Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile K… (NCT04771416) | Clinical Trial Compass
SuspendedPhase 1/2
Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease
Stopped: The study was stopped due to changes in the company strategy.
United States, Brazil24 participantsStarted 2022-02-24
Plain-language summary
PBKR03 is a gene therapy for Krabbe Disease (Globoid cell leukodystrophy) intended to deliver a functional copy of the GALC gene to the brain and peripheral tissues. This study will evaluate the safety, tolerability and efficacy of this treatment by first evaluating two different doses in two different age groups, then confirming the optimal dose to be used for confirmation of safety and efficacy.
Who can participate
Age range1 Month – 9 Months
SexALL
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Inclusion criteria
✓. \> 1 month and \< 9 months of age at enrollment, either presymptomatic or symptomatic with first symptoms of Krabbe Disease at \< 6 months of age
✓. Leukocyte GALC activity below lower limit of normal (LLN)
✓. Whole blood psychosine \> 10 nM
✓. Biallelic pathogenic GALC gene variants previously associated with early infantile Krabbe Disease or variants classified as likely pathogenic
✓. Parents or the subject's legally authorized representative provide written informed consent prior to any study-related procedures, including screening evaluations
✓. Symptomatic subjects must exhibit a minimum level of neurological and developmental function that indicates that they have the potential to benefit from treatment, at least with slowing or stabilization of their disease. In particular, the subject must demonstrate the following clinical features (when age-appropriate):
Exclusion criteria
✕. Any clinically significant neurocognitive deficit not attributable to Krabbe disease.
✕
What they're measuring
1
Number of participants with treatment-related adverse events (AEs) and serious adverse events (SAEs) at Grade 3 or higher within 24 months of dosing
Timeframe: Up to 5 years (multiple visits)
2
Change from baseline in nerve conduction and velocity in motor nerve conduction studies
Timeframe: From baseline to 5 years (multiple visits)
3
Change from baseline in nerve conduction and velocity in sensory nerve conduction studies
Timeframe: From baseline to 5 years (multiple visits)
4
Number of Participants with Clinically Significant Laboratory Abnormalities as Measured Using Hematology, Chemistry and Coagulation Tests
Timeframe: Up to 5 years (multiple visits)
5
Assess Humoral Response Against the Vector and Transgene in Serum
Timeframe: Up to 5 years (multiple visits)
6
Assess Humoral Response Against the Vector and Transgene in CSF
. An acute illness requiring hospitalization within 30 days of enrollment.
✕. History of chronic ventilation assisted respiratory support (defined as more than 12 hours/day of bilevel positive airway pressure, continuous positive airway pressure (CPAP) or ventilator) or a need for tracheostomy as a result of their disease. Note: This does not exclude patients who use respiratory vests.
✕. Intractable seizure or uncontrolled epilepsy defined as having had an episode of status epilepticus, or seizures requiring hospitalization.
✕. Family history of seizures or epilepsy of infantile or childhood onset, other than febrile seizures. This does not exclude subjects with a family history of Krabbe disease.
✕. Any contraindication to the ICM administration procedure, including contraindications to fluoroscopic imaging, intrathecal contrast and anesthesia or any condition that would increase the risk of adverse outcomes from the ICM procedure including, but not limited to, the presence of a space occupying lesion, aberrant vascular anatomy or congenital anatomical abnormalities such as a Chiari malformation.
✕. Any contraindication to MRI or lumbar puncture (LP).