← Back to Search

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

Must not have

Uncontrolled bacterial, fungal or viral infections

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 year post transplant

Awards & highlights

No Placebo-Only Group

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.

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.

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.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

My heart, kidneys, and liver meet the health requirements, and I don't have any active infections.

Select...

I am between 6 months and 25 years old.

Select...

I have IPEX syndrome, Sickle cell, Thalassemia major, or Bone marrow failure.

Select...

I have a severe immune system disorder like HLH, SCID, or Wiskott-Aldrich syndrome.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I do not have any untreated infections.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 year post transplant

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

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Engraftment

Secondary study objectives

Survival

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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

0 / 5Eligibility criteria met