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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03733249
Recruitment Status : Enrolling by invitation
First Posted : November 7, 2018
Last Update Posted : October 14, 2019
Information provided by (Responsible Party):
Bellicum Pharmaceuticals

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.

Condition or disease Intervention/treatment Phase
Acute Lymphoblastic Leukemia Leukemia, Acute Myeloid (AML), Child Lymphoma, Non-Hodgkin Myelodysplastic Syndromes Primary Immunodeficiency Anemia, Aplastic Hemoglobinopathies Cytopenia Fanconi Anemia Diamond Blackfan Anemia Thalassemia Anemia, Sickle Cell Drug: Rimiducid Biological: rivogenlecleucel Phase 1 Phase 2

Detailed Description:
Subjects enrolled on the BP-004 study who have completed 6 months active treatment with BPX-501 T-cells (rivogenlecleucel) 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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 193 participants
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 : January 2017
Estimated Primary Completion Date : June 2034
Estimated Study Completion Date : June 2035

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 Name: AP1903

Biological: rivogenlecleucel
donor T-cells modified with iCasp safety switch
Other Name: BPX-501

Primary Outcome Measures :
  1. Survival [ Time Frame: two years after rivogenlecleucel infusion ]
    Incidence of disease free survival at 2 years

Secondary Outcome Measures :
  1. Adverse events [ Time Frame: Up to 15 years after rivogenlecleucel infusion ]
    Incidence of delayed adverse events suspected to be related to rivogenlecleucel or rimiducid

  2. Number of patients with replication competent retrovirus [ Time Frame: every 6 months after rivogenlecleucel infusion for 5 years and yearly thereafter for another 10 years ]
    Presence of replication competent retrovirus in patient peripheral blood mononuclear cells measured by quantitative PCR

  3. Vector copy number [ Time Frame: every 6 months after rivogenlecleucel infusion for 5 years and yearly thereafter for another 10 years ]
    Copy number of integrated retroviruses in patient peripheral blood mononuclear cells measured by quantitative PCR

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   1 Month to 18 Years   (Child, Adult)
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 and completed 6 months of active treatment

Exclusion Criteria:

  • Lack of parents'/guardian's informed consent for children who are minors
  • Loss of allograft prior to 6 months
  • Progression of malignant disease

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03733249

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IRCCS Ospedale Pediatrico Bambino Gesù
Rome, Italy, 00161
Saudi Arabia
King Abdullah International Medical Research Center (KAIMRC)
Riyadh, Saudi Arabia, 11426
United Kingdom
Institute of Child Health & Great Ormond Street Hospital
London, United Kingdom, WC1N 1EH
Great North Children's Hospital
Newcastle Upon Tyne, United Kingdom, NE1 4LP
Sponsors and Collaborators
Bellicum Pharmaceuticals
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Study Director: Bellicum Pharmaceuticals Clinical Development Sponsor GmbH

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Responsible Party: Bellicum Pharmaceuticals Identifier: NCT03733249    
Other Study ID Numbers: BP-404
First Posted: November 7, 2018    Key Record Dates
Last Update Posted: October 14, 2019
Last Verified: October 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Keywords provided by Bellicum Pharmaceuticals:
Long term follow up
Gene-modified cells
hematologic neoplasms
hematologic malignancies
congenital cytopenia
primary immune deficiencies
Additional relevant MeSH terms:
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Precursor Cell Lymphoblastic Leukemia-Lymphoma
Lymphoma, Non-Hodgkin
Leukemia, Myeloid, Acute
Myelodysplastic Syndromes
Fanconi Anemia
Anemia, Sickle Cell
Anemia, Diamond-Blackfan
Anemia, Aplastic
Neoplasms by Histologic Type
Hematologic Diseases
Bone Marrow Diseases
Leukemia, Lymphoid
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Genetic Diseases, Inborn
Anemia, Hypoplastic, Congenital
DNA Repair-Deficiency Disorders
Metabolic Diseases
Red-Cell Aplasia, Pure
Leukemia, Myeloid