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4 Participants NeededMy employer runs this trial

APR-2020 for Diamond-Blackfan Anemia

Recruiting at 1 trial location
NS
Overseen ByNova Silver
Age: < 65
Sex: Any
Trial Phase: Phase 1
Sponsor: Apriligen, Inc.
Must not be taking: Prohibited medications
Disqualifiers: Viral serology, Infections, Malignancy, Heart disease, others
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

Brief summary

The goal of this clinical trial is to learn if APR-2020 is safe and can help treat Diamond-Blackfan Anemia (DBA) in adolescents and children. The main questions it aims to answer are:

* Is APR-2020 safe and well tolerated?

* Does APR-2020 modify or correct an underlying genetic condition which causes DBA?

* Does APR-2020 reduce or eliminate the need for blood transfusions and/or restore certain blood counts affected by DBA?

Participants will:

* Take the drug one time as an infusion.

* Undergo two rounds of a cellular harvest procedure in which their own cells will be used in the manufacturing of their own participant-specific product.

* Initially return to the clinic for two years of follow up at increasingly sparse intervals.

Are You a Good Fit for This Trial?

Inclusion Criteria

My bone marrow tests are normal except for RPS19-deficient DBA.
I am between 2 and 18 years old.
I have been diagnosed with DBA caused by RPS19 deficiency.
See 7 more

Exclusion Criteria

I have abnormal genetic changes in my blood cells.
I have a close family member with a known or suspected inherited cancer syndrome.
Evidence of significant pulmonary hypertension, per Investigator assessment
See 18 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Cellular Harvest

Participants undergo two rounds of a cellular harvest procedure to collect their own cells for manufacturing the participant-specific product.

4 weeks

Treatment

Participants receive a one-time infusion of APR-2020 gene therapy.

1 day

Follow-up

Participants initially return to the clinic for two years of follow-up at increasingly sparse intervals to monitor safety and efficacy.

24 months

What Are the Treatments Tested in This Trial?

Interventions

  • APR-2020

How Is the Trial Designed?

1

Treatment groups

Experimental Treatment

Group I: APR-2020Experimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Apriligen, Inc.

Lead Sponsor

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