SBI Pharmaceuticals Co., Ltd., (Head office: Minato-ku, Tokyo; Representative Director & President: Yoshitaka Kitao, hereinafter “SBI Pharma”), a subsidiary of SBI Holdings, Inc., engaged in the research and development of pharmaceuticals and medical devices regarding 5-aminolevulinic acid (5-ALA) *, hereby announces the publication of the results of investigator-initiated clinical trials of SPP-004 (5-Aminolevulinic acid hydrochloride and sodium ferrous citrate) for pediatric patients with mitochondrial disease, which were supported with SBI Pharma, in Life, an international scientific journal on life sciences.
| Journal | Life |
| Title | Efficacy and Safety of 5-Aminolevulinic Acid Hydrochloride Combined with Sodium Ferrous Citrate in Pediatric Patients with Leigh Syndrome and Central Nervous System Disorders: An Initial Exploratory Trial with a Double-Blind Placebo-Controlled Period, Followed by an Open-Label Period and a Subsequent Long-Term Administration Study |
| Authors | Yuichi Abe (Department of Pediatrics, Saitama Medical University , Present address: Division of Neurology, National Center for Child Health and Development) et al (Corresponding author： Akira Ohtake). |
| URL | https://www.mdpi.com/2075-1729/15/8/1168 |
| Abstract | The safety and efficacy of SPP-004 (5-ALA hydrochloride and SFC) was evaluated in 10 patients under the age of 2 with Leigh syndrome, one of the major pediatric mitochondrial diseases.
An exploratory study (SPED-ALA-001 study) consisting of a 12-week double‑blind period receiving SPP-004 or Placebo and a 12-week open-label period receiving SPP-004, and a subsequent long‑term administration study (SPED‑ALA‑002 study) in which SPP-004 was administered continuously for up to 180 weeks were conducted. There was no significant difference in NPMDS scores (criterion for assessing the severity of mitochondrial disease), between the SPP-004 and placebo groups at the end of the double-blind period, but the symptoms suggested to be stabilized or improved with subsequent long-term administration of SPP‑004 from the open-label period until the end of the long-term administration study. Blood lactate levels increased in the placebo group during the double-blind period, but the average level was normalized with long‑term administration of SPP-004. One patient died of heart failure presumably due to underlying disease, but there were few serious adverse drug effects, and seven patients safely completed long-term treatment. These results suggest that SPP-004 may contribute to stabilization of symptoms in pediatric patients with mitochondrial disease. |
*1 5-aminolevulinic acid: An amino acid produced in mitochondria. It is an important substance that serves as a functional molecule related to energy production in the form of heme and cytochromes, and its productivity is known to decrease with age. 5-aminolevulinic acid is contained in food such as shochu lees, red wine, and Asian ginseng. It is also known as a material forming chloroplasts in plants.
*2 This new release is an introduction to the research publication. It does not recommend the use of unapproved drugs.
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