DUOC-01 for Adrenoleukodystrophy

Phase-Based Progress Estimates
Duke University Medical Center, Durham, NCAdrenoleukodystrophy+11 MoreDUOC-01 - Biological
1 - 22
All Sexes
What conditions do you have?

Study Summary

This trial is testing a new treatment for inborn errors of metabolism that includes transplanting stem cells from umbilical cords and injecting them into the spinal cord. The goal is to see if this is a safe and effective treatment for early demyelinating disease in the central nervous system.

Eligible Conditions
  • Adrenoleukodystrophy
  • Batten Disease
  • Hunter Syndrome
  • Krabbe Disease
  • Metachromatic Leukodystrophy
  • Niemann-Pick Disease
  • Sandhoff Disease
  • Tay-Sachs Disease
  • Inborn Metabolic Brain Diseases
  • Alpha-Mannosidosis
  • Sanfilippo Syndrome
  • Pelizaeus-Merzbacher Disease

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

2 Primary · 1 Secondary · Reporting Duration: 1-5 years

1 month after infusion
Evaluate for Neuro Toxicity
1-5 years
Efficacy determination
24 hours after infusion
Evaluate for Infusional Toxicity

Trial Safety

Safety Progress

1 of 3

Trial Design

1 Treatment Group

Intrathecal administration of DUOC-01
1 of 1

Experimental Treatment

40 Total Participants · 1 Treatment Group

Primary Treatment: DUOC-01 · No Placebo Group · Phase 1

Intrathecal administration of DUOC-01
Experimental Group · 1 Intervention: DUOC-01 · Intervention Types: Biological

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 1-5 years

Who is running the clinical trial?

Joanne Kurtzberg, MDLead Sponsor
18 Previous Clinical Trials
703 Total Patients Enrolled
The Marcus FoundationOTHER
15 Previous Clinical Trials
1,492 Total Patients Enrolled

Eligibility Criteria

Age 1 - 22 · All Participants · 8 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You do not have a brother, sister, or other relative who is a suitable bone marrow donor.

Frequently Asked Questions

Does this medical trial permit participation from individuals aged 70 or older?

"This research is searching for participants aged between 1 Week and 22 years." - Anonymous Online Contributor

Unverified Answer

Who has the opportunity to partake in this investigation?

"For this investigation, 40 individuals with Tay-Sachs disease between the ages of 1 week and 22 are being accepted. To qualify for participation in the trial, potential candidates must meet a number of stringent criteria such as having an abnormal EEG or brain MRI; displaying three or more early clinical markers like sleep issues or behavioural difficulties; renal performance indicated by serum creatinine levels under 2.0mg/dl; hepatic functioning suggested by transaminases (ALT/AST) lower than 5x normal and bilirubin less than 2.0 mg/dl (unless they have Gilbert's Disease); cardiac health established through ech" - Anonymous Online Contributor

Unverified Answer

Is this research endeavor presently recruiting participants?

"Affirmative. Clinicaltrials.gov indicates that this trial is currently searching for participants, having been first posted on September 1st 2014 with the most recent update being made October 24th 2022. 40 patients are expected to be enrolled from one site." - Anonymous Online Contributor

Unverified Answer

Has DUOC-01 been certified by the FDA?

"Our safety assessment of DUOC-01 gave it a score of 1, which indicates that the clinical evidence regarding this medication's efficacy and safety is quite limited." - Anonymous Online Contributor

Unverified Answer

What is the scale of this research trial in terms of participants?

"Affirmative, the information presented on clinicaltrials.gov indicates that recruitment for this trial is currently underway. This experiment was first posted in September 2014 and most recently updated October 24th 2022. It requires 40 participants to be recruited from 1 site." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.