Results from clinical studies
Clinical Studies
Before SKYSONA was made available, two clinical studies (Studies 1 and 2) were conducted to determine efficacy (how good the treatment is at achieving its intended result) and safety. These studies were designed to determine MFD-free survival in children with cerebral ALD who were treated with SKYSONA.
The MFDs are defined as:
- loss of communication
- cortical blindness
- requirement for tube feeding
- total incontinence
- wheelchair dependence
- complete loss of voluntary movement
Study Information
| Study | Treatment | Patients Enrolled | Duration |
|---|---|---|---|
| STUDY 1 (Completed) |
SKYSONA | 32 patients with early cerebral ALD
|
24 months |
| STUDY 2 (Completed) |
SKYSONA | 35 patients with early cerebral ALD
|
24 months |
| LTF-304 (Long-term follow-up study) |
SKYSONA | Patients from Study 1 and Study 2 | 13 years |
| STUDY 3 (Completed) |
|
Existing medical records for patients with early, active CALD at diagnosis | N/A |
| STUDY 4 | Allo-HSCT | Retrospective allo-HSCT data collection study | N/A |
Study Information
| Study | Treatment | Patients Enrolled | Duration |
|---|---|---|---|
| STUDY 1 (Completed) |
SKYSONA | 32 patients with early cerebral ALD
|
24 months |
| STUDY 2 (Completed) |
SKYSONA | 35 patients with early cerebral ALD
|
24 months |
| LTF-304 (Long-term follow-up study) |
SKYSONA | Patients from Study 1 and Study 2 | 13 years |
| STUDY 3 (Completed) |
|
Existing medical records for patients with early, active CALD at diagnosis | N/A |
| STUDY 4 | Allo-HSCT | Retrospective allo-HSCT data collection study | N/A |
Clinical Results
Treatment efficacy was determined based on an analysis in symptomatic patients that compared time from onset of symptoms to time to first MFD or death (ie, MFD-free survival) in SKYSONA treated and untreated (natural history) patients.
To be included in the analysis, patients had to have symptoms at baseline or be asymptomatic at baseline and have developed symptoms during the course of the study. Additionally, they had to have at least 24 months of follow-up after becoming symptomatic or have had an event (MFD or death).
Because of the risk of cancer caused by SKYSONA, it is recommended that patients are evaluated by a hematologist to determine if they have underlying risk factors that could further increase their risk for cancer.
ESTIMATED MFD-FREE SURVIVAL RATE WAS HIGHER WITH SKYSONA
AT MONTH 24 FROM TIME OF SYMPTOM ONSET*†
| MFD-FREE SURVIVAL RATE IN SYMPTOMATIC SKYSONA PATIENTS was 72% (n=11) |
MFD-FREE SURVIVAL RATE IN SYMPTOMATIC UNTREATED PATIENTs was 43% (n=7) |
| MFD-FREE SURVIVAL RATE IN SYMPTOMATIC SKYSONA PATIENTS was 72% (n=11) |
MFD-FREE SURVIVAL RATE IN SYMPTOMATIC UNTREATED PATIENTs was 43% (n=7) |
*Based on post hoc enrichment analysis in symptomatic patients that compared time from onset of symptoms (NFS ≥ 1) to time to first MFD or death (ie, MFD-free survival) in SKYSONA treated and untreated natural history controls whose data derived from existing medical records for patients with CALD.
†To be included in the analysis, patients had to have symptoms at baseline (NFS=1) or be asymptomatic (NFS=0) at baseline and have developed symptoms (NFS ≥ 1) during the course of follow-up in the study. Additionally, they had to have at least 24 months of follow-up after initial NFS ≥ 1 or have had an event (MFD or death).
Safety FROM CLINICAL STUDIES
Safety should always be a consideration for any type of treatment. You need to know how it will interact with someone’s body, and what the side effects or risks are.
The most important information to know about SKYSONA is that the treatment may cause cancer of the blood and bone marrow, which can be life-threatening and lead to death. Blood cancer has resulted from treatment with SKYSONA, because cancer-causing genes have been turned on by the gene therapy. Patients have developed cancer as early as 1 year to as late as 7.5 years after SKYSONA administration.
THE RISK OF graft-versus-host disease (GVHD) WITH ALLOGENEIC TRANSPLANTS
With any sort of allogeneic transplant (even ones from a matched sibling donor), there’s a risk that the body will reject and possibly attack these foreign cells. As a gene therapy, SKYSONA utilizes a child’s own blood stem cells to treat cerebral adrenoleukodystrophy. By using their own cells, SKYSONA does not carry the same risk of GVHD commonly associated with transplants of donor blood stem cells.‡
NO PATIENTS TREATED WITH SKYSONA EXPERIENCED GVHD§
NO PATIENTS TREATED WITH SKYSONA EXPERIENCED GVHD§
NO PATIENTS TREATED WITH SKYSONA EXPERIENCED GVHD§
NO PATIENTS TREATED WITH SKYSONA EXPERIENCED GVHD§
‡Immunological risks defined here as GVHD and graft rejection only.
§Acute (≥grade II) GVHD or chronic GVHD.
Connect with a Patient Navigator –
a specialist from my bluebird support who can help you navigate through the SKYSONA treatment process:
Connect with a
Patient Navigator –
a specialist from my bluebird support who can help you navigate through the SKYSONA treatment process: