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AADC Deficiency - Healing Genes
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AADC Deficiency

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A Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients (AADC)

Single-Stage, Open-Label, Safety and Efficacy Study of Adeno-Associated Virus Encoding Human Aromatic L-Amino Acid Decarboxylase by Magnetic Resonance MR-guided Infusion Into Midbrain in Pediatric Patients With AADC Deficiency


Phase 1

DESCRIPTION:

Doctors at the University of California San Francisco, Benioff Children’s Hospital seek patients with Aromatic l-amino acid decarboxylase (AADC) deficiency caused by mutations in the DDC gene, to trial a gene-modified stem cell therapy designed to deliver a normal copy of the DDC gene. The normal gene produces an enzyme that helps make key brain molecules called neurotransmitters.

Study participants will undergo a blood draw treatment that sorts out specific stem cells and returns the blood to the patient. After the gene modification in lab, the stem cells will be injected into 2 portions of the center of the brain, guided by MRI. The patient will be closely assessed during treatment, with follow-up for 2+ years.


PATIENT MUST:

  • Be 5 to 18 years of age
  • Have definite diagnosis of AADC deficiency
  • Be unable to ambulate independently (with or without assistive device)

THE STUDY INVOLVES:

  1. Screening before the treatment
  2. Leukapheresis will draw the needed immune cells but return blood cells to the body
  3. Researchers will use a virus in the lab to deliver the gene to the immune cells
  4. A dose of chemotherapy will be delivered
  5. The gene-modified immune cells will be re-infused to the patient
  6. The patient will be closely assessed during treatment, with follow-up for 2 years.

LOCATIONS AND CONTACTS:

The study center is at the University of California San Francisco, Benioff Children’s Hospital. Map.

 

Contacts:

Jill A Imamura-Ching, RN, BSN  |  415-476-3446  |  [email protected]

Waldy E San Sebastian, PhD  |  415-502-4219  |  [email protected]

 

SPONSOR INFORMATION:

Krystof Bankiewicz

The National Institutes of Health (NIH)

 

Or go online:

https://clinicaltrials.gov/ct2/show/NCT02852213

 

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