A Trial of a Single ProHema-CB Product Transplant in Pediatric Patients With Inherited Metabolic Disorders

NCT02354443 | Terminated Phase 1 Results DMC

• 1 other identifier: FT1050-05
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 2

Brief Summary

The purpose of this study is to describe the safety profile of ProHema-CB as part of a single cord blood unit transplant after a myeloablative conditioning regimen in pediatric patients with inherited metabolic disorders. The safety profile will primarily be assessed by neutrophil engraftment.

Conditions

Metabolic Disorders

Outcome Measures

Primary Outcomes (1)

• Safety Profile, Assessed Primarily by Neutrophil Engraftment

  Engraftment by Day 42 following study transplant procedure

Study Arms (1)

ProHema-CB

EXPERIMENTAL

ProHema-CB represents Ex Vivo Modulated Human Cord Blood Cells. Each subject will receive one administration of ProHema-CB unit transplant.

Biological: ProHema-CB Transplant

Interventions

ProHema-CB Transplant BIOLOGICAL

ProHema-CB, the cellular product, represents the cell populations contained within a human UCB unit after modulation on the day of transplantation by an ex vivo incubation process with the prostaglandin derivative, 16,16-dimethyl prostaglandin E2 (also referred to as FT1050). The cell populations include hematopoietic stem and progenitor cells.

ProHema-CB

Eligibility Criteria

• Age: 1 Year - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Patients must have a confirmed diagnosis of an inherited metabolic disorder (IMD) and be amenable to treatment by hematopoietic cell transplantation:
• Mucopolysaccharidoses: Hurler Syndrome (MPS IH), MPS I-HS (Hurler-Scheie Syndrome), Hunter Syndrome (MPS II), Sanfilippo Syndrome (MPS III), or MPS VI (Maroteaux-Lamy syndrome) with early neurologic involvement and/or sensitization to enzyme replacement therapy (ERT); or
• Leukodystrophies: Krabbe disease (Globoid Leukodystrophy), Metachromatic Leukodystrophy (MLD), Adrenoleukodystrophy (ALD and AMN); or
• Other IMD with lysosomal storage disorder including glycoproteinoses (Alpha-Mannosidosis, Mucolipidosis II or I-Cell disease), sphingo- and other lipidoses (Sandhoff disease, Tay Sachs disease, Pelizaeus Merzbacher (PMD), Niemann-Pick disease, GM1 gangliosidosis, Wolman's disease.
• Male and female subjects aged 1 to 18 years, inclusive.
• Lack of 4 6/6 HLA matched non-carrier related UCB or 8/8 HLA A, B, C, DRß1 matched non-carrier related or 8/8 unrelated bone marrow donor; or donor not available within appropriate timeframe, as determined by the transplant physician.
• Availability of suitable primary and secondary umbilical cord blood (UCB) units.
• Adequate performance status, defined as:
• Subjects ≥ 16 years: Karnofsky score ≥ 70%.
• Subjects \< 16 years: Lansky score ≥ 70%.
• Cardiac: Left ventricular ejection fraction at rest must be \> 40%, or shortening fraction \> 26%.
• Pulmonary:
• Subjects \> 10 years: DLCO (diffusion capacity) \> 50% of predicted (corrected for hemoglobin)
• FEV1, FVC \> 50% of predicted; Note: If unable to perform pulmonary tests, then O2 saturation \> 92% on room air.
• Renal: Serum creatinine within normal range for age, or if serum creatinine outside normal range for age, then renal function (creatinine clearance or GFR) \> 70mL/min/1.73m2.

You may not qualify if:

• Evidence of HIV infection or HIV positive serology.
• Current uncontrolled bacterial, viral or fungal infection (progression of clinical symptoms despite therapy).
• Requirement for continuous respiratory supportive therapy (e.g. ventilator). Patients on intermittent respiratory support should be discussed with the Sponsor.
• Active problems related to chronic aspiration.
• Uncontrolled seizures.
• Any active malignancy or myelodysplastic syndrome or any history of malignancy.
• Inability to give informed consent/assent or to comply with the requirements for care after allogeneic stem cell transplantation.
• Female subjects that are breastfeeding or with a positive pregnancy (HCG) test at Screening.
• Use of an investigational drug within 30 days prior to screening for the primary IMD.

Sponsors & Collaborators

• Fate Therapeutics: lead

Study Sites (2)

Boston Children's Hospital

Boston, Massachusetts, 02115-5450, United States

Location

Duke University Medical Center

Durham, North Carolina, 27705, United States

Location

MeSH Terms

Conditions

Metabolic Diseases

Condition Hierarchy (Ancestors)

Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Chris Storgard, Chief Medical Officer
• Organization: Fate Therapeutics, Inc.

chris.storgard@fatetherapeutics.com

866-875-1833

Study Officials

• Chris Storgard, MD

  Fate Therapeutics

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 16, 2015
• First Posted: February 3, 2015
• Study Start: June 1, 2015
• Primary Completion: December 1, 2016
• Study Completion: February 1, 2017
• Last Updated: October 10, 2018
• Results First Posted: October 10, 2018

Record last verified: 2018-02

Locations

US (2)