: 1 unit vehicle controlled placebo infused a total of 5 times 2 weeks apart.
Sponsors
Celularity Incorporated
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
* • Males and females 18 - 75 years of age at the time of signing the informed consent document. * Minimum weight of subject is 40 kg at screening. * Subject must have inflammatory Crohn's Disease (CD) diagnosed at least 6 months but no greater than 15 years prior to treatment with Investigational Product (IP). * Subject must have confirmation of ongoing CD by ileocolonoscopy at screening. * Subject must have a Crohn's Disease Activity Index (CDAI) score ≥ 220 and ≤ 450 as assessed between Visit 1 and Visit 2.
Exclusion criteria
* Subject has any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from participating in the study including but not limited to * Liver Function Tests Aspartate aminotransferase (AST) or Alanine aminotransferase (ALT) \> 2.5 x the upper limit of normal at screening. * Serum creatinine concentration \> 2.0 mg/dl at screening. Alkaline phosphatase \> 2.5 x the upper limit of normal at screening. * Bilirubin level \> 2 mg/dL (unless subject has known Gilbert's disease). * Pregnant or lactating females. * Morbidly obese subjects Body Mass Index (BMI) \> 35 at screening). * Subject has untreated chronic infection including Clostridium difficile toxin positive at screening or treatment of any infection with antibiotics within 4 weeks prior to dosing with IP (other than a treated urinary tract infection or drained perianal abscess). Note: Stable doses of antibiotics used to treat Crohn's Disease are allowed. * Subject has organic heart disease (eg, congestive heart failure), clinically significant arrhythmia or clinically significant abnormal findings on Electrocardiograms (ECG). * Subject has a history of other malignancies within 5 years (except basal cell carcinoma of the skin that is surgically cured, remote history of cancer now considered cured or positive Pap smear with subsequent negative follow up). * Subject has had a stricture of the bowel requiring hospitalization within 182 days prior to treatment with IP. * Subject has had bowel surgery other than perianal (for example, fistulotomy, seton placement, or abscess drainage) or previous abscess drainage within 182 days prior to treatment with IP. * Subject has had any surgery within 28 days prior to treatment with IP. * Subject has a colostomy, ileostomy or ileal pouch anal anastomosis. * Subject has received an investigational agent -an agent or device not approved by FDA for marketed use in any indication-within 90 days (or 5 half-lives, whichever is longer) prior to treatment with investigational product. * Subject has received previous cell therapy. * Subject is expecting to have elective surgery at any time between Visit 1 (screening) and Visit 7 (end of induction phase). * Subject has concurrent diagnosis of ulcerative colitis. * Subjects with protein C or S deficiency. * Subjects with prior history of thrombophlebitis or other pathological arterial or venous thrombosis.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Adverse Events | Up to 1 year | Number of participants experiencing adverse events during the initial and extended follow-up periods |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Clinical Response | Up to 1 year | A clinical response is defined as a reduction from baseline by 25% and/or ≥ 100 points in the Crohn's Disease Activity Index (CDAI) score. The Crohn's Disease Activity Index or CDAI is a research tool used to quantify the symptoms of patients with Crohn's disease. |
| Clinical Remission | Up to 1 year | Clinical remission is defined as a Crohn's Disease Activity Index (CDAI) score CDAI score of ≤ 150 points |
Countries
United States
Outcome results
None listed