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Gene Therapy

Cohort C for Hemophilia A (KEYSTONE 1 Trial)

Phase 3

Recruiting

Research Sponsored by Spark Therapeutics, Inc.

Eligibility Criteria Checklist

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

Must have

Have ≥150 documented exposure days to an FVIII protein product such as recombinant, plasma-derived, or extended half-life FVIII product

If human immunodeficiency virus (HIV)-positive at screening, have an adequate cluster of differentiation 4 (CD4) count (>200/mm3) and undetectable viral load (<50 genome copies [gc]/mL), are on an antiretroviral drug regimen, and have completed at least 12 weeks of this treatment regimen prior to screening

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to approximately 10 years

Awards & highlights

KEYSTONE 1 Trial Summary

This trial aims to test if SPK-8011 is better at preventing bleeding episodes in people with hemophilia A who do not have inhibitors compared to the standard treatment with FVIII prophylaxis

Who is the study for?

Adult males with severe or moderately severe Hemophilia A, having FVIII activity ≤3%. They must have used FVIII products for at least 150 days and not be allergic to them. No history of liver disease, cirrhosis, or inhibitors against FVIII in the past 5 years. HIV-positive participants need controlled virus levels and CD4 count >200/mm3.Check my eligibility

What is being tested?

The trial is testing SPK-8011's ability to prevent bleeding episodes in comparison with routine prophylaxis using Factor VIII (FVIII) in men with Hemophilia A without inhibitors. It will assess if gene therapy can reduce the frequency of bleeds better than standard treatments.See study design

What are the potential side effects?

Potential side effects are not specified here but may include reactions similar to other gene therapies such as immune responses, injection site reactions, headaches, muscle aches, fever or changes in liver function tests.

KEYSTONE 1 Trial Eligibility Criteria

Inclusion Criteria

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

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I have used a Factor VIII product for at least 150 days.

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I am HIV-positive with a CD4 count over 200, an undetectable viral load, and have been on HIV medication for at least 12 weeks.

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I haven't had issues with FVIII inhibitors or low FVIII activity in the past 5 years.

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I have been treated with FVIII for at least 6 months and had 5 or more bleeds treated in the last 6 months.

KEYSTONE 1 Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to approximately 10 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to approximately 10 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to approximately 10 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

Annualized Bleed Rate (ABR) for All Bleeds [Cohort A]

Secondary outcome measures

ABR for treated bleeds [Cohort A]

ABR for treated spontaneous, joint, and target joint bleeds [Cohort A]

Annualized FVIII dosage [Cohort A]

+17 more

KEYSTONE 1 Trial Design

3Treatment groups

Experimental Treatment

Group I: Cohort CExperimental Treatment1 Intervention

Participants with severe or moderately severe hemophilia A without FVIII inhibitors using emicizumab prophylaxis

Group II: Cohort BExperimental Treatment1 Intervention

Participants with severe or moderately severe hemophilia A without FVIII inhibitors using on-demand FVIII replacement therapy

Group III: Cohort A (Primary Cohort)Experimental Treatment1 Intervention

Participants with severe or moderately severe hemophilia A without FVIII inhibitors using routine FVIII prophylaxis

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

SPK-8011

2017

Completed Phase 2

~30

0 / 4Eligibility criteria met

Find a Location

Who is running the clinical trial?

Spark Therapeutics, Inc.Lead Sponsor

14 Previous Clinical Trials

354 Total Patients Enrolled

6 Trials studying Hemophilia A

91 Patients Enrolled for Hemophilia A

Frequently Asked Questions

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

What is the level of risk associated with Cohort C for individuals?

"Based on our assessment at Power, the safety rating for Cohort C in this trial is 3. This reflects a Phase 3 investigation with existing efficacy data and robust records of safety profiles."

Answered by AI

What is the current number of participants being enrolled in this research project?

"Affirmative. The information on clinicaltrials.gov confirms that this study is actively seeking participants. Originally shared on March 13, 2024, and most recently revised on April 15, 2024, the trial aims to enroll a total of 85 individuals across nine designated sites."

Answered by AI

At how many different venues can this study be accessed?

"At the moment, this study is ongoing in 9 different locations. These sites are dispersed among cities such as Walnut Creek, Sacramento, and Oakland along with an additional 6 undisclosed areas. Opting for the nearest site minimizes travel obligations for potential participants."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Study of a Gene Therapy Treatment for Hemophilia A

2024. "Study of a Gene Therapy Treatment for Hemophilia A". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06297486.

All > Hemophilia A > Hemophilia