NEPHSTROM for Diabetic Kidney Disease

NCT ID: NCT02585622

Last Updated: 2024-11-29

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

29 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-12-11

Study Completion Date

2024-01-03

Brief Summary

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The study will investigate, primarily, the safety, feasibility and tolerability and, secondarily, the preliminary efficacy of an allogeneic bone marrow-derived Mesenchymal Stromal Cell (MSC) therapy (ORBCEL-M) in study subjects with type 2 diabetes (T2D) and progressive diabetic kidney disease (DKD).

Detailed Description

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Conditions

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Diabetic Kidney Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

OTHER

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Bone marrow-derived Mesenchymal Stromal Cells

Group Type EXPERIMENTAL

Mesenchymal Stromal Cells

Intervention Type BIOLOGICAL

Cells will be administered intravenously at 3 different doses (80, 160, or 240 x 10\^6, fixed dose) over 10-20 minutes. Volume total of fluid infused: 40 ml

Cryostor CS10

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

Volume total of fluid infused: 40 ml

Interventions

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Mesenchymal Stromal Cells

Cells will be administered intravenously at 3 different doses (80, 160, or 240 x 10\^6, fixed dose) over 10-20 minutes. Volume total of fluid infused: 40 ml

Intervention Type BIOLOGICAL

Placebo

Volume total of fluid infused: 40 ml

Intervention Type OTHER

Other Intervention Names

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Allogeneic Cellular Therapy (NEPHSTROM ORBCEL-M) Cryostor CS10

Eligibility Criteria

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Inclusion Criteria

* Male and female ≥ 40 years and \<85 years old. ;
* T2D for 3 or more years under a clinician with mandated responsibility for management of the patients to national guidelines;
* Urinary albumin excretion (UAE) ≥ 60 µg/min (in a 24 hour urine collection) and urine albumin-to-creatinine ratio (UACR) ≥ 88 mg/g (≥ 10 mg/mmol) (in a spot morning urine collection);
* Estimated GFR (eGFR) 30-50 ml/min/1.73 m\^2 by the CKD-EPI equation on 2 or more consecutive measurements at least 30 days apart within the past 6 months;
* A documented decline of eGFR of ≥ -10ml/min/1.73 m\^2 over the past 3 years or documented rate of eGFR decline of ≥ -5 ml/min/1.73 m\^2 year based on 3 or more consecutive readings at least 90 days apart in the past 18 months;
* Lack of suspicion of renal diagnosis other than DKD;
* Willing and able to provide written informed consent.

Exclusion Criteria

1. Current resting systolic BP ≥ 150 mmHg and current resting diastolic BP ≥ 90 mmHg in a clinical setting, despite treatment with 3 hypertensive agents of different classes (including one diuretic), measured in a quiet environment with morning medications already taken;
2. Initiation of a new anti-hypertensive agent within the past 6 months
3. Increase the dose of an anti-hypertensive agent by ≥ 100% of the previous dose within the past 3 months

4. Current HbA1c \> 75 mmol/mol (\> 9%)
5. Initiation of a new hypoglycaemic agent within the past 6 months
6. Increase the dose of a hypoglycaemic agent by ≥ 100% of the previous dose within the past 3 months

7. Current fasting total cholesterol \> 7 mmol/l
8. Current fasting total triglycerides \> 3.5 mmol/l
9. Initiation of a new lipid lowering agent within the past 6 months

10. Chronic lung or liver disease;
11. Cardiovascular events (myocardial infarction, stroke or acute limb ischemia) within 6 months prior to enrolment;
12. Current or history within 6 months prior to enrolment of NYHA class III or IV heart failure;
13. Other concomitant disease or conditions in the opinion of the investigator that are likely to pose risk to the patient and that would render the patient unsuitable for participation or that could impair patient safety or ability to participate in the study, such as active malignancy;
14. Irreversible disease or condition for which 6-month mortality is estimated to be greater than 50%;
15. Positive screening test for clinically significant anti-HLA antibodies. An initial antibody screening with Luminex® multi-antigen beads to detect class I and class II MHC antibodies followed by a Luminex single antigen bead assay to determine the specificity of any antibody detected. Potential study subjects with positive screening for any clinically significant anti-HLA antibody will be excluded and will not be eligible to participate in the NEPHSTROM clinical study (MFI\>1500);
16. History or presence of any medical condition or disease which, in the opinion of the Investigator may place the participant at unacceptable risk for study participation;
17. Childbearing potential without use of effective acceptable methods of contraception. Women of childbearing potential can only be included in the study if a pregnancy test is negative at the screening visit (V1) and at baseline visit (V2) if they agree to use adequate contraception. Adequate contraception is defined as any combination of at least two effective methods of birth control, of which at least one is a physical barrier (e.g. condoms with hormonal contraception or implants or combined oral contraceptives, certain intrauterine devices). Women are considered post-menopausal and not of child-bearing potential if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g. age appropriate) or 6 months of spontaneous amenorrhea with serum FSH levels \> 40 mIU/mL or have had surgical treatment such as bilateral tubal ligation, bilateral oophorectomy, or hysterectomy.
18. Pregnancy or lactating;
19. Participation in other investigational medicinal product (IMP) trials within 30 days before the inclusion or concurrent to this study (18 month follow-up);
20. Inability to understand the potential risks and benefits of the study;
21. Legal incapacity.
Minimum Eligible Age

40 Years

Maximum Eligible Age

85 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Leiden University Medical Center

OTHER

Sponsor Role collaborator

Papa Giovanni XXIII Hospital

OTHER

Sponsor Role collaborator

Istituto Di Ricerche Farmacologiche Mario Negri

OTHER

Sponsor Role collaborator

Belfast Health and Social Care Trust

OTHER

Sponsor Role collaborator

National University of Ireland, Galway, Ireland

OTHER

Sponsor Role collaborator

University Hospital Birmingham, NHS Foundation Trust, Hospital, Birmingham, UK

UNKNOWN

Sponsor Role collaborator

NHS Blood and Transplant

OTHER_GOV

Sponsor Role collaborator

Mario Negri Institute for Pharmacological Research

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Giuseppe Remuzzi, MD

Role: STUDY_CHAIR

ASST Papa Giovanni XXIII, Bergamo, Italy/IRCCS - Mario Negri Institute for Pharmacological Research

Mattew Griffin, MD

Role: PRINCIPAL_INVESTIGATOR

National University of ireland - Galway University Hospital -Regenerative Medicine Institute

Paul Cockwell, MD

Role: PRINCIPAL_INVESTIGATOR

University Hospital Birmingham NHS Foundation Trust

Peter Maxwell, MD

Role: PRINCIPAL_INVESTIGATOR

Belfast Health and Social Care Trust - Belfast City Hospital

Locations

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National University of ireland - Galway University Hospital -Regenerative Medicine Institute

Galway, , Ireland

Site Status

ASST - Papa Giovanni XXIII - U.O. Nefrologia e Dialisi/ Mario Negri Institute for Pharmacological Research - Clinical Research Center for Rare Diseases Aldo e Cele Daccò

Bergamo, BG, Italy

Site Status

Belfast Health and Social Care Trust - Belfast City Hospital

Belfast, , United Kingdom

Site Status

University Hospital Birmingham NHS Foundation Trust - Queen Elizabeth Medical Centre

Birmingham, , United Kingdom

Site Status

Countries

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Ireland Italy United Kingdom

References

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Perico N, Remuzzi G, Griffin MD, Cockwell P, Maxwell AP, Casiraghi F, Rubis N, Peracchi T, Villa A, Todeschini M, Carrara F, Magee BA, Ruggenenti PL, Rota S, Cappelletti L, McInerney V, Griffin TP, Islam MN, Introna M, Pedrini O, Golay J, Finnerty AA, Smythe J, Fibbe WE, Elliman SJ, O'Brien T; NEPHSTROM Trial Consortium. Safety and Preliminary Efficacy of Mesenchymal Stromal Cell (ORBCEL-M) Therapy in Diabetic Kidney Disease: A Randomized Clinical Trial (NEPHSTROM). J Am Soc Nephrol. 2023 Oct 1;34(10):1733-1751. doi: 10.1681/ASN.0000000000000189. Epub 2023 Aug 10.

Reference Type BACKGROUND
PMID: 37560967 (View on PubMed)

Lu B, Lerman LO. MSC therapy for diabetic kidney disease and nephrotic syndrome. Nat Rev Nephrol. 2023 Dec;19(12):754-755. doi: 10.1038/s41581-023-00776-z. No abstract available.

Reference Type DERIVED
PMID: 37783947 (View on PubMed)

Other Identifiers

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2016-000661-23

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

NEPHSTROM

Identifier Type: -

Identifier Source: org_study_id