CL20-i4-EF1α-hγc-OPT for X-Linked Severe Combined Immunodeficiency (SCID)

Phase-Based Progress Estimates
X-Linked Severe Combined Immunodeficiency (SCID)CL20-i4-EF1α-hγc-OPT - Genetic
< 18
What conditions do you have?

Study Summary

This trial is testing a new way to treat SCID-X1, a genetic disorder that prevents the development of a functioning immune system. The new method, called lentiviral gene transfer, involves transferring a normal copy of the common gamma chain gene into the patient's bone marrow stem cells. The investigators want to determine if the procedure is safe and effective.

Eligible Conditions
  • X-Linked Severe Combined Immunodeficiency (SCID)

Treatment Effectiveness

Study Objectives

5 Primary · 0 Secondary · Reporting Duration: up to 10 years post gene transfer

Day 42
Number of patients with successful reconstitution
Number of patients with treatment failure
Number of patients without Grade 4 adverse event (AE)
Week 52
B-cell function evaluated by Immune response
Number of NK cells
Day 0
Number of patients who achieve the desired therapeutic busulfan AUC
Number of patients with adequate cell collection and processing
Day 42
Number of patients with adequate neutrophil count recovery after busulfan conditioning
Days -2 and -1 prior to therapy
Pharmacokinetic (PK) variables of busulfan
Year 10
Event-free survival (EFS)
Year 10
Overall survival (OS)
Vector copy number by location of vector-integration sites in sorted blood cells

Trial Safety

Side Effects for

Allogeneic Transplant
5%Upper gastrointestinal haemorrhage
5%Hepatic enzyme increased
5%Small intestinal obstruction
5%Gastrointestinal haemorrhage
5%Hepatic failure
5%Supraventricular tachycardia
5%Acute respiratory distress syndrome
This histogram enumerates side effects from a completed 2018 Phase 2 trial (NCT01410344) in the Allogeneic Transplant ARM group. Side effects include: Upper gastrointestinal haemorrhage with 5%, Hepatic enzyme increased with 5%, Small intestinal obstruction with 5%, Sepsis with 5%, Hypotension with 5%.

Trial Design

1 Treatment Group

1 of 1

Experimental Treatment

28 Total Participants · 1 Treatment Group

Primary Treatment: CL20-i4-EF1α-hγc-OPT · No Placebo Group · Phase 1 & 2

TreatmentExperimental Group · 3 Interventions: CL20-i4-EF1α-hγc-OPT, CliniMacs, Busulfan · Intervention Types: Genetic, Device, Drug
First Studied
Drug Approval Stage
How many patients have taken this drug
FDA approved

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: up to 10 years post gene transfer

Who is running the clinical trial?

California Institute for Regenerative Medicine (CIRM)OTHER
59 Previous Clinical Trials
3,232 Total Patients Enrolled
St. Jude Children's Research HospitalLead Sponsor
402 Previous Clinical Trials
5,301,318 Total Patients Enrolled
National Heart, Lung, and Blood Institute (NHLBI)NIH
3,592 Previous Clinical Trials
46,945,766 Total Patients Enrolled
Assisi FoundationOTHER
10 Previous Clinical Trials
363 Total Patients Enrolled
Stephen Gottschalk, MDPrincipal InvestigatorSt. Jude Children's Research Hospital
5 Previous Clinical Trials
195 Total Patients Enrolled

Eligibility Criteria

Age < 18 · Male Participants · 8 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
The patient has been diagnosed with SCID-X1.
The patient was age two months to one year old when they received busulfan.
If the number of CD3+ T-cells is less than 300 by flow cytometry or if there is evidence of maternal engraftment as supported by peripheral blood FISH analysis for XY and XX, the patient is not a candidate for transplant.
The patient's lymphocyte proliferation to phytohemagglutinin is significantly below the normal range for the laboratory.
Children who are younger than two years old at the time of enrollment are eligible for the program.
The patient had never undergone allogeneic stem cell transplantation therapy before.
A mutation that has been proven to exist in the common gamma chain gene, as determined by sequencing the patient's DNA.