What is the mechanism of action of this treatment?

Common treatments for telomere shortening, particularly in the context of bone marrow failure and telomere biology disorders, often involve the use of telomerase activators and gene therapy. Telomerase activators work by upregulating the activity of telomerase, an enzyme that adds telomeric DNA to the ends of chromosomes, thereby maintaining telomere length and promoting cell longevity. Gene therapy approaches may involve the introduction of genes that encode for telomerase or other proteins that protect telomeres from degradation. These treatments are crucial for patients with telomere shortening because they help to stabilize telomere length, prevent premature cellular aging, and improve the function of rapidly dividing cells, such as those in the bone marrow, which are essential for producing blood cells.

What side effects are associated with this type of intervention?

Common treatments for telomere shortening, particularly in the context of bone marrow failure, include androgens, anabolic steroids, and telomerase activators. Known side effects of these treatments can include liver toxicity, virilization, fluid retention, hypertension, and potential cardiovascular issues. Additionally, telomerase activators may carry risks of promoting cancerous growth due to their role in cell proliferation. Patients undergoing these treatments should be closely monitored for these adverse effects.

What prior FDA approvals exist?

There is limited information provided about FDA-approved treatments specifically targeting telomere shortening. However, the context mentions a Phase I/II study of EXG34217 for patients with telomere biology disorders with bone marrow failure, indicating ongoing research in this area. Broadly, treatments for telomere-related conditions often focus on managing symptoms and complications, such as bone marrow failure, rather than directly targeting telomere shortening itself.

What other types of research exist?

Promising alternatives for telomere shortening patients include gene therapy approaches, such as those using adeno-associated virus (AAV) vectors to deliver telomerase reverse transcriptase (TERT) or other telomere-stabilizing genes. Additionally, treatments like danazol, a synthetic androgen, have shown efficacy in improving telomere length and hematologic parameters in patients with telomere biology disorders. Another potential option is the use of RNA-based therapies, such as antisense oligonucleotides, to modulate telomere-related gene expression.

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EXG34217 for Bone Marrow Failure

Phase 1

Recruiting

Led By Kasiani Myers, MD

Research Sponsored by Elixirgen Therapeutics, Inc.

Eligibility Criteria Checklist

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

Must have

Mild or moderate bone marrow failure defined by satisfying specific conditions

Diagnosis of telomere biology disorders

Must not have

Women of child bearing potential or breastfeeding

Patients who are not eligible for G-CSF and plerixafor dosing

Timeline

Screening 3 weeks

Treatment Varies

Follow Up screening, month1,3,6 and 12

Awards & highlights

Summary

This trial tests the safety and tolerability of EXG34217 in patients with bone marrow failure due to telomere biology disorders. The treatment involves collecting, processing, and reinfusing the patient's own blood cells to help improve their bone marrow function.

Who is the study for?

This trial is for adults over 18 with mild or moderate bone marrow failure due to telomere biology disorders. It's not for those with severe bone marrow failure, certain genetic abnormalities, uncontrolled infections, previous transplants, or who can't undergo specific treatments. Pregnant or breastfeeding women and patients on recent trials or cancer treatment are also excluded.

What is being tested?

The study tests the safety and effects of a drug called EXG34217 in patients with bone marrow failure linked to short telomeres. As an early-stage (Phase I/II) trial at a single center, it's designed to see how well participants tolerate this medication.

What are the potential side effects?

Since EXG34217 is being studied primarily for its safety and tolerability, potential side effects aren't listed but may include typical reactions related to new medications such as allergic responses, gastrointestinal issues, fatigue, blood count changes among others.

Eligibility Criteria

Inclusion Criteria

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

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My bone marrow is not working well, but it's not severe.

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I have been diagnosed with a telomere biology disorder.

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I am older than 18 years.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am a woman who could become pregnant or am currently breastfeeding.

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I cannot receive G-CSF and plerixafor treatments.

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My bone marrow does not produce enough blood cells.

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I have had a bone marrow or stem cell transplant from a donor.

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I am currently receiving chemotherapy for my cancer.

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My bone marrow test shows changes linked to MDS or AML.

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I do not have any untreated serious infections.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ screening, month1,3,6 and 12

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~screening, month1,3,6 and 12

This trial's timeline: 3 weeks for screening, Varies for treatment, and screening, month1,3,6 and 12 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Number of participants with a change in Electrocardiography (ECG)

Number of participants with a change in clinical laboratory evaluations

Number of participants with a change in in physical examination

+2 more

Secondary study objectives

Number of participants with a change in telomere length

Number of participants with improvement of blood counts.

Trial Design

1Treatment groups

Experimental Treatment

Group I: EXG34217Experimental Treatment1 Intervention

single autologous CD34+ cells contacted ex vivo with EXG-001

0 / 10Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for telomere shortening, particularly in the context of bone marrow failure and telomere biology disorders, often involve the use of telomerase activators and gene therapy. Telomerase activators work by upregulating the activity of telomerase, an enzyme that adds telomeric DNA to the ends of chromosomes, thereby maintaining telomere length and promoting cell longevity. Gene therapy approaches may involve the introduction of genes that encode for telomerase or other proteins that protect telomeres from degradation. These treatments are crucial for patients with telomere shortening because they help to stabilize telomere length, prevent premature cellular aging, and improve the function of rapidly dividing cells, such as those in the bone marrow, which are essential for producing blood cells.