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, Brazil, Canada24 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 range

1 Month – 9 Months

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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):

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

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)

Change from baseline in nerve conduction and velocity in motor nerve conduction studies

Timeframe: From baseline to 5 years (multiple visits)

Change from baseline in nerve conduction and velocity in sensory nerve conduction studies

Timeframe: From baseline to 5 years (multiple visits)

Number of Participants with Clinically Significant Laboratory Abnormalities as Measured Using Hematology, Chemistry and Coagulation Tests

Timeframe: Up to 5 years (multiple visits)

Assess Humoral Response Against the Vector and Transgene in Serum

Timeframe: Up to 5 years (multiple visits)

Assess Humoral Response Against the Vector and Transgene in CSF

Timeframe: Up to 5 years (multiple visits)

Trial details

NCT IDNCT04771416

SponsorGemma Biotherapeutics

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2027-01

View on ClinicalTrials.gov More Leukodystrophy, Globoid Cell trials

Plain-language summary

Who can participate

Age range

1 Month – 9 Months

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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):

What they're measuring

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)

Change from baseline in nerve conduction and velocity in motor nerve conduction studies

Timeframe: From baseline to 5 years (multiple visits)

Change from baseline in nerve conduction and velocity in sensory nerve conduction studies

Timeframe: From baseline to 5 years (multiple visits)

Number of Participants with Clinically Significant Laboratory Abnormalities as Measured Using Hematology, Chemistry and Coagulation Tests

Timeframe: Up to 5 years (multiple visits)

Assess Humoral Response Against the Vector and Transgene in Serum

Timeframe: Up to 5 years (multiple visits)

Assess Humoral Response Against the Vector and Transgene in CSF

Timeframe: Up to 5 years (multiple visits)

Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease

Plain-language summary

Who can participate

Inclusion criteria

Questions worth asking your doctor

Questions for the trial coordinator

What they're measuring

Trial details

Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease

Plain-language summary

Who can participate

Inclusion criteria

Questions worth asking your doctor

Questions for the trial coordinator

What they're measuring

Trial details

Exclusion criteria