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Gene Therapy

Gene Therapy for Krabbe Disease (GALax-C Trial)

Q: What is the exact aggregate of participants engaged in this trial?

Passage Bio, Inc., the study's sponsor, will be administering this trial out of multiple locations including University of <a href="https://www.withpower.com/clinical-trials/utah">Utah</a> School of Medicine in Salt Lake City and The Children's Hospital Philadelphia. To ensure its success, 24 participants that meet <a href="https://www.withpower.com/clinical-trials/all">all</a> eligibility requirements must partake in it.

Q: Are more participants being sought for this research endeavor?

Affirmative. The latest information on clinicaltrials.gov states that this investigation, which was first published on February 24th 2022, is currently enrolling members. Approximately 24 volunteers need to be recruited from 5 distinct sites.

Q: Could I potentially join this research endeavor?

This experiment is recruiting 24 babies, between 1 and 9 months old, who are currently coping with leukodystrophy. Moreover, there are other prerequisites that need to be satisfied in order to participate: thrusting of the legs during playtime; smiling when a speaker gains their attention; lifting of the head; tracking movement with eyes; being either presymptomatic or displaying initial Krabbe Disease symptoms before 6 months of age.

Q: What are the projected outcomes of this scientific experiment?

This clinical trial's primary objective is to assess the prevalence of adverse events and serious adverse effects at Grade 3 or higher within 24 months from treatment. Its secondary objectives focus on biomarkers of GALC activity in Cerebrospinal fluid, ventilator-free survival compared with natural history data, as well as quality of life changes measured by the Pediatric Quality of Life Scale (Peds QL). These outcomes will be tracked over a period 5 years consisting multiple visits.

Q: Does this research project accept participants who are beyond the age of fifty?

To be considered for enrollment in this medical trial, potential participants must fall between 1 Month and 9 Months old. Additionally, there are 5 other studies available to applicants under the age of 18 years while a separate study is accessible to those who exceed 65 years of age.

Q: Are there ample locations across North America administering this trial?

Patients are being recruited from a selection of medical centres, including University of <a href="https://www.withpower.com/clinical-trials/utah">Utah</a> School of Medicine in Salt Lake City, The Children's Hospital of Philadelphia in <a href="https://www.withpower.com/clinical-trials/pennsylvania">Pennsylvania</a> and <a href="https://www.withpower.com/clinical-trials/new-york">New york</a>-Presbyterian located in the state of New York.

Phase 1 & 2

Waitlist Available

Research Sponsored by Passage Bio, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years (multiple visits)

Awards & highlights

GALax-C Trial Summary

This trial will test a gene therapy for Krabbe Disease, a rare neurological disorder, in two different age groups to see if it is safe and effective.

Who is the study for?

This trial is for infants aged 1-9 months with early infantile Krabbe Disease, showing certain neurological functions and having specific genetic markers. They must have blood psychosine levels above a threshold and low leukocyte GALC activity. Those with significant neurocognitive deficits not due to Krabbe, prior gene therapy or stem cell transplants, recent participation in other trials, chronic ventilation support, recent vaccines, MRI or lumbar puncture contraindications, kidney issues, abnormal blood counts or liver enzymes cannot participate.Check my eligibility

What is being tested?

The study tests PBKR03 gene therapy intended to deliver a functional GALC gene to combat Krabbe Disease. It will first assess safety and tolerability of two doses across different age groups before confirming the best dose for further evaluation of its effectiveness in slowing down or stabilizing the disease progression.See study design

What are the potential side effects?

While specific side effects are not listed here as it's an investigational treatment, potential risks may include reactions at the injection site, immune responses to the new genes introduced into the body (like inflammation), complications from procedures like lumbar punctures or anesthesia used during administration.

GALax-C Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years (multiple visits)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years (multiple visits)

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years (multiple visits) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Assess Humoral Response Against the Vector and Transgene in CSF

Assess Humoral Response Against the Vector and Transgene in Serum

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

+3 more

Secondary outcome measures

Change from Baseline in Developmental Milestones as Assessed by the Bayley Scale of Infant and Toddler Development, Third Edition

Change from Baseline in Developmental Milestones as Assessed by the Vineland Adaptive Behavior Scale-II

Change in Biomarkers of GALC Activity in Blood

+10 more

GALax-C Trial Design

2Treatment groups

Experimental Treatment

Group I: Part 2: Expansion Cohort designed to confirm the safety and efficacy of PBKR03Experimental Treatment1 Intervention

Cohort 5: Subjects aged >1 to <9 months Drug: PBKR03 Single dose of PBKR03, via intra cisterna magna Dose to be used for the confirmatory cohorts in Part 2 will be defined after a review of data from Part 1. *GC/g: genome copiesy per gram of estimated brain weight

Group II: Part 1: Dose Escalation Cohorts designed to identify the optimal dose of PBKR03Experimental Treatment1 Intervention

Cohort 1: Subjects aged >4 to <9 months Drug: PBKR03 1.5 x 10^11 GC/g* Single dose of PBKR03, via intra cisterna magna Cohort 2: Subjects aged >4 to <9 months Drug: PBKR03 5.0 x 10^11 GC/g* Single dose of PBKR03, via intra cisterna magna Cohort 3: Subjects aged >1 to <4 months Drug: PBKR03 1.5 x 10^11 GC/g* Single dose of PBKR03, via intra cisterna magna Cohort 4: Subjects aged >1 to <4 months Drug: PBKR03 5.0 x 10^11 GC/g* Single dose of PBKR03, via intra cisterna magna *GC/g: genome copiesy per gram of estimated brain weight

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

Passage Bio, Inc.Lead Sponsor

5 Previous Clinical Trials

187,081 Total Patients Enrolled

D. Elizabeth McNeil, MD, MScStudy DirectorPassage Bio, Inc.

Samiah Al-Zaidy, MDStudy DirectorPassage Bio, Inc.

1 Previous Clinical Trials

26 Total Patients Enrolled

Media Library

PBKR03 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04771416 — Phase 1 & 2

Krabbe Disease Research Study Groups: Part 2: Expansion Cohort designed to confirm the safety and efficacy of PBKR03, Part 1: Dose Escalation Cohorts designed to identify the optimal dose of PBKR03

Krabbe Disease Clinical Trial 2023: PBKR03 Highlights & Side Effects. Trial Name: NCT04771416 — Phase 1 & 2

PBKR03 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04771416 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What is the exact aggregate of participants engaged in this trial?

"Passage Bio, Inc., the study's sponsor, will be administering this trial out of multiple locations including University of Utah School of Medicine in Salt Lake City and The Children's Hospital Philadelphia. To ensure its success, 24 participants that meet all eligibility requirements must partake in it."

Answered by AI

Are more participants being sought for this research endeavor?

"Affirmative. The latest information on clinicaltrials.gov states that this investigation, which was first published on February 24th 2022, is currently enrolling members. Approximately 24 volunteers need to be recruited from 5 distinct sites."

Answered by AI

Could I potentially join this research endeavor?

"This experiment is recruiting 24 babies, between 1 and 9 months old, who are currently coping with leukodystrophy. Moreover, there are other prerequisites that need to be satisfied in order to participate: thrusting of the legs during playtime; smiling when a speaker gains their attention; lifting of the head; tracking movement with eyes; being either presymptomatic or displaying initial Krabbe Disease symptoms before 6 months of age."

Answered by AI

What are the projected outcomes of this scientific experiment?

"This clinical trial's primary objective is to assess the prevalence of adverse events and serious adverse effects at Grade 3 or higher within 24 months from treatment. Its secondary objectives focus on biomarkers of GALC activity in Cerebrospinal fluid, ventilator-free survival compared with natural history data, as well as quality of life changes measured by the Pediatric Quality of Life Scale (Peds QL). These outcomes will be tracked over a period 5 years consisting multiple visits."

Answered by AI

Does this research project accept participants who are beyond the age of fifty?

"To be considered for enrollment in this medical trial, potential participants must fall between 1 Month and 9 Months old. Additionally, there are 5 other studies available to applicants under the age of 18 years while a separate study is accessible to those who exceed 65 years of age."

Answered by AI

Are there ample locations across North America administering this trial?

"Patients are being recruited from a selection of medical centres, including University of Utah School of Medicine in Salt Lake City, The Children's Hospital of Philadelphia in Pennsylvania and New york-Presbyterian located in the state of New York."

Answered by AI