TerminatedPhase 1/2

Stem Cell Transplantation for Stiff Person Syndrome (SPS)

Stopped: Could not predict who would respond, relapse or go into remission

United States23 participantsStarted 2014-10

Plain-language summary

Non-myeloablative regimens (as the investigators use herein) are designed to maximally suppress the immune system without destruction of the bone marrow stem cell compartment. When using a non-myeloablative regimen recovery occurs without infusion of stem cells and the stem cells are autologous. While not necessary for recovery, stem cell infusion may shorten the interval of neutropenia and attendant complications. Thus in reality there is no transplant only an autologous supportive blood product. Based on our encouraging results of non-myeloablative hematopoietic stem cell transplantation, for patients with multiple sclerosis and chronic inflammatory demyelinating polyneuropathy, the investigators will investigate the role of non-myeloablative hematopoietic stem cell transplantation for patients with SPS who require assistance to ambulate.

Who can participate

Age range18 Years – 60 Years

SexALL

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Inclusion criteria

✓. Diagnosis of Stiff-person Syndrome and
✓. Diagnosis of a SPS variant- Progressive Encephalomyelitis with Rigidity and Myoclonus (PERM) defined as:
✓. Diagnosis of a SPS variant - anti-GAD positive cerebellar ataxia

What they're measuring

Overall Survival

Timeframe: Mean 3.6 years

Trial details

NCT IDNCT02282514

SponsorNorthwestern University

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2019-08-19

Results submitted2020-11-16

View on ClinicalTrials.gov More Stiff-Person Syndrome trials