ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With In… (NCT00654433) | Clinical Trial Compass
TerminatedPhase 3
ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases
Stopped: Terminated - Sponsor Decision
United States40 participantsStarted 2008-03
Plain-language summary
Eligible research subjects will receive an unrelated umbilical cord blood transfusion as a possible cure for their inherited metabolic disease. A portion of cord blood cells (ALD-101) will be separated from the cord blood unit and given approximately 4 hours after the standard cord blood transfusion.
The study will test if the supplemental cells will increase the speed at which normal levels of circulating blood cells are re-established after transplant.
Who can participate
Age range
16 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* confirmed diagnosis of inherited metabolic diseases; including the following:
* Hurler Syndrome (MPS I)
* Hurler-Scheie Syndrome
* Hunter Syndrome (MPS II)
* Sanfilippo Syndrome A and B(MPS III)
* Maroteaux-Lamy Syndrome (MPS VI)
* Krabbe Disease (Globoid Leukodystrophy)
* Metachromatic Leukodystrophy (MLD)
* Adrenoleukodystrophy (ALD and AMN)
* Sandhoff Disease
* Tay Sachs Disease
* Pelizaeus Merzbacher (PMD)
* Niemann-Pick Disease
* Alpha-mannosidosis
* I-Cell Disease (ML II)
* Fucosidosis
* GM I Gangliosidosis
* Canavan Disease
* must be \<16 years of age at the time of study enrollment
* must have a good performance status (Lansky ≥80%)
* must have adequate function of other organ systems including: kidney, liver, heart and lungs
* must have given valid written informed consent
* must have a minimum life expectancy of at least 6 months
* must be determined to be a good candidate for a standard umbilical cord blood transplant
* must have an IQ \>70 or if too young for IQ testing the potential to reach this endpoint by age 5
Exclusion Criteria:
* HIV, Hepatitis B and/or Hepatitis C positive
* concurrently involved in any other clinical study that affects engraftment or immune reconstitution
* uncontrolled seizures, apnea, evidence of aspiration pneumonia, or evidence of brain stem involvement
* uncontrolled infections
* prior allogeneic stem cell transplant with cytoreduction preparative therapy within 12 months of…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
To assess the efficacy of adjuvant therapy of ALD-101 in accelerating platelet engraftment in patients also receiving a standard unrelated UCBT for treatment of inherited metabolic diseases