Metachromatic LeukodystrophyBoostNCT02021266
Single Patient Expanded Access Protocol: Metabolic Boost
A study testing Enriched Hematopoetic Stem Cell Infusion in people with Metachromatic Leukodystrophy, run by Talaris Therapeutics Inc. at 1 site in United States.
Trial summary
This is a single patient expanded access protocol to investigate the effects of a second dose of facilitating cell-enhanced hematopoietic stem cell product. Read more about this study
- Who is it for?
- Any age, women only
- Conditions the study is for
- Metachromatic Leukodystrophy
- What is being tested?
- Enriched Hematopoetic Stem Cell Infusion
- Type of treatment
- Injection or infusion
- How many people
- Not given
- Where
- 1 siteUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- 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
- Women only
- Child, Adult, Older adult
Who may be able to join
- Subject was previously enrolled and qualified for transplant under IND 14070. Subject must be free from infection and have normal liver, kidney, heart and pulmonary function to proceed to a second transplant.
- Patient must have adequate function of other organ systems as measured by:
- Creatinine < 2.0 mg/dl and creatinine clearance ≥60 cc/min/1.73m2. Newborns must have a creatinine clearance > 25 cc/min. For babies < 3 months of age, the raw value on GFR must be > 1 cc/kg/min.
- Hepatic transaminases (ALT/AST) 2.5 x normal, bilirubin <2.0mg/dl
- Normal cardiac function by echocardiogram or radionuclide scan (ejection fraction or shortening fraction >80% of normal value for age)
- Pulmonary function tests (PFT) demonstrating forced expiratory volume at one second (FEV1) of >50% of predicted for age. If child is too young or unable to perform PFTs, crying vital capacity result of >50% of normal value for age or resting pulse oximeter >92% on room air or clearance by pulmonologist will be required.
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- Patient, and parent, or legal guardian must have given written informed consent according to FDA guidelines.
- Patient must have a minimum life expectancy of at least 6 months.
- Female patients of childbearing potential cannot be pregnant or lactating/breast-feeding and must be either surgically sterile, postmenopausal (no menses for the previous 12 months), or must be practicing an effective method of birth control as determined by the investigator (e.g., oral contraceptives, double barrier methods, hormonal injectable or implanted contraceptives, tubal ligation, or partner with vasectomy).
- Recipient screening to include glomerular filtration rate (GFR), chest X-ray (CXR), hepatic and renal chemistries, coagulation studies, pulmonary function testing, and ECHO if clinically relevant, chimerism testing, type and screen, and enzyme levels within 30 days of retransplant.
Who cannot join
- Uncontrolled seizures, apnea, evidence of recurrent or uncontrolled aspiration, or need for chronic mechanical ventilation.
- Subject must not have had previous radiation therapy that would preclude total body irradiation (TBI) (as determined by radiation therapist)
- Uncontrolled infection or severe concomitant diseases, which in the judgment of the Principal Investigator, could not tolerate reduced intensity transplantation.
- Subject with a positive human immunodeficiency virus (HIV) antibody test result
- Subject who are pregnant, as indicated by a positive serum human chorionic gonadotropin (HCG) test
- Subject whose only donor is pregnant at the time of intended transplant
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- Subject of childbearing potential who are not practicing adequate contraception as defined by the investigator at the site
- Jehovah's witnesses being unwilling to be transfused
- Patient that have any comorbid condition which, in the view of the Principal Investigators, renders the patient at too high a risk from treatment complications and regimen related morbidity/mortality.
- Insufficient funds for bone marrow processing.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Single Patient Expanded Access Protocol: Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
This is a single patient expanded access protocol to investigate the effects of a second dose of facilitating cell-enhanced hematopoietic stem cell product.
Read the detailed description
Hematopoietic stem cell transplantation (HSCT) has been established as an important therapeutic option for patients with a variety of inherited metabolic disorders (IMD). The potential life-threatening complications of conventional myeloablative HSCT have limited its application. Additionally, conventional HSCT is only available to the small minority of medically suitable candidates who have histocompatibility leukocyte antigen (HLA)-identical siblings to donate bone marrow or mobilized peripheral blood stem cells.
Donor mobilized peripheral blood stem cells or bone marrow will be processed via a novel technology to deplete mature immune cells while retaining hematopoietic stem cells (HSC) and graft facilitating cells (FC).
A now-standard reduced intensity, nonmyeloablative recipient conditioning regimen will be used to promote mixed allogeneic chimerism, thereby significantly reducing morbidity and mortality.
These two enhancements are intended to significantly improve the benefit:risk ratio of HSCT for patients with IMDs. If successful, transplantation will become a more feasible option for patients without HLA-identical siblings to donate stem cells, and could be offered to patients early in disease progression.
The objective for the study is to establish chimerism following reduced intensity conditioning with no grade III/IV graft-versus-host disease (GVHD). The primary endpoint we will follow is production of the missing enzyme at ≥ 10% of the normal level at day 180 post-transplant as for investigational new drug (IND) 14070. This expanded access protocol details the approach to providing a second dose of the product.
- Conditions
- Metachromatic Leukodystrophy
- Being tested
- Enriched Hematopoetic Stem Cell Infusion
- Medical subject headings
- Leukodystrophy, Metachromatic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Enriched Hematopoetic Stem Cell InfusionBiological
Enriched Hematopoetic Stem Cell Infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Dec 2013 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Metachromatic LeukodystrophyStudies 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
- Expanded access
Sponsor and investigators
- Lead sponsor
- Talaris Therapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Duke University
- Responsible party
- Sponsor
- Investigators
- Suzanne T Ildstad, MD, Study director, Talaris Therapeutics Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02021266
- Sponsor's protocol ID
- ICT-14070-120611-Exp Access
- Acronym
- Boost
- Status last verified
- Oct 2020
- First submitted
- 19 Dec 2013
- Last updated
- 8 Oct 2020
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Oct 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Single Patient Expanded Access Protocol: Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
- ClinicalTrials.gov ID
- NCT02021266
- Acronym / sponsor ID
- Boost / ICT-14070-120611-Exp Access
- Conditions
- Metachromatic Leukodystrophy
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- Talaris Therapeutics Inc.Industry (pharma, biotech, devices)
- Collaborators
- Duke University
- First posted
- 27 Dec 2013 (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 is a single patient expanded access protocol to investigate the effects of a second dose of facilitating cell-enhanced hematopoietic stem cell product.
Detailed description
Hematopoietic stem cell transplantation (HSCT) has been established as an important therapeutic option for patients with a variety of inherited metabolic disorders (IMD). The potential life-threatening complications of conventional myeloablative HSCT have limited its application. Additionally, conventional HSCT is only available to the small minority of medically suitable candidates who have histocompatibility leukocyte antigen (HLA)-identical siblings to donate bone marrow or mobilized peripheral blood stem cells.
Donor mobilized peripheral blood stem cells or bone marrow will be processed via a novel technology to deplete mature immune cells while retaining hematopoietic stem cells (HSC) and graft facilitating cells (FC).
A now-standard reduced intensity, nonmyeloablative recipient conditioning regimen will be used to promote mixed allogeneic chimerism, thereby significantly reducing morbidity and mortality.
These two enhancements are intended to significantly improve the benefit:risk ratio of HSCT for patients with IMDs. If successful, transplantation will become a more feasible option for patients without HLA-identical siblings to donate stem cells, and could be offered to patients early in disease progression.
The objective for the study is to establish chimerism following reduced intensity conditioning with no grade III/IV graft-versus-host disease (GVHD). The primary endpoint we will follow is production of the missing enzyme at ≥ 10% of the normal level at day 180 post-transplant as for investigational new drug (IND) 14070. This expanded access protocol details the approach to providing a second dose of the product.
Study design
Who may qualify
- Sex
- Female only
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- Subject was previously enrolled and qualified for transplant under IND 14070. Subject must be free from infection and have normal liver, kidney, heart and pulmonary function to proceed to a second transplant.
- Patient must have adequate function of other organ systems as measured by:
- Patient, and parent, or legal guardian must have given written informed consent according to FDA guidelines.
- Patient must have a minimum life expectancy of at least 6 months.
- Female patients of childbearing potential cannot be pregnant or lactating/breast-feeding and must be either surgically sterile, postmenopausal (no menses for the previous 12 months), or must be practicing an effective method of birth control as determined by the investigator (e.g., oral contraceptives, double barrier methods, hormonal injectable or implanted contraceptives, tubal ligation, or partner with vasectomy).
Key exclusion criteria (10 listed)
- Uncontrolled seizures, apnea, evidence of recurrent or uncontrolled aspiration, or need for chronic mechanical ventilation.
- Subject must not have had previous radiation therapy that would preclude total body irradiation (TBI) (as determined by radiation therapist)
- Uncontrolled infection or severe concomitant diseases, which in the judgment of the Principal Investigator, could not tolerate reduced intensity transplantation.
- Subject with a positive human immunodeficiency virus (HIV) antibody test result
- Subject who are pregnant, as indicated by a positive serum human chorionic gonadotropin (HCG) test
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 |
|---|---|---|
| Enriched Hematopoetic Stem Cell InfusionEnriched Hematopoetic Stem Cell Infusion | Biological | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Oct 2020.
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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.
Suzanne T Ildstad, MD study director
Talaris Therapeutics Inc.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Enriched Hematopoetic Stem Cell Infusion in people with Metachromatic Leukodystrophy, run by Talaris Therapeutics Inc. at 1 site 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 10 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: status not confirmed, as of the sponsor's last update on 8 Oct 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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.
Are results available for this study?
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 8 Oct 2020.