RP-L201 for Tissue Adhesions

Phase-Based Estimates
1
Effectiveness
1
Safety
Hospital Infantil Universitario Niño Jesús (HIUNJ), Madrid, Spain
Tissue Adhesions+1 More
RP-L201 - Biological
Eligibility
Any Age
All Sexes
Eligible conditions
Tissue Adhesions

Study Summary

This study is evaluating whether a gene therapy may help treat a type of leukemia.

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Eligible Conditions

  • Tissue Adhesions
  • Leukocyte Adhesion Defect - Type I

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Compared to trials

Study Objectives

This trial is evaluating whether RP-L201 will improve 3 primary outcomes and 6 secondary outcomes in patients with Tissue Adhesions. Measurement will happen over the course of 2 years.

2 years
Assessment of LAD-I-associated neutrophilia after infusion of RP-L201
Assessment of overall survival after infusion of RP-L201
Assessment of skin rash or periodontal abnormalities after infusion of RP-L201
CD18 expression after infusion of RP-L201
Genetic correction after infusion of RP-L201
Incidence of infections after infusion of RP-L201
Phase I: Number of participants with treatment-related adverse events as assessed by United States (US) National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v.5.0
Phase II: Number of participants with treatment-related adverse events as assessed by CTCAE v.5.0
Phase II: Survival following infusion of RP-L201

Trial Safety

Safety Estimate

1 of 3

Compared to trials

Trial Design

2 Treatment Groups

Control
RP-L201

This trial requires 9 total participants across 2 different treatment groups

This trial involves 2 different treatments. RP-L201 is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 1 & 2 and have already been tested with other people.

RP-L201
Biological
RP-L201 is a gene therapy product containing autologous genetically modified CD34+ hematopoietic cells transduced with Chim-CD18-WPRE lentiviral vector administered as a single intravenous infusion
ControlNo treatment in the control group

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 2 years
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 2 years for reporting.

Closest Location

University of California, Los Angeles - Los Angeles, CA

Eligibility Criteria

This trial is for patients born any sex of any age. There are 6 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Considered to be an appropriate candidate for autologous transplantation of hematopoietic stem cells.
A competent custodial parent with legal capacity to execute an institutional review board (IRB)/ethics committee (EC)-approved consent form must be available to participate in the consent process. (Informed assent will be sought from capable subjects, in accordance with the directive of the IRB/EC and with local requirements).
Ability to comply with trial procedures including investigational therapy and follow-up evaluations.
At least one (1) prior significant bacterial or fungal infection US NCI CTCAE, v5.0, Grade ≥2). This criterion is not required for subjects with documented family history who meet the above inclusion criteria.
A confirmed diagnosis of severe LAD-I as demonstrated by flow cytometry indicating CD18 expression on <2% neutrophils (polymorphonuclear neutrophils (PMNs)). Subjects in which CD18+ PMNs are >2% will be considered eligible with <2% CD11a or CD11b expressing PMNs and if there is a documented ITGB2 mutation and clinical history consistent with LAD-I (or known family history).
Age ≥3 months.

Patient Q&A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many people get tissue adhesions a year in the United States?

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About 15% of US adults have at least one peritoneal adhesion at the time of their procedure. The incidence of these surgically repairable adhesions does not fluctuate over the course of the year.

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What causes tissue adhesions?

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Adhesion formation may be due to the response of mesothelial cells to surgical manipulation and may be independent of inflammation. The significance of these observations in the development of adhesions has not been fully established.

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What are the signs of tissue adhesions?

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Tissue adhesions, although initially smooth, can develop into adhesive masses and lead to pain in the muscles that connect tissues with other tissues. They may form at a site of surgery (e.g. periperitoneal, abdominal, pelvic, pleural, and pericardial surfaces), or they can be of the general kind (between tissue planes), which tend to be deeper than the outer surfaces of the tissues involved. As the tissues are adhaerensed, they lose the ability to glide easily across one another. Tissue adhesions may also occur as a result of infection or injury; they can adhere tissues and form new scar tissue.

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Can tissue adhesions be cured?

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[Colicocleisis] and [subcutaneous mesh repair] are safe and effective for treating non-specific peritoneal adhesions in children. The authors state: "Further work is required to demonstrate the extent of long-term benefits of peritoneal sclerosis to other abdominal surgery.

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What is tissue adhesions?

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At the time of cholecystectomy, it is most likely to occur early in the procedure. Early postoperatively, during the closure of the incision the tissue of the wound collapses and adheres to the surrounding organ and other adjacent structures. Although most adhesions occur during the operation themselves, there are several risk factors which contribute to the formation of adhesions: excessive bleeding, prior adhesions elsewhere, and manipulation at a number of times.

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What are common treatments for tissue adhesions?

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In general, most treatments do not affect the incidence of long-term complications. However, some techniques, especially those using microthermal bipolar RF technology with a low heat rate, may potentially help control the formation of adhesions.

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Does rp-l201 improve quality of life for those with tissue adhesions?

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The rp-l201 formulation is effective in improving the quality of life of those with persistent tissue adhesions and, in this way, might prove to be both a safe and well tolerated therapy.

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What is the latest research for tissue adhesions?

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The cause of tissue adhesions is not clear; it is likely that multiple variables interact to produce these phenomena. There are multiple treatment modalities to address the different causes. The pathophysiology needs to be understood prior to developing a treatment algorithm.

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Has rp-l201 proven to be more effective than a placebo?

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Recent findings indicated that the administration of rp-l201 was better than placebo in decreasing adhesion formation after abdominal hysterectomy plus salpingectomy. It was concluded that adjunctive use of rp-l201 is more effective than a placebo in reducing the number of adhesion sites and postoperative pain in patients who undergo non-opioid-related surgery with adhesion formation.

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Have there been any new discoveries for treating tissue adhesions?

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Current surgical approaches have not yet shown effectiveness in adhesion prevention. In contrast, more research is needed to determine the effectiveness of novel therapy options of tissue adhesions.

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What does rp-l201 usually treat?

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As per our experience, application of rp-l201 has not been helpful in the treatment of uterine fibroid. Because we do not understand the mechanism, we are going to explore the mechanism of action so that we know the therapeutic potential of rp-l 201.

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Is rp-l201 typically used in combination with any other treatments?

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In a recent study, findings indicate that most frequently Rp-l(alpha) is associated with chemotherapy. In a recent study, findings show the clinical usefulness of Rp-l(alpha) in combination with chemotherapy for patients with advanced breast cancer.

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