Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study

Sponsor
Bellicum Pharmaceuticals (Industry)
Overall Status
Active, not recruiting
CT.gov ID
NCT03733249
Collaborator
(none)
193
1
1
197
1

Study Details

Study Description

Brief Summary

This is a long-term follow up study evaluating the safety of BPX-501 T cells (rivogenlecleucel) and infused in pediatric patients previously enrolled on the BP-004 study.

Detailed Description

Subjects enrolled on the BP-004 study who have completed or discontinued from the study, and are beyond Day 180 will be requested to enroll on this long-term follow up protocol. Long term follow up for gene therapy clinical and safety endpoints will continue up to 15 years.

Study Design

Study Type:
Interventional
Anticipated Enrollment :
193 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
Follow-up of Phase 1/2 Study of CaspaCIDe T Cells (BPX-501) From an HLA-partially Matched Family Donor After Negative Selection of TCR αβ+T Cells in Pediatric Patients Affected by Hematological Disorders
Actual Study Start Date :
Jan 1, 2017
Actual Primary Completion Date :
Jun 30, 2020
Anticipated Study Completion Date :
Jun 1, 2033

Arms and Interventions

Arm Intervention/Treatment
Experimental: Rimiducid and Rivogenlecleucel

Rimiducid: to treat uncontrolled GVHD in patients who have received rivogenlecleucel Rimiducid will be given at 0.4 mg/kg weight (intravenous infusion) No further rivogenlecleucel infusions are planned. Patients who received rivogenlecleucel in the BP-004 study will be evaluated for long-term safety and efficacy.

Drug: Rimiducid
Rimiducid is administered to treat chronic graft versus host disease
Other Names:
  • AP1903
  • Biological: rivogenlecleucel
    donor T-cells modified with iCasp safety switch
    Other Names:
  • BPX-501
  • Outcome Measures

    Primary Outcome Measures

    1. Survival [two years after rivogenlecleucel infusion]

      Incidence of disease free survival at 2 years

    Secondary Outcome Measures

    1. Adverse events [Up to 15 years after rivogenlecleucel infusion]

      Incidence of delayed adverse events suspected to be related to rivogenlecleucel or rimiducid

    2. Rimiducid Pharmacokinetics [Up to 15 years after rivogenlecleucel infusion]

      Assessment of PK (rimiducid) and PD (BPX-501) profiles after treatment with rimiducid for aGVHD.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    1 Month to 18 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:
    • Signed written informed consent by the patient or the patient's guardian for children who are minors

    • Enrolled on BP-004 protocol, received BPX-501 infusion, completed or discontinued from the study, and are beyond Day +180.

    Exclusion Criteria:
    • Lack of parents'/guardian's informed consent for children who are minors

    • Loss of allograft prior to 6 months

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 IRCCS Ospedale Pediatrico Bambino Gesù Rome Italy 00161

    Sponsors and Collaborators

    • Bellicum Pharmaceuticals

    Investigators

    • Study Director: Bellicum Pharmaceuticals, Bellicum Pharmaceuticals, Inc.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Bellicum Pharmaceuticals
    ClinicalTrials.gov Identifier:
    NCT03733249
    Other Study ID Numbers:
    • BP-404
    First Posted:
    Nov 7, 2018
    Last Update Posted:
    Jul 12, 2022
    Last Verified:
    Jul 1, 2022

    Study Results

    No Results Posted as of Jul 12, 2022