NCT03639844

Brief Summary

Providing access of BPX-501 gene modified T cells and rimiducid to pediatric patients who do not meet the eligibility criteria of the BP-U-004 study.

Trial Health

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Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Geographic Reach
1 country

2 active sites

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

August 17, 2018

Completed
4 days until next milestone

First Posted

Study publicly available on registry

August 21, 2018

Completed
Last Updated

October 5, 2020

Status Verified

October 1, 2020

First QC Date

August 17, 2018

Last Update Submit

October 1, 2020

Conditions

Hurler SyndromeInherited Metabolic DisorderLysosomal Storage DisorderMetachromatic LeukodystrophyInborn Errors of Metabolism

Interventions

rivogenlecleucelBIOLOGICAL

BPX-501 T cells are genetically modified with a suicide safety switch. The cells are infused after T cell-depleted HSCT to potentially enhance immune reconstitution while reducing severity and duration of GVHD.

Also known as: BPX-501 T cells
rimiducidDRUG

Rimiducid induces activation of the Caspase 9 suicide gene in BPX-501 T cells inducing apoptosis of the modified T cells in case of GVHD

Also known as: AP1903, Rimiducid for Injection, AP1903 for Injection

Eligibility Criteria

Age3 Months - 21 Years
Sexall
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Males or females
  • Age \< 21 years and \> 3 months
  • Life expectancy \> 10 weeks
  • Patients deemed eligible for allogeneic stem cell transplantation.
  • Non-malignant disorders including:
  • inherited metabolic disorders such as adrenal leukodystrophy;
  • lysosomal storage disorders such as Hurler syndrome or metachromatic leukodystrophy
  • other inborn errors of metabolism
  • Lack of suitable conventional donor (HLA identical sibling or HLA phenotypically identical relative evaluated using high resolution molecular typing).
  • A minimum genotypic identical match of 5/10 is required.
  • The donor and recipient must be identical, as determined by high resolution typing, at least one allele of each of the following genetic loci: HLA-A, HLA-B, HLA-Cw, and HLA- DRB1.
  • Lansky/Karnofsky score \> 50
  • Signed written informed consent

You may not qualify if:

  • Age \< 3 months or \>21 years
  • Patients with non-malignant disorders eligible for treatment on the BP-U-004 study:
  • primary immune deficiencies,
  • severe aplastic anemia not responding to immune suppressive therapy,
  • osteopetrosis,
  • selected cases of hemoglobinopathies and
  • congenital/hereditary cytopenia, including Fanconi Anemia before any clonal malignant evolution (MDS, AML)
  • Dysfunction of liver (ALT/AST \> 5 times normal value, or bilirubin \> 3 times normal value), or of renal function (creatinine clearance \< 30 ml / min)
  • Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or left ventricular ejection fraction \< 40%)
  • Current active infectious disease (including positive HIV serology or viral RNA)
  • Serious concurrent uncontrolled medical disorder
  • Pregnant or breast feeding female patient
  • Lack of parents'/guardian's informed consent.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

Stanford University; Division of Pediatric Stem Cell Transplant & Regenerative Medicine

Palo Alto, California, 94304, United States

Location

MeSH Terms

Conditions

Mucopolysaccharidosis ILysosomal Storage DiseasesLeukodystrophy, MetachromaticMetabolism, Inborn Errors

Interventions

AP 1903 reagentInjections

Condition Hierarchy (Ancestors)

MucopolysaccharidosesCarbohydrate Metabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMucinosesConnective Tissue DiseasesSkin and Connective Tissue DiseasesMetabolic DiseasesNutritional and Metabolic DiseasesHereditary Central Nervous System Demyelinating DiseasesBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesSulfatidosisSphingolipidosesLysosomal Storage Diseases, Nervous SystemLeukoencephalopathiesDemyelinating DiseasesLipidosesLipid Metabolism, Inborn ErrorsLipid Metabolism Disorders

Intervention Hierarchy (Ancestors)

Drug Administration RoutesDrug TherapyTherapeutics

Study Officials

  • Bellicum Pharmaceuticals

    Bellicum Pharmaceuticals, Inc.

    STUDY DIRECTOR

Study Design

Study Type
expanded access
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 17, 2018

First Posted

August 21, 2018

Last Updated

October 5, 2020

Record last verified: 2020-10

Locations

US(2)