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NCT03857841

A Safety Study of IV Stem Cell-derived Extracellular Vesicles (UNEX-42) in Preterm Neonates at High Risk for BPD

Terminated Phase 1 Results posted Last updated 12 October 2021
What this trial tests

Phase 1 trial testing UNEX-42 in Bronchopulmonary Dysplasia in 3 participants. Terminated before completion.

Timeline
9 October 2019
Primary endpoint
20 May 2021
20 May 2021

Quick facts

Lead sponsorUnited Therapeutics
PhasePhase 1
StatusTerminated
Study typeINTERVENTIONAL
Allocationrandomized
Designsequential
Maskingquadruple
Primary purposetreatment
Enrollment3
Start date9 October 2019
Primary completion20 May 2021
Estimated completion20 May 2021
Sites6 locations across United States

Drugs / interventions tested

Conditions studied

Sponsor

United Therapeutics — full company profile →

Who can join

Adults 3 Days to 14 Days, any sex, with Bronchopulmonary Dysplasia. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Number of Subjects With Treatment-emergent Adverse Events During the Post-treatment Phase (Safety and Tolerability) Primary · From Day 1 to 40 Weeks Post-menstrual Age or Hospital Discharge, whichever came first

The safety and tolerability of UNEX-42 in subjects with BPD was evaluated by the number of subjects with treatment-emergent adverse events, including death, computed by dose cohort and overall during the Post-treatment Phase.

GroupValue95% CI
20 Pmol Phospholipid/kg Body Weight2
60 Pmol Phospholipid/kg Body Weight0
200 Pmol Phospholipid/kg Body Weight0
Placebo1

Adverse events — posted to ClinicalTrials.gov

Time frame: From Day -2 to 40 Weeks Post-menstrual Age or Hospital Discharge, whichever came first. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

20 Pmol Phospholipid/kg Body Weight
Serious: 1/2 (50%)
Deaths: 1/2
60 Pmol Phospholipid/kg Body Weight
Serious: 0
Deaths: 0
200 Pmol Phospholipid/kg Body Weight
Serious: 0
Deaths: 0
Placebo
Serious: 0/1 (0%)
Deaths: 0/1

Serious adverse events (1 terms)

ReactionSystem20 Pmol Phospholipid/kg Bo…60 Pmol Phospholipid/kg Bo…200 Pmol Phospholipid/kg B…Placebo
Necrotizing colitisGastrointestinal disorders
Other adverse events (26 terms — click to expand)

ReactionSystem20 Pmol Phospholipid/kg Bo…60 Pmol Phospholipid/kg Bo…200 Pmol Phospholipid/kg B…Placebo
HypotensionVascular disorders
Generalized edemaGeneral disorders
Atrial septal defectCongenital, familial and genetic disorders
Dermatitis diaperSkin and subcutaneous tissue disorders
CholestasisHepatobiliary disorders
CoagulopathyBlood and lymphatic system disorders
HypovolemiaMetabolism and nutrition disorders
Gastroesophageal reflux diseaseGastrointestinal disorders
HyperbilirubinemiaHepatobiliary disorders
HyperkalemiaMetabolism and nutrition disorders
HypernatremiaMetabolism and nutrition disorders
HypoalbuminemiaMetabolism and nutrition disorders
HypocalcemiaMetabolism and nutrition disorders
HyponatremiaMetabolism and nutrition disorders
Intestinal obstructionGastrointestinal disorders
Beta hemolytic streptococcal infectionInfections and infestations
Pleural effusionRespiratory, thoracic and mediastinal disorders
HydrocephalusNervous system disorders
Head injuryInjury, poisoning and procedural complications
Renal impairmentRenal and urinary disorders
Thrombophlebitis superficialVascular disorders
Transaminases increasedInvestigations
Vitamin D deficiencyMetabolism and nutrition disorders
HypoglycemiaMetabolism and nutrition disorders
Wound dehiscenceInjury, poisoning and procedural complications
Oral candidiasisInfections and infestations

Most-reported serious reactions: Necrotizing colitis.

Data from ClinicalTrials.gov NCT03857841 adverse events section.

Sponsor's own description

A multicenter, placebo-controlled, randomized, dose escalation, safety, and tolerability study of UNEX-42 in infants born at \<27 weeks of gestational age (GA) at high risk for bronchopulmonary dysplasia (BPD).

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Advances in therapeutic applications of extracellular vesicles.
    Wiklander OPB, Brennan MÁ, Lötvall J, Breakefield XO, et al · · 2019 · cited 791× · PMID 31092696 · DOI 10.1126/scitranslmed.aav8521
  2. Challenges and advances in clinical applications of mesenchymal stromal cells.
    Zhou T, Yuan Z, Weng J, Pei D, et al · · 2021 · cited 489× · PMID 33579329 · DOI 10.1186/s13045-021-01037-x
  3. Mesenchymal stem cell derived-exosomes: a modern approach in translational medicine.
    Nikfarjam S, Rezaie J, Zolbanin NM, Jafari R. · · 2020 · cited 309× · PMID 33246476 · DOI 10.1186/s12967-020-02622-3
  4. Therapeutic roles of mesenchymal stem cell-derived extracellular vesicles in cancer.
    Weng Z, Zhang B, Wu C, Yu F, et al · · 2021 · cited 282× · PMID 34479611 · DOI 10.1186/s13045-021-01141-y
  5. A Comprehensive Review on Factors Influences Biogenesis, Functions, Therapeutic and Clinical Implications of Exosomes.
    Gurunathan S, Kang MH, Kim JH. · · 2021 · cited 242× · PMID 33628021 · DOI 10.2147/ijn.s291956
  6. Mesenchymal Stem Cell Derived Extracellular Vesicles for Tissue Engineering and Regenerative Medicine Applications.
    Tsiapalis D, O'Driscoll L. · · 2020 · cited 238× · PMID 32316248 · DOI 10.3390/cells9040991
  7. A Brief Overview of Global Trends in MSC-Based Cell Therapy.
    Jovic D, Yu Y, Wang D, Wang K, et al · · 2022 · cited 186× · PMID 35344199 · DOI 10.1007/s12015-022-10369-1
  8. Pulmonary hypertension in bronchopulmonary dysplasia.
    Hansmann G, Sallmon H, Roehr CC, Kourembanas S, et al · · 2021 · cited 171× · PMID 32521539 · DOI 10.1038/s41390-020-0993-4

Verify or expand the search:

Other recruiting trials for Bronchopulmonary Dysplasia

Currently open trials in the same condition.

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