Bisphosphonate Studies

H.O.R.I.Z.O.N. (Health Outcomes and Reduced Incidence with Zoledronic Acid ONce Yearly), sponsored by the Novartis Pharmaceuticals Corporation, is designed to determine the effectiveness and safety of a new experimental investigational bisphosphonate drug compared to an established bisphosphonate drug, both given intravenously.

Pediatric patients, between 3 months and 17 years of age, who meet the following criteria may be eligible for this study: 1) Any child diagnosed with OI type III or IV. Any child diagnosed with type I OI who has had more than 3 fractures caused by minimal trauma [including vertebral (spine) fractures] in the previous 2 years. 2) Child has not taken other bisphosphonate therapies for at least one year prior to the study.

The study is planned in various cities throughout the United States, Canada, Europe and Africa. Dr. Francis H. Glorieux is the Principal Investigator of this trial.

If you, or your child, are interested in participating in this study please go to http://clinicaltrials.gov/show/NCT00063479.

P.O.I.S.E (Pediatric Osteogenesis Imperfecta Safety and Efficacy) study, sponsored by Procter & Gamble Pharmaceuticals and sanofi-aventis, is currently recruiting children with osteogenesis imperfecta (OI) to participate in a three-year study of an oral investigational bisphosphonate, risedronate, for the treatment of patients with OI. For the first year of the study patients will be randomly divided into three groups. Two groups will receive risedronate and one group will receive placebo. For the second and third years of the study all patients will receive risedronate.

In a single dose pharmacokinetic study, data showed that risedronate was well tolerated in 28 children with OI. The POISE study will evelauate the safety and effectiveness of risedronate in the treatment of children with OI. Pediatric patients, between 4 years and 15 years of age, who meet the following criteria, may be eligible for this study:

1) Have been diagnosed with OI
2) Have an increased risk of fractures
3) Have not taken other bisphosphonate therapies for at least one year prior to the study

The POISE study will be conducted in a number of sites in North America, Europe and Australia. If you would like to see the sites participating in this study or for more information to see if your child would qualify access the following link: http://www.clinicaltrials.gov/ct/show/NCT00106028

Pamidronate Study. At the National Institutes of Health (NIH) in Bethesda, Md., Dr. Joan Marini of NICHD is conducting a controlled study of pamidronate infusions for children with types III and IV osteogenesis imperfecta. This is a controlled study with four arms, so that patients may be randomly assigned to groups with (a) pamidronate, (b) growth hormone, (c) both drugs, or (d) no drug. Children ages 2 years through 15 years old may be eligible to enroll. Children would need to separately qualify for growth hormone treatment. The protocol involves visits to the NIH Clinical Center every 3 months. There is no charge for any of the services involved in this protocol since it is Federally funded. Travel funds are also available for appropriate patients. As of summer 2001, the protocol is half enrolled and is actively recruiting additional participants.

Physicians or parents of prospective patients should contact Dr. Marini's office (301-496-6683) for more information.

Alendronate Study. The Montreal Shriners Hospital and several U.S. Shriners Hospitals began a study in June 1999 on alendronate treatment for children with severe OI. This is a double-blind study; one of every four children participating will receive a sugar pill that looks just like an alendronate pill. The patients, their parents , and the doctors will not know which type of pill each child is receiving. After one year, if there is a group whose OI-related symptoms are clearly improving, the study will become open, and all of the children will receive alendronate. This study is still accepting children with Type III, Type IV, or severe Type I OI between 4 and 18 years of age.

For more information on which Shriners Hospitals are participating, call the Shriners information line at (800) 237-5055.

Pamidronate Study. The Montreal Shriners Hospital is continuing to study the effects of pamidronate administered to children with OI. This is an open study with no control group; everyone who participates receives pamidronate treatment. This study is recruiting children with OI under 4 years old. The youngest patient participating so far is 4 weeks old. The Shriners Hospitals will cover the costs of the treatments, but participants are usually responsible for their own transportation costs to and from the study site. (The OI Foundation is unable to provide financial assistance to individuals.)

For more information on the Montreal study and the Shriners Hospitals for Children, call the Montreal Shriners Hospital at (514) 842-5964 or visit www.shrinershq.org.

Potential NIH Bisphosphonate Study: At the National Institutes of Health, Dr. Joan Marini is planning a bisphosphonate trial in the near future. Criteria for inclusion have not yet been determined. Participation in NIH trials is at no cost to the patients, and funds are available for transportation.

If your child is not currently participating in NIH treatment protocols, you may call (301) 496-0741 for more information.

Pamidronate Protocol at the Kennedy-Krieger Institute: Drs. Jay Shapiro, Paul Sponseller, and Michael Wright have previously administered pamidronate, in a total dose of 30 mg, to a group of 10 adults with Type IA OI (one adult had Type IV). Treatment continued for two years at three-month intervals. Bone mineral density increased in the Ward's triangle area (an area of diminished bone density) of the proximal femur (the part of the femur near the hip), but did not increase in the femur neck or trochanter (the bony protrusion at the hip area of the femur). Lumbar vertebral bone density increased and remained stable. Bone biopsy results showed consistent improvement after two years of therapy.

Currently, the OI Clinic at the Kennedy-Krieger Institute in Baltimore, Md. is conducting a treatment protocol with two purposes: 1) to continue to evaluate the effect of pamidronate treatment on bone density and fracture rate in OI, and 2) to determine the age of puberty in younger people with OI. (Puberty is associated with increased bone strength in all OI clinical types, but the age at which puberty occurs in people with OI has not been clearly defined.)

The Kennedy-Krieger Institute is eager for additional participants. They are interested in hearing from people with OI of any age (both children and adults), and with any type of OI. At this time, this treatment protocol is not supported by research funding, and participants are responsible for the costs of treatment. Participants have been encouraged to apply to insurance companies for coverage, and to date, most companies have agreed to cover the treatment, based on recognized experience in treating OI with pamidronate.

To find out more about this protocol, contact the OI Clinic at the Kennedy-Krieger Institute at (410) 502-8102.

Pamidronate Study at Shriners Hospital, Los Angeles: The OI Clinic at Shriners Hospital, Los Angeles, is accepting patients who wish to receive intravenous pamidronate, using the same treatment protocol developed at the Shriners Hospital in Montreal. For information, contact Gayle H. Tyerman, M.D., by e-mail, phone (213-388-3151), or mail (Shriners Hospital for Children, 3160 Geneva Street, Los Angeles, CA 90020).

For more information on controlled clinical trials, how they work, and why they're important, see the Foundation's fact sheet on clinical trials.

 
 
 
 
 
 
 
 
 
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