Multicenter trial of SPP-004 in mitochondrial diseases

JMACCT ID: JMA-IIA00200

Registered date:09/12/2014

Basic Information

• Recruitment status: Completed
• Health condition(s) or Problem(s) studied: Mitochondrial disease, mainly to cranial nerve symptoms
• Date of first enrollment: 10/12/2014
• Target sample size: 10
• Countries of recruitment: Japan
• Study type: INTERVENTIONAL

Intervention(s)

Intervention type:DRUG. Intervention1:SPP-004, Dose form:CAPSULE, Route of administration:ORAL, intended dose regimen:A capsule of each investigational drug [5-ALA HCl capsule (25 mg) and SFC capsule (39.22 mg)] is administered twice a day orally or by a feeding tube . After Week 13, a capsule of each investigational drug (5-ALA HCl capsule and SFC capsule) is administered twice a day orally and by a feeding tube. If capsules cannot be swollen, the content should be dissolved at the time of administration.. Control intervention1:Placebo, Dose form:CAPSULE, Route of administration:ORAL, Intended dose regimen:A capsule of each investigational drug [5-ALA HCl placebo capsule (25 mg) and SFC capsule (39.22 mg)] is administered twice a day orally or by a feeding tube.
If capsules cannot be swollen, the content should be dissolved at the time of administration.
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Outcome(s)

• Primary Outcome: The Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Section I-III. Timepoint:Screening, Treatment period 8,12 and 20,24 weeks or discontinuation.

Secondary Outcome

Life prognosis
NPMDS Section I-IV
FGF21
Weight
Height
Head circumference
Respiratory chain complexes enzyme activity in dermal fibroblast (if informed consent is obtained). Timepoint: Screening, Treatment period 8,12 and 20,24 weeks or discontinuation.

Key inclusion & exclusion criteria

• Age minimum: >=3 YEARS
• Age maximum: <2 YEARS
• Gender: BOTH

Include criteria

Japanese patients who meet all of the following criteria are included in this clinical trial.
1)Patients who were confirmed to be respiratory chain enzyme deficiency or mitochondrial gene abnormality by clinical findings in patients with suspected mitochondrial disease*
2)Patients aged ? 3 months and < 2 years at the acquisition of consent forms (premature infants are dealt as corrected age in month)
3) Patients with blood lactate-pyruvate ratio ?15.0
4) Patients with written informed consent from their legal representatives (persons in parental authority or guardians)
*Patients with all of the followings 1 to 3, as well as one or more of the conditions in 4
1. Progressive neurological disorders associated with delayed motor and mental development
2. Symptoms or signs of disease in brain stem and/or cerebral basal ganglia
3. Increased lactate levels in blood and/or cerebrospinal fluid
4. Patients who meet one or more of the followings
1. Patients who have characteristic findings in images. (bilateral and symmetrical lesion of the brain stem or basal ganglia)
2. Patients with typical neuropathological changes (spongiform degeneration)
3. Patients with a sibling with similar symptoms

Exclude criteria

Patients who meet any of the following criteria are excluded from this trial.
1) Patients with mitochondrial cardiomyopathy
2) Patients with severe cardiac function or renal function disorders
3) Patients complicated by sepsis
4) Patients with a history of drug allergy
5) Patients with a history of hypersensitivity to any ingredients of the investigational drugs
6) Patients participated in another clinical study within 12 weeks prior to the informed consent.
7) Patients whom the investigator and subinvestigator considered inappropriate to participating this trial

Related Information

• Primary Sponsor: Saitama Medical University Hospital
• Source(s) of Monetary Support: Japan Medical Association