Rheumatoid ArthritisPhase 2NCT01261403
Study of Human Placenta-derived Cells (PDA001) to Evaluate the Safety and Effectiveness for Patients With Active Rheumatoid Arthritis
A Phase 2 study testing PDA001 in people with Rheumatoid Arthritis, run by Celularity Incorporated at 16 sites in United States.
Trial summary
The primary objective of the study is to assess the safety and efficacy of 2 dose groups (PDA001 versus vehicle control) in subjects with active rheumatoid Arthritis. The secondary objectives of the study are to determine the clinical response at defined visit intervals, determine the time to flare of RA symptoms and… Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Rheumatoid Arthritis
- 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
- 26 participants
- Where
- 16 sitesUnited States
- Study dates
- Dec 2010to Sep 2013
- Phase
- Phase 2
- 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
- Subjects must satisfy the following criteria to be enrolled in the study:
- Diagnosis of RA as defined by the 1987-revised ACR criteria.
- RA, characterized by at least 6 out of 66 swollen joints and 6 out of 68 tender joints at Screening and Baseline, and at least 2 of the following: a C reactive protein level (CRP), greater than the upper limit of normal (ULN) ³ 1 mg/dl, an erythrocyte sedimentation rate (ESR) of at least 28 mm per hour, or morning stiffness lasting longer than 45 minutes.
- Non responsive or intolerant to a minimum of 2 RA therapies which are classified as DMARDs, biologics, or corticosteroids.
- Biological medication must be discontinued 30 day prior to the first dose of study drug, except golimumab and actemra which are 60 days, and infliximab, alefacept and efalizumab, which must have been discontinued 90 days prior to the first dose of IP.
- Cytotoxic agents, including but not limited to chlorambucil, cyclophosphamide, nitrogen mustard, or other alkylating agents must have been discontinued 90 days prior to the first dose of IP.
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- Subjects must be able to tolerate intravenous infusions in both arms.
- There should be no change in RA medication dose anticipated during the initial treatment and re treatment periods and the respective 12 week follow-up period for each dosing treatment period. The following rules apply for anti-rheumatic medications taken during the study:
- DMARDs must have must have been stable for least 90 days prior to dosing with IP.
- Low -dose corticosteroids are permitted (prednisolone ≤ 10 mg per day or equivalent). Corticosteroids must have been stable for at least 30 days prior to dosing with IP.
- DMARDs and corticosteroids must remain stable throughout the initial 3 months of study and throughout subsequent treatment periods
- The presence of any of the following will exclude a subject from enrollment:
- Prior use of rituximab and other B-cell depleting therapies, abatacept, prior use of more than 2 biologic therapies.
- Subject has received an investigational agent in any indication-within 60 days (or 5 half-lives, whichever is longer) prior to treatment with IP.
- Subject has received previous cell therapy.
- Serum creatinine concentration > 2.0 mg/dl at screening.
- Alkaline phosphatase > 2.5x the upper limit of normal at screening.
- Bilirubin level > 1.5 mg/dL (unless subject has known Gilbert's disease).
- Untreated chronic infection or treatment of any infection with antibiotics within 4 weeks prior to dosing with IP.
- Positive HIV test at Screening. Positive Hepatitis B surface antigen at Screening. Positive Hepatitis C antibody at Screening.
- Organic heart disease (e.g., congestive heart failure), myocardial infarction within six (6) months prior to screening or clinically significant findings on ECG at screening. Clinically significant arrhythmia.
- Primary or secondary immunodeficiency.
Who cannot join
None listed.
Trial locations and contacts
16 sites in 1 country.
16 sites in 1 country
United States16 sites
Pinnacle Research Group
Anniston, Alabama, 36207
Advanced Pain Research Institute
Arcadia, California, 91007
Sanitas Research
Coral Gables, Florida, 33134
Metroplex Clinical Research Center
Dallas, Texas, 75231
Altoona Center for Clinical Research
Duncansville, Pennsylvania, 16635
St. Paul Rheumatology, PA
Eagan, Minnesota, 55121
SNS Rheumatology
Lakewood, New Jersey, 08701
UCLA
Los Angeles, California, 90095
David R. Mandel, MD, Inc.
Mayfield, Ohio, 44143
Arthritis and Diabetes Clinic, Inc
Monroe, Louisiana, 71203
Health Research of Oklahoma
Oklahoma City, Oklahoma, 73103
Health Research Institute
Oklahoma City, Oklahoma, 73109
Compass Research, LLC
Orlando, Florida, 32806
Four Rivers Clinical Research Inc.
Paducah, Kentucky, 42003
Desert Medical Advances
Palm Desert, California, 92260
Progressive Medical Research
Port Orange, Florida, 32127
About this study
Official title: A Phase 2, Randomized, Double- Blind, Placebo-controlled, Multi-center Study to Evaluate the Safety and Efficacy of Intravenous Infusion of Human Placenta-Derived Cells (PDA001) for the Treatment of Adults With Active Rheumatoid Arthritis
The primary objective of the study is to assess the safety and efficacy of 2 dose groups (PDA001 versus vehicle control) in subjects with active rheumatoid Arthritis. The secondary objectives of the study are to determine the clinical response at defined visit intervals, determine the time to flare of RA symptoms and to quantify changes in inflammatory markers including C-reactive protein (CRP), erythrocyte sedimentation rate (ESR), serum amyloid A (SAA), and IL-6.
- Conditions
- Rheumatoid Arthritis
- Being tested
- PDA001, Vehicle Controlled Placebo
- Keywords
- Human Placenta-Derived Cells
- Medical subject headings
- Arthritis, Rheumatoid
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PDA001Biological
Dose escalation study: Subjects will be assigned to 1 of 2 treatment groups (1 unit or 4 units vs. vehicle control) based on the order in which they enroll in the study. Intravenous infusion will be administered on days 0 and 7. Nonresponders will be unblinded after 12 weeks of study. Non- responders on vehicle control will be re-dosed with the active PDA001 dose assigned at baseline (2 infusions, 7 days apart). Nonresponders taking active PDA001 will enter the safety follow-up portion of the study. Responders at 12 weeks will continue in the safety and efficacy follow-up portion of the study until 12 months of study. Responders will be treated for RA flare between 3-9 months of study with the active PDA001 dose assigned at baseline (2 infusions, 7 days apart).
Vehicle Controlled PlaceboDrug
Cohort Dose Level 1: 4 units vehicle controlled placebp infused on Day 0 and Day 7
Cohort Dose Level 2: 4 units vehicle controlled placebo infused on Day 0 and Day 7
| Study group | Role | What they receive |
|---|---|---|
| Group 1 | Experimental | 1 Unit of PDA001 or 4 units Vehicle Control intravenous on Day 0 and Day 7 Receives: Biological: PDA001; Drug: Vehicle Controlled Placebo |
| Group 2 | Experimental | 4 Units PDA001 or 4 units Vehicle Control intravenous (Placebo) on Day 0 and Day 7 Receives: Biological: PDA001; Drug: Vehicle Controlled Placebo |
| Vehicle control | Placebo comparator | Placebo - Vehicle Control Arm Receives: Drug: Vehicle Controlled Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number/Percentage of Participants With an American College of Rheumatology 20% (ACR20) Response within 12 weeks following the initial dose of study drug
A participant is a responder if the following 3 criteria for improvement from Baseline were met: • ≥ 20% improvement in 68 tender joint count; • ≥ 20% improvement in 66 swollen joint count; and • ≥ 20% improvement in at least 3 of the 5 following parameters: o Patient's assessment of pain (measured on a 100 mm visual analog scale [VAS]) o Patient's global assessment of disease activity (measured on a 100 mm VAS) o Physician's global assessment of disease activity (measured on a 100 mm VAS) o Patient's self-assessment of physical function (Health Assessment Questionnaire - Disability Index (HAQ-DI)) o C-Reactive Protein
Measured: Baseline through Week 12
Secondary outcomes (21)
Number/Percentage of Participants With an American College of Rheumatology 20% (ACR20) Response at 6 months
A participant is a responder if the following 3 criteria for improvement from Baseline were met: • ≥ 20% improvement in 68 tender joint count; • ≥ 20% improvement in 66 swollen joint count; and • ≥ 20% improvement in at least 3 of the 5 following parameters: o Patient's assessment of pain (measured on a 100 mm visual analog scale [VAS]) o Patient's global assessment of disease activity (measured on a 100 mm VAS) o Physician's global assessment of disease activity (measured on a 100 mm VAS) o Patient's self-assessment of physical function (Health Assessment Questionnaire - Disability Index (HAQ-DI)) o C-Reactive Protein
Measured: Baseline and 6 months
Number/percent of subjects achieving an ACR 20 clinical response at each scheduled visit during the first 12 months following the first infusion of study drug.
A participant is an ACR 20 responder if the following 3 criteria for improvement from Baseline were met: • ≥ 20% improvement in 68 tender joint count; • ≥ 20% improvement in 66 swollen joint count; and • ≥ 20% improvement in at least 3 of the 5 following parameters: o Patient's assessment of pain (measured on a 100 mm visual analog scale [VAS]) o Patient's global assessment of disease activity (measured on a 100 mm VAS) o Physician's global assessment of disease activity (measured on a 100 mm VAS) o Patient's self-assessment of physical function (Health Assessment Questionnaire - Disability Index (HAQ-DI)) o C-Reactive Protein
Measured: Baseline and each scheduled visit through 12 months following the first infusion of study drug
Number/percent of subjects achieving an ACR 50 clinical response at each scheduled visit during the first 12 months following the first infusion of study drug.
A participant is an ACR responder if the following 3 criteria for improvement from Baseline were met: • ≥ 50% improvement in 68 tender joint count; • ≥ 50% improvement in 66 swollen joint count; and • ≥ 50% improvement in at least 3 of the 5 following parameters: o Patient's assessment of pain (measured on a 100 mm visual analog scale [VAS]) o Patient's global assessment of disease activity (measured on a 100 mm VAS) o Physician's global assessment of disease activity (measured on a 100 mm VAS) o Patient's self-assessment of physical function (Health Assessment Questionnaire - Disability Index (HAQ-DI)) o C-Reactive Protein
Measured: Baseline and each scheduled visit through 12 months following the first infusion of study drug.
Number/percent of subjects achieving an ACR 70 clinical response at each scheduled visit during the first 12 months following the first infusion of study drug.
A participant is an ACR 70 responder if the following 3 criteria for improvement from Baseline were met: • ≥ 70% improvement in 68 tender joint count; • ≥ 70% improvement in 66 swollen joint count; and • ≥ 70% improvement in at least 3 of the 5 following parameters: o Patient's assessment of pain (measured on a 100 mm visual analog scale [VAS]) o Patient's global assessment of disease activity (measured on a 100 mm VAS) o Physician's global assessment of disease activity (measured on a 100 mm VAS) o Patient's self-assessment of physical function (Health Assessment Questionnaire - Disability Index (HAQ-DI)) o C-Reactive Protein
Measured: Baseline and each scheduled visit through 12 months following the first infusion of study drug.
Number/percent of subjects achieving a clinical response based on their Clinical Disease Activity Index (CDAI) at each scheduled visit during the first 12 months following the first infusion of study drug.
A subject is considered a responder based on the Clinical Disease Activity Index (CDAI): A composite index where CDAI = SJC+TJC+SGA+PGA. SJC and TJC are the numbers of swollen and tender joints based on 28-joint counts; SGA and PGA represent the subject's and physician's global assessment of disease activity. The CDAI score ranges from 0-76. Clinical disease activity is defined according to CDAI scores where Remission is ≤ 2.8, Low Disease Activity > 2.8 and ≤ 10, Moderate Disease Activity > 10 and ≤ 22, and High Disease Activity > 22
Measured: Baseline and each scheduled visit through 12 months following the first infusion of study drug
Number/percent of subjects achieving a clinical response based on their Disease Activity Score (DAS 28) score at each scheduled visit during the first 12 months following the first infusion of study drug
A subject is considered a responder based on Disease Activity Score 28 (DAS 28): A measure of severity of disease derived from the following: 28 tender joint count (TJC28); 28 swollen joint count (SJC28), which do not include the distal interphalangeal (DIP) joints, the hip joint, or the joints below the knee, CRP, and SGA. A DAS 28 score > 5.1 indicates high disease activity, a DAS28 score < 3.2 indicates low disease activity, and a DAS28 score < 2.6 indicates clinical remission
Measured: Baseline and each scheduled visit through 12 months following the first infusion of study drug
Number/Percent of Participants Who Achieve the European League Against Rheumatism (EULAR) Response Criteria Using CRP at Month 12
A subject is considered a responder based on EULAR response: The EULAR response criteria classify each subject as a good, moderate or non-responder to treatment based on the degree of improvement from baseline and the level of disease activity at the endpoint. EULAR response is derived using the individual subject's DAS28 as the measure of severity of disease. Good or moderate response is defined as follows: Good response: DAS28 at the time point ≤ 3.2 and improvement from baseline > 1.2 Moderate response: DAS28 at the time point > 3.2 and improvement from baseline > 1.2, or DAS28 at the time point ≤ 5.1 and improvement from baseline > 0.6 and ≤ 1.2
Measured: Baseline and each scheduled visit through 12 months following the first infusion of study drug.
The change from baseline in the ACR core components. ACR Core Component #1: Percentage Change From Baseline in the Swollen Joint Count at each scheduled visit.
Joint tenderness is the presence of pain in a joint when pressure is applied by the examiner to elicit tenderness. The 68 tender joint count evaluates the following joints: upper-temporomandibular, sternoclavicular, acromioclavicular, shoulder, elbow, wrist, meta-carpophalangeal, proximal interphalangeal and distal interphalangeal; lower: hip, knee, ankle, midtarsal, metatarsophalangeal and proximal interphalangeal.
Measured: Baseline and each scheduled visit through 12 months following the first infusion of study drug
ACR Core Component #2: Percentage Change From Baseline in the Swollen Joint Count at each scheduled visit through Month 12 following the initial infusion of study drug
Joint swelling is soft tissue swelling that is detectable along the joint margins and is assessed by inspection and direct palpation of the joint, by the examiner. The ACR 66 swollen joint count evaluates the following joints: upper-temporomandibular, sternoclavicular, acromioclavicular, shoulder, elbow, wrist, metacarpophalangeal, proximal interphalangeal and distal interphalangeal; lower: knee, ankle, midtarsal, metatarsophalangeal and proximal interphalangeal.
Measured: Baseline and at each scheduled visit through Month 12 following the initial infusion of study drug
ACR Core Component #3 Percentage Change From Baseline in the Subject Assessment of Pain at each scheduled visit through Month 12 following the initial dose of study drug
The Subject Assessment of Pain was measured asking the participant to place a vertical line on a 100-mm visual analog scale on which the left-hand boundary (score = 0 mm) represents "no pain," and the right-hand boundary (score = 100 mm) represents "pain as severe as can be imagined." The distance from the mark to the left-hand boundary was recorded in millimeters.
Measured: Baseline and at each scheduled visit through Month 12 following the initial dose of study drug
ACR component #4 Percentage Change From Baseline in the Subject Global Assessment of Disease Activity at each scheduled visit through Month 12 following the initial dose of study drug
The Subject Global Assessment of Disease Activity was measured asking the participant to place a vertical line on a 100-mm visual analog scale on which the left-hand boundary (score = 0 mm) represents " lowest disease activity," and the right-hand boundary (score = 100 mm) represents " highest disease activity." The distance from the mark to the left-hand boundary was recorded in millimeters.
Measured: Baseline and at each scheduled visit through Month 12 following the initial dose of study drug
ACR Component #5 Percentage Change From Baseline in the Physician Global Assessment of Disease Activity at each scheduled visit through Month 12 following the initial dose of study drug
The Physician Global Assessment of Disease Activity was measured asking the physician to assess the subject's current arthritis disease activity by placing a vertical line on a 100-mm visual analog scale on which the left-hand boundary (score = 0 mm) represents " lowest disease activity," and the right-hand boundary (score = 100 mm) represents " highest disease activity." The distance from the mark to the left-hand boundary was recorded in millimeters
Measured: Baseline and Month 12
ACR Component #6; Percentage Change From Baseline in the Health Assessment Questionnaire-Disability Index (HAQ-DI) Score at each scheduled visit through Month 12 following the initial dose of study drug
The Health Assessment Questionnaire - Disability Index (HAQ-DI) was a patient-reported questionnaire consisting of 20 questions referring to eight domains: dressing/grooming, arising, eating, walking, hygiene, reach, grip, and usual activities. Participants assessed their ability to do each task over the past week using the following response categories: without any difficulty (0); with some difficulty (1); with much difficulty (2); and unable to do (3). Scores on each task are summed and averaged to provide an overall score ranging from 0 to 3, where zero represents no disability and three very severe, high-dependency disability. Negative mean changes from baseline in the overall score indicate improvement in functional ability.
Measured: Baseline and at each scheduled visit through Month 12 following the initial dose of study drug
ACR Component#7 Percentage Change From Baseline in the High Sensitivity C-Reactive Protein (CRP) at each scheduled visit through Month 12 following the initial dose of study drug
C-Reactive Protein (CRP) is a substance produced by the liver that increases in the presence of inflammation in the body. An elevated CRP level is identified with blood tests and is considered a non-specific "marker" for disease
Measured: Baseline at each scheduled visit through Month 12 following the initial dose of study drug
ACR Component #8 Percentage Change From Baseline in Erythrocyte Sedimentation Rate (ESR) at each scheduled visit through Month 12 following the initial dose of study drug
The erythrocyte sedimentation rate (ESR) is a blood test that can reveal inflammatory activity. The subject's blood is placed in a tall, thin tube, red erythrocytes gradually settle to the bottom. Inflammation can cause the cells to clump together. Because these clumps of cells are denser than individual cells, they settle to the bottom more quickly. The ESR test measures the distance red blood cells fall in a test tube in one hour. The farther the red blood cells have descended, the greater the inflammatory response
Measured: Baseline and at each scheduled visit through Month 12 following the initial dose of study drug
Change from baseline in rheumatoid factors at each scheduled visit through Month 12 following the initial dose of study drug
Rheumatoid factor is an antibody that is measurable in the blood. Rheumatoid factor is actually an antibody that can bind to other antibodies. High levels of rheumatoid factor can indicate a tendency toward more aggressive disease and/or a tendency to develop rheumatoid nodules and rheumatoid lung disease.
Measured: Baseline and each scheduled visit through Month 12 following the initial dose of study drug
Time to flare of Rheumatoid Arthritis (RA) symptoms
A flare is defined as a 20% increase in both tender and swollen joint counts, an increase in Physician Global Assessment of Disease ≥ 20 mm and an increase in at least 1 of 2 criteria (Patient Global Assessment of Disease ≥ 20 mm, Patient Assessment of Pain ≥ 20 mm) when compared with corresponding values from the subject's best response. Best response is determined by the subject's lowest CDAI score
Measured: At defined intervals
Changes in inflammatory markers including C-reactive protein (CRP), erythrocyte sedimentation rate (ESR), serum amyloid A (SAA), and IL-6
Inflammatory markers are certain laboratory tests performed using the subjects' serum that indicate the presence of RA. Changes in these test results may indicate an improvement in RA
Measured: At defined intervals
Change From Baseline in the Disease Activity Score 28 (DAS 28) Using CRP
The DAS28 measures the severity of disease at a specific time and is derived from the following variables: • 28 tender joint count (TJC28) • 28 swollen joint count (SJC28), which do not include the distal interphalangeal (DIP) joints, the hip joint, or the joints below the knee; • C-reactive protein (CRP) • Subject's global assessment of disease activity (SGA). A DAS28 score higher than 5.1 indicates high disease activity, a DAS28 score less than 3.2 indicates low disease activity, and a DAS28 score less than 2.6 indicates clinical remission. Remission: ≤ 2.8; Low Disease Activity: > 2.8 and ≤ 10; Moderate Disease Activity: > 10 and ≤ 22; High Disease Activity: > 22
Measured: Baseline and Month 6
Change From Baseline in the Clinical Disease Activity Index (CDAI) at Month 3
Clinical Disease Activity Index (CDAI): A simplified composite index as defined by: CDAI = SJC + TJC + SGA + PGA) • SJC and TJC are the numbers of swollen and tender joints based on 28-joint counts. • SGA and PGA represent the subject's and physician's global assessment of disease activity. o The CDAI score ranges from 0-76. Clinical disease activity is categorized according to CDAI scores where Remission ≤ 2.8, Low Disease Activity > 2.8 and ≤ 10, Moderate Disease Activity > 10 and ≤ 22, and High Disease Activity > 22
Measured: Baseline and Month 3
Change From Baseline in the Clinical Disease Activity Index (CDAI) at Month 6
Clinical Disease Activity Index (CDAI): A simplified composite index as defined by: CDAI = SJC + TJC + SGA + PGA) • SJC and TJC are the numbers of swollen and tender joints based on 28-joint counts. • SGA and PGA represent the subject's and physician's global assessment of disease activity. o The CDAI score ranges from 0-76. Clinical disease activity is categorized according to CDAI scores where Remission ≤ 2.8, Low Disease Activity > 2.8 and ≤ 10, Moderate Disease Activity > 10 and ≤ 22, and High Disease Activity > 22
Measured: Baseline and Month 6
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Dec 2010 (estimated)
- Study startDec 2010
- Primary completionFeb 2012
- Study completionSep 2013
Why it stopped: Enrollment terminated pending additional Phase 1 data
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Rheumatoid ArthritisStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- Celularity Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Celgene Corporation
- Responsible party
- Sponsor
- Investigators
- Solveig Ericson, MD, Study director, Celularity Incorporated
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01261403
- Sponsor's protocol ID
- CCT-PDA001-RA-001
- Status last verified
- Jul 2014
- First submitted
- 15 Dec 2010
- Last updated
- 22 Jul 2020
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 22 Jul 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Randomized, Double- Blind, Placebo-controlled, Multi-center Study to Evaluate the Safety and Efficacy of Intravenous Infusion of Human Placenta-Derived Cells (PDA001) for the Treatment of Adults With Active Rheumatoid Arthritis
- ClinicalTrials.gov ID
- NCT01261403
- Acronym / sponsor ID
- CCT-PDA001-RA-001
- Conditions
- Rheumatoid Arthritis
- Keywords
- Human Placenta-Derived Cells
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Enrollment terminated pending additional Phase 1 data
- Enrolment
- 26 participants (actual)
- Lead sponsor
- Celularity IncorporatedIndustry (pharma, biotech, devices)
- Collaborators
- Celgene Corporation
- Study start
- Dec 2010
- Primary completion
- Feb 2012 (actual)
- Study completion
- Sep 2013 (actual)
- First posted
- 16 Dec 2010 (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.
The primary objective of the study is to assess the safety and efficacy of 2 dose groups (PDA001 versus vehicle control) in subjects with active rheumatoid Arthritis. The secondary objectives of the study are to determine the clinical response at defined visit intervals, determine the time to flare of RA symptoms and to quantify changes in inflammatory markers including C-reactive protein (CRP), erythrocyte sedimentation rate (ESR), serum amyloid A (SAA), and IL-6.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Group 1 | Experimental | Biological: PDA001, Drug: Vehicle Controlled Placebo1 Unit of PDA001 or 4 units Vehicle Control intravenous on Day 0 and Day 7 |
| Group 2 | Experimental | Biological: PDA001, Drug: Vehicle Controlled Placebo4 Units PDA001 or 4 units Vehicle Control intravenous (Placebo) on Day 0 and Day 7 |
| Vehicle control | Placebo comparator | Drug: Vehicle Controlled PlaceboPlacebo - Vehicle Control Arm |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (22 listed)
- Diagnosis of RA as defined by the 1987-revised ACR criteria.
- RA, characterized by at least 6 out of 66 swollen joints and 6 out of 68 tender joints at Screening and Baseline, and at least 2 of the following: a C reactive protein level (CRP), greater than the upper limit of normal (ULN) ³ 1 mg/dl, an erythrocyte sedimentation rate (ESR) of at least 28 mm per hour, or morning stiffness lasting longer than 45 minutes.
- Non responsive or intolerant to a minimum of 2 RA therapies which are classified as DMARDs, biologics, or corticosteroids.
- Biological medication must be discontinued 30 day prior to the first dose of study drug, except golimumab and actemra which are 60 days, and infliximab, alefacept and efalizumab, which must have been discontinued 90 days prior to the first dose of IP.
- Cytotoxic agents, including but not limited to chlorambucil, cyclophosphamide, nitrogen mustard, or other alkylating agents must have been discontinued 90 days prior to the first dose of IP.
Key exclusion criteria (0 listed)
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 |
|---|---|---|
| PDA001Dose escalation study: Subjects will be assigned to 1 of 2 treatment groups (1 unit or 4 units vs. vehicle control) based on the order in which they enroll in the study. Intravenous infusion will be administered on days 0 and 7. Nonresponders will be unblinded after 12 weeks of study. Non- responders on vehicle control will be re-dosed with the active PDA001 dose assigned at baseline (2 infusions, 7 days apart). Nonresponders taking active PDA001 will enter the safety follow-up portion of the study. Responders at 12 weeks will continue in the safety and efficacy follow-up portion of the study until 12 months of study. Responders will be treated for RA flare between 3-9 months of study with the active PDA001 dose assigned at baseline (2 infusions, 7 days apart). | Biological | Group 1, Group 2 |
| Vehicle Controlled PlaceboCohort Dose Level 1: 4 units vehicle controlled placebp infused on Day 0 and Day 7 Cohort Dose Level 2: 4 units vehicle controlled placebo infused on Day 0 and Day 7 | Drug | Group 1, Group 2, Vehicle control |
Primary outcome
Number/Percentage of Participants With an American College of Rheumatology 20% (ACR20) Response within 12 weeks following the initial dose of study drug
Time frame: Baseline through Week 12
A participant is a responder if the following 3 criteria for improvement from Baseline were met: • ≥ 20% improvement in 68 tender joint count; • ≥ 20% improvement in 66 swollen joint count; and • ≥ 20% improvement in at least 3 of the 5 following parameters: o Patient's assessment of pain (measured on a 100 mm visual analog scale [VAS]) o Patient's global assessment of disease activity (measured on a 100 mm VAS) o Physician's global assessment of disease activity (measured on a 100 mm VAS) o Patient's self-assessment of physical function (Health Assessment Questionnaire - Disability Index (HAQ-DI)) o C-Reactive Protein
21 secondary outcomes
Number/Percentage of Participants With an American College of Rheumatology 20% (ACR20) Response at 6 months
Time frame: Baseline and 6 months
Number/percent of subjects achieving an ACR 20 clinical response at each scheduled visit during the first 12 months following the first infusion of study drug.
Time frame: Baseline and each scheduled visit through 12 months following the first infusion of study drug
Number/percent of subjects achieving an ACR 50 clinical response at each scheduled visit during the first 12 months following the first infusion of study drug.
Time frame: Baseline and each scheduled visit through 12 months following the first infusion of study drug.
Number/percent of subjects achieving an ACR 70 clinical response at each scheduled visit during the first 12 months following the first infusion of study drug.
Time frame: Baseline and each scheduled visit through 12 months following the first infusion of study drug.
Number/percent of subjects achieving a clinical response based on their Clinical Disease Activity Index (CDAI) at each scheduled visit during the first 12 months following the first infusion of study drug.
Time frame: Baseline and each scheduled visit through 12 months following the first infusion of study drug
Number/percent of subjects achieving a clinical response based on their Disease Activity Score (DAS 28) score at each scheduled visit during the first 12 months following the first infusion of study drug
Time frame: Baseline and each scheduled visit through 12 months following the first infusion of study drug
Number/Percent of Participants Who Achieve the European League Against Rheumatism (EULAR) Response Criteria Using CRP at Month 12
Time frame: Baseline and each scheduled visit through 12 months following the first infusion of study drug.
The change from baseline in the ACR core components. ACR Core Component #1: Percentage Change From Baseline in the Swollen Joint Count at each scheduled visit.
Time frame: Baseline and each scheduled visit through 12 months following the first infusion of study drug
ACR Core Component #2: Percentage Change From Baseline in the Swollen Joint Count at each scheduled visit through Month 12 following the initial infusion of study drug
Time frame: Baseline and at each scheduled visit through Month 12 following the initial infusion of study drug
ACR Core Component #3 Percentage Change From Baseline in the Subject Assessment of Pain at each scheduled visit through Month 12 following the initial dose of study drug
Time frame: Baseline and at each scheduled visit through Month 12 following the initial dose of study drug
ACR component #4 Percentage Change From Baseline in the Subject Global Assessment of Disease Activity at each scheduled visit through Month 12 following the initial dose of study drug
Time frame: Baseline and at each scheduled visit through Month 12 following the initial dose of study drug
ACR Component #5 Percentage Change From Baseline in the Physician Global Assessment of Disease Activity at each scheduled visit through Month 12 following the initial dose of study drug
Time frame: Baseline and Month 12
ACR Component #6; Percentage Change From Baseline in the Health Assessment Questionnaire-Disability Index (HAQ-DI) Score at each scheduled visit through Month 12 following the initial dose of study drug
Time frame: Baseline and at each scheduled visit through Month 12 following the initial dose of study drug
ACR Component#7 Percentage Change From Baseline in the High Sensitivity C-Reactive Protein (CRP) at each scheduled visit through Month 12 following the initial dose of study drug
Time frame: Baseline at each scheduled visit through Month 12 following the initial dose of study drug
ACR Component #8 Percentage Change From Baseline in Erythrocyte Sedimentation Rate (ESR) at each scheduled visit through Month 12 following the initial dose of study drug
Time frame: Baseline and at each scheduled visit through Month 12 following the initial dose of study drug
Change from baseline in rheumatoid factors at each scheduled visit through Month 12 following the initial dose of study drug
Time frame: Baseline and each scheduled visit through Month 12 following the initial dose of study drug
Time to flare of Rheumatoid Arthritis (RA) symptoms
Time frame: At defined intervals
Changes in inflammatory markers including C-reactive protein (CRP), erythrocyte sedimentation rate (ESR), serum amyloid A (SAA), and IL-6
Time frame: At defined intervals
Change From Baseline in the Disease Activity Score 28 (DAS 28) Using CRP
Time frame: Baseline and Month 6
Change From Baseline in the Clinical Disease Activity Index (CDAI) at Month 3
Time frame: Baseline and Month 3
Change From Baseline in the Clinical Disease Activity Index (CDAI) at Month 6
Time frame: Baseline and Month 6
Results and safety
The study was stopped early (reason given: Enrollment terminated pending additional Phase 1 data). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jul 2020.
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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.
Solveig Ericson, MD study director
Celularity Incorporated
16 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing PDA001 in people with Rheumatoid Arthritis, run by Celularity Incorporated at 16 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 22 inclusion and 0 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: stopped early, as of the sponsor's last update on 22 Jul 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 was stopped early (reason given: Enrollment terminated pending additional Phase 1 data). Results are not available in the source record.
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?
Number/Percentage of Participants With an American College of Rheumatology 20% (ACR20) Response within 12 weeks following the initial dose of study drug (time frame: Baseline through Week 12). 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 22 Jul 2020.