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Gene Therapy

Gene Transfer for Pompe Disease

Phase 1 & 2

Waitlist Available

Led By Tahseen Mozaffar, MD

Research Sponsored by Spark Therapeutics, Inc.

Eligibility Criteria Checklist

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

Must have

Males and Females ≥18 years of age with late-onset Pompe disease

Have clinically moderate, late-onset Pompe disease characteristics

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

Study Summary

This trial is testing a new drug for people with Pompe disease who are already receiving enzyme replacement therapy. The goal is to see if it is safe, tolerated, and effective.

Who is the study for?

Adults over 18 with moderate, late-onset Pompe disease who've been on enzyme replacement therapy (ERT) for at least 2 years can join. They must not be pregnant or nursing, have no active infections including hepatitis B/C and HIV, no history of liver cancer or significant liver disease, and agree to use contraception.Check my eligibility

What is being tested?

The trial is testing SPK-3006's safety and effectiveness in adults with Pompe disease. It involves a single intravenous dose of the gene therapy drug given to different groups one after another at increasing dose levels while they continue their regular ERT.See study design

What are the potential side effects?

Specific side effects are not listed but generally may include reactions related to infusion such as discomfort or pain at the injection site, potential immune responses to the treatment like fever or chills, and other unforeseen complications due to genetic nature of therapy.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am 18 or older with late-onset Pompe disease.

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I have moderate, late-onset Pompe disease.

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I have been on enzyme replacement therapy for at least 2 years.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

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

Treatment Details

Study Objectives

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

Primary outcome measures

Number of adverse and serious adverse events (AEs/SAEs), including clinically significant abnormal laboratory values.

Occurrence of immune response against AAV capsid

Occurrence of immune response against GAA transgene

Trial Design

1Treatment groups

Experimental Treatment

Group I: SPK-3006Experimental Treatment1 Intervention

All participants who meet the eligibility criteria will receive a single intravenous (i.v.) administration of SPK-3006.

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

Spark Therapeutics, Inc.Lead Sponsor

14 Previous Clinical Trials

430 Total Patients Enrolled

Spark TherapeuticsLead Sponsor

13 Previous Clinical Trials

350 Total Patients Enrolled

Tahseen Mozaffar, MDPrincipal InvestigatorUniversity of California Irvine Health

2 Previous Clinical Trials

982 Total Patients Enrolled

Media Library

Pompe Disease Research Study Groups: SPK-3006

Frequently Asked Questions

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

Are there a plethora of locations in this city that are hosting the experiment?

"At present, the trial is operating at 13 different sites across North America - including Salt Lake City, Ottawa and Atlanta. To minimize travel requirements for participants, it may be beneficial to select a clinic that is closest to you in order to enroll."

Answered by AI

Is this scientific experiment currently accepting volunteers?

"The clinical trial, which initially went live on October 1st 2020 and was last updated July 22nd 2022 is not accepting participants at this time. However, 1046 other trials are presently open for recruitment."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

2020. "A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04093349.

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