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Gene Therapy

RP-L301 for Pyruvate Kinase Deficiency

Phase 1
Waitlist Available
Led By Julián Sevilla Navarro, MD, PhD
Research Sponsored by Rocket Pharmaceuticals Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
PKD diagnosis with a confirmed PKLR mutation.
PKD diagnosis with a confirmed PKLR mutation
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 2 years
Awards & highlights

Study Summary

This trial is testing a new gene therapy for people with Pyruvate Kinase Deficiency, a rare blood disorder. The goal is to find out if the therapy is safe.

Who is the study for?
This trial is for people with Pyruvate Kinase Deficiency (PKD) who are between 8-50 years old, depending on enrollment phase. They must have a history of multiple blood transfusions and low hemoglobin levels despite treatment like splenectomy. Participants need to be in good health regarding heart, lung, kidney, and liver function.Check my eligibility
What is being tested?
The trial is testing RP-L301, a gene therapy aimed at treating PKD. It's an early-phase study focusing on the safety of this new treatment where all participants receive the intervention without any comparison group.See study design
What are the potential side effects?
As it's an early-stage trial for gene therapy, specific side effects aren't listed but may include reactions related to the body’s acceptance of new genes and general risks associated with bone marrow procedures.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been diagnosed with PKD and have a confirmed PKLR mutation.
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I have been diagnosed with PKD and have a confirmed PKLR mutation.
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I am within the age range specified for my enrollment group.
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I have had severe anemia requiring blood transfusions.
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My hemoglobin levels have been below 8.0 without transfusions.
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My heart, lungs, kidneys, and liver are functioning well.
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My hemoglobin levels have been below 9.5 despite having my spleen removed.
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I've needed multiple blood transfusions in the past few years without major surgery or infections.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~2 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and 2 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
Evaluation of the safety and toxicity of RP-L301: number of participants with treatment-related adverse events
Secondary outcome measures
Clinically significant reduction in anemia
Genetic correction following administration of RP-L301
Blood Transfusion
+2 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: RP-L301Experimental Treatment1 Intervention
RP-L301 is a gene therapy product containing autologous genetically modified CD34+ hematopoietic stem cells containing the corrected PKD gene

Find a Location

Who is running the clinical trial?

Rocket Pharmaceuticals Inc.Lead Sponsor
14 Previous Clinical Trials
375 Total Patients Enrolled
Julián Sevilla Navarro, MD, PhDPrincipal InvestigatorHospital Universitario Fundación Jiménez Díaz
4 Previous Clinical Trials
24 Total Patients Enrolled
José Luis López Lorenzo, MDPrincipal InvestigatorHospital Universitario Fundación Jiménez Díaz

Frequently Asked Questions

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

What potential risks does RP-L301 pose for patients?

"Due to the Phase 1 trial nature of RP-L301, our team at Power estimates its safety as a 1. Data regarding efficacy and safety is still limited in this case."

Answered by AI

How many participants are currently undergoing this research project?

"Affirmative, according to clinicaltrials.gov information this medical trial is presently seeking out potential participants. This experiment was initially posted on July 6th 2020 and recently updated on September 22nd 2021, with a requirement for the recruitment of six patients from one site."

Answered by AI

Who is eligible to enroll in this trial?

"6 applicants will be accepted into this clinical trial, provided they meet the criteria of pyruvate metabolism and inborn errors disorders as well as being between 8 to 50 years old."

Answered by AI

Are there any open slots available for participation in this investigation?

"According to the clinicaltrials.gov data, this research is recruiting patients presently; it was posted on July 6th 2020 and last updated September 22nd 2021."

Answered by AI

Does the age restriction for participants in this study extend to minors?

"This research project is offering enrollment to individuals aged 8 and over, but not exceeding 50 years of age."

Answered by AI
~1 spots leftby May 2025