HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02

Akashi Therapeutics

Status and phase: Terminated; Phase 2

Conditions: Duchenne Muscular Dystrophy

Treatments: Drug: HT-100

Study type: Interventional

Funder types: Industry

Identifiers

NCT02525302

HALO-DMD-03

Details and patient eligibility

About

This study, HALO-DMD-03, is a follow-on study to HALO-DMD-01 and HALO-DMD-02, and allows continued open-label access to HT-100 for subjects who have completed these studies. HALO-DMD-03 will provide safety and strength and function data on continuous long-term dosing. Data from this study will be used to inform the safety, tolerability, and dose selection for a future trial of HT-100 in boys with Duchenne Muscular Dystrophy (DMD).

Full description

As a follow-on study to the initial clinical studies of HT-100 in DMD (Protocols HALO-DMD-01 and HALO-DMD-02), this open-label study is designed to provide data on continuous long-term dosing. Subjects will be entered into the study without cessation of dosing, in a staggered fashion, into the same cohort assignment they had in the predecessor studies. Up to 30 subjects who have completed dosing in HALO-DMD-02 will be offered the opportunity to continue on the same dose regimen until market approval of HT-100 or termination of the study by the Sponsor. Reasons for termination could include, among others, safety concerns or lack of efficacy, based on analysis of combined data from all HT-100 studies. Safety data from subjects approaching the end the HALO-DMD-02 participation will be individually reviewed by the Medical Monitor and the subject's physician (Principal Investigator [PI]). If the Medical Monitor and the PI agree there are no clinically significant safety signals (absence of clinically significant laboratory or clinical abnormalities to date), the subject will be considered eligible and offered continuation of dosing. To avoid an interruption in dosing, subjects will immediately be screened for participation and enrolled upon completing the predecessor trial, HALO-DMD-02. Participation is in this study HALO-DMD-03 is optional. Safety and pharmacodynamics (PD) monitoring will continue throughout the subject's study participation. Dose reduction/modification might occur or individual subjects' participation in the trial may be discontinued if any Adverse Events (AEs) suggest that HT-100 is not sufficiently well tolerated.

Enrollment: 10 patients

Sex: Male

Ages: 6 to 20 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Completed both previous studies HALO-DMD-01 and HALO-DMD-02
2. Ability to provide written informed consent
3. Ability to understand and follow site and protocol instruction for the entire duration of the study

Exclusion criteria

Answering yes to any of the following make the subject NOT eligible to participate in the study.

1. Clinically significant major disease not related to DMD that would make it not safe to be in the study or affect ability to follow the protocol
2. History of severe allergic or anaphylactic reactions
3. Recent report of drug/alcohol abuse

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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10 participants in 5 patient groups

Cohort 1: HT-100 tablet, Dose 1

Experimental group

Description:

HT-100 multiple dose administration (dose 1).

Treatment:

Drug: HT-100

Cohort 1: HT-100 tablet, Dose 2

Experimental group

Description:

HT-100 multiple dose administration (dose 1).

Treatment:

Drug: HT-100

Cohort 1: HT-100 tablet, Dose 3

Experimental group

Description:

HT-100 multiple dose administration (dose 1).

Treatment:

Drug: HT-100

Cohort 1: HT-100 tablet, Dose 4

Experimental group

Description:

HT-100 multiple dose administration (dose 1).

Treatment:

Drug: HT-100

Cohort 1: HT-100 tablet, Dose 5

Experimental group

Description:

HT-100 multiple dose administration (dose 1).

Treatment:

Drug: HT-100