A Randomized, Double-Blind, Placebo-Controlled Dose-Escalation Study to Determine the Safety and Efficacy of Intravenous Infusion of Human Placenta-Derived Cells (PDA001) for the Treatment of Crohn's Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01769755
• Date of registration: 15/01/2013
• Prospective Registration: Yes
• Primary sponsor: Celularity Incorporated
• Public title: A Randomized, Double-Blind, Placebo-Controlled Dose-Escalation Study to Determine the Safety and Efficacy of Intravenous Infusion of Human Placenta-Derived Cells (PDA001) for the Treatment of Crohn's Disease
• Scientific title: A Randomized, Double-Blind, Placebo-Controlled Dose Escalation Study to Determine the Safety and Efficacy of Intravenous Infusion of Human Placenta-Derived Cells (PDA001) for the Treatment of Crohn's Disease
• Date of first enrolment: March 2013
• Target sample size: 14
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01769755
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Monica E Luchi, MD
• Affiliation: Celularity Incorporated

Key inclusion & exclusion criteria

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.



Age minimum: 18 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Crohns Disease

Intervention(s)

Biological: PDA001

Drug: Vehicle Controlled Placebo

Primary Outcome(s)

Adverse Events [Time Frame: Up to 1 year]

Secondary Outcome(s)

Clinical Response [Time Frame: Up to 1 year]

Clinical Remission [Time Frame: Up to 1 year]

Secondary ID(s)

CCT-PDA001-CD-003

Source(s) of Monetary Support

Please refer to primary and secondary sponsors