Sickle Cell DiseasePhase 1NCT03298399
Mesenchymal Stromal Cells for Haplo Hematopoietic Cell Transplantation for Sickle Cell Disease
A Phase 1 study testing Autologous MSCs in people with Sickle Cell Disease, run by Emory University.
Trial summary
This trial is being conducted as a step toward testing the long-term hypothesis that freshly cultured, autologous mesenchymal stromal cells (MSCs) grown in platelet lysate-containing medium will modulate recipient T-cell immune responses and promote engraftment in haploidentical hematopoietic cell transplant (HCT)… Read more about this study
- Who is it for?
- Ages 12 to 40 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Autologous MSCs
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 21 Dec 2017to 25 Oct 2018
- 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 12 to 40 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Must weigh >25 kg at the time of study entry.
- Must have undergone puberty at the time of study entry to allow pre-transplant fertility preservation to occur, if desired. Puberty will be defined as Tanner III or more in male patients (typically age ≥ 13 years) and menarche in female patients.
- Have severe sickle cell disease (SCD) defined as 1 or more of the following:
- Clinically significant neurologic event (stroke) or any neurological deficit lasting > 24 hours;
- History of ≥2 episodes of acute chest syndrome (ACS) in the 2-year period preceding enrollment despite the institution of supportive care measures (i.e. asthma therapy and/or hydroxyurea);
- History of ≥3 severe pain crises per year in the 2-year period preceding enrollment despite the institution of supportive care measures (i.e. a pain management plan and/or treatment with hydroxyurea);
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- Administration of regular red blood cell (RBC) transfusion therapy, defined as receiving ≥8 transfusions per year for ≥1 year to prevent vaso-occlusive clinical complications (i.e. pain, stroke, and acute chest syndrome);
- An echocardiographic finding of tricuspid valve regurgitant jet (TRJ) velocity ≥2.7 m/sec in adult patients.
- Have adequate physical function as measured by:
- Lansky or Karnofsky performance score ≥60
- Cardiac function: left ventricular ejection fraction (LVEF) >40% or LV shortening fraction > 26% by cardiac echocardiogram or by multigated acquisition (MUGA) scan.
- Pulmonary function: pulse oximetry with a baseline O2 saturation of ≥90% and DLCO >40% (corrected for hemoglobin)
- Renal function: serum creatinine ≤1.5 x the upper limit of normal for age as per local laboratory and 24 hour urine creatinine clearance >70 mL/min/1.73 m2 or glomerular filtration rate (GFR) >70 mL/min/1.73 m2 by radionuclide GFR.
- Hepatic function: serum conjugated (direct) bilirubin <2x upper limit of normal for age as per local laboratory and alanine aminotransferase (ALT) and aspartate aminotransferase (AST) <5x upper limit of normal as per local laboratory. Patients with hyperbilirubinemia as a consequence of hyperhemolysis or who experience a sudden, profound change in the serum hemoglobin after a RBC transfusion are not excluded.
- In patients who have received chronic transfusion therapy for ≥1 year and who have clinical evidence of iron overload by serum ferritin or MRI, evaluation by liver biopsy is required. Histological examination of the liver must document the absence of cirrhosis, bridging fibrosis and active hepatitis. The absence of bridging fibrosis will be determined using the histological grading and staging scale as described by Ishak and colleagues (1995).
- Must be HLA typed at high resolution using DNA based typing at HLA-A, -B, -C and DRB1 and have an available related haploidentical bone marrow donor with 2, 3, or 4 (out of 8) HLA-mismatches. A unidirectional mismatch in either the graft versus host or host versus graft direction is considered a mismatch.
Who cannot join
- Availability of an 8 of 8 (HLA-A, B, C and DRB1) human leukocyte antigen (HLA) matched sibling or matched unrelated donor
- Presence of donor directed HLA antibodies.
- Severe pulmonary disease (despite above oxygen saturation and DLCO) including severe and uncontrolled asthma (per 2007 NHLBI Guidelines for the Diagnosis and Treatment of Asthma Expert Panel Report 3; http://www.nhlbi.nih.gov/health-pro/guidelines/current/asthma-guidelines/full-report), chronic obstructive pulmonary disease, and/or pulmonary hypertension (PH). A diagnosis of pulmonary hypertension (PH) will be made by finding of mean pulmonary artery pressure (mPAP) <25 mm Hg on right heart catheterization. In patients unable and/or unwilling to undergo cardiac catheterization, patients will be excluded with the following constellation of findings based upon presumptive diagnosis of PH (PPV of 62%): TRJ velocity >2.5 m/sec AND either N-terminal pro-brain natriuretic peptide (NT-pro-BNP) ≥160 pg/ml OR 6-minute walk distance <333 m.
- Uncontrolled bacterial, viral or fungal infection in the 6 week before enrollment
- Seropositivity for human immunodeficiency virus (HIV)
- Previous hematopoietic cell transplantation (HCT)
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- Participation in a clinical trial in which the patient received an investigational drug or device or the off-label use of a drug or device within 3 months of enrollment
- Demonstrated lack of compliance with prior medical care
- Unwilling to use approved contraception for at least 6 months following transplant
- Pregnant or breastfeeding females
- Allergy to any component of mesenchymal stromal cell (MSC) suspension (such as human albumin) and/or allergy to any drugs used in HCT conditioning regimen.
About this study
Official title: A Phase I Study of Mesenchymal Stromal Cells to Promote Stem Cell Engraftment in Patients With Severe Sickle Cell Disease Undergoing Haploidentical Hematopoietic Cell Transplantation
This trial is being conducted as a step toward testing the long-term hypothesis that freshly cultured, autologous mesenchymal stromal cells (MSCs) grown in platelet lysate-containing medium will modulate recipient T-cell immune responses and promote engraftment in haploidentical hematopoietic cell transplant (HCT) recipients. As a phase I, dose escalation trial of autologous MSCs in patients with sickle cell disease (SCD) undergoing haploidentical HCT, the main aim is to evaluate the safety of this therapy with a secondary aim to evaluate its effects on engraftment and graft-versus-host disease (GVHD).
Read the detailed description
This is a single center, phase I, open label dose escalation study designed to determine the safety and tolerability of autologous, bone marrow-derived MSCs (EPIC2016-MSC003) in patients with SCD undergoing haploidentical HCT.
Study participants are assigned to one of three MSC dose levels: four infusions of MSCs given once per week, four infusions given twice per week, or six infusions given twice per week. Bone marrow (1-2 ml/kg, max 60 ml) will be collected from study participants for autologous MSC expansion a minimum of 28 days prior to first planned MSC infusion. MSCs will be expanded ex vivo in human platelet lysate to the specified dose level. All MSC infusions will be dosed at 2 x 10^6 MSCs/kg recipient weight, with first infusion given on day 0 (day of haploidentical HCT) or day +1. This phase I trial will enroll 12-18 patients with severe SCD undergoing haploidentical HCT, with subjects followed for 1 year following HCT (and MSC infusions).
Prior to MSC infusions, study participants will undergo transplant conditioning and GVHD prophylaxis as follows:
Day -100 to -10: Hydroxyurea 30 mg/kg PO Qday
Day -9: Rabbit anti-thymocyte globulin (ATG) 0.5 mg/kg IV
Day -8: Rabbit ATG 2 mg/kg IV
Day -7: Rabbit ATG 2 mg/kg IV; Thiotepa 10 mg/kg IV
Day -6: Fludarabine 30 mg/m2 IV; Cyclophosphamide 14.5 mg/kg IV
Day -5: Fludarabine 30 mg/m2 IV; Cyclophosphamide 14.5 mg/kg IV
Day -4: Fludarabine 30 mg/m2 IV
Day -3: Fludarabine 30 mg/m2 IV
Day -2: Fludarabine 30 mg/m2 IV
Day -1: Total body irradiation (TBI) 200 centigray (cGy)
Day 0: Haploidentical bone marrow stem cell infusion
Day +3: Cyclophosphamide 50 mg/kg IV
Day +4: Cyclophosphamide 50 mg/kg IV
Day +5: Sirolimus (through day +365); mycophenolate mofetil (MMF) 15 mg/kg/dose three times per day (TID) (through day +35)
- Conditions
- Sickle Cell Disease
- Being tested
- Autologous MSCs
- Keywords
- Hematopoietic cell transplantation (HCT), Haploidentical, Mesenchymal stromal cells
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous MSCsBiological
Participants at dose level 1 receive four infusions of MSCs, given once per week. The first MSC infusion will be given no sooner than 4 hours after the bone marrow transplant. The total number of cells delivered to each patient will depend on their weight and assigned dose level. The maximal individual dose of MSCs any patient will receive is 2 x 10^6 cells/kg, which will be delivered via intravenous infusion. The infusion can be administered to patients in the inpatient or outpatient setting.
Autologous MSCsBiological
Participants at dose level 2 receive four infusions of MSCs, given twice per week. The first MSC infusion will be given no sooner than 4 hours after the bone marrow transplant. The total number of cells delivered to each patient will depend on their weight and assigned dose level. The maximal individual dose of MSCs any patient will receive is 2 x 10^6 cells/kg, which will be delivered via intravenous infusion. The infusion can be administered to patients in the inpatient or outpatient setting.
Autologous MSCsBiological
Participants at dose level 3 receive six infusions of MSCs, given twice per week. The first MSC infusion will be given no sooner than 4 hours after the bone marrow transplant. The total number of cells delivered to each patient will depend on their weight and assigned dose level. The maximal individual dose of MSCs any patient will receive is 2 x 10^6 cells/kg, which will be delivered via intravenous infusion. The infusion can be administered to patients in the inpatient or outpatient setting.
| Study group | Role | What they receive |
|---|---|---|
| MSC dose level 1 | Experimental | The first three subjects (minimum) will receive four weekly infusions of MSCs. Receives: Biological: Autologous MSCs |
| MSC dose level 2 | Experimental | If no significant side effects are encountered at dose level 1, then subsequent subjects will receive four infusions of MSCs given twice weekly. Receives: Biological: Autologous MSCs |
| MSC dose level 3 | Experimental | If dose level 2 is well tolerated, then subsequent subjects will receive six infusions MSCs given twice weekly. Receives: Biological: Autologous MSCs |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability of EPIC2016-MSC003 based upon dose limiting toxicities (DLTs)
DLTs will be defined as any grade ≥3 adverse reaction that is unexpected or considered attributable to the MSC infusion (attribution listed as at least probable). Because of the medical complexity of subjects on this trial and the lack of described DLTs to MSC infusion, all reported DLTs will be reviewed by the Data and Safety Monitoring Committee (DSMC).
Measured: 30 days after last MSC infusion
Secondary outcomes (11)
Primary graft rejection
Defined as the absence of donor cells assessed by peripheral blood chimerism assays on day 42.
Measured: 42 days after HCT
Late graft rejection
Defined as the absence of donor hematopoietic cells in peripheral blood beyond day 42 in a patient who had initial evidence of hematopoietic recovery with > 20% donor cells.
Measured: One year after HCT
Time to neutrophil engraftment
Defined as the first of 3 measurements on different days when the patient has an absolute neutrophil count of 500/µL after conditioning.
Measured: Up to one year after HCT
Time to platelet engraftment
Defined as the first day of a minimum of 3 measurements on different days that the patient has achieved a platelet count > 50,000/µL AND did not receive a platelet transfusion in the previous 7 days.
Measured: Up to one year after HCT
Lineage specific donor chimerism
Genomic deoxyribonucleic acid (DNA) extracted from peripheral blood will be analyzed for variable number of tandem repeats (VNTR) to detect donor engraftment in myeloid and lymphoid fractions.
Measured: Up to one year after HCT
Immune reconstitution
Immune reconstitution will be assessed post-transplant by standard clinical testing and research testing.
Measured: Up to one year after HCT
Acute GVHD
Incidence of grade II-IV and III-IV acute GVHD
Measured: One year after HCT
Chronic GVHD
Incidence and severity of chronic GVHD
Measured: One year after HCT
Transplant-related mortality (TRM)
Defined as any death occurring in continuous complete remission
Measured: One year after HCT
Event-free survival (EFS)
Defined as survival with stable donor erythropoiesis and no new clinical evidence SCD. Primary or late graft rejection with disease recurrence or death will count as events for this endpoint.
Measured: One year after HCT
Overall survival (OS)
Defined as survival with or without SCD after HCT
Measured: One year after HCT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Oct 2017
- Study start21 Dec 2017
- Primary completion25 Oct 2018
- Study completion25 Oct 2018
Why it stopped: Study stopped due to a competing trial.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by Emory UniversityThe sponsor's other registered studies.
- 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
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Principal investigator, Elizabeth Stenger, Emory University
- Investigators
- Elizabeth Stenger, MD, Principal investigator, Emory UniversityLakshmanan Krishnamurti, MD, Principal investigator, Emory University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03298399
- Sponsor's protocol ID
- IRB00090514
- Other IDs
- 1K23HL133446
- Status last verified
- Nov 2018
- First submitted
- 27 Sep 2017
- Last updated
- 7 Nov 2018
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Nov 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Study of Mesenchymal Stromal Cells to Promote Stem Cell Engraftment in Patients With Severe Sickle Cell Disease Undergoing Haploidentical Hematopoietic Cell Transplantation
- ClinicalTrials.gov ID
- NCT03298399
- Acronym / sponsor ID
- IRB00090514
- Conditions
- Sickle Cell Disease
- Keywords
- Hematopoietic cell transplantation (HCT), Haploidentical, Mesenchymal stromal cells
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Study stopped due to a competing trial.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- 21 Dec 2017 (actual)
- Primary completion
- 25 Oct 2018 (actual)
- Study completion
- 25 Oct 2018 (actual)
- First posted
- 2 Oct 2017 (actual)
- 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.
This trial is being conducted as a step toward testing the long-term hypothesis that freshly cultured, autologous mesenchymal stromal cells (MSCs) grown in platelet lysate-containing medium will modulate recipient T-cell immune responses and promote engraftment in haploidentical hematopoietic cell transplant (HCT) recipients. As a phase I, dose escalation trial of autologous MSCs in patients with sickle cell disease (SCD) undergoing haploidentical HCT, the main aim is to evaluate the safety of this therapy with a secondary aim to evaluate its effects on engraftment and graft-versus-host disease (GVHD).
Detailed description
This is a single center, phase I, open label dose escalation study designed to determine the safety and tolerability of autologous, bone marrow-derived MSCs (EPIC2016-MSC003) in patients with SCD undergoing haploidentical HCT.
Study participants are assigned to one of three MSC dose levels: four infusions of MSCs given once per week, four infusions given twice per week, or six infusions given twice per week. Bone marrow (1-2 ml/kg, max 60 ml) will be collected from study participants for autologous MSC expansion a minimum of 28 days prior to first planned MSC infusion. MSCs will be expanded ex vivo in human platelet lysate to the specified dose level. All MSC infusions will be dosed at 2 x 10^6 MSCs/kg recipient weight, with first infusion given on day 0 (day of haploidentical HCT) or day +1. This phase I trial will enroll 12-18 patients with severe SCD undergoing haploidentical HCT, with subjects followed for 1 year following HCT (and MSC infusions).
Prior to MSC infusions, study participants will undergo transplant conditioning and GVHD prophylaxis as follows:
Day -100 to -10: Hydroxyurea 30 mg/kg PO Qday
Day -9: Rabbit anti-thymocyte globulin (ATG) 0.5 mg/kg IV
Day -8: Rabbit ATG 2 mg/kg IV
Day -7: Rabbit ATG 2 mg/kg IV; Thiotepa 10 mg/kg IV
Day -6: Fludarabine 30 mg/m2 IV; Cyclophosphamide 14.5 mg/kg IV
Day -5: Fludarabine 30 mg/m2 IV; Cyclophosphamide 14.5 mg/kg IV
Day -4: Fludarabine 30 mg/m2 IV
Day -3: Fludarabine 30 mg/m2 IV
Day -2: Fludarabine 30 mg/m2 IV
Day -1: Total body irradiation (TBI) 200 centigray (cGy)
Day 0: Haploidentical bone marrow stem cell infusion
Day +3: Cyclophosphamide 50 mg/kg IV
Day +4: Cyclophosphamide 50 mg/kg IV
Day +5: Sirolimus (through day +365); mycophenolate mofetil (MMF) 15 mg/kg/dose three times per day (TID) (through day +35)
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| MSC dose level 1 | Experimental | Biological: Autologous MSCsThe first three subjects (minimum) will receive four weekly infusions of MSCs. |
| MSC dose level 2 | Experimental | Biological: Autologous MSCsIf no significant side effects are encountered at dose level 1, then subsequent subjects will receive four infusions of MSCs given twice weekly. |
| MSC dose level 3 | Experimental | Biological: Autologous MSCsIf dose level 2 is well tolerated, then subsequent subjects will receive six infusions MSCs given twice weekly. |
Who may qualify
- Ages
- 12 to 40 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (16 listed)
- Must weigh >25 kg at the time of study entry.
- Must have undergone puberty at the time of study entry to allow pre-transplant fertility preservation to occur, if desired. Puberty will be defined as Tanner III or more in male patients (typically age ≥ 13 years) and menarche in female patients.
- Have severe sickle cell disease (SCD) defined as 1 or more of the following:
- Have adequate physical function as measured by:
- Must be HLA typed at high resolution using DNA based typing at HLA-A, -B, -C and DRB1 and have an available related haploidentical bone marrow donor with 2, 3, or 4 (out of 8) HLA-mismatches. A unidirectional mismatch in either the graft versus host or host versus graft direction is considered a mismatch.
Key exclusion criteria (11 listed)
- Availability of an 8 of 8 (HLA-A, B, C and DRB1) human leukocyte antigen (HLA) matched sibling or matched unrelated donor
- Presence of donor directed HLA antibodies.
- Severe pulmonary disease (despite above oxygen saturation and DLCO) including severe and uncontrolled asthma (per 2007 NHLBI Guidelines for the Diagnosis and Treatment of Asthma Expert Panel Report 3; http://www.nhlbi.nih.gov/health-pro/guidelines/current/asthma-guidelines/full-report), chronic obstructive pulmonary disease, and/or pulmonary hypertension (PH). A diagnosis of pulmonary hypertension (PH) will be made by finding of mean pulmonary artery pressure (mPAP) <25 mm Hg on right heart catheterization. In patients unable and/or unwilling to undergo cardiac catheterization, patients will be excluded with the following constellation of findings based upon presumptive diagnosis of PH (PPV of 62%): TRJ velocity >2.5 m/sec AND either N-terminal pro-brain natriuretic peptide (NT-pro-BNP) ≥160 pg/ml OR 6-minute walk distance <333 m.
- Uncontrolled bacterial, viral or fungal infection in the 6 week before enrollment
- Seropositivity for human immunodeficiency virus (HIV)
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 |
|---|---|---|
| Autologous MSCsParticipants at dose level 1 receive four infusions of MSCs, given once per week. The first MSC infusion will be given no sooner than 4 hours after the bone marrow transplant. The total number of cells delivered to each patient will depend on their weight and assigned dose level. The maximal individual dose of MSCs any patient will receive is 2 x 10^6 cells/kg, which will be delivered via intravenous infusion. The infusion can be administered to patients in the inpatient or outpatient setting. | Biological | MSC dose level 1 |
| Autologous MSCsParticipants at dose level 2 receive four infusions of MSCs, given twice per week. The first MSC infusion will be given no sooner than 4 hours after the bone marrow transplant. The total number of cells delivered to each patient will depend on their weight and assigned dose level. The maximal individual dose of MSCs any patient will receive is 2 x 10^6 cells/kg, which will be delivered via intravenous infusion. The infusion can be administered to patients in the inpatient or outpatient setting. | Biological | MSC dose level 2 |
| Autologous MSCsParticipants at dose level 3 receive six infusions of MSCs, given twice per week. The first MSC infusion will be given no sooner than 4 hours after the bone marrow transplant. The total number of cells delivered to each patient will depend on their weight and assigned dose level. The maximal individual dose of MSCs any patient will receive is 2 x 10^6 cells/kg, which will be delivered via intravenous infusion. The infusion can be administered to patients in the inpatient or outpatient setting. | Biological | MSC dose level 3 |
Primary outcome
Safety and tolerability of EPIC2016-MSC003 based upon dose limiting toxicities (DLTs)
Time frame: 30 days after last MSC infusion
DLTs will be defined as any grade ≥3 adverse reaction that is unexpected or considered attributable to the MSC infusion (attribution listed as at least probable). Because of the medical complexity of subjects on this trial and the lack of described DLTs to MSC infusion, all reported DLTs will be reviewed by the Data and Safety Monitoring Committee (DSMC).
11 secondary outcomes
Primary graft rejection
Time frame: 42 days after HCT
Late graft rejection
Time frame: One year after HCT
Time to neutrophil engraftment
Time frame: Up to one year after HCT
Time to platelet engraftment
Time frame: Up to one year after HCT
Lineage specific donor chimerism
Time frame: Up to one year after HCT
Immune reconstitution
Time frame: Up to one year after HCT
Acute GVHD
Time frame: One year after HCT
Chronic GVHD
Time frame: One year after HCT
Transplant-related mortality (TRM)
Time frame: One year after HCT
Event-free survival (EFS)
Time frame: One year after HCT
Overall survival (OS)
Time frame: One year after HCT
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Nov 2018.
- Individual participant dataSharing planned: No
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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.
Elizabeth Stenger, MD principal investigator
Emory University
Lakshmanan Krishnamurti, MD principal investigator
Emory University
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Autologous MSCs in people with Sickle Cell Disease, run by Emory University.
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 16 inclusion and 11 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: withdrawn, as of the sponsor's last update on 7 Nov 2018. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. 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 was withdrawn before enrolling participants, so there are no results.
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?
Safety and tolerability of EPIC2016-MSC003 based upon dose limiting toxicities (DLTs) (time frame: 30 days after last MSC infusion). 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 7 Nov 2018.