Open Label Extension Study of HT-100 in Patients With DMD

Processa Pharmaceuticals

Status and phase: Terminated; Phase 2

Conditions: Duchenne Muscular Dystrophy

Treatments: Drug: HT-100

Study type: Interventional

Funder types: Industry

Identifiers

NCT01978366

HALO-DMD-02

HALO (Other Identifier)

Details and patient eligibility

About

This study is designed to provide 6-months continuous dosing with the study medication, called HT-100, on participants who successfully completed the predecessor study (HALO-DMD-01). The main purpose of this study is to assess chronic safety, tolerability, pharmacodynamic activity (testing the drug's effect on DMD) and population pharmacokinetics (measuring how much drug is in the bloodstream) in participants with a broad spectrum of Duchenne muscular dystrophy (DMD).

Enrollment: 17 patients

Sex: Male

Ages: 6 to 20 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Completed both the single ascending dose (SAD) and multiple ascending dose (MAD) phases of predecessor study HALO-DMD-01
• Maintained the same corticosteroid therapy from the predecessor study HALO-DMD-01
• Ability to provide written informed consent
• Ambulatory or non-ambulatory

Exclusion criteria

• Recent, substantial change in use of cardiac medications or medications affecting muscle function
• Clinically significant major disease, not related to DMD
• Significantly compromised cardio-respiratory function
• History of severe allergic or anaphylactic reactions
• Prior treatment with another investigational product in past 6 months
• Inability to undergo magnetic resonance imaging (MRI)
• Current drug or alcohol abuse or prior treatment for abuse

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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17 participants in 5 patient groups

Cohort 1: HT-100 tablet, Dose 1

Experimental group

Description:

• Multiple dose administration: Dose 1

Treatment:

Drug: HT-100

Cohort 2: HT-100 tablet, Dose 2

Experimental group

Description:

• Multiple dose administration: Dose 2

Treatment:

Drug: HT-100

Cohort 3: HT-100 tablet, Dose 3

Experimental group

Description:

• Multiple dose administration: Dose 3

Treatment:

Drug: HT-100

Cohort 4: HT-100 tablet, Dose 4

Experimental group

Description:

• Multiple dose administration: Dose 4

Treatment:

Drug: HT-100

Cohort 5: HT-100 tablet, Dose 5

Experimental group

Description:

• Multiple dose administration: Dose 5

Treatment:

Drug: HT-100