EUCTR2011-001953-10-DE
Active, not recruiting
Phase 1
A phase 2/3 study of the efficacy and safety of hematopoietic stem cells transduced with Lenti D lentiviral vector for the treatment of cerebral adrenoleukodystrophy (CALD)
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- Cerebral Adrenoleukodystrophy (CALD)
- Sponsor
- bluebird bio, Inc.
- Enrollment
- 32
- Status
- Active, not recruiting
- Last Updated
- 5 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •1\. Informed consent is obtained from a competent custodial parent or guardian with legal capacity to execute a local IRB/Independent Ethics Committee (IEC) approved consent (informed assent will be sought from capable subjects, in accordance with the directive of the IRB/IEC and with local requirements).
- •2\. Males aged 17 years and younger, at the time of parental/guardian consent and, where appropriate, subject assent.
- •3\. Active cerebral ALD as defined by:
- •\-Elevated VLCFA values, and
- •\-Active CNS disease established by central radiographic review
- •of brain MRI demonstrating:
- •i. Loes score between 0\.5 and 9 (inclusive) on the 34\-point scale, and
- •ii. Gadolinium enhancement on MRI of demyelinating lesions.
- •4\. Neurological Function Score (NFS) \= 1\.
- •Are the trial subjects under 18? yes
Exclusion Criteria
- •1\. Receipt of an allogeneic transplant or gene therapy.
- •2\. Availability of a willing 10/10 HLA\-matched sibling donor (excluding female heterozygotes).
- •3\. Use of statins, Lorenzo's Oil, or dietary regimens used to lower VLCFA of consent.
- •4\. Receipt of an investigational study drug or procedure within 3 months before Screening that might confound study outcomes. Use of investigational study drugs is prohibited throughout the course of the study.
- •5\. Any conditions that make it impossible to perform MRI studies (including allergies to anesthetics or contrast agents).
- •6\. Hematological compromise as evidenced by:
- •\- Peripheral blood ANC count \< 1500 cells/mm3,
- •\- Platelet count \< 100,000 cells/mm3, or
- •\- Hemoglobin \< 10 g/dL.
- •\- Uncorrected bleeding disorder.
Outcomes
Primary Outcomes
Not specified
Similar Trials
Active, not recruiting
Phase 1
Clinical study to assess the efficacy and safety of gene therapy for the treatment of childhood cerebral adrenoleukodystrophyChildhood Cerebral Adrenoleukodystrophy (CCALD)MedDRA version: 18.0Level: PTClassification code 10051260Term: AdrenoleukodystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]EUCTR2011-001953-10-FRbluebird bio, Inc.15
Not yet recruiting
Phase 2
A clinical study of a herbal formulation DRDC/2022/040 on symptoms of Gastroesophageal reflux disease (GERD)CTRI/2023/08/056221Dabur Research Development Centre Dabur India Limited
Completed
Not Applicable
A Pilot Study to Evaluate Safety and Efficacy of HLA-mismatched Granulocyte Transfusions in the Treatment of Patients with Solid Malignant Tumors Who Are Resistant to Standard Cancer Therapy or for Whom No Standard Cancer Therapy ExistsHealth Condition 1: null- CANCERCTRI/2012/11/003116Munogenics Inc USA22
Completed
Not Applicable
Phase III study on the efficacy and safety of Hypericum extract WS® 5570 in patients with a Major Depressive EpisodeMajor depressive disorderMental and Behavioural DisordersDepressionISRCTN77277298Schwabe Pharmaceuticals (Germany)320
Completed
Not Applicable
Clinical exploratory study on the usefulness of hemodynamic preloading parameters using noninvasive cardiac output measuring device (ClearSight) for perioperative fluid managementJPRN-UMIN000025497Hokkaido Universtiy Graduate School of Medicine, Department of Gastroenterological SurgeryII18