Safety, Tolerability, and Preliminary Effectiveness of CTH120 in Fragile X Syndrome

Summary

The purpose of this Phase IIa study is to evaluate the safety, tolerability, and effectiveness of CTH120 in adult males with Fragile X syndrome.

Conditions

• Fragile X Syndrome (FXS)
• Fragile X Syndrome

Interventions

DRUG

CTH120

15 participants will be randomized to receive CTH120 75 mg hard capsules. Capsules of CTH120 will be administered by the oral route to participants twice a day, one capsule in the morning and one capsule in the evening, at approximately the same times each day, after breakfast (morning dose) and after dinner (evening dose), with a glass of water. The evening dose will be taken approximately 12 h from the morning dose, and preferably before midnight. The evening dose must not be taken before at least 8 hours from the morning dose.

OTHER

CTH120 placebo hard capsules

15 participants will be randomized to receive CTH120 placebo hard capsules. Capsules of CTH120 matching placebo will be administered by the oral route to participants twice a day, one capsule in the morning and one capsule in the evening, at approximately the same times each day, after breakfast (morning dose) and after dinner (evening dose), with a glass of water. The evening dose will be taken approximately 12 h from the morning dose, and preferably before midnight. The evening dose must not be taken before at least 8 hours from the morning dose.

Sponsors & Collaborators

• Hospital del Mar Research Institute (IMIM)

  collaborator OTHER

• Corporacion Parc Tauli

  collaborator OTHER

• Ministry of Science and Innovation, Spain

  collaborator OTHER_GOV

• Astrum CRO, S.L.

  collaborator INDUSTRY

• Connecta Therapeutics, S.L.

  lead INDUSTRY

Principal Investigators

• Rafael De la Torre, Pharm, PhD · Hospital del Mar Medical Research Institute

• Ana María Aldea, MD, PhD · Hospital del Mar Medical Research Institute

• Ana Roche Martínez, MD, PhD · Consorci Corporació Sanitària Parc Taulí. Institut d'Investigació i Innovació Parc Taulí (I3PT)

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Model

PARALLEL

Eligibility

Min Age

18 Years

Max Age

45 Years

Sex

MALE

Healthy Volunteers

No

Timeline & Regulatory

Start

2026-05-21

Primary Completion

2027-05-31

Completion

2027-05-31

Countries

• Spain

Study Locations