Crohns DiseasePhase 1NCT01769755
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
A Phase 1 study testing PDA001 in people with Crohns Disease, run by Celularity Incorporated at 12 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Crohns Disease
- What is being tested?
- PDA001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 14 participants
- Where
- 12 sitesUnited States
- Study dates
- Mar 2013to Nov 2014
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- • 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.
Who cannot join
- 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).
Show all 19Show fewer
- 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.
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
United States12 sites
Erlanger Baroness Hospital
Chattanooga, Tennessee, 37403
University of Cincinnati Medical Center
Cincinnati, Ohio, 45267
Case Western Reserve University Hospitals of Cleveland
Cleveland, Ohio, 44106
University of Colorado Health Science Center
Denver, Colorado, 80220-3706
University of Florida
Gainesville, Florida, 32610
Baylor College of Medicine
Houston, Texas, 77030
Cedars Sinai Medical Center, Inflammatory Bowel Disease Center
Los Angeles, California, 90048
University of Miami School of Medicine
Miami, Florida, 33136
Vanderbilt - Ingram Cancer Center
Nashville, Tennessee, 37232-5505
McGuire Veterans Affairs Medical Center
Richmond, Virginia, 23249
Rochester General Hospital
Rochester, New York, 14621
University of Utah
Salt Lake City, Utah, 84132
About this study
Official 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
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.
Read the 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.
- Conditions
- Crohns Disease
- Being tested
- PDA001, Vehicle Controlled Placebo
- Keywords
- Crohn's disease, Fistula, Diarrhea, Colon, Stem cells
- Medical subject headings
- Crohn Disease, Fistula, Diarrhea
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PDA001Biological
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.
Vehicle Controlled PlaceboDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Human Placenta-Derived Cells PDA001 Intravenous Infusion | Experimental | Intravenous infusion of Human Placenta-Derived Cells PDA001 over the course of 2 hours. Receives: Biological: PDA001 |
| Vehicle controlled placebo | Placebo comparator | Intravenous infusion of Vehicle Controlled Placebo over the course of 2 hours Receives: Drug: Vehicle Controlled Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events
Number of participants experiencing adverse events during the initial and extended follow-up periods
Measured: Up to 1 year
Secondary outcomes (2)
Clinical Response
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.
Measured: Up to 1 year
Clinical Remission
Clinical remission is defined as a Crohn's Disease Activity Index (CDAI) score CDAI score of ≤ 150 points
Measured: Up to 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jan 2013 (estimated)
- Study startMar 2013
- Primary completionNov 2014
- Study completionNov 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Crohns DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- Celularity Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Monica E Luchi, MD, Study director, Celularity Incorporated
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01769755
- Sponsor's protocol ID
- CCT-PDA001-CD-003
- Status last verified
- Aug 2015
- First submitted
- 15 Jan 2013
- Last updated
- 1 Mar 2018
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 1 Mar 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT01769755
- Acronym / sponsor ID
- CCT-PDA001-CD-003
- Conditions
- Crohns Disease
- Keywords
- Crohn's disease, Fistula, Diarrhea, Colon, Stem cells
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Celularity IncorporatedIndustry (pharma, biotech, devices)
- Study start
- Mar 2013
- Primary completion
- Nov 2014 (actual)
- Study completion
- Nov 2014 (actual)
- First posted
- 17 Jan 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Human Placenta-Derived Cells PDA001 Intravenous Infusion | Experimental | Biological: PDA001Intravenous infusion of Human Placenta-Derived Cells PDA001 over the course of 2 hours. |
| Vehicle controlled placebo | Placebo comparator | Drug: Vehicle Controlled PlaceboIntravenous infusion of Vehicle Controlled Placebo over the course of 2 hours |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- • Males and females 18 - 75 years of age at the time of signing the informed consent document.
Key exclusion criteria (19 listed)
- 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
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PDA001Cohort 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. | Biological | Human Placenta-Derived Cells PDA001 Intravenous Infusion |
| Vehicle Controlled PlaceboCohort 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. | Drug | Vehicle controlled placebo |
Primary outcome
Adverse Events
Time frame: Up to 1 year
Number of participants experiencing adverse events during the initial and extended follow-up periods
2 secondary outcomes
Clinical Response
Time frame: Up to 1 year
Clinical Remission
Time frame: Up to 1 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Mar 2018.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Monica E Luchi, MD study director
Celularity Incorporated
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing PDA001 in people with Crohns Disease, run by Celularity Incorporated at 12 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 Mar 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse Events (time frame: Up to 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Mar 2018.