- ICH GCP
- Amerikanska kliniska prövningsregistret
- Klinisk prövning NCT02549625
Phase I Safety and Feasibility Study of Intracoronary Delivery of Autologous Bone Marrow Derived Mononuclear Cells
12 maj 2021 uppdaterad av: Timothy J Nelson, MD, PhD
Phase I Safety and Feasibility Study of Intracoronary Delivery of Autologous Bone Marrow Derived Mononuclear Cells for Systemic, Single Right Ventricular Failure Due to Congenital Heart Disease
The objective of this study is to determine the safety and feasibility of autologous mononuclear cells (MNC) collected from bone marrow (BM) and intracoronary delivery for individuals with declining performance of their single right ventricle systemic pumps.
This procedure has the potential to foster a new strategy for congenital heart patients.
This is an open-label study of autologous MNC derived from bone marrow with a 2-year follow-up to document 1) related serious adverse events and 2) monitor changes in cardiac structure and function.
Studieöversikt
Status
Avslutad
Betingelser
Intervention / Behandling
Detaljerad beskrivning
This study is an open label Phase I trial to determine the safety and feasibility of bone marrow-derived mononuclear cells delivered into the coronary circulation of subjects with Fontan circulation and right ventricular dysfunction.
Subjects will be screened at outpatient clinic visits at Mayo Clinic and interested qualified subjects will be consented and offered participation in this trial.
Once informed consent has been obtained; preoperative values will be established and a selection committee will review subjects within three days prior to planned procedure to confirm inclusion and exclusion criteria.
This will require a non-study cardiologist to review the clinical case and confirm the baseline cardiac function with evidence of disease progression with right ventricle dysfunction.
However, individuals with decreased cardiac function requiring inotropic support and immediate listing for cardiac transplantation will NOT be included such that the risk of this research procedure may not be acceptable.
Following cell-based product delivery, subjects will be followed for 6-months according to a pre-determined schedule that includes imaging studies along with questionnaires, electrophysiology and laboratory studies.
Subjects will be contacted for an additional 18-months for surveillance by phone.
Studietyp
Interventionell
Inskrivning (Faktisk)
10
Fas
- Fas 1
Kontakter och platser
Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.
Studieorter
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Minnesota
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Rochester, Minnesota, Förenta staterna, 55905
- Mayo Clinic
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Deltagandekriterier
Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.
Urvalskriterier
Åldrar som är berättigade till studier
2 år till 30 år (Barn, Vuxen)
Tar emot friska volontärer
Nej
Kön som är behöriga för studier
Allt
Beskrivning
Inclusion Criteria:
- Individuals with Fontan circulation with right ventricular dysfunction
- Ejection fraction equal to or less than 40% despite optimal outpatient medical management for at least 3 months
- Able to undergo bone marrow aspirate according to clinical consultation with hematology (INR must be maintained at or below 1.5)
- Able to undergo an MRI or CT examination
Exclusion Criteria:
- Individuals or parents of minors unwilling to consent to participation
- Individuals with severe chronic diseases, extra-cardiac syndromes, or cancer
- Females 10 years and older with positive pregnancy test or lack of effective birth control method
- Individuals currently requiring IV inotropes
- Individuals with bleeding disorders or history of thrombosis
- Individuals not eligible for MRI and have elevated serum creatinine level (eGFR less that 45 mL/min) and therefore cannot have a CT examination
- Individuals not eligible for MRI and have a prior significant reaction to intravenous contrast required for CT examination
Individuals with the following conditions within 60 days prior to procedure:
- Cardiogenic shock or extracorporeal circulation;
- New arrhythmia that required medication for control;
- Documented infection requiring treatment with antibiotics, and/or current infection being treated with antibiotics;
- Cardiac condition requiring emergency procedure;
- Cardiovascular surgery;
- Seizures or history of significant neurological injury;
- Multi-system organ failure including acute or chronic renal failure
Studieplan
Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.
Hur är studien utformad?
Designdetaljer
- Primärt syfte: Behandling
- Tilldelning: N/A
- Interventionsmodell: Enskild gruppuppgift
- Maskning: Ingen (Open Label)
Vapen och interventioner
Deltagargrupp / Arm |
Intervention / Behandling |
|---|---|
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Experimentell: Single-arm
Intracoronary delivery of autologous bone marrow-derived mononuclear cells using catheterization procedure
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Andra namn:
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Vad mäter studien?
Primära resultatmått
Resultatmått |
Tidsram |
|---|---|
|
Number of related serious adverse events
Tidsram: 6 months post cell delivery
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6 months post cell delivery
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Sekundära resultatmått
Resultatmått |
Tidsram |
|---|---|
|
Number of subjects who died
Tidsram: 6 months post cell delivery
|
6 months post cell delivery
|
|
Number of subjects with sustained or symptomatic ventricular arrhythmias
Tidsram: 6 months post cell delivery
|
6 months post cell delivery
|
|
Number of subjects with acute decompensated heart failure
Tidsram: 6 months post cell delivery
|
6 months post cell delivery
|
|
Number of subjects with myocardial infarction
Tidsram: 6 months post cell delivery
|
6 months post cell delivery
|
|
Number of subjects with cardiac infection
Tidsram: 6 months post cell delivery
|
6 months post cell delivery
|
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Number of subjects with unexpected cardiovascular procedures within 6 months following cellular transplantation
Tidsram: 6 months post cell delivery
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6 months post cell delivery
|
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Percentage of subjects that have cells delivered
Tidsram: 6 months post cell delivery
|
6 months post cell delivery
|
|
Percentage of subjects completing the 6-month follow-up
Tidsram: 6 months post cell delivery
|
6 months post cell delivery
|
|
Change in CT/MRI derived right ventricle ejection fraction
Tidsram: Baseline, 6 months post cell delivery
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Baseline, 6 months post cell delivery
|
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Change in diastolic ventricular volume
Tidsram: Baseline, 6 months post cell delivery
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Baseline, 6 months post cell delivery
|
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Change in systolic ventricular volume
Tidsram: Baseline, 6 months post cell delivery
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Baseline, 6 months post cell delivery
|
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Change in echocardiography derived right ventricle ejection fraction
Tidsram: Baseline, 6 months post cell delivery
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Baseline, 6 months post cell delivery
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Samarbetspartners och utredare
Det är här du hittar personer och organisationer som är involverade i denna studie.
Sponsor
Samarbetspartners
Utredare
- Huvudutredare: Muhammad Y Qureshi, MBBS, Mayo Clinic
- Studierektor: Timothy J Nelson, M.D., Ph.D., Mayo Clinic
Publikationer och användbara länkar
Den som ansvarar för att lägga in information om studien tillhandahåller frivilligt dessa publikationer. Dessa kan handla om allt som har med studien att göra.
Användbara länkar
Studieavstämningsdatum
Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.
Studera stora datum
Studiestart
1 augusti 2015
Primärt slutförande (Faktisk)
1 november 2020
Avslutad studie (Faktisk)
1 november 2020
Studieregistreringsdatum
Först inskickad
10 september 2015
Först inskickad som uppfyllde QC-kriterierna
11 september 2015
Första postat (Uppskatta)
15 september 2015
Uppdateringar av studier
Senaste uppdatering publicerad (Faktisk)
14 maj 2021
Senaste inskickade uppdateringen som uppfyllde QC-kriterierna
12 maj 2021
Senast verifierad
1 maj 2021
Mer information
Termer relaterade till denna studie
Nyckelord
Ytterligare relevanta MeSH-villkor
Andra studie-ID-nummer
- 13-006427
Plan för individuella deltagardata (IPD)
Planerar du att dela individuella deltagardata (IPD)?
NEJ
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