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- Klinische Studie NCT01484470
Umbilical Cord Transplantation for the Elderly Population
19. August 2019 aktualisiert von: Patrick Stiff, Loyola University
While cord blood transplants have been performed safely in elderly patients, many still relapse.
The investigators propose to intensify the preparative regimen for this patient group in an attempt to decrease relapses, and combine this with an ex vivo expanded Umbilical Cord Blood (UCB) unit.
Studienübersicht
Status
Abgeschlossen
Bedingungen
Intervention / Behandlung
Detaillierte Beschreibung
Allogeneic stem cell transplantation is a life saving procedure in selected high-risk or recurrent hematologic malignancies and marrow failure syndromes.
However its wide application is limited by availability of suitably HLA matched adult donors.
Umbilical Cord Blood (UCB) has been increasingly used as an alternative hematopoietic stem cell source for these patients.
To date, over 10,000 UCB transplants have been performed in both children32-38 and adults.35,39-44
Its advantages include easier procurement, decreased risk to donors, reduced risk of transmitting infections, the immediate availability of cryopreserved units, and acceptable HLA mismatches.
The transplantation of UCB allows a greater degree of HLA mismatching without an unacceptably high incidence of graft versus host disease (GVHD).
Adult patients receiving myeloablative cord blood transplants have a 90% chance of engraftment, but carry a 50% rate of transplant related mortality.
Studientyp
Interventionell
Einschreibung (Tatsächlich)
18
Phase
- Phase 2
Kontakte und Standorte
Dieser Abschnitt enthält die Kontaktdaten derjenigen, die die Studie durchführen, und Informationen darüber, wo diese Studie durchgeführt wird.
Studienorte
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Illinois
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Maywood, Illinois, Vereinigte Staaten, 60153
- Loyola University Medical Center
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Teilnahmekriterien
Forscher suchen nach Personen, die einer bestimmten Beschreibung entsprechen, die als Auswahlkriterien bezeichnet werden. Einige Beispiele für diese Kriterien sind der allgemeine Gesundheitszustand einer Person oder frühere Behandlungen.
Zulassungskriterien
Studienberechtigtes Alter
55 Jahre bis 73 Jahre (Erwachsene, Älterer Erwachsener)
Akzeptiert gesunde Freiwillige
Nein
Studienberechtigte Geschlechter
Alle
Beschreibung
Inclusion Criteria:
- Ages 55-73
Patients will have one of the following malignancies:
- Acute myelogenous leukemia (AML) deNovo in first CR with adverse cytogenetic abnormalities, M0, M6, M7 subtypes, extramedullary disease in remission or high CD34+ disease (> 50%)
- AML in early relapse (5-10% blasts on bone marrow aspirate or biopsy), or beyond CR-1 with no circulating blasts
- AML at any time if resulting from a previous myelodysplasia
- Acute lymphocytic leukemia or lymphoblastic lymphoma (ALL) in first CR with adverse prognostic features: t (9; 22), extra medullary disease, or mature B cell phenotype
- Acute lymphoid leukemia or lymphoblastic lymphoma in early relapse (5- 10% blasts on aspirate), or beyond CR-1
- Acute Undifferentiated Leukemia or biphenotypic leukemia in CR1 or CR2
- Transfusion dependent myelodysplastic syndrome (MDS) or refractory anemia with excess blasts (RAEB) or RAEB-in transition, CMMOL, or any myelodysplasia with 7q-, 5q-, 7-, 5- or resulting from prior anti cancer therapy.
- Relapsed Non-Hodgkin's Lymphoma (NHL), including those that have relapsed after an autologous marrow/blood stem cell transplant
- Chronic lymphocytic leukemia (CLL) patient who has had fludarabine and either failed or relapsed. Prior autologous transplant patients are eligible.
- Patients with adequate organ function and performance status criteria
Subject must have at least one or the following back-up stem cell sources in case of engraftment failure:
- Subject is willing to undergo BM harvest or peripheral blood progenitor cells (PBPC) collection for use in case of engraftment failure (when clinically applicable).
- Subject has a second CBU as a possible back up.
- Subject's haploidentical family member has been identified and agreed (by signing a written informed consent) to donate hematopoietic stem cells in case of engraftment failure.
- Evaluation by social service/psychologist
- Subject signs the written informed consent after being aware of the nature of the subject's disease and willingly consents to the treatment program after being informed of alternative treatments, potential risks, benefits and discomforts.
- Ability to understand and agree to compliance with strict evaluation, isolation,and medication schedules
- Designated primary care giver.
- Dental evaluation/treatment completed.
- ENT evaluation/treatment completed.
- All patient who survive to day 90 are eligible for measurement of T and B cell function and lymphocyte subset numbers to determine immune reconstitution post UCB transplantation with or without StemEx®
Exclusion Criteria:
- Patient with suitable related donor as defined per institutional guidelines
- Chemotherapy resistant or active AML, ALL, AUL, biphenotypic leukemia
- AML evolved from myelofibrosis
- MDS with 20% or greater bone marrow blasts at pre-transplant workup. Patients may receive therapy and if in remission, are eligible
- Prior allogeneic hematopoeitic stem cell transplant at any time
- Less than twenty-one days have elapsed since the subject's last radiation or chemotherapy prior to conditioning (except for hydroxyurea)
- Uncontrolled bacterial, fungal or viral infection at the time of study enrollment
- Seropositive or NAT positive for HIV, HTLV-1 and Hepatitis C
- Subjects with signs and symptoms of active central nervous system (CNS) disease
- Females who are pregnant or breastfeeding
- Allergy to bovine proteins or to aminoglycoside antibiotics (e.g. gentamicin) or to any product, which may interfere with the treatment.
- Patient unable to give informed consent or unable to comply with the treatment protocol including appropriate supportive care, follow-up and research tests.
- Enrolled in another clinical trial or received an investigational treatment during the last 30 days, unless approved by the primary investigator.
Studienplan
Dieser Abschnitt enthält Einzelheiten zum Studienplan, einschließlich des Studiendesigns und der Messung der Studieninhalte.
Wie ist die Studie aufgebaut?
Designdetails
- Hauptzweck: Behandlung
- Zuteilung: Nicht randomisiert
- Interventionsmodell: Parallele Zuordnung
- Maskierung: Keine (Offenes Etikett)
Waffen und Interventionen
Teilnehmergruppe / Arm |
Intervention / Behandlung |
|---|---|
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Kein Eingriff: Unmanipulated arm
Participants that do not meet criteria for StemEx®, will be registered into the unmanipulated UCB arm and receive the standard conditioning regimen.
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Experimental: Stemx Arm
StemEx is a stem/progenitor cell-based product of ex-vivo expanded allogeneic UCB, which is administered to the subject in combination with the non-manipulated portion of the same cord blood unit (CBU).
The CBU must be cryopreserved in two portions of which the larger (or equal) CBU portion contains at least 1.5 x 107 total nucleated cells (TNC)/Kg.
This portion remains unmanipulated and is transplanted on Day 0. StemEx is derived from the smaller (or equal) CBU portion, which is expanded ex vivo for 21 days starting pre-transplant in the presence of cytokines TPO, IL-6, Flt-3L and SCF at a concentration of 50ng/ml and 5μM tetraethylenepentamine (TEPA)
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For patients allocated to StemEx® arm:
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Was misst die Studie?
Primäre Ergebnismessungen
Ergebnis Maßnahme |
Maßnahmenbeschreibung |
Zeitfenster |
|---|---|---|
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Efficacy of StemEx
Zeitfenster: 100 days
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The primary endpoint is to demonstrate the efficacy of StemEx® vs. unmanipulated UCB transplantation in the elderly population (>55years of age) following RIC regimen by demonstrating engraftment with full donor chimerism (>98%) by Day 100 of more than 60% of the patients who received transplants expanded by the StemEx method.
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100 days
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Sekundäre Ergebnismessungen
Ergebnis Maßnahme |
Maßnahmenbeschreibung |
Zeitfenster |
|---|---|---|
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Time to engraftment
Zeitfenster: 42 days
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The day of neutrophil engraftment is defined as the first day of 3 consecutive days of an ANC greater than 500/microliter.
The platelet recovery is the first of 3 consecutive measurements tested on different days of a platelet count greater than or equal to 20,000 without requiring platelet transfusions in the previous 7 days.
Patients will be monitored for donor cell engraftment as evidenced by neutrophil recovery and donor chimerism in the marrow and/or peripheral blood at serial time points post transplant.
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42 days
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Mitarbeiter und Ermittler
Hier finden Sie Personen und Organisationen, die an dieser Studie beteiligt sind.
Sponsor
Mitarbeiter
Ermittler
- Hauptermittler: Patrick Stiff, MD, Loyola Universtiy Medical Center
Studienaufzeichnungsdaten
Diese Daten verfolgen den Fortschritt der Übermittlung von Studienaufzeichnungen und zusammenfassenden Ergebnissen an ClinicalTrials.gov. Studienaufzeichnungen und gemeldete Ergebnisse werden von der National Library of Medicine (NLM) überprüft, um sicherzustellen, dass sie bestimmten Qualitätskontrollstandards entsprechen, bevor sie auf der öffentlichen Website veröffentlicht werden.
Haupttermine studieren
Studienbeginn
1. Januar 2010
Primärer Abschluss (Tatsächlich)
21. November 2018
Studienabschluss (Tatsächlich)
21. November 2018
Studienanmeldedaten
Zuerst eingereicht
30. November 2011
Zuerst eingereicht, das die QC-Kriterien erfüllt hat
1. Dezember 2011
Zuerst gepostet (Schätzen)
2. Dezember 2011
Studienaufzeichnungsaktualisierungen
Letztes Update gepostet (Tatsächlich)
21. August 2019
Letztes eingereichtes Update, das die QC-Kriterien erfüllt
19. August 2019
Zuletzt verifiziert
1. August 2019
Mehr Informationen
Begriffe im Zusammenhang mit dieser Studie
Schlüsselwörter
Zusätzliche relevante MeSH-Bedingungen
Andere Studien-ID-Nummern
- 202041
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Klinische Studien zur StemEx
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Gamida Cell -Teva Joint Venture Ltd.AbgeschlossenAkute myeloische Leukämie | Myelodysplastische Syndrome | Non-Hodgkin-Lymphom | Chronisch-myeloischer Leukämie | Hämatologische Malignome | Morbus Hodgkin | Lymphatische LeukämieVereinigte Staaten, Israel, Ungarn, Italien, Spanien