Safety & Efficacy of AlloRx SC® in PTHS Patients

NCT05165017 | Unknown Phase 1 DMC FDA Drug

• 1 other identifier: ALLORX-PTHS
• Study Type: interventional
• Target: 26
• Locations: 0 countries
• Sites: N/A

Brief Summary

This study will evaluate the safety of AlloRx Stem Cells® in subjects with Pitt Hopkins syndrome ages 2-45 with a molecularly confirmed pathogenic mutation in TCF4 or 18q deletion including TCF4. Participants will receive mesenchymal stem cells infusions every 3 months for a year with completion of outcome measures to assess the efficacy of the product.

Conditions

Pitt Hopkins Syndrome

Outcome Measures

Primary Outcomes (2)

• Safety: Incidence of adverse events improvement) in one or more areas of ailments related to PTHS

  Collection of all adverse events (AEs)

  Change from baseline to day 456 (end of study)

• Safety: Incidence of serious adverse events

  Collection of all serious adverse events

  Change from baseline to day 456 (end of study)

Secondary Outcomes (10)

• Change in motor function in individuals with PTHS

  Change from baseline to day 456 (end of study)

• Change in communication abilities in individuals with PTHS.

  Change from baseline to day 456 (end of study)

• Change in sleep habits

  Change from baseline to day 456 (end of study)

• Change in gastrointestinal health

  Change from baseline to day 456 (end of study)

• Change in breath holding spells

  Change from baseline to day 456 (end of study)

Study Arms (2)

Intervention Arm 1

ACTIVE COMPARATOR

AlloRx Stem Cells IV infusion treatment

Biological: AlloRx Stem Cells®

Intervention Arm 2

PLACEBO COMPARATOR

IV infusion of normal saline

Other: Placebo control

Interventions

AlloRx Stem Cells® BIOLOGICAL

Umbilical cord-derived allogeneic mesenchymal stem cells

Intervention Arm 1

Placebo control OTHER

Placebo infusion without mesenchymal stem cells

Intervention Arm 2

Eligibility Criteria

• Age: 2 Years - 45 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Signed informed consent from parent(s) or legal guardian(s)/authorized representative(s) (LAR)
• Documented genetic confirmation of mutation in TCF4, with clinical diagnosis of Pitt Hopkins Syndrome (PTHS)
• Stable seizure control (defined as clinically stable with no changes in antiepileptic medications or use of rescue medication over the prior 1 month before the screening visit, other than weight associated dose adjustments)
• Normal renal function with serum creatinine and spot urine protein within normal limits
• Willing and able to comply with scheduled visits, drug administration plan, laboratory tests, study restrictions, and all study procedures, including intravenous infusion

You may not qualify if:

• Any change in medications or diet/supplements intended to treat symptoms of PTHS (e.g., sleeping aids, supplements, cannabidiol products) over the prior 3 months before screening
• Inability to ambulate independently or with an assistive device or caregiver handhold
• Any bleeding or platelet disorder
• Any clinically significant (CS) cardiovascular, endocrine, hepatic, renal, pulmonary, gastrointestinal, neurologic, malignant, metabolic, psychiatric, or other condition that, in the judgment of the Investigator, will pose a safety risk, make the patient unsuitable for participation in, and/or unable to complete the study procedures
• Any laboratory abnormality, that, in the Investigator's opinion, could adversely affect the safety of the patient, make it unlikely that the course of treatment or follow up would be completed, or impair the assessment of study result
• Known positive for hepatitis B virus, hepatitis C virus, or human immunodeficiency virus (HIV). Patient is pregnant or lactating
• Usage of drugs that increase the risk of bleeding (e.g., heparin, low molecular weight heparin, platelet inhibitors).
• Use of any investigational oligonucleotide and any investigational drugs in the past 6 months
• Any prior use of gene therapy

Sponsors & Collaborators

• Vitro Biopharma Inc.: lead

MeSH Terms

Conditions

Pitt-Hopkins syndrome

Study Design

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

Model Details: Randomized Placebo Controlled Double Blinded

Study Record Dates

• First Submitted: October 29, 2021
• First Posted: December 21, 2021
• Study Start: December 1, 2021
• Primary Completion: December 1, 2022
• Study Completion: December 1, 2023
• Last Updated: December 21, 2021

Record last verified: 2021-12

Data Sharing

• IPD Sharing: Will not share