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出境医 / 临床实验 / Wharton´s Jelly Derived Mesenchymal Stromal Cell Repeated Treatment of Adult Patients Diagnosed With Type I Diabetes

Wharton´s Jelly Derived Mesenchymal Stromal Cell Repeated Treatment of Adult Patients Diagnosed With Type I Diabetes

Study Description
Brief Summary:
An open label, parallel single centre trial of Wharton's Jelly derived allogenic mesenchymal stromal cells repeated treatment to preserve endogenous insulin production in adult patients diagnosed with type 1 diabetes

Condition or disease Intervention/treatment Phase
Type1diabetes Drug: ProTrans Phase 1 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open Label, Parallel Single Center Trial of Wharton's Jelly Derived Allogeneic Mesenchymal Stromal Cells Repeatedly Treated to Preserve Endogenous Insulin Production in Adult Patients Diagnosed With Type 1 Diabetes
Actual Study Start Date : May 17, 2019
Estimated Primary Completion Date : October 17, 2020
Estimated Study Completion Date : October 17, 2024
Arms and Interventions
Arm Intervention/treatment
Experimental: ProTrans-Repeat

Patients 1-3: 25 x10e6 cells Patients 4-6:100 x10e6 cells Patients 7-9:200 x10e6 cells

Control group 9 patients= non treated

 Drug: ProTrans
ProTrans is an pooled allogenic mesenchymal stromal cell treatment
Outcome Measures
Primary Outcome Measures :
  1. The primary safety endpoint in this study is; safety parameters include adverse events, hypoglycemia and allergic reactions [ Time Frame: 372 days ]
    To investigate the safety and tolerance after a repeated allogeneic infusion of Whartons Jelly Mesenchymal Stromal Cells (WJMSCs) intravenously in adult patients diagnosed with type 1 diabetes after one year following the repeated treatment.

  2. Delta-change of C-peptide AreaUnder the Curve (AUC) (0-120 min) for Mixed Meal Tolerance Test (MMTT) at day 372 following WJMSC infusion when compared to test performed before start of treatment. [ Time Frame: 372 days ]

Secondary Outcome Measures :
  1. Number of patients insulin independent (ADA criteria) at day 372. [ Time Frame: 372 days ]
  2. Number of patients with daily insulin needs <0.25U/kg at day 372. [ Time Frame: 372 days ]
  3. HbA1c at day 372 [ Time Frame: 372 days ]
  4. Glucose variability (mean amplitude of glycaemic excursions and glycaemic lability index) duration derived from the continuous glucose monitoring system® at day 372 [ Time Frame: 372 days ]
  5. Delta change of levels of fasting C-peptide at day 372 when compared to test before start of treatment [ Time Frame: 372 days ]
  6. Numbers of patients with peak C-peptide >0.20 nmol/l, in response to the MMTT, at day 372. [ Time Frame: 372 days ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years to 64 Years   (Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. A new written informed consent for participation of the study is required to be given before undergoing any study-specific procedures.
  2. Only patients that have previously been dosed by the IMP according to protocol Protrans-1 are eligible for a second dose of Protrans.
  3. No identified IMP related on-going adverse event, neither history of any adverse event that is evaluated potentially to be related to the previous IMP dosing in Protrans I.
  4. Clinical history compatible with type 1 diabetes diagnosed less than 3 years before enrolment. This also includes control patients not receiving IMP.
  5. Only male patients between 18-41 years of age will be included.
  6. Mentally stable and, in the opinion of the investigator, able to comply with the procedures of the study protocol.

Exclusion Criteria:

  1. Inability to provide informed consent
  2. Patients with body mass index (BMI) > 30, or weight >100 kg
  3. Patients with weight <50 kg
  4. Patients with unstable cardiovascular status incl. NYHA class III/IV or symptoms of angina pectoris.
  5. Patients with uncontrolled hypertension (≥160/105 mmHg).
  6. Patients with active infections unless treatment is not judged necessary by the investigators
  7. Patients with latent or previous as well as on-going therapy against tuberculosis, or exposed to tuberculosis or has travelled in areas with high risk of tuberculosis or mycosis within the last 3 months.
  8. Patients with serological evidence of infection with HIV, Treponema pallidum, hepatitis B antigen (patients with serology consistent with previous vaccination and a history of vaccination are acceptable) or hepatitis C.
  9. Patients with any immune suppressive treatment
  10. Patients with known demyelinating disease or with symptoms or physical examination findings consistent with possible demyelinating disease.
  11. Patients with known, or previous, malignancy.
  12. Taking oral anti-diabetic therapies or any other concomitant medication which may interfere with glucose regulation other than insulin
  13. Patients with GFR <80 ml/min/1.73 m2 body surface
  14. Patients with proliferative retinopathy
  15. Patient with any condition or any circumstance that in the opinion of the investigator would make it unsafe to undergo treatment with MSC.
  16. Known hypersensitivity against any excipients, i.e. dimethyl sulfoxide (DMSO).
Contacts and Locations

Contacts
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Contact: Mathias SVAHN, PhD +4687355595 mathias.svahn@nextcellpharma.com
Contact: Leo Groenewegen +4687355595 leogroenewegen@gmail.com

Locations
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Sweden
Karolinska Trial Alliance, Fas 1 enheten, Karolinska Universitetssjukhuset Huddinge Recruiting
Huddinge, Sweden
Contact: Sofia Sisay, PhD    +46(0)8-51771644    sofia.sisay@sll.se   
Sponsors and Collaborators
NextCell Pharma Ab
Investigators
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Principal Investigator: Per-Ola Carlsson, PhD Uppsala University
Tracking Information
First Submitted Date  ICMJE May 16, 2019
First Posted Date  ICMJE June 4, 2019
Last Update Posted Date June 5, 2019
Actual Study Start Date  ICMJE May 17, 2019
Estimated Primary Completion Date October 17, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 3, 2019)
  • The primary safety endpoint in this study is; safety parameters include adverse events, hypoglycemia and allergic reactions [ Time Frame: 372 days ]
    To investigate the safety and tolerance after a repeated allogeneic infusion of Whartons Jelly Mesenchymal Stromal Cells (WJMSCs) intravenously in adult patients diagnosed with type 1 diabetes after one year following the repeated treatment.
  • Delta-change of C-peptide AreaUnder the Curve (AUC) (0-120 min) for Mixed Meal Tolerance Test (MMTT) at day 372 following WJMSC infusion when compared to test performed before start of treatment. [ Time Frame: 372 days ]
Original Primary Outcome Measures  ICMJE
 (submitted: June 3, 2019)
  • The primary safety endpoint in this study is; safety parameters include adverse events, hypoglycemia and allergic reactions [ Time Frame: 372 days ]
    To investigate the safety and tolerance after a repeated allogeneic infusion of WJMSCs intravenously in adult patients diagnosed with type 1 diabetes after one year following the repeated treatment.
  • Delta-change of C-peptide AreaUnder the Curve (AUC) (0-120 min) for Mixed Meal Tolerance Test (MMTT) at day 372 following WJMSC infusion when compared to test performed before startof treatment. [ Time Frame: 372 days ]
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE
 (submitted: June 3, 2019)
  • Number of patients insulin independent (ADA criteria) at day 372. [ Time Frame: 372 days ]
  • Number of patients with daily insulin needs <0.25U/kg at day 372. [ Time Frame: 372 days ]
  • HbA1c at day 372 [ Time Frame: 372 days ]
  • Glucose variability (mean amplitude of glycaemic excursions and glycaemic lability index) duration derived from the continuous glucose monitoring system® at day 372 [ Time Frame: 372 days ]
  • Delta change of levels of fasting C-peptide at day 372 when compared to test before start of treatment [ Time Frame: 372 days ]
  • Numbers of patients with peak C-peptide >0.20 nmol/l, in response to the MMTT, at day 372. [ Time Frame: 372 days ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Wharton´s Jelly Derived Mesenchymal Stromal Cell Repeated Treatment of Adult Patients Diagnosed With Type I Diabetes
Official Title  ICMJE An Open Label, Parallel Single Center Trial of Wharton's Jelly Derived Allogeneic Mesenchymal Stromal Cells Repeatedly Treated to Preserve Endogenous Insulin Production in Adult Patients Diagnosed With Type 1 Diabetes
Brief Summary An open label, parallel single centre trial of Wharton's Jelly derived allogenic mesenchymal stromal cells repeated treatment to preserve endogenous insulin production in adult patients diagnosed with type 1 diabetes
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Type1diabetes
Intervention  ICMJE Drug: ProTrans
ProTrans is an pooled allogenic mesenchymal stromal cell treatment
Study Arms  ICMJE Experimental: ProTrans-Repeat

Patients 1-3: 25 x10e6 cells Patients 4-6:100 x10e6 cells Patients 7-9:200 x10e6 cells

Control group 9 patients= non treated

Intervention: Drug: ProTrans
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: June 3, 2019) 		18
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE October 17, 2024
Estimated Primary Completion Date October 17, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. A new written informed consent for participation of the study is required to be given before undergoing any study-specific procedures.
  2. Only patients that have previously been dosed by the IMP according to protocol Protrans-1 are eligible for a second dose of Protrans.
  3. No identified IMP related on-going adverse event, neither history of any adverse event that is evaluated potentially to be related to the previous IMP dosing in Protrans I.
  4. Clinical history compatible with type 1 diabetes diagnosed less than 3 years before enrolment. This also includes control patients not receiving IMP.
  5. Only male patients between 18-41 years of age will be included.
  6. Mentally stable and, in the opinion of the investigator, able to comply with the procedures of the study protocol.

Exclusion Criteria:

  1. Inability to provide informed consent
  2. Patients with body mass index (BMI) > 30, or weight >100 kg
  3. Patients with weight <50 kg
  4. Patients with unstable cardiovascular status incl. NYHA class III/IV or symptoms of angina pectoris.
  5. Patients with uncontrolled hypertension (≥160/105 mmHg).
  6. Patients with active infections unless treatment is not judged necessary by the investigators
  7. Patients with latent or previous as well as on-going therapy against tuberculosis, or exposed to tuberculosis or has travelled in areas with high risk of tuberculosis or mycosis within the last 3 months.
  8. Patients with serological evidence of infection with HIV, Treponema pallidum, hepatitis B antigen (patients with serology consistent with previous vaccination and a history of vaccination are acceptable) or hepatitis C.
  9. Patients with any immune suppressive treatment
  10. Patients with known demyelinating disease or with symptoms or physical examination findings consistent with possible demyelinating disease.
  11. Patients with known, or previous, malignancy.
  12. Taking oral anti-diabetic therapies or any other concomitant medication which may interfere with glucose regulation other than insulin
  13. Patients with GFR <80 ml/min/1.73 m2 body surface
  14. Patients with proliferative retinopathy
  15. Patient with any condition or any circumstance that in the opinion of the investigator would make it unsafe to undergo treatment with MSC.
  16. Known hypersensitivity against any excipients, i.e. dimethyl sulfoxide (DMSO).
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 18 Years to 64 Years   (Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Mathias SVAHN, PhD +4687355595 mathias.svahn@nextcellpharma.com
Contact: Leo Groenewegen +4687355595 leogroenewegen@gmail.com
Listed Location Countries  ICMJE Sweden
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03973827
Other Study ID Numbers  ICMJE ProTrans-Repeat
2018-004158-11 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party NextCell Pharma Ab
Study Sponsor  ICMJE NextCell Pharma Ab
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Per-Ola Carlsson, PhD Uppsala University
PRS Account NextCell Pharma Ab
Verification Date June 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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