Inherited Metabolic DiseasesPhase 3UCBT-002NCT00654433
ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases
A Phase 3 study testing ALD-101 in people with Inherited Metabolic Diseases and Lysosomal Storage Disorders, run by Aldagen at 3 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 16 years
- Conditions the study is for
- Inherited Metabolic Diseases, Lysosomal Storage Disorders, Peroxisomal Storage Diseases
- What is being tested?
- ALD-101
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 3 sitesUnited States
- Study dates
- Mar 2008to Nov 2011
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 16 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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)
Show all 25Show fewer
- 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
Who cannot join
- 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 enrollment
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Duke University
Durham, North Carolina, 27705
Mattel Children's Hospital at UCLA
Los Angeles, California, 90095
Mt. Sinai Medical Center
New York, New York, 27705
About this study
Official title: A Phase III Trial of ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transplantation (UCBT) in Patients With Inborn Errors of Metabolism
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.
Read the detailed description
Subjects will be hospitalized and undergo high doses of chemotherapy which will destroy the child's normal cells including their bone marrow (which forms blood cells) in order to prepare their body for the umbilical cord blood transplant. The cord blood transplant is intended to rescue your child's bone marrow from the bad effects of the procedure. The child will receive 80% of a standard cord blood transplant followed by 20% supplemental stem cell called ALD-101.
The study will evaluate if these cells (ALD-101) will repopulate the bone marrow more rapidly after transplant. This would shorten the period of time that the transplanted subject would be at risk for infection and bleeding and would also decrease the number of red blood cell and platelet transfusions needed.
ALD-101 has been used as a supplement to cord blood transplant in twenty-eight children with genetic diseases and malignancy in one previous clinical study that is on-going.
The main purpose of this research study is to test whether a portion of cord blood cells that have been separated from a cord blood unit (ALD-101) will increase the speed at which normal levels of circulating blood cells are re-established after transplant. This is the experimental part of this study. ALD-101 is also being tested to see if it is safe.
- Conditions
- Inherited Metabolic Diseases, Lysosomal Storage Disorders, Peroxisomal Storage Diseases, Inborn Errors of Metabolism, Mucopolysaccharidosis
- Being tested
- ALD-101
- Keywords
- Hurler Syndrome, MPS I, Hurler-Scheie Syndrome, Hunter Syndrome, MPS II, Sanfilippo Syndrome, MPS III, Maroteaux-Lamy Syndrome, MPS VI, Krabbe Disease, Globoid Leukodystrophy, Metachromatic Leukodystrophy, MLD, Adrenoleukodystrophy, ALD, Adrenomyeloneuropathy, AMN, Sandhoff Disease, Tay Sachs Disease, Pelizaeus Merzbacher
- Medical subject headings
- Lysosomal Storage Diseases, Metabolism, Inborn Errors, Mucopolysaccharidoses, Mucopolysaccharidosis I, Mucopolysaccharidosis II, Sudden Infant Death, Mucopolysaccharidosis III, Mucopolysaccharidosis VI
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALD-101Biological
A subpopulation of cord blood cells composed of cells that express a high level of the intracellular enzyme aldehyde dehydrogenase (ALDH).
| Study group | Role | What they receive |
|---|---|---|
| I | Experimental | Not described Receives: Biological: ALD-101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 180 Days
Secondary outcomes (2)
To assess the efficacy of ALD-101 in accelerating neutrophil engraftment
Measured: 180 Days
To assess the safety of adjuvant therapy of ALD-101 in infusional toxicity, adverse events, and primary graft failure.
Measured: 180 Days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Apr 2008 (estimated)
- Study startMar 2008
- Primary completionJul 2011
- Study completionNov 2011
Why it stopped: Terminated - Sponsor Decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Inherited Metabolic DiseasesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Aldagen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- James Hinson, MD, Study director, AldagenJoanne Kurtzberg, MD, Principal investigator, Duke University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00654433
- Sponsor's protocol ID
- UCBT-002
- Other IDs
- BB-IND#13502
- Acronym
- UCBT-002
- Status last verified
- Jul 2014
- First submitted
- 3 Apr 2008
- Last updated
- 8 Jul 2014
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Prasad VK, Kurtzberg J. Emerging trends in transplantation of inherited metabolic diseases. Bone Marrow Transplant. 2008 Jan;41(2):99-108. doi: 10.1038/sj.bmt.1705970. Epub 2008 Jan 7.
PubMed 18176609Martin PL, Carter SL, Kernan NA, Sahdev I, Wall D, Pietryga D, Wagner JE, Kurtzberg J. Results of the cord blood transplantation study (COBLT): outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with lysosomal and peroxisomal storage diseases. Biol Blood Marrow Transplant. 2006 Feb;12(2):184-94. doi: 10.1016/j.bbmt.2005.09.016.
PubMed 16443516Escolar ML, Poe MD, Martin HR, Kurtzberg J. A staging system for infantile Krabbe disease to predict outcome after unrelated umbilical cord blood transplantation. Pediatrics. 2006 Sep;118(3):e879-89. doi: 10.1542/peds.2006-0747. Epub 2006 Aug 21.
PubMed 16923928Gentry T, Deibert E, Foster SJ, Haley R, Kurtzberg J, Balber AE. Isolation of early hematopoietic cells, including megakaryocyte progenitors, in the ALDH-bright cell population of cryopreserved, banked UC blood. Cytotherapy. 2007;9(6):569-76. doi: 10.1080/14653240701466347.
PubMed 17882722
Study at a glance
From the registry record, last updated by the sponsor on 8 Jul 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase III Trial of ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transplantation (UCBT) in Patients With Inborn Errors of Metabolism
- ClinicalTrials.gov ID
- NCT00654433
- Acronym / sponsor ID
- UCBT-002 / UCBT-002
- Conditions
- Inherited Metabolic Diseases, Lysosomal Storage Disorders, Peroxisomal Storage Diseases, Inborn Errors of Metabolism, Mucopolysaccharidosis
- Keywords
- Hurler Syndrome, MPS I, Hurler-Scheie Syndrome, Hunter Syndrome, MPS II, Sanfilippo Syndrome, MPS III, Maroteaux-Lamy Syndrome
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Terminated - Sponsor Decision
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- AldagenIndustry (pharma, biotech, devices)
- Study start
- Mar 2008
- Primary completion
- Jul 2011 (actual)
- Study completion
- Nov 2011 (actual)
- First posted
- 8 Apr 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Subjects will be hospitalized and undergo high doses of chemotherapy which will destroy the child's normal cells including their bone marrow (which forms blood cells) in order to prepare their body for the umbilical cord blood transplant. The cord blood transplant is intended to rescue your child's bone marrow from the bad effects of the procedure. The child will receive 80% of a standard cord blood transplant followed by 20% supplemental stem cell called ALD-101.
The study will evaluate if these cells (ALD-101) will repopulate the bone marrow more rapidly after transplant. This would shorten the period of time that the transplanted subject would be at risk for infection and bleeding and would also decrease the number of red blood cell and platelet transfusions needed.
ALD-101 has been used as a supplement to cord blood transplant in twenty-eight children with genetic diseases and malignancy in one previous clinical study that is on-going.
The main purpose of this research study is to test whether a portion of cord blood cells that have been separated from a cord blood unit (ALD-101) will increase the speed at which normal levels of circulating blood cells are re-established after transplant. This is the experimental part of this study. ALD-101 is also being tested to see if it is safe.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| I | Experimental | Biological: ALD-101 |
Who may qualify
- Ages
- Up to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (25 listed)
- confirmed diagnosis of inherited metabolic diseases; including the following:
- 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
Key exclusion criteria (5 listed)
- 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 enrollment
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ALD-101A subpopulation of cord blood cells composed of cells that express a high level of the intracellular enzyme aldehyde dehydrogenase (ALDH). | Biological | I |
Primary outcome
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
Time frame: 180 Days
2 secondary outcomes
To assess the efficacy of ALD-101 in accelerating neutrophil engraftment
Time frame: 180 Days
To assess the safety of adjuvant therapy of ALD-101 in infusional toxicity, adverse events, and primary graft failure.
Time frame: 180 Days
Results and safety
The study was stopped early (reason given: Terminated - Sponsor Decision). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jul 2014.
- Background: PubMed 18176609Prasad VK, Kurtzberg J. Emerging trends in transplantation of inherited metabolic diseases. Bone Marrow Transplant. 2008 Jan;41(2):99-108. doi: 10.1038/sj.bmt.1705970. Epub 2008 Jan 7.
- Background: PubMed 16443516Martin PL, Carter SL, Kernan NA, Sahdev I, Wall D, Pietryga D, Wagner JE, Kurtzberg J. Results of the cord blood transplantation study (COBLT): outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with lysosomal and peroxisomal storage diseases. Biol Blood Marrow Transplant. 2006 Feb;12(2):184-94. doi: 10.1016/j.bbmt.2005.09.016.
- Background: PubMed 16923928Escolar ML, Poe MD, Martin HR, Kurtzberg J. A staging system for infantile Krabbe disease to predict outcome after unrelated umbilical cord blood transplantation. Pediatrics. 2006 Sep;118(3):e879-89. doi: 10.1542/peds.2006-0747. Epub 2006 Aug 21.
- Background: PubMed 17882722Gentry T, Deibert E, Foster SJ, Haley R, Kurtzberg J, Balber AE. Isolation of early hematopoietic cells, including megakaryocyte progenitors, in the ALDH-bright cell population of cryopreserved, banked UC blood. Cytotherapy. 2007;9(6):569-76. doi: 10.1080/14653240701466347.
- Sponsor's WebsiteLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
James Hinson, MD study director
Aldagen
Joanne Kurtzberg, MD principal investigator
Duke University
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing ALD-101 in people with Inherited Metabolic Diseases and Lysosomal Storage Disorders, run by Aldagen at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 25 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 8 Jul 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Terminated - Sponsor Decision). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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 (time frame: 180 Days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00654433, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jul 2014.