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

NCT03406585 | Completed Phase 1

• 2 other identifiers: ProTrans-T1D2017-002766-50
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

To investigate the safety and tolerance after allogeneic infusion of WJMSCs intravenously in adult patients diagnosed with type 1 diabetes.

Conditions

Type1 Diabetes Mellitus

Outcome Measures

Primary Outcomes (2)

• Safety; measured through set safety parameters

  measured through the registration of adverse events and other safety parameters such as hypoglycemia, allergic reactions, ophthalmologic examination, ECG, vital signs and laboratory assessments.

  throughout the study untill day 372

• Efficacy; comparison of the intervention versus placebo at day 372 versus start of treatment

  Delta-change of C-peptide Area Under the Curve (AUC) (0-120 min) for Mixed Meal Tolerance Test (MMTT) at day 372 following WJMSC/Placebo infusion when compared to test performed before start of treatment.

  Day 372

Secondary Outcomes (7)

• Number of patients insulin independent (ADA criteria) at days 187 and 372

  Days 187 and 372 following WJMSC/Placebo infusion

• Number of patients with daily insulin needs <0.25U/kg at days 187 and 372

  Days 187 and 372 following WJMSC/Placebo infusion

• Insulin requirement/kg BW at days 187 and 372

  Days 187 and 372 following WJMSC/Placebo infusion

• HbA1c at days 187 and 372.

  Days 187 and 372 following WJMSC/Placebo infusion

• Glucose variability at day 372

  Day 372 following WJMSC/Placebo infusion

Study Arms (2)

Allogeneic transplantation with WJMSCs

EXPERIMENTAL

Single infusion of 200 million cells per patient.

Drug: ProTrans: Allogeneic transplantation with WJMSCs

Sham transplantation (placebo)

PLACEBO COMPARATOR

Single infusion with albumin and dmso in sodium chloride (identical concentrations as active treatment)

Drug: Placebos

Interventions

ProTrans: Allogeneic transplantation with WJMSCs DRUG

The drug is a cell suspension with allogeneic mesenchymal stromal cells derived from umbilical cord tissue.

Also known as: Protrans

Allogeneic transplantation with WJMSCs

Placebos DRUG

Placebo treatment

Also known as: Placebo

Sham transplantation (placebo)

Eligibility Criteria

• Age: 18 Years - 40 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• Written informed consent for participation of the study, given before undergoing any study-specific procedures
• Clinical history compatible with type 1 diabetes diagnosed less than 2 years before enrolment
• In the first part of the study patients 1-9 only male patients between 18-40 years of age will be included. In the second part of the study, patients 10-24, both male and female patients 18 to 40 years of age (inclusive at both ends) will be included.
• Mentally stable and, in the opinion of the investigator, able to comply with the procedures of the study protocol
• Fasting plasma C-peptide concentration \>0.12 nmol/L.
• Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, if they are using effective methods of contraception during the study. Acceptable birth control methods are those with a failure rate of less than 1% per year when used consistently and correctly. Such methods include (in "Recommendations related to contraception and pregnancy testing in clinical trials", supplied from www.hma.eu/):
• Combined (estrogen and progestogen containing hormonal contraception associated with inhibition of ovulation.
• oral
• intravaginal
• transdermal
• progestogen-only hormonal contracption associated with inhibition of ovulation
• oral
• injectable
• implantable
• intrauterine device (IUD)

You may not qualify if:

• Inability to provide informed consent
• Patients with body mass index (BMI) \> 30, or weight \>100 kg
• Patients with weight \<50 kg
• Patients with unstable cardiovascular status incl. NYHA class III/IV or symptoms of angina pectoris.
• Patients with uncontrolled hypertension (≥160/105 mmHg).
• Patients with active on-going infections.
• 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.
• 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.
• Patients with any immune suppressive treatment
• Patients with known demyelinating disease or with symptoms or physical examination findings consistent with possible demyelinating disease-
• 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.
• Patients with known, or previous, malignancy.
• Taking oral anti-diabetic therapies or any other concomitant medication which may interfere with glucose regulation other than insulin.
• Patients with GFR \<80 ml/min/1.73 m2 body surface.
• Patients with proliferative retinopathy.

Sponsors & Collaborators

• NextCell Pharma Ab: lead

Study Sites (1)

Karolinska Trial Alliance, Fas 1 enheten, Karolinska Universitetssjukhuset Huddinge

Stockholm, 141 86, Sweden

Location

Related Publications (1)

• Carlsson PO, Espes D, Sisay S, Davies LC, Smith CIE, Svahn MG. Umbilical cord-derived mesenchymal stromal cells preserve endogenous insulin production in type 1 diabetes: a Phase I/II randomised double-blind placebo-controlled trial. Diabetologia. 2023 Aug;66(8):1431-1441. doi: 10.1007/s00125-023-05934-3. Epub 2023 May 24.

  PMID: 37221247 DERIVED

Study Officials

• Per-Ola Carlsson, MD, PhD

  NextCell Pharma

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 27, 2017
• First Posted: January 23, 2018
• Study Start: November 28, 2017
• Primary Completion: July 1, 2020
• Study Completion: September 4, 2020
• Last Updated: April 3, 2023

Record last verified: 2023-03

Data Sharing

• IPD Sharing: Will not share

Locations

SE (1)