Clinical trial

Long Term Follow-up Study in Patients With Telomere Biology Disorders With Bone Marrow Failure Who Completed Study EXG-US-01

ID

Name

EXG-US-02

Description

This is a long-term rollover follow-up study for Phase I/II study (Protocol EXG-US-01).

Trial arms

Trial start

2023-07-01

Estimated PCD

2027-01-30

Trial end

2027-01-30

Status

Not yet recruiting

Phase

Early phase I

Treatment

EXG34217

Single infusion

Arms:

EXG34217

Size

12

Primary endpoint

Number of participants with adverse events -Safety by Incidence of Treatment-Emergent

Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72

umber of participants with a change in in physical examination

Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72

Number of participants with a change in Electrocardiography (ECG)

Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72

Number of participants with a change in clinical laboratory evaluations

Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72

Number of participants with a change of Immunogenicity

Change from Baseline Systolic Blood Pressure at Month 18, 24,30,36,42,48,60 and 72

Eligibility criteria

Inclusion Criteria: 1. Signed an Institutional Review Board (IRB) approved informed consent document indicating that they understand the purpose of, and procedures required by the study and are willing to participate in the study and comply with all study procedures and restrictions. Informed consent must be obtained from the subject prior to initiating procedures. 2. Have completed the 12-month visit of Study EXG-US-01. Exclusion Criteria: -

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'Sequential Assignment', 'primaryPurpose': 'SUPPORTIVE_CARE', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 12, 'type': 'ESTIMATED'}}

Updated at

2023-05-22

1 organization

1 product

1 indication

Organization

Elixirgen Therapeutics

Product

Indication

Dyskeratosis Congenita