Mesenchymal Stromal Cells to Treat Type 1 Diabetes in Children and Adolescents

April 27, 2026 updated by: Per-Ola Carlsson, Uppsala University Hospital

A Double-blinded, Randomized, Parallel, Placebo-controlled Trial of Wharton's Jelly-derived Allogeneic Mesenchymal Stromal Cells to Treat Type 1 Diabetes in Children and Adolescents

This is a combined phase 1 and 2 study in 66 subjects, male or female, between 7-21 years of age that have recently (< 6 months) been diagnosed with type 1 diabetes. The first phase 1 part of the study includes six subjects openly receiving allogeneic Wharton's jelly derived mesenchymal stromal cells as the Advanced Therapy Medicinal Product (ATMP) Protrans, three each in the age ranges 7-11 and 12-18.The second part is a randomized, double-blinded placebo-controlled phase 2 study in parallel design comparing allogeneic Wharton's jelly derived mesenchymal stromal cells treatment (as Protrans) to placebo in children and adolescent subjects (7-21 years of age) diagnosed with type 1 diabetes, The primary objectives of this study will be to investigate the safety, tolerance and efficacy after an allogieneic infusion of Wharton's jelly derived mesenchymal stromal cells.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
66

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Sweden
      • Uppsala, Sweden, 75185
        • Uppsala University Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

7 years to 21 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Written informed consent for participation of the study (for subjects below 18 years of age also from both caregivers), given before undergoing any study-specific procedures
  2. Clinical history compatible with type 1 diabetes diagnosed less than 6 months before enrolment
  3. In the first part of the study, six subjects, three between 7-11 and three between 12-18 years of age (both groups inclusive at both ends), will be included. The sixty subjects in the second part of the study are stratified by age (12-21 and 7-11 years, respectively) and randomized to one of two treatment arms (active or placebo), with a 6-month safety delay for the younger stratum.
  4. Mentally stable and, in the opinion of the investigator, able to comply with the procedures of the study protocol.
  5. Fasting plasma C-peptide concentration >0.12 nmol/L.

  6. Subjects of child-bearing potential must agree to using adequate contraception until one year after the administration of WJMSC/Placebo. Adequate contraception is as follows:

    1. oral (except low-dose gestagen (lynestrenol and noretisteron), injectable or implanted hormonal contraceptives.
    2. intrauterine device
    3. intrauterine system (for example progestin-releasing coil)
    4. vasectomized male (with appropriate postvasectomy documentation of the absence of sperm in the ejaculate)

Exclusion Criteria:

  1. Subjects with body weight >100 kg
  2. Subjects with unstable cardiovascular status incl. NYHA class III/IV or symptoms of angina pectoris.
  3. Subjects with uncontrolled hypertension (≥160/105 mmHg).
  4. Subjects with active on-going infections.
  5. Subjects with latent or previous as well as on-going therapy against tuberculosis, or exposed to tuberculosis or has traveled in areas with a high risk of tuberculosis or mycosis within the last 3 months.
  6. Subjects with serological evidence of infection with HIV, Treponema pallidum, hepatitis B antigen (subjects with serology consistent with previous vaccination and a history of vaccination are acceptable), or hepatitis C.
  7. Subjects with any systemic immune suppressive treatment
  8. Subjects with a known demyelinating disease or with symptoms or physical examination findings consistent with possible demyelinating disease.
  9. Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test.
  10. Subjects with known, or previous, malignancy.
  11. Taking oral anti-diabetic therapies or any other concomitant medication which may interfere with glucose regulation other than insulin.
  12. Subjects with GFR <60 ml/min/1.73 m2 body surface.
  13. Subject with any condition or any circumstance that, in the opinion of the investigator, would make it unsafe to undergo treatment with MSC.
  14. Known hypersensitivity against any excipients, i.e., dimethyl sulfoxide (DMSO).

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Active Comparator: Wharton's jelly derived mesenchymal stromal cells (Protrans)
Cells are dissolved in saline and given intravenously over a period of 20-40 min. 100 million cells to subjects < 50 kg and 200 million cells to subjects 50-100 kg (>100 kg is an exclusion criterion).
Biological: the ATMP Protrans
Protrans consists of Wharton's jelly derived mesenchymal stromal cells
Placebo Comparator: Placebo
Placebo (saline) is given intravenously over a period of 20-40 min.
Biological: the ATMP Protrans
Protrans consists of Wharton's jelly derived mesenchymal stromal cells

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Safety at one year evaluated as adverse events
Time Frame: One year
Safety parameters will be evaluated at each study visit and recorded as adverse events.
One year
Safety at five years evaluated as adverse events
Time Frame: Five years
Safety parameters will be evaluated at each study visit and recorded as adverse events.
Five years
Efficacy measured as change in C-peptide Area under the curve to a mixed mealtolerance test.
Time Frame: One year
Change in C-peptide Area under the curve (AUC) (0-120 min) for mixed meal tolerance test (MMTT) at 12 months following Protrans/Placebo infusion when compared to test performed before the start of treatment (baseline).
One year

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Insulin independency
Time Frame: One year
The proportion of study participants independent of insulin at 6 months
One year
Insulin independency
Time Frame: One year
The proportion of study participants independent of insulin at 12 months
One year
Low insulin needs
Time Frame: 6 months
The proportion of study participants with daily insulin needs <0.25 U/kg at 6 months
6 months
Low insulin needs
Time Frame: 12 months
The proportion of study participants with daily insulin needs <0.25 U/kg at 12 months
12 months
Insulin needs
Time Frame: 6 months
Insulin requirement/kg body weigh at 6 months
6 months
Insulin needs
Time Frame: 12 months
Insulin requirement/kg body weigh at 12 months
12 months
HbA1c
Time Frame: 6 months
HbA1c at 6 months
6 months
HbA1c
Time Frame: 12 months
HbA1c at 12 months
12 months
Time in target
Time Frame: 6 months
Time in target (4-8 mmol/l) as measured by flash glucose monitoring for 14 days at 6 months
6 months
Time in target
Time Frame: 12 months
Time in target (4-8 mmol/l) as measured by flash glucose monitoring for 14 days at 12 months
12 months
Time in range
Time Frame: 6 months
Time in target (3.9-10 mmol/l) as measured by flash glucose monitoring for 14 days at 6 months
6 months
Time in range
Time Frame: 12 months
Time in target (3.9-10 mmol/l) as measured by flash glucose monitoring for 14 days at 12 months
12 months
C-peptide
Time Frame: 6 months
Change in C-peptide Area under the curve (AUC) (0-120 min) for mixed meal tolerance test (MMTT) at 6 months following Protrans/Placebo infusion when compared to test performed before the start of treatment (baseline).
6 months
Change in peak C-peptide
Time Frame: 6 months
Change in peak C-peptide concentration during the first 6 months
6 months
Change in peak C-peptide
Time Frame: 12 months
Change in peak C-peptide concentration during the first 12 months
12 months

Other Outcome Measures

Other Outcome Measures

For clinical trials, a planned measurement described in the protocol that is used to determine the effect of an intervention/treatment on participants. For observational studies, a measurement or observation that is used to describe patterns of diseases or traits, or associations with exposures, risk factors, or treatment. Types of outcome measures include primary outcome measure and secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Gender differences
Time Frame: 6 months
Differences in parameters of primary and secondary endpoints between genders
6 months
Gender differences
Time Frame: 12 months
Differences in parameters of primary and secondary endpoints between genders
12 months
HLA class 1 genotypes
Time Frame: 6 months
Differences in parameters of primary and secondary endpoints between HLA class 1 genotypes
6 months
HLA class 1 genotypes
Time Frame: 12 months
Differences in parameters of primary and secondary endpoints between HLA class 1 genotypes
12 months
age
Time Frame: 6 months
Differences in parameters of primary and secondary endpoints between ages 7-11 and 12-21
6 months
age
Time Frame: 12 months
Differences in parameters of primary and secondary endpoints between ages 7-11 and 12-21
12 months
Autoantibodies
Time Frame: 6 months
Change of levels of diabetes-related autoantibodies when compared to test before the start of treatment (baseline)
6 months
Autoantibodies
Time Frame: 12 months
Change of levels of diabetes-related autoantibodies when compared to test before the start of treatment (baseline)
12 months
Peripheral blood mononuclear cells
Time Frame: 6 months
Change in reactivity and cytokine production of peripheral blood mononuclear cells when compared to test before the start of treatment (baseline)
6 months
Peripheral blood mononuclear cells
Time Frame: 12 months
Change in reactivity and cytokine production of peripheral blood mononuclear cells when compared to test before the start of treatment (baseline)
12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Uppsala University Hospital

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Per-Ola Carlsson, MD, PhD, Uppsala University Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
January 14, 2022

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
September 1, 2028

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 1, 2028

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
September 20, 2021

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 20, 2021

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
September 29, 2021

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
April 28, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 27, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • WJMSC-P01

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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