Clinical Trials Logo

Clinical Trial Details — Status: Terminated

Administrative data

NCT number NCT02354443
Other study ID # FT1050-05
Secondary ID
Status Terminated
Phase Phase 1
First received
Last updated
Start date June 2015
Est. completion date February 2017

Study information

Verified date February 2018
Source Fate Therapeutics
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


Description:

This study is an open-label trial of the safety of a single cord blood transplant using ProHema-CB following busulfan/cyclophosphamide/ATG conditioning for pediatric patients with inherited metabolic disorders.

A maximum of 12 eligible male and female subjects (1 to 18 years old, inclusive) will be enrolled and treated in the trial at approximately 1 to 3 centers within the U.S.

All subjects will be admitted to the hospital, per institutional practice and will receive a conditioning regimen, after which they will receive a HLA-matched or partially matched ProHema -CB unit on Day 0.

They will receive study follow up assessments weekly following Day 0 through Day 100 and study visit Days 180, 270, 365 and 730.


Recruitment information / eligibility

Status Terminated
Enrollment 1
Est. completion date February 2017
Est. primary completion date December 2016
Accepts healthy volunteers No
Gender All
Age group 1 Year to 18 Years
Eligibility Inclusion Criteria:

1. 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.

2. Male and female subjects aged 1 to 18 years, inclusive.

3. 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.

4. Availability of suitable primary and secondary umbilical cord blood (UCB) units.

5. Adequate performance status, defined as:

- Subjects = 16 years: Karnofsky score = 70%.

- Subjects < 16 years: Lansky score = 70%.

6. Cardiac: Left ventricular ejection fraction at rest must be > 40%, or shortening fraction > 26%.

7. 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.

8. 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.

9. Hepatic: Bilirubin = 2.5 mg/dL (except in the case of Gilbert's syndrome, ongoing hemolytic anemia, or due to the primary IMD); and ALT, AST and Alkaline Phosphatase = x 3 ULN (all elevations beyond the ULN must be secondary to the primary IMD and not a comorbid condition).

10. Signed IRB approved Informed Consent Form (ICF).

Exclusion Criteria:

1. Evidence of HIV infection or HIV positive serology.

2. Current uncontrolled bacterial, viral or fungal infection (progression of clinical symptoms despite therapy).

3. Requirement for continuous respiratory supportive therapy (e.g. ventilator). Patients on intermittent respiratory support should be discussed with the Sponsor.

4. Active problems related to chronic aspiration.

5. Uncontrolled seizures.

6. Any active malignancy or myelodysplastic syndrome or any history of malignancy.

7. Inability to give informed consent/assent or to comply with the requirements for care after allogeneic stem cell transplantation.

8. Female subjects that are breastfeeding or with a positive pregnancy (HCG) test at Screening.

9. Use of an investigational drug within 30 days prior to screening for the primary IMD.

Study Design


Related Conditions & MeSH terms


Intervention

Biological:
ProHema-CB Transplant
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.

Locations

Country Name City State
United States Boston Children's Hospital Boston Massachusetts
United States Duke University Medical Center Durham North Carolina

Sponsors (1)

Lead Sponsor Collaborator
Fate Therapeutics

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Safety Profile, Assessed Primarily by Neutrophil Engraftment Engraftment by Day 42 following study transplant procedure
See also
  Status Clinical Trial Phase
Completed NCT02311270 - Survey on Cardiovascular Events in Patients With Metabolic Disease on Chronic Carnitine Supplementation N/A
Completed NCT02039596 - Nutritional Metabolomics: the Search for Dietary Exposure Variables N/A
Completed NCT04142424 - A Study to Understand the Safety, Tolerability, and Activity of Drug in Body Over a Period of Time of AZD2693, in Subjects of Non-Childbearing Potential in Overweight But Otherwise Healthy Subjects, and Healthy Chinese and Japanese Subjects Phase 1
Recruiting NCT05398783 - A Natural History Study of Metabolic Sizing in Health and Disease
Completed NCT00387166 - Socioeconomic Status, Psychosocial Factors, and CVD Risk in Mexican-American Women N/A
Completed NCT02130908 - A Study on the Possible Health Effects of Lean Fish, Fatty Fish and Lean Meat Intake in Non-obese Adults N/A
Recruiting NCT03128996 - Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders Phase 1/Phase 2
Not yet recruiting NCT06358118 - Buyuan-zhixiao Formula in the Treatment of Elderly Patients With Diabetes and Multiple Metabolic Disorders N/A
Recruiting NCT02503384 - Clinical and Basic Study for Pediatric Liver Transplantation N/A
Active, not recruiting NCT01894139 - An Optimized Programming of Healthy Children (APPROACH) N/A
Completed NCT04868240 - Concurrent Training and Metabolic Profile, Lung Function, Quality of Life and Stress N/A
Terminated NCT02231710 - Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases Phase 1
Recruiting NCT00920972 - Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases Phase 1/Phase 2
Active, not recruiting NCT03188107 - Interrater Reliability of Infant Motor Profile N/A
Withdrawn NCT03284658 - Biomarker for the Early Diagnosis and Monitoring in Tyrosinemia Type 1 (BioTyrosin)
Completed NCT06067126 - Post-delivery Maternal-offspring Obesity and Metabolic Risk After a Prepregnancy Weight-loss Intervention
Completed NCT02107872 - Study to Assess the Safety and Tolerability of Multiple Ascending Doses of REGN1500 Phase 1
Recruiting NCT02419898 - Oxfordshire Women and Their Children's Health
Terminated NCT01343680 - Trial of Two Central Venous Catheter (CVC) Flushing Schemes in Pediatric Hematology and Oncology Patients Phase 3