Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

Related Clinical Trial
A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN) Study to Assess PXL065 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD) Study to Assess PXL770 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD) Study of Disease Progression in Adults With Inherited Forms of Spastic Paraplegia Treating Leg Symptoms in Women With X-linked Adrenoleukodystrophy Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy and Metachromatic Leukodystrophy SMART-ALD – A New Lifestyle Intervention to Improve Quality of Life in Women With X-linked Adrenoleukodystrophy (X-ALD) Quality of Life in Women With X-linked Adrenoleukodystrophy A Clinical Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT) Plasma Exchange With Albumin in AMN Patients Stem Cell Transplant for Inborn Errors of Metabolism Human Placental-Derived Stem Cell Transplantation Precision Exercise in Children With Malignant Hemopathies Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy Effect of Pioglitazone Administered to Patients With Adrenomyeloneuropathy MD1003-AMN MD1003 in Adrenomyeloneuropathy A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients. Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD) A Clinical Trial for AMN: Validation of Biomarkers of Oxidative Stress, Efficacy and Safety of a Mixture of Antioxidants Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy Repetitive Transcranial Magnetic Stimulation as Therapy in Hereditary Spastic Paraplegia and Adrenomyeloneuropathy Clinical Study and Gene Mutation Analysis of Adrenoleukodystrophy in Taiwanese Children Early Diagnosis Of Childhood Cerebral ALD Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy Multi-center Clinical Study on the Diagnosis and Treatment Management of Rare Neurological Disease in Children HSCT for High Risk Inherited Inborn Errors Longitudinal Natural History Study of Patients With Peroxisome Biogenesis Disorders (PBD) UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases The Effect of Bezafibrate on the Level of Very Long Chain Fatty Acids (VLCFA) in X-linked Adrenoleukodystrophy (X-ALD) A Phase III Trial of Lorenzo’s Oil in Adrenomyeloneuropathy Lentiviral Gene Therapy for X-ALD MT2014-14 IT-MSC for Advanced Cerebral Adrenoleukodystrophy (cALD) Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD) Newborn Screening for Adrenoleukodystrophy A Study to Prospectively Assess Disease Progression in Male Children With X-ALD Long-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy Expanded Access for Lorenzo’s Oil (GTO/GTE) in Adrenoleukodystrophy Minnesota Adrenoleukodystrophy Registry Study (MARS) and Biobank A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

Brief Title

Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

Official Title

Allogeneic Hematopoietic Stem Cell Transplantation for Standard Risk Inherited Metabolic Disorders

Brief Summary

      Rationale: Chemotherapy administration before a donor stem cell transplant is necessary to
      stop the patient's immune system from rejecting the donor's stem cells. When healthy stem
      cells from a donor are infused into the patient, the donor white blood cells can provide the
      missing enzyme that causes the metabolic disease. Sometimes the transplanted cells from a
      donor can make an immune response against the body's normal cells. Giving a monoclonal
      antibody, alemtuzumab, before transplant and cyclosporine and mycophenolate mofetil before
      and after transplant may stop this from happening. This may be an effective treatment for
      inherited metabolic disorders.

      Purpose: The design of this study is to achieve donor cell engraftment in patients with
      standard-risk inherited metabolic diseases with limited peri-transplant morbidity and
      mortality. This will be achieved through the administration of the chemotherapy regimen
      described. The intention is to follow transplanted patient for years after transplant
      monitoring them for complications of their disease and assisting families with a multifaceted
      interdisciplinary approach.

Detailed Description

      Primary Objective:

        -  To estimate the proportion of patients with donor derived engraftment at day 100 post
           transplant as defined by 80% or greater donor cells in the CD3 (T cell) fraction

      Secondary Objectives:

        -  To determine the incidence and severity of graft-versus-host disease (GVHD) by day 100

        -  To determine the incidence of peri-transplant mortality (death by day 100)

        -  To monitor donor cell chimerism at various time points following allogeneic
           transplantation with this transplant regimen as determined at day 28, 42, 100, 6 months
           and yearly for 5 years.

Study Phase

Phase 2

Study Type


Primary Outcome

Number of Patients With Donor Derived Engraftment

Secondary Outcome

 Number of Patients With Grade 0 Graft-Versus-Host Disease (GVHD)





Study Arms / Comparison Groups

 Transplant Patients
Description:  Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.


* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status


Estimated Enrollment


Start Date

December 2009

Completion Date

June 2017

Primary Completion Date

June 2015

Eligibility Criteria

        Inclusion Criteria:

          -  Must have diagnosis of one of the following: mucopolysaccharidosis disorder,
             glycoprotein metabolic disorder, sphingolipidoses or inherited leukodystrophy,
             peroxisomal disorder or other inherited diseases of metabolism

          -  Must have an acceptable graft source as defined by University of Minnesota criteria

          -  Adequate organ function

        Exclusion Criteria:

          -  Pregnant - menstruating females must have a negative serum pregnancy test within 14
             days of treatment start

          -  Evidence of human immunodeficiency virus (HIV) infection or known HIV positive




N/A - 21 Years

Accepts Healthy Volunteers



Paul Orchard, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations



Organization ID


Secondary IDs


Responsible Party


Study Sponsor

Masonic Cancer Center, University of Minnesota

Study Sponsor

Paul Orchard, MD, Principal Investigator, Masonic Cancer Center, University of Minnesota

Verification Date

September 2017