Creatine and CMT trials in San Francisco

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Sherry,

There is a trial going on at UCSF Division of Child Neurology in San

Francisco. I am not sure if it has concluded or not, since

recruitment was in 2004 and limited to 18 participants between ages 4

and 18. However, it is listed as an Active Neuromuscular Trial/Study

and includes many other disorders. I am posting the info for you,

follwed by the numerous disorders included. ~ Gretchen

NAME OF CLINICAL TRIAL/STUDY:

Creatine monohydrate -- safety in children with neuromuscular

disorders (UCSF)

PURPOSE AND RATIONALE:

Creatine monohydrate is a dietary supplement used by body builders to

help increase muscle mass. Many people are using this supplement to

see if it helps with nerve and muscle disorders. Dietary supplements

are not regulated by the Food and Drug Administration. The

investigators say that, since there may be significant side effects

from this supplement, especially in children, they are undertaking

this study to determine the safety of this treatment.

STUDY DETAILS:

Participants will be admitted to the University of California at San

Francisco (UCSF) Children's Hospital for three overnight visits in a

three-month period. This will take place at the start of the trial,

after one month of creatine supplementation and after being off

creatine for one month. All participants will receive creatine.

After each six patients enrolled in the study, the dose of creatine

given to the participants will be increased. The creatine is given

four times a day for a five-day loading period and then once a day

for the rest of the month.

During each hospital stay the participants will undergo strength and

function testing. Basic blood tests will be performed once during

each admission, and urine will be collected for 24 hours.

Participants will not be compensated.

more on this at http://www.mdausa.org/research/view_ctrial.cfm?id=119

DISEASE CLASSIFICATION(S):

SMA Type 1 (SMA1), SMA Type 2 (SMA2), Hypokalemic PP (HYPOKPP),

Hyperkalemic PP (HYPERKPP), KSS (KSS), MELAS (MELAS), MERRF (MERRF),

Carnitine Deficiency (CD), Carnitine Palmityl Transferase Deficiency

(CPT), Lactate Dehydrogenase Deficiency (LDHA), Myoadenylate

Deaminase Deficiency (MAD), Phosphorylase Deficiency (MPD),

Phosphofructokinase Deficiency (PFKM), Phosphoglycerate Kinase

Deficiency (PGK), Phosphoglycerate Mutase Deficiency (PGAM), MNGIE

(MNGIE), NARP (NARP), PEO (PEO), Acid Maltase Deficiency (Pompe's

Disease) (AMD), Becker Muscular Dystrophy (BMD), Congenital Muscular

Dystrophy (CMD), Spinal Muscular Atrophy General (SMA), Spinal Bulbar

Muscular Atrophy (SBMA), Polymyositis (PM), Periodic Paralysis (PP),

Paramyotonia Congenita (PC), Oculopharyngeal Muscular Dystrophy

(OPMD), Nemaline Myopathy (NM), Myotubular Myopathy (MM), Limb-Girdle

Muscular Dystrophy (LGMD), Myotonia Congenita (MC), Distal Muscular

Dystrophy (DD), Myasthenia Gravis (MG), Duchenne Muscular Dystrophy

(DMD), Emery-Dreifuss Muscular Dystrophy (EDMD), Lambert-Eaton

Syndrome (LES), Central Core Disease (CCD), Mitochondrial Myopathy

General (MITO), Myotonic Muscular Dystrophy (MMD), Charcot-Marie-

Tooth Disease (CMT), Congenital Myasthenic Syndrome (CMS),

Hypothyroid Myopathy (HYPOTM), Hyperthyroid Myopathy (HYPTM),

Dermatomyositis (DM), General (GENERAL), Dejerine-Sottas Disease

(DS), Friedreich's Ataxia (FA), SMA Type 3 (SMA3), Congenital

Myotonic Muscular Dystrophy (CMMD), Facioscapulohumeral Muscular

Dystrophy (FSHD), Juvenile Dermatomyositis (JDM), Leigh's Syndrome,

Pearson Syndrome