A Phase 1/2a Study of Oral PRO-1562 in Healthy Subjects and Patients With Relapsing Multiple Sclerosis

Progentos Therapeutics

Status and phase: Begins enrollment this month; Phase 2; Phase 1

Conditions: Multiple Sclerosis

Treatments: Drug: Placebo Oral Capsule; Drug: PRO-1562 Oral Capsules

Study type: Interventional

Funder types: Industry

Identifiers

NCT07826689

PRO-1562-101

Details and patient eligibility

About

Phase 1 will test single oral doses of PRO-1562 in healthy adult subjects to characterize the safety and the amount of drug that is absorbed into the body. Some subjects will have lumbar punctures performed to measure the amount of drug that crosses the blood-brain barrier since that is the site of drug activity.

Phase 2a will be a 6-month trial to test the ability of once monthly dosing of PRO-1562 to promote remyelination of the CNS and provide clinical benefit to adult patients with relapsing multiple sclerosis (RMS) who also have long-term vision problems diagnosed as chronic optic neuropathy. The benefits of remyelination will be measured using tests of visual acuity, speed of optic nerve conduction signals, and clinical assessments of cognitive and motor function. PRO-1562 will be added on to a stable regimen of MS disease modifying therapy.

Full description

Phase 1 is a randomized, double-blind, placebo-controlled, single-ascending dose (SAD) trial of PRO-1562 in healthy adult participants at a single clinical research unit to characterize the safety and pharmacokinetics of a range of oral doses of PRO-1562. Phase 2a is a randomized, double-blind, placebo-controlled, multi-center, 6-month trial of PRO-1562 in adult patients with relapsing multiple sclerosis who also have chronic optic neuropathy. Patients will remain on their stable regimen of an approved MS disease modifying therapy during the trial. Oral PRO-1562 or placebo will be dosed once a month at scheduled clinic visits. Participants will be given the option to continue on their assigned study treatment after completing the 6-month treatment period until the last patient completes the 6-month treatment period.

Enrollment: 160 estimated patients

Sex: All

Ages: 18 to 65 years old

Volunteers: Accepts Healthy Volunteers

Inclusion and exclusion criteria

Phase 1 Inclusion Criteria:

1. Able and willing to provide written informed consent
2. Body weight ≥ 48 kg (105.6 lbs) and body mass index (BMI) ≥ 18.5 and ≤ 30 kg/m2
3. Must be in good health and without clinically significant abnormalities by review of medical and surgical history, physical examination, vital signs measurement, and 12-lead ECG
4. No clinically-significant laboratory abnormalities
5. Willing and able to use highly-effective forms of contraception for at least 30 days after the end of study visit.

Phase 1 Exclusion Criteria:

1. Ongoing medical condition requiring systemic therapy.
2. Ongoing infection
3. mRNA or live vaccine during the past 60 days
4. History of significant hypersensitivity, intolerance, or allergy to any drug/medication
5. Participation in another clinical trial with an investigational agent within 30 days prior to Check-in or 5 half-lives (if known) of the investigational drug's PK, PD, or biological activity (if known), whichever is longer
6. Positive alcohol breath test or urine screen for drugs of abuse.
7. Use or intent to use natural products or nutritional/dietary supplements (including St. John's wort), vitamins, minerals, and phytotherapeutic /herbal/plant-derived preparations within or received within 14 days or 5 half-lives prior to Check-in, whichever is longer.
8. Poor peripheral venous access.
9. Receipt of blood products within 2 months prior to Check-in.
10. Donation of blood or blood products during the 4 weeks prior to Check-in.
11. Participants who in the opinion of the Principal Investigator (or designee), should not participate in this study

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Single Group Assignment

Masking: Quadruple Blind

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160 participants in 10 patient groups, including a placebo group

Phase 1, PRO-1562 Dose level 1

Experimental group

Description:

PRO-1562 oral capsule

Treatment:

Drug: PRO-1562 Oral Capsules

Phase 1, PRO-1562 Dose level 2

Experimental group

Description:

PRO-1562 oral capsule

Treatment:

Drug: PRO-1562 Oral Capsules

Phase 1, PRO-1562 Dose level 3

Experimental group

Description:

PRO-1562 oral capsule

Treatment:

Drug: PRO-1562 Oral Capsules

Phase 1, PRO-1562 Dose level 4

Experimental group

Description:

PRO-1562 oral capsules

Treatment:

Drug: PRO-1562 Oral Capsules

Phase 1, PRO-1562 Dose level 5

Experimental group

Description:

PRO-1562 oral capsule

Treatment:

Drug: PRO-1562 Oral Capsules

Phase 1, PRO-1562 Dose level 6

Experimental group

Description:

PRO-1562 oral capsule

Treatment:

Drug: PRO-1562 Oral Capsules

Phase 1, Placebo

Placebo Comparator group

Description:

Placebo oral capsule

Treatment:

Drug: Placebo Oral Capsule

Phase 2, PRO-1562 Oral Capsules Dose level 1

Experimental group

Description:

PRO-1562 low dose level selected from Phase 1 (oral capsule)

Treatment:

Drug: PRO-1562 Oral Capsules

Phase 2, PRO-1562 Oral Capsules Dose level 2

Experimental group

Description:

PRO-1562 high dose level selected from Phase 1 (oral capsule)

Treatment:

Drug: PRO-1562 Oral Capsules

Phase 2, Placebo

Placebo Comparator group

Description:

Placebo oral capsule

Treatment:

Drug: Placebo Oral Capsule