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Study to Evaluate of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure

Primary Purpose

Telomere Shortening, Bone Marrow Failure

Status
Recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
EXG34217
Sponsored by
Elixirgen Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Telomere Shortening

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Age > 18 years.
  • Mild or moderate bone marrow failure defined by satisfying specific conditions.
  • Diagnosis of telomere biology disorders

Exclusion Criteria:

  • Women of child bearing potential or breastfeeding.
  • Patients with cancer who are on active chemotherapeutic treatment.
  • Patients with severe bone marrow failure.
  • Clonal cytogenetic abnormalities associated with MDS or AML on bone marrow examination.
  • Uncontrolled bacterial, viral or fungal infections.
  • Prior allogeneic marrow or stem cell transplantation.
  • Patients who are not eligible for G-CSF and plerixafor dosing.
  • Patients who are not eligible for the apheresis.
  • Patients currently taking or have taken danazol and androgens within 60 days prior to Day 1.
  • Patients with any other clinically relevant acute or chronic diseases which could interfere with the patients' safety during the trial, expose them to undue risk, or which could interfere with study objectives.
  • Patients who have participated in another clinical trial with an investigational drug within the previous 30 days.

Sites / Locations

  • Cincinnati Children's HospitalRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

EXG34217

Arm Description

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

Outcomes

Primary Outcome Measures

Number of participants with adverse events -Safety by Incidence of Treatment-Emergent Adverse Events
Number of participants with treatment-related adverse events as assessed by CTCAE v4.03. Incidence and nature of adverse events, vital signs, weight.
Number of participants with a change in in physical examination
Physical examination changes General appearance ,Head, eyes, ears, nose, and throat, Respiratory, Cardiovascular, Musculoskeletal, Abdomen, Neurologic, Extremities, Dermatologic, Lymphatic)
Number of participants with a change in Electrocardiography (ECG)
ECG (standard digital 12-lead in singlicate)
Number of participants with a change in clinical laboratory evaluations
Changes in clinical laboratory evaluations (Hematology, Blood chemistry, Coagulation, and Urinalysis)
Number of participants with a change of Immunogenicity
Change in Antibody against virus vector and transgene

Secondary Outcome Measures

Number of participants with a change in telomere length
Change in telomere length in any peripheral blood cells
Number of participants with improvement of blood counts.
Blood counts: neutrophils,platelets, or hemoglobin

Full Information

First Posted
December 6, 2019
Last Updated
May 8, 2023
Sponsor
Elixirgen Therapeutics, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT04211714
Brief Title
Study to Evaluate of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure
Official Title
A Phase I/II Study to Evaluate the Safety and Tolerability of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure
Study Type
Interventional

2. Study Status

Record Verification Date
May 2023
Overall Recruitment Status
Recruiting
Study Start Date
April 8, 2021 (Actual)
Primary Completion Date
April 8, 2027 (Anticipated)
Study Completion Date
April 8, 2027 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Elixirgen Therapeutics, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
This is a Phase I/II, open label, single center study to assess the safety and tolerability of EXG34217 in bone marrow failure patients with telomere biology disorders.
Detailed Description
This is a Phase I/II, open label study in up to 12 subjects with telomere biology disorders with bone marrow failure. The study is open to all participants regardless of gender or ethnicity. Subjects who are enrolled but not evaluable will be replaced. Subjects will sign a consent form prior to any study related procedure and will complete baseline screening assessments. Subjects for this study will not require any preparative regimen such as chemotherapy or radiation. The study will be conducted in three parts Peripheral blood mononuclear cells (PBMNCs) collection; mobilization and apheresis, Ex vivo cell processing Processed cell infusion and post-infusion safety monitoring, Follow-up (Week 2, 3,4,5, Months 1, 2,3,4,5,6,9 and 12)

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Telomere Shortening, Bone Marrow Failure

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
12 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
EXG34217
Arm Type
Experimental
Arm Description
single autologous CD34+ cells contacted ex vivo with EXG-001
Intervention Type
Biological
Intervention Name(s)
EXG34217
Intervention Description
Single infusion
Primary Outcome Measure Information:
Title
Number of participants with adverse events -Safety by Incidence of Treatment-Emergent Adverse Events
Description
Number of participants with treatment-related adverse events as assessed by CTCAE v4.03. Incidence and nature of adverse events, vital signs, weight.
Time Frame
Multiple times for the duration of the study (baseline through Month 12)
Title
Number of participants with a change in in physical examination
Description
Physical examination changes General appearance ,Head, eyes, ears, nose, and throat, Respiratory, Cardiovascular, Musculoskeletal, Abdomen, Neurologic, Extremities, Dermatologic, Lymphatic)
Time Frame
Multiple times for the duration of the study (baseline through Month 12)
Title
Number of participants with a change in Electrocardiography (ECG)
Description
ECG (standard digital 12-lead in singlicate)
Time Frame
Multiple times for the duration of the study (baseline through Month 12)
Title
Number of participants with a change in clinical laboratory evaluations
Description
Changes in clinical laboratory evaluations (Hematology, Blood chemistry, Coagulation, and Urinalysis)
Time Frame
Multiple times for the duration of the study (baseline through Month 12)
Title
Number of participants with a change of Immunogenicity
Description
Change in Antibody against virus vector and transgene
Time Frame
Multiple times for the duration of the study (baseline through Month 12)
Secondary Outcome Measure Information:
Title
Number of participants with a change in telomere length
Description
Change in telomere length in any peripheral blood cells
Time Frame
Screening, Month1,3,6 and 12
Title
Number of participants with improvement of blood counts.
Description
Blood counts: neutrophils,platelets, or hemoglobin
Time Frame
Multiple times for the duration of the study (baseline through Month 12)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Age > 18 years. Mild or moderate bone marrow failure defined by satisfying specific conditions. Diagnosis of telomere biology disorders Exclusion Criteria: Women of child bearing potential or breastfeeding. Patients with cancer who are on active chemotherapeutic treatment. Patients with severe bone marrow failure. Clonal cytogenetic abnormalities associated with MDS or AML on bone marrow examination. Uncontrolled bacterial, viral or fungal infections. Prior allogeneic marrow or stem cell transplantation. Patients who are not eligible for G-CSF and plerixafor dosing. Patients who are not eligible for the apheresis. Patients currently taking or have taken danazol and androgens within 60 days prior to Day 1. Patients with any other clinically relevant acute or chronic diseases which could interfere with the patients' safety during the trial, expose them to undue risk, or which could interfere with study objectives. Patients who have participated in another clinical trial with an investigational drug within the previous 30 days.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Martine Francis
Phone
301-343-8894
Email
martine@mafinc.com
First Name & Middle Initial & Last Name or Official Title & Degree
Minako Koga
Phone
202-615-6004
Email
mkoga@kmphc.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Kasiani Myers, MD
Organizational Affiliation
Cincinnati Children Hospital Medical Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
Cincinnati Children's Hospital
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Kasiani Myers, MD
Phone
513-803-3218
Email
Kasiani.Myers@cchmc.org
First Name & Middle Initial & Last Name & Degree
Monica Trapp
Phone
(513) 803-3218
Email
Monica.Trapp@cchmc.org
First Name & Middle Initial & Last Name & Degree
Kasani Myers, MD

12. IPD Sharing Statement

Plan to Share IPD
No

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Study to Evaluate of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure

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