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New treatment option for Adrenoleukodystrophy
Official title UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
ClinicalTrials.gov ID: NCT02254863
What this study is testing
What is DUOC-01?
DUOC-01 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for adrenoleukodystrophy.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The primary objective of the study is to determine the safety and feasibility of intrathecal administration of DUOC-01 as an adjunctive therapy in patients with inborn errors of metabolism who have evidence of early demyelinating disease in the central nervous system (CNS) who are undergoing standard treatment with unrelated umbilical cord blood transplantation (UCBT). The secondary objective of the study is to describe the efficacy of UCBT with intrathecal administration of DUOC-01 in these patients.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 1 to 22
You may be able to join if
- Patients must be age ≥1 week to ≤21 years.
- Patients must have one of the following inherited metabolic diseases detected by enzyme or mutation analysis, and confirmed by repeat testing on a...
- Patients must have neurologic evidence of their disease, either clinically or via neuroimaging or neurophysiological testing. Examples of evidence of...
- Abnormal EEG, Brainstem Auditory Evoked Response (BAER), and/or Visual Evoked Potentials (VEP).
- Abnormal brain MRI, ie. increased Loes score (measure of white matter damage, demyelination, and brain atrophy) and/or abnormal corticospinal tracts...
You likely can't join if
- Prior organ, tissue, or stem cell transplant within 3 years of study entry.
- Prior participation in any gene or regenerative cell therapy study.
- Inability to have an MRI scan or lumbar puncture.
- Intractable seizures.
- Chronic aspiration.
- Bleeding disorder.
See the full eligibility criteria
- Patients must be age ≥1 week to ≤21 years.
- Patients must have one of the following inherited metabolic diseases detected by enzyme or mutation analysis, and confirmed by repeat testing on a separately obtained sample: Adrenoleukodystrophy (ALD) Batten Disease...
- Patients must have neurologic evidence of their disease, either clinically or via neuroimaging or neurophysiological testing. Examples of evidence of neurologic involvement include, but are not limited to the following:
- Abnormal EEG, Brainstem Auditory Evoked Response (BAER), and/or Visual Evoked Potentials (VEP).
- Abnormal brain MRI, ie. increased Loes score (measure of white matter damage, demyelination, and brain atrophy) and/or abnormal corticospinal tracts as assessed by MRI with diffusion tensor imaging (DTI).
- Three or more of the early clinical markers: problems sleeping, increased activity, behavior difficulties, seizure-like activity, chewing behavior, inappropriate bladder training, inappropriate bowel training.
- Patients must have adequate organ function as measured by:
- Renal: Serum creatinine ≤ 2.0 mg/dl
- Hepatic: Hepatic transaminases (ALT/AST) ≤ 5 x normal, bilirubin ≤ 2.0 mg/dl (except in patients with Gilbert's disease or newborns with physiological or breast milk associated jaundice).
- Cardiac: Normal cardiac function by echocardiogram or radionuclide scan (shortening fraction or ejection fraction
- 80% of normal value for age). Patients with acquired or congenital cardiomyopathy may receive melphalan as a substitute for cyclophosphamide.
- Pulmonary: Pulmonary function tests demonstrating FVC, FEV1, and DLCO ≥ 60% of predicted in patients who can complete the testing. If patient cannot perform PFT's, an O2 sat must be \>90% on room air.
- Patients must have an available, suitably matched, banked UCB unit for transplant.
- Patients must have a performance status as follows: Lansky ≥ 40%, or Karnofsky ≥ 40%
- Patients must have a life expectancy of ≥ 6 months.
- Prior organ, tissue, or stem cell transplant within 3 years of study entry.
- Prior participation in any gene or regenerative cell therapy study.
- Inability to have an MRI scan or lumbar puncture.
- Intractable seizures.
- Chronic aspiration.
- Bleeding disorder.
- Evidence of HIV infection or HIV positive serology.
- Uncontrolled bacterial, viral, or fungal infection at the time of pre-UCBT cytoreduction.
- Inability to obtain patient's, parent's or legal guardian's consent.
- Requirement of ventilatory support.
- Pregnant or breastfeeding.
- Active concurrent malignancy, or receiving concurrent radiotherapy, immunosuppressive medications, or cytotoxic chemotherapy
The study team makes the final eligibility decision.
Where it's taking place
- Durham, North Carolina, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 1 week to 22 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Durham, North Carolina, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.