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The only pill approved in the U.S. for treatment of children with type 2 diabetes is proving surprisingly ineffective, according to a new study, heightening worries about the fast-growing and largely preventable disease.

The research, reported Sunday, is one of the first long-term studies to test the effectiveness of drugs for diabetic children—estimated in the U.S. to number in the tens of thousands. It tested three different drug-based regimens aimed at controlling the disease and found that only about half the participants successfully controlled their blood sugar—despite relatively good compliance.

Researchers said the findings suggested a majority of youth with …