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Chemotherapy

RIC Regimen for Nonmalignant Diseases (RIC Trial)

Phase 2
Waitlist Available
Led By Timothy J Olson, MD
Research Sponsored by Children's Hospital of Philadelphia
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Organ criteria: Cardiac - Echocardiogram shortening fraction >27%, Renal - Serum creatinine less than 1.5 times the upper limit of normal for age, Hepatic - liver function tests must be less than 5 times the upper limit of normal, No active infections
Age >6 months- 25 years
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year post transplant
Awards & highlights

RIC Trial Summary

This trial will use a RIC regimen in an effort to improve outcomes in patients with non-malignant diseases.

Who is the study for?
This trial is for young people aged between 6 months and 25 years with certain non-cancerous diseases like severe immune disorders, blood conditions (like sickle cell disease), or bone marrow failure. They must have a heart that pumps well, normal-ish kidney function, liver tests not too high, and no ongoing serious infections.Check my eligibility
What is being tested?
The study is testing different levels of Campath (a medication used to prepare the body for transplant) and a smaller dose of Busulfan (chemotherapy) in patients getting donor stem cells from bone marrow or cord blood. The goal is to see if these reduced intensity treatments help the body accept new stem cells with fewer complications.See study design
What are the potential side effects?
Possible side effects include reactions related to Campath such as fever, chills, nausea; Busulfan can cause issues like mouth sores or liver problems. There's also a risk of graft-versus-host disease where the new cells attack the patient's body.

RIC Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My heart, kidneys, and liver meet the health requirements, and I don't have any active infections.
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I am between 6 months and 25 years old.
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I have IPEX syndrome, Sickle cell, Thalassemia major, or Bone marrow failure.
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I have a severe immune system disorder like HLH, SCID, or Wiskott-Aldrich syndrome.

RIC Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 year post transplant
This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year post transplant for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Engraftment
Secondary outcome measures
Survival

RIC Trial Design

3Treatment groups
Experimental Treatment
Group I: RIC:Intermediate CampathExperimental Treatment1 Intervention
Day Treatment Day - 14 to-10 Inpatient: Alemtuzumab (Campath) IV or SQ (subcutaneously) Day - 7 to -3 Fludarabine IV Day - 2 Melphalan 140 mg/m2 IV Day - 1 Cyclosporine infusion starts Day 0 Transplant: Bone marrow or cord blood infusion
Group II: RIC: Mini BusulfanExperimental Treatment1 Intervention
Day Treatment Day - 8 Alemtuzumab (Campath) IV or SQ (subcutaneously) Day - 7 Alemtuzumab (Campath) IV or SQ (subcutaneously) Day - 6 Alemtuzumab (Campath) IV or SQ (subcutaneously) Busulfan IV Fludarabine IV Day - 5 Alemtuzumab (Campath) IV or SQ (subcutaneously) Busulfan IV Fludarabine IV Day - 4 Alemtuzumab (Campath) IV or SQ (subcutaneously) Fludarabine IV Day - 3 Fludarabine IV Day - 2 Fludarabine IV Cyclosporine infusion Day - 1 Rest Day 0 Transplant: Bone marrow or cord blood infusion
Group III: RIC: Distal CampathExperimental Treatment1 Intervention
Day Treatment Day - 22 Inpatient: Alemtuzumab (Campath) test dose IV or SQ (subcutaneously) (subcutaneously) over 2 hours Day - 21 to-19 Alemtuzumab IV/ SQ (subcutaneously) Day - 7 to -3 Readmission to hospital Fludarabine IV Day - 2 Melphalan IV Day - 1 Begin cyclosporine infusion Day 0 Transplant: Bone marrow or cord blood infusion

Find a Location

Who is running the clinical trial?

Children's Hospital of PhiladelphiaLead Sponsor
705 Previous Clinical Trials
8,571,310 Total Patients Enrolled
Timothy J Olson, MDPrincipal InvestigatorChildren's Hospital of Philadelphia

Media Library

Busulfan (Chemotherapy) Clinical Trial Eligibility Overview. Trial Name: NCT01050855 — Phase 2
Nonmalignant Diseases Research Study Groups: RIC: Distal Campath, RIC:Intermediate Campath, RIC: Mini Busulfan
Nonmalignant Diseases Clinical Trial 2023: Busulfan Highlights & Side Effects. Trial Name: NCT01050855 — Phase 2
Busulfan (Chemotherapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT01050855 — Phase 2

Frequently Asked Questions

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

Are individuals aged 30 and over being recruited for this experiment?

"This research is open to participants aged between 6 months and 25 years old."

Answered by AI

What is the number of participants currently enrolled in this clinical experiment?

"Yes, the details posted to clinicaltrials.gov confirm that recruitment is actively underway for this medical trial. Initially published on January 1st 2008 and updated most recently March 8th 2022, 50 individuals are being sought from one research site."

Answered by AI

Is enrollment for this experiment open at the moment?

"Affirmative. According to clinicaltrials.gov, this medical study is still recruiting patients - first posted on January 1st 2008 and last updated March 8th 2022. There are 50 participants needed at one trial site."

Answered by AI

Has the research concerning RIC:Intermediate Campath been extensive?

"Presently, 125 clinical trials are actively studying the effectiveness of RIC:Intermediate Campath. Out of those investigations, 16 have reached Phase 3; with Philadelphia's being the main hub for these experiments and 1255 other sites running their own tests on this medication."

Answered by AI

What criteria must a participant meet in order to be included in this research?

"This research initiative is recruiting 50 individuals with hemoglobinopathies aged 6-25 months. To be considered, patients must have Sickle cell disease; Hemophagocytic lymphohistiocytosis other macrophage activation syndromes; severe Langerhans histiocytosis; Severe combined immune deficiency; adenosine deaminase deficiency and/or common variable immunodeficiency."

Answered by AI

What is the predominant purpose of RIC:Intermediate Campath treatment?

"RIC:Intermediate Campath is primarily employed to manage autoimmune hemolytic anemia, but can be utilized in the treatment of multiple sclerosis, transplant rejection; kidney issues and b-lymphocytes."

Answered by AI

Has the RIC:Intermediate Campath drug been officially sanctioned by the FDA?

"After a thorough assessment, the team at Power put RIC:Intermediate Campath's safety rating at 2. This trial is still in its Phase 2 stage, meaning there are limited reports of efficacy but data exists supporting safety."

Answered by AI
~7 spots leftby Dec 2025