NCT04042844

A Single Dose of BRTX 100 for Patients With Chronic Lumbar Disc Disease (cLDD)

A Phase 2, Double-Blind, Controlled, Randomized Study to Evaluate the Safety and Preliminary Efficacy of a Single Dose Intradiscal Injection of BRTX-100 for Patients With Chronic Lumbar Disc Disease (cLDD)

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 99 (estimated)
• Sponsor: BioRestorative Therapies · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is a double-blind, controlled, randomized study with blinded assessments using a single dose. Subjects that have a current diagnosis of chronic lumbar disc disease and meet eligibility criteria will be enrolled. Chronic lumbar disc disease is defined as back and/or radicular pain with degeneration of the disc confirmed by patient history, physical examination, and radiographic measures such as computed tomography (CT), magnetic resonance imaging (MRI), plain film, myelography, discography, or other acceptable means.

Detailed description

This is a double-blind, controlled, randomized study with blinded assessments using a single dose. Subjects that have a current diagnosis of chronic lumbar disc disease and meet eligibility criteria will be enrolled. Chronic lumbar disc disease is defined as back and/or radicular pain with degeneration of the disc confirmed by patient history, physical examination, and radiographic measures such as computed tomography (CT), magnetic resonance imaging (MRI), plain film, myelography, discography, or other acceptable means. Subjects randomized to active treatment will undergo bone marrow harvest for processing into BRTX- 100 for intradiscal injection. Subjects randomized to control will also undergo a bone marrow and blood harvest but only undergo a sham intradiscal injection procedure. Subjects will return to the study site for a visit at Week 2, Week 12, Week 26, Week 52 and Week 104/Early Termination. The trial will have a Safety Run-In component that will insert a 3+3 design for the initial subjects dosed with BRTX-100 at 40 × 10\^6 cells. Specifically, the randomization scheme will be briefly shifted from the overall trial 2:1 randomization to an initial 3:1 allotment of intradiscal BRTX-100 versus control. As such, four subjects will initially be randomized and administered their agents. There will be a 14-day safety follow-up period that must elapse between dosing of each of the first four (4) subjects. Dosing of each subsequent subject in the Safety Run-In component cannot occur until the independent Medical Monitor (MM) reviews the previously dosed subject's blinded data, including but not limited to physical examination findings, laboratory values and reported adverse events (AEs) and serious adverse events (SAEs), at the completion of the 14-day visit and documents the findings. If no potential dose-limiting toxicity (DLT) is noted by the MM, the MM will approve the dosing of the next subject. If a potential DLT is noted by the MM, the MM will request that an ad hoc Data Safety Monitoring Board (DSMB) review of unblinded data occur per DSMB Charter before the next subject is dosed.

Conditions

• Lumbar Disc Disease

Interventions

Type	Name	Description
BIOLOGICAL	BRTX-100	Hypoxic cultured mesenchymal stem cells (MSCs) from autologous bone marrow with autologous platelet lysate.
DRUG	Saline	Sodium Chloride (0.9%) intravenous infusion preparation is a sterile and non-pyrogenic solution

Timeline

• Start date: 2022-06-30
• Primary completion: 2026-12-01
• Completion: 2027-12-01
• First posted: 2019-08-02
• Last updated: 2025-08-26

Locations

19 sites across 1 country: United States

Regulatory

• FDA-regulated drug study