Inherited Metabolic Storage DiseasesPhase 1/2NCT00056979
Monoclonal Antibody Conditioning for Allogeneic Stem Cell Transplantation of Patients With Inherited Metabolic Storage Diseases
A Phase 1/2 study testing CAMPATH-1H, Anti-CD45 and FK506 in people with Inherited Metabolic Storage Diseases, run by Baylor College of Medicine at 2 sites in United States.
Trial summary
This study treats patients with an inherited disease that prevents the body from making a specific protein or enzyme needed for the body's metabolism. Lack of this enzyme causes accumulation of harmful or toxic substances in the body, which leads to deterioration and failure of organs such as the brain or the heart. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Inherited Metabolic Storage Diseases
- What is being tested?
- CAMPATH-1H, Anti-CD45 and FK506
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 2 sitesUnited States
- Study dates
- Jun 2002to Jun 2003
- Phase
- Phase 1/2
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients with inherited metabolic storage diseases of all ages are eligible.
- Diagnosis of inherited metabolic storage disease confirmed by standard biochemical and genetic studies in consultation with the Department of Genetics at the Baylor College of Medicine
- Inherited metabolic storage diseases which may be stabilized or improved by stem cell transplantation include: Hurler, Hunter, Maroteaux-Lamy, Sly, Wolman, Gaucher, Farber, Nieman-Pick, Mannosidosis, Aspartylglucosaminuria, Fucosidosis, Neuronal Ceroid-Lipofuscinosis, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, and Adrenoleukodystrophy
- Availability of an HLA matched or mismatched (up to one haplotype) donor who is not an obligate carrier for the inherited condition or an unrelated HLA matched stem cell donor. Fully matched is defined as 6/6 match by high resolution DR based DNA typing.
- Female patients of childbearing age must have a negative pregnancy test and be willing to use an effective means of birth control.
Who cannot join
- Patients with a life expectancy (<6 weeks) limited by diseases other than inherited metabolic storage disease
- Patients with advanced inherited metabolic storage disease, which has not been stabilized or improved by hematopoietic stem cell transplantation.
- Patients with symptomatic cardiac disease, or evidence of significant cardiac disease by echocardiogram (i.e., shortening fraction <25%)
- Patients with severe renal disease (Creatinine >2 x normal for age)
- Patients with known allergy to rat serum products
- Patients with a Karnofsky or Lansky score <50%.
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- Patients with a severe infection that on evaluation by the Principal Investigator precludes ablative chemotherapy or successful transplantation
- Patients with severe personality disorder or mental illness or neuropsychological evaluation indicating too much damage for the transplant to be of benefit.
- Patients with documented HIV positivity.
- Patients with grade III-IV liver toxicity not related to metabolic storage disease.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Texas Children's Hospital
Houston, Texas
The Methodist Hospital
Houston, Texas
About this study
Official title: Anti-CD45 (YTH-24 & YTH 54) and ANTI-CD52 (CAMPATH-1H) Monoclonal Antibody Conditioning Regimen for Allogeneic Stem Cell Transplantation of Patients With Inherited Metabolic Storage Diseases
This study treats patients with an inherited disease that prevents the body from making a specific protein or enzyme needed for the body's metabolism. Lack of this enzyme causes accumulation of harmful or toxic substances in the body, which leads to deterioration and failure of organs such as the brain or the heart. This disease can be fatal.
Some patients with inherited metabolic storage disease may benefit from an allogeneic stem cell transplant ('allogeneic' means that the stem cells come from another person). Stem cells are created in the bone marrow. They mature into different types of blood cells that are needed including red blood cells, white blood cells, and platelets. Stem cells, when transplanted, can make a new blood system. Donor stem cells can make the protein or enzyme patients with this disease cells cannot. The donor cells may prevent further accumulation of toxic substances. It is hoped that the donor cells can prevent or stop the disease from progressing.
This research study uses a new pre-treatment combination of two drugs, Anti-CD45 and CAMPATH-1H. Anti-CD45 and CAMPATH-1H are antibodies against certain types of blood cells. CAMPATH-1H is particularly important because it stays active in the body for a long time after infusion, which means it may work longer at preventing GVHD symptoms. In addition to antibodies, patients will receive Fludarabine, which is a chemotherapy drug. Fludarabine kills bone marrow cells and is given to reduce the bone marrow cells so that donor stem cells may 'take.'
Read the detailed description
Fludarabine will be given as a daily IV (intravenous, by vein) infusion for a total of 5 days. CAMPATH-1H will be given as a daily 4-hour IV (intravenous, by vein) infusion for three days. Anti-CD45 will be given as a daily 6-hour IV infusion over the next 4 days. Then patients will have a one-day rest period before receiving the stem cell transplant. To help prevent the body from rejecting the transplant, patients will also receive the drug FK506, starting two days before the transplant and continuing for three months. If there is no GVHD, the amount of FK506 patients are taking will be reduced by 20% every 2 weeks until this medication is stopped.
- Conditions
- Inherited Metabolic Storage Diseases
- Being tested
- CAMPATH-1H, Anti-CD45, FK506, Fludarabine
- Keywords
- Metabolic diseases
- Medical subject headings
- Metabolic Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CAMPATH-1HDrug
Anti-CD45Drug
FK506Drug
FludarabineDrug
| Study group | Role | What they receive |
|---|---|---|
| Fludarabine, CAMPATH-1H , Anti-CD45, FK506 | Experimental | Fludarabine will be given as a daily IV (intravenous, by vein) infusion for a total of 5 days. CAMPATH-1H will be given as a daily 4-hour IV (intravenous, by vein) infusion for three days. Anti-CD45 will be given as a daily 6-hour IV infusion over the next 4 days. To help prevent body from rejecting the transplant, the drug FK506 will be given, starting two days before the transplant and continuing for three months. Receives: Drug: CAMPATH-1H; Drug: Anti-CD45; Drug: FK506; Drug: Fludarabine |
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Mar 2003 (estimated)
- Study startJun 2002
- Primary completionJun 2003
- Study completionJun 2003
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Inherited Metabolic Storage DiseasesStudies of the same condition, open and under way.
- More trials run by Baylor College of MedicineThe sponsor's other registered studies.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baylor College of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- The Methodist Hospital Research Institute
- Responsible party
- Principal investigator, Robert Krance, Baylor College of Medicine
- Investigators
- Malcolm K Brenner, MD, Study chair, Baylor College of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00056979
- Sponsor's protocol ID
- H11909
- Other IDs
- MAID
- Status last verified
- Sep 2015
- First submitted
- 26 Mar 2003
- Last updated
- 23 Sep 2015
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Sep 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Anti-CD45 (YTH-24 & YTH 54) and ANTI-CD52 (CAMPATH-1H) Monoclonal Antibody Conditioning Regimen for Allogeneic Stem Cell Transplantation of Patients With Inherited Metabolic Storage Diseases
- ClinicalTrials.gov ID
- NCT00056979
- Acronym / sponsor ID
- H11909
- Conditions
- Inherited Metabolic Storage Diseases
- Keywords
- Metabolic diseases
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Baylor College of MedicineUniversity, hospital or other
- Collaborators
- The Methodist Hospital Research Institute
- Study start
- Jun 2002
- Primary completion
- Jun 2003 (actual)
- Study completion
- Jun 2003 (actual)
- First posted
- 27 Mar 2003 (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.
This study treats patients with an inherited disease that prevents the body from making a specific protein or enzyme needed for the body's metabolism. Lack of this enzyme causes accumulation of harmful or toxic substances in the body, which leads to deterioration and failure of organs such as the brain or the heart. This disease can be fatal.
Some patients with inherited metabolic storage disease may benefit from an allogeneic stem cell transplant ('allogeneic' means that the stem cells come from another person). Stem cells are created in the bone marrow. They mature into different types of blood cells that are needed including red blood cells, white blood cells, and platelets. Stem cells, when transplanted, can make a new blood system. Donor stem cells can make the protein or enzyme patients with this disease cells cannot. The donor cells may prevent further accumulation of toxic substances. It is hoped that the donor cells can prevent or stop the disease from progressing.
This research study uses a new pre-treatment combination of two drugs, Anti-CD45 and CAMPATH-1H. Anti-CD45 and CAMPATH-1H are antibodies against certain types of blood cells. CAMPATH-1H is particularly important because it stays active in the body for a long time after infusion, which means it may work longer at preventing GVHD symptoms. In addition to antibodies, patients will receive Fludarabine, which is a chemotherapy drug. Fludarabine kills bone marrow cells and is given to reduce the bone marrow cells so that donor stem cells may 'take.'
Detailed description
Fludarabine will be given as a daily IV (intravenous, by vein) infusion for a total of 5 days. CAMPATH-1H will be given as a daily 4-hour IV (intravenous, by vein) infusion for three days. Anti-CD45 will be given as a daily 6-hour IV infusion over the next 4 days. Then patients will have a one-day rest period before receiving the stem cell transplant. To help prevent the body from rejecting the transplant, patients will also receive the drug FK506, starting two days before the transplant and continuing for three months. If there is no GVHD, the amount of FK506 patients are taking will be reduced by 20% every 2 weeks until this medication is stopped.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Fludarabine, CAMPATH-1H , Anti-CD45, FK506 | Experimental | Drug: CAMPATH-1H, Drug: Anti-CD45, Drug: FK506, Drug: FludarabineFludarabine will be given as a daily IV (intravenous, by vein) infusion for a total of 5 days. CAMPATH-1H will be given as a daily 4-hour IV (intravenous, by vein) infusion for three days. Anti-CD45 will be given as a daily 6-hour IV infusion over the next 4 days. To help prevent body from rejecting the transplant, the drug FK506 will be given, starting two days before the transplant and continuing for three months. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Patients with inherited metabolic storage diseases of all ages are eligible.
- Diagnosis of inherited metabolic storage disease confirmed by standard biochemical and genetic studies in consultation with the Department of Genetics at the Baylor College of Medicine
- Inherited metabolic storage diseases which may be stabilized or improved by stem cell transplantation include: Hurler, Hunter, Maroteaux-Lamy, Sly, Wolman, Gaucher, Farber, Nieman-Pick, Mannosidosis, Aspartylglucosaminuria, Fucosidosis, Neuronal Ceroid-Lipofuscinosis, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, and Adrenoleukodystrophy
- Availability of an HLA matched or mismatched (up to one haplotype) donor who is not an obligate carrier for the inherited condition or an unrelated HLA matched stem cell donor. Fully matched is defined as 6/6 match by high resolution DR based DNA typing.
- Female patients of childbearing age must have a negative pregnancy test and be willing to use an effective means of birth control.
Key exclusion criteria (10 listed)
- Patients with a life expectancy (<6 weeks) limited by diseases other than inherited metabolic storage disease
- Patients with advanced inherited metabolic storage disease, which has not been stabilized or improved by hematopoietic stem cell transplantation.
- Patients with symptomatic cardiac disease, or evidence of significant cardiac disease by echocardiogram (i.e., shortening fraction <25%)
- Patients with severe renal disease (Creatinine >2 x normal for age)
- Patients with known allergy to rat serum products
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 |
|---|---|---|
| CAMPATH-1H | Drug | Fludarabine, CAMPATH-1H , Anti-CD45, FK506 |
| Anti-CD45 | Drug | Fludarabine, CAMPATH-1H , Anti-CD45, FK506 |
| FK506 | Drug | Fludarabine, CAMPATH-1H , Anti-CD45, FK506 |
| Fludarabine | Drug | Fludarabine, CAMPATH-1H , Anti-CD45, FK506 |
Primary outcome
Not reported in the source record.
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Sep 2015.
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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.
Malcolm K Brenner, MD study chair
Baylor College of Medicine
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing CAMPATH-1H, Anti-CD45 and FK506 in people with Inherited Metabolic Storage Diseases, run by Baylor College of Medicine at 2 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 5 inclusion and 10 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 23 Sep 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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. 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.
Where can I read the original study information?
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 23 Sep 2015.