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

NCT07826689 | Recruiting Phase 1 DMC

• 1 other identifier: PRO-1562-101
• Study Type: interventional
• Target: 160
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Multiple Sclerosis

Keywords

relapsing multiple sclerosis; chronic optic neuropathy; Remyelination

Outcome Measures

Primary Outcomes (2)

• Phase 1 Incidence of ≥Grade 2 treatment-emergent adverse events

  Incidence of treatment-emergent adverse events including clinically significant laboratory abnormalities of ≥Grade 2 in healthy participants

  From enrollment until 7 days post dose

• Phase 2 Change from baseline P100 VEP latency

  The change from baseline on visual evoked potential (VEP) in affected eye of RMS patients after 6 months of study treatment

  From enrollment until the end of 6 months of treatment

Secondary Outcomes (3)

• Area under the concentration-time curve of PRO-1562 (Phase 1)

  From enrollment until 7 days post dose.

• Brain myelin content, change from baseline

  From enrollment to the end of 6 months of treatment

• Safety Phase 2 Incidence of ≥Grade 2 treatment-emergent adverse events

  From enrollment until the end of 6-months of treatment

Study Arms (10)

Phase 1, PRO-1562 Dose level 1

EXPERIMENTAL

PRO-1562 oral capsule

Drug: PRO-1562 Oral Capsules

Phase 1, PRO-1562 Dose level 2

EXPERIMENTAL

PRO-1562 oral capsule

Drug: PRO-1562 Oral Capsules

Phase 1, PRO-1562 Dose level 3

EXPERIMENTAL

PRO-1562 oral capsule

Drug: PRO-1562 Oral Capsules

Phase 1, PRO-1562 Dose level 4

EXPERIMENTAL

PRO-1562 oral capsules

Drug: PRO-1562 Oral Capsules

Phase 1, PRO-1562 Dose level 5

EXPERIMENTAL

PRO-1562 oral capsule

Drug: PRO-1562 Oral Capsules

Phase 1, PRO-1562 Dose level 6

EXPERIMENTAL

PRO-1562 oral capsule

Drug: PRO-1562 Oral Capsules

Phase 1, Placebo

PLACEBO COMPARATOR

Placebo oral capsule

Drug: Placebo Oral Capsule

Phase 2, PRO-1562 Oral Capsules Dose level 1

EXPERIMENTAL

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

Drug: PRO-1562 Oral Capsules

Phase 2, PRO-1562 Oral Capsules Dose level 2

EXPERIMENTAL

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

Drug: PRO-1562 Oral Capsules

Phase 2, Placebo

PLACEBO COMPARATOR

Placebo oral capsule

Drug: Placebo Oral Capsule

Interventions

PRO-1562 Oral Capsules DRUG

Oral, CNS-penetrant small molecule designed to induce remyelination

Phase 1, PRO-1562 Dose level 1; Phase 1, PRO-1562 Dose level 2; Phase 1, PRO-1562 Dose level 3; Phase 1, PRO-1562 Dose level 4; Phase 1, PRO-1562 Dose level 5; Phase 1, PRO-1562 Dose level 6; Phase 2, PRO-1562 Oral Capsules Dose level 1; Phase 2, PRO-1562 Oral Capsules Dose level 2

Placebo Oral Capsule DRUG

Placebo filled capsules that look like the active intervention

Phase 1, Placebo; Phase 2, Placebo

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Able and willing to provide written informed consent
• Body weight ≥ 48 kg (105.6 lbs) and body mass index (BMI) ≥ 18.5 and ≤ 30 kg/m2
• 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
• No clinically-significant laboratory abnormalities
• Willing and able to use highly-effective forms of contraception for at least 30 days after the end of study visit.

You may not qualify if:

• Ongoing medical condition requiring systemic therapy.
• Ongoing infection
• mRNA or live vaccine during the past 60 days
• History of significant hypersensitivity, intolerance, or allergy to any drug/medication
• 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
• Positive alcohol breath test or urine screen for drugs of abuse.
• 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.
• Poor peripheral venous access.
• Receipt of blood products within 2 months prior to Check-in.
• Donation of blood or blood products during the 4 weeks prior to Check-in.
• Participants who in the opinion of the Principal Investigator (or designee), should not participate in this study

Sponsors & Collaborators

• Progentos Therapeutics: lead

Study Sites (1)

CMAX Clinical Research

Adelaide, South Australia, 5000, Australia

RECRUITING

MeSH Terms

Conditions

Multiple Sclerosis

Condition Hierarchy (Ancestors)

Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Paul A Frohna, MD, PhD, PharmD

  Progentos Therapeutics

  STUDY CHAIR

Central Study Contacts

Chief Medical Officer

CONTACT

858-306-2085; pfrohna@progentos.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 8, 2026
• First Posted: September 17, 2026
• Study Start: October 1, 2026
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): February 1, 2029
• Last Updated: September 29, 2026

Record last verified: 2026-09

Data Sharing

• IPD Sharing: Will not share

Locations

AU (1)