Status:
COMPLETED
AAVRh.10 Administered to Children With Late Infantile Neuronal Ceroid Lipofuscinosis
Lead Sponsor:
Weill Medical College of Cornell University
Conditions:
Late Infantile Neuronal Ceroid Lipofuscinosis
Batten Disease
Eligibility:
All Genders
3-18 years
Phase:
PHASE1
PHASE2
Brief Summary
The investigators propose to assess the safety and efficacy of a new administration method to deliver a biologic to children with a form of Batten disease using an experimental gene transfer procedure...
Detailed Description
This study is designed to run parallel to our currently IRB approved protocol #0810010013 entitled "Direct CNS Administration of a Replication Deficient Adeno-associated Virus Gene Transfer Vector Ser...
Eligibility Criteria
Inclusion
- Inclusion criteria.
- Definitive diagnosis of LINCL, based on clinical phenotype and genotype. The study does not limit to one specific genotype (genetic constitution).
- The subject must be between the age of 3 and 18 years.
- Subjects will have an average total score of less than 4 but at least 1, and/or an uncommon genotype defined as any genotype that does not include at least one of the 5 most common mutant CLN2 genotypes: C3670T (nonsense Arg208 to stop), G3556C (intron 7 splice), G5271C (Gln422His), T4396G (aberrant splicing, intron8), and G4655A. The total LINCL score should not be outside the 95th percentile confidence limits for age based on our historic data.
- The subject will not previously have participated in a gene transfer or stem cell study.
- Parents of study participants must agree to comply in good faith with the conditions of the study, including attending all of the required baseline and follow-up assessments, and both parents or legal guardians must give consent for their child's participation.
- Sexually active subjects will have to use contraception during the treatment and for 2 months after completion of the treatment.
- Exclusion criteria.
- Presence of other significant medical or neurological conditions may disqualify the subject from participation in this study, particularly those which would create an unacceptable operative risk or risk to receiving the AAVrh.10CUhCLN2 vector, e.g., malignancy, congenital heart disease, liver or renal failure.
- Subjects without adequate control of seizures.
- Subjects with heart disease that would be a risk for anesthesia or a history of major risk factors for hemorrhage.
- Subjects who cannot participate in MRI studies.
- Concurrent participation in any other FDA approved Investigational New Drug.
- Subjects with history of prolonged bleeding or abnormal platelet function or taking aspirin.
- Renal disease or altered renal function as defined by serum creatinine \> 1.5 mg/dl at admission.
- Abnormal serum sodium, potassium calcium, magnesium, phosphate at grade III or IV by Division of AIDS Toxicity Scale
- Hepatic disease or altered liver function as defined by SGPT \> 150 U/L, and or T.Bilirubin\> 1.3 mg/dL
- Immunosuppression as defined by WBC \< 3,000 at admission
- Uncorrected coagulopathy during the baseline period defined as INR \> 1.4; PTT \> 35 sec; PLT \< 150,000/mm3
- Anemia (hemoglobin \< 11.0 mg/dl at \> 2 years of age, with normal serum iron studies)
Exclusion
Key Trial Info
Start Date :
April 15 2010
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
February 8 2017
Estimated Enrollment :
8 Patients enrolled
Trial Details
Trial ID
NCT01414985
Start Date
April 15 2010
End Date
February 8 2017
Last Update
August 31 2020
Active Locations (1)
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1
Weill Cornell Medical College
New York, New York, United States, 10065